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Glucagon-like peptide1 receptor agonist treatment of cystic fibrosis-related diabetes complicated by obesity: A cases series and literature review.
J Clin Transl Endocrinol
December 2024
Division of Endocrinology Diabetes and Metabolism, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
Cystic fibrosis-related diabetes (CFRD) is the most common non-pulmonary comorbidity in people with cystic fibrosis (CF). Current guidelines recommend insulin therapy as the treatment of choice for people with CFRD. In the past, obesity and overweight were uncommon in individuals with CF.
View Article and Find Full Text PDFRespiratory Outcomes and Aspergillus Serology Following Elexacaftor/Tezacaftor/Ivacaftor Therapy in People with Cystic Fibrosis and a History of Aspergillus fumigatus Infection.
Lung
January 2025
Mother and Child Department, Cystic Fibrosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Purpose: The study evaluated the effects of elexacaftor/tezacaftor/ivacaftor (ETI) therapy in people with cystic fibrosis (pwCF) and a clinical history of Aspergillus fumigatus (AF) infection.
Methods: This prospective cohort study included pwCF who initiated ETI therapy and had received antifungal treatment in the preceding five years due to allergic bronchopulmonary aspergillosis (ABPA group) or other AF-related clinical manifestations (AF group). A control group of pwCF with no prior respiratory cultures positive for AF was also included.
Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial.
Lancet Respir Med
December 2024
Population Policy and Practice Department, UCL Great Ormond Street Institute of Child Health, University College London, London, UK; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK. Electronic address:
Article Synopsis
- Vanzacaftor-tezacaftor-deutivacaftor is a new CFTR modulator showing safety and effectiveness in phase 2 trials for adults with cystic fibrosis, leading to a study evaluating its use in children aged 6-11.
- This phase 3 trial, called RIDGELINE, involved participants from 33 clinical sites across eight countries, focusing on children with specific CFTR variants and stable health conditions.
- The study aimed to assess the drug's safety, tolerability, and efficacy over 24 weeks, with primary outcomes evaluated through various health metrics and participant feedback.
Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials.
Lancet Respir Med
December 2024
Division of Infection, Immunity and Respiratory Medicine, University of Manchester, Manchester, UK. Electronic address:
Background: The goal of cystic fibrosis transmembrane conductance regulator (CFTR) modulators is to reach normal CFTR function in people with cystic fibrosis. Vanzacaftor-tezacaftor-deutivacaftor restored CFTR function in vitro and in phase 2 trials in participants aged 18 years and older resulting in improvements in CFTR function, as measured by sweat chloride concentrations and lung function as measured by spirometry. We aimed to evaluate the efficacy and safety of vanzacaftor-tezacaftor-deutivacaftor compared with standard of care elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older.
View Article and Find Full Text PDFSweat chloride reflects CFTR function and correlates with clinical outcomes following CFTR modulator treatment.
J Cyst Fibros
January 2025
Cystic Fibrosis Therapeutics Development Network Coordinating Center, Seattle Children's Hospital, Seattle, WA, USA; Department of Pediatrics, University of Washington, Seattle, WA, USA; Department of Biostatistics, University of Washington, Seattle, WA, USA.
Background: Highly effective CFTR modulators improve CFTR function and lead to dramatic improvements in health outcomes in many people with cystic fibrosis (pwCF). The relationship between measures of CFTR function, such as sweat chloride concentration, and clinical outcomes in pwCF treated with CFTR modulators is poorly defined. We conducted analyses to better understand the relationships between sweat chloride and CFTR function in vitro, and between sweat chloride and clinical outcomes following CFTR modulator treatment.
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