Impact of CFTR Modulator Use on Outcomes in People with Severe Cystic Fibrosis Lung Disease

Overview

Journal Eur Respir Rev

Specialty Pulmonary Medicine

Date 2020 Mar 22

PMID 32198216

Citations 46

Authors

Michal Shteinberg

Jennifer L Taylor-Cousar

Affiliations

Soon will be listed here.

Abstract

Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation. While the hope is that these drug compounds will prevent lung damage when started early in life, there is an ongoing need to care for people with advanced lung disease. The focus of this review is the accumulating data from clinical trials and case series regarding the benefits of CFTR modulator therapy in people with advanced pulmonary disease. We address the impact of treatment with ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor on lung function, pulmonary exacerbations, nutrition and quality of life. Adverse events of the different CFTR modulators, as well as the potential for drug-drug interactions, are discussed.

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