Exploring the Lived Experiences of Individuals with Cystic Fibrosis After the Implementation of Trikafta® for Treatment.

Exploring the Lived Experiences of Individuals with Cystic Fibrosis After the Implementation of Trikafta® for Treatment. | Research Square window.SnipcartSettings = { analytics: { enabled: false } }; (function() { var accessVector = localStorage.getItem('access_vector') || ''; window.dataLayer = window.dataLayer || []; if (accessVector) { window.dataLayer.push({ user: { profile: { profileInfo: { snid: accessVector } } } }); } })(); (function(w,d,s,l,i){w[l]=w[l]||[];w[l].push({'gtm.start':new Date().getTime(),event:'gtm.js'});var f=d.getElementsByTagName(s)[0],j=d.createElement(s),dl=l!='dataLayer'?'&l='+l:'';j.async=true;j.src='https://www.googletagmanager.com/gtm.js?id='+i+dl;f.parentNode.insertBefore(j,f);})(window,document,'script','dataLayer','GTM-K279D39R'); Browse Preprints In Review Journals COVID-19 Preprints AJE Video Bytes Research Tools Research Promotion AJE Professional Editing AJE Rubriq About Preprint Platform In Review Editorial Policies Our Team Advisory Board Help Center Sign In Submit a Preprint Cite Share Download PDF Research Article Exploring the Lived Experiences of Individuals with Cystic Fibrosis After the Implementation of Trikafta ® for Treatment. Alyssa Riggle, Annelise Page, Erica Roesch, Marsha Michie, Rebecca Darrah This is a preprint; it has not been peer reviewed by a journal. https://doi.org/ 10.21203/rs.3.rs-7989348/v1 This work is licensed under a CC BY 4.0 License Status: Posted Version 1 posted You are reading this latest preprint version Abstract Background: CFTR-modulators are a class of medications prescribed to people with cystic fibrosis (pwCF) who have certain genetic mutations. In 2019, Trikafta ® was approved by the FDA and lead to significant health improvements for pwCF including increased pulmonary function, a decrease in sweat chloride levels, and an overall increase in health-related quality of life. Studies done on prior CFTR-modulators have found that modulator therapies can not only impact the physical health of pwCF, but also psychological and social aspects of life. Despite Trikafta ® showing significant health improvements compared to prior modulators, little is known about how Trikafta ® impacts the lived experience of pwCF. The purpose of this study is to explore how the implementation of Trikafta ® to cystic fibrosis treatment has impacted the lived experience of pwCF and what additional resources healthcare providers should be offering to their patients Methods: Nine men and women with CF who were over the age of 18 and have been on Trikafta ® for at least a year were recruited from an adult CF center. A semi-structured interview guide was used to conduct interviews over the Zoom platform. The data were then analyzed through inductive coding rooted in grounded theory. Results: Four themes were constructed from the data in this study: (1) “transformation in health” refers to the change in physical health the participants have noticed since starting Trikafta ® , (2) “future planning and opportunities” highlights new opportunities becoming available and a new desire to plan for the future, (3) “impact on relationships” describes how participants felt their relationships with their partner, family, and care teams, were impacted by Trikafta ® , (4)“new worries and struggles” describes new struggles and concerns that participants have faced. Conclusions: The data from this study provide evidence that Trikafta ® has not only impacted pwCF on a physical level, but may also have an impact on relationships, mental health, and future planning. It also highlights the need for additional resources that CF care teams should be providing to their patients such as mental health resources, increased financial support, and shifting to a preventative care approach as pwCF grow older. Cystic fibrosis Trikafta® (elexacaftor/tezacaftor/ivacaftor) Lived experience CFTR modulators Physical health Mental health Self-identity Relationships Future planning Healthcare needs Background Cystic fibrosis (CF) is one of the most common life-threatening monogenetic diseases, particularly in populations of Northern European descent. 1 CF is caused by variants in the CF transmembrane conductance regulator (CFTR) gene which results in dysfunctional or absent CFTR protein. 2 – 4 Despite there being hundreds of different disease-causing mutations seen in people with CF (pwCF), the most common is the Phe508del CFTR mutation. Around 90% of pwCF have at least one copy of the Phe508del mutation 5 , 6 Historically, CF was once considered to be a childhood fatal disease. In the 1930s,85% of infants diagnosed with CF were expected to die before their second birthday. 7 Over time, improvements in medical management, newborn screening, and drug therapies changed CF from a fatal disease to a chronic condition, with more adults than children now living with CF. 8,9 Most people with cystic fibrosis begin to experience symptoms shortly after birth, including respiratory disease, pancreatic insufficiency, poor weight gain, excessive salt loss, and male infertility. Respiratory disease and chronic lung infections are the most concerning for pwCF as it is the most frequent cause of lung-transplantation or death in early adult life. 1 Treatments initially targeted CF symptoms: enzyme supplementation for pancreatic insufficiency, antibiotics to prevent or treat lung infection, and mucolytics and airway clearance techniques to help remove mucus from the airway. However, recent CFTR modulator therapies target the underlying defect in CF, the absent or dysfunctional CFTR protein. CFTR modulators include the potentiator ivacaftor and correctors lumacaftor, tezecaftor, elexacaftor and vanzacaftor. 10 Potentiators correct the CFTR channel so that more ions can flow through while correctors help improve the CFTR folding and allow it to be trafficked to the surface of the cell. 2 In 2019, the combination of the drugs elexacaftor/tezacaftor/ivacaftor (ETI), otherwise known as Trikafta®, was approved by the FDA for individuals who have at least one Phe508del allele or other qualifying mutation, potentially benefiting up to 90% of pwCF. 5 , 11 , 12 A clinical trial studied pwCF who took Trikafta® for 24 weeks compared to a placebo group. Participants who were on Trikafta® experienced a significant improvement in FEV 1, sharp decrease in sweat chloride concentration, decrease in pulmonary exacerbations, improvements in quality of life, and improvements in BMI. Although side effects were reported in a subset of the participants who were on Trikafta®, the severity of the events were milder compared to prior CFTR modulators. 5 The introduction of Trikafta® to CF treatment led to major improvements in the clinical health of pwCF. 5 , 11 , 12 However, although majority of pwCF taking Trikafta® report positive outcomes on their health, there are still a subset of individuals whose health has worsened. One side effect reported by pwCF taking Trikafta® is neuropsychiatric changes including depression and anxiety. In a recent survey, healthcare providers from CF care centers reported that while a majority of their patients had positive health outcomes from Trikafta® such as reductions in cough, fewer pulmonary exacerbations, fewer hospitalizations, improved lung function, and increased optimism, a subset of patients had negative health outcomes, including difficulty sleeping, brain fog, memory difficulties, depression, anxiety, and mood swings. 12 Participants also reported that patients shared concerns about attending college, job opportunities, food insecurity, copays, marriage and financial planning. Although this study identified some of the negative health outcomes that pwCF are experiencing from Trikafta®, it is still unknown whether these effects are related to only the medication itself or may also be related to life changes pwCF have experienced since being on Trikafta®. Studies conducted prior to the introduction of CFTR-modulators have found that the illness-related experiences pwCF face can play a profound role in their lives. 7 , 13 , 14 After the introduction of CFTR-modulators such as ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), and tezacaftor/ivacaftor (Symdeko®), studies explored how patient’s lived experience was impacted by modulator therapy. One qualitative study explored relationships between CFTR-modulator drugs and the psychological and social aspects of the lives of individuals with cystic fibrosis including career, family planning, relationships, and psychological functioning. 10 The study found several overlapping themes: stability of physical symptoms; changes in self-identity; desires and motivations to achieve new goals; and hope for the future with hope being the most prominent theme. 10 With little data being present about the lived experiences of young adults with CF, this study provided evidence that targeted CFTR-modulator treatments may have negative impacts on the mental, emotional, and social functioning of pwCF. However, at the time of that study Trikafta® was not yet approved for treatment by the FDA. Compared to previous modulator therapies, Trikafta® has shown many more significant health improvements for pwCF with fewer adverse health outcomes. Therefore, it is possible that Trikafta® may have a stronger impact on lived experiences of pwCF compared to prior CFTR modulators. 10 Another study in the United Kingdom examined the psychological implications and health risks of cystic fibrosis pre- and post elexacaftor/tezacaftor/ivacaftor modulator therapy. The study found that while many patients “felt lucky to be living”, they still identified significant challenges identified in regard to their psychological well-being. 9 The study did not explore what individuals felt was contributing to their mental health challenges or what resources they had used to address these challenges. Despite prior studies showing how CFTR-modulators impact the lived experience of pwCF, little data is present that explores the healthcare needs pwCF are facing after the approval of Trikafta ® in 2019 and what, if any, resources may be lacking for this population. The purpose of this study is to explore how the addition of Trikafta® to cystic fibrosis treatment has impacted the lived experiences of people with cystic fibrosis and to identify additional resources healthcare providers should offer. Understanding how Trikafta® has influenced these experiences can help reveal emerging needs within this population. By assessing current support systems and identifying gaps, this study aims to investigate the impact of Trikafta on the lived experience of pwCF and guide healthcare providers in adapting care and resources to best suit their patient’s needs. Methods Study design This study used qualitative methodology based on principles drawn from grounded theory. Grounded theory is a qualitative approach that uses data to construct new theoretical constructs, rather than interpreting data using a prior framework 15 Recruitment and Participants Study participants were recruited from a CF care center in the midwest between September 2024 and January 2025 dates. A list of potential subjects was generated from the Cystic Fibrosis Foundation Registry using the following inclusion criteria: diagnosis of cystic fibrosis, age 18 years or older, English speaking, and treatment with Trikafta® for at least 1 year. Potential subjects were contacted by electronic health record messaging (MyChart) or in-person during one of their regularly scheduled visits to the CF clinic. For participants who were recruited through MyChart, an initial message was sent that included details of the study and next steps if they were interested in participating. A consent form was then sent electronically to the participant to print out and sign and an interview over the Zoom platform was scheduled (see supplementary material). For individuals who were recruited in person, the researcher provided information about the study, obtained written consent and contact information from participants, and scheduled an interview over the Zoom platform. Data Collection Semi-structured interviews used an interview guide focused on the aims of this research (see supplementary material) Each question in the interview guide was targeted towards understanding the individual’s lived experience, needs, and resources that they found helpful/lacking. Interviews were conducted over the Zoom platform and recorded with permission from the participant. Interviews ranged in length from 35 to 72 minutes with an average time of 51 minutes. All data collected from participants was stored securely on University Hospital’s REDcap system. The study was approved by the Institutional Review Board of University Hospitals Cleveland Medical Center (STUDY20240547). Data Analysis Each interview was recorded and a transcript was autogenerated by Zoom. Each transcript was reviewed by the firth author to correct any errors in the auto-generated transcript and edited to remove filler words and repeated words to increase clarity and readability. A number was assigned to each interview transcript, and any identifying information was removed. Once the transcripts were completed, they were coded electronically using the Dedoose qualitative analysis software. The coding process for the interviews was rooted in principles derived from grounded theory and used inductive coding methodology to create codes and themes based on the raw data. Coding began by going line-by-line through each interview and was then followed by reanalysis of each interview to implement newly created codes. A codebook was developed by the researcher that was edited and tested as coding continued. A separate researcher reviewed two of the transcripts using the final codebook to ensure consistency. Coded excerpts were grouped into themes for further analysis. Results Sample Demographics One hundred and twenty-one individuals met the inclusion/exclusion criteria. All 121 individuals were contacted through MyChart a maximum of three times with follow-up messages sent to individuals who expressed initial interest. In addition, the firth author attended CF clinics to aid in recruitment. Nine participants who met the inclusion criteria for the study agreed to participate. Written informed consent was obtained from all nine participants. Six participants were recruited through MyChart while three participants were recruited in-person during a scheduled CF appointment. Participants’ ages ranged from 19 to 66 with five participants being between the ages of 18–35 and four participants being over the age of 36. The data are presented in four themes: (1) transformation in health, (2) future planning and opportunities, (3) impact on relationships, (4) new worries and struggles. Additional demographic data for the participants can be found in Table 1 which is located on page 6. Table 1 Demographic data of participants Participant Sex Assigned at Birth (M/F) Age Race Relationship Status Children (Y/N) Currently Pregnant (Y/N) On prior modulators 1 F 31 White Married N N No 2 F 34 White Married Y N Yes, Orkambi and Symdeko 3 F 19 White Single N N No 4 M 44 White Single N N Yes, Symdeko 5 M 66 White Married N N No 6 F 26 White Married N Y Yes, Symdeko 7 M 24 White Single N N No 8 M 56 White Married Y N No 9 F 51 White In a relationship N N Yes, Kalydeco Theme 1. Transformation in Health The theme “transformation in health” refers to changes in physical health that participants noticed after starting Trikafta®. Throughout the interviews, participants expressed positive changes including improved lung function, easier weight gain, decrease symptom burden and fewer hospitalizations. Some subjects also noted side effects from the medication affecting their physical health. One participant shared: Since [starting Trikafta®] I've had probably within the first year gained 15 to 20% of my lung function back. I gained, I think, around 15 pounds or so that first year that I was on it. And then I went a whole year without having IV antibiotics which was kind of crazy for me. It was the longest I had gone since middle school maybe. (Participant 2) Some participants mentioned decreased symptom burden after starting Trikafta®. Yeah, since then I rarely cough. I rarely am wheezing. Only when I'm sick am I wheezing or crackling, which it used to be very common. More often than not I would be wheezing. I know we did a sweat test too. My salt levels are way down everything like that... and since then I've been able to lower how often I'm doing my breathing treatment. (Participant 3) Some participants not only noticed a change in their lung function, but also an increase in their ability to do physical activities. But I do feel like it is just easier to do activities. So laundry or going upstairs, like that kind of stuff. Making my bed, all of that would make me winded... Now I can go get laundry downstairs, go upstairs, make my bed, get food, all of that in an hour and it sounds silly that it is really cool that I can do that better and faster than before. (Participant 6) Some participants who had taken other CFTR modulators prior to Trikafta® compared the changes in physical health they have experienced on both medications. “Yeah, I mean you can't even compare them. Like the prior modulators, they gave you maybe 5% of what Trikafta did. Like you felt a little more stable. You could tell maybe you didn't cough as much or you gained weight a little bit easier but it was night and day from Trikafta. Trikafta was like an immediate reaction. You felt your lungs clearing out. I mean, people gained like me 10 pounds in a week. It was just like everything your body had been deprived of suddenly it was able to do.” (Participant 2) However, some participants reported experiencing side effects while taking Trikafta ®. Four years, maybe five years in, I realized I was having memory issues. So details are not important to me because they'll drive me crazy. Just like if you ask me something and then somebody says, “Well, what did she ask you?” I won’t remember. (Participant 9) Like this summer I suddenly out of nowhere had super high blood pressure for a couple of months. And I was in and out of the ER with high blood pressure. And we think it might be from Trikafta but obviously we don't know because it's never been documented. And yeah, so it's just like.....you kinda have to keep rolling with the punches and figuring out what's happening. (Participant 2) Theme 2. Future Planning and Opportunities Many participants expressed a new desire to plan for the future since starting Trikafta®. Participants mentioned opportunities that have become available to them such as starting a family, changing careers, or preparing for retirement. One participant shared: I would say that the biggest improvements have been less worrying about the future. You never know. That’s something that I do worry about, you know, what's going to happen down the road and stuff like that. But it's a little bit easier to make better long-term plans now than before. (Participant 4) Another participant shared being able to switch to a more physically demanding career since starting Trikafta®, which has brought him a new sense of purpose. Like hooking my feet onto a ladder and swinging through houses or climbing up in attics without a single thought is definitely not something I could have done. That would have winded me a long time ago. But I also really have a hard time saying like what I could do and what I couldn't do cause I was lucky enough to get it [Trikafta] just as I was hitting the point where I was really getting serious about working and being an adult and actually having to do things. (Participant 7) Participant 8 shared he has begun to think about retiring from his career and pursuing other interests. Well, yeah pre-Trikafta I was wondering how much longer I'd be able to work. But since Trikafta I'm thinking about retiring actually this year from [job]... and then just do something different. Maybe back into nursing, but maybe it's just something totally different. (Participant 8) Another participant expressed hope that she will be able to conceive children and carry a pregnancy. My doctors, even before all the modulators, they were like “We can try our best. Here's what we know.” And so they were always hopeful that it would happen. And then of course with Trikafta, they actually talked to me once I was on Trikafta and they knew I was married like, “Hey, just a heads up Trikafta is actually kind of causing a baby boom. If you don't want kids like just know that.” And I was like, oh, that's good to know that it is causing a lot more pregnancy. (Participant 6) Theme 3. Impact on Relationships Participants described impacts on their relationships with their partner, family, and care teams since starting Trikafta®. For example, one participant shared the positive impacts her improvements in physical health have had on her relationship with her husband. So I started Trikafta when I was with my husband. We were still dating at the time. But with him, I think he's more just seen a difference in me, like a positive aspect of “Yeah, you feel like you can do more activities” and he that we were dating when I used to get so sick every other week and it's kind of nice, I'm sure for him. Not that he minds taking care of me but it's hard to see someone that you love like go through that all the time so he's like, “Oh, that's so nice that you get to feel more healthy. (Participant 6) Some participants discussed an improved ability to begin a romantic relationship. So actually, ironically, with meeting so many people through these apps right now, cause, nothing's really stuck, my thing used to just be like, “Hey, I have cystic fibrosis.... I can happily educate you about it. If that's a deal breaker, though I completely understand.” Ironically, that's changed now to the same thing, but I used to not be healthy. But I'm healthier than I've ever been and there's still downsides, but it's significantly lessened. (Participant 7) Some participants shared how Trikafta® has not had a major impact on their relationship with their family members. Yeah, I'd say it has a bit, just because I don't have to do as much maintenance and treatments day to day. So I'd say it's changed a bit, but I don't think there's any major changes since starting Trikafta in the family dynamic. (Participant 3) Several participants also mentioned how Trikafta® did not have a strong impact on their relationship with their healthcare team. I wouldn't say it's changed our relationship, but we have to focus now on different things, if that makes sense. Like we're not so concerned about keeping my lungs super healthy. We're concerned more about big picture stuff. (Participant 2) I started out from almost day one, Dr. [Name] was my doctor. So as far as a relationship goes, I mean, he’s my doctor. I didn’t really think too much beyond that. (Participant 5) Theme 4. New Worries and Struggles Participants also highlighted a wide range of struggles and new concerns after starting Trikafta® including finances, changes in self-identity, and fear of new health concerns arising. For example, one participant shared having a difficult time adjusting to her “new normal”. Like when you have your whole life you're told, “This is what your life is going to be like, this is what your life expectancy is going to be like,” and you kind of plan. And then when something like this comes out that can substantially, you know, much longer......you're going to live and you're going to like.......you could do all these things that you didn't think maybe before you could, so it's very hard adjusting. (Participant 1) Another participant expressed fear over developing cancer in the future now that her lung health is no longer her biggest concern. Also that both my parents have died from cancer and suddenly I'm going to continue to get older and that probably is the reason that I will pass away. Like I've started thinking about that as well. Yeah, I think that's the biggest, the biggest worries that I have. (Participant 2) Some participants shared concerns about the cost of Trikafta® and fear of losing disability payments. That is the one thing with Trikafta that I worry about is am I going to be told that I am no longer considered disabled. Because I have read that there have been people that they are telling that to. And then at my age you're going to tell a 51-year-old lady that she has to now go find a career. And if I was to get really sick and need a disability again, I would have to fight for it. It's not something they just put you back on. (Participant 9) Thank god they passed the ADA and thank god the state has some helping because Trikafta is ungodly high. So that is paid for. If it wasn’t, I wouldn’t be taking it. (Participant 5) Other participants expressed concerns for their finances, including a lack of financial planning for the future. Yeah because I mean, it's not funny, but I tell my husband “Well, I didn't think I was ever going to retire so I never saved anything”. I mean, honestly, so now it's like “Oh, well, crap, maybe I will work until I'm maybe 65.” So now I should probably start saving. So yeah, that's definitely a big switch with Trikafta too, because before I was like “I don't have to worry about it. (Participant 1) Another participant discussed having to advocate for herself when receiving obstetric care. Yeah, so obviously OBGYN care is a struggle when you have CF because.......and it's even gotten worse because of that big baby boom after Trikafta....they just aren't. Thankfully, we have a really good maternal fetal medicine team at UH that has some experience with CF but it is just crazy. Like they don't know how to treat people with CF. There's a lot of advocating you have to do for yourself. (Participant 2) Discussion The purpose of this qualitative study was to explore how the implementation of Trikafta® to cystic fibrosis treatment has impacted the lived experiences of people with cystic fibrosis and what additional resources healthcare providers should be offering to their patients. From the data collected from this study, four themes emerged: transformation in health, future planning and opportunities, impact on relationships, and new worries and struggles. These findings contribute to the growing body of literature on how CFTR-modulators can impact pwCF beyond clinical health. Participants expressed overwhelmingly positive changes in their physical health that they attributed to being on Trikafta®. Many participants described improvements in lung function, less frequent hospitalizations, decreased coughing, and the ability to perform more physical activities. The positive changes in health reported by participants align with previous interventional and observational studies in pwCF taking Trikafta that showed significant increases in FEV 1 , decreases in sweat chloride concentration, decreases in the amount of pulmonary exacerbations, increases in BMI, and an overall increase in health related quality of life. 8,11 An observational study conducted in 2021 for pwCF Trikafta® found similar results and highlighted the clinical benefits that pwCF experienced after being on Trikafta® for 6 months. 11 Participants in this study who were on prior CFTR-modulators also emphasized that the considerable improvements to their health that they observed from Trikafta® far exceeded those from prior modulators they were on. Overall, participants reported not only stabilizing effects of Trikafta® on health, but also significant improvements in physical health. However, several participants mentioned side effects that they believe to be related to treatment with Trikafta®, including challenges with memory and incidents of high blood pressure. Prior research has found that some pwCF experience neuropsychiatric side effects such as brain fog, sleep disturbances, and mood changes. 13 More research is needed to determine the impact that Trikafta® has on other body systems. Study participants also discussed the ways in which Trikafta® opened the door for new opportunities and created a new desire to plan for the future. Some participants described pursuing new careers or being able to work physically demanding jobs, while others described planning for retirement or starting a family. The shifts that participants had toward long-term planning aligns with prior qualitative studies observing the effect of CFTR-modulators on the lived experience of pwCF. 10 , 16 Therefore, the ability for participants to envision and plan for a future that was previously deemed uncertain sheds light on the similar psychosocial aspects Trikafta® has on pwCF. Throughout the interviews, participants reported variable effects on how Trikafta® impacted their personal relationships. While participants reported that Trikafta® had a positive impact on their dating life and romantic relationships, relationships with family members and CF care teams remained largely unchanged. These findings partially align with results from previous studies that found improvements in friendships and romantic relationships due to participants having increased energy and less frequent hospitalizations. 4 Another study conducted in the United Kingdom explored how ETI therapy (elexacaftor/tezacaftor/ivacaftor) impacted the relationships of pwCF with their CF care teams. The study found mixed results: participants discussed their CF care teams not taking their input seriously, a lack of support around changes to self-identity, and a need for routine psychological support at every CF visit. 19 These findings suggest that although Trikafta® has been found to have a strong impact on physical health and lifestyle, its effects on relationships with others may be variable. The purpose of this study was not only to identify how Trikafta® has impacted the lived experience of pwCF, but also to inveestigate any new needs that pwCF are experiencing. Some of the worries or concerns mentioned by participants included the lack of financial planning for the future, fear of losing disability benefits, changes in self-identity, and the fear of new health concerns arising now that the life span for pwCF is increasing. These themes align with results from a 2023 study that found pwCF experienced new challenges including increased anxiety about life planning, struggles with finances, and uncertainty about their long-term health. 4 Similarly, individuals in our study also reported difficulties adjusting to their new identity now that they are no longer as sick as they were before. Lastly, some participants expressed having to self-advocate for themselves with other healthcare providers, including with OBGYNs lacking education on how to care for pwCF. These reported experiences align with research highlighting the need for a better transition from adolescent to adult CF care. As the number of adults living with CF continues to increase, priorities are shifting to a long-term care approach centered around monitoring chronic conditions such as CF related diabetes and liver disease, optimizing nutrition, educating about sexual and reproductive health, and evaluating mental health concerns. 17 This study highlights the need for additional psychological support and resources for pwCF as well as increased education about CF care to adult providers outside of the CF care team. Compared to other CFTR-modulator therapies, Trikafta® has shown significant improvements in the clinical health of pwCF. 5 , 11 , 12 Prior to this study, little research has investigated impacts of Trikafta® on the lived experiences of pwCF. A 2022 qualitative study of the lived experience of pwCF on CFTR-modulators, before the approval of Trikafta® by the FDA, found prominent feelings of hope among participants regarding the CFTR-modulator they were prescribed and advancing medical research. Although participants in the study did not see dramatic improvements in their physical health, they shared a sense of hope for the future and a desire to achieve new goals. Participants also reported changes to their self-identity, such as no longer seeing CF as their defining feature, as well as a new potential to accomplish specific goals. 10 Participants in our study expressed similar feelings of hope, such as hopes to start a family, switch careers, retire, and explore new interests. However, unlike the prior study in which participant’s hopes were centered around the potential for future positive health, our participants attributed their hopes to the significant positive changes in health that they had already experienced. Participants in our study had also begun taking steps to achieve these goals such as switching to a more physically demanding job, putting aside money to plan for a family, and beginning retirement planning. While participants in both studies reported some similar experiences, more research is needed to determine how Trikafta® impacts the lived experience of pwCF over time. Study Limitations This study’s findings are limited due to the small sample size of participants, which did not allow us to reach data saturation. The inclusion criteria (18 years or older, English speaking, and being on Trikafta® for at least 1 year) limited our sample since Trikafta® has only been available since 2019 and many pwCF do not qualify for this therapy. Study participants were only recruited through a single CF treatment center in the Midwest region of the US, and these findings may not be representative of the experiences of pwCF who live in other geographic areas or visit other hospital systems for their CF care. However, despite the small sample size, the participants in this study spanned diverse ages, sexes, and life experiences such as relationship status, use of prior modulators, and parenting. Lastly, the experiences shared by participants in this study only captured a moment in time of their CF journey. Perceptions and experiences of pwCF on this therapy are likely to change over time. Practice Implications Limited research has investigated how Trikafta® impacts the lived experiences of pwCF. The data from this study provides evidence that Trikafta® has impacted pwCF not only physically, but may also have an impact on relationships, mental health, and future planning. It also highlights areas in which healthcare providers can improve care for pwCF. This study highlights potential impacts of Trikafta® on mental health, including anxiety about the future, changes to self-identity, and concerns about new changes in health as pwCF get older. Although anxiety and depression screenings and management protocols are often already implemented by CF care teams, additional resources surrounding mental health and referrals to other health professionals who understand CF-related challenges may help to lessen some of these concerns. 17 Participants in the study also expressed challenges with long-term financial planning, affordability of CF care/medications, and concerns about losing disability benefits. While CF care teams often include social workers to assist with finances, it may be beneficial to connect pwCF with other professionals surrounding CF-related finances. For instance, healthcare providers may find it beneficial to collaborate with financial advisors to help assist their patients with financial planning, find payment plans for medications/care, and advocate for patients as they transition to being healthier. Some participants in the study also experienced side effects and new health concerns while taking Trikafta®. As life expectancy for pwCF has improved since the implementation of CFTR-modulators, it is important for providers to not only monitor side effects associated with the start of the medication but also shift to a lifelong approach centered around preventative care and maintaining long term health as pwCF grow older. This may include additional education to other health providers about CF-related health concerns and increasing the number of adult-care providers that are part of the CF team. Research Recommendations Research centered around the lived experience of pwCF after taking Trikafta® is lacking. Currently, around 90% of CF patients have a genotype that makes them eligible for the use of Trikafta®. 18 Therefore understanding how Trikafta® impacts the lived experience of members of the CF community is important in enhancing the care provided to these individuals. Future research should also investigate how the lived experience of pwCF is impacted for individuals who do not qualify for Trikafta® or have decided against or discontinued this treatment. Although this study adds to the body of literature surrounding the lived experience for pwCF who are on Trikafta®, this study only includes responses from a few members of the CF community. Therefore, this study is not generalizable to all individuals with CF who are on Trikafta®. A study investigating the lived experience for pwCF who are on Trikafta® in a larger population size may be able to provide insight to these experiences in a way that is more generalizable. Declarations Human Ethics and Consent to Participate This study was conducted in accordance with the Declaration of Helsini and was approved by the Institutional Review Board of University Hospitals Cleveland Medical Center (STUDY20240547). Clinical trial number: not applicable Consent for Publication Each participant in the study provided their consent to participate and signed an in-person consent form that was approved by the Institutional Review Board of University Hospitals Cleveland Medical Center (STUDY20240547). Availability of data and materials The datasets used and/or analyzed during the current study are available from the corresponding author on reasonable request. Competing interests The authors declare that they have no competing interests. Funding No funding was obtained in the conduction of this research. Acknowledgements I would like to express my deepest gratitude to my thesis committee advisor (Rebecca Darrah) and thesis committee members (Annelise Page, Erica Roesch, Marsha Michie) for dedicating their time and expertise to my research. Without their valuable feedback and encouragement, this work would not have been possible. I would also like to thank the incredible staff at Leroy W. Matthews Cystic Fibrosis Center, my graduate program co-director (Michelle Merrill), and my wonderful classmates who provided endless support and insightful ideas throughout the research process. References De Boeck K. Cystic fibrosis in the year 2020: A disease with a new face. Acta Paediatr Oslo Nor 1992. 2020;109(5):893–9. 10.1111/apa.15155 . Baroud E, Chaudhary N, Georgiopoulos AM. Management of neuropsychiatric symptoms in adults treated with elexacaftor/tezacaftor/ivacaftor. Pediatr Pulmonol. 2023;58(7):1920–30. 10.1002/ppul.26412 . Cantin AM, Hartl D, Konstan MW, Chmiel JF. Inflammation in cystic fibrosis lung disease: Pathogenesis and therapy. J Cyst Fibros Off J Eur Cyst Fibros Soc. 2015;14(4):419–30. 10.1016/j.jcf.2015.03.003 . Rafeeq MM, Murad HAS. Cystic fibrosis: current therapeutic targets and future approaches. J Transl Med. 2017;15(1):84. 10.1186/s12967-017-1193-9 . Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. N Engl J Med. 2019;381(19):1809–19. 10.1056/NEJMoa1908639 . De Boeck K, Vermeulen F, Dupont L. The diagnosis of cystic fibrosis. Presse Médicale . 2017;46(6, Part 2):e97-e108. 10.1016/j.lpm.2017.04.010 Higham L, Ahmed S, Ahmed M. Hoping to Live a Normal Life Whilst Living with Unpredictable Health and Fear of Death: Impact of Cystic Fibrosis on Young Adults. J Genet Couns. 2013;22(3):374–83. 10.1007/s10897-012-9555-1 . Ladores S, Polen M. Lingering Identity as Chronically Ill and the Unanticipated Effects of Life-Changing Precision Medicine in Cystic Fibrosis: A Case Report. J Patient Exp. 2021;8:2374373521996971. 10.1177/2374373521996971 . Keyte R, Kauser S, Mantzios M, Egan H. The psychological implications and health risks of cystic fibrosis pre- and post- CFTR modulator therapy. Chronic Illn. 2023;19(3):539–56. 10.1177/17423953221099042 . Page A, Goldenberg A, Matthews AL. Lived experiences of individuals with cystic fibrosis on CFTR-modulators. BMC Pulm Med. 2022;22(1):42. 10.1186/s12890-022-01825-2 . Bacalhau M, Camargo M, Magalhães-Ghiotto GAV, Drumond S, Castelletti CHM, Lopes-Pacheco M. Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis. Pharm Basel Switz. 2023;16(3):410. 10.3390/ph16030410 . Nichols DP, Paynter AC, Heltshe SL et al. Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial. Am J Respir Crit Care Med. 205(5):529–39. 10.1164/rccm.202108-1986OC Badlan K. Young people living with cystic fibrosis: an insight into their subjective experience. Health Soc Care Community. 2006;14(3):264–70. 10.1111/j.1365-2524.2006.00619.x . Cuneo AA, Outram S, Marsac ML, et al. Medical traumatic stress in cystic fibrosis: A qualitative analysis. J Cyst Fibros Off J Eur Cyst Fibros Soc. 2023;22(4):763–71. 10.1016/j.jcf.2022.12.008 . Chun Tie Y, Birks M, Francis K. Grounded theory research: A design framework for novice researchers. SAGE Open Med. 2019;7:2050312118822927. 10.1177/2050312118822927 . Bathgate CJ, Muther E, Georgiopoulos AM, et al. Positive and negative impacts of elexacaftor/tezacaftor/ivacaftor: Healthcare providers’ observations across US centers. Pediatr Pulmonol. 2023;58(9):2469–77. 10.1002/ppul.26527 . Singh J, Towns S, Jayasuriya G, et al. Transition to adult care in cystic fibrosis: The challenges and the structure. Paediatr Respir Rev. 2022;41:23–9. 10.1016/j.prrv.2020.07.009 . Additional Declarations No competing interests reported. Supplementary Files RiggleIRBInterviewGuide.docx Cite Share Download PDF Status: Posted Version 1 posted You are reading this latest preprint version Research Square lets you share your work early, gain feedback from the community, and start making changes to your manuscript prior to peer review in a journal. As a division of Research Square Company, we’re committed to making research communication faster, fairer, and more useful. We do this by developing innovative software and high quality services for the global research community. Our growing team is made up of researchers and industry professionals working together to solve the most critical problems facing scientific publishing. Also discoverable on Platform About Our Team In Review Editorial Policies Advisory Board Help Center Resources Author Services Accessibility API Access RSS feed Manage Cookie Preferences © Research Square 2026 | ISSN 2693-5015 (online) Privacy Policy Terms of Service Do Not Sell My Personal Information {"props":{"pageProps":{"initialData":{"identity":"rs-7989348","acceptedTermsAndConditions":true,"allowDirectSubmit":true,"archivedVersions":[],"articleType":"Research Article","associatedPublications":[],"authors":[{"id":553784635,"identity":"9759e011-a8e8-43f8-acfe-39a311c10b8c","order_by":0,"name":"Alyssa Riggle","email":"data:image/png;base64,iVBORw0KGgoAAAANSUhEUgAAAZAAAAAyAQMAAABI0h/eAAAABlBMVEX///8AAABVwtN+AAAACXBIWXMAAA7EAAAOxAGVKw4bAAAA/UlEQVRIiWNgGAWjYDACZiB+wMAgA+Z8AGI2dmK0JDAw8IDYjDNAWpiJsQmmhZkHZgg+YN7OfvlFQoUdj8H54w8/2/zaJs/HzMD44WMObi0yh3nKLBLOJPMY3Mgxls7tu23YxszALDlzG24tEsw8aQaJbcxALTwM0rk9txmBWtiYeQlq+VcPctjj35Y9t+2J0MJ++EFiw2EegwMJZtIMP24nEmMLG0PCseM8kjdyzCx7G24ntzEzNuP3C//xxx8+1FTL8QEdduPHn9u289ubD374iEcLMEbMJECUwgEgwdgGYjI24FMPBOyPQcmEQR6s7g8BxaNgFIyCUTAiAQCLVU7o9iQWBgAAAABJRU5ErkJggg==","orcid":"","institution":"Case Western Reserve University","correspondingAuthor":true,"prefix":"","firstName":"Alyssa","middleName":"","lastName":"Riggle","suffix":""},{"id":553784636,"identity":"19d14e0b-fcf1-4ab2-991b-dfb065b2bf8d","order_by":1,"name":"Annelise 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09:11:51","extension":"html","order_by":5,"title":"","display":"","copyAsset":false,"role":"acdc-reference","size":86416,"visible":true,"origin":"","legend":"","description":"","filename":"earlyproof.html","url":"https://assets-eu.researchsquare.com/files/rs-7989348/v1/1aa9911d06960dd992d0cef1.html"},{"id":99313467,"identity":"320cd8ba-5648-424d-acef-3337de4eb75d","added_by":"auto","created_at":"2025-12-31 16:20:13","extension":"pdf","order_by":0,"title":"","display":"","copyAsset":false,"role":"manuscript-pdf","size":615720,"visible":true,"origin":"","legend":"","description":"","filename":"manuscript.pdf","url":"https://assets-eu.researchsquare.com/files/rs-7989348/v1/7350847f-aa97-4a90-b0f2-134ff245e331.pdf"},{"id":97377732,"identity":"d5cfde2e-83f9-4301-ac9f-13bdec1a107a","added_by":"auto","created_at":"2025-12-03 17:02:43","extension":"docx","order_by":0,"title":"","display":"","copyAsset":false,"role":"supplement","size":18691,"visible":true,"origin":"","legend":"","description":"","filename":"RiggleIRBInterviewGuide.docx","url":"https://assets-eu.researchsquare.com/files/rs-7989348/v1/11a961cfedecfa233d5e67d2.docx"}],"financialInterests":"No competing interests reported.","formattedTitle":"\u003cp\u003eExploring the Lived Experiences of Individuals with Cystic Fibrosis After the Implementation of Trikafta\u003csup\u003e®\u003c/sup\u003e for Treatment.\u003c/p\u003e","fulltext":[{"header":"Background","content":"\u003cp\u003eCystic fibrosis (CF) is one of the most common life-threatening monogenetic diseases, particularly in populations of Northern European descent.\u003csup\u003e\u003cspan citationid=\"CR1\" class=\"CitationRef\"\u003e1\u003c/span\u003e\u003c/sup\u003e CF is caused by variants in the CF transmembrane conductance regulator (CFTR) gene which results in dysfunctional or absent CFTR protein.\u003csup\u003e\u003cspan additionalcitationids=\"CR3\" citationid=\"CR2\" class=\"CitationRef\"\u003e2\u003c/span\u003e\u0026ndash;\u003cspan citationid=\"CR4\" class=\"CitationRef\"\u003e4\u003c/span\u003e\u003c/sup\u003e Despite there being hundreds of different disease-causing mutations seen in people with CF (pwCF), the most common is the Phe508del CFTR mutation. Around 90% of pwCF have at least one copy of the Phe508del mutation\u003csup\u003e\u003cspan citationid=\"CR5\" class=\"CitationRef\"\u003e5\u003c/span\u003e,\u003cspan citationid=\"CR6\" class=\"CitationRef\"\u003e6\u003c/span\u003e\u003c/sup\u003e Historically, CF was once considered to be a childhood fatal disease. In the 1930s,85% of infants diagnosed with CF were expected to die before their second birthday.\u003csup\u003e\u003cspan citationid=\"CR7\" class=\"CitationRef\"\u003e7\u003c/span\u003e\u003c/sup\u003e Over time, improvements in medical management, newborn screening, and drug therapies changed CF from a fatal disease to a chronic condition, with more adults than children now living with CF.\u003csup\u003e8,9\u003c/sup\u003e\u003c/p\u003e\u003cp\u003eMost people with cystic fibrosis begin to experience symptoms shortly after birth, including respiratory disease, pancreatic insufficiency, poor weight gain, excessive salt loss, and male infertility. Respiratory disease and chronic lung infections are the most concerning for pwCF as it is the most frequent cause of lung-transplantation or death in early adult life.\u003csup\u003e\u003cspan citationid=\"CR1\" class=\"CitationRef\"\u003e1\u003c/span\u003e\u003c/sup\u003e Treatments initially targeted CF symptoms: enzyme supplementation for pancreatic insufficiency, antibiotics to prevent or treat lung infection, and mucolytics and airway clearance techniques to help remove mucus from the airway. However, recent CFTR modulator therapies target the underlying defect in CF, the absent or dysfunctional CFTR protein. CFTR modulators include the potentiator ivacaftor and correctors lumacaftor, tezecaftor, elexacaftor and vanzacaftor.\u003csup\u003e\u003cspan citationid=\"CR10\" class=\"CitationRef\"\u003e10\u003c/span\u003e\u003c/sup\u003e Potentiators correct the CFTR channel so that more ions can flow through while correctors help improve the CFTR folding and allow it to be trafficked to the surface of the cell.\u003csup\u003e\u003cspan citationid=\"CR2\" class=\"CitationRef\"\u003e2\u003c/span\u003e\u003c/sup\u003e\u003c/p\u003e\u003cp\u003eIn 2019, the combination of the drugs elexacaftor/tezacaftor/ivacaftor (ETI), otherwise known as Trikafta\u0026reg;, was approved by the FDA for individuals who have at least one Phe508del allele or other qualifying mutation, potentially benefiting up to 90% of pwCF.\u003csup\u003e\u003cspan citationid=\"CR5\" class=\"CitationRef\"\u003e5\u003c/span\u003e,\u003cspan citationid=\"CR11\" class=\"CitationRef\"\u003e11\u003c/span\u003e,\u003cspan citationid=\"CR12\" class=\"CitationRef\"\u003e12\u003c/span\u003e\u003c/sup\u003e A clinical trial studied pwCF who took Trikafta\u0026reg; for 24 weeks compared to a placebo group. Participants who were on Trikafta\u0026reg; experienced a significant improvement in FEV\u003csub\u003e1,\u003c/sub\u003e sharp decrease in sweat chloride concentration, decrease in pulmonary exacerbations, improvements in quality of life, and improvements in BMI. Although side effects were reported in a subset of the participants who were on Trikafta\u0026reg;, the severity of the events were milder compared to prior CFTR modulators. \u003csup\u003e5\u003c/sup\u003e\u003c/p\u003e\u003cp\u003eThe introduction of Trikafta\u0026reg; to CF treatment led to major improvements in the clinical health of pwCF.\u003csup\u003e\u003cspan citationid=\"CR5\" class=\"CitationRef\"\u003e5\u003c/span\u003e,\u003cspan citationid=\"CR11\" class=\"CitationRef\"\u003e11\u003c/span\u003e,\u003cspan citationid=\"CR12\" class=\"CitationRef\"\u003e12\u003c/span\u003e\u003c/sup\u003e However, although majority of pwCF taking Trikafta\u0026reg; report positive outcomes on their health, there are still a subset of individuals whose health has worsened. One side effect reported by pwCF taking Trikafta\u0026reg; is neuropsychiatric changes including depression and anxiety. In a recent survey, healthcare providers from CF care centers reported that while a majority of their patients had positive health outcomes from Trikafta\u0026reg; such as reductions in cough, fewer pulmonary exacerbations, fewer hospitalizations, improved lung function, and increased optimism, a subset of patients had negative health outcomes, including difficulty sleeping, brain fog, memory difficulties, depression, anxiety, and mood swings. \u003csup\u003e12\u003c/sup\u003e Participants also reported that patients shared concerns about attending college, job opportunities, food insecurity, copays, marriage and financial planning. Although this study identified some of the negative health outcomes that pwCF are experiencing from Trikafta\u0026reg;, it is still unknown whether these effects are related to only the medication itself or may also be related to life changes pwCF have experienced since being on Trikafta\u0026reg;.\u003c/p\u003e\u003cp\u003eStudies conducted prior to the introduction of CFTR-modulators have found that the illness-related experiences pwCF face can play a profound role in their lives.\u003csup\u003e\u003cspan citationid=\"CR7\" class=\"CitationRef\"\u003e7\u003c/span\u003e,\u003cspan citationid=\"CR13\" class=\"CitationRef\"\u003e13\u003c/span\u003e,\u003cspan citationid=\"CR14\" class=\"CitationRef\"\u003e14\u003c/span\u003e\u003c/sup\u003e After the introduction of CFTR-modulators such as ivacaftor (Kalydeco\u0026reg;), lumacaftor/ivacaftor (Orkambi\u0026reg;), and tezacaftor/ivacaftor (Symdeko\u0026reg;), studies explored how patient\u0026rsquo;s lived experience was impacted by modulator therapy. One qualitative study explored relationships between CFTR-modulator drugs and the psychological and social aspects of the lives of individuals with cystic fibrosis including career, family planning, relationships, and psychological functioning. \u003csup\u003e10\u003c/sup\u003e The study found several overlapping themes: stability of physical symptoms; changes in self-identity; desires and motivations to achieve new goals; and hope for the future with hope being the most prominent theme.\u003csup\u003e\u003cspan citationid=\"CR10\" class=\"CitationRef\"\u003e10\u003c/span\u003e\u003c/sup\u003e With little data being present about the lived experiences of young adults with CF, this study provided evidence that targeted CFTR-modulator treatments may have negative impacts on the mental, emotional, and social functioning of pwCF. However, at the time of that study Trikafta\u0026reg; was not yet approved for treatment by the FDA. Compared to previous modulator therapies, Trikafta\u0026reg; has shown many more significant health improvements for pwCF with fewer adverse health outcomes. Therefore, it is possible that Trikafta\u0026reg; may have a stronger impact on lived experiences of pwCF compared to prior CFTR modulators. \u003csup\u003e10\u003c/sup\u003e Another study in the United Kingdom examined the psychological implications and health risks of cystic fibrosis pre- and post elexacaftor/tezacaftor/ivacaftor modulator therapy. The study found that while many patients \u0026ldquo;felt lucky to be living\u0026rdquo;, they still identified significant challenges identified in regard to their psychological well-being.\u003csup\u003e\u003cspan citationid=\"CR9\" class=\"CitationRef\"\u003e9\u003c/span\u003e\u003c/sup\u003e The study did not explore what individuals felt was contributing to their mental health challenges or what resources they had used to address these challenges. Despite prior studies showing how CFTR-modulators impact the lived experience of pwCF, \u003cem\u003elittle data is present that explores the healthcare needs pwCF are facing after the approval of Trikafta\u003c/em\u003e\u0026reg; \u003cem\u003ein 2019 and what, if any, resources may be lacking for this population.\u003c/em\u003e\u003c/p\u003e\u003cp\u003eThe purpose of this study is to explore how the addition of Trikafta\u0026reg; to cystic fibrosis treatment has impacted the lived experiences of people with cystic fibrosis and to identify additional resources healthcare providers should offer. Understanding how Trikafta\u0026reg; has influenced these experiences can help reveal emerging needs within this population. By assessing current support systems and identifying gaps, this study aims to investigate the impact of Trikafta on the lived experience of pwCF and guide healthcare providers in adapting care and resources to best suit their patient\u0026rsquo;s needs.\u003c/p\u003e"},{"header":"Methods","content":"\u003cdiv id=\"Sec3\" class=\"Section2\"\u003e\u003ch2\u003eStudy design\u003c/h2\u003e\u003cp\u003eThis study used qualitative methodology based on principles drawn from grounded theory. Grounded theory is a qualitative approach that uses data to construct new theoretical constructs, rather than interpreting data using a prior framework \u003csup\u003e\u003cspan citationid=\"CR15\" class=\"CitationRef\"\u003e15\u003c/span\u003e\u003c/sup\u003e\u003c/p\u003e\u003c/div\u003e\n\u003ch3\u003eRecruitment and Participants\u003c/h3\u003e\n\u003cp\u003eStudy participants were recruited from a CF care center in the midwest between September 2024 and January 2025 dates. A list of potential subjects was generated from the Cystic Fibrosis Foundation Registry using the following inclusion criteria: diagnosis of cystic fibrosis, age 18 years or older, English speaking, and treatment with Trikafta\u0026reg; for at least 1 year. Potential subjects were contacted by electronic health record messaging (MyChart) or in-person during one of their regularly scheduled visits to the CF clinic. For participants who were recruited through MyChart, an initial message was sent that included details of the study and next steps if they were interested in participating. A consent form was then sent electronically to the participant to print out and sign and an interview over the Zoom platform was scheduled (see supplementary material). For individuals who were recruited in person, the researcher provided information about the study, obtained written consent and contact information from participants, and scheduled an interview over the Zoom platform.\u003c/p\u003e\n\u003ch3\u003eData Collection\u003c/h3\u003e\n\u003cp\u003eSemi-structured interviews used an interview guide focused on the aims of this research (see supplementary material) Each question in the interview guide was targeted towards understanding the individual\u0026rsquo;s lived experience, needs, and resources that they found helpful/lacking. Interviews were conducted over the Zoom platform and recorded with permission from the participant. Interviews ranged in length from 35 to 72 minutes with an average time of 51 minutes. All data collected from participants was stored securely on University Hospital\u0026rsquo;s REDcap system. The study was approved by the Institutional Review Board of University Hospitals Cleveland Medical Center (STUDY20240547).\u003c/p\u003e\u003cdiv id=\"Sec6\" class=\"Section2\"\u003e\u003ch2\u003eData Analysis\u003c/h2\u003e\u003cp\u003eEach interview was recorded and a transcript was autogenerated by Zoom. Each transcript was reviewed by the firth author to correct any errors in the auto-generated transcript and edited to remove filler words and repeated words to increase clarity and readability. A number was assigned to each interview transcript, and any identifying information was removed. Once the transcripts were completed, they were coded electronically using the Dedoose qualitative analysis software. The coding process for the interviews was rooted in principles derived from grounded theory and used inductive coding methodology to create codes and themes based on the raw data. Coding began by going line-by-line through each interview and was then followed by reanalysis of each interview to implement newly created codes. A codebook was developed by the researcher that was edited and tested as coding continued. A separate researcher reviewed two of the transcripts using the final codebook to ensure consistency. Coded excerpts were grouped into themes for further analysis.\u003c/p\u003e\u003c/div\u003e"},{"header":"Results","content":"\u003cdiv id=\"Sec8\" class=\"Section2\"\u003e\u003ch2\u003eSample Demographics\u003c/h2\u003e\u003cp\u003eOne hundred and twenty-one individuals met the inclusion/exclusion criteria. All 121 individuals were contacted through MyChart a maximum of three times with follow-up messages sent to individuals who expressed initial interest. In addition, the firth author attended CF clinics to aid in recruitment. Nine participants who met the inclusion criteria for the study agreed to participate. Written informed consent was obtained from all nine participants. Six participants were recruited through MyChart while three participants were recruited in-person during a scheduled CF appointment. Participants\u0026rsquo; ages ranged from 19 to 66 with five participants being between the ages of 18\u0026ndash;35 and four participants being over the age of 36. The data are presented in four themes: (1) transformation in health, (2) future planning and opportunities, (3) impact on relationships, (4) new worries and struggles. Additional demographic data for the participants can be found in Table\u0026nbsp;\u003cspan refid=\"Tab1\" class=\"InternalRef\"\u003e1\u003c/span\u003e which is located on page 6.\u003c/p\u003e\u003cp\u003e\u003cdiv class=\"gridtable\"\u003e\u003ctable float=\"Yes\" id=\"Tab1\" border=\"1\"\u003e\u003ccaption language=\"En\"\u003e\u003cdiv class=\"CaptionNumber\"\u003eTable 1\u003c/div\u003e\u003cdiv class=\"CaptionContent\"\u003e\u003cp\u003eDemographic data of participants\u003c/p\u003e\u003c/div\u003e\u003c/caption\u003e\u003ccolgroup cols=\"8\"\u003e\u003cdiv align=\"left\" class=\"colspec\" colname=\"c1\" colnum=\"1\"\u003e\u003c/div\u003e\u003cdiv align=\"left\" class=\"colspec\" colname=\"c2\" colnum=\"2\"\u003e\u003c/div\u003e\u003cdiv align=\"char\" char=\".\" class=\"colspec\" colname=\"c3\" colnum=\"3\"\u003e\u003c/div\u003e\u003cdiv align=\"left\" class=\"colspec\" colname=\"c4\" colnum=\"4\"\u003e\u003c/div\u003e\u003cdiv align=\"left\" class=\"colspec\" colname=\"c5\" colnum=\"5\"\u003e\u003c/div\u003e\u003cdiv align=\"left\" class=\"colspec\" colname=\"c6\" colnum=\"6\"\u003e\u003c/div\u003e\u003cdiv align=\"left\" class=\"colspec\" colname=\"c7\" colnum=\"7\"\u003e\u003c/div\u003e\u003cdiv align=\"left\" class=\"colspec\" colname=\"c8\" colnum=\"8\"\u003e\u003c/div\u003e\u003cthead\u003e\u003ctr\u003e\u003cth align=\"left\" colname=\"c1\"\u003e\u003cp\u003eParticipant\u003c/p\u003e\u003c/th\u003e\u003cth align=\"left\" colname=\"c2\"\u003e\u003cp\u003eSex Assigned at Birth (M/F)\u003c/p\u003e\u003c/th\u003e\u003cth align=\"left\" colname=\"c3\"\u003e\u003cp\u003eAge\u003c/p\u003e\u003c/th\u003e\u003cth align=\"left\" colname=\"c4\"\u003e\u003cp\u003eRace\u003c/p\u003e\u003c/th\u003e\u003cth align=\"left\" colname=\"c5\"\u003e\u003cp\u003eRelationship Status\u003c/p\u003e\u003c/th\u003e\u003cth align=\"left\" colname=\"c6\"\u003e\u003cp\u003eChildren (Y/N)\u003c/p\u003e\u003c/th\u003e\u003cth align=\"left\" colname=\"c7\"\u003e\u003cp\u003eCurrently Pregnant (Y/N)\u003c/p\u003e\u003c/th\u003e\u003cth align=\"left\" colname=\"c8\"\u003e\u003cp\u003eOn prior modulators\u003c/p\u003e\u003c/th\u003e\u003c/tr\u003e\u003c/thead\u003e\u003ctbody\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e1\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eF\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e31\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eMarried\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eNo\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e2\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eF\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e34\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eMarried\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eY\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eYes, Orkambi and Symdeko\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e3\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eF\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e19\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eSingle\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eNo\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e4\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eM\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e44\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eSingle\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eYes, Symdeko\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e5\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eM\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e66\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eMarried\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eNo\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e6\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eF\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e26\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eMarried\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eY\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eYes, Symdeko\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e7\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eM\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e24\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eSingle\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eNo\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e8\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eM\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e56\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eMarried\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eY\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eNo\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003ctr\u003e\u003ctd align=\"left\" colname=\"c1\"\u003e\u003cp\u003e9\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c2\"\u003e\u003cp\u003eF\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e\u003cp\u003e51\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c4\"\u003e\u003cp\u003eWhite\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c5\"\u003e\u003cp\u003eIn a relationship\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c6\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c7\"\u003e\u003cp\u003eN\u003c/p\u003e\u003c/td\u003e\u003ctd align=\"left\" colname=\"c8\"\u003e\u003cp\u003eYes, Kalydeco\u003c/p\u003e\u003c/td\u003e\u003c/tr\u003e\u003c/tbody\u003e\u003c/colgroup\u003e\u003c/table\u003e\u003c/div\u003e\u003c/p\u003e\u003c/div\u003e\n\u003ch3\u003eTheme 1. Transformation in Health\u003c/h3\u003e\n\u003cp\u003eThe theme \u0026ldquo;transformation in health\u0026rdquo; refers to changes in physical health that participants noticed after starting Trikafta\u0026reg;. Throughout the interviews, participants expressed positive changes including improved lung function, easier weight gain, decrease symptom burden and fewer hospitalizations. Some subjects also noted side effects from the medication affecting their physical health. One participant shared:\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eSince [starting Trikafta\u0026reg;] I've had probably within the first year gained 15 to 20% of my lung function back. I gained, I think, around 15 pounds or so that first year that I was on it. And then I went a whole year without having IV antibiotics which was kind of crazy for me. It was the longest I had gone since middle school maybe.\u003c/p\u003e\u003cp\u003e(Participant 2)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eSome participants mentioned decreased symptom burden after starting Trikafta\u0026reg;.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eYeah, since then I rarely cough. I rarely am wheezing. Only when I'm sick am I wheezing or crackling, which it used to be very common. More often than not I would be wheezing. I know we did a sweat test too. My salt levels are way down everything like that... and since then I've been able to lower how often I'm doing my breathing treatment.\u003c/p\u003e\u003cp\u003e(Participant 3)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eSome participants not only noticed a change in their lung function, but also an increase in their ability to do physical activities.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eBut I do feel like it is just easier to do activities. So laundry or going upstairs, like that kind of stuff. Making my bed, all of that would make me winded... Now I can go get laundry downstairs, go upstairs, make my bed, get food, all of that in an hour and it sounds silly that it is really cool that I can do that better and faster than before.\u003c/p\u003e\u003cp\u003e(Participant 6)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eSome participants who had taken other CFTR modulators prior to Trikafta\u0026reg; compared the changes in physical health they have experienced on both medications.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003e\u0026ldquo;Yeah, I mean you can't even compare them. Like the prior modulators, they gave you maybe 5% of what Trikafta did. Like you felt a little more stable. You could tell maybe you didn't cough as much or you gained weight a little bit easier but it was night and day from Trikafta. Trikafta was like an immediate reaction. You felt your lungs clearing out. I mean, people gained like me 10 pounds in a week. It was just like everything your body had been deprived of suddenly it was able to do.\u0026rdquo;\u003c/p\u003e\u003cp\u003e(Participant 2)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eHowever, some participants reported experiencing side effects while taking Trikafta \u0026reg;.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eFour years, maybe five years in, I realized I was having memory issues. So details are not important to me because they'll drive me crazy. Just like if you ask me something and then somebody says, \u0026ldquo;Well, what did she ask you?\u0026rdquo; I won\u0026rsquo;t remember.\u003c/p\u003e\u003cp\u003e(Participant 9)\u003c/p\u003e\u003cp\u003eLike this summer I suddenly out of nowhere had super high blood pressure for a couple of months. And I was in and out of the ER with high blood pressure. And we think it might be from Trikafta but obviously we don't know because it's never been documented. And yeah, so it's just like.....you kinda have to keep rolling with the punches and figuring out what's happening.\u003c/p\u003e\u003cp\u003e(Participant 2)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\n\u003ch3\u003eTheme 2. Future Planning and Opportunities\u003c/h3\u003e\n\u003cp\u003eMany participants expressed a new desire to plan for the future since starting Trikafta\u0026reg;. Participants mentioned opportunities that have become available to them such as starting a family, changing careers, or preparing for retirement. One participant shared:\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eI would say that the biggest improvements have been less worrying about the future. You never know. That\u0026rsquo;s something that I do worry about, you know, what's going to happen down the road and stuff like that. But it's a little bit easier to make better long-term plans now than before.\u003c/p\u003e\u003cp\u003e(Participant 4)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eAnother participant shared being able to switch to a more physically demanding career since starting Trikafta\u0026reg;, which has brought him a new sense of purpose.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eLike hooking my feet onto a ladder and swinging through houses or climbing up in attics without a single thought is definitely not something I could have done. That would have winded me a long time ago. But I also really have a hard time saying like what I could do and what I couldn't do cause I was lucky enough to get it [Trikafta] just as I was hitting the point where I was really getting serious about working and being an adult and actually having to do things.\u003c/p\u003e\u003cp\u003e(Participant 7)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eParticipant 8 shared he has begun to think about retiring from his career and pursuing other interests.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eWell, yeah pre-Trikafta I was wondering how much longer I'd be able to work. But since Trikafta I'm thinking about retiring actually this year from [job]... and then just do something different. Maybe back into nursing, but maybe it's just something totally different.\u003c/p\u003e\u003cp\u003e(Participant 8)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eAnother participant expressed hope that she will be able to conceive children and carry a pregnancy.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eMy doctors, even before all the modulators, they were like \u0026ldquo;We can try our best. Here's what we know.\u0026rdquo; And so they were always hopeful that it would happen. And then of course with Trikafta, they actually talked to me once I was on Trikafta and they knew I was married like, \u0026ldquo;Hey, just a heads up Trikafta is actually kind of causing a baby boom. If you don't want kids like just know that.\u0026rdquo; And I was like, oh, that's good to know that it is causing a lot more pregnancy.\u003c/p\u003e\u003cp\u003e(Participant 6)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cdiv id=\"Sec11\" class=\"Section2\"\u003e\u003ch2\u003eTheme 3. Impact on Relationships\u003c/h2\u003e\u003cp\u003eParticipants described impacts on their relationships with their partner, family, and care teams since starting Trikafta\u0026reg;. For example, one participant shared the positive impacts her improvements in physical health have had on her relationship with her husband.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eSo I started Trikafta when I was with my husband. We were still dating at the time. But with him, I think he's more just seen a difference in me, like a positive aspect of \u0026ldquo;Yeah, you feel like you can do more activities\u0026rdquo; and he that we were dating when I used to get so sick every other week and it's kind of nice, I'm sure for him. Not that he minds taking care of me but it's hard to see someone that you love like go through that all the time so he's like, \u0026ldquo;Oh, that's so nice that you get to feel more healthy.\u003c/p\u003e\u003cp\u003e(Participant 6)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eSome participants discussed an improved ability to begin a romantic relationship.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eSo actually, ironically, with meeting so many people through these apps right now, cause, nothing's really stuck, my thing used to just be like, \u0026ldquo;Hey, I have cystic fibrosis.... I can happily educate you about it. If that's a deal breaker, though I completely understand.\u0026rdquo; Ironically, that's changed now to the same thing, but I used to not be healthy. But I'm healthier than I've ever been and there's still downsides, but it's significantly lessened.\u003c/p\u003e\u003cp\u003e(Participant 7)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eSome participants shared how Trikafta\u0026reg; has not had a major impact on their relationship with their family members.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eYeah, I'd say it has a bit, just because I don't have to do as much maintenance and treatments day to day. So I'd say it's changed a bit, but I don't think there's any major changes since starting Trikafta in the family dynamic.\u003c/p\u003e\u003cp\u003e(Participant 3)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eSeveral participants also mentioned how Trikafta\u0026reg; did not have a strong impact on their relationship with their healthcare team.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eI wouldn't say it's changed our relationship, but we have to focus now on different things, if that makes sense. Like we're not so concerned about keeping my lungs super healthy. We're concerned more about big picture stuff.\u003c/p\u003e\u003cp\u003e(Participant 2)\u003c/p\u003e\u003cp\u003eI started out from almost day one, Dr. [Name] was my doctor. So as far as a relationship goes, I mean, he\u0026rsquo;s my doctor. I didn\u0026rsquo;t really think too much beyond that.\u003c/p\u003e\u003cp\u003e(Participant 5)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003c/div\u003e\u003cdiv id=\"Sec12\" class=\"Section2\"\u003e\u003ch2\u003eTheme 4. New Worries and Struggles\u003c/h2\u003e\u003cp\u003eParticipants also highlighted a wide range of struggles and new concerns after starting Trikafta\u0026reg; including finances, changes in self-identity, and fear of new health concerns arising. For example, one participant shared having a difficult time adjusting to her \u0026ldquo;new normal\u0026rdquo;.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eLike when you have your whole life you're told, \u0026ldquo;This is what your life is going to be like, this is what your life expectancy is going to be like,\u0026rdquo; and you kind of plan. And then when something like this comes out that can substantially, you know, much longer......you're going to live and you're going to like.......you could do all these things that you didn't think maybe before you could, so it's very hard adjusting.\u003c/p\u003e\u003cp\u003e(Participant 1)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eAnother participant expressed fear over developing cancer in the future now that her lung health is no longer her biggest concern.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eAlso that both my parents have died from cancer and suddenly I'm going to continue to get older and that probably is the reason that I will pass away. Like I've started thinking about that as well. Yeah, I think that's the biggest, the biggest worries that I have.\u003c/p\u003e\u003cp\u003e(Participant 2)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eSome participants shared concerns about the cost of Trikafta\u0026reg; and fear of losing disability payments.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eThat is the one thing with Trikafta that I worry about is am I going to be told that I am no longer considered disabled. Because I have read that there have been people that they are telling that to. And then at my age you're going to tell a 51-year-old lady that she has to now go find a career. And if I was to get really sick and need a disability again, I would have to fight for it. It's not something they just put you back on.\u003c/p\u003e\u003cp\u003e(Participant 9)\u003c/p\u003e\u003cp\u003eThank god they passed the ADA and thank god the state has some helping because Trikafta is ungodly high. So that is paid for. If it wasn\u0026rsquo;t, I wouldn\u0026rsquo;t be taking it.\u003c/p\u003e\u003cp\u003e(Participant 5)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eOther participants expressed concerns for their finances, including a lack of financial planning for the future.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eYeah because I mean, it's not funny, but I tell my husband \u0026ldquo;Well, I didn't think I was ever going to retire so I never saved anything\u0026rdquo;. I mean, honestly, so now it's like \u0026ldquo;Oh, well, crap, maybe I will work until I'm maybe 65.\u0026rdquo; So now I should probably start saving. So yeah, that's definitely a big switch with Trikafta too, because before I was like \u0026ldquo;I don't have to worry about it.\u003c/p\u003e\u003cp\u003e(Participant 1)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003cp\u003eAnother participant discussed having to advocate for herself when receiving obstetric care.\u003cdiv class=\"BlockQuote\"\u003e\u003cp\u003eYeah, so obviously OBGYN care is a struggle when you have CF because.......and it's even gotten worse because of that big baby boom after Trikafta....they just aren't. Thankfully, we have a really good maternal fetal medicine team at UH that has some experience with CF but it is just crazy. Like they don't know how to treat people with CF. There's a lot of advocating you have to do for yourself.\u003c/p\u003e\u003cp\u003e(Participant 2)\u003c/p\u003e\u003c/div\u003e\u003c/p\u003e\u003c/div\u003e"},{"header":"Discussion","content":"\u003cp\u003eThe purpose of this qualitative study was to explore how the implementation of Trikafta\u0026reg; to cystic fibrosis treatment has impacted the lived experiences of people with cystic fibrosis and what additional resources healthcare providers should be offering to their patients. From the data collected from this study, four themes emerged: transformation in health, future planning and opportunities, impact on relationships, and new worries and struggles. These findings contribute to the growing body of literature on how CFTR-modulators can impact pwCF beyond clinical health.\u003c/p\u003e\u003cp\u003eParticipants expressed overwhelmingly positive changes in their physical health that they attributed to being on Trikafta\u0026reg;. Many participants described improvements in lung function, less frequent hospitalizations, decreased coughing, and the ability to perform more physical activities. The positive changes in health reported by participants align with previous interventional and observational studies in pwCF taking Trikafta that showed significant increases in FEV\u003csub\u003e1\u003c/sub\u003e, decreases in sweat chloride concentration, decreases in the amount of pulmonary exacerbations, increases in BMI, and an overall increase in health related quality of life. \u003csup\u003e8,11\u003c/sup\u003e An observational study conducted in 2021 for pwCF Trikafta\u0026reg; found similar results and highlighted the clinical benefits that pwCF experienced after being on Trikafta\u0026reg; for 6 months.\u003csup\u003e\u003cspan citationid=\"CR11\" class=\"CitationRef\"\u003e11\u003c/span\u003e\u003c/sup\u003e Participants in this study who were on prior CFTR-modulators also emphasized that the considerable improvements to their health that they observed from Trikafta\u0026reg; far exceeded those from prior modulators they were on. Overall, participants reported not only stabilizing effects of Trikafta\u0026reg; on health, but also significant improvements in physical health. However, several participants mentioned side effects that they believe to be related to treatment with Trikafta\u0026reg;, including challenges with memory and incidents of high blood pressure. Prior research has found that some pwCF experience neuropsychiatric side effects such as brain fog, sleep disturbances, and mood changes.\u003csup\u003e\u003cspan citationid=\"CR13\" class=\"CitationRef\"\u003e13\u003c/span\u003e\u003c/sup\u003e More research is needed to determine the impact that Trikafta\u0026reg; has on other body systems.\u003c/p\u003e\u003cp\u003eStudy participants also discussed the ways in which Trikafta\u0026reg; opened the door for new opportunities and created a new desire to plan for the future. Some participants described pursuing new careers or being able to work physically demanding jobs, while others described planning for retirement or starting a family. The shifts that participants had toward long-term planning aligns with prior qualitative studies observing the effect of CFTR-modulators on the lived experience of pwCF.\u003csup\u003e\u003cspan citationid=\"CR10\" class=\"CitationRef\"\u003e10\u003c/span\u003e,\u003cspan citationid=\"CR16\" class=\"CitationRef\"\u003e16\u003c/span\u003e\u003c/sup\u003e Therefore, the ability for participants to envision and plan for a future that was previously deemed uncertain sheds light on the similar psychosocial aspects Trikafta\u0026reg; has on pwCF.\u003c/p\u003e\u003cp\u003e Throughout the interviews, participants reported variable effects on how Trikafta\u0026reg; impacted their personal relationships. While participants reported that Trikafta\u0026reg; had a positive impact on their dating life and romantic relationships, relationships with family members and CF care teams remained largely unchanged. These findings partially align with results from previous studies that found improvements in friendships and romantic relationships due to participants having increased energy and less frequent hospitalizations.\u003csup\u003e\u003cspan citationid=\"CR4\" class=\"CitationRef\"\u003e4\u003c/span\u003e\u003c/sup\u003e Another study conducted in the United Kingdom explored how ETI therapy (elexacaftor/tezacaftor/ivacaftor) impacted the relationships of pwCF with their CF care teams. The study found mixed results: participants discussed their CF care teams not taking their input seriously, a lack of support around changes to self-identity, and a need for routine psychological support at every CF visit.\u003csup\u003e19\u003c/sup\u003e These findings suggest that although Trikafta\u0026reg; has been found to have a strong impact on physical health and lifestyle, its effects on relationships with others may be variable.\u003c/p\u003e\u003cp\u003eThe purpose of this study was not only to identify how Trikafta\u0026reg; has impacted the lived experience of pwCF, but also to inveestigate any new needs that pwCF are experiencing. Some of the worries or concerns mentioned by participants included the lack of financial planning for the future, fear of losing disability benefits, changes in self-identity, and the fear of new health concerns arising now that the life span for pwCF is increasing. These themes align with results from a 2023 study that found pwCF experienced new challenges including increased anxiety about life planning, struggles with finances, and uncertainty about their long-term health. \u003csup\u003e4\u003c/sup\u003e Similarly, individuals in our study also reported difficulties adjusting to their new identity now that they are no longer as sick as they were before. Lastly, some participants expressed having to self-advocate for themselves with other healthcare providers, including with OBGYNs lacking education on how to care for pwCF. These reported experiences align with research highlighting the need for a better transition from adolescent to adult CF care. As the number of adults living with CF continues to increase, priorities are shifting to a long-term care approach centered around monitoring chronic conditions such as CF related diabetes and liver disease, optimizing nutrition, educating about sexual and reproductive health, and evaluating mental health concerns.\u003csup\u003e\u003cspan citationid=\"CR17\" class=\"CitationRef\"\u003e17\u003c/span\u003e\u003c/sup\u003e This study highlights the need for additional psychological support and resources for pwCF as well as increased education about CF care to adult providers outside of the CF care team.\u003c/p\u003e\u003cp\u003eCompared to other CFTR-modulator therapies, Trikafta\u0026reg; has shown significant improvements in the clinical health of pwCF.\u003csup\u003e\u003cspan citationid=\"CR5\" class=\"CitationRef\"\u003e5\u003c/span\u003e,\u003cspan citationid=\"CR11\" class=\"CitationRef\"\u003e11\u003c/span\u003e,\u003cspan citationid=\"CR12\" class=\"CitationRef\"\u003e12\u003c/span\u003e\u003c/sup\u003e Prior to this study, little research has investigated impacts of Trikafta\u0026reg; on the lived experiences of pwCF. A 2022 qualitative study of the lived experience of pwCF on CFTR-modulators, before the approval of Trikafta\u0026reg; by the FDA, found prominent feelings of hope among participants regarding the CFTR-modulator they were prescribed and advancing medical research. Although participants in the study did not see dramatic improvements in their physical health, they shared a sense of hope for the future and a desire to achieve new goals. Participants also reported changes to their self-identity, such as no longer seeing CF as their defining feature, as well as a new potential to accomplish specific goals.\u003csup\u003e\u003cspan citationid=\"CR10\" class=\"CitationRef\"\u003e10\u003c/span\u003e\u003c/sup\u003e Participants in our study expressed similar feelings of hope, such as hopes to start a family, switch careers, retire, and explore new interests. However, unlike the prior study in which participant\u0026rsquo;s hopes were centered around the potential for future positive health, our participants attributed their hopes to the significant positive changes in health that they had already experienced. Participants in our study had also begun taking steps to achieve these goals such as switching to a more physically demanding job, putting aside money to plan for a family, and beginning retirement planning. While participants in both studies reported some similar experiences, more research is needed to determine how Trikafta\u0026reg; impacts the lived experience of pwCF over time.\u003c/p\u003e\u003cdiv id=\"Sec14\" class=\"Section2\"\u003e\u003ch2\u003eStudy Limitations\u003c/h2\u003e\u003cp\u003eThis study\u0026rsquo;s findings are limited due to the small sample size of participants, which did not allow us to reach data saturation. The inclusion criteria (18 years or older, English speaking, and being on Trikafta\u0026reg; for at least 1 year) limited our sample since Trikafta\u0026reg; has only been available since 2019 and many pwCF do not qualify for this therapy. Study participants were only recruited through a single CF treatment center in the Midwest region of the US, and these findings may not be representative of the experiences of pwCF who live in other geographic areas or visit other hospital systems for their CF care. However, despite the small sample size, the participants in this study spanned diverse ages, sexes, and life experiences such as relationship status, use of prior modulators, and parenting. Lastly, the experiences shared by participants in this study only captured a moment in time of their CF journey. Perceptions and experiences of pwCF on this therapy are likely to change over time.\u003c/p\u003e\u003c/div\u003e\u003cdiv id=\"Sec15\" class=\"Section2\"\u003e\u003ch2\u003ePractice Implications\u003c/h2\u003e\u003cp\u003eLimited research has investigated how Trikafta\u0026reg; impacts the lived experiences of pwCF. The data from this study provides evidence that Trikafta\u0026reg; has impacted pwCF not only physically, but may also have an impact on relationships, mental health, and future planning. It also highlights areas in which healthcare providers can improve care for pwCF. This study highlights potential impacts of Trikafta\u0026reg; on mental health, including anxiety about the future, changes to self-identity, and concerns about new changes in health as pwCF get older. Although anxiety and depression screenings and management protocols are often already implemented by CF care teams, additional resources surrounding mental health and referrals to other health professionals who understand CF-related challenges may help to lessen some of these concerns.\u003csup\u003e\u003cspan citationid=\"CR17\" class=\"CitationRef\"\u003e17\u003c/span\u003e\u003c/sup\u003e Participants in the study also expressed challenges with long-term financial planning, affordability of CF care/medications, and concerns about losing disability benefits. While CF care teams often include social workers to assist with finances, it may be beneficial to connect pwCF with other professionals surrounding CF-related finances. For instance, healthcare providers may find it beneficial to collaborate with financial advisors to help assist their patients with financial planning, find payment plans for medications/care, and advocate for patients as they transition to being healthier. Some participants in the study also experienced side effects and new health concerns while taking Trikafta\u0026reg;. As life expectancy for pwCF has improved since the implementation of CFTR-modulators, it is important for providers to not only monitor side effects associated with the start of the medication but also shift to a lifelong approach centered around preventative care and maintaining long term health as pwCF grow older. This may include additional education to other health providers about CF-related health concerns and increasing the number of adult-care providers that are part of the CF team.\u003c/p\u003e\u003c/div\u003e\u003cdiv id=\"Sec16\" class=\"Section2\"\u003e\u003ch2\u003eResearch Recommendations\u003c/h2\u003e\u003cp\u003eResearch centered around the lived experience of pwCF after taking Trikafta\u0026reg; is lacking. Currently, around 90% of CF patients have a genotype that makes them eligible for the use of Trikafta\u0026reg;.\u003csup\u003e18\u003c/sup\u003e Therefore understanding how Trikafta\u0026reg; impacts the lived experience of members of the CF community is important in enhancing the care provided to these individuals. Future research should also investigate how the lived experience of pwCF is impacted for individuals who do not qualify for Trikafta\u0026reg; or have decided against or discontinued this treatment. Although this study adds to the body of literature surrounding the lived experience for pwCF who are on Trikafta\u0026reg;, this study only includes responses from a few members of the CF community. Therefore, this study is not generalizable to all individuals with CF who are on Trikafta\u0026reg;. A study investigating the lived experience for pwCF who are on Trikafta\u0026reg; in a larger population size may be able to provide insight to these experiences in a way that is more generalizable.\u003c/p\u003e\u003c/div\u003e"},{"header":"Declarations","content":"\u003cp\u003e\u003cem\u003eHuman Ethics and Consent to Participate\u003c/em\u003e\u003c/p\u003e\n\u003cp\u003eThis study was conducted in accordance with the Declaration of Helsini and was approved by the Institutional Review Board of University Hospitals Cleveland Medical Center (STUDY20240547).\u0026nbsp;\u003c/p\u003e\n\u003cp\u003eClinical trial number: not applicable\u003c/p\u003e\n\u003cp\u003e\u003cem\u003eConsent for Publication\u003c/em\u003e\u003c/p\u003e\n\u003cp\u003eEach participant in the study provided their consent to participate and signed an in-person consent form that was approved by the Institutional Review Board of University Hospitals Cleveland Medical Center (STUDY20240547).\u003c/p\u003e\n\u003cp\u003e\u003cem\u003eAvailability of data and materials\u003c/em\u003e\u003c/p\u003e\n\u003cp\u003eThe datasets used and/or analyzed during the current study are available from the corresponding author on reasonable request.\u003c/p\u003e\n\u003cp\u003e\u003cem\u003eCompeting interests\u003c/em\u003e\u003c/p\u003e\n\u003cp\u003eThe authors declare that they have no competing interests.\u003c/p\u003e\n\u003cp\u003e\u003cem\u003eFunding\u003c/em\u003e\u003c/p\u003e\n\u003cp\u003eNo funding was obtained in the conduction of this research.\u0026nbsp;\u003c/p\u003e\n\u003cp\u003e\u003cem\u003eAcknowledgements\u003c/em\u003e\u003c/p\u003e\n\u003cp\u003eI would like to express my deepest gratitude to my thesis committee advisor (Rebecca Darrah) and thesis committee members (Annelise Page, Erica Roesch, Marsha Michie) for dedicating their time and expertise to my research. Without their valuable feedback and encouragement, this work would not have been possible. I would also like to thank the incredible staff at Leroy W. Matthews Cystic Fibrosis Center, my graduate program co-director (Michelle Merrill), and my wonderful classmates who provided endless support and insightful ideas throughout the research process.\u003c/p\u003e"},{"header":"References","content":"\u003col\u003e\u003cli\u003e\u003cspan\u003eDe Boeck K. Cystic fibrosis in the year 2020: A disease with a new face. Acta Paediatr Oslo Nor 1992. 2020;109(5):893\u0026ndash;9. \u003cspan class=\"ExternalRef\"\u003e\u003cspan class=\"RefSource\"\u003e10.1111/apa.15155\u003c/span\u003e\u003cspan address=\"10.1111/apa.15155\" targettype=\"DOI\" class=\"RefTarget\"\u003e\u003c/span\u003e\u003c/span\u003e.\u003c/span\u003e\u003c/li\u003e\u003cli\u003e\u003cspan\u003eBaroud E, Chaudhary N, Georgiopoulos AM. 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Transition to adult care in cystic fibrosis: The challenges and the structure. Paediatr Respir Rev. 2022;41:23\u0026ndash;9. \u003cspan class=\"ExternalRef\"\u003e\u003cspan class=\"RefSource\"\u003e10.1016/j.prrv.2020.07.009\u003c/span\u003e\u003cspan address=\"10.1016/j.prrv.2020.07.009\" targettype=\"DOI\" class=\"RefTarget\"\u003e\u003c/span\u003e\u003c/span\u003e.\u003c/span\u003e\u003c/li\u003e\u003c/ol\u003e"}],"fulltextSource":"","fullText":"","funders":[],"hasAdminPriorityOnWorkflow":false,"hasManuscriptDocX":true,"hasOptedInToPreprint":true,"hasPassedJournalQc":"","hasAnyPriority":false,"hideJournal":true,"highlight":"","institution":"","isAcceptedByJournal":false,"isAuthorSuppliedPdf":false,"isDeskRejected":"","isHiddenFromSearch":false,"isInQc":false,"isInWorkflow":false,"isPdf":false,"isPdfUpToDate":true,"isWithdrawnOrRetracted":false,"journal":{"display":true,"email":"[email protected]","identity":"researchsquare","isNatureJournal":false,"hasQc":true,"allowDirectSubmit":true,"externalIdentity":"","sideBox":"","snPcode":"","submissionUrl":"/submission","title":"Research Square","twitterHandle":"researchsquare","acdcEnabled":true,"dfaEnabled":false,"editorialSystem":"","reportingPortfolio":"","inReviewEnabled":false,"inReviewRevisionsEnabled":true},"keywords":"Cystic fibrosis, Trikafta® (elexacaftor/tezacaftor/ivacaftor), Lived experience, CFTR modulators, Physical health, Mental health, Self-identity, Relationships, Future planning, Healthcare needs","lastPublishedDoi":"10.21203/rs.3.rs-7989348/v1","lastPublishedDoiUrl":"https://doi.org/10.21203/rs.3.rs-7989348/v1","license":{"name":"CC BY 4.0","url":"https://creativecommons.org/licenses/by/4.0/"},"manuscriptAbstract":"\u003cp\u003e\u003cstrong\u003eBackground: \u003c/strong\u003eCFTR-modulators are a class of medications prescribed to people with cystic fibrosis (pwCF) who have certain genetic mutations. In 2019, Trikafta\u003csup\u003e® \u003c/sup\u003ewas approved by the FDA and lead to significant health improvements for pwCF including increased pulmonary function, a decrease in sweat chloride levels, and an overall increase in health-related quality of life. Studies done on prior CFTR-modulators have found that modulator therapies can not only impact the physical health of pwCF, but also psychological and social aspects of life. Despite Trikafta\u003csup\u003e® \u003c/sup\u003eshowing significant health improvements compared to prior modulators, little is known about how Trikafta\u003csup\u003e® \u003c/sup\u003eimpacts the lived experience of pwCF. The purpose of this study is to explore how the implementation of Trikafta\u003csup\u003e®\u003c/sup\u003e to cystic fibrosis treatment has impacted the lived experience of pwCF and what additional resources healthcare providers should be offering to their patients\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eMethods: \u003c/strong\u003eNine men and women with CF who were over the age of 18 and have been on Trikafta\u003csup\u003e® \u003c/sup\u003efor at least a year were recruited from an adult CF center. A semi-structured interview guide was used to conduct interviews over the Zoom platform. The data were then analyzed through inductive coding rooted in grounded theory.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eResults: \u003c/strong\u003eFour themes were constructed from the data in this study: (1) “transformation in health” refers to the change in physical health the participants have noticed since starting Trikafta\u003csup\u003e®\u003c/sup\u003e, (2) “future planning and opportunities” highlights new opportunities becoming available and a new desire to plan for the future, (3) “impact on relationships” describes how participants felt their relationships with their partner, family, and care teams, were impacted by Trikafta\u003csup\u003e®\u003c/sup\u003e, (4)“new worries and struggles” describes new struggles and concerns that participants have faced.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eConclusions: \u003c/strong\u003eThe data from this study provide evidence that Trikafta\u003csup\u003e® \u003c/sup\u003ehas not only impacted pwCF on a physical level, but may also have an impact on relationships, mental health, and future planning. It also highlights the need for additional resources that CF care teams should be providing to their patients such as mental health resources, increased financial support, and shifting to a preventative care approach as pwCF grow older.\u003c/p\u003e","manuscriptTitle":"Exploring the Lived Experiences of Individuals with Cystic Fibrosis After the Implementation of Trikafta® for Treatment.","msid":"","msnumber":"","nonDraftVersions":[{"code":1,"date":"2025-12-03 17:02:38","doi":"10.21203/rs.3.rs-7989348/v1","editorialEvents":[{"type":"communityComments","content":0}],"status":"published","journal":{"display":true,"email":"[email protected]","identity":"researchsquare","isNatureJournal":false,"hasQc":true,"allowDirectSubmit":true,"externalIdentity":"","sideBox":"","snPcode":"","submissionUrl":"/submission","title":"Research Square","twitterHandle":"researchsquare","acdcEnabled":true,"dfaEnabled":false,"editorialSystem":"","reportingPortfolio":"","inReviewEnabled":false,"inReviewRevisionsEnabled":true}}],"origin":"","ownerIdentity":"9b5e708d-8e5f-4efe-85b7-9a66fa0ecd9f","owner":[],"postedDate":"December 3rd, 2025","published":true,"recentEditorialEvents":[],"rejectedJournal":[],"revision":"","amendment":"","status":"posted","subjectAreas":[],"tags":[],"updatedAt":"2025-12-26T07:09:17+00:00","versionOfRecord":[],"versionCreatedAt":"2025-12-03 17:02:38","video":"","vorDoi":"","vorDoiUrl":"","workflowStages":[]},"version":"v1","identity":"rs-7989348","journalConfig":"researchsquare"},"__N_SSP":true},"page":"/article/[identity]/[[...version]]","query":{"redirect":"/article/rs-7989348","identity":"rs-7989348","version":["v1"]},"buildId":"8U1c8b4HqxoKbykW_rLl7","isFallback":false,"isExperimentalCompile":false,"dynamicIds":[84888],"gssp":true,"scriptLoader":[]}