Retirement (term) is defined as an action or fact of leaving one’s job and ceasing to work.

I’m not sure I can truly comprehend that word—or that action. To me, “retirement” feels like the end, like being put out to pasture. But my life has never been about endings—it’s always been about just one more day.

Living with cystic fibrosis since childhood and surviving three organ transplants, I’ve never taken life for granted. I’ve always focused on what I call a living list, not a bucket list. My goal has never been to stop—it’s been to keep going, to keep working, to keep inspiring. Work, for me, isn’t just survival—it’s what gives me purpose.

Growing up in the 1960s with cystic fibrosis, I was told the average life expectancy was 16. Then 21. Then 25. Today, I’m 69 years old and will turn 70 on June 27, 2026—a milestone I never thought possible. I’ve made it here through discipline, exercise, positive thinking, compliance with treatments, and an unshakable belief in living fully.

My journey has been long: diagnosed at age 11, countless hospitalizations, a career in the fashion industry, years coaching pole vaulters, and three organ transplants (lungs, liver, and kidney). Along the way, I’ve also had a hip and knee replacement. But through it all, I’ve never stopped moving forward.

Today, I proudly continue my work at the Boomer Esiason Foundation as Director of Team Boomer, where I encourage a healthy lifestyle through exercise, advocate for organ donation, raise awareness about cystic fibrosis, and inspire others through speaking, fundraising, and coaching. Every day that I can be active, engaged, and connected with people, I feel truly alive.

For me, there is no finish line in sight. My passion remains the same: to LIVE, BREATHE & SUCCEED, while encouraging others in the CF and rare disease communities to embrace the power of exercise and perseverance.

So no, I don’t see myself ever “retiring.” Instead, I’ll keep lacing up, keep showing up, and keep running the race of life. Because this isn’t a sprint—it’s a marathon. And I’m still going strong, one mile at a time.

Living with cystic fibrosis (CF) can sometimes feel isolating. Between daily treatments, hospital visits, and just trying to live life, the idea of a “clinical trial” might sound like one more thing to juggle. But here’s the truth: you’re not alone in this, and clinical trials are one of the reasons we have the treatments we do today.

This article is here to break it down, not in medical jargon, but in real, plain language. We’ll look at how trials are designed, why they matter, what it’s like to take part, and how researchers are working to make them easier and more accessible for everyone with CF. My hope is that by the end, you’ll feel a little less intimidated and a little more empowered to see if a trial could be right for you.

Where We’re At Right Now

Let’s start with the big picture.

• 162,428 people worldwide are estimated to be living with CF, as of 2022. (Guo et al., 2022,1)

• Only about 65% are diagnosed, which means a big chunk of people may still not know they have CF. (Guo et al., 2022,1)

• And just 12% are receiving triple combination therapies. (Guo et al., 2022,1)

If you’ve been on a triple-combo, you already know how life-changing it can be. These therapies don’t just treat symptoms, they go after the root problem, the faulty CFTR protein. For people with at least one F508del mutation, they can mean stronger lungs, better quality of life, and slower disease progression.

The reality is, these breakthroughs don’t happen by accident, they’re the result of years of hard work, not just from researchers, but from patients who signed up for trials. Every advance in CF care exists because real people with CF were willing to step up, even when they weren’t sure if they’d benefit directly.

What Trials Really Look Like

So, what actually happens in a trial? An American Thoracic Society (ATS) study followed 8,735 people with CF over a six-year period using data from the Cystic Fibrosis Foundation Registry database (Goss et al., 2005,2). Here’s what they found:

• About 1 in 3 people with CF took part in at least one official clinical trial.

• Those who joined tended to be sicker, with lower lung functions [FEV1% predicted (68 vs. 77%, p < 0.001)] and more infections [Pseudomonas aeruginosa infection (71 vs. 65%, p < 0.01)].

• They were also more likely to have private insurance and nearly twice as likely to be white.

What’s this all mean? Even though people in trials were generally sicker to begin with, their lung function declined more slowly than those who didn’t take part. That means these new treatments are powerful, and being in a trial can sometimes offer real benefits.

What else does it mean? People with cystic fibrosis who are not white and are uninsured may have more difficulty accessing clinical trials. That’s not right, and it needs to change.

So how can underserved communities participate without being overwhelmed by logistics or finances? The answer starts with clarity. Finding and evaluating trials must be explained in plain language with support that makes participation realistic. Researchers can go further by offering resources and working with patient-centered platforms, ensuring trials meet people where they are, instead of asking them to fit into a system that leaves too many behind.

Why Access Matters

If trials are so important, why isn’t everyone signing up? A lot of it comes down to access.

• Some people can’t afford the travel or time off work.

• Others don’t have nearby clinical trial centers.

• Some aren’t given information in a way that feels clear and achievable.

The good news is, researchers are starting to realize this. More trials are being designed with flexibility, remote check-ins, at-home monitoring, and financial support, so it’s not such a burden to take part.

Still, we have a long way to go. Until every person with CF has a fair chance at participation, the system isn’t working the way it should.

Finding a Trial That Fits

Okay, so let’s say you’re curious - how do you even find a trial?

• Clinicaltrials.gov: This is the official government database. It lists every active trial, but it can feel overwhelming and difficult to read.

• Private directories like pRxEngage: These are designed to be more user-friendly. They put everything into plain language, help you figure out what’s nearby, and even walk you through the steps so you don’t have to go it alone.

Questions to Ask Before Joining

Here are some good questions to bring to your care or research team if you’re considering a trial:

• Who is this trial designed for? Trials often have inclusion (who can join) and exclusion (who can’t) criteria. Knowing these upfront saves time and helps you see if it’s worth a conversation with your care team.

• What are the researchers hoping to learn? Understanding the main goal of the trial helps give you clarity about how your participation contributes to the bigger picture.

• Is there a possibility of receiving a placebo? Ask what that means for your care, whether you’ll know which treatment you’re getting, and how placebos are used in this specific trial.

• How will your health and safety be monitored? Make sure you understand what tests, check-ins, or safeguards are in place to protect you during the study.

• How often will you need to visit the hospital or clinic? Ask about the schedule of clinic visits, whether any require overnight stays, and how this will fit into your daily life.

• Will there be any costs for participating, or will you be reimbursed for expenses like travel or time? Clinical trials should not leave you responsible for out-of-pocket costs. Confirm whether expenses such as travel, meals, parking, or time away from work or school will be reimbursed.

Remember: no question is silly. If something feels confusing or stressful, speak up. This is your health, your time, and your decision.

Taking the Mystery Out of the Process

Trials can sound intimidating, but once you know the lingo, it’s a lot less scary. By simply breaking down some of the key terms and processes, clinicians and administrators can help bridge that knowledge gap:

Here are some terms you might hear:

• Screening: This is the “check-up” before the trial begins. The medical team reviews your history, asks about your daily health, and may run tests such as pulmonary function tests or blood work. The purpose isn’t to keep you out, but to be sure the trial is safe for you and truly a good fit.

• Run-in: This is the “practice round” before treatment starts. You follow your usual routine so the team can see your baseline health and be sure you’re comfortable with the trial tasks, like taking medicine on schedule or using equipment at home.

• Washout: Sometimes you’ll need a short break from certain medicines before the trial. This clears them from your system so the new treatment can be tested safely without unexpected interactions.

• Visit frequency: The schedule of when you’ll come in for appointments or check-ins. Early on, visits may be more frequent to monitor your safety closely, then spaced out once things are stable.

• Decentralized or remote options: Some trial activities can be done from home or a local lab instead of traveling to the main clinic. This may include video visits, at-home breathing tests, or wearables to make participation easier.

• Reimbursements: Trials should not cost you money to join or take part in. Most cover expenses like travel, meals, parking, or childcare. Ask what’s included so you can plan with peace of mind.

• Support: You won’t go through it alone. Many trials offer help with paperwork, transport, or questions. Emotional support and peer connections are often available, too.

Knowing these ahead of time can take away a lot of the fear.

The Emotional Side

Trials aren’t just about science. They’re about people. It can feel scary to join something new, especially when your health is on the line. Anxiety, doubt, and “what if” questions are normal.

The thing that helps? Support. Most trials connect you with people who’ve been through it before or with groups where you can talk it out. The CF community is strong—participating in trials is another way our community comes together to support and empower one another.

Why Say Yes?

So why should you consider joining a clinical trial?

• You might not see direct benefits yourself. That’s the hard truth.

• But others after you will. Every single advance in CF care happened because patients said yes to being part of a trial.

• Trials also sometimes give you sooner access to treatments, extra monitoring, and a chance to shape the future of CF care.

At the end of the day, novel treatments and procedures cannot be developed without trials, and trials need patients to test possible treatments. Every step forward brings us closer to a world where CF isn’t all-consuming.

Final Thoughts

Every breakthrough in cystic fibrosis, every new drug, every treatment, has been made possible because people chose to take part in trials not just for themselves, but for the whole community. Data shows that the CF community has proven its desire to come together and support future generations of people with CF.

By making trials easier to join and more inclusive, we can ensure no one is left behind in the search for better treatments. Together, each step we take brings us closer to the day when cystic fibrosis is no longer a life-defining condition, but a challenge we’ve overcome.

References

Goss, C. H., Rubenfeld, G. D., Ramsey, B. W., & Aitken, M. L. (2005). Clinical Trial Participants Compared with Nonparticipants in Cystic Fibrosis. American Journal of Respiratory and Critical Care Medicine, 173(1), https://www.atsjournals.org/doi/10.1164/rccm.200502-273OC

Guo, J., Garratt, A., & Andrew Hill. (2022). Worldwide rates of diagnosis and effective treatment for cystic fibrosis. Journal of Cystic Fibrosis, 21(3), https://www.sciencedirect.com/science/article/pii/S1569199322000315

Tobi and Pulmozyme ushered me into my CF life. At my first clinic visit I was introduced to new medications, treatments, and instructions. I left clinic that June day thinking about the new treatments and worrying about the cost of it all without a thought about what it took for these meds to come into my hands. In time I began to understand that progress in CF care was the result of the efforts of many: parents who were persistent in seeking help for their children and raised so much money to make it happen, scientists and doctors who asked questions and moved research forward, and those with CF who volunteered for studies of new drugs. It has taken me time to appreciate all of this since I was handed that first box of Tobi—gifts from the legions of people in the CF community who have come before me.

When I began connecting with other adults online, I learned a lot about living with CF. My new friends talked about research studies, new meds on the horizon, and the pipeline. Pipeline? In clinic I asked about research studies and learned that travel was a barrier; I live 700 miles and two states away from my CF center. When study sponsors began to pay travel expenses for CF patients in research studies I wanted to be involved.

Finding a Clinical Trial

Participation in a clinical research study may begin with an invitation from someone on the CF team. Another way to learn about research studies is checking out these two websites that describe current studies for CF and give the contact information for participating centers:

• The Cystic Fibrosis Foundations clinical trials finder: https://apps.cff.org/trials/finder

• Clinical Trial Connect (CTC) at Emily’s Entourage https://www.emilysentourage.org/ctc

After finding an interesting or promising study the next step is to contact the research site and speak with a research coordinator about the study. When patients meet the criteria for the study, they can be scheduled for a screening visit to assess if the study is appropriate for the participant. The clinical research coordinator becomes the main contact going forward.

Screening for a Study

At the screening visit the purpose of the study is explained. Details such as possible risks and benefits of participating in the study, the timing for each study visit, the plan for administering the study drug/placebo, assessments such as blood tests and other clinical procedures, and compensation are spelled out clearly. The informed consent document will address common questions like: How long will this study take? Does everyone get the new drug or treatment? Are different doses of the study drug being tried in this study? Will I know if I am getting the study drug? What happens if I decide I want to stop the study? There will be time to ask questions and address concerns before signing the informed consent. Typical tests that will be done at this visit are spirometry and bloodwork but there may also be additional specific tests for different studies. The next hurdle is assessment of the screening visit and approval - the green light to begin the study. Delays can happen, such as the time my fecal elastase test was hung up in a hurricane!

Participating in Clinical Research

Like many of our relationships in CF care, clinical research is a partnership. The study team includes the company sponsoring the study (sponsor), one or more clinical research coordinators, a principal investigator (PI), most often a doctor at the CF center, and the research subject, the CF patient. Our shared efforts continue over weeks, months, or years for the active and follow-up phases of the study.

An important step in preparing for a study is aligning the visits with everyone’s schedule and making sure the necessary spaces in the research center, the study drug, and equipment are ready when needed for each visit. Research coordinators really earn their title. Participants who travel for the study may work with a travel agency to arrange flights, car rentals, and lodging with expenses paid for by the sponsor. Preparing to travel to my center begins the process of a study visit—packing all the things I need for one or more nights away from home then usually heading to the airport the day before I need to be there. A delayed or cancelled flight can throw a wrench into a carefully planned study day.

For me, each study visit has begun with the CF quality-of-life questionnaire. Most studies include spirometry and blood draws throughout. So much blood! I’ve swallowed pills, breathed into a bag, had a study drug placed in my nose, spit into a test tube, inhaled different study meds, collected and mailed stool samples to two different labs, and once I wore a fanny pack with a study drug and pump connected to an IV for five days. I would do it all again.

In research I’ve participated in different testing procedures used in CF care and evaluation such as the Nasal Potential Difference (NPD) and Lung Clearance Index (LCI). The NPD test looks for abnormal function of the CFTR gene in the nose via electrodes attached to a voltmeter and the subject’s nasal mucosa and forearm. NPD can be used to diagnosis CF. A specially trained person is needed to operate the equipment, yet another scheduling job for the research coordinator! In one study I had the lengthy and seemingly complicated NPD test several times. Another diagnostic tool I’ve been introduced to during research is the LCI. The LCI measures how quickly an inhaled gas is cleared from the lungs during exhalation, a measure of pulmonary function. Now when someone mentions NPD or LCI in a presentation or community discussion I understand what they are talking about.

Being in a clinical research study is an investment of time. Most studies have several visits that can last a few to several hours. There are visits for dosing of the study drug, observation, and follow up. The effects of the study drug, procedures, or treatments can be challenging for participants. All studies offer an option to stop at any time. On the plus side we are compensated for our time, discomfort, and effort.

I’ve Finished the Study. Now What?

There may be a sense of let down or relief when the study is finished. Then we wait to learn the outcome. Has the drug been shown to be safe and effective? Will it progress through the pipeline? Did I get the drug? Some studies may show minimal or no benefit and the drug doesn’t move forward. There may be continued research before new drugs are approved and available to the CF community. I feel some pride seeing data from studies I’ve participated in show up in a presentation at a conference. In time there will be notice about the outcome of the study, including whether you received the study drug in a placebo-controlled trial. I was recently surprised to learn that I didn’t receive the study drug in a 2019 study. I was sure I’d had the drug.

I’ll keep raising my hand for studies. I have time and it feels good to make a contribution to the CF community. I learn new things about my health and CF by taking part in research and trying new meds and treatments. I like to follow “my” drugs in the pipeline. I am hopeful when I feel improvement from a study drug, yet it can be a long wait until it is available for me and for others. Those who are ineligible for or unable to tolerate the side effects of Trikafta are especially eager for progress in modulator therapy or different treatment avenues. We will soon learn if Alyftrek, the newest modulator approved by the FDA, will have fewer challenging side effects. And we continue to watch and wait for the research that will bring highly effective treatments to all people with CF.

Finally, we need clinical research to improve our lives with CF and researchers need us to make it happen.

Clinical trials are so important for the advancement of medicine. While deciding whether to partake in one is a personal decision, if I see a clinical trial that I may qualify for I will always go through the necessary screening. If I end up qualifying, I ask the necessary questions (mainly the potential risks to me) and decide whether to take part in it accordingly. I have no problem being a test subject so that these pharmaceutical companies that have developed medications and/or treatments can have the appropriate data to ensure their safety or effectiveness for generations to come. While it is bittersweet that I had to grow up in and out of the hospital due to lack of treatment and medications available, clinical trials continue to elevate the quality of life for newer generations. The newer generations, hopefully, will not have to endure what I (and many others) had to go through with our cystic fibrosis care regimen.

I have been in various clinical trials for cystic fibrosis throughout the years. I participated in a phage clinical trial, a clinical trial for a drug that was for managing swelling in the lungs, and a clinical trial that was simply observational based on exercise. The different trials are for different purposes, but they all help assess cystic fibrosis. The first two were drug-related trials. In a drug trial you’re given either a placebo or the actual drug itself but you don’t know which one you received until after the study has ended. For the phage study I was given a placebo but for the other study I did receive the actual drug. I think it’s cool that the test subjects don’t know whether they are getting the placebo or the actual drug. I feel like people overall do better/try harder not knowing because of the “placebo effect.” I think some people do better on the placebo because mentally they think they are taking the drug in the trial or could be taking it. In both studies my starting lung function was not great; I was very sick and literally wanted to try anything at that point. In my head, nothing could hurt. My lung function at the time of those two drug trials was 30-40% and the bacteria in my lungs had become resistant to most medications, so I was willing to try anything. My lung function and chest x-rays were better after the second drug clinical trial, so I was hopeful it would become available to me, but unfortunately the FDA never approved it.

Another risk with clinical trials is that you could do all that work and testing for it only for the drug to not be approved by the FDA. I remember it being a definite bummer that the drug never came to fruition because it did help me. The exercise clinical trial was purely observational, but I found it interesting that even though there was no drug involved, I found myself trying to exercise more and take better care of myself physically so I could perform well for the categories they observed. In that trial I had a six-minute walk test, a test to determine how many squats I could do in one minute, a glucose test, lung function tests, and monitoring of my blood pressure & oxygen levels while doing the exercises. I loved being a part of that study as the purpose was to see how people with CF did after exercising for a little bit compared to a person who didn’t have CF.

As stated above, you have to decide for yourself if you should participate in clinical trials. For me, I look at the purpose of the study and how long the study is. The purpose of the study is important because I need to know if it’s worth it for me and most of the time it is if it helps me and the CF community. Short studies (a few months) are mostly an automatic yes, and some studies are for a year or two and those are more of a time commitment. You don’t know what life will throw your way, but the great part about clinical trials is that you can stop at any time. The data can still be used for the trial.

Another factor in deciding is weighing the risks and benefits. Do the risks outweigh the benefits? Sometimes looking at the risks can be overwhelming because the drugs are experimental and could potentially have adverse reactions. In the three trials I have been a part of, the benefits outweighed the risks. For the observational one, there weren’t any risks except fatigue or being out of breath from the activities that needed to be performed. My heart rate may have increased, I might have had bruising from bloodwork, or had high blood pressure. For the two drug trials the benefits also outweighed the risks. The phage study was specifically for the Pseudomonas I was growing and at that point the bacteria had become resistant to all medications except one and that one drug wasn’t working that well on the bacteria, so I was willing to do anything. The other drug was beneficial as well because it targeted inflammation which was a reason why my lung function wasn’t great–everything was inflamed.

The risks associated with these drugs, aside from potentially affecting bloodwork, were minimal so I decided to do the studies. The main reason for me was compensation. Clinical trials are voluntary and there are risks involved; compensation is therefore a part of the decision to do them. I am volunteering to use my body as a test subject for science and for future CF treatments. Compensation is an incentive for participating in clinical trials and I had to decide whether it was worth putting myself through the necessary tests performed, drugs taken, activities performed, doctor visits needed, and the time committed.

One last deciding factor for a clinical trial is how my health is at that time. When I was constantly sick and my health was all over the place, I was eager to do specific clinical trials related to my lungs and the bacteria in my lungs. I wanted to try anything and everything that could potentially boost my lung function and quality of life as I was sick and tired of being sick and tired. I really didn’t care to do any clinical trials that may have to do with my GI system or sugar levels as that was not on the forefront of my mind. I needed trials that were specifically targeted towards lung function and attacking the Pseudomonas colonization in my lungs. As my health got better, I have been more open to doing a variety of clinical trials (if I qualify). It doesn’t have to be solely for the lungs, it can be for physique, weight, mental health, my pancreas, GI system, etc. My health is much more stable now, so I’d be willing to try different trials.

Overall clinical trials are not a one-size-fits-all and there are various reasons why one might choose (or not choose) to participate in one. I am continuously on the lookout for new trials I could be a part of as I enjoy all aspects of doing clinical trials. I think it’s cool that I can be a part of new medical findings.

As a kid, I didn’t have much of a say in my medical care—my mom made most of the decisions for me. Living with cystic fibrosis (CF) was challenging, but she was determined to give me every chance at a better life. That meant enrolling me in a few clinical trials. At the time, I didn’t fully grasp what was happening; I was just along for the ride.

As I grew into young adulthood, my priorities shifted. Like many others in their late teens and early twenties, I was busy figuring out who I was and what I wanted from life. College, work, friends, and adventures filled my days. Honestly, my health wasn’t always at the forefront of my mind. For several years, clinical trials—and even the idea of actively managing my CF—weren’t on my radar. Then, I met my wife.

A Wake-Up Call

Meeting the person you want to spend your life with has a way of shifting your perspective. For me, it was a wake-up call. After we got married, the realization hit me hard: my health wasn’t just about me anymore. It was about us—our future, our plans, and the life we were building together. I owed it to her to take my health seriously, not only for myself but also for the promise of our shared life.

That’s when I started taking an active role in managing my CF. Together, my wife and I dove into researching potential treatments and clinical trials, particularly those targeting my specific mutations. I’m part of the roughly 10% of CF patients who don’t yet have modulators available for their gene mutations. That reality has been both frustrating and motivating—it drives me to stay involved and push for progress.

The Ups and Downs of Clinical Trials

Participating in clinical trials isn’t always easy. I’ve been in studies where I’m fairly certain I received a placebo. It’s hard not to feel discouraged when you don’t see any improvement and wonder if your efforts are making a difference. But then I remind myself why placebos are part of the process: they’re essential to ensuring new treatments are safe and effective. Even if I didn’t benefit directly, my participation is contributing to the bigger picture—getting new treatments approved for the CF community.

That mindset keeps me going, especially when I think about the people who will benefit from these treatments down the line. In those moments, I feel like a small but vital piece of a much larger puzzle.

A Promising New Chapter

Recently, I joined a trial that feels particularly hopeful. This one targets one of my specific mutations, and I know I’ll be receiving the actual drug—not a placebo. For the first time, I feel like there’s a tangible chance for improvement, not just for me but for others with the same mutation.

Of course, being part of clinical trials comes with risks. The early stages can be nerve-wracking; you’re venturing into the unknown with a treatment that hasn’t been widely tested. Will it work? How will my body react? Those are questions I grapple with every time. But in the end, the potential benefits outweigh the fears.

Trust and Transparency Are Key

One thing that makes a huge difference is having a research team I trust. The doctors and staff at my research site have been incredible—they’re open, honest, and supportive every step of the way. I’ve never felt judged for asking questions or voicing concerns, and that level of trust has been crucial for me. Knowing that they genuinely have my best interests at heart helps me feel more confident about participating.

Doing It for Myself—and for Others

When I take a step back and think about why I do this, the reasons are clear. I’m doing this for myself, to give myself the best chance at a longer, healthier life. I’m doing it for my wife, who has been my rock and my greatest supporter. I’m doing it for my family, who have always wanted the best for me.

But perhaps most importantly, I’m doing it for the CF community. I know there are people out there who, for one reason or another, can’t participate in trials. Maybe they don’t qualify, or maybe the demands of the trial—like frequent travel or time off work—make it impossible. I feel a responsibility to step up for them because if I don’t, how can I ask someone else to?

The Challenges Are Worth It

Don’t get me wrong—this journey isn’t always smooth. Clinical trials can be physically and emotionally taxing. There are travel requirements, hours spent at research sites, and sometimes side effects that make you question your decision. But in those moments, I remind myself of the bigger picture. Each blood draw, every test, and even the uncertainty are steps toward progress. It’s not always fun, but it’s worth it.

Looking Ahead

As I continue this journey, I feel a deep sense of pride. I’m proud of myself for stepping up and taking an active role in my health. I’m proud to be part of something bigger than myself, contributing to advancements that will benefit not just me but the entire CF community.

Participating in clinical trials isn’t just about finding a cure or treatment for my own mutations—it’s about paving the way for others. It’s about hope, resilience, and the belief that we can make a difference.

If you’ve ever considered joining a clinical trial, I encourage you to take that step. It’s not always easy, and it might feel daunting at times, but the impact you can have is immeasurable. Together, we can push the boundaries of what’s possible and bring hope to those who need it most.

I had my 81st birthday this past October 20th. I had been looking forward to this special birthday for a long time. I remember as a teen telling a friend that I didn’t think that I would live to be 40 and now I’ve more than doubled that lifetime expectancy!

As a teen, I didn’t know why I was always so sick. I was not diagnosed until I was 53, probably because one of my chromosomes is an undetected CFTR mutation or DNA variant (IVS8-5T) while the other is the F508del. Nature ran an article about the clinical consequences of the 5T variant several decades ago . The IVS8-5T variant causes, among other things, severe sinusitis.

In 1997, at the age of 53, my ENT said I was fine after yet another surgery, but I knew I wasn’t fine. As I sat in the patient chair I started crying because I knew something was very wrong. He told me that I was still young so he would send me to the Hospital of the University of Pennsylvania in Philadelphia near where we lived at that time. The day I saw the ENT specialist, Dr. Lanza, he immediately suspected I had CF. He ordered my first ever culture which showed Pseudomonas, MRSA, and a few other bacteria. He also ordered my first CT scan which showed the extent of malformation of my sinuses. He then ordered a sweat test, which was done at the Children’s Hospital of Pennsylvania because they don’t diagnose adults with CF. The sweat test was overwhelmingly positive and confirmed the diagnosis. I was put on gentamicin and another IV for my infection but this permanently damaged my inner ear sense of balance.

In 2010 I had a frontal sinus obliteration, which finally eradicated most of the bacteria. During this surgery, my scalp was cut ear to ear and pulled down to expose the frontal sinuses so that I would not have a scar visible on my face. The surgeon used a diamond drill bit to excavate the sinus cavity and infection. The empty space was then filled with disinfected fat from my abdomen. I had many friends offer to donate their fat as well for this procedure but the surgeon did not get the joke.

My proudest achievement was answering the call to start a CF Walk for a Cure in 2007 in McAllen, Texas where we then lived because the warmer weather was better for my health. I’m so proud of all those who came to support me. We raised tens of thousands of dollars each year. I connected with so many CF families with the same drive to find a cure. I’m proud to say that all these years later, even after we moved to Atlanta in 2013 to be near family, this walk continues to be very successful.

Now with the advances in CF care—especially with newborn screenings and new CFTR modulators like Trikafta—I hope those with CF will have more normal lives. I hope to be a model for others—they too can survive CF with near normal lives and live a good life without a short life expectancy just by doing their life-saving treatments and taking their meds.

QUESTION

Do I have the ability to obtain Medicare benefits by just proving I have cystic fibrosis? What are the ways a person with CF can become eligible for Medicare?

ANSWER

Some people with CF are incorrectly told that they can obtain Medicare benefits simply because they have a CF diagnosis but this is not correct.

Typically, there are three ways a person usually becomes eligible for Medicare benefits. The first way to obtain Medicare coverage is by reaching the age of 65 and meeting other eligibility criteria related to citizenship and work history. The largest number of Medicare recipients receive Medicare because they are 65 years of age or older.

The second way to obtain Medicare benefits if a person is not yet 65 years old is based on the receipt of Social Security Disability Insurance (SSDI) benefits. More people with CF are becoming eligible for Medicare after they stop work and the person has received SSDI benefits for 24 months and continues to receive SSDI benefits. If a person loses SSDI benefits before 24 months of benefit payments have occurred then the person will not receive Medicare unless they become eligible for SSDI in the future and meet the 24-month waiting period for Medicare eligibility.

Third, a person over the age of 18 who receives a Social Security Disability benefit based on a deceased, retired, or disabled parent’s work record will become eligible for Medicare after 24 months of receiving the Social Security benefit. Some Social Security offices will approve eligibility for Medicare if a person was receiving another type of Social Security Disability benefit for more than 24 months and then became eligible for the parent benefit.

There are two medical conditions that result in eligibility for Medicare benefits based on diagnosis with the condition even if a person has not yet received SSDI benefits for more than 24 months. One condition is Amyotrophic Lateral Sclerosis (ALS). The other condition is end-stage renal disease, which is permanent kidney failure that requires dialysis or a transplant.

A person must also meet the following conditions:

• The person’s kidneys no longer work,
  AND

• The person needs regular dialysis or has a transplanted kidney.

However, if a person has Medicare because of permanent kidney failure then Medicare coverage will end either 12 months after the month the person stops dialysis treatments or 36 months after the month the person has a kidney transplant. However, if a person is receiving Social Security Disability Insurance (SSDI) benefits for more than 24 months, Medicare will continue for as long as the person receives SSDI benefits in most circumstances.

In addition, if a person starts dialysis again or gets a kidney transplant within 12 months after the month the person stopped dialysis, Medicare resumes. If a person starts dialysis or gets another kidney transplant within 36 months after the month the person received their first kidney transplant then Medicare coverage would restart.

Lastly, if a person only receives Supplemental Security Income (SSI) benefits then the person would only be eligible for SSI benefits and Medicaid coverage. A person is not eligible for Medicare if the person only receives SSI benefits.

QUESTION

Does Medicare provide coverage for vision benefits and eyeglasses?

ANSWER

Traditional Medicare coverage does not provide coverage for eyeglasses or general eye appointments. Medicare Advantage plans may provide such coverage.

Even though I teach research methods at a medical school, I’ve never participated in a clinical trial myself—not for CF-specific drugs or for any other kind of innovative therapy that might help with some of my symptoms. Why? As I often tell my students, participating in medical research requires more than just interest. And whether one feels interested in the first place can depend on a lot of factors before even getting into the many external barriers that can prevent people from getting involved with trials.

Working with different CF organizations over the years has shown me the diversity of perspective in our adult community surrounding the idea of a cure. Personally, I don’t focus my own health care activities around the idea of one day not having CF anymore. Although I understand that desire, I don’t share it. My overarching goal has always been to live as well as I can with my CF while still being a person who has this disease and is shaped by it. I don’t want to know who I would be without CF. Moreover, I don’t want to live in a world “free” of people with genetic conditions—because that doesn’t seem very free at all to me.

Family history—going back to how I joined the world in the first place as well as the legacies of genocide and ethnic cleansing in multiple branches of my ancestry—makes me innately suspicious of efforts to rid the world of a given genetic disease. I don’t look at health conditions that people live with from birth the same way I look at infectious diseases like polio or malaria. Lifelong chronic disease shapes who we are and who we become. Whether or not that influence always makes people better than we might otherwise have been doesn’t matter in my view. What does matter is the message to the world at large that people with CF and other genetic diseases are not problems to be solved. When people suggest that I should chase a cure for a condition that is inextricably intertwined with so much of who I am and what I give back to the world, I question the motivations behind that thinking.

That said, my interest in living as well as I can with my CF certainly invites plenty of interest in participating in research when suitable opportunities arise. I say “suitable” because thus far I’ve only contributed data to studies that don’t involve an interventional component. We often use that term in the clinical research world to mean “doing something to address an underlying issue” like giving someone a drug or providing them with counseling.

Over the years I’ve contributed samples of different bodily fluids and secretions, plus the occasional tissue biopsy. I’ve also benefited immensely from certain drugs that my providers have used off-label as part of my home care regimen. That term refers to taking an already-approved drug for a purpose outside the one for which it generally gets prescribed. For example, inhaled corticosteroids give me a lot of help with controlling pleuritic inflammation—swelling in the muscle walls of the lungs—that can make it much more difficult and painful to breathe well even when my airways are fairly clear. Some people with CF deal with pleurisy more than others. Off-label use of beclomethasone has made a massive difference in my daily quality of life and made it easier to stay physically active, which in turn helps to keep my airways clear and free of infection.

I know quite a few other folks in the 10 percent of adult CF community members not currently eligible for CFTR protein modulator drugs who have likewise benefited strongly from off-label use of medications initially developed for other conditions. I also know several fellow “10 percenters” who have participated in trials of the triple combination modulator Trikafta for people with very rare mutations not initially included in the eligibility criteria, as well as people with double lung transplants whose genotypes do fit the initial eligibility profile for that therapy. Some of them have wound up taking Trikafta steadily and benefiting from it substantially. Others have regretted ever taking it in the first place.

Concern about potential side effects that would reduce my quality of life looms large in my own thinking about what I would and would not be willing to take for experimental purposes. Trying a completely new drug always involves a bit of a gamble before one even considers that a given trial participant may not get the study drug as opposed to a placebo or what we call standard care in medical research. A “standard care” scenario for a drug trial usually means participants in the control group—that’s the group of folks who get compared to people taking the new therapy—continue taking whatever medication they were using before. Getting the study drug raises a whole host of additional concerns about unanticipated adverse events, which may range from temporary brain fog or minor stomach upset to severe allergic reactions or permanent organ damage.

For me to take a totally new drug, I’d have to weigh the potential risks carefully and do a lot of background reading on the active ingredients. I’d need to consult with my CF team and get their input while discussing my own concerns with the providers who know me and my body best. Ideally, I would also be able to coordinate my participation in a trial through my CF clinic. In many cases this isn’t an option, which I’ll address in more detail along with other instrumental barriers like transportation and availability. I’d need to consider the broader social context of both the initial drug development and the planning of human trials for later stages of testing its safety and efficacy—how well the treatment works under ideal conditions closely controlled by researchers. I’d also want to know how thoroughly the research team planned intentionally for use of the drug by a racially and ethnically diverse population of patients, as well as patients of all sexes and genders.

I should note that clinical trials aren’t always testing drugs. Medical devices, including those that many of us use for routine airway clearance, also go through clinical trials before getting authorized for widespread use. Having been an early adopter of a couple different physiotherapy devices over the years that have helped me with my own health maintenance, I’ve often felt more open to the idea of participating in a device trial than a drug trial. But my interest and willingness will ultimately depend on the specifics of a given trial and that all-important ratio of potential benefits to known risks. After many years both conducting and reviewing research in the health sciences, including providing human subjects review for drug and device trials, I think about those same considerations for myself as a potential study participant.

Of course, work commitments introduce several other considerations as well for participation in clinical trials. Medical research studies can differ quite vastly in their logistics and time requirements. And as several of my USACFA colleagues noted while we were planning the Focus Topic for this issue, not all CF centers in the United States participate equally in trials for new drugs or devices. Some do not participate at all! If a center does participate in trials, the many people in today’s adult CF community who work in paid or volunteer positions must consider how the design of a given trial fits with our responsibilities for those commitments.

All the usual logistical factors involved in routine care also apply here. If we are going to a clinic site physically, how are we getting there and is that transportation affordable? Do buses or trains run when we need them to get where we’re going? And if we’re driving, is parking available and accessible? If we are self-administering medication or using a device at home, how do those activities impact our ability to do other daily tasks? Do we have adequate instructions available in our native languages to ensure that we’re taking the medication or using the device as intended? Can we have someone else at home with us in case we experience a serious adverse event and need immediate assistance? What about our own responsibilities at home and how our health care activities intersect with those? Can we participate in the trial and still provide needed care for children, elders, pets? And how do all these nuances, along with whatever type of observation and monitoring we are under as trial participants, impact our mental and social well-being?

Thanks to many advancements in care that got their start in clinical trials, including but certainly not limited to CFTR protein modulators, more adults with CF than ever before are living dreams that might once have seemed unattainable to us. We’re building our careers, furthering our education, buying our homes, expanding our families, caring for others, exploring new hobbies, and more. This necessarily involves thinking about what we want for our futures as well as the day-to-day of our lives in the present. How clinical trials fit into that can depend on a great deal of factors.

I’ve seen promising innovation and creativity from medical researchers in recent years, especially those who partner closely with patient scientists and patient advisors from outside the scientific community in planning their drug and device trials, aimed at making research participation more feasible and accessible for people with CF and a host of other chronic diseases. Thanks to digital technology and vastly improved connectivity, today’s clinical trials often present fewer barriers related to transportation and time. Fair compensation for trial participants—and awareness of us as people with full lives of our own that have value and meaning—has increased as well thanks to activism by patients and our families. That doesn’t always mean participating in a given trial would be worth it with all factors considered. But it creates a favorable landscape for making those choices as empowered consumers of our own health care.

So to the medical researchers reading this, regardless of whether you have CF yourselves: Keep innovating! The more creativity and ingenuity you bring to designing the patient participation experience for drug and device trials, the more you’ll welcome a diverse group of participants who can help you demonstrate the full potential of your interventions. Don’t hesitate to get us involved in the planning and to get our honest input about potential barriers and solutions, even if your funders don’t require that sort of outreach. These days you may well connect with a few of us who work in different areas of medical research ourselves and can offer insight from multiple perspectives!

And to all fellow adults with CF reading this, whether you have any background in medical research or not: Your time is valuable, as is your comfort! Living with CF and benefiting from prior advancements in care does not obligate you to put yourself in harmful situations for the sake of contributing data. I encourage knowing yourselves and your worth—and trusting your own judgment about whether to participate in any clinical trials you get the opportunity to join. The right research opportunities for each of us are the ones that work for our lives and allow us to thrive in the process.

When last I left off in the Autumn 2024 CFR, I was recovering from a lobectomy for lung cancer in July 2024. I also had some superficial squamous cell cancer (SCC) scraped off my head. No big deal.

Well, it became a very big deal very quickly. A bump started to form where the scraping had been done. I took a picture and sent that to the doctor who had done the scraping. He brought me in and did a culture. Long story short, this thing grew fast and was literally sucking the blood out of me, so much so that I became anemic and completely exhausted. Taking a shower was draining (pun intended); I couldn’t do anything else except lie on the couch. At the beginning of September 2024, I had the SCC growth removed but the surgeon could not get all the cancer out safely, and it came back two weeks later. I then saw a radiation oncologist as well as an oncologist. They decided the best treatment plan was to do proton therapy. However, the tumor was growing so fast that the measurements were obsolete within a week. I did a couple of rounds of chemotherapy to try to slow the tumor growth down, but that didn’t work. In desperation, I sent the tumor picture to my head and neck cancer surgeon (he had removed a very aggressive SCC from my head in 2011) and explained the situation. He replied immediately and said it looked like he would need to do another surgery on me. Yay!

I had the tumor removed at the end of October. This time, the surgeon got most of it out but there were a few microscopic cancer pieces on the dura of my brain that he did not want to touch for fear of giving me a stroke. To cover the spot left over from the tumor removal, he took a big piece of skin off my wrist along with a vein for a blood supply. Unfortunately, I have small vessels and that skin ended up dying. I had another surgery in early December to replace the dead skin with skin and a vein from my other wrist, and that healed beautifully.

No rest for the weary though. I had a CT scan of my lungs in mid-December and to my surprise, my lungs were full of cancer. “Too many nodules to count” was one of the findings. I assumed my lung cancer came back, but nope. It was the SCC from my head, which metastasized to my lungs. I really thought I was done for. I immediately started getting rid of stuff, thinking about what my husband needed to know once I was gone. It was very rough and by mid-January I was in a deep depression. My psychiatrist increased my depression medication, and it took a couple of months, but I was feeling better mentally by the end of March. I was so anxious about cancer and dying that I lost 15 lbs that I could not afford to lose. Christmas presents for my friends were left unwrapped because I just could not do anything, even something as simple as that. It was horrible. I am now doing immunotherapy, which scared me to death when I started it in February 2025, because I have transplanted lungs and a transplanted kidney. Essentially turning on my immune system could have caused my transplanted organs to go into rejection, and if that happened, I would die. But, I’m happy to say, so far so good! Most of my cancer is gone, and my lungs and kidney are doing fine. I don’t know what the next day or the next test will show, so I take one day at a time and enjoy feeling good enough to travel and get back to volunteering.

Thank goodness I was retired. And thank goodness we did not get another dog. We were both exhausted every day, especially when I was in the hospital and Scott was there every day, all day. He had to drive me to all my appointments. I had to stop all my volunteer work. If I was still working, I would have had to take leave without pay because I surely would have used up any sick leave I had left. I barely had any energy, nowhere near enough to also do my high-stress job. I always knew I could only reach a certain level at work and never applied for jobs I knew my health would eventually keep me from doing. That was my reality, and yes people questioned (and understood) that decision, but I never did. I’m grateful for the jobs I have had over the years but I’m also grateful for being retired during the fight of, and for, my life.

I met my younger self for coffee today.

I was on time.

She came in ten minutes late, the backpack with her portable oxygen weighing her shoulders down.

I wore my hair down, freshly washed, natural auburn strands draping loosely past my shoulders.

Her hair was faded purple, still braided from two days earlier, loose pieces blowing across her forehead.

I wore a long, flowing skirt with a sleeveless knit top framing my torso.

She had on a sweatshirt and pajama pants, both soft and perfectly worn in, with faint stains from her tube feeds.

She sat across from me, staring open-mouthed, searching inside for the memory of the stranger in front of her.

I remembered everything she felt.

She looked down at her mocha, sharing that she’d just moved back in with her mom.

I untangled my fingers from around my latte, watching her intently as I showed photos of the life we live in
San Francisco, of the apartment we love that’s become our safe space.

She broke down, describing through tears I could somehow feel on my own cheeks how scared she was to live with another denial for the lung transplant she needed to survive.

I smiled faintly, placing my warm hand on top of her chilled one, softly telling her next week is our two and a half-year lung transplant anniversary.

She snapped that she doesn’t want to hear any details,
the sting of thrashed hope still splintering her future.

“I know.” I remembered. I understood.

She said she’s been thinking of giving up.

I reassured her that being afraid to keep going,
of what’s coming, isn’t the same as wanting to give up.

She asked if one day it would be worth all of this pain.

I told her she will never think the pain had a purpose. She will never make sense of it.

But one day, the grip on her chest will loosen. She’ll let go of the breath she’s holding.

Her ground will feel even again.

We stood up together, my hand finding its way to her forearm.

I drove her home, walked her to her front door, shouldering her portable oxygen and the weight of her fears
for just those steps.

She thanked me for the coffee, shutting the cherry red door.

I waved goodbye, left to wait for when we meet again.

This piece is inspired by the poem, “I Met My Younger Self for Coffee,” from Deep in My Feels by Jennae Cecelia. It’s a reflection on survival, grief, and hope, drawn from my experiences with cystic fibrosis, through end-stage lung disease, and now navigating life post-transplant. Reimagined through the voice of Matison Deaton, February 2025.

I was listening to a podcast about writing and publishing and the speaker talked about setting boundaries to protect their writing time. The example given was that when a doctor’s office calls to set up an appointment, the speaker never just accepts the appointment; rather, he waits until the doctor’s office has an appointment that is convenient for his writing schedule.

I felt a bit angry. I wasn’t angry at the podcast—boundaries are wonderful when you have the privilege to set them. Perhaps I’m a bit angry that I don’t have the opportunity to prioritize things other than my family’s health. One thing I took from the discussion is that he doesn’t have any significant health issues. Perhaps I’m wrong, but I doubt it! It reminded me of reading White Privilege: Unpacking the Invisible Knapsack by Peggy McIntosh. In her book, McIntosh identifies various aspects of daily life that she had once taken for granted and through the exercise of writing them down, recognized the many opportunities not granted to others simply because of their skin color.

Privilege is an interesting phenomenon and not one we have a lot of control over. I am a white female in the United States. For most of my life, I have been comfortably middle class. I grew up with two college educated parents with stable blue-collar jobs with excellent health benefits and retirement pensions. I’m also disabled with cystic fibrosis, diabetes, and asthma. My medical conditions are significant and life threatening, but they are primarily invisible. While I am open about my disabilities, their physical invisibility provides additional privileges that I wouldn’t have if my disabilities were visible.

Here is my list of privilege wishes:

• I wish I could leave the house with a small “clutch” pocketbook.

• I wish I could go to a “no cell phone” event but I need mine to manage my insulin.

• I wish I could travel without worrying about my meds being confiscated.

• I wish I could travel with just a carry-on bag.

• I wish I could travel without researching the best hospital in the area.

• I wish I could go to an interview or a meeting without threat of my medical equipment beeping.

• I wish I could go to an interview without risk of needing to disclose my disabilities.

• I wish I could choose employment without needing to analyze the health benefits options in great detail.

• I wish I didn’t need to use all my paid sick days to attend medical appointments.

• I wish other people going to work sick didn’t put my life at risk.

• I wish I could choose the lower cost health plan.

• I wish I could get life insurance to protect my family.

• I wish I could get 90-day supplies of all my meds.

• I wish I could go to the pharmacy once a month at the most.

• I wish I could coordinate my medical appointments easily.

• I wish my medical appointments could be less than an hour away from my home.

• I wish health care wasn’t connected to employment.

• I wish insurance wasn’t so difficult.

• I wish insurance didn’t require prior authorizations.

• I wish insurance didn’t require me to use five different specialty pharmacies.

• I wish I didn’t have to deal with insurance issues on a weekly basis.

• I wish my medications came already organized for A.M. and P.M.

• I wish I knew that I would always have access to needed medications.

• I wish they gave you enough medications to actually make it to the next refill.

• I wish everything could be on automatic refill.

• I wish that all needed medications showed up at my door.

• I wish that all medications did not require a signature upon delivery.

• I wish I didn’t have to call the manufacturer every time a pod falls off.

• I wish I didn’t have to “save” the pod to read them a bunch of numbers to get a replacement.

• I wish I would have a chance during a “doomsday scenario.”

• I wish I could count on society protecting me during a pandemic.

• I wish I could count on having access to life-saving ventilation during a pandemic.

• I wish I could count on society not deprioritizing my care during a pandemic.

• I wish I could request curbside pickup during a pandemic and be easily accommodated.

• I wish I could request a workplace accommodation based on my disabilities and have confidence that
  I would be treated fairly.

• I wish the Americans with Disabilities Act had more teeth.

• I wish my disability identities were seen by society as assets.

• I wish I could watch tv and see disability represented.

• I wish the representation I did see was authentic and empowering.

• I wish I could buy magazines, books, and toys that feature people who share my and my children’s disability identities.

• I wish I did not have to risk being seen as a bitch while advocating for my needs.

• I wish I did not have to risk being seen as a bitch while advocating for my children’s needs.

• I wish I could hang out in person with people that share my identity without putting my health (or their health) at risk.

• I wish I could plan on retirement.

• I wish I could plan for a comfortable and healthy retirement.

• I wish I could plan on living to “retirement.”

Roseann Greco

Age: 41 (at the time of the interview)

Resides in: Smithtown, NY

Hobbies: book clubs, reading, book journaling, traveling, and spending time with my nieces and nephews

You were listed for a lung transplant at Duke, Columbia, and at a center in Philadelphia. Tell us what that experience was like. What were your PFTs at that time? How was your quality of life?

My journey with being listed for a double lung transplant began with Columbian Presbyterian in 2011. My FEV1 was ranging between 32 to 37 percent. I remember at that time my lowest was a 24% FEV1. During that time, I was on 10 liters of oxygen and had a portable oxygen tank. I looked so frail; I didn’t even recognize the person staring back at me in the mirror. I would say where did the vibrant, energetic girl go? I stayed in bed for almost two years. I struggled with getting dressed, with being on treatments, with eating, and with being on constant IV antibiotics. I started to lose hope. I anxiously waited for my call and pictured what my new life would be like with having healthy lungs. At the same time, I was having a hard time coming to terms with parting with my own lungs because they were mine and I was accustomed to living with CF. My dad, being a mechanic, used to tell me getting a pair of new lungs was like getting new tires put on a car.

Three calls came from Columbia Presbyterian. Each call felt like I was getting closer and closer. I remember my first call was on New Year’s Eve. My parents’ friends and their kids were visiting at the time. When we got the call, we had to send them home. That call was a false alarm as they had mistakenly called me instead of another person. The second call resulted in the lungs being no good; they weren’t going to hold the oxygen and they would just fail.

On the third call, it was determined that the lungs were too big. They needed to be small because I am only 4 feet and 10 inches tall and petite.

Your CF care team is Columbia Presbyterian. Why were you listed at two other transplant centers?

All the hospitals were telling me the lung size was going to be a challenge for me. Since I needed lungs from a small child or someone petite, I was listed at multiple hospitals because I wanted to keep my options open. Also, I had signed papers at the time to accept a high-risk donation (examples include: organ donation from donors with exposure to HIV, donors who are in prison, donors with history of intravenous drug use, donors with a history of prostitution, donors with high-risk sexual history, and donors with hemophilia).

What was your view on transplant while waiting for one? What is it now? Initially, you indicated you would not want one, why?

Back then, I don’t think I was thinking about anything other than being able to breathe, coming off IV antibiotics, and being able to have somewhat of a normal life. When you are that sick, you just want relief. I also lived with the fear of wondering how long my lungs would last. As much as I suffered, I knew this was not what God had planned for me; he had better plans for me written in my book. As I look back now, I’m happy that the transplant wasn’t an option for me. I read so many stories about rejection and how the necessary immunosuppression pills cause cancer in transplant recipients. I’m glad I didn’t have to worry about rejection and being up against the clock. I have been through plenty as it is—everything from bowel obstruction to kidney stones, three times.

Did they test you for your mutations? Did you know that you were a good candidate for modulators?

Looking back at my pediatrician’s medical records, I was tested as an infant having two copies of the F508del mutation, which is both the worst and most common mutation. They offered me a chance to try the modulators for Orkambi and then for Trikafta. At first, I was hesitant. I wanted to say no more drugs and no more pills because I was on every single drug you can think of! I kept thinking, how is this going to work? Am I just wasting my time?

Can you share your experience with modulators? Were you in a clinical trial for any of them? Which ones did you try? Did you feel a difference right away? Were there any side effects?

I was in several clinical trials for various modulators. I knew right away that I had the real medication because within three to four days I felt a great difference. I did my first clinical trial with Orkambi back in 2015. I remember constantly keeping track of my SpO2 which was a steady 97-98%, and I felt fantastic. I noticed less mucus production as well. I did notice a change faster with Trikafta than with Orkambi. I felt a slight difference with the switch from Orkambi to Trikafta, and I thought, wow! If this is what my life was going to be like on this medication, I am home free! Then, when I finished the clinical trial and reflected on my time on the medication, I felt like I had just lost the greatest thing that ever happened in my life. I wondered if I would ever get this opportunity again. I am so thankful for having been given the chance to see what it is like to just simply breathe. My time on the medication was short and I hoped that I would get the chance to take Trikafta again.

Talk about being on Trikafta. How did it change your life?

After being on Trikafta, I felt instantly better. I went dancing, I gained weight, and I can now exercise better and longer.

What can you do now that you could not do on modulators?

When Tikafta came out, I was able to start before it was approved because the government granted me permission since I was on a lung transplant list. My journey with Trikafta was amazing. I felt like the life I was waiting for had finally arrived. The medication was a total game changer for me. Right after I started the medication, I put myself to the test. I celebrated with my family and went dancing with my cousins the following night. I am able to enjoy nights out watching my friend sing in her wedding band and also dance with them. I can sing my favorite songs in the car, especially my favorite singer Celine Dion’s songs, and hold such high notes while singing her songs in the car. The joy is watching everyone in other cars look at me.

Do you still do CF treatments? What about airway clearance? What happens if you get a cold? What do you do now that you’re on Trikafta?

I wish I didn’t have to do any respiratory treatments, but, unfortunately, I am in the category of advanced lung disease, so I’m not totally off of treatments or airway clearance. I still do the vest for a half hour in the morning, usually accompanied with a cup of coffee or a cup of tea. When I’m not sick and feel back to my baseline, I do very few aerosol treatments. I can stick to the inhalers and one machine treatment. If a virus or a simple cold finds its way into my lungs (as they do) then I’m back to 45 minutes to an hour of treatments each day until I am feeling better. I have noticed a huge change with my oxygen saturation levels being in a steady range of 98% to 100% and I’ve also noticed less clubbing in the fingers since being on Trikafta. Every so often I see the clubbing when fighting an exacerbation. When the clubbing returns I call my fingers monster fingers.

Being on Trikafta has helped me get over the hurdles of not being on long courses of intravenous antibiotics, so instead of a 28-day intravenous antibiotic course it lasts a week to 10 days, depending on the severity of the cold or germ I caught.

When you travel, do you still need supplemental oxygen? What about with air travel?

Being able to travel without oxygen has been the greatest accomplishment since I have been on Trikafta. For my 40th birthday, I went to Paris and saw P!nk in concert. This year I climbed Mount Washington in New Hampshire, which has an elevation of 6,288 feet, in New Hampshire. I’ve traveled to Connecticut, Delaware, and Ocean City, Maryland. We are currently planning a trip to Vegas, a trip to Italy, and a trip to Maine. Before being on Trikafta I didn’t enjoy traveling because of all the extra respiratory treatments I needed to bring and being fearful of forgetting something. Where I am right now in my life, if I forget to get something, it’s really not a big deal because I feel I can skip a treatment here and there and not worry. I realize now that I can relax and enjoy my life rather than worry all the time. In Connecticut, I have been to the Pez Factory and Mystic Seaport in Mystic Connecticut. I’ve visited several casinos in Delaware, Massachusetts, and in upstate NY around the Lake George area. Mike and my family both enjoy gambling—I guess it’s an Italian thing. Our grandparents both loved it. His grandmother won the state lottery of Connecticut on the same day as my birthday. Every September, we go to a festival called the The Big E in Massachusetts.

Every summer we go upstate to Alpine Lake with Mike’s family and ours and I am able to walk, jog around the lake, and ride the bike trails. This past October, I was able to practice soccer with my nephew and run the whole length of the soccer field for a good half hour straight. Trikafta has been the greatest gift that has been given to me and it has tremendously changed my life, allowing me to do all these things.

Tell us about your cancer scare with your appendix.

In October of 2023, right after I turned 40 and went on my trip to Paris, I was diagnosed with Stage IV Goblet cell carcinoid appendiceal cancer. Having a family member come to your bed side with tears in their eyes, telling you that you have cancer is a horrible experience. For me, that family member was my sister. She has a medical degree and was therefore in charge of corresponding with the doctors and surgeons. She has not only saved my life once, but multiple times. I was having stomach issues for quite some time and feeling bloated all the time. I felt full very quickly after I ate, which drastically reduced my appetite. My stomach was so bloated that I thought I was having another bowel obstruction. I contacted the doctor and an x-ray seemed to indicate it was just some stool. I developed an addiction to exercising at the time because my mom thought I was gaining unnecessary weight and my clothes were not fitting right—I always looked pregnant. I often experienced a burning sensation like I had eaten something spicy and I found myself in the bathroom frequently. One night I finally realized the pain would not be so low if it were gas or stool. The next day I advocated for myself and called my CF doctor and requested a referral to a gastroenterologist. I called and they gave me the new patient run around that they couldn’t fit me in and his next appointment wasn’t until February 2024. They informed me that my doctor needed to write them an email stating why the appointment was so critical. When I finally saw the gastroenterologist, he sent me for a CT scan. The results came relatively quickly; I knew it wasn’t going to be good news. I remember reading the report and telling Michael that what I was reading didn’t look good—very large tumors, but not necessarily cancerous. I got a hold of my sister right away and she spoke to my gynecologist, since I was scheduled for an emergency hysterectomy. During the procedure they found that the cancer had originally started in my appendix so they had another surgeon remove my appendix while I was under anesthesia for the hysterectomy. I had eight lymph nodes removed from the omentum, a layer of adipose tissue that nestles on intra-peritoneal organs. They saw over 50 little tumors on my small intestines, none of which they could remove as the surgeons were afraid of perforating my bowel. The recovery post-hysterectomy was extremely painful. If I hadn’t been proactive and advocated for myself I don’t think I would be here today telling my story.

Are you now in menopause from the hysterectomy? Was that a shock to you?

Getting a hysterectomy a long time ago should have been on my to do list. It was already hard for me to accept the fact that I couldn’t have a family on my own and let my parents become grandparents in the process. In the end, having a hysterectomy because of cancer wasn’t a shocker for me—just another heartache in the end. I was not ready to lose that part of my womanhood. I was very emotional and it was difficult to accept, plus the recovery wasn’t an easy one. However, I had tons of family plans for the summer and I wasn’t going to give them up. Every time I look down at my scar I’m reminded of everything I’ve endured—scars represent the battles that you have overcome. The benefit of this unwanted surgery is the lack of a menstrual period. Through everything, my parents have been and continue to be my greatest supporters in life. When I’m struggling with what has transpired they tell me that they are just happy and relieved that with all that I’ve overcome from childhood and into adulthood, that I am still alive. They know I will never be 100% healthy, but one day there will be a cure and I hope they will be around to see it. For me, that would be the greatest gift I could give to both my parents.

How was being on chemotherapy, and what were the side effects?

Being on chemo was a very painful experience—both emotionally and physically. It was like a horrible, ongoing nightmare. I couldn’t believe this was actually happening to me. I wasn’t sure I was going to make it through all six months of 12 rounds of chemo. Chemo treatment was every other Monday for a total of two to three chemo sessions per month depending on the month. As time went on the exhaustion took hold. I usually slept the rest of the day following my chemo treatments. I developed horrible jaw pain when chewing. The pain was so intense that I was barely able to enjoy anything I put into my mouth. My appetite got worse and worse as time went on. I couldn’t use utensils because they left a metallic taste in my mouth. Eventually, my swallowing was delayed too. It was a struggle to get any food down as eating became more of an effort rather than an enjoyment. Even now, I still have issues when chewing—there’s a slight burning sensation in the back of my jaw.

Can you tell us about your brain injury? How long did it take to recover from this?

In 2015 I wound up in the hospital from an overdose of intravenous Tobramycin antibiotic, which left me in the hospital ICU for a month. It was just an ordinary CF day with the pills and whole morning routine, until later when I didn’t feel well. I felt a bad burning sensation, almost like really bad acid reflux. My mom gave me some Pepcid on her way out the door, thinking it was nothing. While I was in the shower, I had my head resting against the tile, barely able to keep my eyes open. At one point, I fell asleep and then I woke up and thought where the hell am I. After I got out of the shower, I tried texting my friend, but I forgot how to type and then I couldn’t talk. Thankfully, my sister came over to check on me. She said I needed to go to the hospital right away so my father drove me to the ER. I passed out before I even made it inside and the last thing I remember was a team of doctors running toward me. I never made it to the emergency room. When I came to I was lying flat on the operating table getting a hemodialysis catheter inserted and a spinal tap, which they needed to do four times. I remember just lying there, in and out of it, not really hearing much but I felt pressure in my neck and saw a bright light. I slipped into a coma for 72 hours. While in the coma, I thought my mom put my phone to my ear and I heard my grandmother’s voice say “Rosie, it’s Grandma” and I woke up.

My mother told me my sister was pinching me, trying to wake me up. Thinking about it now, I believe I had an out of body experience. I went to heaven, but never really saw God or any past family members. The music was so peaceful and the grass was greener than ever. It was like I was a bird flying as high as I could. Then there was the darkness, like I was in hell. All the animals growled at me and it was dark. When I woke from the 72-hour coma I experienced a lot of hallucinations. All the nurses who took care of me looked like they were dressed in costumes from Disneyland. I needed to learn to get up and walk all over again. The doctors thought I would never be able to talk again. Recovery was tough, I don’t know what was worse, going through six months of chemo or doing dialysis for a month. I finished dialysis right before my 31st birthday. Today I suffer with bit of brain damage and hearing loss. I forget where I put things and sometimes when driving around it takes me a while to remember where I am on the road—familiarity doesn’t come to me right away. I always need to drive with a GPS on.

Part II of Andrea’s interview with Roseann Greco will be printed in our Summer/Autumn 2026 issue.

Caries (Tooth Decay) In A Cohort Of Adults With Cystic Fibrosis: A Cross-Sectional Study

https://tinyurl.com/2c96prrm 

A cross-sectional study of 92 adults with CF and 92 adults without CF was undertaken in Cork University Dental School and Hospital. The median age for study group and control group participants was 31 years and 27 years, respectively. All participants completed a detailed questionnaire before undergoing a clinical examination that recorded demographic, social and oral health variables. Caries were recorded using the Decayed, Missing and Filled Teeth (DMFT) index. All data were statistically analysed using the Wilcoxon rank-sum test, chi-squared test and Fisher’s test. Negative binomial models were also used to analyse data. The study group had a higher mean DMFT score compared to the control group. While the study group had a higher DMFT, the only component that was statistically significant between the groups was the Decayed Teeth component. In this study, the cohort of people with CF had more caries than people without CF. Further research is required to establish if underlying systemic conditions, social and behavioural factors, or a combination of the aforementioned are responsible for a higher caries experience in this study group.

A Chronic Pseudomonas Aeruginosa Mouse Lung Infection Modeling The Mucus Obstruction, Lung Function, And Inflammation Of Human Cystic Fibrosis

https://tinyurl.com/tn59buke

Mouse models of CF have been used to study chronic lung infections; however, these models have lacked the airway mucus that defines human CF pathophysiology and required the use of mucoid Pseudomonas aeruginosa. Here, researchers combined alternative models and established a chronic P. aeruginosa lung infection model to recapitulate nutrient and mucus characteristics of the human CF lung environment and test the effects of chronic infections on bacterial burden, lung function, and the immune response. Using wild-type SCFM2-C57BL/6 mice as controls, SCFM2-Scnn1b-Tg mice failed to clear bacterial infections, and lung function measurements showed that infected the mice had decreased inspiratory capacity and compliance, elevated airway resistance, and significantly reduced forced expiratory volumes. Flow cytometry and cytokine arrays showed that, like people with CF, the mice developed inflammation characterized by neutrophil and eosinophil infiltration and Th2 lymphocytic cytokine responses. Chronically infected mice developed an exacerbated mix of innate and Th1, Th2, and Th17-mediated inflammation, causing higher lung cellular damage and elevated numbers of unusual Siglec F+ neutrophils. These types of mice will be useful for investigating bacterial pathogenesis by non-mucoid P. aeruginosa, including treatments and the roles of Siglec F+ neutrophils in CF inflammation.

Vancomycin Monitoring for Treatment of Acute Pulmonary Exacerbations of Adult Cystic Fibrosis Patients

https://tinyurl.com/294sej5a

Therapeutic drug monitoring (TDM) for vancomycin (VAN) in adult pwCF historically has utilized trough concentrations. Recent VAN TDM guidelines recommend area under the curve (AUC) monitoring to reduce the risk of acute kidney injury (AKI), despite limited evidence to support this practice in adult pwCF. Receipt of concurrent nephrotoxins was more common in the AUC cohort than in the trough cohort, but the rate of AKI was similar. AUC monitoring was associated with earlier achievement of TDM goal vs. 2 days, lower total daily doses vs. 57.5 mg/kg/day, and fewer regimen changes vs. 2 changes. In patients with MRSA, pulmonary function recovery, readmission, and mortality were similar. In adult pwCF, the incidence of AKI was similar between AUC and trough monitoring cohorts; however, AUC monitoring achieved therapeutic targets sooner with fewer regimen modifications without significantly increasing the number of concentrations compared to trough monitoring.

Proteostasis Landscapes Of Cystic Fibrosis Variants Reveal Drug Response Vulnerability

https://tinyurl.com/35s6jp7f

Approximately 3% of persons with CF harbor poorly responsive CFTR variants. In this study researchers used affinity purification mass spectrometry proteomics to profile the protein homeostasis (proteostasis) changes of CFTR variants during correction to assess modulated interactions with protein folding and maturation pathways. Responsive variant interactions converged on similar proteostasis pathways during correction. In contrast, poorly responsive variants subtly diverged, revealing a partial restoration of protein quality control surveillance and partial correction. Computational structural modeling showed that corrector VX-445 failed to confer enough NBD1 stability to poor responders. NBD1 secondary stabilizing mutations rescued poorly responsive variants, revealing structural vulnerabilities in NBD1 required for treating poor responders. This study provides a framework for discerning the underlying protein quality control and structural defects of CFTR variants not reached with existing drugs to expand therapeutics to all susceptible CFTR variants.

Diseases Common in Persons With Cystic Fibrosis Among CFTR Heterozygotes

https://tinyurl.com/cy56wst7

CF is one of the most commonly diagnosed autosomal recessive disorders in the US. It is estimated that more than 10 million individuals are heterozygous for a pathogenic CFTR gene variant in the US (heterozygotes). The phenotypic risk of these heterozygotes is not well defined, particularly among populations of predominantly non-European genetic ancestry. Understanding disease risk across each population can improve management strategies for all. The objective of this study was to examine associations of diseases across the phenome with CFTR heterozygotes.

In this genetic association study, most heterozygotes did not appear to have a substantially higher risk of CF–associated diseases during their adulthood compared to noncarriers. Additional studies are needed to investigate the underlying factors for the elevated risk of respiratory and infectious diseases in some heterozygotes.

Clarametyx Biosciences Announces Positive Interim Analysis in Phase 2A Study Evaluating CMTX-101 for Infections Associated With Cystic Fibrosis

https://tinyurl.com/2kzkx8m2

Clarametyx announced in June that it is advancing a Phase 1b/2a clinical trial evaluating its novel immune-enabling antibody therapy CMTX-101 to treat CF-associated pulmonary infections, based on the DMC’s approval to proceed following results of a pre-specified interim analysis.

Revealing The Impact Of Pseudomonas Aeruginosa Quorum Sensing Molecule 2’aminoacetophenone On The Human Bronchial-Airway Epithelium And Pulmonary Endothelium Using A Human Airway-On-A-Chip

https://tinyurl.com/dmrkjwu

Pseudomonas aeruginosa (PA) causes severe respiratory infections utilizing multiple virulence functions. The earlier work of this team of researchers demonstrated that the Pseudomonas aeruginosa quorum-sensing molecule 2′-aminoacetophenone (2-AA) reprograms host immune and metabolic pathways, thereby facilitating the pathogen’s long-term persistence.However, its specific impact on the bronchial airway epithelium and pulmonary endothelium remains unknown. To evaluate the spatiotemporal changes in 2-AA within the human airway, considering endothelial cells as the primary point of contact when the route of lung infection is hematogenous, these researchers utilized the airway-on-a-chip platform lined by polarized human bronchial airway epithelium and pulmonary endothelium. The effects of 2-AA on epithelial and endothelial primary cells studied within a dynamic microphysiological environment that mimics the human lung airway deepen our understanding of this quorum sensing (QS) signaling molecule. This study offers novel insights into the functions and interactions of 2-AA, paving the way for innovative, cellspecific therapeutic strategies to combat Pseudomonas aeruginosa lung infections.

Antibacterial Siderophores of Pandoraea Pathogens and Their Impact on the Diseased Lung Microbiota

https://tinyurl.com/puts825z

Antibiotic-resistant bacteria of the genus Pandoraea, frequently acquired from the environment, are an emerging cause of opportunistic respiratory infections, especially in CF patients. However, their specialized metabolites, including niche and virulence factors, remained unknown. Through genome mining of environmental and clinical isolates of diverse Pandoraea species, these researchers identified a highly conserved biosynthesis gene cluster (pan) that codes for a nonribosomal peptide synthetase (NRPS) assembling a new siderophore. Using bioinformatics-guided metabolic profiling of wild type and a targeted null mutant, they discovered the corresponding metabolites, pandorabactin A and B. Their structures and chelate (gallium) complexes were elucidated by a combination of chemical degradation, derivatization, NMR, and MS analysis. Metagenomics and bioinformatics of sputum samples of CF patients indicated that the presence of the pan gene locus correlates with the prevalence of specific bacteria in the lung microbiome. Bioassays and mass spectrometry imaging showed that pandorabactins have antibacterial activities against various lung pathogens (Pseudomonas, Mycobacterium, and Stenotrophomonas) through depleting iron in the competitors. Taken together, these findings offer first insight into niche factors of Pandoraea and indicate that pandorabactins shape the diseased lung microbiota through the competition for iron.

Researchers Develop Wearable Sensor To Help Diagnose Cystic Fibrosis

https://tinyurl.com/hhdxfv2f

A group of US researchers has developed a wearable device capable of accurately tracking chloride ion levels in sweat, which is essential for evaluating hydration status and health conditions like cystic fibrosis and more. This sensor will allow for real-time tracking of an exercising person’s sweat through a hydrogel-based design that allows the device to operate with enhanced sensitivity, accuracy and efficiency, all while being reusable.

Symptom Factors And Their Clinical Correlates Among Adults With Cystic Fibrosis

https://tinyurl.com/3b349h7f

Despite CFTR modulator therapy that may dramatically alter the course of disease, many people living with CF experience co-occurring symptoms that may be interrelated and may synergistically degrade quality of life. These researchers sought to identify symptom factors, or groups of correlated symptoms, connected by underlying latent variable(s). They then examined demographic and clinical characteristics associated with these groups among PwCF. Among 262 participants, median age was 33 years, and 78 % were prescribed a CFTR modulator. Researchers identified three symptom factors: respiratory-energy, mood-gastrointestinal irritability, and pain-gastrointestinal dysmotility. High symptom severity in each factor was associated with specific demographic and clinical characteristics. CF symptom management strategies have historically focused on single-symptom approaches. Findings from this study may prompt clinicians to consider co-occurring symptoms, and ensure their assessment and management is tailored to the unique experiences of PwCF.

Successful Pregnancy in a Patient With Advanced Cystic Fibrosis Lung Disease and Burkholderia Cenocepacia Colonization

https://tinyurl.com/d4aemcmn

CF affects multiple aspects of health, including reproduction and fertility. This report details a case of a 38-year-old woman with several complications from her CF, including advanced CF lung disease, pancreatic exocrine insufficiency, asthma, and airway colonization with Burkholderia cenocepacia, who became pregnant and successfully delivered a baby boy without significant complications. This was an unplanned pregnancy 4 years after initiation of elexaftor/tezacaftor/ivacaftor therapy, a CF transmembrane conductance regulator modulator therapy that has significantly improved various health outcomes in patient populations with CF, including the reproductive health of women.

Gut Dysbiosis Driven by CFTR Gene Mutations in Cystic Fibrosis Patients: From Genetic Disruption to Multisystem Consequences and Microbiota Modulation

https://tinyurl.com/mpn6xwfw

CFTR protein modulators represent a promising approach to enhancing lower GI function in patients with CF. The aim of the review is to present the complex relationships between the presence of CFTR gene mutations and the gut microbiota dysbiosis in patients with CF. Mutations in the CFTR gene, the molecular basis of CF, disrupt epithelial ion transport and profoundly alter the gastrointestinal environment. Defective chloride and bicarbonate secretion leads to dehydration of the mucosal layer, increased mucus viscosity, and the formation of biofilms that favour microbial persistence, which together promote gut microbiota dysbiosis. This dysbiotic state contributes to impaired epithelial barrier function, chronic intestinal inflammation, and abnormal immune activation, thereby reinforcing disease progression. The interplay between CFTR dysfunction and microbial imbalance appears to be bidirectional, as dysbiosis may further exacerbate epithelial stress and inflammatory signalling. Therapeutic interventions with CFTR protein modulators offer the potential to partially restore epithelial physiology, improve mucus hydration, and foster a microbial milieu more consistent with intestinal homeostasis. The aim of this review is to elucidate the complex relationships between CFTR gene mutations and gut microbiota dysbiosis in patients with CF, with a particular emphasis on the clinical implications of these interactions and their potential to inform novel therapeutic strategies.

Patients With Cystic Fibrosis Face Higher Non-Pulmonary Comorbidity Risk

https://tinyurl.com/3z3nvbak 

Patients with CF who did not receive a lung transplant developed cardiovascular disease, kidney problems, and cancer at higher rates than those without CF. Those with CF who did receive a transplant experienced similar rate of these complications compared with transplant recipients without CF, but they occurred about three decades earlier in those with CF. According to the authors of this research, this study has clinical implications highlighting the need for CF care models to identify and address these emerging non-pulmonary comorbidities early to improve care for pwCF. It will continue to be important to monitor rates of these complications in the future, particularly in the context of increased availability of CFTR.

Brief Parent-Report Measure Of Slowness In Eating Is Associated With Weight Status In Children With Cystic Fibrosis Over A 3-Year Follow-Up

https://tinyurl.com/4vdvr4dw 

Eating behaviors are potential targets to improve outcomes including metabolic health in those with CF. These researchers aimed to test whether slowness in eating was associated with weight status over 3 year follow-up in children with CF, using the slowness in eating subscale from the Child Eating Behavior Questionnaire (CEBQ), a brief parent-report instrument. Low slowness in eating at baseline was associated with high BMIz 1, 2, and 3 years later in children with CF. The CEBQ-SE subscale could identify children who could benefit from early intervention to optimize weight status and eating behavior.

Utility of Continuous Glucose Monitors for Improved Detection of Cystic Fibrosis-Related Diabetes

https://tinyurl.com/yphb4k88

CFRD can be associated with decline in pulmonary function and nutritional status. Earlier diagnosis of CFRD than offered by annual recommended oral glucose tolerance test (OGTT) and earlier initiation of insulin may help prevent clinical decline. This retrospective study investigates the utility of continuous glucose monitoring (CGM) for detection of hyperglycemia in patients with CF. Multiple CGM measures correlated with components of the OGTT. Across glucose-tolerance groups, significant differences were observed for the OGTT 2-h glucose, mean of daily differences from CGM, and standard deviation from CGM. Approaching significance was the lability index from the CGM data. Glucose management indicator, continuous overlapping net glycemic action, glycemic risk assessment in diabetes equation, and average daily risk range showed negative correlations with change in forced expiratory volume over 1 (FEV1) over the year before OGTT. Markers of glycemic variability may be important variables distinguishing between degrees of abnormal glucose tolerance, including CFRD. This area warrants further research with a larger sample size.

A Pilot Study Of Ultra-Low-Dose Chest CT Combined With Co-Production In Cystic Fibrosis Care

https://tinyurl.com/2z8een9y

This pilot study determined whether ultra-low-dose chest tomography (ULDCT) is a feasible tool to assess structural airway abnormalities in adolescents and young adults with CF taking elexacaftor/tezacaftor/ivacaftor (ETI). These researchers explored if reviewing ULDCT findings with PwCF would impact adherence and satisfaction with airway clearance therapy (ACT). In all, 20 subjects completed baseline questionnaires and 17 completed ULDCT and post-ULDCT surveys. Findings revealed 13 subjects had bronchiectasis. Baseline surveys revealed 50% of participants reported not completing ACT the week prior to enrollment. Post-ULDCT, 82% reported completing ACT in the prior week. Reported unintentional and purposeful nonadherence to ACT decreased post-ULDCT. ULDCT is feasible for assessing structural lung abnormalities in adolescents and young adults with CF taking ETI. Incorporating ULDCT with co-production techniques may improve patient satisfaction and align the treatment goals between PwCF and their care team.

Relationship Between Skeletal Muscle Indicators And Clinical Outcomes In Cystic Fibrosis Is Modified By Adiposity And CFTR Modulator Use: A Cross-Sectional Analysis

https://tinyurl.com/yt62nc9s

Muscle mass predicts clinical outcomes in people with PwCF, but muscle quality remains insufficiently characterized. Furthermore, although CFTR modulators promote weight gain, their impact on the relationship between muscle and clinical outcomes is unknown. The objective of this study was to quantify the relationships between skeletal muscle indicators and clinical outcomes in adults with CF and determine whether CFTR modulators and/or adiposity influence the relationships. The muscle mass, strength, and quality exhibited are lower in PwCF than in controls, with muscle function linked to glucose tolerance. Lung function relationships with muscle mass differ by CFTR modulator use, and excess adiposity attenuates strength-related functional benefits. This study highlights the need for personalized approaches to CF management.

Dyspnea-Related Kinesiophobia As A Barrier On Cystic Fibrosis: The Role Of Children And Parents

https://tinyurl.com/3vy6a2py

Children with CF may be physically inactive due to disease progression and infection risk, with parents sometimes being overly protective. This study aimed to explore the relationship between dyspnea-related kinesiophobia in children and their parents and the child’s respiratory muscle strength, physical activity, fitness, and quality of life. Children with CF and their parents show high levels of dyspnea-related kinesiophobia. In children, it was associated with lower respiratory muscle strength and reduced quality of life, while parental dyspnea-related kinesiophobia was more strongly associated with the child’s physical activity, fitness, and quality of life. Parental dyspnea-related kinesiophobia may be more closely related to the child’s functional abilities than the child’s dyspnea-related kinesiophobia, highlighting the potential value of addressing psychosocial factors in CF rehabilitation.

Arterialized Oxygen Tension And Unfavorable Clinical Outcomes In Pediatric Cystic Fibrosis

https://tinyurl.com/3nwu22h4

Maintaining good lung function is a primary goal in managing CF. As spirometry lacks sensitivity for detecting mild lung disease, early progression often remains unrecognized. To overcome this limitation, more sensitive monitoring tools are needed. Researchers evaluated arterialized oxygen tension (pO2) as an easily accessible, and widely applicable surveillance method. Early abnormal pO2 at age 5 significantly correlated with accelerated FEV1 decline and a greater probability for CF-related complications. Implementing arterialized oxygen tension may offer valuable insights beyond spirometry alone in identifying high-risk patients.

“All My Food Is Customized*”: Barriers & Facilitators Concerning Nutrition For Persons Living With Cystic Fibrosis

https://tinyurl.com/44hxpfst

Maintaining optimal nutrition is often an important concern for PWCF. With the introduction of effective modulators, the focus has now shifted from preventing malnourishment to instead stabilizing weight and preventing further weight gain. Research on how PWCF experience and manage their nutrition in this new era remains limited. Understanding these supports and challenges is essential for developing effective and personalized nutritional strategies for PWCF. Enhancing access to knowledgeable dietitians, simplifying nutritional recommendations, and addressing cost barriers can significantly improve nutritional outcomes. Future research should focus on applicable solutions that utilize existing successful strategies while addressing common barriers many with CF face.

Real -World Impact Of Previous Exposure To CFTR Modulators On Clinical Parameters And Chronic Sinus Disease In People With Cystic Fibrosis On Elexacaftor-Tezacaftor-Ivacaftor

https://tinyurl.com/43327xhu

ETI therapy is associated with improved %predicted FEV1 and BMI and decreased Pseudomonas and MRSA colonization in pwCF. With ETI therapy, sinus CT scans in pwCF showed improvement using both LM and SL scoring systems. These changes were independent of previous exposure to CFTR modulator therapies.

Medication Reconciliation And Drug Interactions In Adult Cystic Fibrosis Patients Attending An Outpatient Clinic: A Cross-Sectional Study

https://tinyurl.com/26ejmd9d

Outpatient medication reconciliation (MR) studies in adult CF patients are scarce. This project aims to evaluate outpatient MR in adult CF individuals and to identify the prevalence of discrepancies and describe associated factors. Additionally, the study examines drug-drug interactions (DDI) and challenges in accessing medications. It’s a cross-sectional study with prospective data collection, involving 65 participants. The median number of medications used was 9. Thirty-five patients used medications prescribed only by the CF outpatient clinic, while 30 also had external prescriptions. Fifty-two patients engaged in self-medication. Fourteen patients had serious DDIs, and 26 had type D DDIs. External prescriptions were significantly associated with serious DDIs. Most patients reported difficulty accessing medications. A high prevalence of discrepancies was observed, linked to the number of medications and self-medication.

A Phase IIa, Single-Blind, Placebo-Controlled, Parallel-Group Study to Assess Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Brensocatib in Adults with Cystic Fibrosis

https://tinyurl.com/bdeccm9s

Brensocatib, an oral, competitive, and reversible inhibitor of dipeptidyl peptidase 1, reduces exacerbations and lung function decline in non-cystic fibrosis bronchiectasis. This study aimed to evaluate the pharmacokinetics, pharmacodynamics, safety, and tolerability of brensocatib in adults with CF, comparing these findings with data from previous trials in healthy adults and in those with NCFBE to inform dose selection for future clinical trials. Brensocatib demonstrated consistent PK profiles independent of CFTR therapy and comparable to those of healthy and NCFBE adults. Brensocatib reduced blood and sputum NSP levels. The safety profile was comparable to previous studies, with no new safety concerns identified, supporting the use of similar dosing for adults with CF as for other populations. These findings advocate for further investigation of brensocatib in CF.

Cystic Fibrosis (CF) and Modulators Based Therapy (CFTR) In Oral And Salivary Perspectives: A Single Center Cross-Sectional Study

https://tinyurl.com/3zxeyhbj

Imbalance in the oral environment due to course of CF (comorbidities), behavioral (acidic diet, poor oral hygiene), pharmacotherapeutic (hyposalivation, salivary composition) factors need to be tackled with medical insights to take control of destructive behaviours. The effects of reduced mucin levels after CFTR modulator therapy should be interpreted as beneficial for oral health.

Regardless of new modulator therapy in CF, the lack of proper oral health care indicates the need for tailored interventions related to oral health home care and nutrition counseling.

Safety, Feasibility And Efficacy Of Exercise As An Airway Clearance Technique In Cystic Fibrosis: A Randomised Pilot Feasibility Trial

https://tinyurl.com/4tjyv5fv

The objective of this trial was to test the feasibility and safety of exercise as an airway clearance technique (ExACT) for pwCF versus usual care. Testing of the primary hypothesis within a feasibility trial showed ExACT to be a safe, acceptable and feasible intervention for pwCF. These data support advancement to a definitive, longer-term, multisite trial evaluating the safety, efficacy and cost-effectiveness of ExACT, following minor refinement.

The Impact of Azithromycin on Lung Function in Children And Adolescents with Cystic Fibrosis: A Systematic Review And Meta-Analysis

https://tinyurl.com/5auxbj6u

This study aims to evaluate the effects of azithromycin on lung function in children with CF through a systematic review and meta-analysis of randomized controlled trials (RCTs). The study primarily focuses on its impact on FEV1, FVC, and the progression of lung function decline. Azithromycin shows potential in improving FEV1 and slowing lung function decline in children with cystic fibrosis, likely through its anti-inflammatory and immunomodulatory effects. Further large-scale studies are warranted to confirm its long-term efficacy, evaluate safety, and optimize treatment strategies, including potential combination therapies.

Mental Health And Transplantation In Cystic Fibrosis

https://tinyurl.com/zkzejtha

People with CF and their caregivers may face the prospect of lung or liver transplantation as CF progresses. Despite the links between psychological distress, poor adherence and survival outcomes, their mental health needs may not be consistently addressed. These researchers conducted a narrative review of mental health aspects of transplantation in PWCF, including health-related quality of life (HRQoL), pre-transplant psychosocial evaluation, neuropsychiatric complications, and psychosocial and psychopharmacologic interventions. As CF progresses, patients and caregivers require preparation for the psychosocial aspects of transplant evaluation,

including the salience of social support, treatment adherence, and the importance of early interventions for mental health and substance use disorders. Specialists in mental health, palliative care, and pain management can be enlisted to improve symptoms and functioning in PWCF and caregivers at all stages of the transplant process. Psychological and psychopharmacologic interventions may require adaptation to target the specific needs of PWCF with advanced disease.

Diet Habits And Body Composition In People With Cystic Fibrosis Under Elexacaftor/Tezacaftor/Ivacaftor Treatment - A Longitudinal, Observational Study

https://tinyurl.com/mr3x52fy

Malnutrition is a significant concern in people with CF. Historically, nutritional goals have focused on increasing energy intake and body weight, with less attention to energy sources. This study evaluates diet habits and body composition in pwCF under elexacaftor/tezacaftor/ivacaftor (ETI) treatment. This is a longitudinal, observational study in adult pwCF using ETI medications in Western Norway. The nutritional status in 27 pwCF using ETI treatment was assessed using dietary assessments and measurements of anthropometrics, body composition and handgrip strength. Post-ETI treatment, mean body mass index increased by 1.2 kg/m2, with more pwCF classified as overweight than underweight. Few pwCF had fat-free mass index, appendicular skeletal muscle mass, or handgrip strength below sarcopenia thresholds, and mean values aligned with general healthy population references. Dietary intake of saturated fat exceeded the daily recommended intake. Although weight and BMI improved following ETI treatment, proactive measures are imperative to address the current trends of weight gain, elevated FM%, and dietary patterns inconsistent with recommendations. Close monitoring and interventions to prevent excessive weight gain and to improve diet quality may become essential.