Journal of Cystic Fibrosis最新文献

{"title":"Coping and learning to Manage Stress with cystic fibrosis (CALM): A multisite telehealth randomized controlled trial to reduce depression and anxiety symptoms in adults with cystic fibrosis","authors":"Christina J. Bathgate ,&nbsp;Elizabeth D. Smith ,&nbsp;Nora H. Murphy ,&nbsp;Alexandra L. Quittner ,&nbsp;Kristin A. Riekert ,&nbsp;Jennifer L. Goralski ,&nbsp;Kristen E. Holm","doi":"10.1016/j.jcf.2024.11.002","DOIUrl":"10.1016/j.jcf.2024.11.002","url":null,"abstract":"<div><h3>Background</h3><div>Adults with cystic fibrosis (AWCF) have higher rates of depression and anxiety than comparable community members. This multisite randomized waitlist-controlled trial tested the efficacy of “Coping and Learning to Manage Stress with CF” (CALM), a 6-session+booster telehealth intervention to improve depression and anxiety symptoms (primary outcomes) and perceived stress, coping self-efficacy, and key health-related quality of life domains (secondary outcomes).</div></div><div><h3>Methods</h3><div>AWCF reporting mild to severe symptoms of depression and/or anxiety were randomized to receive CALM immediately (<em>immediate, n</em> = 66) or after a 13-week delay (<em>waitlist, n</em> = 66). Group differences post-intervention and at 1-month were examined via linear mixed models. Maintenance of treatment gains from baseline to 3-month follow-up was examined using combined data from both groups. Effect size calculations using Cohen's <em>d</em> assessed treatment effect magnitude.</div></div><div><h3>Results</h3><div>Compared to the waitlist group, those that received CALM immediately reported lower depression and anxiety symptoms post-intervention and at 1-month follow-up (ps&lt;0.001). For depression there was a large effect size post-intervention (<em>d</em> = 0.85) and a medium effect size at 1-month follow-up (<em>d</em> = 0.70); anxiety had a medium effect size post-intervention (<em>d</em> = 0.65) and at 1-month follow-up (<em>d</em> = 0.66). The immediate group also reported significantly higher coping self-efficacy, less stress, and increased vitality post-CALM and at 1-month follow-up (ps&lt;0.01). Treatment gains were maintained at 3-month follow-up for all outcomes.</div></div><div><h3>Conclusions</h3><div>CALM was efficacious for AWCF in reducing symptoms of depression, anxiety, and perceived stress while improving coping self-efficacy and vitality with evidence of treatment sustainability. Next steps are dissemination and implementation to CF psychosocial clinicians.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 310-318"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709541","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Supporting adherence to the cystic fibrosis regimen: Development and validation of The Daily Care Check-In (DCC)","authors":"Kristin A. Riekert ,&nbsp;Christine Ford ,&nbsp;Andrea Goodman ,&nbsp;Thomas Eckmann ,&nbsp;Angela Green ,&nbsp;Alexandra L. Quittner ,&nbsp;Success with Therapies Research Consortium Barriers Study Investigators","doi":"10.1016/j.jcf.2024.10.011","DOIUrl":"10.1016/j.jcf.2024.10.011","url":null,"abstract":"<div><h3>Background</h3><div>The cystic fibrosis (CF) regimen is time-consuming and burdensome leading to barriers to self-management. This mixed-methods study developed the Daily Care Check-in (DCC) that is specific to the barriers faced by people with CF (PWCF) and evaluated its validity.</div></div><div><h3>Methods</h3><div>Qualitative methods were used to identify barriers to self-management and develop items, with “think aloud” cognitive interviews conducted to refine the items. A multisite, cross-sectional study was conducted to test the internal consistency, test-retest reliability, and validity of the DCC scores, comparing them to objective medication adherence (composite medication possession ratio (cMPR)) and psychosocial measures (self-efficacy, medication beliefs, executive functioning, depressive and anxiety symptoms, treatment burden, and treatment complexity).</div></div><div><h3>Results</h3><div>The DCC (18 items) includes two scales: Occurrence (score range 0–18) and Interference (score range 0–90). 405 participants completed the DCC, 344 (85 %) completed the survey, and 365 (90 %) had a cMPR calculated. On average, 6.8 barriers were reported (SD = 4.2 Occurrence Scale), and the Interference Scale had a mean score of 18.4 (SD = 14.0). Reliability was acceptable to good. cMPR was negatively correlated with the DCC (rho=-0.26, Occurrence and rho = -0.31, Interference, p-values&lt;0.0001). <em>A priori</em> hypotheses between the DCC and the other measures were supported and demonstrated construct validity.</div></div><div><h3>Conclusions</h3><div>This study provides evidence supporting the validity of the DCC for assessing the presence and impact of barriers to CF self-management, including medication adherence. Formal screening of self-management barriers (e.g., using the DCC) should be considered to facilitate conversations with the care team and identify tailored interventions to support CF self-management.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 326-333"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564561","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pseudo-Bartter syndrome: A CFTR-related disorder?","authors":"Noelia Rodriguez Mier ,&nbsp;Virginie Antoons ,&nbsp;Senne Cuyx ,&nbsp;Anabela Santo Ramalho ,&nbsp;Mieke Boon ,&nbsp;Marijke Proesmans ,&nbsp;Djalila Mekahli ,&nbsp;François Vermeulen","doi":"10.1016/j.jcf.2024.10.007","DOIUrl":"10.1016/j.jcf.2024.10.007","url":null,"abstract":"<div><div>This case report presents a 14-month-old boy with a history of cystic fibrosis (CF) carrier status, diagnosed following a positive newborn screening for CF (CF-NBS), who developed symptoms suggestive of Pseudo-Bartter syndrome (PBS). Despite initial evaluations not meeting CF diagnostic criteria, subsequent investigations revealed an intermediate sweat chloride concentration, a second CFTR mutation, and CFTR dysfunction through rectal organoid morphology analysis (ROMA) consistent with CFTR-related disorder (CFTR-RD). This case raises important considerations regarding the diagnosis and management of CFTR-RD. PBS can be considered as a rare presentation of CFTR-RD and can occur in children with sweat chloride below the CF range. Functional testing of CFTR by ROMA enabled a more accurate diagnosis. Despite the negative work-up after CF-NBS, this infant developed CFTR-RD, but this should not be considered as a screen failure. Follow-up of children with CFTR-RD at a CF centre is preferred, because of the risk of developing CF.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 401-403"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142557883","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"AI-facilitated home monitoring for cystic fibrosis exacerbations across pediatric and adult populations","authors":"Henryk Mazurek ,&nbsp;Andrzej Emeryk ,&nbsp;Kamil Janeczek ,&nbsp;Eric Derom ,&nbsp;Barbara Kuźnar-Kamińska ,&nbsp;Tomasz Grzywalski ,&nbsp;Adam Biniakowski ,&nbsp;Krzysztof Szarzyński ,&nbsp;Anna Pastusiak ,&nbsp;Dominika Kaminiarczyk-Pyzałka ,&nbsp;Dick Botteldooren ,&nbsp;Honorata Hafke-Dys ,&nbsp;Jędrzej Kociński","doi":"10.1016/j.jcf.2025.02.011","DOIUrl":"10.1016/j.jcf.2025.02.011","url":null,"abstract":"<div><h3>Background</h3><div>AI-aided home stethoscopes offer the opportunity of continuous remote monitoring of cystic fibrosis (CF) patients, reducing the need for clinic visits.</div></div><div><h3>Aim</h3><div>This study aimed to analyze the possibility of detecting CF pulmonary exacerbations (PEx) at home using an AI-aided stethoscope (AIS).</div></div><div><h3>Materials and Methods</h3><div>In a six-month study, 129 CF patients (85 children, 44 adults) used AIS for at least weekly self-examinations, recording various parameters: wheezes, rhonchi, crackles intensity, respiratory and heart rate, and inspiration-to-expiration ratio. Health state surveys were also completed. Physicians evaluated 5160 examinations to identify PEx. Machine learning models were trained using those parameters, and AUCs were calculated for PEx detection.</div></div><div><h3>Results</h3><div>522 self-examinations were diagnosed clinically as exacerbated. AI-aided home stethoscopes detected 415 exacerbated self-examinations (sensitivity 79.5 % at specificity 89.1 %). Among the single-parameter discriminators, coarse crackles intensity exhibited an AUC of 70 % (95% CI: 65–75) for young children, fine crackles intensity demonstrated an AUC of 75 % (95 % CI: 72–78) for older children, and an AUC of 93 % (95 % CI: 92–93) was achieved for adults using fine crackles intensity. The combination of parameters yielded the highest efficacy, with AUC exceeding 83% for objective parameters from the AI module alone and exceeding 90 % when incorporating both objective and subjective parameters across all groups.</div></div><div><h3>Conclusions</h3><div>The AI-aided home stethoscope has proven to be a reliable tool for detecting PEx with greater accuracy than self-assessment alone. Implementing this technology in healthcare systems has the potential to provide valuable insights for timely intervention and management of PExes.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 390-397"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mycobacterium abscessus treatment outcomes in cystic fibrosis: A single centre experience","authors":"Ieuan Evans ,&nbsp;Aaron Weimann ,&nbsp;Timothy Baird ,&nbsp;Charles Haworth ,&nbsp;Andres Floto","doi":"10.1016/j.jcf.2024.07.016","DOIUrl":"10.1016/j.jcf.2024.07.016","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 364-367"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141788215","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Elexacaftor/tezacaftor/ivacaftor and mental health: A workshop report from the Cystic Fibrosis Foundation's Prioritizing Research in Mental Health working group","authors":"CJ Bathgate ,&nbsp;DA Fedele ,&nbsp;EM Tillman ,&nbsp;J He ,&nbsp;RS Everhart ,&nbsp;LR Reznikov ,&nbsp;FF Liu ,&nbsp;K Kirby ,&nbsp;K Raffensperger ,&nbsp;K Traver ,&nbsp;KA Riekert ,&nbsp;SW Powers ,&nbsp;AM Georgiopoulos","doi":"10.1016/j.jcf.2024.11.006","DOIUrl":"10.1016/j.jcf.2024.11.006","url":null,"abstract":"<div><h3>Background</h3><div>This report summarizes the 2023 inaugural annual meeting of the Cystic Fibrosis Foundation's <em>Prioritizing Research in Mental Health (PRIME)</em> working group. This workshop focused on mental health and elexacaftor/tezacaftor/ivacaftor (ETI).</div></div><div><h3>Methods</h3><div>We reviewed existing literature and identified key gaps and study design considerations in preclinical work, pharmacokinetics/pharmacodynamics, mood/anxiety, quality of life/self-perception, neuropsychological symptoms, sleep, and symptom management.</div></div><div><h3>Results</h3><div>Limited studies have identified behavioral changes with modulator exposure in rodent models of depression, anxiety, and cognition. Longitudinal human studies reporting mean changes generally show no change or improvement. However, case reports and single-center studies identify subgroups reporting new or worsening symptoms.</div></div><div><h3>Conclusions</h3><div>Future studies should focus on understanding the role of CFTR in the nervous system, defining ETI impacts in preclinical models, and mechanistic investigations. Innovative methods with larger samples and comprehensive assessments are needed to determine the incidence of new/worsening symptoms throughout the lifespan and effective management strategies.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 301-309"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142729103","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advertisement for Editor-in-Chief, Journal of Cystic Fibrosis","authors":"","doi":"10.1016/j.jcf.2025.02.006","DOIUrl":"10.1016/j.jcf.2025.02.006","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 201-202"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143408108","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mental health and adherence in CF: Self-efficacy and perceived barriers as mediators","authors":"Robin S. Everhart ,&nbsp;Milene T. Saavedra ,&nbsp;Christine R. Ford ,&nbsp;Sidney L. Gibson ,&nbsp;Felicia Reid ,&nbsp;Emily F. Muther ,&nbsp;Christina L. Duncan ,&nbsp;Rachel Cravens ,&nbsp;Angela Green ,&nbsp;Kristin A. Riekert","doi":"10.1016/j.jcf.2025.02.016","DOIUrl":"10.1016/j.jcf.2025.02.016","url":null,"abstract":"<div><h3>Background</h3><div>Symptoms of depression and anxiety can contribute to lower medical treatment adherence. Given that people with cystic fibrosis (PWCF) have higher rates of depressive and anxiety symptoms than those without cystic fibrosis (CF), this study examined factors that mediated the association between mental health and adherence.</div></div><div><h3>Methods</h3><div>Participants were 294 adults (<em>M</em> age=25 years) with CF who were enrolled in the Daily Care Check-in Validation Study. Participants completed in-clinic questionnaires that assessed depressive and anxiety symptoms, perceived barriers to self-management, and medication self-efficacy. Medication adherence was measured by pharmacy refill data. Parallel mediation models assessed perceived barriers and medication self-efficacy as mediators between depressive symptoms and adherence, and between anxiety symptoms and adherence.</div></div><div><h3>Results</h3><div>Perceived interference of barriers to self-management significantly mediated the association between depressive symptoms and adherence (β =-0.005, SE=0.002, 95 % CI [-0.009, -0.001]), and between anxiety symptoms and adherence (β=-0.005, SE=0.003, 95 % CI [-0.008, -0.001]). Additionally, self-efficacy significantly mediated the association between depressive symptoms and adherence (β=-0.004, SE=0.001, 95 % CI [-0.007, -0.002]), and between anxiety symptoms and adherence (β=-0.004, SE=0.001, 95 % CI [-0.007, -0.001]).</div></div><div><h3>Conclusions</h3><div>This study found that when PWCF experienced mental health symptoms (either anxiety or depression), they were likely to report more interference from barriers to disease management or experience less medication self-efficacy, which was related to worse adherence. Building self-efficacy around taking medications may reduce the impact that mental health symptoms have on adherence. Care teams should also work with PWCF to minimize the impact of barriers on daily therapies.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 334-340"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Racial disparities in lung transplantation for cystic fibrosis in the era of highly effective modulator therapy","authors":"Jessica M. Ruck ,&nbsp;Shi Nan Feng ,&nbsp;Alexandra H. Toporek ,&nbsp;Pali D. Shah ,&nbsp;Erin Tallarico ,&nbsp;Noah Lechtzin ,&nbsp;Allan B. Massie ,&nbsp;Dorry L. Segev ,&nbsp;Errol L. Bush ,&nbsp;Christian A. Merlo","doi":"10.1016/j.jcf.2025.01.006","DOIUrl":"10.1016/j.jcf.2025.01.006","url":null,"abstract":"<div><h3>Background</h3><div>Highly effective modulator therapies (HEMT) including ivacaftor (IVA) and elexacaftor/tezacaftor/ivacaftor (ETI) have transformed treatment for people with cystic fibrosis (pwCF). However, non-HEMT-responsive mutations are more common in pwCF of non-White race/ethnicity; introduction of HEMT might have exacerbated racial/ethnic disparities in CF care.</div></div><div><h3>Methods</h3><div>Using the Scientific Registry of Transplant Recipients, we identified all lung transplant candidates and recipients 05/2005–12/2022 and categorized them by diagnosis (CF/non-CF), race/ethnicity (non-Hispanic White/Black/Hispanic) and era [Pre-HEMT (2005–1/30/2012), IVA (1/31/2012–10/30/2019), ETI (10/31/2019–12/31/2022)]. We compared the percentage of patients listed, delisted/died, or transplanted by race/ethnicity and era.</div></div><div><h3>Results</h3><div>34,659 lung transplants were performed: 10,521 pre-HEMT, 15,944 in IVA era, and 7,888 in ETI era. Over the three eras, the percentage of lung recipients with CF of White race decreased (94.5 % to 92.4 % to 78.4 %) and of Black race (1.7 % to 2.4 % to 5.7 %) or Hispanic ethnicity increased (3.5 % to 4.6 % to 14.2 %; <em>p</em> &lt; 0.001). Similarly, among candidates listed for CF over the three eras, the percentage that were of White race decreased (82.0 % vs. 78.6 % vs. 71.0 %) and of Black race (9.2 % vs. 10.0 % vs. 10.3 %) or Hispanic ethnicity increased (6.4 % vs. 8.6 % vs. 13.6 %; <em>p</em> &lt; 0.001).</div></div><div><h3>Conclusion</h3><div>The introduction of HEMT appears to have benefitted CF lung transplant candidates and recipients of Black race or Hispanic ethnicity less than those of White race. This is likely due to the higher prevalence of HEMT-ineligible CFTR mutations among Black and Hispanic patients and underscores the need for therapies aimed at non-HEMT-responsive mutations prevalent in these racial/ethnic populations.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 278-283"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world outcomes of generic elexacaftor/tezacaftor/ivacaftor (gETI) in South Africans (SA) with CF using standard versus clarithromycin-boosted gETI, modulator-sparing strategies to reduce cost","authors":"Marco Zampoli ,&nbsp;Janine Verstraete ,&nbsp;Cathy Baird ,&nbsp;Tony Biebuyck ,&nbsp;Greg Calligaro ,&nbsp;Marina Coetzee ,&nbsp;Carla Els ,&nbsp;Marlize Frauendorf ,&nbsp;Paul Gebers ,&nbsp;Brenda Morrow ,&nbsp;Dave Richards ,&nbsp;Hanri Truter ,&nbsp;Andrew Hill","doi":"10.1016/j.jcf.2025.02.002","DOIUrl":"10.1016/j.jcf.2025.02.002","url":null,"abstract":"<div><h3>Objective</h3><div>Access to highly effective modulator therapies (HEMT) in resource-limited countries is limited by prohibitive cost and restrictive patents. We report the clinical outcomes of a cost-reduction strategy in South Africa (SA), where generic elexacaftor/tezacaftor/ivacaftor (gETI) was pharmacokinetically enhanced with clarithromycin (gETI/c) for people with CF (pwCF) eligible for HEMT.</div></div><div><h3>Methods</h3><div>A multi-center observational study from December 2021 to May 2024. Analysis of variance (ANOVA) and linear mixed effects analyses were conducted to describe and compare change in sweat chloride (SC), FEV1pp, BMI (m/kg²) and adverse events (AE) over 18-months follow-up for different gETI dose categories: a) standard, full or b) modulator sparing dose (gETI/c at 25–50 % recommended dose, twice/thrice weekly).</div></div><div><h3>Results</h3><div>70/413 (17 %) eligible pwCF [median age 27 years (range 6–52); 68 (97 %) with ≥ one copy F508del] received gETI with standard (<em>n</em> = 38) or modulator-sparing doses (<em>n</em> = 32); 29 changed dosing regimens across the study period. The overall mean (SD) reduction in SC after 1-month of treatment was -52.9 (16.9) mmol/L (<em>p</em> &lt; 0.001), with no evidence of difference between dose groups (<em>p</em> = 0.2). Overall mean (SD) FEV1pp and BMI increased at 1-month by 14.9 (95 % CI 11.49–18.40) and 0.84 (95 % CI 0.16–1.49), respectively. Improvements in FEV1pp and BMI were sustained throughout follow-up, with no evidence of difference between dosing groups. No serious AEs were reported.</div></div><div><h3>Conclusion</h3><div>Our experience with gETI is similar to real-world reports using the originator product. Boosting ETI with CYP3A-inhibitors is a safe and effective strategy to increase access to ETI in settings where access to HEMT is restricted.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 2","pages":"Pages 284-289"},"PeriodicalIF":5.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143254339","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}