Results 51 to 60 of about 3,103 (172)

A descriptive case series of hepatotoxicity associated with CFTR modulators and possible relevance of pharmacogenetic polymorphisms in cystic fibrosis patients

British Journal of Clinical Pharmacology, EarlyView.
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are widely used in patients with cystic fibrosis and significantly improve respiratory function and quality of life. However, their effectiveness may be limited by liver damage, which sometimes leads to treatment discontinuation, and the mechanisms underlying this remain poorly ...
Clara Laffitte Redondo, Venceslas Bourdin, Abd El Kader Ait Tayeb, Sihem Benaboud, Inès Martins, Caroline Joyau, Myriam Benhamida, Isabelle Sermet‐Gaudelus, Clémence Martin, Pierre Régis Burgel, Céline Verstuyft, Laurent Chouchana, Estelle Ayme‐Dietrich   +12 more
wiley   +1 more source

Impact of cystic fibrosis transmembrane conductance regulator modulator therapies on liver stiffness and liver enzymes: An observational perspective single‐center cohort study

JPGN Reports, EarlyView.
Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano, Elisabetta Augustoni, Antonio Pizzol, Anna Opramolla, Cristina Chiadò, Anna Zagaria, Irene Esposito, Giulia Roberto, Alice Ponte, Andrea Evangelista, Paola Quarello, Michele Pinon, Pier Luigi Calvo   +12 more
wiley   +1 more source

Clinical efficacy of elexacaftor-tezacaftor-ivacaftor in an adolescent with homozygous G85E cystic fibrosis

, 2022
Elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) is a triple combination drug therapy approved for individuals with cystic fibrosis (CF) who possess at least one copy of the F508del cystic fibrosis transmembrane conductance regulator (CFTR) variant.
David Saul, Aaron Chidekel, Elena Stekolchik   +2 more
core   +1 more source

Triple combination cystic fibrosis transmembrane receptor modulator effects on glycaemia and insulin kinetics in cystic fibrosis with and without diabetes

Internal Medicine Journal, EarlyView.
Abstract Background Greater insight into the effects of cystic fibrosis (CF) transmembrane modulators such as elexacaftor‐tezacaftor‐ivacaftor (ETI) on glucose metabolism can support a more dynamic and individualised approach to CF‐related dysglycaemia.
Yi W. Chen, Rachel Prasad, Peter G. Middleton, D. Jane Holmes‐Walker   +3 more
wiley   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor in 2 cystic fibrosis adults homozygous for M1101K with end-stage lung disease

Respiratory Medicine Case Reports, 2023
Elexacaftor-tezacaftor-ivacaftor (ETI) therapy is shown to improve the health of individuals with cystic fibrosis (CF) who have the F508del variant. There are in vitro studies showing benefit with ETI for select rare CF variants.
Winnie M. Leung, Parastoo Molla Davoodi, Ashten Langevin, Clare Smith, Michael D. Parkins   +4 more
doaj   +1 more source

Comparison of Single‐Breath and Multi‐Breath Xe‐MRI in the Longitudinal Assessment of Treatment in Children With Cystic Fibrosis

Journal of Magnetic Resonance Imaging, Volume 63, Issue 6, Page 1652-1661, June 2026.
ABSTRACT Background Elexacaftor/tezacaftor/ivacaftor (ETI) is a current standard therapy for pediatric cystic fibrosis (CF). Multiple‐breath washout 129Xe MRI (MBW Xe‐MRI) is improved following 1 month of treatment. However, the utility of MBW Xe‐MRI over extended ETI treatment and its comparison to single‐breath Xe‐MRI and pulmonary function tests ...
Faiyza S. Alam, Samal Munidasa, Brandon Zanette, Daniel Li, Sharon Braganza, Felix Ratjen, Giles Santyr   +6 more
wiley   +1 more source

Impact of elexacaftor-tezacaftor-ivacaftor in lung transplantation for cystic fibrosis in the United States

JHLT Open
Background: Cystic fibrosis (CF) is an autosomal recessive condition leading to progressive lung disease and often necessitating lung transplantation. Historically, CF has been one of the leading indications for lung transplants in the United States. The
Tahuanty A. Pena, MD, MS, Brittany Wright, PharmD, Kalpaj R. Parekh, MBBS, Julia Kleney-Tait, MD, PhD   +3 more
doaj   +1 more source

Clinical Use of Home Spirometry in Children With Cystic Fibrosis

Pediatric Pulmonology, Volume 61, Issue 6, June 2026.
ABSTRACT Background The use of home spirometry (HSPIR) has increased in pediatric cystic fibrosis (CF) care, but how it has been used clinically and its impact on clinical care have not been described. The purpose of this study was to address this knowledge gap through a secondary analysis of data from a HSPIR quality improvement project to ...
Lucy Tan, Andrew Borowiec, James A. Reed, Mark Rath, Reid Masi, Laura Bennett, Don B. Sanders, Daniel J. Weiner, Clement L. Ren   +8 more
wiley   +1 more source

Cystic fibrosis-related diabetes in the era of modern treatment using CFTR modulators in pediatric patients—a systematic review

Frontiers in Pediatrics
BackgroundCystic fibrosis-related diabetes (CFRD) is a common comorbidity in cystic fibrosis (CF), particularly in the pediatric population. As cystic fibrosis transmembrane conductance regulator modulators (CFTRm) become widely used, there is growing ...
Anna Pietrzykowska, Jerzy Darmoń, Katarzyna Szymocha, Wiktoria Donocik, Mateusz Tarasiewicz, Julia Stadnik, Przemysława Jarosz-Chobot   +6 more
doaj   +1 more source

Elexacaftor–Tezacaftor–Ivacaftor Therapy for Cystic Fibrosis Patients with The F508del/Unknown Genotype

Antibiotics, 2021
The new CFTR modulator combination, elexacaftor/tezacaftor/ivacaftor (Trikafta) was approved by the FDA in October 2019 for treatment of Cystic Fibrosis in patients 6 years of age or older who have at least one F508del mutation in one allele and a ...
Marika Comegna, Vito Terlizzi, Donatello Salvatore, Carmela Colangelo, Antonella Miriam Di Lullo, Immacolata Zollo, Giovanni Taccetti, Giuseppe Castaldo, Felice Amato   +8 more
doaj   +1 more source