Abstract
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.
Footnotes
Cite 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).
- Copyright ©the authors 2021
