Fersiynau electronig

Dogfennau

Dangosydd eitem ddigidol (DOI)

  • Paulos Gebrehiwet
    Cytokinetics
  • Lisa Meng
    Cytokinetics
  • Phil Saroccoa
    Cytokinetics
  • Jenny Wei
    Cytokinetics
  • Andrew A. Wolff
    Cytokinetics
  • Michael Butzner
    Cytokinetics
  • Adriano Chio
    University of Turin
  • Jinsey A. Andrews
    Columbia University, New York
  • Angela Genge
    Montreal Neurological Institute-Hospital
  • Dyfrig Hughes
  • Carlayne E. Jackson
    University of Texas at San Antonio
  • Noah Lechtzin
    The Johns Hopkins University School of Medicine
  • Timothy M. Miller
    Washington University in St Louis
  • Jeremy M. Shefner
    Creighton University, Phoenix
Aims
To estimate the health utilities and quality-adjusted life years (QALYs) in patients with amyotrophic lateral sclerosis (ALS) receiving reldesemtiv versus placebo in FORTITUDE-ALS.

Materials and methods
We performed a post hoc analysis of clinical trial data from FORTITUDE-ALS (NCT03160898). This Phase IIb, double-blind, randomized, dose-ranging, placebo-controlled, parallel-group, 12-week trial evaluated reldesemtiv in patients with ALS. Health utilities from the five-level version of the EuroQol five-dimensional questionnaire (EQ-5D-5L) were estimated using ALS Functional Rating Scale-Revised (ALSFRS-R) scores collected during the trial. QALYs were estimated using the area under the curve method.

Results
The full analysis set consisted of 456 patients (reldesemtiv n = 342, placebo n = 114), who received at least one dose of the double-blind study drug, and had ALSFRS-R assessed at baseline and at least one post-baseline assessment. The difference in EQ-5D-5L utility least-squares (LS) mean change from baseline to week 12 for reldesemtiv versus placebo, adjusted for baseline values, was statistically significant (0.03, 95% confidence interval [CI]: 0.01, 0.05; p = .0008). The incremental QALY of reldesemtiv versus placebo adjusted for baseline utility values showed a modest, but statistically significant, difference (0.004, 95% CI: 0.001, 0.007; p = .0058).

Conclusions
This post hoc analysis of FORTITUDE-ALS suggests that reldesemtiv showed a modest but significant benefit in health utilities and QALYs compared with placebo. Future long-term studies that include direct collection of EQ-5D-5L data will be needed to confirm our findings.

ClinicalTrials.gov identifier
NCT03160898
Iaith wreiddiolSaesneg
Tudalennau (o-i)488-493
Nifer y tudalennau6
CyfnodolynJournal of Medical Economics
Cyfrol26
Rhif y cyfnodolyn1
Dyddiad ar-lein cynnar17 Maw 2023
Dynodwyr Gwrthrych Digidol (DOIs)
StatwsCyhoeddwyd - 31 Maw 2023

Cyfanswm lawlrlwytho

Nid oes data ar gael
Gweld graff cysylltiadau