Real-World Outcomes of Elexacaftor/Tezacaftor/Ivacaftor in Children with Cystic Fibrosis

Lucy Perrem^{1

2}, Paul McNally^{1

3}

Affiliations

Editorial

Lucy Perrem et al. Ann Am Thorac Soc. 2025 Nov.

. 2025 Nov;22(11):1650-1651.

doi: 10.1513/AnnalsATS.202509-988ED.

Lucy Perrem^{1

2}, Paul McNally^{1

3}

No abstract available

Figures

Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in 6- to 11-Year-Olds with Cystic Fibrosis: An Observational Study.
Pittman JE, Morgan S, McCreary M, Vu PT, Jorth P, Heltshe S, Hoffman LR, Kelly A, Sagel SD, Singh PK, Solomon GM, Rosenfeld M, Ratjen F; PROMISE Pediatric substudy Investigators. Pittman JE, et al. Ann Am Thorac Soc. 2025 Nov;22(11):1696-1708. doi: 10.1513/AnnalsATS.202501-103OC. Ann Am Thorac Soc. 2025. PMID: 40540670

1. Zemanick ET, Taylor-Cousar JL, Davies J, Gibson RL, Mall MA, McKone EF. et al. A phase 3 open-label study of elexacaftor/tezacaftor/ivacaftor in children 6 through 11 years of age with cystic fibrosis and at least one F508del allele. Am J Respir Crit Care Med . 2021;203:1522–1532. - PMC - PubMed
1. Mall MA, Brugha R, Gartner S, Legg J, Moeller A, Mondejar-Lopez P. et al. Efficacy and safety of elexacaftor/tezacaftor/ivacaftor in children 6 through 11 years of age with cystic fibrosis heterozygous for F508del and a minimal function mutation: a phase 3b, randomized, placebo-controlled study. Am J Respir Crit Care Med . 2022;206:1361–1369. - PMC - PubMed
1. Pittman JE, Morgan S, McCreary M, Vu PT, Jorth P, Heltshe S. et al. Clinical effectiveness of elexacaftor/tezacaftor/ivacaftor in 6 to 11-year-olds with cystic fibrosis. Ann Am Thorac Soc . 2025;22:1696–1708. - PMC - PubMed
1. Stahl M, Dohna M, Graeber SY, Sommerburg O, Renz DM, Pallenberg ST. et al. Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or two F508del alleles. Eur Respir J . 2024;64:2400004. - PMC - PubMed
1. Nichols DP, Paynter AC, Heltshe SL, Donaldson SH, Frederick CA, Freedman SD. et al. PROMISE Study; Group. Clinical effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: a clinical trial. Am J Respir Crit Care Med . 2022;205:529–539. - PMC - PubMed