Journal of Cystic Fibrosis最新文献

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ETD001: A novel inhaled ENaC blocker with an extended duration of action in vivo ETD001:一种新型吸入式 ENaC 阻断剂,可延长体内作用时间。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.06.002
Henry Danahay , Clive McCarthy , Thomas Schofield , Roy Fox , Holly Charlton , Sarah Lilley , Juan Sabater , Matthias Salathe , Nathalie Baumlin , Stephen P Collingwood , Martin Gosling
{"title":"ETD001: A novel inhaled ENaC blocker with an extended duration of action in vivo","authors":"Henry Danahay ,&nbsp;Clive McCarthy ,&nbsp;Thomas Schofield ,&nbsp;Roy Fox ,&nbsp;Holly Charlton ,&nbsp;Sarah Lilley ,&nbsp;Juan Sabater ,&nbsp;Matthias Salathe ,&nbsp;Nathalie Baumlin ,&nbsp;Stephen P Collingwood ,&nbsp;Martin Gosling","doi":"10.1016/j.jcf.2024.06.002","DOIUrl":"10.1016/j.jcf.2024.06.002","url":null,"abstract":"<div><h3>Background</h3><div>Inhibiting ENaC in the airways of people with cystic fibrosis (pwCF) is hypothesized to enhance mucociliary clearance (MCC) and provide clinical benefit. Historically, inhaled ENaC blockers have failed to show benefit in pwCF challenging this hypothesis. It is however unknown whether the clinical doses were sufficient to provide the required long duration of action in the lungs and questions whether a novel candidate could offer advantages where others have failed?</div></div><div><h3>Methods</h3><div>Dose-responses with the failed ENaC blockers (VX-371, BI 1265162, AZD5634, QBW276) together with ETD001 (a novel long acting inhaled ENaC blocker) were established in a sheep model of MCC and were used to predict clinically relevant doses that would provide a long-lasting enhancement of MCC in pwCF. In each case, dose predictions were compared with the selected clinical dose.</div></div><div><h3>Results</h3><div>Each of the failed candidates enhanced MCC in the sheep model. Translating these dose-response data to human equivalent doses, predicted that substantially larger doses of each candidate, than were evaluated in clinical studies, would likely have been required to achieve a prolonged enhancement of MCC in pwCF. In contrast, ETD001 displayed a long duration of action (≥16 h) at a dose level that was well tolerated in Phase 1 clinical studies.</div></div><div><h3>Conclusions</h3><div>These data support that the ENaC blocker hypothesis is yet to be appropriately tested in pwCF. ETD001 has a profile that enables dosing at a level sufficient to provide a long duration of action in a Phase 2 clinical study in pwCF scheduled for 2024.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 72-78"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141293424","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}
引用次数: 0
What does it mean to be “healthy” when taking elexacaftor/tezacaftor/ivacaftor (ETI)? A qualitative study 服用 elexacaftor/tezacaftor/ivacaftor (ETI) 时,"健康 "意味着什么?定性研究。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.11.003
Robin S. Everhart , Emma McWilliams , Jill Maggs , Gregory S. Sawicki , Takeera Sconiers , Kyle Smith , Dana Yablon , Jennifer Butcher , Michelle Prickett , Callie Bacon , Andrea Goodman , Alex H. Gifford , Nicole Mayer-Hamblett , David P. Nichols , Kristin A. Riekert
{"title":"What does it mean to be “healthy” when taking elexacaftor/tezacaftor/ivacaftor (ETI)? A qualitative study","authors":"Robin S. Everhart ,&nbsp;Emma McWilliams ,&nbsp;Jill Maggs ,&nbsp;Gregory S. Sawicki ,&nbsp;Takeera Sconiers ,&nbsp;Kyle Smith ,&nbsp;Dana Yablon ,&nbsp;Jennifer Butcher ,&nbsp;Michelle Prickett ,&nbsp;Callie Bacon ,&nbsp;Andrea Goodman ,&nbsp;Alex H. Gifford ,&nbsp;Nicole Mayer-Hamblett ,&nbsp;David P. Nichols ,&nbsp;Kristin A. Riekert","doi":"10.1016/j.jcf.2024.11.003","DOIUrl":"10.1016/j.jcf.2024.11.003","url":null,"abstract":"<div><h3>Background</h3><div>Elexacaftor/tezacaftor/ivacaftor (ETI) has profoundly affected the health and lives of many people with CF (pwCF). The rapid change in health for pwCF taking ETI provided them an opportunity to reflect on what “being healthy” means. The goal of this secondary analysis was to document changes in the health and well-being of pwCF after starting ETI, beyond the expected physical benefits.</div></div><div><h3>Methods</h3><div>The Qualitative Understanding of Experiences with the SIMPLIFY Trial (QUEST) study evaluated pwCF's experiences on ETI, including participation in a withdrawal study, treatment burden, and experiences of health. Two sixty-minute interviews were conducted approximately four months apart, audio-recorded and transcribed. A phenomenological approach was used to identify text of interest and create a formal codebook.</div></div><div><h3>Results</h3><div>Ninety-one pwCF (mean age=27.8 years; range 14–67; 51 % male) and 23 caregivers of the teenagers completed at least one interview. Beyond the expected benefits of ETI, four themes were identified: (1) Night and Day Change in Health, (2) Reduced Cognitive Burden, (3) Shifting to Managing Overall Wellness and Co-Morbidities, and (4) Social/Self Identity Changes.</div></div><div><h3>Conclusion</h3><div>ETI has raised pwCF's expectations for their health and well-being. Current symptom and QOL measures may no longer capture the less perceptible ways pwCF on ETI experience changes in symptoms or health. CF care may need to adapt to focus more on general health, managing other comorbidities, and planning for the future.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 187-192"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142692902","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}
引用次数: 0
Bridging the gap: Challenging lung infections and clinical trial development in cystic fibrosis 缩小差距:挑战性肺部感染与囊性纤维化的临床试验开发。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.11.001
Damian G. Downey , Nicholas J. Simmonds , Silke van-Koningsbruggen-Rietschel , Jutta Bend , Fiona Dunlevy , Kate Hill , Lieven Dupont
{"title":"Bridging the gap: Challenging lung infections and clinical trial development in cystic fibrosis","authors":"Damian G. Downey ,&nbsp;Nicholas J. Simmonds ,&nbsp;Silke van-Koningsbruggen-Rietschel ,&nbsp;Jutta Bend ,&nbsp;Fiona Dunlevy ,&nbsp;Kate Hill ,&nbsp;Lieven Dupont","doi":"10.1016/j.jcf.2024.11.001","DOIUrl":"10.1016/j.jcf.2024.11.001","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 79-82"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709236","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}
引用次数: 0
Cardiometabolic risk factors in adults with cystic fibrosis undergoing elexacaftor/tezacaftor/ivacaftor therapy 成人囊性纤维化患者接受elexaftor /tezacaftor/ivacaftor治疗的心脏代谢危险因素
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.11.009
Andrea Gramegna , Massimiliano Ruscica , Gloria Leonardi , Chiara Macchi , Isabella Fichtner , Gianmarco Putti , Margherita Carnevale Schianca , Leonardo Terranova , Gianfranco Alicandro , Francesco Blasi
{"title":"Cardiometabolic risk factors in adults with cystic fibrosis undergoing elexacaftor/tezacaftor/ivacaftor therapy","authors":"Andrea Gramegna ,&nbsp;Massimiliano Ruscica ,&nbsp;Gloria Leonardi ,&nbsp;Chiara Macchi ,&nbsp;Isabella Fichtner ,&nbsp;Gianmarco Putti ,&nbsp;Margherita Carnevale Schianca ,&nbsp;Leonardo Terranova ,&nbsp;Gianfranco Alicandro ,&nbsp;Francesco Blasi","doi":"10.1016/j.jcf.2024.11.009","DOIUrl":"10.1016/j.jcf.2024.11.009","url":null,"abstract":"<div><div>The introduction of elexacaftor/tezacaftor/ivacaftor (ETI) therapy has further extended life expectancy of adults with cystic fibrosis (awCF), highlighting the need for increased attention to potential long-term health issues. Given the increasing prevalence of cardiovascular diseases in the ageing population and the presence of cardiovascular risk factors associated with CF, understanding the impact of ETI on cardiometabolic risk factors is a crucial clinical concern. The aim of our prospective observational study was to explore early changes in cardiac and metabolic biomarkers after 6 months of ETI therapy. A total of 58 consecutive awCF were enrolled during clinical stability at the Adult CF Center of the Policlinico Hospital in Milan, Italy between January 2021 and June 2022. Blood samples were obtained before ETI initiation and after 6 months, and underwent central processing for an extended panel of cardiometabolic biomarkers. We observed a rise in cholesterol, triglycerides, apolipoprotein-B and adipokine levels, while inflammatory markers decreased. The direct relationship between leptin and adiponectin suggest a disruption in the normal regulatory mechanisms that control these hormones, potentially leading to metabolic imbalances, such as increased risk of obesity and cardiovascular events. The impact of ETI on cardiovascular risk in awCF is heterogeneous and while it improves some risk factors, such as chronic inflammation, it has a worsening effect on lipoproteins. Our findings suggest that the dysregulation of adipokines could be a potential cause of the metabolic disturbances observed in awCF.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 53-56"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791736","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}
引用次数: 0
CF Ferrets exposed to in utero ivacaftor do not develop lens abnormalities 子宫内接触过 ivacaftor 的 CF 雪貂不会出现晶状体异常。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.09.007
Jennifer L. Taylor-Cousar , Shahab Fakhari , Lacina Allison , Doug J. Bartels , Raksha Jain , Sushan Han
{"title":"CF Ferrets exposed to in utero ivacaftor do not develop lens abnormalities","authors":"Jennifer L. Taylor-Cousar ,&nbsp;Shahab Fakhari ,&nbsp;Lacina Allison ,&nbsp;Doug J. Bartels ,&nbsp;Raksha Jain ,&nbsp;Sushan Han","doi":"10.1016/j.jcf.2024.09.007","DOIUrl":"10.1016/j.jcf.2024.09.007","url":null,"abstract":"","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 19-20"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288298","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}
引用次数: 0
Changes in factors associated with inhaled antibiotic prescriptions for people with cystic fibrosis over time in the U.S. 美国囊性纤维化患者吸入抗生素处方相关因素随时间推移的变化。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.09.017
Marianne S. Muhlebach , Jane She , Eric Y. Zhang , Jonathan D. Cogen , Michael R. Kosorok
{"title":"Changes in factors associated with inhaled antibiotic prescriptions for people with cystic fibrosis over time in the U.S.","authors":"Marianne S. Muhlebach ,&nbsp;Jane She ,&nbsp;Eric Y. Zhang ,&nbsp;Jonathan D. Cogen ,&nbsp;Michael R. Kosorok","doi":"10.1016/j.jcf.2024.09.017","DOIUrl":"10.1016/j.jcf.2024.09.017","url":null,"abstract":"<div><h3>Rationale</h3><div>CF care guidelines recommend chronic inhaled antibiotics for chronic <em>Pseudomonas aeruginosa</em> (Pa) lung infection. These medications are costly, time consuming and prescription needs may change with improved outcomes.</div></div><div><h3>Objectives</h3><div>We determined the proportion of pwCF with chronic, intermittent or negative Pa infection categories, their clinical and demographic characteristics, factors associated with inhaled antibiotic prescription and changes between 2011 and 2019.</div></div><div><h3>Methods</h3><div>This cohort study using the U.S. CF Foundation patient registry for pwCF &gt;2 years, no prior lung transplant, and with ≥3 respiratory cultures/year determined chronic inhaled antibiotics (≥3 months per calendar year) and Pa infection status from encounter level data. Outcomes and odds of prescription for relevant clinical factors were evaluated using generalized estimating equation models with additional interaction between the predictor and the calendar year to examine changes of predictors over time.</div></div><div><h3>Results</h3><div>Proportion of pwCF with chronic and intermittent Pa decreased and antibiotic prescription rates increased for these groups and decreased for Pa negative pwCF. Hispanic ethnicity, female sex, pancreatic insufficiency, CF diabetes, and ivacaftor/lumacaftor were associated with higher antibiotic prescriptions for each Pa status. Among Pa-negative pwCF prescriptions were higher with <em>Burkholderia spp</em>. (1.17, (CI<sub>95</sub> 1.03,1.34)) or MRSA (OR 1.45, (1.26,1.68)) but decreased between 2011 and 2019. For <em>Aspergillus</em> OR increased to 1.6,(1.3,1.8) in 2019. Prescriptions for pwCF on ivacaftor decreased, becoming lower in 2019 for chronic (OR 0.7, (0.5,0.8)) and Pa-negative pwCF (OR 0.7, (0.5,0.8)).</div></div><div><h3>Conclusions</h3><div>Factors predicting inhaled antibiotic prescription differed between 2011 and 2019 indicating changes in health and care for pwCF even prior to triple-modulators.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 98-104"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142400430","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}
引用次数: 0
Glucagon-like-peptide-1 agonist therapy in adults with cystic fibrosis 成人囊性纤维化患者的胰高血糖素样肽-1 激动剂治疗。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.08.005
Sanghoon Park , Raksha Jain , Sasan Mirfakhraee
{"title":"Glucagon-like-peptide-1 agonist therapy in adults with cystic fibrosis","authors":"Sanghoon Park ,&nbsp;Raksha Jain ,&nbsp;Sasan Mirfakhraee","doi":"10.1016/j.jcf.2024.08.005","DOIUrl":"10.1016/j.jcf.2024.08.005","url":null,"abstract":"<div><div>Glucagon-like-peptide-1 (GLP-1) agonists are commonly used to improve glycemic control and promote weight loss in individuals with type 2 diabetes mellitus (T2DM) and/or obesity. However, there is a paucity of evidence regarding GLP-1 agonist use in people with cystic fibrosis (pwCF). We present 11 people with CF (males: 3, females: 7; age range 24–47; BMI range 25.7–43.7) treated with GLP-1 agonists (semaglutide: 9,tirzepatide: 2) for variable duration (1–50 months). All experienced weight loss on GLP- 1 agonist therapy (median change in weight = -7.2 kg; change in BMI [kg/m2] = -0.9 to -8.1). Eight pwCF showed improvement in percent predicted forced expiratory volume in 1 second (ppFEV1) [change = -5 to + 18] and nine pwCF showed improvement in percent predicted forced vital capacity (ppFVC) [change= +1 to + 26]. Of the 7 pwCF with CFRD, all reduced their insulin quantity (mean, 31.5 % decrease in total daily insulin dose), and glucose time in range improved for most (mean, +11 % increase from baseline). Four pwCF stopped using GLP-1 agonists: 2 due to severe nausea/vomiting, 1 due to lack of perceived benefit, and 1 due to change in insurance coverage. This report is the largest published series to date of pwCF treated with GLP-1 agonist therapy. With the addition of GLP-1 agonists, all individuals experienced weight loss and a reduction in daily insulin dose, and most had improvement in pulmonary function. Future multi-center studies are needed to corroborate the efficacy and safety of these agents in the CF population.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 40-46"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142107829","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}
引用次数: 0
Self-reported chronic therapy use after 24-weeks of follow-up by participants who completed the simplify randomized, controlled trial 完成简化随机对照试验的参与者在随访 24 周后自我报告的慢性疗法使用情况。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.08.008
Alex H. Gifford , Katherine Odem-Davis , Margaret Kloster , Brian P. O'Sullivan , Gregory J. Omlor , Susan L. Millard , John P. Clancy , Gregory S. Sawicki , Kristin Riekert , Nicole Mayer-Hamblett , David P. Nichols , SIMPLIFY Study Group
{"title":"Self-reported chronic therapy use after 24-weeks of follow-up by participants who completed the simplify randomized, controlled trial","authors":"Alex H. Gifford ,&nbsp;Katherine Odem-Davis ,&nbsp;Margaret Kloster ,&nbsp;Brian P. O'Sullivan ,&nbsp;Gregory J. Omlor ,&nbsp;Susan L. Millard ,&nbsp;John P. Clancy ,&nbsp;Gregory S. Sawicki ,&nbsp;Kristin Riekert ,&nbsp;Nicole Mayer-Hamblett ,&nbsp;David P. Nichols ,&nbsp;SIMPLIFY Study Group","doi":"10.1016/j.jcf.2024.08.008","DOIUrl":"10.1016/j.jcf.2024.08.008","url":null,"abstract":"<div><h3>Background</h3><div>Highly effective CFTR modulator therapy (HEMT) has improved the health of many people with cystic fibrosis (pwCF), offering opportunities to discontinue burdensome therapies. SIMPLIFY included randomized, controlled trials that confirmed non-inferiority of discontinuing versus continuing dornase alfa (DA) or hypertonic saline (HS) for 6 weeks in pwCF on HEMT. In this study of post-trial treatment use by SIMPLIFY participants, we hypothesized that randomization to discontinue DA or HS during the trial would be associated with a higher likelihood of non-use of each medication during follow-up.</div></div><div><h3>Methods</h3><div>We electronically surveyed SIMPLIFY participants every 4 weeks for 24 weeks after trial completion but before the main trial results were publicly disclosed. We asked them how often they used medications during the previous week. We estimated covariate-adjusted odds ratios (ORs) of DA or HS non-use by logistic regression with generalized estimating equations.</div></div><div><h3>Results</h3><div>After exclusions mostly due to lack of any surveys, 472 participants were included in the analysis population, 181 from the HS trial and 291 from the DA trial. Approximately half of the analysis population completed all six surveys. At every month of follow-up in both trials, the percentage of individuals reporting non-use of DA or HS during the previous week was greater among those randomized to discontinue therapy. Among participants with responses at 24 weeks, 30/122 (24.6 %) in the HS trial and 79/222 (35.6 %) in the DA trial reported non-use of the respective study medication. After adjusting for covariates, participants randomized to discontinue DA were 8.7-times (95 % CI: 4.3–17.7) more likely to not use DA during follow-up than those randomized to continue DA, and participants randomized to discontinue HS were 5.2-times (95 % CI: 2.1–12.8) more likely to not use HS during follow-up compared to those randomized to continue.</div></div><div><h3>Conclusions</h3><div>In healthy pwCF on ETI, randomization to discontinue DA or HS during SIMPLIFY was associated with greater odds of not using each medication after the trial compared to randomization to continue. These findings suggest that participation in a treatment discontinuation trial can influence participants’ post-trial treatment decisions. This possibility may be relevant during discussions about research participation and clinical care.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 91-97"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142288303","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}
引用次数: 0
Development of metabolic syndrome in people with Cystic Fibrosis one year after exposure to elexacaftor-tezacaftor-ivacaftor 囊性纤维化患者在使用 elexacaftor-tezacaftor-ivacaftor 一年后出现代谢综合征。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.09.022
Gregory A. Ratti , Hannah Smith , Sasan Mirfakhraee , Joan Reisch , Leah Cohen , Raksha Jain , James D. Finklea
{"title":"Development of metabolic syndrome in people with Cystic Fibrosis one year after exposure to elexacaftor-tezacaftor-ivacaftor","authors":"Gregory A. Ratti ,&nbsp;Hannah Smith ,&nbsp;Sasan Mirfakhraee ,&nbsp;Joan Reisch ,&nbsp;Leah Cohen ,&nbsp;Raksha Jain ,&nbsp;James D. Finklea","doi":"10.1016/j.jcf.2024.09.022","DOIUrl":"10.1016/j.jcf.2024.09.022","url":null,"abstract":"<div><h3>Background</h3><div>The constellation of hypertension, truncal obesity, impaired fasting glucose, low high-density lipoprotein, and hypertriglyceridemia is known as metabolic syndrome (MetSyn) and is associated with cardiovascular and other diseases. Elexacaftor-tezacaftor-ivacaftor (ETI) in people with cystic fibrosis (pwCF) is associated with weight gain but effects on cardiovascular risk are unknown. This study sought to investigate ETI exposure and risk for development of MetSyn in pwCF.</div></div><div><h3>Methods</h3><div>A prospective cohort study including pwCF ≥ 18 years old exposed to ETI was performed. All data for calculating MetSyn was collected from the electronic medical record at initiation and 1 year ± 3 months after starting ETI. A total of 152 pwCF exposed to ETI and 34 pwCF never exposed to CF transmembrane conductance regulator modulators were included in the analysis. Changes to hypertension classification was also examined over this period.</div></div><div><h3>Results</h3><div>After 1 year of ETI there was an increase in MetSyn from 13 to 30 pwCF, <em>p</em> &lt; 0.0001. No new cases of MetSyn were seen in the group not exposed to ETI. After 1 year of ETI, more people met criteria for class 1 (BP 130–139/90–99 mm Hg) or class 2 hypertension (BP ≥140/≥90 mm Hg) regardless of prior modulator exposure, <em>p</em> &lt; 0.0001.</div></div><div><h3>Conclusions</h3><div>Exposure to ETI for 1 year resulted in an increased number of cases of MetSyn. There was an increased incidence of hypertension associated with ETI exposure. Additional studies are needed to further examine this trend and to determine if these changes will translate to cardiovascular complications over time.</div></div>","PeriodicalId":15452,"journal":{"name":"Journal of Cystic Fibrosis","volume":"24 1","pages":"Pages 47-52"},"PeriodicalIF":5.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142466564","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}
引用次数: 0
Exogenous insulin does not reduce protein catabolism in pre-diabetic cystic fibrosis patients: A randomized clinical trial 外源性胰岛素不会减少糖尿病前期囊性纤维化患者的蛋白质分解:随机临床试验。
IF 5.4 2区 医学
Journal of Cystic Fibrosis Pub Date : 2025-01-01 DOI: 10.1016/j.jcf.2024.10.005
Michele Schiavon , Claudio Cobelli , K. Sreekumaran Nair , Katherine Klaus , Gianna Toffolo , Lin Zhang , Antoinette Moran
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