MSR6 Modeling Survival of People with Cystic Fibrosis (PWCF) Aged ≥6 Years Heterozygous for the F508DEL Mutation with a Minimal Function Mutation (F/MF) in Brazil Treated with Elexacaftor/Tezacaftor/Ivacaftor and Ivacaftor (ELX/TEZ/IVA)

G Pedra,C Daly,La Pinto,A Lopez,G Vega-Hernandez,Jl Rubin

doi:10.1016/j.jval.2022.04.1213

MSR6 Modeling Survival of People with Cystic Fibrosis (PWCF) Aged ≥6 Years Heterozygous for the F508DEL Mutation with a Minimal Function Mutation (F/MF) in Brazil Treated with Elexacaftor/Tezacaftor/Ivacaftor and Ivacaftor (ELX/TEZ/IVA)

G Pedra, C Daly + Show 4 more

Open Access

https://doi.org/10.1016/j.jval.2022.04.1213

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Journal: Value in Health	Publication Date: Jun 25, 2022
License type: publisher-specific-oa

Affiliation: Vertex Pharmaceuticals (United Kingdom), Pontifícia Universidade Católica do Rio Grande do Sul, Vertex Pharmaceuticals (United States)

#People With Cystic Fibrosis #Cause Of Cystic Fibrosis + Show 8 more

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Abstract

ELX/TEZ/IVA is a breakthrough therapy targeting the underlying cause of cystic fibrosis that is currently not available in Brazil. ELX/TEZ/IVA has been shown to substantially improve lung function and nutritional outcomes. Although survival projections are available in non-Brazilian settings, the potential long-term impact of ELX/TEZ/IVA on Brazilian pwCF is of interest given regional variation in survival. Here we estimate survival in Brazilian F/MF pwCF aged ≥6 years receiving ELX/TEZ/IVA+best supportive care (BSC) versus BSC-alone.

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