Impact of age at ivacaftor initiation on pulmonary outcomes among people with cystic fibrosis.

Autor: Merlo CA; Johns Hopkins School of Medicine, Baltimore, Maryland, USA cmerlo@jhmi.edu., McGarry LJ; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA., Thorat T; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA., Nguyen C; Analysis Group Inc, Los Angeles, California, USA., DerSarkissian M; Analysis Group Inc, Los Angeles, California, USA., Muthukumar A; Analysis Group Inc, Menlo Park, California, USA., Healy J; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA., Brookhart MA; Duke University, Durham, North Carolina, USA., Rubin JL; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA.
Jazyk: angličtina
Zdroj: Thorax [Thorax] 2024 Sep 18; Vol. 79 (10), pp. 915-924. Date of Electronic Publication: 2024 Sep 18.
DOI: 10.1136/thorax-2023-220559
Abstrakt: Background: Ivacaftor (IVA) improves lung function and other extrapulmonary outcomes in people with cystic fibrosis (CF). However, the effect of initiating IVA at earlier versus later ages has not been studied.
Methods: We conducted an observational cohort study of people in the US CF Foundation Patient Registry aged ≥6 years with ≥1 CF transmembrane conductance regulator-gating mutation to compare the effects of initiating IVA at earlier ages on per cent predicted forced expiratory volume in 1 s (ppFEV 1 ) and pulmonary exacerbation (PEx) outcomes. People with CF were grouped by age at IVA initiation (ages 6-10, 11-15, 16-20 and 21-25 years) to perform three analyses of younger versus older IVA initiation (6-10 vs 11-15, 11-15 vs 16-20 and 16-20 vs 21-25 years). For each analysis, baseline characteristics assessed over 1-year periods at the same age prior to IVA initiation were balanced by standardised mortality/morbidity ratio (SMR) weighting. For each analysis, outcomes were compared over a 5-year outcome assessment period when both groups were in the same age range and receiving IVA.
Findings: Baseline characteristics were well balanced between younger and older IVA initiator groups after SMR weighting. In the outcome assessment period, younger IVA initiators had significantly higher mean ppFEV 1 than older initiators across all comparisons, and those initiating IVA between ages 6-10 and 11-15 years had significantly lower PEx rates.
Interpretation: Study findings showed the importance of early IVA initiation in people with CF.
Competing Interests: Competing interests: All authors received nonfinancial support (assistance with manuscript preparation) from ArticulateScience, LLC, which was funded by Vertex Pharmaceuticals Incorporated. CAM reports funding from Vertex Pharmaceuticals. CN, MD and AM are employees of Analysis Group, Incorporated, which received research funding from Vertex Pharmaceuticals. LJM, TT, 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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