Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis.
| Autor: | Merlo CA; Johns Hopkins School of Medicine, Baltimore, Maryland, USA cmerlo@jhmi.edu., Thorat T; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA., DerSarkissian M; Analysis Group Inc, Los Angeles, California, USA., McGarry LJ; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA., Nguyen C; Analysis Group Inc, Los Angeles, California, USA., Gu YM; Analysis Group Inc, Los Angeles, California, USA., Healy J; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA., Rubin JL; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA., Brookhart MA; Duke University, Durham, North Carolina, USA. |
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| Jazyk: | angličtina |
| Zdroj: | Thorax [Thorax] 2024 Sep 18; Vol. 79 (10), pp. 925-933. Date of Electronic Publication: 2024 Sep 18. |
| DOI: | 10.1136/thorax-2023-220558 |
| Abstrakt: | Background: Ivacaftor (IVA) has been shown to improve lung function and other clinical outcomes in people with cystic fibrosis (CF). A decade of real-world IVA availability has enabled the examination of long-term outcomes with this treatment. This retrospective, longitudinal cohort study investigated the impact of IVA on mortality rate and health outcomes among people with CF in the US. Methods: Data from the US CF Foundation Patient Registry from January 2010 to December 2019 were analysed. The IVA-treated cohort included people with a CF transmembrane conductance regulator ( CFTR ) gating mutation (excluding R117H ); age-matched comparator cohort included people with a F508del and a minimal function CFTR mutation who had no prior CFTR modulator treatment. Baseline characteristics were balanced between cohorts using standardised mortality ratio weighting generated from propensity scores. Outcomes of interest were overall survival, lung transplant, percent predicted forced expiratory volume in 1 s (ppFEV Findings: Over a maximum follow-up of 7.9 years, the IVA-treated cohort (N=736) had lower rates of mortality (hazard ratio [HR] (95% CI): 0.22 (0.09 to 0.45)), lung transplant (HR: 0.11 (95% CI 0.02 to 0.28)), PEx (rate ratio: 0.49 (95% CI 0.42 to 0.55)) and all-cause hospitalisations (rate ratio: 0.50 (95% CI 0.43 to 0.56)) as well as better lung function (mean difference in ppFEV Interpretation: Our analysis suggests that IVA provides sustained clinical benefits in people with CF over a follow-up period of approximately 8 years. These findings reinforce the existing real-world evidence that IVA can slow disease progression and decrease the healthcare burden of CF over the long term. Competing Interests: Competing interests: All authors received non-financial support (assistance with manuscript preparation) from ArticulateScience, which was funded by Vertex Pharmaceuticals. CAM reports funding from Vertex Pharmaceuticals. MD, CN and YMG are employees of Analysis Group, which received research funding/consultancy fees from Vertex Pharmaceuticals. TT, LJM, JH and JLR are current or former employees of Vertex Pharmaceuticals and may own stock or stock options in that company. MAB has served as a scientific advisory committee member for Amgen, Astellas/Seagen, Atara Biotherapeutics, Brigham and Women’s Hospital, Kite, Gilead, Intercept, NIDDK, and Vertex Pharmaceuticals and has consulting fees/equity with Target RWE and equity with Accompany Health. (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.) |
| Databáze: | MEDLINE |
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