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Editorial
. 2023 Nov 1;208(9):911-913.
doi: 10.1164/rccm.202309-1646ED.

Sustained Improvement of Cystic Fibrosis Lung Disease following Initiation of Elexacaftor/Tezacaftor/Ivacaftor Therapy: Lessons from Real-World Studies

Eva Steinke  1   2   3 , Mirjam Stahl  1   2   3
Affiliations
Editorial

Sustained Improvement of Cystic Fibrosis Lung Disease following Initiation of Elexacaftor/Tezacaftor/Ivacaftor Therapy: Lessons from Real-World Studies

Eva Steinke et al. Am J Respir Crit Care Med. .
No abstract available

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Figures

Figure 1.
Figure 1.
Real-world studies adding to our understanding of treatment effects after the initiation of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in people with cystic fibrosis (CF). (A and B) Current real-world evidence for the effects of ELX/TEZ/IVA on clinical signs and symptoms and CF-typical pathologies in adolescents and adults (A) and children (B). Small circles reflect short-term changes, and large circles represent long-term changes produced by ELX/TEZ/IVA. The green background highlights novel findings of the study by McNally and colleagues (13); the question marks highlight unknown effects. The upward arrow represents an improvement or increase, and the downward arrow denotes a reduction of the respective parameter.
See this image and copyright information in PMC

Comment on

References

    1. Graeber SY, Vitzthum C, Pallenberg ST, Naehrlich L, Stahl M, Rohrbach A, et al. Effects of elexacaftor/tezacaftor/ivacaftor therapy on CFTR function in patients with cystic fibrosis and one or two F508del alleles. Am J Respir Crit Care Med . 2022;205:540–549. - PubMed
    1. Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, et al. VX17-445-103 Trial Group Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet . 2019;394:1940–1948. - PMC - PubMed
    1. Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, et al. VX17-445-102 Study Group Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med . 2019;381:1809–1819. - PMC - PubMed
    1. Barry PJ, Mall MA, Álvarez A, Colombo C, de Winter-de Groot KM, Fajac I, et al. VX18-445-104 Study Group Triple therapy for cystic fibrosis Phe508del-gating and -residual function genotypes. N Engl J Med . 2021;385:815–825. - 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