Background: Strategic Targeting of Registries and International Database of Excellence (STRIDE; NCT02369731) is an ongoing, multicenter, observational registry providing data on ataluren use in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients in routine clinical practice.
Objective: We investigated whether nmDMD patients receiving ataluren plus standard of care (SoC) in the STRIDE Registry experienced a lesser decline in pulmonary function versus DMD patients receiving SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Natural History Study (NCT00468832).
Methods: Data were extracted on January 31, 2023. Propensity score matching identified STRIDE and CINRG patient cohorts (N=277) comparable in established predictors of disease progression: age at first symptoms; age at initiation of corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use. Patients from CINRG who had received investigational drugs for DMD were excluded from this analysis. Kaplan–Meier analyses were used to estimate ages at %-predicted forced vital capacity (FVC) <60% and 50%. Results: The mean (SD) ages at onset of first symptoms (STRIDE vs CINRG; N=277 per cohort) were 2.8 (1.7) and 2.9 (1.5) years, respectively. Most patients (STRIDE vs CINRG) received corticosteroids for ≥12 months (88.8% vs 89.2%), with a similar proportion receiving deflazacort (52.0% vs 47.7%) or other corticosteroids (44.8% vs 48.4%). Median (95% confidence interval [CI]) ages at %-predicted FVC <60% (STRIDE vs CINRG) were 17.7 (16.9, 19.9) and 15.6 (15.1, 16.4) years, respectively (HR [95% CI] 0.630 [0.440, 0.903]; p=0.0086). Median (95% CI) ages at %-predicted FVC <50% (STRIDE vs CINRG) were 19.2 (17.8, not estimable) and 17.7 (16.5, 18.6) years, respectively (HR [95% CI] 0.630 [0.426, 0.932]; p=0.0206). Conclusions: These real-world, multi-country data suggest that treatment with ataluren and SoC in routine clinical practice slows disease progression in pulmonary function in nmDMD patients.