Summer 2024

Pearls of wisdom:
Things to Do in Denver When You’re Dead—Lessons Learned at 5,280 Feet Above
Sea Level

Adventures Abound:
The Expansion of You

Culinary Corner:
Easy Egg Salad

Transplant Talk:
Traveling to New York City

salty parenting:
Cape Cod to Quebec City,
June 2024

in the spotlight:
Marina Forstmann Day

Focus topic:
CF and Travel

Air Travel With
a New Insulin Pump

Why I Haven’t Traveled as Much as
I Wish and Small Changes I am Making to
Try to Change That

Medical Exemptions for Carry-on Luggage:
A Cautionary Tale

WIlliam Coon Jr. Scholarship winners

Scholarship for the arts winners

Navigating Healthcare and Hospitals for
CF Travelers

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.

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.

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.

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.

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.

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.

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.

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.

Corcoran

Ask The Attorney:
Understanding Medicare Basics for People With CF, Part 1

By Beth Sufian, J.D.

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.

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.

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.

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.

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.

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.

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.

attorney

Pearls of wisdom:
Things to Do in Denver When You’re Dead—Lessons Learned
at 5,280 Feet Above
Sea Level

By Xan Nowakowski,
Ph.D., M.P.H.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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?

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.

pearls

Adventures abound:
The Expansion of You

By Marcus Miller

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.

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.

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.

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.

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.

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.

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.

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.

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.

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?

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.

Adventures

Culinary Corner:
Easy Egg Salad

By Maggie Williamson

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!

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.

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

Culinary

Transplant Talk:
Traveling to
New York City

By colleen Adamson

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.

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.

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.

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.

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.

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.

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.

transplant talk

salty

salty parenting:
Cape Cod to Quebec City, June 2024

By Katie Lockwood

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.

• 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.

• 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.

• 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!

• 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.

• 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.

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.

• Double check all the expiration dates on said paperwork.

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

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

• Count the clothes and take the minimum needed.

• 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.

• 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.

• 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.

• 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.

• Pack Tylenol, just in case.

• Grab the toothbrushes and toothpaste.

• Get the diapers and wet wipes.

• Can’t forget the Desitin!

• Oh! The bottles and sippy cups too.

• Sunscreen. Need bathing suits too.

• Toys for car rides and picture books.

• 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.

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.

In the Spotlight:
With Marina Forstmann Day

By andrea eisenman

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.

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.

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.

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.”

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.

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.

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.

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.”

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!

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.

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.

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.

ITS Day

Eisenman focus

Air Travel With a
New Insulin Pump

By Andrea Eisenman

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.

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.

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.

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.

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.

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.

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.

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.

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.

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?

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.

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.

armstrong

Why I Haven’t Traveled as Much as I Wish and Small Changes I am Making to Try to Change That

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.

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.

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.

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.

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.

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.

By Brian Armstrong

marshall

Medical Exemptions
for Carry-on Luggage:
A Cautionary Tale

By sydna marshall

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.

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.

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.

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.

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.

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.

coon scholarship

Announcing the Recipients of The William Coon Jr. Business Scholarship

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.

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.

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.

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.

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.

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.

Announcing The
Recipients Of The Scholarship for the Arts

Arts scholarship

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.

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.

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!

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.

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.”

Stenzel scholarship

Announcing the Recipient of
The Stenzel Scholarship

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.

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!

research roundup

round up

Compiled by Aimee Lecointre

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

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.

summer 2024

Focus topic: CF and Travel

Navigating Healthcare and Hospitals for CF Travelers

Ask The Attorney: Understanding Medicare Basics for People With CF, Part 1

Pearls of wisdom: Things to Do in Denver When You’re Dead—Lessons Learned at 5,280 Feet Above Sea Level

Adventures abound: The Expansion of You

Culinary Corner: Easy Egg Salad

Transplant Talk: Traveling to New York City

salty parenting: Cape Cod to Quebec City, June 2024

In the Spotlight: With Marina Forstmann Day

Air Travel With a New Insulin Pump

Why I Haven’t Traveled as Much as I Wish and Small Changes I am Making to Try to Change That

Medical Exemptions for Carry-on Luggage: A Cautionary Tale

Announcing the Recipients of The William Coon Jr. Business Scholarship

Announcing The Recipients Of The Scholarship for the Arts

Announcing the Recipient of The Stenzel Scholarship

research roundup

Registered Charity Number : EIN # 93-1036770

Focus topic:
CF and Travel

Navigating Healthcare and Hospitals for
CF Travelers

Ask The Attorney:
Understanding Medicare Basics for People With CF, Part 1

Pearls of wisdom:
Things to Do in Denver When You’re Dead—Lessons Learned
at 5,280 Feet Above
Sea Level

Adventures abound:
The Expansion of You

Culinary Corner:
Easy Egg Salad

Transplant Talk:
Traveling to
New York City

salty parenting:
Cape Cod to Quebec City, June 2024

In the Spotlight:
With Marina Forstmann Day

Air Travel With a
New Insulin Pump

Medical Exemptions
for Carry-on Luggage:
A Cautionary Tale

Announcing The
Recipients Of The Scholarship for the Arts

Announcing the Recipient of
The Stenzel Scholarship