biostudies-literatureUnknown57(12)Cornet MFondation pour la Recherche Médicale<h4>Introduction</h4>Clinical trials for CFTR modulators consider mean changes of clinical status at the cohort level, and thus fail to assess the heterogeneity of the response. We aimed to study the different response profiles to lumacaftor-ivacaftor according to age in children with cystic fibrosis (CF).<h4>Methods</h4>A mathematical framework, including principal component analysis, data clustering, and data completion, was applied to a multicenter cohort of 112 children aged 6-18 years, treated with lumacaftor-ivacaftor. Studied parameters at baseline and 6 months included body mass index (BMI), number of days of antibiotics (ATB), Sweat test (ST), forced expiratory volume in 1 s expressed in percentage predicted (ppFEV₁ ), forced vital capacity (ppFVC), and forced expiratorPediatric pulmonology2992-2999https://www.ebi.ac.uk/biostudies/studies/S-EPMC9826158biostudies-literatureProfiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.PMC9826158Ciciriello FRoy VChedevergne FSermet-Gaudelus IBihouee TStoven VCornet MRobin GMarguet CLucidi VLebourgeois MBonnel ASReix PKelly MfalseProfiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.<h4>Introduction</h4>Clinical trials for CFTR modulators consider mean changes of clinical status at the cohort level, and thus fail to assess the heterogeneity of the response. We aimed to study the different response profiles to lumacaftor-ivacaftor according to age in children with cystic fibrosis (CF).<h4>Methods</h4>A mathematical framework, including principal component analysis, data clustering, and data completion, was applied to a multicenter cohort of 112 children aged 6-18 years, treated with lumacaftor-ivacaftor. Studied parameters at baseline and 6 months included body mass index (BMI), number of days of antibiotics (ATB), Sweat test (ST), forced expiratory volume in 1 s expressed in percentage predicted (ppFEV₁ ), forced vital capacity (ppFVC), and forced expirator2022-01-01T00:00:00Z2022 Dec2025-04-05T10:26:52.47Z2025-04-05T10:26:52.47ZS-EPMC98261583599621410.1002/ppul.26123