subscription manager
subscription manager
subscription manager
subscription manager
subscription manager
subscription manager
subscription manager
subscription manager
AUTUMN 2019
A CF Friendship:
Chance Or Destiny
This story has two main characters: Tori and me. We met at the University of North Carolina at Greensboro after joining the same sorority. We barely knew each other when we were assigned to live together. In August of 2014, I stepped foot into a room the size of a walk-in closet. There sat Tori, cross-legged, unpacking, dressed in just a sports bra and shorts. Quickly I noticed two scars across her stomach. “Oh, yeah, I guess I should tell you I have cystic fibrosis,” she said as if it was a way to start small talk. I didn’t know what that really meant, just as I didn’t know what Tori would become to me. Throughout that year, I was introduced to nebulizers, vest treatments, coughing attacks in the middle of the night, high-calorie diets, and the dozens of medicines taken every day. I found out that the scars were from a bowel resection and a feeding tube that Tori had needed at some point in her childhood. That year, I was also introduced to a girl who was brilliant in her kindness, despite her dark, sad story. I was introduced to a girl who was utterly admirable in her strength, and who was unlike anything I can describe. It was during this year that I decided “best friends” is an overused label. Tori wasn’t merely a “best friend.” She became so much more. She was comfort. She was honesty. She was a voice of patience and understanding and reason. Her brain seemed entwined with mine — we both viewed the world with the same angry, questioning, yet awestruck, eyes. I don’t think there is a word strong enough to describe what we became to each other. Our stories before we met each other were heartbreaking, and sometimes I think the world knew I needed someone whose bitterness matched mine, but who still saw the beauty in the world. I think there was more than luck in meeting Tori.
We lived together the following year in an apartment. I slept in her bed more than I did in my own. We remained close. I went with her family to New Orleans for New Year’s Eve. I watched her oldest sister get married on Bald Head Island. Tori shaved my head for St. Baldrick’s, while together we raised thousands of dollars for childhood cancer. I went to Disney World, proudly claiming the title “Megan Tamborino.” It was during this year that Tori was hospitalized with a lung infection. I remember calling my dad sobbing while sitting in the hospital lobby. I remember crying and saying something like, “she actually has it, dad. She has cystic fibrosis.” It had never registered until that moment. Sure, I watched her do the medicines and knew she went to clinic appointments, but I had never seen her like that. It’s amazing to me that when our loved ones get that sick, it somehow feels like we’re the ones who are going to die.
Fast forward a bit. Tori and I both graduated college in December of 2016. Knowing something about sinus disease from being around Tori and her many medical issues relating to CF, I knew I needed sinus surgery. I was deaf in my left ear from many ear infections. I had polyps, underdeveloped sinuses, and needed to figure out my health before I went abroad. My ENT took a full history — from the day I was born until that very second sitting there talking to him. That history consisted of hospitalizations for pneumonia; many ear infections; sinus infections; bronchitis; being underweight as a child; inexplicably passing out multiple times at track practice; going into pulmonary arrest after getting my gallbladder out at age 21; multiple GI problems; and many other issues I dealt with while growing up. He looked me dead in the face and said, “I don’t think anyone is this unlucky to have had so many random health issues in their life. I think there’s a bigger issue.” The “biggest thing” it could be was cystic fibrosis. Two days later, I got a sweat test. One day after that, I was diagnosed with cystic fibrosis.
Tori cried about my diagnosis and left work early the day I told her. I think she was sad that I would now enter that part of her world and would have to feel some of the pain she feels on a daily basis.
I’m sure if you somehow were able to calculate the odds of two girls living together in a school in North Carolina, while they both (one of them unknowingly at the time) have CF, you would find that it is something like a one-in-a-billion situation. Eighty percent of CFers are diagnosed before age two. Now, CF screening is a part of newborn testing. I, on the other hand, was diagnosed at age 23. I was almost relieved to be diagnosed. Everything in my life made a little more sense. I passed out at track practice because I wasn’t replenishing the salt I lost. I was underweight and small because my body couldn’t absorb vitamins and nutrients like a normal person. My colds turned into pneumonia or bronchitis because my lungs couldn’t rid themselves of the mucus trapped there. I was never afraid. I knew everything I needed to know. I immediately started on Pulmozyme and hypertonic saline. I knew how to set up nebulizers. I knew how often to do vest treatments. I knew everything about CF because of Tori. I wasn’t mad. I was okay because of Tori. I often wonder how to thank her for that. How do you thank a person for preparing you so fully for the hardest thing life will hand you?
In the last two years, I have been hospitalized a dozen times for lung infections, surgeries, kidney infections, bowel obstructions, and kidney failure. Tori has had multiple PICC lines for IV antibiotics. I am currently on 37 medications. Tori is on “20 something.” In the cystic fibrosis community, they say we’re supposed to remain six feet away from each other at all times. They say we grow bacteria in our lungs and that by being so close to other people with CF, we are at risk of sharing our bacteria and getting each other sick. Tori and I say, “who cares?” I would rather live in a world where Tori and I laugh too hard and talk too much and wonder about the crazy universe that merged our stories together, than live a dull life far from her.
This story is centered on a kind of bond that has yet to be labeled, but is felt by so many. It’s a story about two girls, who had no idea how that small dorm would change them. It is a story about the unknown and the unquestionable, the dark and the light, and illness and the silver lining. I don’t know what we want out of this story being told: awareness, maybe, of CF and the importance of friendship. Or maybe we just want others to wonder with us about whatever force or God or universe brought us together. Or perhaps, we just want our story out there so anyone else who has a friend who is more than a “best friend” can smile and laugh. I only know I was compelled to share this story about our magic, and I hope that whoever is reading this enjoyed this incredible CF story.
Megan is 25 and has CF. She graduated with her Bachelor’s of Science Degree in Kinesiology: Sports Medicine from the University of North Carolina at Greensboro in 2016. She is currently pursuing a Master’s of Science Degree in Athletic Training at Azusa Pacific University. Megan would like to continue her education in pursuit of a Doctorate Degree, focusing on research involving cystic fibrosis and athletes. She enjoys all things sports, is an avid runner and book reader, and loves spending time with her friends and siblings. You may contact her at meganmcarroll4@gmail.com.
speeding past 50:
It was worth a try
So another summer has come and, almost, gone. It was a beautiful summer here. There were only a few days over 90 degrees and not too much rain. The nights were cool enough to sleep comfortably. I think we are blessed with great weather here. I know that many people would rather have hotter weather, but I like it like this. The autumn crocuses are coming up and the leaves of the walnut trees are coming down. Soon the driveway will look as if it is paved with gold because the black walnut tree has such golden leaves.
As is usual for me, July was a little problematic with increased coughing and some days where I felt less than grand, but I didn’t get sick. When I was young, March, July, and October were the months when I would get pneumonia and feel really crummy. I hope those reactions are over. I like feeling well.
I noticed that the focus topic of this issue is CFTR Modulators. I thought that I would tell you about my experience with taking a CFTR modulator.
In November 2018, I began a regimen of Orkambi. My doctor felt that it was worth a try, even though my mutations were not an exact match for this medicine. He had read articles that made him feel it might work well for me. Prior to this, my doctor had urged me to try Orkambi for a few years, but I was reluctant because of the cost. Even though I have excellent insurance that covers my prescriptions with a very low copay, I felt that such an expensive prescription would take a large bite out of the money that is there for all the insureds to access. I really didn’t feel that it was fair to all the others in my insurance pool to use so much of the funds for one prescription. Friends explained to me that the money is there for just such meds and that I should give it a try. After hearing that so many times, I gave in and said yes.
When I started Orkambi in November 2018, I only got a one-month supply (as opposed to the normal three-month supply of prescriptions) as I didn’t want to potentially waste multi-thousands of dollars worth of medicine if it didn’t work for me. I admit that I was concerned about some of the potential side effects. Still, I decided to go ahead. I am a compulsive record keeper, so I kept a journal of my experience with Orkambi.
The literature says to take it with a fatty meal such as peanut butter, avocado, or egg. It was a real challenge to figure out what I could take with it that would be fatty. (More about this, later.) I chose cream cheese on a cracker for my first dose. Since I am a night owl, I took it at 10 p.m. and 10 a.m.
Within a very short time — maybe only minutes — I noticed that I was getting rid of a copious amount of mucus. It wasn’t difficult to bring it up and it just kept coming. I had a good night of sleep with minimal coughing. After the third dose, I had very unpleasant hand pain and diarrhea. Those lasted a day or two and then subsided. I skipped my polyethylene glycol for a couple of days until my innards went back to my normal. The next day, I had a minor bout of hemoptysis. For about four days I had a little blood in my mucus, but it was nothing overly concerning. I did call and talk with my doctor when it first appeared and worked out how to handle it. For the next few days I had what I call “Christmas” mucus. Just little stripes or bits of red in the green mucus. That had disappeared by the end of the eighth day and I have had none since then.
During the first two weeks of taking Orkambi, in addition to the diarrhea and hemoptysis, I had a headache, some nausea, and a little dizziness. These all may have been caused by a compression fracture that I have in my neck. They did not seem to be directly related to Orkambi, but one never knows. By the end of those first two weeks, all of the various symptoms were gone and I have had no further problems with it. One thing that really pleased me was that I slept a full eight hours without being awakened by anything. I felt so much better than I had been feeling. If I am able to take this drug for the rest of my life, I will be delighted.
I am able to be up and active all day now. For years I had been unable to stay active for more than an hour or two per day. The rest of the day, I would be sitting in a chair with my legs up and doing things that didn’t take much energy. I would sleep a couple of hours on most days. I found it difficult to accomplish much. Now, I am up and doing things that I want and am able to go away from home and enjoy what I am doing.
It is amazing to me that I awaken feeling rested and ready to meet the day. I love the feeling of being an almost-normal old lady. I still may take a nap in the afternoon and it may last anywhere from 20 minutes to an hour and a half, depending on how I’m feeling and how comfortable I am while I’m sleeping. I think that I may just be lazy, which is okay with me. I enjoy being able to rest as needed.
While I was feeling less well, my muscles had gotten quite out of shape. Before my various surgeries that rendered exercising difficult, I had been able to walk some distance and had been doing various muscle-strengthening exercises. But by the time I started Orkambi, I had very little muscle tone. Now, I like being able to do some exercise on the stair-stepper and will get back to walking on the treadmill. I was all ready to start my daily walks when our treadmill died. Since it is nearly 25 years old, there are no parts available to repair it. So, my walking will have to wait until we can afford a new treadmill. That’s okay because I am able to get in some walking in my house to fill in. I know that my legs need the exercise because they are somewhat weak. Walking will help build them back up.
One thing that has been an adjustment for me is having to eat something fatty with every dose of Orkambi. I have tried to vary what I use for the fatty food. Sometimes I eat peanut butter with a little mayonnaise on a cracker. Other times I have a little Norwegian cured salmon with cream cheese on a bagel. I also like to eat avocado or have a little sun-dried tomato pesto or cheddar cheese on a cracker. My favorite fatty food is the popcorn that Paul makes for me using an old-fashioned recipe he got from a friend. It calls for cooking the corn in a deep saucepan and melting butter in the pan to pour over the popped corn. It is delicious! I am sure that it has enough fat to last for several doses, but I use it for only one dose every week or two. Fortunately, I have not had a big weight gain from all of the added fat. I am one of the people who doesn’t have a failure to thrive. I have to watch my weight or I can balloon up quite quickly. I have maintained my normal weight since I started Orkambi, with a fluctuation of only two or three pounds up or down.
I almost forgot to mention I am coughing less than I used to and do not have to cough so hard. Because of that, the muscle along the right side of my spine has relaxed and I am able to stand up straighter. It also has lessened the pain I was feeling from that knotted muscle.
I say that Orkambi has been a good medicine for me. I appreciate the positive changes it has made in my life. I am happy that my doctor was able to prevail and get me to try it. It was worth a try. Thanks, Dr. H.
In this column in the last issue of CF Roundtable I spoke of the changes that my ENT doc was making to my sinus regimen. When I next saw him, he decided to stop the hypertonic saline irrigations, since they were so harsh on my throat. Instead, I am irrigating with plain normal saline. He will check again next month and, if my sinuses still are looking as good, he plans to discontinue all irrigations. He said that my sinuses look like normal sinuses except for the scarring and surgical changes. I am not sure I would know how to act if I didn’t have to irrigate my sinuses. I am quite willing to find out, however. I have no idea if this is a result of taking Orkambi, but I do feel that it could be.
So, over all, life is good and I am happy to be living it. Wahoo! I hope to live as long as life still is enjoyable.
Stay healthy and happy,
Kathy
Kathy is 75 and has CF. She is a former director of USACFA and has served as Treasurer, President and Managing Editor of CF Roundtable. She and her husband, Paul, live in Gresham, OR. You may contact her at krussell@usacfa.org.
Family Matters:
Handling Anxiety In Children Of CF Parents
There are plenty of things about parenting with CF for which I wasn’t prepared. The mind-numbing exhaustion, for one. My daughter licking the hospital floor during one of my admissions, for another. I did try to stop her! But among the most difficult and unexpected parts of parenting has been dealing with my six-year-old daughter’s anxiety — especially when it’s so often triggered by my cystic fibrosis.
My daughter started showing signs of being anxiously tuned in to my health startlingly young. From the time she was four or five months old, when I got sick, she’d quickly become fractious and fussy, not wanting to be held by anyone but me. As she grew into a spirited, high-needs toddler, her anxiety manifested most often as aggression toward me: hitting, yelling, and biting. While many of my CF mom friends gushed about how their young children were sweet and thoughtful, bringing them blankets when they were sick and helping to sort their pills, my daughter at age three was spending my whole treatment time on my lap, screaming at me to stop and trying to pull my nebulizer out of my mouth by force.
Thankfully, at six at a half, my daughter is much more understanding of the need for me to rest, do breathing treatments, and have occasional IVs. But even now, her anxiety plays a big role in our lives together. She frequently struggles with intense separation anxiety, worried that any time we’re apart — whether that’s during the school day, while my husband and I have a date night, or simply when I put her to bed in the evening — my health might suddenly decline and I might have to go to the emergency room. When I’m coughing more because of an exacerbation, she watches me suspiciously, drilling me with questions about why my cough is worse.
While my daughter’s anxiety is definitely more than a normal amount — she has a diagnosed anxiety disorder — some level of anxiety is very common in children whose parents have CF. Being confronted by the daily realities of CF can cause a lot of different symptoms of anxiety in a child: panic attacks (which often look like large-scale tantrums in toddlers and babies); spiraling worries; obsessive-compulsive behaviors like excessive handwashing, aggression, or acting out at home or school; disturbed sleep; physical signs like stomach aches; and separation anxiety that goes well beyond a normal level. Even if your child doesn’t have an anxiety disorder, a major event in your life such as a hospitalization, exacerbation, transplant, or other health emergency can lead to a lot of anxiety in your child.
In this article, I wanted to share a few strategies that my husband and I have found helpful in parenting our daughter, as well as ideas gathered from talking to other conversations with CF parents.
1. Be honest and age appropriate. Anxiety is often exacerbated by fear of the unknown, and kids are usually much more perceptive than we give them credit for! Answering your child’s questions in an honest, but age-appropriate, way can help alleviate anxiety. I often joke that my approach to talking to my daughter about CF is a lot like my approach to talking to her about sex: I try to answer every question she asks me honestly, without deflecting, but also without elaborating or adding additional information for which she hasn’t asked. Practically speaking, this means that when she was three and asked me if CF was ever going to go away, I told her honestly that it was not — but I didn’t volunteer more information than that, like the fact that CF has a shortened life expectancy.
2. Involve them in your healthcare. My daughter frequently sees me doing treatments and sometimes even helps me load nebulizers or turn on my vest. When I’m on IVs, she loves to help flush my line. She’s even been my assistant when I’ve flushed my port before, handing me syringes when I needed them! I’ve taken her with me to clinic visits since she was little, and even pulled her out of school to come with me one month when she was feeling especially anxious about my health and I thought that reassurance from my CF team would help.
My daughter also has a “doctor kit” with medical equipment she finds fascinating — old nose clips from CF clinics, paper masks, empty syringes, an old nebulizer, and a disposable stethoscope. Sometimes when she’s struggling with my health, she’ll pull the doctor kit out a lot and pretend she’s giving her stuffed animals PFT tests and nebulizer treatments.
3. Allow them space and support for their emotions. It’s normal for a kid to experience heightened emotions when they’re trying to process something difficult. Sometimes this looks like tears or worries; other times, it looks like aggression or “acting out.” Give them space to feel whatever they’re feeling, and make sure they know that it’s okay to be sad, angry, upset, or fearful because of CF. Sometimes, when my daughter is really spiraling, I talk to her about how I also often feel mad, sad, or afraid because of CF and the effects it has on my life. I don’t allow my daughter to hurt me or others when she’s upset, but I try to be present and ready to talk, snuggle, or play with her when she needs me. We love to read books together, and I’ve found that seeking out children’s books about dealing with emotions serves as a useful tool in working through and talking about her feelings when they’re overwhelming her. Other ideas that can help a child process their emotions are therapeutic coloring (have them color a picture that represents how they’re feeling and tell you about it) or role playing with dolls or stuffed animals.
4. Make time for connection. My daughter’s anxiety levels are noticeably lower if I try to make at least a few minutes here and there to connect with her in whatever way she wants — playing dolls, reading books, or watching her do tricks on our backyard swing. When I’m sick, these moments of connection can become much harder, but I’ve found that in those moments it’s even more important for me to find ways to connect to her, even if that just means snuggling on the couch while we watch a movie.
5. Learn coping techniques together. Because my daughter’s anxiety plays such a big role in our lives, we’ve spent several years learning calming strategies to use when she’s having a panic attack or meltdown. The internet is full of resources for teaching kids deep breathing strategies and simple meditation or visualization! A key for us has been practicing these techniques when my daughter is not feeling anxious, so that she’s ready to deploy them when she is anxious. We have also really liked the What To Do When You series from Magination Press, which are workbooks focused on helping teach kids cognitive behavioral therapy strategies for anxiety. Our favorites are What To Do When You Worry Too Much by Dawn Huebner and What To Do When You Don’t Want To Be Apart by Kristen Lavallee and Silvia Schneider.
6. Try to preserve your child’s routine as much as possible. For many anxious kids, routine is king. My daughter does much better if she knows what to expect, both in the immediate and the more distant future. Of course, preserving her routine is way harder when I get sick, and we often have to make adjustments, like someone else picking her up from school. We try to talk about any changes with her before they happen, so that she’s prepared. Now that she can read, she especially loves organizational tools like calendars, to-do lists, schedules, and maps, and sometimes when she’s feeling anxious a good to-do list or schedule of what to expect that day can be enough to calm her down!
7. Involve the other adults in your child’s life. Make sure that other important adults in your kid’s life know what’s going on, whether that’s school teachers, family friends, grandparents, or aunts and uncles. I try to keep my daughter’s school teacher in the loop about what’s happening both with my health and with my daughter’s anxiety, so that she knows when my daughter might need a little extra intervention. I also share what things have helped us at home, so that she can employ the same strategies in the classroom.
8. Enlist professional help. Having a parent with CF is a pretty big deal and, for many kids, enlisting a therapist can help. For younger children, therapists can use techniques like play therapy, parent-child interaction therapy, sand therapy, and other methodologies to help kids cope with their feelings. Techniques like cognitive behavioral therapy can help equip older children with the tools they need to handle anxiety-provoking situations. A trusted therapist can also talk a child through difficult things they may be dealing with, putting issues like exacerbations, hospital stays, and transplantation into better perspective.
Whether a child has an anxiety disorder or not, it’s completely normal for children of CF parents to experience at least occasional anxiety. I hope these tips will better equip you and your child to get through the ups and downs of your cystic fibrosis together!
Cindy is 31 with CF. She lives just outside Portland, Oregon, with her husband and spitfire of a daughter (who never makes CF care easy!). She is the author of Where the Watermelons Grow and the forthcoming Beginners Welcome, both with HarperCollins Children’s Books.
ask the attorney
CF Roundtable readers have asked many questions related to the Americans with Disabilities Act and the Family Medical Leave Act. No reference made in this column is to a specific situation. Nothing in this column is meant to be legal advice about a specific situation but is only meant to be information.
The CF Legal Information Hotline can answer questions related to Social Security benefits, Medicare, Medicaid, health insurance, and rights in employment and education. If you have questions, please email CFLegal@sufianpassamano.com to schedule a time to speak with an attorney.
1. Is an employer required to allow a person with CF to work from home if there are people in the office who are coming to work sick?
This is a frequent question. A discussion of the American with Disabilities Act is helpful in answering this question. The Americans with Disabilities Act (ADA) applies to employers with 15 or more employees. The ADA protects employees who are qualified individuals with disabilities. A “qualified individual with a disability” is a person who can perform the essential functions of the job, either with or without a reasonable accommodation.
• A disability is any physical or mental impairment that substantially limits a major life activity. For a person with CF, a limited major life activity may be breathing or digestion.
• A reasonable accommodation is any change to the job, work environment, or employment practices that enables the disabled person to perform the essential functions of the job, or to receive equal opportunities of employment.
• A reasonable accommodation must be requested by the employee, who should: (1) identify the limitation that needs accommodation; and (2) suggest a method of accommodation that will overcome that limitation.
• The employer must agree to the request.
• A reasonable accommodation is any change to the job, work environment, or employment practices that enables the disabled person to perform the essential functions of the job, or to receive equal opportunities of employment.
An employee who wants to request a reasonable accommodation of working from home while other employees, who are sick, are attending work, would make the request in writing. Some employers have specific forms the employee must use to request a reasonable accommodation. The employer is allowed to request follow-up information from the treating physician.
Some employers routinely allow employees to work from home; others do not like employees to do so. It may be harder to get approval to work from home if the employer does not allow anyone to work from home, or if the job must be done at the office. The employee requesting the ability to work from home does need to have a job that can be actually performed at home.
2. If a person with CF is sick, when can he/she qualify to receive Family Medical Leave?
Family Medical Leave can be very helpful for a person with CF who needs time off from work due to health issues. In order to be eligible for FMLA leave, a person must meet three conditions:
The employer must have 50 or more employees. The 50 employees must be employed within a 75-mile radius of the workplace for at least 20 calendar weeks out of the year.
Covered employees must have worked for the employer for at least 12 months at the time the FMLA leave is requested.
Covered employees must have worked at least 1250 hours in the 12 months prior to the leave being requested.
Medical leave must be for a serious medical condition that requires the employee to take care of him/herself, the employee’s spouse, the employee’s child, or the employee’s parent. An employee who meets the criteria listed above will be entitled to receive: 12 weeks of unpaid leave that can be taken all at once or be broken up into segments of time.
The employee has the right to return to his/her same or similar job at the end of the FMLA leave period.
The employee cannot be fired while she/he is out on FMLA leave. Upon return to the job, the employer may decide to terminate the employee if the employee can no longer perform the job with or without a reasonable accommodation. The employee can be terminated upon return to work if the employer is eliminating the positon.
The employee could ask if there is a similar position the employee could perform if the employee’s position is eliminated. The employer does not have to create a job for the employee if the current positon is eliminated.
The employee has the right to maintain employee benefits during the FMLA leave, such as health benefits and other employee benefits.
FMLA leave is unpaid, but the employer is allowed to provide paid leave.
The employer may run any paid sick or vacation time leave at the same time the FMLA time is running.
Beth is 54 and has CF. She is an attorney who specializes in disability law and is a Director and current Vice President of USACFA. Her contact information is on page 2. You may contact her with your legal questions about CF-related issues at CFLegal@sufianpassamano.com.
spirit medicine:
The Spirit Of Ambivalence
I was sitting at the CFRI Conference this past July, listening to the speaker talking about the significant results of the upcoming triple combo Vertex drug trial. For several years now, I’ve heard about the results of the last few Vertex drugs for people with certain genetic mutations. Now, this triple combo was directed for all patients with one delta F508 mutation. That included me. I felt overwhelmed with excitement. I was witnessing a moment in history. For the majority of patients, cystic fibrosis was becoming a controllable disease. CF was on the verge of being cured. All my life, my bedtime prayers begged for a cure for this disease. And finally, in 2019, this was happening.
At the same time, I felt a veil of sadness overcome me. I felt a weight of sorrow and grief for all the friends I’ve lost, especially the recent deaths for people who came so close to this next hopeful treatment. I felt profoundly sad for all of us CFers who needed a transplant before these drugs could help us breathe. I felt sad for myself, even though I’m 15 years post-transplant and would never have lived long enough for these drugs. These mixed emotions are not unfamiliar. I’ve been holding this kind of emotional ambivalence all my life. I’m happy to be alive, but I hate the effort of this CF life. I’m happy to still be here, but I mourn that I don’t get to reap the benefits of these drugs.
In this Spirit Medicine, I’d like to explore ambivalence. Ambivalence is the state of carrying two opposing experiences at the same time. It means the simultaneous and contradictory attitudes or feelings towards a person or experience.
Ambivalence can cause angst, discomfort, and worry. I can judge myself, wondering how heartless I am that I’m not exclusively happy about the new Vertex drugs. Juggling two opposite feelings is hard. Sometimes, it’s easier to take one side and suppress or avoid the other feeling that is trying to creep up, too. Trying to avoid a state of ambivalence is a way to avoid the discomfort.
Ambivalence is often undervalued. It has a purpose and role in our lives. In the case of political or ethical dilemmas, ambivalence protects us from rejection; it serves as self-protection in case someone on one side of an issue resents the opposing side we might take. I also believe humans cultivate ambivalence to protect their feelings in the face of uncertainty.
Ambivalence is also confusing. Sometimes ambivalence might be mistaken as indifference, which it is not. Ambivalence is marked by an excess of feelings or opinions, not an absence of these things. It is a sign of maturity to be in agreement with two contradictory sides. F. Scott Fitzgerald believed that the ability to hold ambivalence was a sign of intelligence: “[t]he test of a first-rate intelligence is the ability to hold two opposed ideas in mind at the same time and still retain the ability to function.” Research shows that ambivalent people are actually more creative.
One of the most maladaptive styles of thinking is black-and-white thinking. That’s like all-or-nothing thinking, which can be easy to do with CF. I’m either healthy or sick; I’m either going to live or going to die. I want to be well and want the specialness of the sick role. I played with this kind of tendency for many years. Eventually I learned that I can be in-between. I need to allow the gray. It makes the loads of life easier to carry if we don’t fall into habits of categorization. I think our tendency to put ideas into boxes probably exists to help us organize the world. But we have to watch out when it gets in the way.
My relationship with CF epitomizes this ambivalence. CF is a pain in the you-know-what. It causes endless suffering, inconvenience, social awkwardness, financial demise, etc. You get the picture. CF is also a blessing. It has connected me to the most amazing people and has awakened a deep sense of awareness and appreciation for life. It makes me special and surrounds me with kindness. It makes me disciplined and grateful for all.
Nancy Mairs, an author with MS, wrote in Carnal Acts, “[t]o view your life as blessed does not require you to deny your pain. It simply demands a more complicated vision; one in which a condition or event is not either good nor bad but is, rather, both good and bad, not sequentially but simultaneously. In my experience, the more such ambivalences you can hold in your head, the better off you are, intellectually and emotionally. Categorical statements become meaningless. The saddest stories are shot through with humor. You come to tolerate people, ideas, and circumstances wholly at odds with your dreams and desires.”
The greatest lesson is to know that it’s okay to feel all sorts of things and to hold them all together. Like red paint and blue paint, when you mix them you get purple. That’s what CF is asking us to do — make purple paint!
There have been times when I’ve struggled with ambivalence in this CF journey. When I was getting sicker, I wanted both to keep working and to quit so I could focus on my health. I wanted a transplant and simultaneously didn’t. Now, I am so happy I’m well enough to work but sure could use some time off to slow down. When my twin sister Ana died, I was both deeply sad and incredibly relieved her intense suffering was over. I missed her advice but was glad I didn’t have to argue with her for wanting her way or telling me what to do.
I remember when Ana got her first lung transplant. She was so happy she could breathe but fell into a deep depression because she didn’t know what her purpose was anymore. For her entire life, her purpose was to combat this disease, to aim for health, and to fight against decline. Now, she was transplanted and well. She was asking, “now what?!”
I can relate to what my sister experienced so many years ago. We are supposed to be happy that we are alive or that a new treatment is saving our lives. And yet, at the same time, sadness exists, too. I’m so incredibly happy for my friends who have worked so hard to stay alive and preserve their lung function for so long, so that this triple combo can help them. I’m happy for them and scared, wondering what if the drug doesn’t work? What if there are terrible side effects? What if they can’t afford it? I’ll be honest: I feel tremendous gratitude for the scientists and corporate executives for choosing to invest in studying and treating CF, and I also feel such deep anger at their profit motive and taking advantage of sick people.
I know many of my CF peers have also felt ambivalence about getting a transplant. There is the intricate weaving between yes and no and not sure and not yet. It takes time to process the decision, gather all the subjective and objective information for the final choice that creates the fabric of your fate. And after transplant, just like many of my CF friends taking the Vertex drugs, they also might be asking, “now what?” We’ve prepared for a shorter life and yet also realize we may be in this game of life for the long haul. I’ve prepared for my death and pretended I’d live a long time as well. Both. And here I am, now, almost 48.
There’s one thing I know for sure: being alive is a wild and wondrous adventure. There is so much to experience — even when a possibility of health brings up sadness and other complex feelings. We are always being asked to train our spiritual muscles in our capacity to carry the good, the bad, the hard, the smooth, the sickness, the health, the blessings, the costs, the opportunities, the closed doors. Maybe we just need to learn to dance between the many sides of this next chapter of the CF experience.
Isabel is 47 and has CF. She lives in San Mateo, California, with her husband of 20-plus years, Andrew. She can be reached at isabear27@hotmail.com.
CFRD Education:
Questions And AnswersFrom Our Educator
Cystic Fibrosis Related Diabetes (“CFRD”) is an arduous journey — a complex disease on top of a complex disease, stretching the term polypharmacy to its maximum capacity. We learn how to manage CF, and then CFRD is placed in our laps like an unwanted perpetrator. The only possible retaliation we have is education — learning to manage what seems unmanageable.
I am so excited and honored to have a new column for CF Roundtable, “Diabetes Specialist.” I look forward to offering education and empowerment around CFRD management. I feel such gratitude to have a role in the CF community, to be able to support the warriors who live with CF, those who have taught me to hope and dream for me and my daughter.
Nothing in this column is meant to be medical advice about a specific situation but is only meant to be information.
Q: What is the difference between type 1/type 2 diabetes and CFRD?
A: Type 1 diabetes (T1DM) is an autoimmune disease where the body attacks the pancreas and destroys the insulin-making beta cells, leading to insulin deficiency and complete dependence on insulin. Type 2 diabetes (T2DM) is caused by a strong genetic component. The gene is often expressed in the presence of excess weight and inactivity. This causes fat and muscle cells to become resistant to the insulin the body makes, ultimately resulting in elevated blood sugars. Oral medications are the first line of therapy in T2DM. CFRD has hallmarks of both type 1 and type 2 diabetes. Patients with CFRD have some insulin insensitivity in addition to insulin deficiency because patients are not making insulin in normal quantities and they are resistant to the insulin present, whether through supplementation or insulin made by the body. This is especially true during times of infection. Let’s break down these areas:
Insulin resistance: having the CFTR gene mutation leads to exogenous glucocorticoids (steroids), chronic inflammation, malnutrition and infection. All of those complications lead to increased insulin resistance and ultimately, hyperglycemia (high blood sugars).
Insulin deficiency: having the CFTR gene mutation leads to thick mucus secretions blocking the pancreatic duct, fatty infiltration, and fibrosis. These complications lead to delayed and dysfunctional insulin secretions. Essentially, impaired insulin production.
CFRD is threefold: individuals with CF don’t make insulin as well as those without CF. They don’t make the levels of insulin the body needs, and the insulin that is made doesn’t work as well as it should.
Q: I have had a “borderline” CF diagnosis for years. I mostly just experience hypoglycemia. Shouldn’t I be on the lookout for hyperglycemia?
A: Individuals who have normal blood sugars without CFRD/Diabetes will vacillate between 60—140 blood sugar readings throughout the day. Hypoglycemia is common in individuals who are in the beginning stages of CFRD development. In the development of CFRD, you lose your first-phase insulin response, meaning the insulin that generally releases upon eating is delayed and less robust. This delayed release results in a spike in blood sugars about one hour after eating, as seen in most people with CFRD. After the delayed first-phase insulin response, the body then makes a robust and overzealous second-phase insulin response in an effort to overcompensate for the abnormally high blood sugars present. This second-phase response drives the blood sugar down, often too far, causing reactive hypoglycemia (postprandial hypoglycemia). This occurs in individuals who are not yet on insulin therapy. Generally, this is not unsafe as the body will rebound from the dip in blood sugars, which is usually short lived. Postprandial hypoglycemia is also generally accompanied by profound symptoms: confusion, fatigue, headache, anxiety, dizziness, and blurred vision, among other things. While this response is generally not unsafe, it is an indication that you need insulin therapy. When you have postprandial hypoglycemia, we know that your first-phase insulin response is compromised. While it seems counter intuitive to take insulin for low blood sugar, it functions as your first phase of insulin, eliminating that second robust insulin response, making you less likely to experience hypoglycemia after eating. This type of hypoglycemia is generally not in need of glucose tabs or glucagon, as it is usually resolved by food. Individuals who do not yet use insulin or require very little can usually eat carbs and not have a hyperglycemic incident, as their bodies are still making some insulin. This is called early dysglycemic CFRD, (which is essentially stage 1) versus early CFRD (stage 2) or full-blown CFRD (stage 3).
Kat Porco is a diabetes educator and the co-founder of Attain Health Foundation. She attended Humboldt State University to complete her Bachelor’s in Social Work, and later Boston University to achieve her Master’s of Science Degree in Health Communications. Her work over the past 10 years has been solely focused on supporting and advocating for the cystic fibrosis community. Throughout these years, she has seen firsthand the disconnect between the recommendations of the medical community and subsequent applicability for the patient community. Understanding this complex relationship, Kat felt that she could assist in bridging the gap to reach the ultimate health goals of both parties through integrative health coaching and diabetes education. Kat attended Duke Integrative Medicine and is a Duke Certified Integrative Health Coach, as well as being Nationally Board Certified in Health & Wellness Coaching (NBC-HWC) through the National Board of Medical Examiners. Kat is a Level II Diabetes Educator through the AADE and is preparing to sit for the Certified Diabetes Educator (CDE) exam in October. For questions about CFRD email kat@attainhealth.org
all in modulation
After I downed the first pill of Kalydeco (aka Ivacaftor) over three years ago, I wrote down all the health changes I experienced. I’ve added to the list over time and share it with you now. The list helped to keep track of the before-and-after effects of Kalydeco. Some improvements were immediate, while other effects were nil, moderate, or took two or more years as my body readjusted to a new normal.
Kalydeco is a CFTR modulator. It stands for CF Transmembrane conductance Regulator. CFTR is the name of a normal protein that transports electrolytes, such as salt and bicarbonate, into and out of the cell to places like the airway, digestive tract, sweat glands, and other organs. A CFTR modulator is a drug that makes the mutant protein work properly.
I found out about Kalydeco from a CF Roundtable director who followed the clinical trials closely and was very knowledgeable about which CF mutations would benefit from it. Kalydeco, by itself, works mostly on two classes of CFTR mutations called class III and IV, in which abnormal CFTR proteins are at the cell membrane or surface, but they don’t work correctly. Kalydeco forces (or modulates) the mutant CFTR proteins to do their job of allowing the normal flow of electrolytes in and out of the cells.
Four years ago, Kalydeco was one of two CFTR modulators available. It was FDA approved for only a handful of specific CFTR mutations, but not my specific class IV mutation. My class IV mutation, D1152H, was shown to respond to Kalydeco in small research studies only. This meant that in order to get it, I’d have to receive it off-label.
I desperately wanted to try it and approached my CF clinic. They had concerns regarding ordering this for off-label use and warned that an appeals process might follow. After conversation, debate and, I must admit, quite a bit of frustration, they ordered Kalydeco at my request. My insurance approved it quickly and, within days, I was thrilled to have this hopeful new drug in my hands.
Because I was receiving it off-label, I didn’t qualify for Vertex’s patient assistance program that covers $110 of my monthly copay, so I had to pay the copay of $125. I thought I’d try it for one month. If there were no improvements, then I’d stop it and save money. But that didn’t happen. What did happen were quite a few large and small miracles.
Lungs: Before Kalydeco I had violent hacking fits of coughing with thick green sputum day and night. I also had frequent lung collapses, bacterial colonization of my lungs that would turn to pneumonia, and I frequently coughed up blood (hemoptysis). After a few short weeks, my cough became less spastic, less frequent, and I had fewer secretions with my cough. My tissue usage from spitting out sputum, blowing my nose, and use during nebulizer treatments, went from going through a large box every two or three days to one box every few weeks. I saved money right away from not buying megapacks of tissue boxes as frequently.
However, Kalydeco did not change the scarring and bronchiectasis in my lungs. As a result, my airways have many areas that are fragile and frequently lead to inflammation, pneumonia, and coughing up blood. In fact, two years ago, I coughed up massive amounts of blood similar to the possessed child in the Exorcist movie, except it was red blood, not green, well, puke. I joke, but this was a life-threatening situation where I had to undergo an emergent, life-saving bronchial artery embolization procedure. It took five hours for them to find all the bronchial blood vessels that were leaking into my airways. Since the history is for the hemoptysis to recur, only time will tell if my new normal of less hemoptysis is due to the embolization procedure or from the modulator. My airways still clog up if I don’t exercise or complete hours of nebulizer treatments and airway clearance every day, but the clogging isn’t so heavy or thick. There are also fewer episodes of lung collapses. Breathing overall is much easier and deeper now
Laughing: Before, every laugh or even little chuckle was accompanied by coughing spasms. Sometimes, I’d hold back a laugh, afraid of the violent cough or flying sputum that might ensue. Since I love to laugh and it’s the best airway clearance, holding back didn’t work. Instead, I’d had to make sure I had a tissue readily available at all times. This is one of my favorite benefits after starting Kalydeco: being able to laugh freely without coughing.
Costochondritis: My rib cage has always taken a beating with the constant cough that caused rib tenderness and pain or costochondritis (inflammation of ribs/sternum). Although the costochondritis still remains an issue, there has been less pain overall. I’m still holding on to the hope that this will improve in time.
Sinuses: My sense of smell has been terrible for about 20 years and I hoped this would improve. Unfortunately, it hasn’t changed much since starting Kalydeco. My olfactory receptors (responsible for the sense of smell) are fickle and sometimes work correctly, while other times, they either interpret odors incorrectly (dysosmia) or fail to detect any odors. I may comment to a friend that it smells particularly good outside, only to find out by their confused look that there’s an awful fertilizer smell. Or vice versa happens: I smell something terrible but am told there’s a wonderful rose-flower aroma. Sinus infections still occur, but they don’t last as long and respond more quickly to antibiotics, which is always good.
Sleep: Pre-Kalydeco, I would have difficulty falling asleep and would also wake up every night from coughing. I often couldn’t return to sleep. I’d stay up for two hours or more and, when I did sleep, it was, without fail, an interrupted sleeping pattern, often leaving me fatigued throughout the day.
Post-Kalydeco the coughing at night is a fraction of what it was. You’d think that I’d be getting a good night’s sleep now. Unfortunately, sleep still eludes me. It seems my faulty biorhythms and habits, which developed from years of chronic steroid use and CF flare-ups, have made it very difficult to break the habit of suddenly waking up in the middle of the night and being unable to fall back to sleep.
Falling asleep has become somewhat easier by not viewing any electronics one hour before bed or at any time during the night. Listening to meditation recordings, such as the Headspace App, also has worked wonders for falling asleep. Making a sleep diary was helpful because I discovered I only need seven to seven and a half hours of sleep every night to sufficiently function. And not worrying about being tired the next day also took away a ton of anxiety associated with not sleeping well. These small restructurings of habits have only been possible once the CFTR modulator reduced the coughing at night. Still, sleeping better is definitely a work in progress.
Exercise: Exercising prior to Kalydeco was difficult because of poor endurance and easy fatigue. After 30 minutes to one hour of forcing myself through any exercise, I’d be dead tired for the rest of the day. Yet, without exercise, my lungs would clog up even more.
It took a while after that first pill but, eventually, I felt excited to exercise since I could keep up during an exercise class. The best part is my muscles returned and my bone density stopped declining. In general, I feel invigorated for the rest of the day.
Eyes/Mouth: This one was a particular surprise. Before Kalydeco, I had scant saliva, dry mouth and eyes, and geographic tongue (where your tongue looks like a map of uncharted territories). I didn’t know this was from CF. Two days after that first pill, I got my first clue that the mouth dryness was indeed due to CF. It was morning and thankfully I was at the table alone having breakfast. I yawned and saliva drooled out of my mouth on to my shirt. What just happened?!? I hoped I hadn’t had a stroke or developed facial palsy. After reassuring myself I was alright, I thought it was just a fluke. Then it almost happened again, but I was ready for it the next time. Who knew that the salivary glands carried CFTR? Well they do. Eventually, my tongue lost the map and I got used to the mouth salivation. And I’ve stopped drooling!
My eye dryness is better. I could stop the daily lubricating eye drops since my lacrimal glands responsible for producing tears now work properly.
Sweat: Prior to Kalydeco, I hardly ever perspired on hot days or during exercise. After starting it, I began sweating up a storm after exercise, looking as if someone had poured a bucket of water over me. I have to drink oodles and oodles of even more water and electrolytes because of it.
Weight gain: All CFTR modulators must be taken with fatty foods every 12 hours. The health improvements led to weight gain — 15 pounds and counting! My body no longer expends all its energy on fighting inflammation and infection or on coughing.
Cost: Kalydeco has since been FDA approved for my CFTR mutation. Through Vertex’s patient assistance program, I now only pay $15/month. Certainly, a miracle!
Psych: Before Kalydeco, I developed regular bouts of anxiety about the future or the possible lack of one. Questions plagued me: what will I be able to do if I get worse? How will my poor health affect my relationships and plans with my family and friends? You get the picture of the road I was going down.
My mental state took its sweet time to improve until recently. Now that I feel confident that the benefits of this drug are sustained, I have much less anxiety. When I do, it’s often situational and brief. I feel that I have a future — one of relative health, one that I’ll be around for, look forward to, and can plan for, as long as I make sure to have my health as my #1 priority.
I’m forever grateful to the CF Roundtable director who was correct in her assessment that this CFTR modulator would be beneficial for me. I can’t thank her enough for helping improve my quality and, I don’t doubt, quantity of life.
Jeanie is 57 and has CF. She is a physician and Treasurer of USACFA. She lives in Los Angeles with her husband and has three adult children living throughout the U.S. She welcomes you to contact her any time. Her contact info is on page 2.
From Planning For Sickness To Planning For Wholesomeness:
The Changing Landscape Of Adult CF Life
The discomfort was becoming quite unbearable. I adjusted my posture again on the recliner, but I just couldn’t seem to get any relief. As I watched a documentary on assisted suicide and dying with dignity in the U.S., I couldn’t help but think of my own fate. My lungs were aching and inflamed, which was causing me to feel an ongoing shortness of breath and chest discomfort. I paused the show so I could go do a treatment and take an anti-inflammatory, hoping for a reprieve. I couldn’t ever remember feeling that bad. I made it through the documentary, and through the rest of the day as best as I could. That night I woke up to sudden coughing and uncontrollable spurting of blood. Although I had become accustomed to bleeding while coughing, this was probably the biggest such bleed I had ever had. I anxiously and gently carried myself to the recliner to finish my evening slumber propped in an upright position. As early as I could, I put in a call to my CF doctor, who told me that it was probably time for me to be hospitalized with IV antibiotics. I was feeling so sick, that I couldn’t wait to get there.
After a successful two-week treatment of IV antibiotics, I was feeling back to my old self. I went back to my normal routine of raising my kids and working full time. However, just a few short weeks later, I was sick again and feeling just as bad as I had been before the hospital visit. So, the doctor ordered another course of antibiotics. The recliner became my IV treatment location at home, so that I could hook up in the early morning or during late-night hours and fall asleep to the rhythmic drips. I didn’t understand what was going on and recognized that my CF might finally be changing. Depression crept in as I wasn’t used to being so sick. My body had a mind of its own.
The FDA had approved Orkambi earlier that same month, and I decided to get started on it as soon as possible. I was hoping that the drug might be able to stop whatever downward spiral my CF had suddenly started to take. As I held those two pink pills in my hands for the first time, I was overwhelmed with emotions. I hoped that my health would change for the better, and yet I wasn’t sure how the drug would affect me, or even if would help me at all. It was all so scary and exciting at the same time.
As the weeks and months went by, I noticed very subtle changes to my health. My sinuses felt a little clearer, my mucus was thinner, and my weight increased. But the best part of all was that my frequent lung bleeds had come to a halt. It was an amazing lift in my mental and physical well-being. However, I had a side effect of chest tightness that would affect me like clockwork every day. But despite that, I was still convinced it was better than having regular episodes of hemoptysis.
A couple of years later, it was thrilling to see the next generation of CFTR modulators coming to the market. I was excited to start Symdeko, as it seemed to be slightly better than Orkambi. As I popped the first yellow pill, I was feeling similar emotions to when I started Orkambi, and I wondered how this new drug would change things yet again. Over the course of a few days, I was feeling quite ill. I was having fevers, coughing, and muscle aches. I was confident that this was temporary due to the medication change, and I pushed through. Those initial side effects subsided, but then my lungs went into an uproar. For approximately two months, I had significant coughing and bleeds. I did two courses of antibiotics during that time, when the recliner once again became my good friend. I was certain, however, that the downturn in my health was just my body adjusting to the modification to my cells.
After pushing through the rocky start, I finally came out on the other side. My sinuses were so clear that I was able to stop sinus rinses. My coughing frequency went down significantly. The lung bleeds stopped once again.
Now, after a year and a half on Symdeko, I am feeling better than I have in a long time. My lung function has gone up 4%, my weight has increased 20 pounds, and I’m no longer bothered by constant crackling sounds in my lungs. It’s an incredible and amazing improvement for me. I am now doing things that I have always wanted to do. In addition to raising my two boys and working full time, I have started my own business providing IT support, in addition to side jobs in gardening, photography, and writing. My first book is in the works and I assist in writing scripts for motivational videos. I regularly volunteer for organizations that help the homeless and people with low incomes. I also gather donations from the surrounding communities to provide to the needy. I am even planning for my retirement — something I had never planned for before. I can travel for work and leisure whenever I want without having to plan it around my sickness. I am now excited to be a part of the challenges that middle-aged women have, like coloring my gray hair, going through menopause, and sending my children off to college. I have so much energy and hope now and have no doubt that I can tackle my entire bucket list.
My living room recliner — once a depressing part of my home that was riddled with memories of sickness, discomfort, and laborious treatments — is now a place of rest after an activity-filled day, while my family gathers around me, and we laugh, play, and plan our future adventures together.
Leah Sands is 38 years old and lives in Detroit, Michigan. She loves photography, sports, and traveling. She can be contacted by email at leah.sands@gmail.com.
Hope Realized With
CFTR Modulators
I was just as excited as the rest of the CF community when Orkambi was finally approved by the FDA. I have two copies of the F508 mutation. I worked with my cystic fibrosis clinic and Vertex to gain access through my health insurance and Vertex’s patient assistance program to start taking Orkambi. It was awful. Within 30 minutes of taking my first purple pill, I could not breathe. My chest was so tight I felt I was gasping for air. I could not walk around. I couldn’t even carry on a conversation while sitting. My CF team was convinced it was psychosomatic. But I was not crazy! They worked with me by quartering the dose and “working my way up” to the full dose. Every time I would try to increase the dosage, the awful symptoms came flooding back to my body. I felt like I was drowning in mucus, despite increasing my therapies to four times a day. I constantly felt like I was breathing through a straw. No matter how many times I reached for my rescue inhaler, I received no relief. The addition of these symptoms to my fatigue and extensive daily regimen of therapy meant that Orkambi wasn’t working for me. I decided to discontinue Orkambi after trying to increase dosage to the recommended amount for over seven months. Did I mention my pulmonary function tests kept dropping over those seven months? Once I discontinued, my PFTs leveled off for a while.
When Symdeko became available, I was much more hesitant but still optimistic. I was encouraged that there were far fewer adverse reactions and side effects with it than with Orkambi.
Symdeko was a different experience for me. I don’t recall ever having the drowning effect. I didn’t have the same shortness of breath or chest tightness. In fact, I didn’t really notice anything at all for the first couple of months. BUT when I went for my CF appointments, we started seeing my PFTs improve. My FEV1 increased from 46% to 57% within four months of starting Symdeko. I experienced fewer exacerbations. Pre Symdeko, I was getting sick monthly. In the past 17 months, I have been on IV antibiotics once. My body can fight most infections just with the help of oral antibiotics.
The most astonishing impact is I feel more energized and now have the allotted energy necessary in my daily life to complete my duties as wife, mom, and optometrist. The ability to exercise has also helped improve my health. Furthermore, I am able to work full time. To be a productive member of society in the traditional sense is rewarding. The last time I worked full time was in my early 20s. I excelled in college earning a BSE in Health, Human Performance, and Outdoor Recreation. I also attended optometry school and earned my Doctor of Optometry. I have run my own optometry practice. Yet, I have never been able to work full time. Fatigue has always been a hindrance. To be afforded the hope that my health doesn’t always have to be in decline goes beyond words for my mental health and outlook on life. When you get to wake up each day with a fighting chance, you are willing to put one foot in front of the other.
I now go for walks, bike rides, lift weights, and even started rollerblading again for fun. Not for my health — for fun. It’s like I’m a teenager again! This medication has been such a blessing. I pray everyone in the Cystic Fibrosis community will have access to a life-changing medication soon.
Summer is 38 and has CF. She’s an optometrist living in sunny Southern California with her six-year-old daughter and husband of 13 years. As a native Texan, she takes advantage of the more moderate weather and every beach activity made available by living on the coast. She takes pleasure in watching her daughter’s personality and skills grow and develop. Her daughter’s extracurricular activities keep her busy outside of the office where Summer enjoys the rewarding, instant gratification of improving patients’ vision. She can be found on Instagram @summasumma.joy
Waiting for better
When you have a disease like cystic fibrosis (CF), you’re always waiting for the next best thing: something to help you breathe better, something to make the pain and symptoms go away, something to give back the days and weeks spent doing treatments in a given year.
As soon as Orkambi came to market, I started communicating with my CF care team to get access. After maybe a month of approvals and back and forth with my insurance company, I got my first box of Orkambi. I remember this out-of-body feeling. Years of research, fundraising, clinical trials, and hope led to this moment. Was I going to be “normal”? Could I stop doing all these time-robbing treatments? It was the first time that people with my CF mutation had a modulator, and my head was riddled with emotions.
Years later, I look back and find myself disappointed by Orkambi’s efficacy and side effects. I was short of breath for years, had chronic hemoptysis, and didn’t see an improvement (or decrease, for that matter) in my FEV1. My lung function remained stable but, due to side effects, my overall health deteriorated within days of starting the drug. In a word, I am disappointed by how the hype surrounding the drug actually translated into a struggle.
I’m not quite sure why I kept taking Orkambi. Perhaps it was the denial that my first “miracle drug” was short of a miracle or maybe it was the “underlying help” that Orkambi is doing — making my body more “normal” even if I can’t feel it.
A couple years later, Symdeko came to market. Once again, I found myself in an out-of-body experience when that first box arrived. The PFT data were better than Orkambi and the side effects didn’t seem as grueling. However, within a month after starting Symdeko, my liver enzymes went through the roof. It is difficult to describe the disconsolateness that came when I learned of the high levels. For months, I got blood draw after blood draw, hoping my LFTs would decrease — they didn’t. They remained elevated for months. My care team eventually took me off Symdeko and left me without a modulator. It was the first time in years that I wasn’t on any sort of Vertex drug, but I didn’t feel terribly different. My sputum was thicker, for sure, but I didn’t feel notably sicker.
I moved to a new state after finishing my Ph.D. and switched to a new CF clinic in Maryland. During my first visit, the doctors wanted me to try Symdeko again. I wasn’t reluctant. I knew that the “triple” drugs in the pipeline were essentially Symdeko with an additional drug. So, for the third time, I started taking a CFTR modulator. This time was different: my liver enzymes didn’t go through the roof. I’ve been on Symdeko ever since.
I choose not to overthink why my second round on Symdeko is going well. My FEV1 has improved by 5% and my liver seems to be in better shape. Were my liver issues related to the drug? I’ll probably never know. In CF, I’ve learned to compartmentalize. Trying to add up the ups and downs of health can eat you alive. Even as a scientist, you can’t fully predict or understand why things happen the way they do.
My experience with CFTR modulators has been far from a fairy tale story. The side effects, combined with my constant battle with my health insurance, leave me feeling neutral at best. But I know that if the triple therapy comes to market, I’ll be thrown back into that same out-of-body experience as before because, with CF, we have been conditioned to believe that the next best thing is on the horizon.
Reid is 27 and lives in Silver Spring, MD, and works as a consultant specializing in regulatory sciences and federal health. Reid enjoys tennis, travel, and trying new foods. Reid received his Ph.D. in Biomedical Engineering and enjoys merging his perspective as a patient with research. Reid can be contacted at reidwdamico@gmail.com.
Six Ways Kalydeco Has Impacted My Life
Six years ago, on August 30, 2013, I started taking Kalydeco. I was underweight, struggling to get pregnant, and my lungs weren’t in the best shape. I was hesitant to start taking these little blue pills twice a day because I didn’t want to rely on yet another medication. I had the right mutation (G551D), but I didn’t want to add more pills to my weekly container, and another medication to my treatment routine. In hindsight, I think I was also worried about getting my hopes up about it actually working. Six years ago, I had no idea what impact one medication could have on my life! In the six years since I’ve started taking Kalydeco...
1. I have gained over 20 pounds and have kept on the weight.
I started Kalydeco when I was 26 years old and I was weighing in around 109 pounds. I struggled to gain and maintain my weight and, like many people with CF, I could lose weight easily and not on purpose. Within two months of beginning Kalydeco, I gained SIX pounds! Today, I weigh about 130–135 pounds. Having said that, at my latest clinic appointment in July I weighed in at almost 138 pounds and felt a little uncomfortable with that number.
I’ve had to have a completely different mindset when it comes to my weight in recent years, and it’s been hard to change my thoughts, to be honest. It’s tough seeing the higher numbers on the scale, noticing the CF (constantly pregnant-looking) belly and tighter clothes. But I know, as a person with CF, this is a good problem to have. I know it’s healthy for me to have a couple extra pounds to spare, especially as fall, winter, and flu season approach and my body works harder to keep itself healthy. It has been nice to see my body finally have some shape, and it is one less thing to worry about when I go to my clinic appointments, so that’s always a plus. This has probably been the biggest physical and noticeable change for me since starting Kalydeco.
2. My overall lung function has stayed “normal.”
Before I started Kalydeco, my FEV1 was 77%. In July 2019, my FEV1 was 86%. My baseline lung function over the last six years has been between 85—88% FEV1, even at one point reaching as high as 92%. That’s “normal” lung function for over six years! I will admit I was a little disappointed when I wasn’t seeing my numbers get much higher than 88% (I wanted to be in the 90s for so long). But when I think back to a typical trend being a slow decline in lung function over the years, gaining about 10% and maintaining that FEV1 for so long has been wonderful and I am truly grateful!
In addition to this increase in my overall lung function, my doctor pointed out to me that my small airway function had increased tremendously. After being on Kalydeco for about two months, my small airway lung function went from 72% to 90%. While my overall lung function only increased about 5% in that time, my small airway function increased by almost 20%. That was huge! I could tell then that this medication was working for me and that it was something I was definitely going to continue for a long, long time.
3. I’ve added six more healthy years to my marriage.
My husband, Tim, and I got married in October of 2010. In that time, we have moved four times, bought a house, bought a couple vehicles, both started new jobs more than once, taken a couple of mini vacations, started a family, and have created and shared many wonderful moments and memories together. Tim will always be one of my greatest motivators in life. I believe it takes a strong person to marry someone with CF, and I feel like I need to do my absolute best in taking care of myself so I can be the best and healthiest partner for Tim. Due to taking Kalydeco over these last six years, I haven’t had to stress about my health as much as I have in the past, and it has allowed me to enjoy the life that we have been creating together.
4. I’ve been pregnant three times.
One of my proudest achievements has been becoming a mother. It is all I ever wanted in life — getting married and having a family — and here I am, living my own dream. Shortly after beginning Kalydeco, while going through fertility treatments, I found out that I was finally pregnant after years of trying. My doctor and I had a long discussion about whether to stay on Kalydeco while I was pregnant. Ultimately, I decided to come off during the first trimester, during the critical weeks of fetal development, and see how I felt. Unfortunately, this was also the middle of winter, so after being off Kalydeco for about nine weeks and coming down with a terrible infection, which led to an abundance of mucus, we decided I needed to get back on Kalydeco right away — I was 15 weeks pregnant at this point. My lung function took a huge hit and dropped to 69% (the lowest it’s ever been) but, luckily after getting back on Kalydeco and adding Tobi to my regimen, by the end of my pregnancy my lung function was back up in the 80s. Other than this hurdle, I had a very textbook “normal” pregnancy. I was able to control my sugars with my diet so I did not end up with gestational diabetes. On August 8, 2014, our daughter Anna entered the world. It was one of the best days of my entire life! I was fortunate enough to be able to breastfeed Anna for 13 months, while maintaining my weight, and I am so proud of that. She was an easy baby and filled our hearts with so much love and joy. Anna is now five years old and in kindergarten. She’s smart, creative, inquisitive, and so silly.
When Anna was around two years old, we decided to go back to our fertility doctor to work on having another baby. Little did we know, we would be surprised with twins! I was so nervous about my body being able to handle a twin pregnancy, and how I would manage two newborns at once, but I was also so excited at the possibility. Unfortunately, our little twin boys were born too early. My body went into “silent labor” when I was just 19 weeks and 4 days pregnant. The boys were born about two hours apart and were too small to survive. Their lungs were not developed enough and both boys were “born sleeping.” It was absolutely one of the most devastating days of our lives. Just a few months after their birth, I got their tiny footprints tattooed on my wrists (Baby B on the left, Baby A on the right — the sides I held them on in the hospital) so I will forever remember our babies. The doctors never really gave an explanation as to why this happened: it was unfortunately a “sometimes those things happen” tragedy, and it had nothing to do with me having CF.
Tim and I couldn’t let this be the end of our parenthood journey. We couldn’t let this memory be the last of us trying to have children. As soon as my body and mind were ready, we tried again. This time we went through IVF (previous two times were IUI) to ensure that I would get pregnant with only one baby this time around. Thankfully, our first round of IVF worked, and I was pregnant again just a few months after having the twins. This third pregnancy was tougher mentally and emotionally, but I had a wonderful set of doctors who monitored me very closely. I was diagnosed with gestational diabetes and controlled my sugars with insulin this time around. My body and health held up wonderfully, and our rainbow baby, Calvin, was born on February 8, 2018, just one year and three days after his twin brothers. Calvin is nothing like his sister — he’s loud, he’s moody, and he’s always getting into everything, but man is he cute, and he laughs all the time! Calvin keeps us on our toes, and he is a complete mama’s boy. I was able to nurse Calvin for 16 months and he’s a very healthy and silly little boy.
Without Kalydeco, I don’t know that my body would have been strong and healthy enough to carry these pregnancies, let alone get pregnant at all. After the lung function dip when coming off it during Anna’s pregnancy, I vowed to myself and my doctor that I would never come off Kalydeco again, and I have kept that word. Kalydeco has allowed me to achieve the dream of motherhood, and I could not be happier.
5. I’ve been able to continue working full time as a teacher.
I graduated with my bachelor’s in 2010 with a degree in Elementary Education. I was so excited to have my own classroom, but finding a teaching job at that point was very difficult. There were hundreds, even thousands, of applicants for any position. For two years I worked as a special education assistant while I applied like crazy for teaching positions. Finally, in 2012, I was offered a kindergarten position and couldn’t have been more excited. My doctor, on the other hand, wasn’t thrilled about my first teaching job being in a kindergarten classroom, but she was excited for me. During my first year of teaching, I lost several pounds and struggled to keep my weight at a healthy point. Luckily, the next year, 2013, I started Kalydeco and I didn’t have to worry so much about my weight anymore.
Since then, I’ve taught kindergarten for two years, first grade for one year, third grade for three years, and just last year I moved to a new school district and I’m now in my second year of teaching fifth grade. Teaching is my passion and I absolutely love it! I love working with children every single day and knowing that I’m making a difference with the students each year.
Of course, I have been exposed to many germs, but I have been very fortunate that I haven’t caught anything too extreme. I have had my fair share of chest infections over the years, but with my annual flu shot and my daily doses of Kalydeco, I have stayed healthy enough to maintain this career, and I am loving every day of it!
6. I have graduated with my master’s degree.
In May 2019, I officially graduated with my Master’s Degree in Teaching and Learning with an emphasis in Engineering and Math. This is something that I never pictured myself doing. The idea of going back to school did not appeal to me whatsoever, especially when I was working full time and starting a family. But in 2018, the school district I was working in presented a professional development opportunity that I couldn’t pass up, and so began the journey towards my master’s. I started when I was pregnant with Calvin and finished when he was 15 months old. Thankfully, I completed the classes all online, and worked with a fabulous, supportive group of teachers who helped and encouraged me along the way.
After completing the master’s degree, I decided to take a break over the summer but, this fall, I have registered for my first class beyond my master’s. The highest I can go on my pay scale is 32 credits beyond a masters, and my plan is to get there, slowly but surely, three credits at a time.
The idea of furthering my education was never an option in my mind. I honestly didn’t think I’d live long enough to reap the benefits of the higher pay or the idea of retirement, but now that I am healthier than I have been in years, I just want to keep going! I want to make the most of my days and years. I don’t want cystic fibrosis determining my future.
The past six years haven’t been completely blissful in terms of my health, and there have been a few bumps in the road. I’ve had my share of lung infections, but fortunately have been able to control them with oral antibiotics. Also, just earlier this year, I battled with six months of random episodes of hemoptysis that baffled my doctor and me for a little while. Thankfully, we got that back under control when I restarted my Azithromycin and began taking Colistin instead. I wish it wasn’t necessary to keep adding new medications to my treatment regimen but, if I want to maintain my lung function, overall health, and busy lifestyle, then that’s worth it to me. I am so lucky and grateful to have these little blue pills in my life. They are keeping my health stable so that I can continue thriving and enjoying my life! For now, for me, this is as close to a cure as I am going to get, so I really can’t complain.
Colleen is 31 years old, and lives with her husband, Tim, their two children, Anna and Calvin, and two cats, Archie and Nellie, in Decatur, IL. In her free time, Colleen likes to spend time with her family and friends, read young adult books (especially ones recommended by her students) and watch crime shows on TV. You may contact her at colleen.veitengruber@gmail.com or on Facebook.
What CFTR?!
Buzz buzz. Psst. Psst. CFTR modulators? What’s that you say?!
Shortly after I popped out of the womb 32 years ago, scientists discovered the gene responsible for my cystic fibrosis. What is CF? What did my future hold? My fate was predetermined according to statistics.
Fast forward to when I’m 10 years old. Doctors told my parents that a cure wasn’t far off, maybe in the next decade. Okay, I thought, I can make it 10 years until a cure is available. Inhale (wheezes). Exhale through the sludge in my airways. I can totally do this.
Fast forward. I’m now 20. Where’s the cure? Is it around the corner like you originally said? No? New treatments, okay. That will sustain me for a while. I hope. And I’ll keep going when my lung functions dip below 50% and they mention the need for a double lung transplant in the future. Okay, yes, no problem. I’m totally fine. I’ve got this.
In the meantime, research articles, opinion pieces, and press releases all proclaim the closest thing to a cure are the new CFTR modulators. They’re the miracle key to fix the broken lock for 80% of the CF population — but not for me. I don’t have the specific genotype the majority of the CF population has: DF508.
It’s a difficult balance between sharing in the enthusiasm for your friends, young kids, and those who are clinging to this hope of a (close enough) cure, while also grieving for myself and the others in my same boat who won’t get these modulators.
Cheering from the sidelines with tears in my eyes and filled with mixed emotions.
Then I’m faced with saying goodbye to my lungs and hello to a stranger’s. Two years ago, I had a double lung transplant. I grieved for the life I envisioned with my own lungs and celebrated that I’m still here with new breath. It’s a teeter-totter of emotion.
Those of us with new lungs won’t experience the dreams of the CFTR modulators in the same way that our fellow CFers have and will. Our dreams have had to shift. Otherwise, we wouldn’t be here to talk about it. It’s bittersweet. We’re grateful for the gift we’ve been given. We realize our dreams have taken an unexpected course. We grieve the loss. We embrace the gain. New life in a different embodiment.
My CF came with aggressive lung disease and relatively few GI issues. I couldn’t wait for the modulators to apply to me and my unique genes. Time ran out, but a transplant saved my life.
It’s been surreal to breathe in a way I never imagined. I live with the constant unknowns of the transplant life. Blocking out the coulda, woulda, shouldas that drive me insane when thoughts wander to if I could have held out a little longer.
But all that does is create unnecessary anxiety because the truth of the matter is that nobody knows. Even though transplant life feels more unpredictable than CF lung life, that is an illusion, too.
I want so desperately for everybody to have the modulator and hope that it works for everyone’s individual needs. I don’t want anyone to experience the transplant life if there’s another way. It’s also true that I wouldn’t change how my story has evolved.
What we know as CFers, and those of us who are on the other side of transplant, is that we are grateful for what we have been granted in any capacity. That looks different for just about everyone. For this moment, I celebrate the fact that dreams are coming true in our little corner of the world: a sustainable solution to alleviate symptoms until CF stands for Cure Found.
Lara Govendo is 32 years old and resides in Vermont (for now) as a wild, adventure enthusiast, while holding a Master’s Degree in Mental Health Counseling. She writes about living out loud and develops educational programs to restore hope to those in need. You can now find Lara traveling on the regular, exploring the glorious outdoors, and belly laughing with her loves. She is passionate about connecting with like-minded people, and you can find her at www.laragovendo.com and on Facebook/Instagram “Lungs4Lovey,” where she flies by the seat of her (no) pants.
How CFTR Modulators Saved Me From Transplant
When I was 13 years old, my family and I were told that I would most likely have to receive a lung transplant before age 18 to successfully make it past the point of being more than a statistic. While living in Indiana, there wasn’t a children’s hospital that performed lung transplants, which meant my family and I would endure a six-hour-plus car ride to St. Louis once every six months. The thought of my family crammed in one vehicle for over six hours was scarier than the thought of transplant.
Luckily for us, not long after we started making our routine trips to Missouri, the VX-809 (aka Orkambi) drug trial started. If you knew someone with CF and don’t recall this time, you were living under a rock. At the time, this was considered as close to a cure the cystic fibrosis community has ever seen. VX-809 is considered a CFTR modulator because it helps correct the underlying genetic defect. The drug would regulate water and chloride intake at the cellular level, ultimately combating our thick and sticky mucus.
Eligibility requirements for the study were minimal — you had to be above the age of 12 and have lung function of 40% or higher. There was also a limited number of patients who could be in the study in each state. It was like the hunger games within the CF community.
My mom, being my number one advocate since before my actual existence, knew everything about the subject. Imagine being in high school and having pressure from your parents to get all As; that was my mom in the PFT lab. I hadn’t reached 40% lung function in a long time, always sitting at 39%. When the day came for me to exhale into the machine with at least a 40%, I was confident. I had been doing everything in my power to get to the goal I was so close to. After three breaths at 38%, on the fourth try I got a 41%. I had outdone myself.
For the next few years my life would consists of blood draws, pulmonary function tests, EKGs, and sputum cultures. Back then, I had no idea how much it would change my life.
From ages 13—18, while in the drug trial, I was driving back and forth to St. Louis. After I turned 18, my doctor was certain I wouldn’t need a transplant for a couple more years so I could continue being on the inactive transplant list with the Indianapolis adult hospital. Right before Symdeko came out, I was taken off the list completely. I’m currently 23 years old and I am still sitting at a 38% baseline lung function.
If you ask most anyone who knows about either one of these drugs, my lung function should have increased by about 12%, and they’re right. I’ve heard from several other CFers that typically you either have too many of the side effects (and therefore go without the new drug) or your numbers increase. I stay the same. For 10 years, I have hovered around the same 40% lung function and I honestly couldn’t be happier. I’m 23 years old and am able to work, go to school, and have a social life all while not requiring oxygen. Knock on wood. I still get sick, I still have hospital stays but, for the most part, I’m healthy. I have CFTR modulators and tiny lab mice to thank for that.
Hannah Schau is 23 years old. She lives in Mishawaka, IN. She loves traveling, reading, shopping, watching football, and staying active!
Autumn 2019 Recipients Of The Lauren Melissa Kelly Scholarship Announced
In our evaluation, we look for students who demonstrate tremendous academic achievement, community involvement, and a powerful understanding of how their CF — matched with these achievements — places them in a unique situation to gain leadership roles within the community. Our scholarship is open to all pursuing any degree, from associates to Ph.Ds. We believe that any higher education is a strong foundation for advocacy and involvement in the CF community.
Nancy Wech established this scholarship in honor of her daughter, Lauren Melissa Kelly. This semester’s winners demonstrated outstanding potential, just like Lauren years ago. Lauren was an inspiration to all who knew her. An incredible leader and scholar, her drive and success are the foundation of her memory. She was transformative in every aspect of her life. She had distinguished herself as a member of the Golden Key Honor Society, Mortar Board, Phi Upsilon Omicron, Gamma Beta Phi, Delta Gamma sorority, and was chosen as one of ten Senior Leads at the University of Georgia. She acted as one of the re-founding members of the Phi Kappa Literary Society and was significant in the metamorphosis of the Z Club into the William Tate Society. Even after losing her battle with cystic fibrosis late in her senior year, her hard work and memory continue to live on through her inspiring involvement.
We are pleased to announce Sarah Salas and Alexandra (“Ally”) Bannon as the recipients of the scholarships for this semester. They will each be awarded $2,500. Congratulations to both!
Sarah Salas is an accomplished young woman pursuing her Master’s Degree in Speech Language Pathology at Texas A&M, where she is building upon her skills earned from her Bachelor of Science Degree in Communication Sciences and Disorders. Her professional goals include regaining and improving people’s communicative abilities by providing speech therapy to those in need. She hopes to grow in her knowledge of clinical research and gain experience in all aspects of speech and language disorders. She has striven to be a leader in various groups in her community, including being young group leader on annual retreats, Vice President for the campus’s Catholic Student Organization, and organizing her yearly Cystic Fibrosis Great Strides Walk team. She has given presentations to CF families, educating them on the importance of compliance with therapy and managing life with CF. It brings her much joy to help others and to be a positive light.
Ally Bannon is a motivated young woman who is studying electrical engineering at the University of Michigan. While still in high school, she completed an Early College program with a Science, Technology, Engineering, and Math (STEM) focus, which gave her an opportunity to experiment with computer and engineering practices. She was also inducted into both the National Honor Society and National Technical Honor Society and plans to be a part of professional organizations on campus. In her community, she has volunteered at the Special Olympics, Toys for Tots, and Project Christmas, as well as supporting cystic fibrosis fundraising with Great Strides Walks and 65 Roses Dinners.
Both Sarah and Ally demonstrated the leadership, intelligence, and drive of Lauren Melissa Kelly. All of us at USACFA look forward to seeing them further develop their leadership and advocacy in the cystic fibrosis community.
Scholarships are offered for both the spring and fall semesters each year. More information, including the application and relevant deadlines, can be found on our website. For questions about future scholarships or anything related to the application process, please contact us at scholarships@usacfa.org.
wellness: magic
I recently traveled to Nebraska for my 40th high school reunion. You read that right…40 years. I am not sure who was more surprised by my presence there: me or the 200 or so classmates who showed up. The funny thing is that all this time, I thought that very few people from my high school class even knew that I had anything wrong with me. Of course, my close friends knew. I mean, I spent a fair amount of time in their bathrooms, so… They were also well aware of how sick my older sister was during that time, so they saw my future just as I did. But of the 500 or so people in my class, I thought only about a half dozen actually knew the full story.
I was wrong. Almost everyone I talked to asked how I was feeling, or commented on how great it was that I was still so healthy, etc. Many people commented on how “inspiring” I was, which I will never actually understand. I haven’t really done anything special other than following my doctors’ advice and committing to exercise as a lifestyle habit. My body is just doing what it is programmed to do and, fortunately for me, there are some modifying genes that are somehow moderating how CF expresses itself.
Two years ago, it looked like CF was starting to express itself with a new vengeance, and that perhaps my good fortune was coming to an end. That year began with major back surgery, and ended with a total of four hospitalizations for pneumonia. That was a new personal record for me, and that little voice in my head that had been pretty quiet until then began to speak more loudly and forcibly, “this is not going to end well, Julie.”
This was also the year that Orkambi turned out to be a big bust for me. I tried it for five days, but it was pretty clear that lying in bed with a fever and feeling like I couldn’t breathe was not a good option, even if I did eventually get the potential 3% bump in my FEV1. Feeling like crap and being unable to exercise for who knows how long for that marginally better number made no sense to me.
I also had the background experience of being in the Phase III clinical trial of the Vertex 770/809 combination that lead to the approval of Orkambi. That experience proved to be exasperating. In retrospect, I know that for the first few months I was on a placebo. Then, the day I rolled over into the open-label extension, less than an hour after swallowing the a.m. dose, I experienced shortness of breath and coughing much more than usual. It felt like an asthma attack. Within a couple of weeks, I was in the hospital. Two months later, I was back in the hospital. Then a month later, once more, and I finally dropped out of the trial.
You might ask yourself, why in the world did she try Orkambi? That is a good question, and the answer is that I didn’t actually know for sure at the time that I was on placebo for the first part of the trial (now I do). So, it could have been coincidence that I got so sick with the open-label extension. I decided to try Orkambi, at half dose, just to see what would happen.
I know that a lot of people experienced this shortness of breath and general feeling of malaise/fever but fought through it and benefitted from the drug in the end. But I knew my history with it and didn’t want a repeat of the previous year. I was also fortunate enough to have pretty stable health at a relatively good level of lung function without a CFTR modulator, so I could afford to wait for the next combination.
Enter the magic pills. I started Symdeko as soon as it was approved, and fortunately experienced absolutely no side effects! I’ve now been taking it for 20 months at the time I type this, and, in that period, have only once needed IV antibiotics for pneumonia. I gave Symdeko my pet name, Magic, a few months into taking it because, although my lung function hasn’t really changed, a few things have.
The most obvious thing is the lack of exacerbations, or at least the markedly diminished frequency of them. But I also initially gained about five pounds, and had a new energy that seemed to appear during workouts. Instead of one exercise session doing me in for the day, it became fairly routine for me to both lift weights or do Pilates and also do cardio in the same day. What limits me in the gym now is not a lack of lung function or energy, but instead the various orthopedic issues that go along with getting old. And that’s nothing to complain about.
So, what is next on the CFTR modulator front? I’m sure that nobody reading this is unaware of the fact that the triple combination therapy from Vertex is now in the hands of the FDA. When I read the results of the Phase III clinical trials of the triple, I could hardly believe my eyes. In each study, treatment with the VX-445 triple combination regimen resulted in statistically significant improvements in the primary endpoint of absolute change in percent predicted forced expiratory volume (FEV1) and all key secondary endpoints. In the study involving people with one copy of F508del and one minimal function mutation, 24 weeks of the triple combination of Elexacaftor (VX 445), Tezacaftor and Ivacaftor, resulted in a mean absolute improvement in FEV1 in one second FEV1 of 14.3 percentage points from baseline (p<0.0001) and a 63% reduction in the annualized rate of pulmonary exacerbations (p<0.0001). This is amazing and shows that this combination is a gamechanger.
Additionally, the second study for those homozygous for F508del, who were already on Symdeko (Ivacaftor + Tezacaftor), the addition of the third corrector (VX445/Elexacaftor) showed a mean absolute improvement in FEV1 of 10.0 percentage points from baseline at week four. Another game changer, similar in magnitude to the experience of those people with G551D who began Kalydeco in 2012. There are 107 people who were in this treatment arm, all of whom chose to enter the 96-week open-label extension. I’m sure many of them are chomping at the bit to talk about their experience with the triple combination therapy, but they can’t talk about it until the FDA makes their approval/disapproval decision.
These numbers are staggering. What also blew my mind was the decrease in sweat chloride in both groups of over 40 mmol/L, again in the same league as what was seen in the G551D patients in the VX 770 trials. Other secondary endpoints, including exacerbation rate, change in CFQ-R Respiratory Domain, and BMI were similarly positive. Bottom line: the triple combination works, and it works as well or better than Ivacaftor works in those with at least one copy of G551D.
I am fairly confident that the FDA will approve this combination, and with that, there will be a highly effective treatment for 90% of people with CF. But that leaves 10% (those with STOP mutations who don’t produce enough CFTR protein on which modulators can work) wondering when will it be their turn?
There is good news on this front as well, although the timeline is not nearly as far along. For people with STOP mutations, there are a few options being explored. The most exciting to me are the inhaled mRNA therapies. A prime example is the Translate Bio drug candidate, MRT 5005. As a quick cell biology review: messenger RNA (mRNA) is the backbone from which proteins are built. In the nucleus of the cell, the CFTR gene is transcribed into mRNA, which travels to the cytoplasm, where ribosomes use it to build the protein CFTR. In people who have a STOP mutation in the CFTR gene, useful mRNA is not made, so the CFTR protein is not made. But if inhaled “normal” or “wildtype” mRNA can make it to the appropriate cells, then normal CFTR can be made, and this not only bypasses the problem with STOP mutations, but also bypasses the need for CFTR modulators. In other words, if the inhaled mRNA therapies being developed are successful, CF is cured (at least in the lung, where the inhaled drug is targeted). A more systemic delivery of mRNA, or gene therapy, would be needed for a global CF cure, one that would target not just the lungs, but the GI tract, pancreas, liver, sweat glands, etc. But since the lung is generally the place where life-threatening damage occurs, it makes sense to develop therapies directed there as expediently as possible.
Translate Bio recently shared the news of a Phase I/II clinical trial which was intriguing and fairly exciting (https://investors.translate.bio/news-releases/news-release-details/translate-bio-announces-interim-results-phase-12-clinical-trial). It was a very small study, so the results must be taken with caution, but they did show encouraging interim results suggesting that the mRNA from a single inhaled dose was safe and effectively delivered to the target cells. Indeed, after a single dose, there were significantly positive changes in ppFEV1 at day eight follow up. It’s too soon to dance in the street, but this would be a “mutation agnostic” treatment. In other words, it would work for everyone with CF.
So, there is hope. Of course, gene therapy would be the ultimate cure, and this is also actively being pursued in the pre-clinical pipeline. Recently, an Italian team successfully used a gene-editing technique, CRISPR-Cas9, to permanently correct two of the mutations in the CFTR gene that cause CF. Instead of animal models, organoids derived from patients’ own cells were used in the study. This makes the findings even more pertinent, as animal model findings do not always transfer to patients. CRISPR is widely considered to be one of the most promising approaches to correct the mutations in the CFTR gene. In this study. The CRISPR gene-editing tool was able to restore the levels of normal CFTR mRNA molecules by more than 60%.
If you have to live with CF, this is a pretty exciting time to do it. I long for the day when there is a Magic pill for everyone. But as the CFF says in its mission statement, “[w]e will not rest until we find a cure for all people with cystic fibrosis.” Indeed, that will be magical. s
Julie Desch is 59 years old and lives in San Rafael, CA. She enjoys meditation, reading, writing, exercise of every variety, and hanging out with her partner, two boys, and three dogs. She can be reached at Juliedesch@gmail.com.
NOURISHED BITES:
Modulator Snacks
I’m so excited, and thankful, to be contributing once again to CF Roundtable, this time with a column dedicated to food and nutrition — something I am so passionate about!
I hope to inspire you all to get in your kitchen and whip up some delicious and nutritious meals. Healthy eating is one of the best things you can do to boost your physical well-being and fitness.
For this first issue back, I wanted to stick with the focus topic and share some higher-fat snack ideas that can be eaten when it’s time to take whatever modulator you’ve been prescribed. I know that some people can struggle with variety, so I hope this list helps a bit.
Modulator Snack Ideas:
• Prosciutto slices wrapped around mozzarella cheese sticks.
• Guacamole with chips or veggies.
• Hummus with crackers, naan, or veggies.
• Dates stuffed with peanut butter, topped with salt flakes and cinnamon.
• Apple or banana with coconut butter or peanut butter.
• Whole milk yogurt with peanut butter drizzle, granola, and coconut flakes.
• Half an avocado with Everything But the Bagel seasoning (found at Trader Joe’s, or you can make your own).
• Avocado toast: toast, half an avocado, salt, red pepper flakes, and Everything But the Bagel seasoning.
• Smoked salmon and cream cheese on toast or a bagel.
• Deli roll-ups, such as cheese wrap, salami, ham, pepperoncini.
• Mini charcuterie plate with crackers, cheese, salami.
• Chavocado pudding: blend half an avocado, 1–2 tbsp cocoa powder, 1 tbsp maple syrup, 1 tsp vanilla, pinch of salt.
• Mini cream cheese and veggie frittatas: mix 4 eggs, ½ cup whole milk, ½ tsp salt, ½ tsp pepper, 1 oz goat cheese, 4 chopped cherry tomatoes. Fill 6 lined muffin tins with mixture. Bake at 375º for about 20 minutes or until cooked through.
• A couple of hard-boiled eggs with prosciutto.
• Deviled eggs.
• Chia seed pudding: one can full-fat coconut milk, ¼ cup chia seeds, 1 tbsp raw honey, ½ tsp vanilla, pinch of Himalayan sea salt. Blend everything but the chia seeds until smooth. Stir in the chia seeds. Pour into one large, or a few smaller, containers. Refrigerate until thickened. Serve topped with your favorite berries.
• Coconut cashew energy balls: 1 cup cashews, ½ cup raw cashew butter, 2 tbsp raw coconut butter, 2 dates (remove the seeds), ½ tsp flaky sea salt, 2 tsp almond milk. Blend in food processor until smooth, scraping the sides as needed. Roll into 1-inch balls then roll in shredded coconut. Store in fridge for up to one week.
• Mixed nuts and coconut flakes.
• Smoothie: 1 cup whole milk, 1 banana, 2 tbsp peanut butter, 1 tbsp cocoa powder or chocolate syrup.
• A couple of dark chocolate squares topped with peanut butter and salt flakes.
• A couple spoonfuls of coconut butter. This is a favorite of mine!
This list is certainly not exhaustive, but hopefully it inspires you to try some new things and switch it up every once in a while. Some of these things can even be made ahead to keep on hand, such as the mini frittatas, chavocado pudding, cashew coconut energy bites, and chia pudding. s
Aimee Lecointre is 34 years old and was diagnosed with CF at two weeks old. She is a certified nutritional therapy practitioner, primal health coach, and registered yoga teacher. She lives in Salt Lake City with her husband, Laurent. She’s passionate about food and nutrition but also loves true crime, tacos, and a good cup of coffee. You can email her at aimee@thenourishedbreath.com with requests for future columns and check out her website at www.thenourishedbreath.com to learn more.
voices from the roundtable: IVF Success Leads To Three Kids
Growing up all I ever wanted was to get married and have kids. Unfortunately, I barely dated during my teenage years and my 20s because I believed that my early death was likely, and I didn’t want to bring a woman into my life to ultimately die on her. After years of therapy, I overcame my dating obstacles and realized that if I’m honest with a woman and disclose my CF early, then the choice of dating me would be hers.
At 30 I met my wife, at 31 we married, and at 32 we were pregnant. My journey to fatherhood began while my future wife and I were dating. We both wanted children and it was a priority for each of us. I didn’t know if I could have children naturally since roughly 95% of men with CF are sterile.
After marriage I went to a urologist to determine if I could have children. I learned that most CF men are either born without a vas deferens (the tube that takes the sperm from the testicles to the urethra) or the tube is clogged with mucus and nonfunctional. Ultimately, either scenario means that sperm can’t travel through the tube. The doctor felt my testicles and immediately determined that I was born without the vas deferens. After some discussion, my wife and I proceeded with in vitro fertilization (IVF).
The IVF process involved retrieving sperm from me and eggs from my wife in order for fertilization to take place in a lab. The embryo would be implanted in my wife’s uterus and, it is hoped, would develop into a baby. My wife went on a protocol of medicine and shots to stimulate her body to produce more eggs for eventual retrieval. These medications were very powerful and had major effects on her hormones and personality. To retrieve my sperm, the doctor cut into my testicles with only local anesthetic for the pain. Halfway through the procedure, the doctor left the room without any communication, leaving only the nurse and me, which caused me a great deal of stress. I later learned that he went to a high-powered microscope to determine if I had viable sperm and if they were motile (both yes!). He closed me back up and I was extremely sore and black and blue for about two weeks. My wife eventually had her eggs removed and our first IVF attempt did not work; however, the second attempt produced my twin boys, who are now 16 years old.
We did our IVF procedures in 2002 and, from a financial perspective, we were lucky because, in 2002, my wife worked for Disney and their insurance covered IVF. Their coverage, however, was limited, and we still had about $4,000 in out-of-pocket expenses. In 2003, Disney eliminated their IVF coverage and, as a result, our IVF doctor did a different protocol between attempts 1 and 2 and we think that was the difference in our success.
My twins were born before my lung transplant, but my third child was born post-transplant via IVF. Post-transplant I’m on 25 different medications and we couldn’t risk using fresh sperm and having a child with health problems. We used frozen embryos from our first IVF process and my wife needed to go back on a minimalized course of medications to try and get pregnant, but our first attempt failed. Our second attempt required my wife going on a full course of medications to retrieve live eggs and we utilized my frozen sperm to create an embryo, which was successful when implanted. My third son is now 12.
Although we didn’t have IVF insurance coverage for my third son, our regular insurance was more generous with medications, doctor visits, and procedures. Our total cost was approximately $8,000. The emotional component of my experience was extensive including fear, anxiety, frustration, hopelessness, worry, pain, joy, awe, love, happiness, elation, accomplishment, satisfaction, wonder, amazement, delight, and enjoyment. I think it’s important to recognize that the process of IVF is long, emotional, and stressful because there are no guarantees of success. Most patients visit IVF doctors because of female-related reproductive issues so knowing it’s a male issue is an advantage and we were successful on our second attempt both times.
My IVF experience produced three amazing kids, whom I love to the moon and back and to infinity and beyond. s
Jeff was diagnosed with CF at age one and is now 50. He is 15 years post-transplant, a dad to three boys, and lives in Los Angeles. He can be reached by email at jeffr530@yahoo.com.
In the Spotlight:
With Lizzie And Bailey Gorman
Sometimes it takes two! For this column we feature two sisters who are two years apart and have CF and CFRD in common, but also have their own individual lives. They have had the shared experience of bilateral lung transplants (two years apart), but both had completely different experiences. They discuss the cruel reality of infection control preventing them from fully being able to physically be there for each other. Even though one of their physicians could not tell them apart, I think you, as the reader, will. With delight, enjoy meeting these two stars. Spotlight, please.
Your ages: Lizzie (LG): 29 or something…but actually, 29.
Bailey (BG): 27 (but apparently, I look 20).
Where do you live? LG: Dallas, TX.
BG: Jacksonville, FL, but home will always be Texas.
Where do you work?
LG: Prior to my transplant, I worked for Citibank in the problem management department doing a lot of data and quality analysis. I was told about one month before my surgery that I would be laid off toward the end of October of 2017. I was living off the disability provided by my employer until September, 2018, when I started thinking about what I wanted to do with my life. It was hard to go back into the industry I was in, because office cubicles don’t provide the best germ containment. I started doing some soul searching and landed on being a barista at Starbucks. I know that’s a totally different direction than I was going in life, but I actually really enjoy it. I chose this job because it was a drive-through and walk-up location only; meaning I would be minimizing my encounters with contagious germs. I always wash my hands in the middle of shifts and take careful precautions if one of my partners is feeling unwell. This is definitely a random stop on my journey, but it was one that felt necessary for the time being.
BG: I work as an electrical engineer for NAVAIR. I love my job and it keeps me busy. Since day one they have been very understanding of my health. Luckily, I have a compressed work schedule so I get every other Friday off, which allows me to not take off for most of my appointments.
When did you have your transplant?
LG: My transplant took place on August 29, 2017. I was listed on April 1, 2017…but it was an awfully real April Fools’ joke, if you ask me.
BG: Got the call on August 31, 2015, at 11:55 p.m. The transplant took place on September 1, 2015.
How important was it to go through transplant with each other?
LG: The crazy thing is that when Bailey had her transplant, I went to the hospital and saw her immediately afterwards. We were then told that we weren’t allowed to see each other for three months, after the prednisone had completely tapered. Even though her lungs no longer had cystic fibrosis, the post-transplant immunosuppressant drugs would leave her susceptible to the bacteria that I still had in mine.
BG: It was hard to be supported by Lizzie. We went from hanging out almost every day to not being able to hang out at all. We still talked, but she didn’t fully understand how I was feeling. I hope I supported Lizzie during her transplant. I was back at school full time when she received her call. I came the next day while the gas crisis from the hurricane was happening. I tried to help her out as much as I could. I remember her telling me that her lips were very chapped and I kept having to apply lip balm for her.
Who else is in your support system?
LG: Our immediate family has always just been a huge support to each other. My parents would easily drop anything to be there if we needed them. There have definitely been some hard calls to make; e,g., when Bailey was listed for transplant, we were supposed to be flying out to Boston, but she got sick and the doctors deemed her too sick to fly. When that happened, we were all disappointed and had to lean on each other. We don’t always see each other, but we always know that we will be there for each other.
BG: I lived with my aunt and uncle before and after my transplant. My aunt, being a wound nurse, really helped me the first couple of weeks, especially with my wounds. My grandfather would come eat lunch with me once a week after my transplant. My parents are also a big support. They had to accept that they couldn’t be in Dallas all the time, but came up as often as they could.
How did your transplant experiences differ?
LG: Well, from my perspective, I saw Bailey go through the transplant (in 2015) and she struggled very much in the beginning, whereas my experience was quite different. I had my breathing tube out in almost half a day; my time in ICU was limited to about three days; and then I was home and out of the hospital within a week. The time flew by, and I was seeing this huge improvement right off the bat. I was even kicked out of physical therapy early! Suddenly, I took this huge dip and it was so scary. I had to have sinus surgery; I had to have my gallbladder removed; another sinus surgery; and then, to top it off, two rejection scares. It was like, I did all of this for this?! I just wanted it to be easy, but with transplants there is no easy…and I had to learn to accept that. Now my lung functions have stayed around 90%, and it’s been two years!
BG: I would say that we had very different experiences. I was in ICU for seven days after my transplant and then seven days in a normal room. I had to go into surgery a day after my transplant because of bleeding due to my old lungs being stuck to my tissue. I wasn’t in a whole lot of pain after I was moved into the normal room, so I decreased my pain meds. I had to go home with a chest tube to get all the fluid out of my lungs. I did get steroid-induced diabetes due to the high dosage of prednisone. I cut out sugar, sodas, and a lot of carbs to help with my diabetes. After a year passed, my diabetes didn’t cause any issues. I monitor my blood sugars now, but I haven’t had to give myself insulin in over two years. My transplant hurdles were at the very beginning of my transplant. As for similarities, we had the same transplant surgeon and doctor and we both graduated out of physical therapy early.
How long have you been this close and what brought you together?
LG: Well, I remember being younger and spending our hospital visits together. We would decorate our joined room together and make it a jungle or whatever we wanted. Our mom, in particular, is a very creative person and wanted us to feel less “sick,” so she tried to make our hospital visits fun. The reality of the situation is that we had this disease in common, but we were also so very different. We have completely different personalities, so we butted heads a lot while we were younger. I don’t think that either of us appreciated the other until I left for college in Missouri and we weren’t able to see each other as often. That’s when we became the closest and actually ended up being roommates for one year of college.
BG: We weren’t really that close until Lizzie moved away to college. Then I realized how much I relied on her. We have always been there for each other but siblings are siblings. We annoyed each other and we wanted our own lives as kids.
How hard was it not to see each other post-transplant?
LG: When I had my transplant, she was no longer living in Dallas. She was back to being a full-time student, so it wasn’t easy for her to get back and see me. However, I do remember her coming down to visit as soon as she could and I was so grateful for her to be there. I kept asking her questions like “is this normal,” and she would legitimately just tell me “I don’t know, ask the nurse!”
BG: Not being able to see each other after my transplant was really hard. We went from hanging out most days to not hanging out at all. When we did hang out, we had to be extra careful. We both wore masks and washed our hands often.
How is managing diabetes post-transplant?
LG: Diabetes — it’s honestly the only bad thing to come post-transplant so far. I think the thing about it is that it is still just so unpredictable to me. And it may always be. Post-transplant and cystic-fibrosis-related diabetics are bound to be more unpredictable because of the medications we take post-transplant and the hormones our bodies experience. I am still learning how to be a diabetic for sure, but the continuous glucose monitor has helped immensely. I don’t know how I did diabetes before having it. I use the Dexcom G6 CGM and the Tandem T:2 Pump for my insulin. They communicate to each other (somewhat) to prevent me from having lows and, it is to be hoped, with future updates we will see it auto-dose for high sugars. It has made the disease more manageable, despite constant beeping at me for doing something wrong, or the battery dying, or I’m low on insulin, or my sugars aren’t in range.
BG: I had never had any problems with my diabetes before my transplant. After my transplant, I had steroidinduced diabetes. My blood sugars were crazy. I decided pretty fast after getting home that I needed to cut out most sugars and carbs from my diet. The problem with completely cutting out sugars resulted in extremely low blood sugars in the morning. I was on oral medication and then I had an insulin pen for meals. It was hard to adjust to having to prick myself five-plus times a day and not having sugar whenever I wanted.
What are your hobbies and/or passions?
LG: I guess a lot of people have hobbies, but I am one of those more “go with the flow” or “try anything at least once” types. I love spending time with my friends doing anything and everything, especially if it includes bringing my two dogs! I enjoy spending time at the movies, going rock climbing with my bestie, driving around with the radio far too loud, and singing along whether it’s by myself or with friends. I just enjoy the small things and appreciate every opportunity that is given to me.
BG: I acquired a passion for reading while being in the hospital. If I am not reading, I am either hanging out with friends, watching tv, singing, and trying new restaurants. I am trying to learn Spanish, but have not gotten very far with that since I am usually tired when I get home from work.
What or who inspires you??
LG: I don’t think I have befriended a single person who didn’t inspire me. Friendships come and go but you always learn something from them and that’s inspiration, right?
BG: My friends and family are mostly who inspire me. But I would also say I find inspiration in the little things in life.
Are you on social media?
LG: I am on social media under @SaltyLizzie on my main Instagram. My newly started, food-themed account is @IvaGottaEat. My blog is QuirkyCyster.Wordpress.com
BG: I am on Facebook, Instagram, and Snapchat. I also blogged after my transplant. I would say that being on social media has been helpful with staying connected with friends and letting them know how I am doing. I am not against following other people with CF, but I wouldn’t say that I follow many people with CF. Most of the people I follow have had a transplant.
What did your non-CF peers think of you two?
LG: Growing up was difficult because, as a kid, you don’t fully understand that you’re sick, or that you’re going through something different than the other kids around you. We spent a lot of time in the hospital and would have to unenroll at our elementary school. Kids just didn’t understand why we would disappear from school for periods of time. Kids can be very mean about it.
BG: I think people with CF learn how to grow up faster. I wasn’t always open about having cystic fibrosis. My parents always let my teachers know. I waited to tell my friends for a while. It was hard to explain why I was sick or bailing on them. I know my friends didn’t really understand. Middle school was probably the hardest time, but I also met my best friends in middle school and we’re in touch today. I also had people who were just mean and said some pretty horrible things to me about my disease. I definitely have found that people don’t always stick around but when you find someone who will, he/she is a keeper.
What do you do for exercise?
LG: I try to go to the gym five times a week, but there are definitely periods of my life when that is a complete pipe dream. I have followed a few exercise regimens and I really enjoy the sisterhood of Tone it Up. I have a bicycle and ride it when it’s between about 65 degrees and 80. Post-transplant, I am so paranoid about the sun that I’ve severely limited my outdoor activity. Often, I work out at home because of the germs inside the gym. They are so dirty!
BG: Lately I haven’t been able to work out as much as I would like. I have been walking my roommates’ dog two miles about three times a week. When I go to the gym, I try to switch up my routine so that I don’t get too bored. I try to do either about 20–30 minutes of cardio or 20–30 minutes of weights. My other options are yoga or Zumba.
What’s your social life and/or dating life post-transplant?
LG: Dating is just so hard in general. Having to explain that you have CF and had a double lung transplant, with a life expectancy of roughly 10 years on average, makes it even harder to get someone to buy into that. Again, it helps weed out the people who won’t be there for you, and I would rather find out before I am invested, but it can be difficult. I’m honestly fine and happy by myself with my two dogs, but it would be nice to have someone to depend on who will be there when times get tough.
BG: I would like to say that I have a social life. I hang out with my friends at the beach, see movies, have game nights, and go out to the bars. It is hard to go out at night because I don’t always want to be the one taking care of my tipsy friends. I am used to being the designated driver, but I don’t like staying out as late as my friends.
I don’t have a dating life mainly because I like being on my own. Also, I don’t really want to be on dating apps, and that seems like the only way people meet nowadays.
Do you have a motto you live by?
LG: “I’m hungry and I need caffeine and/or a nap” is the closest thing I have to a motto/mantra.
BG: Here are probably my top four:
1. “Enjoy the little things…” — Robert Brault
2. “Life is not measured by the number of breaths we take, but by the moments that take our breath away.” — Attributed to Hitch (Will Smith), among others
3. “Live everyday as if it were your last.” — Muhammad Ali
4. Try to find the positive side in everything. s
Andrea Eisenman is 54 and has CF. She is a Director of USACFA and is both the Webmaster and Executive Editor of CF Roundtable. Her contact information is on page 2. Jeanie Hanley is 57 and is a physician who has CF. She is a Director and the President of USACFA. Her contact information is on page 2. If you would like to be interviewed for “In The Spotlight,” please contact either Andrea or Jeanie.
CF: The MIND Game
Pitfalls On The CF Road
Of Life: Substance Abuse
And Dependency
Nineteen years ago, when I was just completing my MPA at Syracuse University, I had a clinic appointment that didn’t go particularly well. Most of you have probably experienced this: my PFTs were down more than 10%, which meant it was time to once again go to the hospital. I don’t know which I hated more at the time: the thought of lying around the hospital yearning to be on campus or taking antibiotics that wrecked my insides and left me dehydrated, nauseated, and drained. However, that time it was partly my fault because, prior to that visit, I had been running myself ragged pushing to complete my MPA in just 18 months while juggling a graduate assistantship (teaching stats and researching states’ Medicaid Managed Care Plans) and running Priority Fitness, the business I co-owned with my brother-in-law. During that clinic visit, in a moment of frank conversation while the doctor was reviewing my disappointing test results, he made a brief, but poignant, comment that has haunted me ever since. My doctor, CF specialist, primary care provider, and the principal investigator for my research project, the one who knew me better than anyone at the time said, “I’m afraid your biggest problem isn’t that you have CF but that you haven’t reconciled the fact that you have a Type A personality with a Type B disease.” For me, one of the key psychological consequences of CF has been the overriding drive to accomplish all my big, hairy, audacious goals as fast as I can for fear that tomorrow, or shortly thereafter, they will be out of reach. In other words, CF mentally manifests as a deep-seated, unquenchable desire to suck the marrow out of each and every day, but that is at odds with the physical presentation of the disease, which seeks to drain the life out of me each and every day.
Fortunately, I did complete my MPA in 18 months and was hospitalized soon thereafter. Many people struggle with finding balance between living every day like it’s their last and lasting long enough to live more days. Consequently, many of us ebb and flow between seasons of remarkable growth and productivity followed by seasons of dormancy and depression as the goals, ambitions, and dreams we so dearly held slip away. With the exception of rhythmic disruptions from medical breakthroughs and transplants, flow times inevitably grow shorter and ebb times grow longer as we get older. Consequently, when most of us experience an exacerbation, have to go inpatient, or end up on IVs, we partly blame ourselves for not doing our treatments enough, for traveling too much, for burning the candle at both ends, or for just pushing ourselves too hard to accomplish that next fantastical goal. For the most part, we’ve all learned to find balance as these two powerful forces push and pull us. We manage to stay on the road of life on a relatively even keel emotionally and try to keep our minds nimble so that we can adjust to the changing seasons.
However, we all live with different levels of risk we’re comfortable with while navigating that road without crashing, losing our way, or veering off. I knew a CF patient who ran a marathon with less than 20% lung function; another who traveled the country giving motivational speeches while also fighting an infection for which there was no known treatment at the time; and still another who became a volunteer firefighter by hiding his disease. In fact, the level of risk I was comfortable with at one time in my life may be well beyond the comfort level for many who are reading this. When I was 28, I deadlifted 945 pounds as a competitive powerlifter. This put so much strain on my body that both my nostrils bled profusely and the blood vessels in my eyes burst while I was completing the lift in preparation for the upcoming national championship a month later. Unfortunately, I did not make it to the national championship that year or any other, even though I had won the Amateur Athletic Union New York State Powerlifting Championship six months prior. The night I hit that personal record, I woke with the worst episode of hemoptysis I’d ever experienced, followed by months in and out of the hospital with a superinfection.
Aside from the calculated risks many of us take by skipping treatments or delaying hospitalizations so that we can pursue our goals while this life-sucking disease salivates at our heels, most patients don’t become overtly self destructive. Nearly every CF adult I’ve known has managed the problem with good results by developing a rich mental/virtual life when their active life wanes, thus enabling them to temporarily escape their CF lives in varying degrees. Escapist coping techniques widely used in the CF community range from reading books, binging shows on Netflix or other streaming services, mastering video games that enable us to become bigger than life, and/or writing, as I do. However, for some, the mental drive to succeed and prove oneself worthy to the world is too much and cannot coexist psychically with the practical reality of a disease that is itself bigger than life and seeks to devour all that we desire and hold dear.
Other patients I’ve known have not adjusted so well to the disease and the changing seasons, despite the abundance of first-world escapist distractions available. In some cases. these patients have abused or become overly dependent on alcohol and/or other drugs as a coping mechanism. Unfortunately, the prevalence of this problem in the CF community is not well known. I cannot emphasize enough the importance of a better understanding of drug and alcohol abuse from a diagnostic, epidemiological, and treatment standpoint. Nevertheless, based on what we know — namely that the prevalence of depression and anxiety disorders in the CF population is three- to four-fold that of the non-CF population — it is reasonable to believe that alcoholism and/or drug addiction, which shares high comorbidity rates with these disorders, is probably more prevalent in the CF population than we think.
I’m sure there are many reasons why we don’t hear more about this problem in the CF population, but a few come instantly to mind: the primary psychological symptom of addiction is denial of the behavior itself; the physical manifestations of CF are so demanding that the mental manifestations often take a back seat; and finally, what I’ve coined as CF denial, which I observed more than 20 years ago when I first began studying psychological adjustment in CF patients. CF denial is best described as a patient’s masking of his or her problems during clinic visits by normalizing their symptoms, underreporting their medical non-adherence, and/or overstating their emotional stability. This behavior has been observed across the entire population but was particularly pernicious among adults who developed sophisticated and effective masking devices over time, and for whom there were often few or no collateral contacts that could provide a glimpse behind the mask.
To be sure, for those CF patients who turn to alcohol and/or drugs to escape depression, anxiety and/or the harsh realities of life with CF, the world is a particularly dark place. Not only are they forced to fight a chronic, terminal illness, but they’re seemingly inescapably trapped in the cycle of addiction, living from one binge to the next. If you’ve read the recent Summer 2019 issue of CF Roundtable, then you’re familiar with my story and the challenges I faced while getting sober when I was 18 years old. Getting sober can be every bit as challenging as fighting CF itself, particularly since long-term sobriety rates hover in the low single digits. CF patients who cannot successfully break the cycle of alcohol and/or drug use as a coping mechanism often experience health declines and early morbidity due to poor medical adherence, poor diet, lack of exercise, as well as mental and psychological decompensation.
I’m convinced that all CF patients know, in some varying degree, what it’s like to have a Type A personality and live with the seemingly insatiable drive to succeed and prove ourselves while also living our lives ensconced in the hourglass of CF and a Type B disease. However, some of us cannot accept the realities of our lives with CF without turning to alcohol and/or drugs to cope. If you or someone you know is facing this dark prospect, know that there is hope and share this article with them! The fact is that many CF patients have faced and overcome alcoholism and/or addiction! However, you have to take the first step by reaching out to someone on your CF team to get the help needed to break the cycle, begin the healing, and find your hope!! s
Mark is 50 and has CF. He lives in Albany, NY, with his wife, MaryGrace, and stepson, Sean. He holds a Master’s in Psychology from Marywood University and a Master’s in Public Administration from Maxwell School of Citizenship and Public Affairs from Syracuse University. He was the Director of Rehabilitation, Vocation, and Family programs at Syracuse Behavioral Health. He is one of the first researchers in the country to study psychological functioning in adults with CF, including a presentation at the 1999 American Thoracic Society International Conference1. His contact information is on page 2.
​
1“Psychological Functioning as Measured by the Minnesota Multiphasic Personality Inventory in Adults with Cystic Fibrosis,” American Journal of Respiratory and Critical Care Medicine, Volume 1, Number 3, March 1999.
​
On The Speakers Bureau
The U.S. Adult Cystic Fibrosis Association (USACFA) is pleased to announce the relaunch of our Speakers Bureau as part of our ongoing mission to serve the population of adults with CF through advocacy and various programs providing support. Historically, individuals with CF and their families spearheaded advocacy within the CF community through community fundraisers and education days, among other things. However, the evolution of our knowledge and understanding of cystic fibrosis, coupled with more adults with CF living longer lives, mandates change in how we advocate, communicate, and connect as a community.
In advocacy, we often wear our hearts on our sleeves. Advocates — and our speakers in Speakers Bureau — are driven by the need to have raw conversations about living with a disease that affects every facet of life. Our speakers are available to speak on a wide range of different topics: balancing school, work, and CF; mental health; and sexuality and CF.
Adults with CF have learned how to speak about life with this disease in many different forms and to many different audiences. As treatments continue to improve, people with CF are living longer, fuller lives and are able to work more and engage with others on a deeper level. It’s common in the CF community to remark on how our lives are not defined by CF, but there’s no doubt that CF empowers us in ways that few can understand. That means our lives are often lived differently: frequent medical appointments teach us how to engage with others as self-advocates; extensive daily treatments, sometimes concurrent with heading off to college, force us to learn responsibility and accountability at a younger age than most; and living with a chronic, terminal disease means we have to confront mortality by the time we hit puberty. Our stories aren’t just stories about the patient experience or life with chronic disease — they are stories about the human condition. With the help of our speakers, your events will feature voices that are experienced not just about life with CF, but about life in all its complexity.
The Bureau hopes to infuse events with the passionate voices of people who are making strides in the community and who have authentic perspectives. Our community is diverse in many ways: from life experience to careers to opinions on current topics. The Bureau offsets the cross-infection concerns and resulting isolation by affording the opportunity to hear the many voices and perspectives in the CF community, thereby making way for deeper conversations, more education, and further dialogue between patients and caregivers.
As a longstanding organization deeply rooted in the community, we’re committed to bridging the communication gap between patients and CF clinic and care teams through an array of speaking opportunities. To this end, USACFA covers the expenses of sending a speaker at your next event, whether that’s a fundraiser, family education day, conference, or any other event that would benefit from having an excited, passionate advocate for the CF community as a whole.
Please reach out to us via e-mail at speakers-bureau@usacfa.org if you’re interested in booking a speaker. Alternatively, you can book online at https://www.cfroundtable.com/speakers-bureau, where you can see every speaker’s experience and preferred topics. We look forward to speaking at your next event!
​
​