In  2007, I was five years post-lung transplant. At 26, I was tired of  almost everything. My anxiety from waiting for the surgery and not  knowing if it would ever happen in addition to the anxiety from the  constant ticking of the mortality clock afterwards had put me in a place  of extreme discomfort and uncertainty. All of us with CF are born with  it, but I hadn’t really gotten sick because of CF until I was 17. It  happened fast. Within a little over a year, I was on the transplant  list. From ages 17 to 25, I felt like I was a living statue—always  walking around in a fog, only thinking about my health. On top of this,  my survivor’s guilt after the transplant was crippling. Getting away  from everything and everyone I knew didn’t seem like a crazy idea to me  at all. In fact, it felt right as rain for a million different reasons.  Everyone who has received a transplant does so in order to survive—to  continue this beautiful life—but I wasn’t quite sure what I was  surviving for. Traveling the world was an idea that, for years, I had  never even let myself entertain and now, without any preparation or even  much thought, I impulsively decided it was time to stop surviving and  start living. I had been abroad many times as a child and in my teens,  but never on my own and never after I had gotten sick. When I was 17 and  got seriously sick for the first time, I happened to be in Ireland.  Irish hospitals became familiar to me. It was a wonderful experience  with incredibly caring, smart, effective, and efficient doctors and  nurses. With this in mind, and, considering the fact that I was the  healthiest I had ever been, traveling around at 26 did not seem risky at  all. And so, over the next decade, I hopped from country to country,  living in different places for a year or three at a time, where,  ultimately, I did become familiar with several hospitals and health care  systems in many countries. Some were fine. Some were bad—very bad.  Others were good, maybe even great.

Europe:

In 2007,  I moved to Madrid, Spain. It is a beautiful city with fantastic food,  gorgeous architecture, great transportation, and hospitals that, at  least for me, wanted nothing to do with my American health problems.

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The  first time I got sick there, I had a small fever and some shortness of  breath. I wasn’t all that concerned because it was flu season and I  thought maybe it just hit me a little harder than others. Of course, as  we all know, a fever and breathing issues should always be checked out,  regardless of my intuition. At home, I had been seeing my transplant  team at Penn in Philadelphia, so being in contact with them was  critical. My nurse practitioner and transplant doctor tried sending in a  prescription for me, but finding a pharmacy in Madrid that would take  it was impossible. There are pharmacies all over Europe—they are mostly  all marked with a large neon green cross sign out front. I had a  prescription from my team that I was able to fax to a pharmacy (it was  20 years ago) but that proved to be completely fruitless. First, it was  in English. That wasn’t a huge problem because translating medications  from English to Spanish is easy; however, none of the pharmacists knew  who my doctor was, where he was, or if the prescription was even real.  Most pharmacists seemed eager to help but unwilling to give me any  medicine. On top of that, I could never get a fax number to the actual  pharmacy or even have my team call or email the pharmacy.

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Ultimately,  I had to go to a hospital in Madrid. I walked in, walked up to  reception, and tried my best to explain my situation. For example, CF in  Spanish is fibrosis quística—a fairly simple translation. I was seen by  a doctor who spoke English (something I came to realize most doctors in  other countries do), however, things quickly took a turn for the worse.  He listened to me for about three minutes, then told me to, “Go back to  America if you want help.” I was shocked. He wasn’t saying this to tell  me that I was so sick I needed to be home, he was saying that his  hospital would not help me because I was American, plain and simple.

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Defeated,  I tried another approach. I found a private pulmonologist with the help  of a friend. This was the ticket. She was lovely and also shocked at  her fellow countrymen. We spoke for a bit and she quickly gave me every  prescription I needed. I continued to see her about once every couple of  months to listen to me and help me with any issues I needed.

Obviously,  Europe is a big place. I’ve been to a few countries, visited some, and  lived in others, but whenever I found the health care system too much  and the hospitals unwilling to care for me, a private practicing doctor  is what I would recommend. And, as I said, I’ve found that most  well-regarded doctors speak English if you are concerned about that.

Latin America:

Around  2010, I moved to Latin America. I lived in Campinas, Brazil because my  girlfriend was Brazilian. That was an immediate advantage in terms of  health care. Like many countries in Latin America, the bigger cities  will always have the best hospitals, best doctors, and best chances of  finding someone with the knowledge and experience a CF and transplant  patient may need. To me, Campinas resembled a “Brazilian Boston.” It is a  college city with lots of young people and lots of English-speaking  professionals. My health issues there were never serious, but enough to  need to see someone to get an opinion and medication. At this point, I  was still technically a patient at Penn and had my same team, although  they were unable to help me much, with the exception of advice and  questions to ask any doctor I may see in Brazil.

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A couple  of things to know: there are clinics, private practices, and hospitals  in every country in Latin America. However, someone with CF or someone  post-transplant should try and avoid clinics if you can. They are not  like urgent care facilities in America. The upside of a clinic is that  it’s usually free; however, the downside is that there are always a lot  of very sick people and, in my experience, it’s almost never not  completely packed. I went once for myself and was unable to really  explain my situation, even with the help of Brazilian friends. Plus, the  doctors at clinics are swamped with patients, so the ability to get  great care is just unfeasible. As a result, I went to the Hospital da  UniCamp, or the University Hospital of Campinas. There are a number of  very good hospitals in Campinas, however, since I was teaching at the  Univserity of Campinas, it was much more convenient to simply go to the  hospital connected to the university. At the hospital, things picked up.  Nearly every doctor spoke English and were eager to help. I was able to  be seen quickly and was given prescriptions. From there, however,  getting the prescriptions filled was difficult. There are plenty of very  nice pharmacies, but the cost of filling any prescription was  outrageous. For Brazilians, this is not the case, but I am American and  had no Brazilian documents, so the cost was astronomical. If I needed a  month’s supply of prednisone, it would have been around $500.  Antibiotics were cheaper but my transplant medications would have cost  me thousands and thousands of dollars for a small quantity. In terms of  health care, Brazil is very similar to most other countries in Latin  America in which I visited or lived. If you are traveling on vacation  and need a doctor in Latin America, I would stick to big cities and big  hospitals.

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I  eventually worked my way up north. I stopped and lived in Mexico for a  while in the early 2010s. Like Brazil, clinics are not a good option,  and like most countries, there are private and public hospitals  everywhere. If you can, I would try and avoid public hospitals in  Mexico. I was in a private hospital in Mexico City for a more serious  issue than my previous encounters. I had a severe bout of pneumonia and  needed quality care. I had friends in Mexico but was at the hospital  alone. However, I was so sick that I was barely able to explain my  entire condition. What I was able to do is write down all my medications  and hand it to a doctor. From there, they work-shopped and started me  on IV antibiotics, which worked great. If I were to do that again, I  would now have all of my medications, strengths, doses, diagnoses, and  contact numbers in my phone before going abroad. The biggest problem  with Mexican private hospitals is that they are expensive—very  expensive. Before I left the hospital, I was presented with my bill.  Just like in a restaurant, I was expected to pay my bill before leaving.  My American insurance would take weeks or months to process the charges  and cover the bill but for some reason, either no one understood that  or they didn’t care – they wanted their money. My total bill was around  $30,000 for five days of care and some outpatient medications.  Obviously, I did not have that money on me, so things got very tricky,  very quickly. In order to leave, my boss at the time, had to use his own  Social Security ID to essentially place my bill on credit. The issue  here was that, although my insurance would eventually cover the cost, he  did not believe that American insurance companies needed time to  properly evaluate the circumstances before paying the balance. It took  months for my insurance to finally approve payment and, by that time, he  believed I was somehow swindling him out of paying the bill. Of course,  that was not the case, but trying to explain the slow-moving processes  of the American health care system to him fell on deaf ears. In the end,  he was paid in full by my insurance, but it took months to accomplish.

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Ultimately,  if someone with CF these days is traveling, it’s now much easier to  communicate with cell phones. An easy thing to do is have all of your  medical and insurance information on your phone ahead of time. Also,  like almost every country, you will find more and better options in big  cities than small ones, as well as hospitals versus clinics. English  speaking countries such as New Zealand, Ireland, and England will always  be easier if you only speak English. Plus, in my experience, the  hospitals there are all wonderful and you will find great care in any of  them. It is other countries where you most likely will find more  difficulties getting the care you may need.

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These  days, my travel years are long past. With the exception of a few minor  complications, finding necessary health care help outside of the U.S.  can happen, sometimes easily, sometimes not, but a strong lifeline to  your team at home helps enormously. People with CF know themselves and  their bodies, so common sense and listening to yourself is the greatest  tool to take with you. Before you go, I would research reliable  hospitals in the city and country to where you are traveling. Have your  medical information on your phone and, most of all, don’t panic. From my  experience, I have found that most people want to help and will go out  of their way for you. Patience and kindness will get you a long way. For  me, it was trust in my body, knowledge of myself, and contact with my  transplant and CF team that made it simple to know when I needed help  and how urgent it was. I expect for all of us with CF, the same applies,  so use your own intuition wisely and don’t be afraid to search out that  help when it is needed.

Andrew  Corcoran is 43 years old and has CF. He received a double-lung  transplant in 2002. He now lives in South Jersey with his family and  friends. He is a writer. Andrew’s email is acorcoran@usacfa.org.

©2024 Beth Sufian. All Rights Reserved.

People  with cystic fibrosis (CF) who have worked the required amount and who  have left the workforce because of a disability may be eligible for  Social Security Disability Insurance (SSDI) benefits. SSDI has two  parts: (1) a monthly cash benefit and (2) Medicare after a waiting  period. The amount of the monthly cash benefit is determined by the  Social Security Administration (SSA) and it depends on the amount the  individual contributed to the Social Security system through payroll  withholdings or self-employment tax payments over a certain number of  years. People who receive SSDI are eligible for Medicare coverage after a  waiting period. Individuals enrolled in Medicare have several choices  regarding Medicare coverage and the choices determine their type of  coverage and the costs they pay for different types of healthcare  services.

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Medicare  is an extremely complex program and this article will provide general  information to help people with CF understand Medicare and some of the  choices available. This is not a comprehensive discussion of Medicare.  More information is available on the Medicare website, which can be  found at www.medicare.gov.

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Medicare  is complicated because it has several parts established in different  years to deliver different types of health care coverage. Medicare  started in 1965 as a benefit that paid for certain hospital costs.  Congress expanded the types of things Medicare covers by adding new  parts. The Medicare program is administered by Centers for Medicare and  Medicaid Services (CMS), which is part of the U.S. Department of Health  and Human Services.

• Traditional Medicare.

Traditional Medicare has three parts: Part A, Part B, and Part D.

1.  Medicare Part A. Generally, Medicare Part A is hospital insurance that  covers inpatient hospital care, skilled nursing facility care, and  limited aspects of home healthcare. Part A also covers emergency  department visits if the emergency results in a hospital admission.

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2.  Medicare Part B. Medicare Part B helps cover outpatient services such as  services by doctors and other healthcare providers, durable medical  equipment, preventative services, some aspects of home health care and a  very limited number of FDA-approved prescription drugs used with  durable medical equipment. Part B premiums are paid by people enrolled  in Medicare Part B. People who have low monthly income and low assets  may be eligible for help paying their Part B premium.

3.  Medicare Part D. Medicare Part D helps cover the cost of outpatient  prescription drugs, excluding the limited number of drugs that are  already covered by Part B, including some vaccines. Medicare Part D has a  monthly premium that is paid by the person. If a person has low income  and assets the person may be eligible for help paying all or part of the  premium.

• Part C

Medicare Advantage Plans  as an Alternative to Traditional Medicare

Congress  authorized CMS to contract with public or private organizations to  offer health plan options as an alternative to traditional Medicare.  These plans are called Medicare Part C plans—more commonly known as  Medicare Advantage Plans. These health plans provide all Medicare Parts A  and B benefits and most Advantage plans include Part D drug coverage.  Many plans even offer additional benefits beyond those covered by  traditional Medicare, such as dental coverage. Medicare Advantage plans  are coordinated care arrangements that deliver benefits through health  maintenance organizations (HMO), provider-sponsored associations (PSOs),  preferred provider organizations (PPOs) and other care arrangements.  Part C Medicare Advantage plans are offered by Medicare-approved private  companies that must follow Medicare rules.

• Medicare Prescription Drug Benefits and the Inflation Reduction Act of 2022

Because  of the importance of prescription drugs for people with CF, the scope of  Part D coverage and the cost of coverage is a primary concern. Part D  is currently in transition because the Inflation Reduction Act (IRA) of  2022 has redesigned the Part D program. Part D prescription drug plans  are available either as a supplement to traditional Medicare or as part  of a Medicare Advantage plan. Part D requires payment of a monthly  premium, deductibles, co-pays, and cost sharing obligations. Individuals  with low income and low assets may be eligible for Part D premium  assistance. High-income beneficiaries may be charged a premium  surcharge. Most Medicare Advantage plans include the prescription drug  benefit in their plan of benefits with no additional premiums; however,  the plans may still require deductibles, co-pays, and cost sharing for  prescription drugs. IRA of 2022 made substantial changes in the Medicare  Part D design, which affects coverage and cost. Most of these changes  make prescription drugs more affordable under Medicare Part D. To  understand Medicare Part D benefits, we will look at how the program was  designed, the changes made, and how the program will work after all the  changes are implemented.

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The  easiest way to understand how Medicare Part D works is to understand  that there are different phases of coverage. In each phase, the  responsibility for funding is spread among the different parties. First,  a beneficiary must pay a monthly premium to enroll in Part D coverage.  Premium subsidies are available to some low-income beneficiaries, but  typically the beneficiary pays 100% of the premium. When an enrolled  beneficiary has a prescription drug expense, the individual must first  pay a deductible. The covered individual pays 100% of prescription drug  cost up to the deductible limit. The deductible limit is currently about  $543. After the deductible has been met, the initial insurance-coverage  phase begins. In this phase the beneficiary and the plan share the cost  of the prescription drugs. The beneficiary pays 25% and the plan pays  75% of the cost. This cost sharing continues up to $5,030 of  prescription drug costs.

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For  prescription drug expenses costs between $5,030 and up to $12,447, there  is a gap in insurance coverage. In the insurance coverage gap, the  beneficiary continues to pay 25% but the remaining cost is covered in  part by product discounts from the drug manufacturers (70%) and 5% by  the Part D plan. Total prescription drug costs that exceed $12,447 are  considered catastrophic costs and the cost-sharing arrangement changes  again. The beneficiary’s share of the $12,447 threshold is approximately  $3,100 in out-of-pocket expenses. In the catastrophic phase of  coverage, the beneficiary pays 5% of costs, the Part D plan pays 15%,  and Medicare pays 80%.

The  problem for individuals with a high prescription drug cost is that there  is no out-of-pocket limit or cap on the beneficiary’s share of the  costs as Part D was originally designed. This is one of the problems  that the IRA changes. The changes in Part D implemented by the IRA will  be phased in over time. In 2024, the IRA eliminated the 5% cost sharing  in the catastrophic phase of coverage. However, during 2024, the  beneficiary’s out-of-pocket limit increases from $3,100 to $3,250.  However, beginning in January 2025, the beneficiary’s out-of-pocket  limit will decrease from $3,250 to $2,000. The beneficiary’s  out-of-pocket limit may increase thereafter annually on an indexed  basis. People with CF who have Medicare Part D may be eligible for  co-pay assistance from certain organizations.  CF Foundation Compass can  provide information to people with CF about possible assistance for  Part D copays. The CF Foundation Compass program can be reached at compass@cff.org or by calling 844-266-7277.

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Nothing  in this article is meant to be legal advice and is only information.  Nothing in this article guarantees Medicare coverage for any service or  medication. If you have questions about laws related to Social Security  benefits, Medicaid, Medicare, health insurance, employment, and  education right you can contact the CF Legal Information Hotline at CFLegal@sufianpassamano.com or 1-800-622-0385.

Beth  Sufian is 58 years old and has CF. She is an attorney who focuses her  law practice on disability law and is the 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.

I never knew how much I’d come to take Florida for granted until I found myself in Denver for a conference.

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Let’s  rewind a bit. The year was 2014, long after my parents and clinicians  first suspected I had CF. I’d been tentatively diagnosed with the  disease at age five and treated as if I had it for the remainder of my  pediatric and adolescent care. After I became an adult and subsequently  moved over 1,200 miles away from my previous providers, my care fell  into complete disarray. I’d been dealing with recurring pneumonia from  mycobacterium avium complex (MAC) for several years on and off—a  never-ending cycle of taking broad-spectrum antibiotics, grudgingly  swallowing a bit of codeine cough syrup to sleep, and then hacking up  green jelly for several minutes in the middle of the night.

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I’d been  doing okay for a few weeks and felt excited to present some of the  evaluation work I’d been doing with the Florida Asthma Program at the  American Evaluation Association’s annual conference that October. I was  also scheduled to speak on a panel with colleagues I’d met through those  networks. Anyone who’s spent a few minutes with me knows I love public  speaking! So it should tell you something that amid a flurry of exciting  things to do at the conference, I spent nearly every moment of my spare  time flat on my back in my hotel room, with the lights out and the  curtains closed, staring at the ceiling and struggling to breathe. I  also fainted during my poster session, crumpling like an accordion onto  the patterned carpet below my board.

This was  quite a change from my first night in town, when, despite feeling dizzy  and lightheaded, I managed to go out with a friend who lived in town  for some delightful Ethiopian food and a tour of his favorite  neighborhoods. My friend was concerned enough by how badly I was  struggling to get around by the end of the evening that he flagged down a  city bus between stops and convinced the driver to let us on. Things  got worse from there. I managed to present my poster and speak on my  panel. Looking at photos from the panel session reveals me smiling  broadly and sitting bolt upright.

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Did I  mention how I have a lot of performing arts training? Convincing people  that you’re more alive than you are is a skill many of us in the CF  community learn well, whether onstage or off. And as Shakespeare once  put it—and any sociologist trained in dramaturgical traditions will  confirm—all the world’s a stage to some extent.

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I don’t  remember much from that panel—or from the remainder of the day that  followed before dragging myself out of bed to head back to the airport  the next morning. Thank goodness I’d already reserved a taxi for the  trip. I could barely wrangle my roller bag over the curb to get into the  terminal. I kept stopping to sit on top of my luggage while I navigated  to my gate. I don’t recall most of the plane ride back to Atlanta,  either. But I do remember the moment I stepped out of the airbridge and  gasped like a fish flopping around on land. I just stood there against a  wall of the concourse, sucking in the biggest lungfuls of air I could  manage until I could walk well enough to get on the tram and find my  connecting flight to Tallahassee.

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I made  it back to my apartment, fell into bed, and slept for an inordinate  amount of time. My spouse, then my partner, came up from Tampa that  weekend to visit me. They spent the entire weekend carrying me around  because I could barely walk. During the night, they would pound on my  back over and over until I coughed out enough foul-smelling muck to be  able to breathe at all. The supply of mucus seemed to be never-ending. I  understood on some level that I was dealing with pulmonary edema, a  condition where the lungs fill rapidly with excess fluid. It certainly  felt very different from the usual “walking pneumonia” symptoms caused  by the MAC that had been living in my lungs for several years.

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I went  to my primary care doctor and was put on antibiotics once again. They  didn’t do much. Being back at only a few feet above sea level helped the  fluid in my lungs dissipate as I continued coughing up massive amounts  of sickly green mucus. But the inflammation in the lung tissue didn’t  subside and the infection didn’t improve. I kept going to work and was  met with horrified looks from colleagues. I had to move offices that  week and was assigned a staff member to help me relocate my books and  files. I’m not entirely sure what happened for the rest of that month.  You get accustomed to living in an altered state and memories just  vanishing without ever forming in the first place. Doing it tired. Doing  it sick. Doing it very nearly dead.

What I  do remember, vividly, is the appointment I had with a local immunology  practice where I was put on whopping doses of inhaled corticosteroids  and an anticholinergic medication to stop the vicious cycle of  nonproductive coughing from increased irritation to membranes in my  respiratory system. I breathed in and out. A nurse gave me a full  battery of pulmonary function tests with scary results. I remember the  exhaled nitric oxide assay the best. “You’re breathing out pure poison”  isn’t something one forgets hearing, I imagine.

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Years of  MAC infections had damaged my lung tissue to the point that my alveoli  (the little sacs at the end of each of our small airways that get oxygen  into our blood) rapidly failed at higher elevation. My diffusion looks  good at sea level but in the “Mile High City” I barely lasted three  days. As ever, context matters. I should never have gone out there  without portable oxygen. I also wouldn’t have had much hope of getting  that set up given I wasn’t even an established patient at an accredited  CF clinic at the time, much less conclusively diagnosed.

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Two  years later, that changed. Inhaled corticosteroids and a lot of manual  airway clearance kept me alive to see that day come. It seems completely  wild now to look back and remember those days of having to cobble  together my own care and experiencing problem after problem with no end  in sight. But after several years of immersion in collaborative science  and advocacy about CF—and especially learning about the dangers of  insufficient diagnosis and discontinuity of care—I know my story is  hardly unusual. Indeed, the most unusual thing about it is my having  survived to tell it at all.

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A lot of  people with my background still don’t make it. Having lighter skin and  parents who taught medical school themselves at least meant I was at  least tested for CF as a child, despite my inability to produce enough  sweat for a valid sample on the chloride screening. I received enough  care to take me into my adult years—but with only a house of cards to  shield me from the weather. When it all came tumbling down, I thought I  might actually go to sleep on the floor of an airport terminal and never  wake up. This didn’t bother me as much as it probably should have at  the time. I was just trying to survive. Now, remembering those days now  feels harrowing. As difficult as things sometimes get for other  reasons—living with complex post-traumatic stress is never a picnic even  when one can breathe—today I can envision my future as more than a  black hole.

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When I  look at those photos from the panel, I see the big smile and remember  how glad I felt to participate. Moreover though, I see the glassy look  in my eyes. The dazed expression beneath brittle hair and skin that had  taken on a strange and discolored hue, with too much blue showing  underneath. I remember getting on that plane in Denver and collapsing  into my seat. Closing my eyes as the flight attendant demonstrated how  to put on the oxygen mask in case of an emergency. I didn’t have the  heart to tell anyone I was already there.

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I  haven’t left Florida for a long time now. Life looks very different than  it did in 2014, in some ways for the better and in others for the  worse. One thing that has changed though is my own willingness to insist  that my basic health needs be met. To expect, rather than request,  required accommodations for my safety guaranteed under the Americans  with Disabilities Act. To persist in getting whatever medications,  devices, and other instrumental and social supports I need to stay well  at a given time. To seek help rather than saying “I’ll get through it”  and expecting myself to be tough and not complain. Other people have it  harder, right?

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Other  people are dead. And I didn’t make it past my 40th birthday to stay  silent about how other people in our community—people with multiethnic  backgrounds, people of color, people with rare and nonsense  mutations—often wind up gone before their stories ever reach the world’s  ears. You might say I feel inclined, as the landscape of CF science and  care finally evolves to embrace our whole patient community, to keep  shouting the dangers of exclusion and apathy from a mountaintop.

Just not without portable oxygen, because now I know much better.

Dr. Alexandra “Xan” Nowakowski is 40 years old and has CF. Xan is a director of CF Roundtable, in addition to being a medical sociologist and public health program  evaluator. They currently serve as an Associate Professor in the  Geriatrics and Behavioral Sciences and Social Medicine departments at  Florida State University College of Medicine. They also founded the  Write Where It Hurts project (www.writewhereithurts.net)  on scholarship engaging lessons from lived experience of illness and  trauma with their spouse, Dr. J Sumerau. You can find their contact  information on page 2.

As I grew stronger and reached tiny goals, my overall scope of what I could do

expanded and evolved exponentially.

As I sit  here on my front porch, I listen to the birds sing their final song for  the evening. I watch the western sky turn a golden hue and an  overwhelming sense of peace begins to fill the air. I gaze through my  binoculars to watch the 25-30 whitetail deer munching on the freshly  sprouted soybeans. The aroma of freshly cut grass still lingers in the  air from when I mowed earlier in the day. The chirping sound of katydids  ignites memories from my childhood in rural Ohio. As the sunlight fades  and gives way to darkness, the first few stars begin to flicker, and  the waxing moon becomes brighter in the sky. It’s a perfect end to the  day and the tranquility refreshes my soul.

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Earlier  in the day, however, it was far from tranquil. My mind and body waged  war against each other. You see, I’m in the latter end of a training  cycle preparing to run another ultramarathon and these are the days that  will determine how quickly or even if I finish this 50-mile race. Some  days, my body and mind are perfectly in tune, relishing every mile,  while other days, I feel like giving up completely. Today was one of  those days and admittedly I gave in to the excuses and the pain and I  quit before I reached my goal.

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In the  past, if I gave up on my training goals, I would spiral into  self-loathing, which, of course, doesn’t help. Rather, I’ve learned to  channel my energy to determine what went wrong and adjust my regimen  accordingly. This helps me find that balance to push myself to the limit  while still achieving my goals. Today’s adjustment in preparation for  tomorrow’s 15-mile run includes different shoes, changing what I eat  prior to running, as well as refueling appropriately with electrolytes  during the run as the hot summer sun takes a much greater toll than I  had been calculating.

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If  you’re wondering what exactly my training schedule looks like pre-race,  here is an example. I still have several weeks left to train and the  intensity will continue to increase in the coming weeks, but this was  the past six days with a plan for the 15-mile run

Saturday morning:

Sunday: run 13 miles.

Monday: walk 5 miles in the sand with a 40-lb vest.

Tuesday: run 10 miles.

Wednesday: run 10 miles.

Thursday: 100 pullups, 200 pushups, no run this day.

Friday: run 8 miles.

Saturday: *Planned run for 15 miles.

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On top  of this, there are many other elements that come into play in this  preparation sequence: many icy cold baths to aid in muscle recovery and  several hours of yoga and stretching each week to prevent any muscle,  joint, or tendon injuries. I also need to ensure my body has the proper  nutrition each day to remain fueled and efficient. Last, and far from  least, is the overarching need to be very cautious and fully attentive  to my body to avoid injury or sickness. At this point, almost any little  incident could disrupt my training and ultimately lead to a drop out or  unsuccessful completion come race day. While this level of discipline  can be difficult to maintain, I also know full well that without these  intense measures, the odds of running a successful race decline  precipitously.

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One may  ask, why do this in the first place? Is it really worth the pain and  effort? Believe me, I ask myself this question at about mile 12 of every  long training run I undertake. I always come back to the same answer  which is really just another question: “What do I gain tomorrow or in  the future by doing this now?” I think Abraham Lincoln put it into the  proper perspective when he said, “Discipline is choosing between what  you want now and what you want most.” So, what do I want most? Of  course, that’s a very loaded question which requires a great deal of  thought to even articulate a decent answer. I guess from the fitness and  health perspective it’s a twofold answer for me. Simply put, I want to  keep my body and lungs healthy as long as possible and, additionally,  prove what CF patients can possibly accomplish with Trikafta or other  modulators.

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Secondly,  I want to continue living a life of adventure; to be able to find the  beauty in desolate places and make memories while exploring mother  earth. My diligence with routinely running and lifting has given me the  opportunity to hike into some areas that are completely untouched by  human civilization, where all things are still truly wild. I’ve been  able to summit the peaks of several mountains that exceed 14,000-foot  elevation. Most importantly, I’ve been able to go on adventures with  friends and family and have discovered that relationships are nurtured  more along the journey up and down the mountains than almost anywhere  else.

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What if  we’re at a point in our journey of life where we haven’t figured out  what we want most? Does that mean we’re just indecisive, lazy, or  unmotivated? No, not at all. It means we just need more time and more  experiences under our belt to calibrate our personal compass. I’d love  to tell you I’ve always been exceedingly disciplined and from the start  have had these grand plans to run races and climb mountains. However,  that couldn’t be further from the truth. In fact, many years ago, when I  first started on this journey, my vision for fitness was completely  undeveloped. At that time, I really had no idea that I wanted to hike to  the peaks of giant rocks or go off-grid for a week at a time living  only with what I could carry on my back. I had no idea I wanted to run  ultramarathons or write about my adventures. All I knew is that I wanted  to keep my lungs healthy as long as possible and keep my body in great  shape. Plus, I always enjoyed the social aspect of working out and  running with friends. That simple objective was enough to get me started  and, looking back, I’m quite grateful for the path my journey has  taken.

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Even  though my original fitness vision wasn’t completely fleshed out, as I  grew stronger and reached tiny goals along the way, the breadth of what I  could do radically expanded and evolved. While there is much to be said  for big picture planning and developing a play-by-play of our goals, I  believe too much rigidity can also be very limiting to our growth.  Several years ago, when I finally hit the milestone of being able to run  five miles, I thought I had reached my personal pinnacle. The thought  of running 10 times that distance hadn’t even entered the realm of  possibility for me.

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I’ve  learned that much of life is like climbing a mountain. For the first  part of the journey to the top, you can’t see the summit at all; you’re  surrounded by coniferous forest and only a small section of trail is  visible ahead of you. As you traverse through switchbacks up the  mountain you begin to see the path laid out ahead of you. You clear the  tree line at roughly 11,500 feet and you finally catch a glimpse of the  peak you’re heading towards. Now your goal starts coming into focus.  Along the way you’ll encounter several false summits, those peaks where  you think you’ve finally made it, only to discover there’s more  elevation to be gained ahead of you. But with each false summit your  view of the world just becomes larger and the beauty more awe inspiring.  Eventually, you make it to the actual summit and it’s like being on top  of the world. The views are mesmerizing and as you look around from  whence you came and realize the enormity of your accomplishment,  euphoria sweeps over you. You feel like you can see the entire world and  as your eyes take in the vast surroundings, you begin to wonder about  all the other peaks visible in the distance. You think to yourself,  what’s the name of that tall, rugged peak over yonder? Next thing you  know, you catch yourself dreaming. I made it to the top of this peak,  perhaps I could climb that one as well?

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I guess  my point is this: in our various endeavors in life, there will be times  that our vision is chaotic and muddled and even times when we’re lost  and desolate. But even the tiniest goal or the littlest ounce of passion  is all it takes to get started. With time, discipline, and consistency,  we can grow stronger and, as our capabilities increase, new  opportunities will begin to appear. The compounding effect of small  successes begin to propel us forward and up, making things that once  seemed like an impossibility begin to appear on the horizon. The next  thing you know you’ve made it to one peak of many peaks. What will you  conquer next?

Marcus  Miller is 31 years old and has CF. He lives outside Wilmington, North  Carolina, about 30 miles from the Atlantic Coast. He has the best pup in  the world, a Siberian Husky, named Emma and she accompanies him on most  of his adventures. His true passions in life are hunting, archery,  running/fitness, hiking and camping, and basically anything that gets  him out in nature. If you’d like to follow his adventures or reach out  to him, you can find him on IG @marcusrmiller or by email at mmiller@usacfa.org.

When  the temps are warmer, I start looking for a quick and easy meal. Summer  is the time to be on the go and I like meals that have very few  ingredients but are also nutritious. This easy egg salad takes your  basic mayo base and uses Caesar dressing instead. You can of course make  your own Caesar dressing, but store bought is just as good! So many of  the ingredients I added to this dish are optional. If you have a  favourite add-in to your egg salad that isn’t on this list, please add  it! This recipe is very customisable. I love a good base recipe that can  be used in so many different ways. Add the egg salad to toast, a  sandwich, crackers, or a lettuce wrap for a low carb option. If you want  to see how I made this step-by-step, I have a reel on Instagram. Please  go to @justasprig on instagram for this recipe and others!

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Easy Egg Salad

Ingredients:

• 4 medium eggs

• 2-4 tbsp Caesar dressing (store-bought or make your own!)

• 1 celery stalk, chopped finely (optional)

• 1 small onion, chopped finely (optional)

• 1 tbsp chopped fresh chives

• 1 tsp dried or fresh dill (optional)

• 1 tbsp chopped capers (optional)

• 1 tbsp lemon juice

• Salt and pepper to taste

Preparation:

Step 1:

In a  small to medium-sized pot, add water until all four whole eggs, in their  shell, are just covered. Once the water comes to boil, set a timer for  eight minutes. Once they have boiled for eight minutes, drain and rinse  the eggs in very cold water for a couple of minutes. Peel the eggs and  cut into rough chunks.

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Step 2:

Add the  eggs to a mixing bowl along with   the remaining ingredients. Mix well,  breaking up the eggs even more, until well combined. Serve and enjoy! s

Maggie  Williamson is 35 years old and has cystic fibrosis. She received a  double lung transplant in 2014. She now lives in the U.K. with her  British husband, Tom, and their Bengal cat, Charlie. You can find her  and all of her cooking delights on Instagram @justasprig

I  am a Fanilow and I’m not ashamed to admit it. According to the TV show  Will & Grace, being a Fanilow means you are a big fan of Barry  Manilow. For those of you who do not know who he is, he is an amazing  singer, songwriter, and musician. He is also an incredible entertainer,  as I can attest to since I have seen him in concert about 20 times.  Seriously, look him up and listen to his music or watch some of his  concert videos. You won’t be sorry, I promise.

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My  parents played the Even Now cassette tape in the car on every road trip  when my brother and I were growing up. I had every song memorized; of  all his songs, Even Now is still my favorite song. That was the  beginning of my Fanilow days.

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What  does Barry Manilow have to do with my trip to New York City? Everything,  really. Since I am (obviously) a member of the Barry Manilow fan club, I  get advance notice and early access to the best tickets at upcoming  shows. When I got an email about him being in NYC for five days of  concerts in April 2024, I contacted my best friend and kidney donor,  Kelly, about going to see him. She had never seen him in concert so I  was determined to show her how great Barry Manilow is live.

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Kelly  and I each took the train to NYC early on Wednesday for the concert that  night at Radio City Music Hall (sold out by the way). Barry was  amazing, of course! I got my usual goosebumps and shed a few tears for  certain songs that have special meaning to me. Kelly loved the show and  can’t wait to see him again! Radio City actually added five more  concerts in October 2024 because the April shows sold out. I may be  back, NYC! People in NYC especially love him because he is a native New  Yorker (he grew up in Brooklyn) and he’s a wonderful person and  performer. Seriously, look.him.up.

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We  stayed until Friday afternoon to do some other things, including going  to the Today Show, attending a couple of museums, touring St. Patrick’s  Cathedral, eating lots of pizza, and having lunch with USACFA’s  Executive Editor and Webmaster extraordinaire, Andrea Eisenman! Andrea  and I have known each other for many years; however, that was our first  time meeting each other in person (you may have seen our six-feet-apart  pictures in the last CF Roundtable issue and/or on USACFA’s social  media). Kelly and I had a wonderful time despite the rain and the cold  and we were exhausted from all the walking and doing so much. I did wear  a mask on the train, in the museums, when walking around in general,  and in the Cathedral. I didn’t wear it during the concert because I knew  I would be singing and screaming.

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We only  brought our backpacks and cross-body purses with us. We didn’t want to  deal with a lot of luggage while walking from the train station to our  hotel, which was about 15 NYC blocks (which can be shorter or longer  than a regular city block). Since we were technically only going to be  gone for two full days, I decided not to bring my nebulizer air  compressor (for hypertonic saline inhalation) and my Monarch vest. I  normally do these treatments twice a day and I did them Wednesday  morning and Friday evening. Plus, I knew we would be walking a lot (not  to mention dancing and screaming at the Barry concert), so I figured it  was ok to miss a few treatments. It also allowed me to have more time  for sightseeing. I think for me personally, missing four treatments was a  good trade-off with being so active while we were in NYC, plus it was  so much easier not lugging those machines around, as many of you can  relate to. My health was fine when I got back home, so the lesson  learned for me is that I can skip a few treatments for the sake of  making travel much easier, but I would not want to skip more than that.  Also, I had a lung transplant and take Trikafta, so I don’t produce much  mucus as it is.

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The  bottom line for traveling with CF is to assess your own health and get  your doctor’s opinion if possible before you travel so you can make an  informed decision on what treatments you can skip and which ones you  should not miss. Also, having someone I know who has CF living in NYC  was great because if I did get sick or injured for whatever reason I  could ask Andrea where to go for the best treatment. When planning your  trip, make sure you know where the nearest CF center is at your  destination and research which hospitals have the best ratings. Know  before you go!

Colleen Adamson is 55 and has CF. She is the Treasurer of USACFA, and lives in Alexandria, VA. Her contact info is on page 2.

It’s  been on my mom’s bucket list to visit Quebec City and with that only  being an 8-hour drive, we decided this year was the year to check that  off the list. So we booked a sweet Airbnb and contemplated if driving an  8-hour stretch there and back was both feasible and wise with a two and  almost-four year old. We decided it wasn’t so we planned on staying in  southern Vermont for a night on the way up and in Burlington, Vermont on  the way back to break up the trip and hours spent in the car.

Itinerary

• Day 1: Drove from Cape Cod to Vermont; 3 hours and 45 minutes. Stopped for dinner en route.

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• Day 2:  Drove to Airbnb near Quebec City; 5 hours. Stopped periodically,  including for lunch. Arrived and checked into the Airbnb and then met  the rest of our group at St. Bernard, a local pub where we had a  reservation.

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• Day 3:  Drove to Montmorency Falls and went on a hike—well, that is what my  brother and his girlfriend did; I walked, slowly. I also followed them  down 500 stairs—that was a mistake! My legs pulsated for days. We then  headed into Quebec City and got on the Hop On & Hop Off bus to tour  the city. After, we headed back closer to our home away from home and  ate at La Grange.

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• Day 4:  One of my favorite things to do when traveling is check out a spa. So, I  booked a massage for half the group and we participated in the Nordic  experience of alternating hot, cold, and relaxation—we loved it!

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• Day 5:  We checked out and headed to Burlington, VT. Spent the evening  exploring the city and had a yummy dinner at a local brewery, Zero  Gravity, and then went to both Lake Champlain chocolates and Ben &  Jerry’s for dessert.

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• Day 6:  Checked out of our Airbnb and ate at August First—highly recommend! My  mom’s friend lives in the area and met us for brunch, which was a nice  treat! We drove home to Cape Cod, MA. It was a lot of driving,  especially with little ones, but it was fun and always nice to connect  with loved ones.

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Packing with CF has Always Been a Project, Yet I've Found a Rhythm:

• Get  the important paperwork together: passport, license, and a credit card  that doesn’t charge foreign transaction fees if leaving the U.S. Also, a  letter from my doctor that says I have CF and need to take a ridiculous  amount of medications daily.

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• Double check all the expiration dates on said paperwork.

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• Take a bunch of stuff out of the closet and throw it all on the bed.

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• Roll up the clothes and arrange them into piles by type.

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• Count the clothes and take the minimum needed.

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• Lay  out all of my medications, weighing the risk of either combining them  into baggies for each day and possibly having a problem with security  versus each pill bottle taking up the majority of space in my suitcase  as taking 15 or so different medications does that.

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•  Contact my doctor to discuss getting an antibiotic on hand in case of  illness as I’ve already been experiencing an increase in asthma symptoms  and my rescue inhaler hasn’t been cutting it so the call has been long  overdue. He recommended the SMART protocol for the next 48 hours (i.e.,  taking my symbicort every four hours instead of the usual a.m. and p.m.  dosing. We also discussed different options for antibiotics.

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• If I  was flying as a part of the trip I would insist on antibiotics because  every time I fly, I get sick. Luckily, this trip is a road trip so I was  less concerned, or at least I would have been if I hadn’t had trouble  breathing prior to leaving.

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• If I  was a “good” CF patient, I would also pack my vest and nebulizer and  those medications but I’ve never been a “good patient,” even prior to  Trikafta. A lot of my CF symptoms are mild so I have been able to get  away with not bringing those things on vacation. This benefits my mental  health.

OK…I’m ready!

Oh wait, I have Two Kids Now... Not Ready

• Throw all their clothes on the bed. Sort. Roll. Pack.

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• Pack Tylenol, just in case.

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• Grab the toothbrushes and toothpaste.

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• Get the diapers and wet wipes.

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• Can’t forget the Desitin!

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• Oh! The bottles and sippy cups too.

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• Sunscreen. Need bathing suits too.

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• Toys for car rides and picture books.

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• New travel car seat (boy, that took a while to figure out!)

Cleaned the house.

Cleaned the car.

Packed up the car, including my husband’s and mom’s stuff.

Packed the cooler for snacks, drinks, and medications that need to be refrigerated.

Started driving…I’m sure I’m forgetting something, I just wish I knew what. Fingers crossed it’s something I can buy en route.

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Katherine  Lockwood is 36 years old and has CF. She lives on Cape Cod with her  husband Arden and her girls, Rose and Magnolia. She is a therapist for  Verge Therapy and focuses on supporting individuals and couples  experiencing disability in the family. She is the author of Why Me, Mama?, an award-winning children’s book about the disability experience. She is currently working on two picture book projects: OUCH! OOPS! & OH NO!, a set of three books to support pre-k to third graders in reducing  bullying and My Body Beeps!, a book about balancing taking care of  yourself while having fun. You can follow Katie’s picture book projects  on Instagram @acorn_cottage_press.

It  has been a privilege and an honor to get to know Marina. Prior to this  interview, I received a book of her artwork. Her images—sculptures,  assemblage (a style of art in which various found objects are assembled  together for the installation), or watercolors—are soothing to look at.  There is whimsy with beautiful, serene colors, sometimes using a  collection of found objects. She is clearly a creative person which, as  she tells it, helped save her life and kept her going through adversity.  In the following interview, you will see she was quite late in getting  diagnosed with CF. This led to much suffering most of her life with  classic CF symptoms—frequent infections and crushing sinus  headaches—which possibly led to her self-medicating. She has achieved a  lot: becoming a nurse, then a therapist, and now a full-time artist who  showcases her art in galleries and gives talks. Throughout her long life  she remained creative, a key part of how she has coped with all her  unknown afflictions. Even while she may not be physically well and is  currently healing, creating art, for her, is a must and still keeps her  going. Read on about her journey—late diagnosis, living abroad,  overcoming addiction, and always caring for others. Please welcome our  newest star, spotlight please!

When and How were You First Diagnosed?

I am 80  years old; a mother, wife, therapist, and artist. I was diagnosed at age  68 by Dr. Jeffrey Suh, who is an amazing Head & Neck Surgeon at  UCLA in Los Angeles, California. I was having recurring sinus infections  which required IV antibiotics and multiple surgeries to treat them. It  was a great relief to finally know why I had been having all these sinus  infections and why I had suffered from headaches, pain, and sickness  all my life!

Did You Have Siblings Who Also Had CF?

I grew  up in Connecticut; number four of six children. One brother, slightly  older than me has struggled with nasal polyps, pneumonia, and repeated  surgeries. He happened to be in the hospital having his lungs drained at  the time of my diagnosis. I called him as soon as I was diagnosed! He  discovered he too has CF.

What Were Your Main Health Issues Growing Up?

As a  child in a bustling family, I tried to keep up as my siblings were very  athletic. I knew early on that I didn’t have their endurance in sports  such as field hockey, volleyball, or tennis. I made the teams but then  couldn’t breathe. What was it? Allergies? Asthma? A missed year of high  school for mono? I was always trying nose drops or steam, or anything  really, something that might help me breathe and take the pain away. I  had severe headaches throughout childhood.

You became a nurse; were you trying to figure out your medical issues? How did being a nurse affect your health?

There  was an element of stress in the family that complicated my situation. My  father was an alcoholic and my mother had very little understanding of  his condition so she certainly didn’t want sick children too. I learned  to just pretend I was okay. I would just go to my room to isolate and  cough.

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As the  first girl, I quite naturally became my father’s companion. I even went  to Georgetown University School of Nursing to “figure out how to get him  well.” Unfortunately, he died during my freshman year but I stayed in  school battling round after round of infections.

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After  university, I married and lived in Berlin, West Germany for two years.  It was cold, damp, and dark. Although a very interesting experience, I  continued to hide how I felt. That is, being ashamed of coughing too  much and being sick. My son was born there. I was in labor for three  days and finally had a Cesarean section. Both the baby and I had  pneumonia.

When we  moved back to New York City, I worked at Lenox Hill Hospital in the  intensive care unit on the night shift. We were usually masked and I  assume that protected both the patients and me. I have always been glad  that I had the nursing background because I have used it with family and  friends throughout my life.

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I moved  to Los Angeles in 1969 and CF definitely came with me. I lost three  babies early in pregnancy. I had endometriosis, pneumonia, polyps,  ectopic pregnancy, meningitis and shingles three times. Finally, after  nine years of trying, I gave birth to a beautiful baby girl.

That all sounds tough to endure, how do you feel about it now?

Now  looking back on my young adult self, I can feel compassion for what I  went through. At the time I had no idea why I was so sick and felt  tremendous guilt and embarrassment about it. I became addicted to  narcotics and alcohol. Being diagnosed so late, at 68, I just thought I  didn’t know how to take care of myself.

What has helped you persevere?

By the  age of 35, I knew my addiction was a problem; the family disease of  alcoholism was also mine. At first, I tried to address it on my own but  am forever thankful that I found both Al-Anon (for family or friends of a  loved one battling alcohol use disorder) and then AA (for an individual  struggling with alcohol abuse)—they’ve both been tremendously important  in my recovery. I share this here because my journey with CF was still  unknown to me so I couldn’t connect that I was self-medicating to  address a serious health problem as I blamed myself for being sick  without a “reason.”

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I have  been sober for 41 years; 41 years in AA and 44 years in Al-Anon. I am  still very active in both programs. At 80 years old I am grateful for  the love and understanding of these invaluable spiritual communities.  They certainly have been by my side throughout my CF journey.  Additionally, I am fortunate to be on the medication Trikafta and to  have the support of the CF team at UCLA. In particular, the  pulmonologist, Dr. Patricia Eshaghian, has shepherded me through a  variety of challenges this year—carotid artery surgery, a nasty broken  hip, and a C6-C7 back fusion surgery to address numbness in both my feet  and hands (which I am still working on—I walk with a cane because the  feeling hasn’t returned.)

What made you decide to become a therapist?

At 47 I  became a therapist. Caregiving has always been a part of me and I  delighted in accompanying others in their journey of self-discovery and  personal growth. I chose a Jungian orientation and found that my healing  and creativity were feeding each other. My medical background prepared  me to accompany three of my brothers on their final journeys.

What part has art played in your journey?

I have  always been an artist. As a child making things and painting stories  were my safe place. In sobriety I had enormous energy and the creativity  allowed me full expression. This year, although unable to walk, I still  made art in bed. I try to go within and allow whatever wants to be  given life to come through. Often it is several months ahead of me but  it brings a feeling of “yes-ness” and a strange quiet peace even if I  don’t quite understand it yet. I was overjoyed when I found one  hundred-year-old paper prescriptions to paint on. I collected maps; old  strange wartime ones to paint and reconfigure—art were helping my inner  war!

What does being creative do you for you currently?

Creativity  lifts me out of today. When I am painting or make collages, I lose all  track of time and access another level of connection. As a child,  struggling with breathing and tummy aches, I would hide quietly in my  room and draw or make clothes for my dolls; it was another world! Now I  experience a spiritual connection when I am creating. I am often totally  surprised by what comes through. If I am encountering a difficulty in a  relationship or circumstance I meditate and wait. Often, an image will  present itself then I try to make it!

What would you say to your younger self?

“Sweetheart,  I wish you hadn’t had to suffer so long and alone. I have such  compassion for you hiding and wish you hadn’t blamed yourself for your  health challenges, but I also see you now as quite a little hero and if  you had known you might never have given birth in Berlin or become an  artist to express your feelings. Certainly, you would never have become a  nurse and exposed yourself to so much danger. How could you have known  the blessings of sobriety, without battling the disease of addiction?  You became a therapist to try to understand your own inner workings and  that enabled you to help others. What a trip!”

What is your life like now?

The  growing edges of my life now are acceptance and gratitude. My dear  husband Paul has taught me so much about love, kindness, gentleness,  art, music (he was a fabulous piano player), friends, family, and simple  things. I miss who he was but I love who he is. His bravery in letting  go and surrendering to Alzheimer’s has given us another dimension of  love—it is an honor to care for him.

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It seems  crazy to say that I may be happier now than at any other time of my  life. I feel that all the threads (I do needlepoint) are coming  together; I see my life more completely and I can relax and breathe. I  am so grateful for my children and their creative journeys, for eleven  grandchildren that surprise me with their antics, for art, new ideas,  and new friends. I still work with AA women who have become my sisters.  Now, rather than hiding, I try to say “yes.” For example, I am part of  the CF Research Institute (CFRI) late diagnosis group with Jeanie and  Laura. We meet monthly—it’s my tribe of women with CF and we all  understand what this disease means and that is a powerful tool in coping  with CF. I stay in close touch with my remaining brother and sister and  a huge extended family.

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What is new for you?

Exercise  is my new mountain! Physical therapy and Pilates are unfolding miracles  for me. I am learning new things about my body at 80! So, I am never  bored. I now believe that we are spiritual beings having a human  experience—life is a gift! CF and all that it has brought with it has  become a precious part of my life.

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My  husband is 86 and has Alzheimer’s disease; he is in his 13th year. I  made calendars for each day, week, and month, so he could orient and  know where he was and what was going on. I wrapped little boxes inside  the calendar pages and placed a magnet inside each box so they would  attach to the metal studio door of my office. This became the canvas for  my piece entitled “Stairway to Heaven.”

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My  husband has four children and I have two. We have been together 33 years  and have 11 grandchildren ranging in ages from 21 years to six months!  My husband practiced law but was also a musician and founded a Waldorf  school. All of our children are creative—architects, designers,  documentary filmmakers, and teachers—and so far, none with CF!

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Do you exhibit your work? Where?

Yes, I  did show my work in various galleries here in Los Angeles, New York, and  in Europe. Generally, I have one solo show and one group show per year.  It has always been a delicate balance between family, therapy, and  creativity.

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In the  past few years, I have been focused on family health; three of my  brothers have died, my husband has declined, and I have had issues  myself, but I continue to make things and feel happier and healthier  when I do.

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Recently  I met Dylan Mortimer, a fellow artist with CF. He was part of a group  show of 17 artists all with CF. The show, “Breath-Taking,” was curated  by Ted Meyer at the dnj Gallery in Santa Monica. As an addition to the  show, I participated in a discussion about creativity and health. I  shared that art is what I’ve always turned to! It was wonderful to meet  fellow artists with CF—we immediately had a kinship!

What was it like being diagnosed so late in life?

When I  was diagnosed with CF my husband was beginning his long journey with  Alzheimer’s. It was so difficult with CF because I was new to nebulizers  and various routines and my coughing and shortness of breath frightened  him. He, with the addition of friends and family, was always supportive  so it was as though we all sighed with relief to finally know my  diagnosis.

What, if any, impact did CF Roundtable have on your life?

CF Roundtable was given to me the day I was diagnosed, which took place in the  pediatric department of UCLA! I read it cover to cover! In fact, I  decided then and there that I would help this publication with support  from my foundation. I am so grateful for the honesty and openness that I  found here. I never knew anyone with CF so I suffered alone! Suddenly I  had a community: friends with CF, parents with CF, artists with CF, and  doctors who understood it—I am so grateful!

If you would like to be interviewed for “In The Spotlight,” please contact us at itsinterviews@usacfa.org. Either Xan Nowakowski or Andrea Eisenman will reach out to coordinate an interview.

First,  let me be clear: I love my new Tandem T-Slim insulin pump. There’s a  bit of a learning curve coming from an older system, Medtronics, where  the pump and Dexcom G7 continuous glucose monitor (CGM) didn’t share  glucose information with each other. My control was decent with an  average A1C of 6.5 but I felt it could have been better.

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Offering  more stability than my older system, the new pump ideally has the  potential to keep my blood sugars stable. Its goal is to keep my glucose  levels between 100-120. Since technology took a giant leap forward, my  diabetes center recommended upgrading to a “closed loop” system like the  Tandem T-Slim. The information from the Dexcom G7 CGM would be  connected via Bluetooth to the T-Slim. The pump then makes decisions  based on what it estimates my glucose will be in 30 minutes, using the  readings it receives from my CGM every five minutes. It’s almost like a  mechanical pancreas.

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With the  old system, I saw my CGM readings on my cell phone then I had to  manually input the number into my pump. If my glucose reading was high  it then figured out the amount of correction for a bolus to bring my  glucose within range, usually 120-170. Similarly, with my old pump,  before I ate any carbs I had to input my glucose readings from the CGM  into my pump and then guess at the amount of carbs and my pump would  tell me how much insulin to give myself to cover the meal.

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This new  system almost cuts me out of the equation, which is a good thing, for  the most part. If my new pump predicts my sugars are heading out of  range, it will give me insulin first by increasing my basal rate, which  is the programmed rate at which small doses of insulin are regularly  delivered. Then, if my glucose is still rising, it gives me an insulin  bolus, which is a more rapid delivery of insulin used to make  corrections. Mainly, the various methods of insulin delivery are unknown  to me when my glucose is rising quickly. This can be bad if I’m about  to exercise or walk the dogs. I do not want to have extra “insulin on  board” to risk getting a low during exertion. So, I must be  conscientious about putting this pump into “exercise” mode before  walking a lot or getting on my stationary bike. In “exercise” or “sleep”  mode it allows me to have slightly higher glucose levels before it  starts correcting it (in theory). Alternatively, this pump will lower my  basal rate if it knows my glucose number will be lower than the  preferred range in 30 mins or turn off delivery of insulin until my  level starts to rise. I’m still trying to figure all of this out.

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It is a  powerful tool which has kept me from having lows in the middle of the  night or just before bed. It is a delicate machine and when I was going  through training, I was warned when traveling by plane to never go  through the x-ray machine at the airport. The disclaimer says they  cannot guarantee it will work properly if it were to be exposed to  x-rays. I initially thought it’s not a big deal as I so rarely fly.

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Then, I  flew. I am not an anxious flyer but being on a plane post-transplant and  post-COVID-19 does give me pause about the increased risk of getting a  virus. To combat that I wear a mask, always. It is not a guarantee but  has kept me healthier. Going through security is never pleasant but it’s  a means to an end; getting on a plane and the subsequent freedom to  travel.

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I got up  super early to arrive at the airport two and a half hours prior to  takeoff, thankfully. I checked in and headed to security at JFK which,  if you are not familiar, is a gigantic, sprawling international airport.  A huge, hectic line unfolded in front of me at JFK. No biggie, I was  early. In the line, I removed my sneakers, placed my iPad, backpack, and  jacket in one bin (cell phone and keys in the pockets) and placed my  sneakers, belt, and purse (my life!) in another bin. I told the TSA  agent I cannot go through the x-ray machine because I have an insulin  pump and that I needed to be patted down. He says, so quietly it was as  if he was whispering into a hurricane force wind, “I need a female  officer here,” presumably for the pat-down. He tells me that someone is  coming. This wait started to look futile, but I remained patient and  hopeful. My stuff was now on the other side of security, as if it was on  the other side of a great divide. As the others who were behind me in  this line were now done being screened and allowed to gather their  belongings, I was not sure my stuff would still be waiting for me.

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I tried  to remain calm as the minutes ticked by. After ten minutes, as others  who were in wheelchairs, who also could not go through the x-ray  machine, were long gone, I again said, “I need to go through, my stuff  is on the other side of security—all my belongings!” Again, I was told  someone is coming, and the agent says “female agent request” to no one  in particular. Then the TSA agent who was there was replaced with  someone else. At this point I was almost hyperventilating. I started to  panic. I beseechingly said, “I need to get to my stuff over there, can  you find a female TSA agent?” He did seem to be trying to get someone. A  woman appeared but must attend to someone else first but, she reassured  me that she is coming back for me.

As I  looked around at the vast array of faces, many disgruntled, I vowed  never to fly again. I am not built for this. I then see a woman who  recently gave birth, judging by the age of the child in her arms. She  was in tears. At that moment, even though I was still panicked, I had  more empathy for this woman and knew my situation could be way worse.  Eventually, the TSA agent came and brought me to the other side of  security and patted me down thoroughly. I saw my purse and sneakers but  not my backpack, jacket or iPad. I told her I do not see them and she  asked me what they look like. It is in that moment when I thought I was  going to cry. I told her what color everything was and she acted like  she has no idea where it could be. I then stepped further into the roped  off area and I see my stuff behind security and pointed it out to the  agent. She said “I was trying to find you, we have to look inside your  bag.” Why didn’t she just look past the scanning machine? I was standing  right there! Obviously, she didn’t look very hard.

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When I  travel I pack all my pills into my carry-on to ensure I arrive with all  of my meds and nothing is lost in transit. This includes my inhalation  machine because my hypertonic saline is worthless unless I have a  nebulizer to inhale it. So, it was in my backpack. That set off all  sorts of worries with TSA. When I flew home, I put the machine in my  checked suitcase. I was not going through this again.

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Even  though I was upset, I did not take it out on the TSA agent. Not only did  I get felt up by her, but I also had to pass the hand swab test where  they test you for explosives. Once I was through the line and so  grateful all my stuff was intact, I had very little time to get to my  flight. As I mentioned, the airport is gargantuan—I had what seemed like  a mile to walk to get to my gate.

I was so  furious and didn’t want it to taint the rest of my trip, so I called  Steve, my husband, to vent. I was ranting, “Don’t they understand about  people with health issues? Why is this so hard? Obviously, the TSA is  understaffed so it happens. Why did I have to get this stupid pump just  before my flight,” I lamented?

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The rest  of my trip was great. I got to see and stay with my cousin and his  family in Florida. My other cousin who is on the other coast of Florida  came to see me as well. I also got to see a dear friend who I used to  work with 30 years ago. I had great visits with everyone and went to the  gym every day.

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As my  return date came closer, I grew more anxious. I knew getting to the  airport early was imperative should anything like my previous experience  happen again. I did think that the Florida airport was smaller and  better run than JFK. My hunch was correct. I only waited five minutes to  get my pat down and hands swabbed. On the first pass my hand swab was  positive. Thankfully, the TSA agent took it in stride and just tested me  again; I passed and was free to board my plane to go home.

Here is  what I learned: remain calm, it will work out. Arrive extra early if you  veer out of the norm—have an insulin pump, travel with a compressor,  etc. Be polite, you attract more bees with honey. Be prepared to have a  problem and know it can always be worse. But most importantly, enjoy  your trip!

Andrea  Eisenman is 59 years old and has CF. She is the Executive Editor of  USACFA. Andrea just celebrated her 24th lung transplant anniversary. She  and her husband, Steve Downey, live in NYC with their three  dogs—Roscoe, Trixie, and Willie. Her contact information is on page 2.

I’ve  said it many times and I’ll say it again—I’ve always wanted to travel  more. I wanted to travel more both before and after my transplant. I  battle persistent anxiety that often ends in me giving up on the idea of  a trip. Sometimes I am able to break through this resultant barrier and  I have yet to regret it when I do. Even though I can’t escape the  burden of care during travel, there is something about a change of  scenery that seemingly lessens that burden. I consider the wins small  steps in overcoming this anxiety and I hope that one day, with  experience and the support of others, I can knock it down completely and  leave all my hesitations behind.

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Prior to  my transplant, I never traveled much. Most traveling was done when my  parents were still the ones managing my care so I never had to think  about the complexities of traveling and juggling my health. Brief family  trips and one Make-A-Wish trip constituted most of my traveling as both  a child and an adult before my transplant. I often thought about travel  as I got older but the ensuing anxiety led me to frequently use CF and  the burden of care as an excuse not to go, which then led to  disappointment. Getting a transplant gave me a temporary boost in my  will to travel.

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Since  transplant I’ve done solo trips to Niagara Falls and Acadia National  Park. I have also traveled with my friend to California for a week in  addition to taking trips with my girlfriend to North Conway, Martha’s  Vineyard, and Las Vegas. Though I am happy that I have been able to  follow through with more travel since my surgery, it has not been as  easy as expected.

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I  thought the idea of traveling would become easier after my transplant  but, as the years have passed, things have become more complex. I am  free of supplemental oxygen but my burden of care has increased greatly  over the years since my transplant surgery. I have had to continue to  adapt and learn about what’s needed when traveling and what I can do to  make myself comfortable. Coming up with a travel version of my intricate  daily routine of workouts, airway clearance, medications, and therapies  has proven to be a challenge.

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I am  starting to learn more and streamline processes as I have eased into  traveling since my transplant. This has helped alleviate my anxiety  about going away for several days or longer. In the past, packing always  caused a lot of anxiety—many times I waited until the morning I was  leaving to finish up my vest therapy, nebulizer treatments, inhalers,  etc., before packing them. My girlfriend encouraged me to get the  Monarch Vest after having learned about it, which then led to getting  travel versions of all other medical equipment: a travel nebulizer with  spare neb cups, a spare Acapella, a spare incentive spirometer, and a  spare inhaler spacer. All of these now sit in my luggage, ready to go.  It can be a financial barrier to accomplish this but I am glad I was  able to afford it. This has helped lessen the burden of packing and the  anxiety of believing I might forget something. Having travel versions of  everything ready to go helps me envision my routine while I am away,  which makes things easier on myself. This has also helped me overcome  some of my internal barriers and it doesn’t just apply to packing.

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I used  to be hesitant or nervous about pre-boarding airplanes—people stare, but  it is our right to do so. I have not flown with the Monarch yet, but on  previous flights I still pre-boarded to sanitize the seats before the  plane filled up. When flying I also travel with a note from my doctor  indicating my surgical clips and sternal wires, lifesaving medications,  and multiple medical devices. The airport is a source of great anxiety  and I needed to start preparing better if I was ever going to travel  more.

I hope  that these things I have learned over the years, along with the support  of my girlfriend, will continue to decrease my anxiety about traveling  and start minimizing that barrier so that I don’t even realize it is  there. There are many trips I want to take and it is never easy to  travel when chronic disease follows you; however, I’m excited for the  future.

Brian  is 35 years old and has cystic fibrosis. He received a double lung  transplant in 2017. He lives in a coastal town north of Boston, MA.  Brian enjoys reading, playing video games, working out, yoga, and going  for walks. He loves spending time with his girlfriend, Syd. He can be  reached at armstrongb@merrimack.edu.

Most  of us probably already know that anything medical is exempt from  airline carry-on rules. In other words, I can have three bags of medical  and none of it counts against the limit. What I didn’t know is that  exemption isn’t always honored.

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Back in  2022 my husband and I flew to NYC with my dad and stepmom. We flew  Delta, first class. For my carry-on luggage, I had my purse, which is  more of a large handbag because I’m one of those women who carries  everything with them, just in case. I had my Frequencer, which is a  device that uses low frequency sound waves that match the resonance of  the lungs, causing them to vibrate, thus decreasing the mucosity in the  lungs and effectively breaking up the mucus. Much like the Vest or  Monarch, it’s wildly expensive and hard to replace. I also had the  litany of pills needed for the week-long trip, along with my nebulizer  and all of my nebulizer medications, including the refrigerated ones  like Pulmozyme and Cayston. Having traveled quite a few times with all  this, I had a good routine and way of packing to make it as easy as  possible to cart this across the airport. My bag of medications,  nebulizer, nebulizer cups, and the various inhaled medications all go in  a bag that says Medication (so it’s clear that it’s a medical  necessity) with the travel letter from my CF clinic readily accessible  in the outside pocket. This loops around the pull handle on the hard  travel case for my Frequencer, which has rolling wheels. Then, I just  have my purse and a small bag for my books, headphones, and chargers—the  usual things one has in their carry-on luggage on a plane. I also have  TSA pre-check and a Global Entry pass through the U.S. Customs and  Border Protection (CBP) Trusted Travelers Program. In other words,  security should be super easy most of the time. I also have TSA  pre-check and I can use the medical line at security if needed. Leaving  from the Austin airport was a breeze.

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Returning  home, however, was a nightmare. We were scheduled to fly out of JFK at  the Delta terminal. When we arrived to the gate, the steward at the gate  told me I had too many bags and must check them. I explained that two  of the bags were medically necessary and therefore exempt. My remaining  bag and purse fit under the seat and were included in the usual limits  for carry-ons. She proceeded to tell me that no, the medical exemption  only applies to things like breast pumps—my airway clearance and nebs  weren’t part of that exemption. She then mimed the appropriate size of  what was exempt, which was a small box, probably the size of the  packaging for said breast pump. At this point, she informed me that my  options were to check both bags or check my purse and carry-on. Hard  pass on that—not checking my purse, period. Also, maybe almost everyone  has forgotten the United-broke-my-guitar jingle from a decade ago, but I  haven’t. Checking my Frequencer (which had to get through customs and  took a month or more to arrive when I got it in the first place) wasn’t  happening either—if they broke it, I’m out the device until they replace  it and with a roughly $20K price tag, and the airline likely wouldn’t  have replaced it anyway. Fortunately, my dad and stepmom only had one  carry-on each so everyone chipped in and took one of my bags as their  own and we boarded.

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Upon  returning, I took the time to lodge a formal written complaint through  the Delta website. I read all the verbiage about medical exemptions on  their website and I explained my situation in full, noting that the  policy said xyz and none of that was honored. Many months later I  finally got a reply from Delta and they told me that it was up to each  individual gate as to whether they allow medically necessary bags to be  exempt from the carry-on luggage limits. They also noted that the gate  attendant was completely within her rights to do what she did. This was  maddening.

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This  fiasco worked out in the end, but it would have been more challenging if  I hadn’t been traveling with other people. I personally will never fly  Delta again, especially since their website said one thing but in  reality they arbitrarily apply their rules; they aren’t consistent from  gate to gate much less airport to airport.

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Traveling  with CF is always a challenge and requires a lot of planning and  organization. It’s easier now because I have permission from my CF  doctors to not do my daily nebulizer treatments now that I’m on  Trikafta. This means I don’t have to lug all that equipment around but I  can if I’m not feeling my best when I need to travel. Nowadays, I just  take my pills, my albuterol inhaler, my advair, and a whole box of  Trikafta (Boy Scout motto—be prepared!)

Sydna Marshall is 43 years old and has CF. She is the President and Managing Editor of USACFA and CF Roundtable. She lives in Austin, TX with her husband, Adam, and her two furbabies,  Cutty (12) and Mickey (1). When she’s not corralling her toddler puppy  she’s reading, working on jigsaw puzzles, cooking up buttery goodness in  the kitchen, and recentering herself on her yoga mat. Her contact  information is on page 2.

William  Coon Jr. established $20,000.00 in scholarship funds to be awarded in  $2,500.00 scholarships. Mr. Coon was both a cystic fibrosis patient and a  businessman who valued the importance of education and “paying it  forward.” Any student seeking a degree in any of the following is  welcome to apply: business, economics, communications, political  science, information, project management, finance, accounting, public  administration, or marketing. We believe that any higher education is a  strong foundation for advocacy and involvement in the CF community.

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We’re  pleased to announce the winners of the William Coon Jr. scholarship for  the 2024-2025 academic cycle: Aaron Pires, Johnny Perkins, and Maggie  Thompson.

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Aaron is  enrolled at Bryant University and starts as a freshman this fall. His  late diagnosis of CF has inspired his passion for self-advocacy. The  many hours spent hospitalized after Aaron’s diagnosis solidified his  dedication to academic resilience. Aaron is very active in the community  and has volunteered many hours of his time to multiple charitable  organizations.

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Johnny  is enrolled at Harvard College, starting this fall. He plans to earn a  Bachelor of Science in Statistics and Data Science. Johnny has a lengthy  list of academic achievements, including being a National Merit Scholar  finalist. He’s also heavily involved in the community and has raised  over $165,000 in funds for cystic fibrosis research.

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Maggie  is enrolled at Gonzaga University, with plans of becoming a realtor and  aiding people in finding the home of their dreams. She’s also enrolled  in a nearby community college to get some of her introductory coursework  completed. Maggie has been involved in various athletic programs, both  through her high school and outside of school, including assistant  coaching for the Boys 10u soccer program.

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Scholarships  are offered in the spring semester 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.

The  U.S. Adult CF Association (USACFA) is pleased to announce the  recipients of the Scholarship for the Arts, offered in memory of Helen  M. Eisenman. We offer this arts scholarship in Helen’s memory—she was a  Holocaust survivor with a passion for the arts. A talented photographer,  she eventually earned a reputation as the “Doyenne of Subtitles” within  the film industry for her skills in subtitling films in multiple  languages. She made many sacrifices over the years so that her daughter,  Andrea, who has cystic fibrosis, could live as long as possible. Helen  always encouraged Andrea to be creative, read books, appreciate museums,  and listen to music.

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In our  evaluation, we look for students who demonstrate tremendous artistic  creativity, originality and achievement, community involvement, and a  powerful understanding of how their CF—matched with their creative  endeavors—places them in a unique situation to impact the world through  their art. The scholarship is open to anyone seeking a degree from  associate to doctoral level in the creative arts. Eligible fields of  study include fine arts, computer graphics, design, music, choral,  photography, filmmaking, creative writing, and poetry—to name a few.

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We are  pleased to announce Avery Flatford and Lillian Queen as the recipients  of the Scholarship for the Arts for the 2024-2025 academic cycle. They  were each awarded $5,000. Congratulations to both!

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Avery is  currently attending the University of Tennessee. She’s pursuing her  degree in 3D Fine Arts. Avery submitted samples of various collections  of her artwork. Her “Collection of Giraffes” utilizes mixed media, 3D  wire sculpture, and digital media. She also submitted a similarly  diverse “Collection of Sunsets” focusing on landscape forms along with  her “Collection of Gingerbread Houses” created for a children’s hospital  fundraising event in Knoxville, TN. Avery plans to also minor in Art  Education to follow in the footsteps of her mother. Her big career goal  is to one day be an art museum curator with the hopes of inspiring  others in the cystic fibrosis community.

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Lillian  is currently enrolled at Queens University in Charlotte, NC. She aspires  to apply to a Ph.D. program that focuses on Victorian and Edwardian  British literature. Lillian plans to focus on narratology, particularly  in the work of James Barrie and Charles Dickens. In her words: “As a  child, I found myself drawn to these genres in times of difficulty. When  I was sitting in my hospital bed, unable to sleep because the plastic  mattress made me sweat and the PICC line in my arm was itching, books  like The Lord of the Rings, His Dark Materials, and The Wind in the  Willows refreshed my spirit more effectively than anything else.”

Scholarships  are offered in the spring semester 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.

The  Stenzel Scholarship was established in 2023 in memory of two amazing  women with CF, Isa Stenzel-Byrnes and Ana Stenzel. Isa was a licensed  social worker and had a Master of Public Health degree. She imparted her  wisdom to CF Roundtable readers for 17 years in her CF Roundtable  column, “Spirit Medicine.” Ana Stenzel was Isa’s twin sister. Ana was a  genetic counselor at Stanford hospital for 16 years. Isa and Ana  dedicated their lives to helping others. They provided education, hope  and comfort to thousands of people throughout their lives. They showed  the world that people with CF could find meaning in their lives by  making a difference in the lives of others. The Ana and Isa Stenzel  Scholarship is awarded once annually to a person with cystic fibrosis  enrolled in a related degree program at a college or university in the  United States during the academic year covered by the award. Eligible  degree programs include health science, social work, mental health  science, genetic counseling, or environmental science.

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We’re  pleased to announce the winner of The Stenzel Scholarship for the  inaugural 2024-2045 academic cycle: Jenny Livingston. In addition to her  Associate of Science from Snow College, Jenny also has a Bachelor of  Science in Psychology from Utah State University, where she’s currently  seeking her Master of Social Work. While attending school, Jenny also  works at Gunnison Valley Home Health and Hospice as part of a Field  Practicum where she provides compassionate support to end-of-life  patients and their families in addition to counseling and grief and  bereavement support. Jenny also connects clients to resources and  services, facilitates end-of-life wishes in the care process, and  provides case management support. She is also very active within her  local CF community and has served on both the Utah Adult CF Advisory  Board since 2019 and the Utah-Idaho CFF Chapter Board for several years.  Congratulations, Jenny!

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Scholarships  are offered in the spring semester 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.

Effectiveness  of Lumacaftor/Ivacaftor Initiation in Children with Cystic Fibrosis  Aged 2 Through 5 Years on Disease Progression: Interim Results From an  Ongoing Registry-Based Study.

Lumacaftor/ivacaftor  (LUM/IVA) has been shown to be safe and efficacious in people with  cystic fibrosis (CF) ≥1 year of age. To assess the impact of early  LUM/IVA initiation on CF disease progression, a 6-year observational  study leveraging data from existing CF patient registries is being  conducted in children with CF homozygous for F508del who were aged 2  through 5 years at treatment initiation. Presented here are the interim  results from this study focusing on data from the European CF Society  Patient Registry (ECFSPR). The LUM/IVA cohort matched to the F/MF  concurrent comparator cohort had 681 children and the LUM/IVA cohort  matched to the F/F concurrent comparator cohort had 183 children.  LUM/IVA cohorts had increases in body mass index percentiles relative to  the matched F/MF and F/F concurrent comparator cohorts. Increases in  height and weight percentiles were also observed in the LUM/IVA cohort  relative to the F/MF and F/F concurrent comparator cohorts. Reductions  in pulmonary exacerbations and hospitalizations relative to baseline and  the F/F concurrent comparator cohort were seen in 2021. In conclusion,  this interim analysis showed favorable trends in clinical outcomes,  including growth parameters, pulmonary exacerbations, and  hospitalizations, suggesting an early beneficial effect of LUM/IVA  treatment in children aged 2 through 5 years at treatment initiation.

https://tinyurl.com/2tyymjy9

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Intestinal  Bacteroides Modulates Inflammation, Systemic Cytokines, and Microbial  Ecology Via Propionate in a Mouse Model of Cystic Fibrosis.

Persons  with cystic fibrosis (CF), starting in early life, show intestinal  microbiome dysbiosis characterized in part by a decreased relative  abundance of the genus Bacteroides. Bacteroides is a major producer of  the intestinal short chain fatty acid propionate. Researchers  demonstrate here that cystic fibrosis transmembrane conductance  regulator-defective (CFTR-/-) Caco-2 intestinal epithelial cells are  responsive to the anti-inflammatory effects of propionate. Furthermore,  Bacteroides isolates inhibit the IL-1β-induced inflammatory response of  CFTR-/- Caco-2 intestinal epithelial cells and do so in a  propionate-dependent manner. The introduction of  Bacteroides-supplemented stool from infants with cystic fibrosis into  the gut of CftrF508del mice results in higher propionate in the stool as  well as the reduction in several systemic pro-inflammatory cytokines.  Bacteroides supplementation also reduced the fecal relative abundance of  Escherichia coli, indicating a potential interaction between these two  microbes, consistent with previous clinical studies. For a Bacteroides  propionate mutant in the mouse model, pro-inflammatory cytokine KC is  higher in the airway and serum compared with the wild-type (WT) strain,  with no significant difference in the absolute abundance of these two  strains. Taken together, the data indicate the potential multiple roles  of Bacteroides-derived propionate in the modulation of systemic and  airway inflammation and mediating the intestinal ecology of infants and  children with CF. The roles of Bacteroides and the propionate it  produces may help explain the observed gut-lung axis in CF and could  guide the development of probiotics to mitigate systemic and airway  inflammation for persons with CF.

https://tinyurl.com/3j3t695t

The Impact of Antimicrobial Resistance in Cystic Fibrosis.

The  phenomenon of antimicrobial resistance (AMR) is a critical global health  challenge, with prospects indicating its potential to become the  leading cause of death worldwide in the coming years. Individuals with  pre-existing conditions, such as neoplastic disease undergoing  chemotherapy, those on immunosuppressive therapy, and individuals with  rare diseases like cystic fibrosis (CF), face heightened challenges due  to AMR. This review delves into the complex relationship between AMR and  climate dynamics, focusing on the unique challenges faced by  individuals with CF. It discusses the methods employed to measure AMR,  its global impact on antibiotic resistance, and the specific microbial  communities present in the CF airway. The review also explores the  intricacies of antimicrobial resistance within the context of cystic  fibrosis, emphasizing the urgent need for research in this field. In the  context of cystic fibrosis (CF), AMR poses a persistent challenge.  Advances in molecular technologies, particularly whole-genome sequencing  (WGS) from next-generation sequencing (NGS) platforms, offer new  prospects for genotypic recognition of bacterial resistance profiles.  This method provides greater discriminatory ability and efficiency,  especially in detecting acquired resistance genes and chromosomal  mutations in polymicrobial communities within the airways of PwCF.  However, research gaps still exist, requiring further elucidation of  factors such as virulence, mechanisms of AMR, systematic treatment  guidelines, and the need for more studies in children to address  pharmacokinetic differences. The complexity of interactions between  different species and the different facets of the respiratory  pathophysiology of CF represent challenges for the development of new  effective prevention and therapeutic tools

https://tinyurl.com/2rupf5h6

SPLUNC1 as a Biomarker of Pulmonary Exacerbations in Children With Cystic Fibrosis.

Short  palate, lung, and nasal epithelium clone 1 (SPLUNC1) is an innate  defense protein that acts as an antimicrobial agent and regulates airway  surface liquid volume through inhibition of the epithelial sodium  channel (ENaC). SPLUNC1 levels were found to be reduced in airway  secretions of adults with cystic fibrosis (CF). The potential of SPLUNC1  as a biomarker in children with CF is unknown. Researchers quantified  SPLUNC1, interleukin-8 (IL-8) and neutrophil elastase (NE) in sputum of  CF children treated with either intravenous antibiotics or oral  antibiotics for a pulmonary exacerbation (PEx)s, and in participants of a  prospective cohort of CF children with preserved lung function on  spirometry, followed over a period of two years. Sputum SPLUNC1 levels  were significantly lower before compared to after intravenous and oral  antibiotic therapy for PEx. In the longitudinal cohort, SPLUNC1 levels  were found to be decreased at PEx visits compared to both previous and  subsequent stable visits. Higher SPLUNC1 levels at stable visits were  associated with longer PEx-free time. SPLUNC1 at PEx visits did not  correlate with IL-8 or NE levels in sputum or forced expiratory volume  in one second (FEV1) but did correlate with the lung clearance index. In  conclusion, SPLUNC1 demonstrates promising clinometric properties as a  biomarker of PEx in children with CF.

https://tinyurl.com/56757pjk

The Changing Landscape of Treatment for Cystic Fibrosis Related Diabetes.

Patients  with Cystic Fibrosis related diabetes [CFRD] are treated with insulin  and high calorie diets to maintain body mass. The combined CFTR  modulator elexacaftor/tezacaftor/ivacaftor [ETI] decreases pulmonary  exacerbations and improves nutritional status. In this study researchers  reviewed the effects of ETI on BMI, HbA1c and diabetes regimen in  patients with CFRD over a period of three years. Twenty-seven patients  with CFRD (15 men/12 women), aged 30.6 ± 11.5,  years, BMI 22.4 ± 4.0  kg/m2, were included. Fifteen patients had low BMI and 12 patients had  at target BMI. Patients with low BMI had an increase in their BMI from  19.5 ± 1.7 to 21.4 ± 2.2 kg/m2 at one year, and 21.8 ± 1.8 kg/m2 at  three years after ETI initiation. Four patients in the low BMI group had  achieved normal BMI by the end of study follow up. There was no change  in weight in the at target BMI group. HbA1c and basal insulin  requirements did not change in either group. Five patients started  non-insulin therapies. In conclusion, BMI increased after ETI therapy in  CFRD patients with low BMI, but not in those with at target BMI. The  use of non-insulin therapies is increasing in CFRD and should be  evaluated in future studies.

https://tinyurl.com/je4p44mn

Exploring Perceptions of and Decision-Making About CFTR Modulators.

Cystic  fibrosis (CF) treatment has increasingly focused on highly effective  modulators. Despite measurable benefits of modulators, there is little  guidance for CF care team members on providing education and support to  patients regarding initiation of these therapies. These researchers  aimed to explore patient, caregiver, and clinician perceptions of  modulators and influences on decisions about starting cystic fibrosis  transmembrane regulator (CFTR) modulators. They conducted semistructured  interviews with CF clinicians, adults with CF, and caregivers of  children with CF. They also reviewed audio recordings and coded  responses to identify central themes. The researchers interviewed 8 CF  clinicians, 9 adults with CF, and 11 caregivers of children with CF.  Themes centered on emotional responses to modulator availability,  influences on decision-making, concerns about side effects, impact of  modulators on planning for the future, the benefits of the  multidisciplinary CF care team in supporting treatment decisions, and  the unique needs of people with CF who are not eligible for modulators.  Clinicians described changes in conversations about modulators since the  approval of elexacaftor/tezacaftor/ivacaftor, specifically greater  willingness to prescribe with less nuanced conversations with patients  and/or caregivers regarding their use. In conclusion, based on  perspectives and experiences of CF clinicians, adults with CF, and  caregivers of children with CF, we suggest clinicians approach  conversations about CFTR modulators thoughtfully and thoroughly,  utilizing the multidisciplinary model of CF care in exploring patient  and caregiver emotions while filling in knowledge gaps, asking about  treatment goals beyond potential clinical benefit, and having  compassionate conversations with those who are ineligible for  modulators.

https://tinyurl.com/37ahdxp5

In Vitro  Stimulation with Nontuberculous Mycobacteria Induced a Stronger  Cytokine Response in Leukocytes Isolated From Individuals with Latent  Tuberculosis Compared to Those Isolated From Active Tuberculosis or  Cystic Fibrosis Patients.

Mycobacterium  tuberculosis and opportunistic environmental non-tuberculous  mycobacteria (NTM) can cause severe infection. Why latent tuberculosis  infection advances to active disease, and why some individuals with CF  develop pulmonary infections with NTM is still poorly understood. The  aim of this study was to investigate the effector function of peripheral  blood mononuclear cells (PBMC) from individuals with active or latent  tuberculosis, individuals with CF with or without pulmonary  NTM-infection and healthy controls, by measuring cytokine response to in  vitro stimulation with different species of NTMs. The cytokine  concentrations of IL-17A, IL-22, IL-23, IL-10, IL12p70 and IFN-γ were  measured in PBMC-culture supernatants after stimulation with NTMs. PBMCs  from individuals with latent tuberculosis infection showed strong  IL-17A, IL-22, and IFN-γ responses compared to individuals with active  tuberculosis or CF. IL-10 production was low in both tuberculosis groups  compared to the CF groups and controls. This study suggests that IL-17A  and IL-22 might be important to keep tuberculosis in a latent phase and  that individuals with CF with an ongoing NTM infection seem to have a  low cytokine response.

https://tinyurl.com/55p44sy9

Galectin-3 Levels in Children With Cystic Fibrosis.

Cystic  fibrosis (CF) is a multisystemic disease in which airway obstruction,  infection, and inflammation play a critical role in the pathogenesis and  progression of CF lung disease. The carbohydrate-binding protein  Galectin-3 is increased in several inflammatory and fibrotic diseases  and has recently been forwarded as a biomarker in these diseases. These  researchers aimed to define the role of serum Galectin-3 in children  with CF by comparison with healthy subjects. This is a cross-sectional,  case–control study. 143 CF and 30 healthy subjects were enrolled in the  study. Peripheral blood and sputum concentrations of Galectins-3,  interleukin (IL)-17A, IL-8, and neutrophil elastase (NE) were determined  with commercial ELISA kits. There was no significant difference between  the groups in age and gender. Serum Galectin-3 and NE concentrations  were higher in the patient group than in healthy controls. There were no  significant differences between groups according to IL-17A and IL-8  concentrations. Serum Galectin-3 was correlated with age and body mass  index (BMI) in children with CF. Sputum Galectin-3 levels are negatively  correlated with percent predictive forced expiratory volume in 1 s  (FEV1), FEV1 z-score,  percent predicted forced vital capacity (FVC),  and FVC z-score. In conclusion, the study shows that serum Galectin-3  levels increased in clinically stable CF patients, and serum Galectin-3  response may depend on age, gender, and BMI. The sputum Galectin-3 was  found to be negatively correlated with patients’ lung functions

https://tinyurl.com/mtjuewzk

Alterations  in the Fecal Microbiota in Patients With Advanced Cystic Fibrosis Liver  Disease After 6 Months of Elexacaftor/Tezacaftor/Ivacaftor.

Cystic  fibrosis associated liver disease (CFLD) carries a significant disease  burden with no effective preventive therapies. According to the  gut-liver axis hypothesis for CFLD pathogenesis, dysbiosis and increased  intestinal inflammation and permeability permit pathogenic bacterial  translocation into the portal circulation, leading to hepatic  inflammation and fibrosis. Evaluating the effect of CFTR modulation with  elexacaftor/tezacaftor/ivacaftor (ETI) may help determine the role of  CFTR in CFLD and increase understanding of CFLD pathogenesis, which is  critical for developing therapies. These researchers  aimed to  characterize the fecal microbiota in participants with CF with and  without advanced CFLD (aCFLD) before and after ETI. This is an ancillary  analysis of stool samples from participants ages ≥12 y/o enrolled in  PROMISE. Included participants had aCFLD (cirrhosis with or without  portal hypertension, or non-cirrhotic portal hypertension) or CF without  liver disease (CFnoLD). Fecal microbiota were defined by shotgun  metagenomic sequencing at baseline and 1 and 6 months post-ETI. They  analyzed 93 samples from 34 participants. Compared to CFnoLD, aCFLD had  significantly higher baseline relative abundances of potential pathogens  Streptococcus salivarius and Vella paeillonrvula. Four of 11 aCFLD  participants had an initially abnormal fecal calprotectin that  normalized 6 months post-ETI, correlating with a significant decrease in  S. salivarius and a trend towards decreasing V. parvula. In conclusion,  these results support an association between dysbiosis and intestinal  inflammation in CFLD with improvements in both post-ETI, lending further  support to the gut-liver axis in aCFLD.

https://tinyurl.com/mrhxkbsb

Discovery  of GLPG2737, a Potent Type 2 Corrector of CFTR for the Treatment of  Cystic Fibrosis in Combination With a Potentiator and a Type 1  Co-Corrector.

CF is  caused by mutations in the CF transmembrane conductance regulator (CFTR)  protein. This epithelial anion channel regulates the active transport  of chloride and bicarbonate ions across membranes. Mutations result in  reduced surface expression of CFTR channels with impaired functionality.  Correctors are small molecules that support the trafficking of CFTR to  increase its membrane expression. Such correctors can have different  mechanisms of action. Combinations may result in a further improved  therapeutic benefit. Researchers describe the identification and  optimization of a new pyrazolol3,4-bl pyridine-6-carboxylic acid series  with high potency and efficacy in rescuing CFTR from the cell surface.  Investigations showed that carboxylic acid group replacement with  acylsulfonamides and acylsulfonylureas improved ADMET and PK properties,  leading to the discovery of the structurally novel co-corrector  GLPG2737. The addition of GLPG2737 to the combination of the potentiator  GLPG1837 and C1 corrector 4 led to an 8-fold increase in the F508del  CFTR activity.

https://tinyurl.com/y9uxvse9

Early Onset of Abnormal Glucose Tolerance in Patients With Cystic Fibrosis: A Systematic Review and Meta-Analysis.

Despite  translational evidences suggesting that cystic fibrosis-related abnormal  glucose tolerance (CF-related AGT) may begin early in life and is known  to be associated with increased morbidity and mortality, current  guidelines recommend screening for AGT only from 10 years of age, thus  missing the opportunity for early detection and intervention. A  systematic review and meta-analysis was conducted on studies that  reported data on the prevalence of AGT or its subtypes in CF  populations. Pooled proportions, risk, and odds ratios with 95 %  confidence intervals (CI) were calculated. One-stage dose-response  random-effect meta-analysis was used to assess the effect of age on  CF-related diabetes (CFRD). The quantitative analysis included 457  studies and data from 520,544 patients. Every third child with CF  (chwCF) and every second adult with CF (awCF) were affected by AGT. Even  in the 5–10 years of age subgroup, the proportion of AGT was 0.42. The  prevalence of prediabetes remained unchanged, whereas the proportion of  CFRD increased with age. In conclusion, CF-related AGT is common under  10 years of age. This study suggests considering earlier AGT screening,  starting from 5 years of age. This highlights the imperative for  additional research for guideline adjustments and provides the  opportunity for early intervention.

https://tinyurl.com/yc397h7e

Airway Commensal Bacteria in Cystic Fibrosis Inhibit The Growth Of P. aeruginosa Via a Released Metabolite.

In CF,  Pseudomonas aeruginosa infection plays a critical role in disease  progression. Although multiple studies suggest that airway commensals  might be able to interfere with pathogenic bacteria, the role of the  distinct commensals in the polymicrobial lung infections is largely  unknown. In this study, researchers aimed to identify airway commensal  bacteria that may inhibit the growth of P. aeruginosa. Through a  screening study with more than 80 CF commensal strains across 21  species, more than 30 commensal strains from various species have been  identified to be able to inhibit the growth of P. aeruginosa. The  underlying mechanisms were investigated via genomic, metabolic and  functional analysis, revealing that the inhibitory commensals may affect  the growth of P. aeruginosa by releasing a large amount of acetic acid.  The data provide information about the distinct roles of airway  commensals and provide insights into novel strategies for controlling  airway infections.

https://tinyurl.com/ycypvanb

Symptoms and Quality of Life in Adults With Cystic Fibrosis: A Cross-Sectional Analysis of the InSPIRe:CF Trial.

People  living with cystic fibrosis experience a high symptom burden. Due to the  changing landscape of CF in the era of modulator therapy, these  researchers sought to examine the epidemiology of symptoms and their  association with quality of life, to help CF clinicians improve symptom  screening in clinic. Using baseline data from a trial of specialist  palliative care in adults with CF, they examined symptom prevalence,  distress, and association with quality of life. Among 262 participants,  median age was 33, and 78% were on modulator therapy. The most common  symptoms were lack of energy and cough, whereas the most distressing  were difficulty sleeping and pain. The symptoms that impaired quality of  life the most were extrapulmonary: lack of energy, feeling sad and  worrying. In conclusion, the symptoms that were associated with the  lowest quality of life were extrapulmonary. CF clinicians may consider  screening for common symptoms that affect quality of life the most (lack  of energy, worrying, difficulty sleeping, feeling irritable, pain, and  shortness of breath). These symptoms may identify people living with CF  who are most at risk for a decreased quality of life and may benefit  from additional support.

https://tinyurl.com/ye25ea63

Elexacaftor/Tezacaftor/Ivacaftor Improves Nasal Nitric Oxide in Patients With Cystic Fibrosis.

In  health, nitric oxide (NO) shows high concentrations in the upper  airways, while nasal NO (nNO) is significantly lower in patients with  sinonasal inflammation, such as people with cystic fibrosis (PwCF). In  PwCF treated with elexacaftor/tezacaftor/ivacaftor (ETI; PwCF-ETI),  clinical improvement of sinonasal symptoms and inflammation was  observed. Researchers therefore hypothesized that ETI may increase nNO  in PwCF. 25 PwCF-ETI underwent nNO measurement at baseline and after 3  to 24 months of ETI treatment. NNO was measured using velum closure (VC)  techniques in cooperative patients and tidal breathing (TB) for all  patients. As controls, 7 CF patients not eligible for ETI (PwCF-non ETI)  and 32 healthy controls (HC) were also repeatedly investigated. Results  showed, in PwCF-ETI, sinonasal symptoms, lung function parameters and  sweat chloride levels improved from baseline to follow-up whereas there  was no change in PwCF-non ETI and HC. NNO increased from a median value  at baseline to follow-up.. At follow-up, PwCF-ETI reached nNO values in  the normal range. In PwCF-non ETI as well as HC, nNO did not change  between baseline and follow-up. In conclusion,  PwCF-ETI, the nNO values  significantly increased after several months of ETI treatment in  comparison to baseline and reached values in the normal range. This  suggests that nNO is a potential non-invasive biomarker to examine  sinonasal inflammatory disease in PwCF and supports the observation of  clinical improvement in these patients.

https://tinyurl.com/59b3tjwh

In Vitro Modulator Responsiveness of 655 CFTR Variants Found in People With Cystic Fibrosis

In 2017,  the US Food and Drug Administration initiated expansion of drug labels  for the treatment of CF to include CF transmembrane conductance  regulator (CFTR) gene variants based on in vitro functional studies.  This study aims to identify CFTR variants that result in increased  chloride (Cl−) transport function by the CFTR protein after treatment  with the CFTR modulator combination elexacaftor/tezacaftor/ivacaftor  (ELX/TEZ/IVA). These data may benefit people with CF (pwCF) who are not  currently eligible for modulator therapies. Plasmid DNA encoding 655  CFTR variants and wild-type (WT) CFTR were transfected into Fisher Rat  Thyroid cells that do not natively express CFTR. After 24 h of  incubation with control or TEZ and ELX, and acute addition of IVA, CFTR  function was assessed using the transepithelial current clamp  conductance assay. Each variant’s forskolin/cAMP-induced baseline Cl−  transport activity, responsiveness to IVA alone, and responsiveness to  the TEZ/ELX/IVA combination were measured in three different  laboratories. Western blots were conducted to evaluate CFTR protein  maturation and complement the functional data. 253 variants not  currently approved for CFTR modulator therapy showed low baseline  activity. For 152 of these variants, treatment with ELX/TEZ/IVA improved  the Cl− transport activity by ≥10 % of normal CFTR function, which is  suggestive of clinical benefit. ELX/TEZ/IVA increased CFTR function by  ≥10 percentage points for an additional 140 unapproved variants with ≥10  % but <50 % of normal CFTR function at baseline. These findings  significantly expand the number of rare CFTR variants for which  ELX/TEZ/IVA treatment should result in clinical benefit.

https://tinyurl.com/ra9xkm54

PREFUL  MRI for Monitoring Perfusion and Ventilation Changes after  Elexacaftor-Tezacaftor-Ivacaftor Therapy for Cystic Fibrosis: A  Feasibility Study.

This  feasibility study demonstrates phase-resolved functional lung, or  PREFUL, MRI as an adequate tool to monitor ventilation and perfusion  improvement in response to therapy in individuals with cystic fibrosis.  The purpose of this study is to assess the feasibility of monitoring the  effects of elexacaftor-tezacaftor-ivacaftor (ETI) therapy on lung  ventilation and perfusion in people with cystic fibrosis (CF), using  phase-resolved functional lung (PREFUL) MRI. This secondary analysis of a  multicenter prospective study was carried out between August 2020 and  March 2021 and included participants 12 years or older with CF who  underwent PREFUL MRI, spirometry, sweat chloride test, and lung  clearance index assessment before and 8–16 weeks after ETI therapy. For  PREFUL-derived ventilation and perfusion parameter extraction,  two-dimensional coronal dynamic gradient-echo MR images were evaluated  with an automated quantitative pipeline. T1- and T2-weighted MR images  and PREFUL perfusion maps were visually assessed for semiquantitative  Eichinger scores. Wilcoxon signed rank test compared clinical parameters  and PREFUL values before and after ETI therapy. Correlation of  parameters was calculated as Spearman ρ correlation coefficient.  Twenty-three participants were included. Quantitative PREFUL parameters,  Eichinger score, and clinical parameters showed significant improvement  after ETI therapy. Ventilation defect percentage of regional  ventilation decreased from 18% and perfusion defect percentage from 26%.  Areas of matching normal (healthy) ventilation and perfusion increased  from 52% to 73%. Visually assessed perfusion scores did not correlate  with PREFUL perfusion nor with ventilation-perfusion match values.

In  conclusion the study demonstrates the feasibility of PREFUL MRI for  semiautomated quantitative assessment of perfusion and ventilation  changes in response to ETI therapy in people with CF.

https://tinyurl.com/3x4e46pb

Akt-driven TGF-β and DKK1 Secretion Impairs F508del CF Airway Epithelium Polarity.

Epithelial  polarity is fundamental in maintaining barrier integrity and tissue  protection. In cystic fibrosis (CF), apicobasal polarity of the airway  epithelium is altered, resulting in increased apical fibronectin  deposition and enhanced susceptibility to bacterial infections. Here,  researchers evaluated the effect of highly effective modulator treatment  (HEMT) on fibronectin apical deposition and investigated the  intracellular mechanisms triggering the defect in polarity of the CF  airway epithelium. To this end, primary cultures of CF (F508del variant)  human airway epithelial cells (HAECs) and a HAEC line, Calu-3,  knocked-down (KD) for CFTR (CFTR KD) were compared to control  counterparts, grown at an air-liquid interface (ALI). They show that  CFTR mutation in primary HAECs and CFTR KD cells promote the  overexpression and over-secretion of TGF-β1 and DKK1 when cultured at  ALI. These dynamic changes result in hyperactivation of the TGF-β  pathway and inhibition of the Wnt pathway through degradation of  β-catenin leading to imbalanced proliferation and polarization. The  abnormal interplay between TGF-β and Wnt signaling pathways is  reinforced by aberrant Akt signaling. Pharmacological manipulation of  TGF-β, Wnt, and Akt pathways restored polarization of the F508del CF  epithelium, a correction that was not achieved by HEMT. This data sheds  new insights into the signaling pathways that fine-tune apicobasal  polarization in primary airway epithelial cells and may provide an  explanation to the mitigated efficacy of HEMT on lung infection in  people with CF.

https://tinyurl.com/2wzjx676

Lung  Clearance Index (LCI2.5) Changes After Initiation of  Elexacaftor/Tezacaftor/Ivacaftor in Children With Cystic Fibrosis Aged  Between 6 and 11 Years: The “Real-World” Differs From Trial Data.

Elexacaftor  in combination with Tezacaftor and Ivacaftor (ETI) became licensed in  the United Kingdom in early 2022 for children aged 6–11 years with  cystic fibrosis (CF) and an eligible mutation. Many in this age group  have excellent prior lung health making quantitative measurement of  benefit challenging. Clinical trials purport that lung clearance index  (LCI2.5) measurement is most suitable for this purpose. This study aimed  to understand the clinical utility of LCI2.5 in detecting change after  commencing ETI in the real world.

Baseline  anthropometric data were collected along with spirometry (FEV1), forced  vital capacityFV and LCI2.5 measures in children aged 6–11 years with  CF before starting ETI. Measures were repeated after a mean of 8.2  months of ETI treatment. The primary endpoint was a change in LCI2.5,  with secondary endpoints including change in FEV1 and change in body  mass index (BMI) also reported. Twelve children were studied. The study  population had a mean LCI2.5 of 7.01 and FEV1 of 96 %predicted at  baseline. Mean changes in LCI2.5 and BMI were observed, along with  changes in FEV1 of +3.1 %predicted. In conclusion, real-world changes in  LCI2.5 are different to those reported in clinical trials. Lower  baseline LCI2.5 as a result of prior modulator exposure, high baseline  lung health, and new LCI2.5 software analyzes all contribute to lower  LCI2.5 values being recorded in the real world of children with CF.

https://tinyurl.com/5kwdfrad

Impact of CFTR Modulator Therapy on Body Composition as Assessed by Thoracic Computed Tomography: A Follow-up Study.

Treatment  with cystic fibrosis transmembrane conductance regulator (CFTR)  modulators in individuals with cystic fibrosis (CF) has brought a  significant change in forced expiratory volume in 1 second (FEV1) and  clinical parameters. However, it also results in weight gain. The aim of  this study is to evaluate the effect of CFTR modulator treatment on  body composition, measured by computed tomography (CT). Adult subjects  with CF under follow-up at La Princesa University Hospital were  recruited. All of them were on elexacaftor–tezacaftor–ivacaftor  (ELX/TEZ/IVA) treatment. Body composition analysis was conducted using  CT scans and an open-source software. The results were then compared  with bioimpedance estimations, as well as other clinical and spirometry  data. The sample consisted of 26 adult subjects. The fat mass  compartments on CT scans correlated with similar compartments on  bioimpedance, and normal-density muscle mass exhibited a strong  correlation with phase angle. Higher levels of very low-density muscle  prior to treatment were associated with lower final FEV1 and less  improvement in FEV1 after therapy. An increase in total body area,  driven by increases in total fat mass, subcutaneous fat, visceral fat,  and intermuscular fat was observed. The only muscle compartment that  showed an increase after treatment was very low-density muscle. In  conclusion, CT scans represent an opportunity to assess body composition  on CF. Combination treatment with CFTR modulators, leads to an  improvement in FEV1 and to an increase in body mass in all compartments  primarily at the expense of fat mass.

https://tinyurl.com/pddsd32z

Safety and Efficacy of Ivacaftor in Infants Aged 1 to Less Than 4 Months With Cystic Fibrosis.

Ivacaftor  (IVA) has been shown to be safe and efficacious in children aged ≥4  months with cystic fibrosis (CF) and CFTR gating variants. Researchers  evaluated safety, pharmacokinetics (PK), and efficacy of IVA in a small  cohort of infants aged 1 to <4 months with CF. In this phase 3,  open-label study, infants 1 to <4 months with CF and an  IVA-responsive CFTR variant received an initial low dose of IVA based on  age and weight. Because IVA is a sensitive CYP3A substrate and CYP3A  maturation is uncertain in infants, doses were adjusted at day 15 to  better match median adult exposures based on individual PK measurements  taken on day 4. Primary endpoints were safety and PK measurements. Seven  infants received ≥1 dose of IVA. Six infants had doses adjusted at day  15 and one infant did not require dose adjustment; subsequent PK  analyses showed mean trough concentrations for IVA and metabolites were  within range of prior clinical experience. Four infants had adverse  events (AEs); no serious AEs were noted. One infant discontinued study  drug due to a non-serious AE of elevated alanine aminotransferase >8x  the upper limit of normal. Mean sweat chloride concentration decreased  through week 24. Improvements in biomarkers of pancreatic function and  intestinal inflammation, as well as growth parameters, were observed. In  this small, open-label study, IVA dosing in infants achieved exposures  previously shown to be safe and efficacious. Because PK was predictable,  a dosing regimen based on age and weight is proposed. IVA was generally  safe and well tolerated, and led to improvements in CFTR function,  markers of pancreatic function and intestinal inflammation, and growth  parameters, supporting use in infants as young as 1 month of age.

https://tinyurl.com/bdfbjr62

Monoclonal Antibodies Derived From B Cells in Subjects With Cystic Fibrosis Reduce Pseudomonas Aeruginosa Burden In Mice.

Pseudomonas  aeruginosa (PA) is an opportunistic, frequently multidrug-resistant  pathogen that can cause severe infections in hospitalized patients.  Antibodies against the PA virulence factor, PcrV, protect from death and  disease in a variety of animal models. However, clinical trials of  PcrV-binding antibody-based products have thus far failed to demonstrate  benefit. Prior candidates were derivations of antibodies identified  using protein-immunized animal systems and required extensive  engineering to optimize binding and/or reduce immunogenicity. Of note,  PA infections are common in people with cystic fibrosis (pwCF), who are  generally believed to mount normal adaptive immune responses. Here  researchers utilized a tetramer reagent to detect and isolate  PcrV-specific B cells in pwCF and, via single-cell sorting and  paired-chain sequencing, identified the B cell receptor (BCR) variable  region sequences that confer PcrV-specificity. They derived multiple  high affinity anti-PcrV monoclonal antibodies (mAbs) from PcrV-specific B  cells across 3 donors, including mAbs that exhibit potent anti-PA  activity in a murine pneumonia model. This robust strategy for mAb  discovery expands what is known about PA-specific B cells in pwCF and  yields novel mAbs with potential for future clinical use.

https://tinyurl.com/n4vxxv4v

A Role for the Stringent Response in Ciprofloxacin Resistance in Pseudomonas Aeruginosa.

Pseudomonas  aeruginosa is a major cause of nosocomial infections and the leading  cause of chronic lung infections in cystic fibrosis and chronic  obstructive pulmonary disease patients. Antibiotic treatment remains  challenging because P. aeruginosa is resistant to high concentrations of  antibiotics and has a remarkable ability to acquire mutations  conferring resistance to multiple groups of antimicrobial agents. Here  researchers report that when P. aeruginosa is plated on ciprofloxacin  (cipro) plates, the majority of cipro-resistant (ciproR) colonies  observed at and after 48 h of incubation carry mutations in genes  related to the Stringent Response (SR). Mutations in one of the major SR  components, spoT, were present in approximately 40% of the ciproR  isolates. Compared to the wild-type strain, most of these isolates had  decreased growth rate, longer lag phase and altered intracellular ppGpp  content. Also, 75% of all sequenced mutations were insertions and  deletions, with short deletions being the most frequently occurring  mutation type. These researchers present evidence that most of the  observed mutations are induced on the selective plates in a  subpopulation of cells that are not instantly killed by cipro. These  results suggests that the SR may be an important contributor to  antibiotic resistance acquisition in P. aeruginosa.

https://tinyurl.com/3dytfsrz s

Aimee  Lecointre is 39 and has CF. She lives in Salt Lake City, UT. She loves  reading, cooking, writing, and spending time with her husband.