Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children &#x2265;2 years with cystic fibrosis: 96-week interim results from a phase 3 open-label extension study

RECOMMENDED

Pulmonary Fibrosis

Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: Results from an open-label extension study

★ 2 Recommended by Dr. med. Martin Frey, Faculty Member

★ 1 Recommended by Dr Marcus Hesse, Faculty Member

Ann Am Thorac Soc

Chmiel JF, McBennett K, Quon BS, Colombo C, De Wachter E, Fajac I, Mondejar-Lopez P, Polineni D, Robinson P, Sutharsan S, Mall MA, McKone EF, Ramsey B, Taylor-Cousar JL, Tullis E, Suresh NE, Zhang Y, Sosnay P, Jennings M, Barry PJ, 445-110 study group

doi: 10.1093/annalsats/aaoag053

Pulmonary HypertensionFurther Publications

Assessment of efficacy and tolerability of elexacaftor-tezacaftor-ivacaftor in an observational cohort study of "aged" people with cystic fibrosis

Ther Adv Respir Dis

Blankenship S, Flueckiger BV, Caleb G, Bailey J, Baker E, Garcia B, Krick S

doi: 10.1177/17534666251410246

RECOMMENDED

Pulmonary Fibrosis

Impact of Elexacaftor-Tezacaftor-Ivacaftor on fungal colonisation in children and adolescents with cystic fibrosis

★ 2 Recommended by Dr. med. Martin Frey, Faculty Member

★ 1 Recommended by Dr Marcus Hesse, Faculty Member

J Cyst Fibros

Sitterlé E, Bonnel AS, Roussey-Bihouee T, Blondé A, Ribault M, Wizla N, Bresson C, Persat F, Maubon D, Leroy J, Hassani F, Benhala L, Mounier M, Znaidi S, Ferroni A, Bougnoux ME, Sermet-Gaudelus I, Mycologist and Modul-CF groups

doi: 10.1016/j.jcf.2026.02.012

Further Publications

Sweat chloride and lung function responses to elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis with two versus one responsive CFTR variants: an analysis of two real-world observational studies

Lancet Respir Med

Burgel PR, Da Silva J, Girodon E, Durieu I, Reynaud-Gaubert M, Murris-Espin M, Chiron R, Grenet D, Ramel S, Mely L, Hamidfar R, Douvry B, Houdouin V, Audousset C, Macey J, Mittaine M, Weiss L, Cosson L, Danner-Boucher I, Reix P, Baravalle M, Burnet E, Sermet-Gaudelus I, Martin C, Paillasseur JL, French Cystic Fibrosis Reference Network study group

doi: 10.1016/S2213-2600(25)00190-0

RECOMMENDED

Pulmonary Fibrosis

Severe hepatotoxicity is uncommon following the introduction of Elexacaftor/Tezacaftor/Ivacaftor: A real-world two-years follow-up study of the Danish cystic fibrosis cohort

★ 1 Recommended by Dr. med. Martin Frey, Faculty Member

J Cyst Fibros

Højte C, Olsen MF, Faurholt-Jepsen D, Jørgensen MH, Pressler T, Leo-Hansen C, Bryrup T, Jeppesen M, Olesen HV, Petersen J, Katzenstein TL, Nielsen BU, Skov M, TransformCF Study Group

doi: 10.1016/j.jcf.2025.08.008

RECOMMENDED

Pulmonary FibrosisBronchi

Effect of elexacaftor-tezacaftor-ivacaftor on bronchial dilatations in adolescents with cystic fibrosis: a multicentre prospective observational study

★ 3 Recommended by Dr. med. Martin Frey, Faculty Member

Lancet Respir Med

Sermet-Gaudelus I, Letierce A, Berteloot L, Bonnel AS, Chen Y, Makani P, Kelly-Aubert M, Kanoun F, Penalva L, Bouleghem N, Bihouee T, Bui S, Corvol H, Houdouin V, Mittaine M, Tatopoulos A, Weiss L, Wizla N, Kapel N, Bessou A, Tiddens HAWM, Caudri D, Reix P, Marguet C, MODUL-CF study group

doi: 10.1016/S2213-2600(25)00248-6

RECOMMENDED

Pulmonary FibrosisFurther Publications

Clinical efficacy of elexacaftor-tezacaftor-ivacaftor in two siblings with homozygous I1234V mutation cystic fibrosis: A prospective case series

★ 1 Recommended by Dr. med. Martin Frey, Faculty Member

Respir Med Case Rep

Alzaid M, Alshahrani T, Alotaibi R, Mukhtar G, Alanazi A, Alsadoon H, Eltahir S, Aldraihem A, Alotaibi W

doi: 10.1016/j.rmcr.2025.102264

RECOMMENDED

Lung Cancer

The Italian Mauris Phase IIIb Trial of Atezolizumab Plus Carboplatin and Etoposide for Patients With Newly Diagnosed Extensive-Stage Small Cell Lung Cancer: 3-Year End-of-Study Results

★ 1 Recommended by Dr. med. Martin Frey, Faculty Member

Clin Lung Cancer

Morgillo F, Del Signore E, Bria E, de Marinis F, Ciardiello F, Garassino MC, Stefani A, Verderame F, Morabito A, Sbrana A, Tonini G, Gilli M, Berardi R, Taveggia PA, Bearz A, Delmonte A, Migliorino MR, Gridelli C, Fadda GM, Iero M, Ardizzoni A

doi: 10.1016/j.cllc.2025.07.003

RECOMMENDED

Further Publications

Impact of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance and Abnormal Glucose Metabolism: A Phase 3b, Open-Label Clinical Trial

★ 2 Recommended by Dr. med. Martin Frey, Faculty Member

Am J Respir Crit Care Med

Durieu I, Clements B, Fabrizzi B, Mall MA, McKone E, Ramsey B, Tullis E, Taylor-Cousar JL, van der Meer R, Bachman E, Chin A, Conner S, Jennings M, Weinstock T, Colombo C, Robinson P

doi: 10.1164/rccm.202411-2312OC

Pneumology

Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children ≥2 years with cystic fibrosis: 96-week interim results from a phase 3 open-label extension study

Related content

Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: Results from an open-label extension study

Assessment of efficacy and tolerability of elexacaftor-tezacaftor-ivacaftor in an observational cohort study of "aged" people with cystic fibrosis

Impact of Elexacaftor-Tezacaftor-Ivacaftor on fungal colonisation in children and adolescents with cystic fibrosis

Sweat chloride and lung function responses to elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis with two versus one responsive CFTR variants: an analysis of two real-world observational studies

Severe hepatotoxicity is uncommon following the introduction of Elexacaftor/Tezacaftor/Ivacaftor: A real-world two-years follow-up study of the Danish cystic fibrosis cohort

Effect of elexacaftor-tezacaftor-ivacaftor on bronchial dilatations in adolescents with cystic fibrosis: a multicentre prospective observational study

Clinical efficacy of elexacaftor-tezacaftor-ivacaftor in two siblings with homozygous I1234V mutation cystic fibrosis: A prospective case series

The Italian Mauris Phase IIIb Trial of Atezolizumab Plus Carboplatin and Etoposide for Patients With Newly Diagnosed Extensive-Stage Small Cell Lung Cancer: 3-Year End-of-Study Results

Impact of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance and Abnormal Glucose Metabolism: A Phase 3b, Open-Label Clinical Trial