PT  - JOURNAL ARTICLE
AU  - Claire Kim
AU  - Christopher Lee
AU  - Rachel Zahigian
AU  - Nitin Nair
AU  - Ase Sewall
AU  - Alexander Elbert
TI  - Real-world effects of lumacaftor/ivacaftor (LUM/IVA) in people with cystic fibrosis (pwCF): interim results of a long-term safety study using US CF Foundation Patient Registry (CFFPR) data
AID  - 10.1183/13993003.congress-2021.PA2098
DP  - 2021 Sep 05
TA  - European Respiratory Journal
PG  - PA2098
VI  - 58
IP  - suppl 65
4099  - http://erj.ersjournals.com/content/58/suppl_65/PA2098.short
4100  - http://erj.ersjournals.com/content/58/suppl_65/PA2098.full
SO  - Eur Respir J2021 Sep 05; 58
AB  - Background: LUM/IVA is approved to treat the underlying cause of CF in pwCF homozygous for F508del (F/F).Aims and objectives: This ongoing 5-year safety surveillance study evaluates safety and disease progression in F/F pwCF treated with LUM/IVA in the real world.Methods: This interim analysis included safety and disease progression cohorts of pwCF in the US CFFPR aged ≥2 y treated with LUM/IVA in 2019 and pwCF aged ≥6 y treated with LUM/IVA from 2015 or 2016 through 2019, respectively. Outcomes were compared to a concurrent comparator (COMP) of pwCF heterozygous for F508del and a minimal function mutation and no history of CFTR modulator use. Outcomes included safety (death and organ transplant) and disease progression (percent predicted [pp] FEV1 and pulmonary exacerbations [PEx]).Results: LUM/IVA safety cohort (n=3,182) had a lower age-adjusted odds ratio of death (OR=0.16; 95% CI: 0.07, 0.38) and organ transplant (OR=0.40; 95% CI: 0.22, 0.70) than COMP (n=4,099). LUM/IVA disease progression cohort had less decline in ppFEV1 than COMP (Figure, A), while PEx remained stable with LUM/IVA but increased in COMP (Figure, B). No new safety signals were identified.Conclusions: These data support the potential for disease modification with CFTR-targeted treatment and long-term LUM/IVA use.Sponsor: Vertex Pharmaceuticals Inc.FootnotesCite this article as: European Respiratory Journal 2021; 58: Suppl. 65, PA2098.This abstract was presented at the 2021 ERS International Congress, in session “Prediction of exacerbations in patients with COPD”.This is an ERS International Congress abstract. No full-text version is available. Further material to accompany this abstract may be available at www.ers-education.org (ERS member access only).