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.
Objectives: We investigated if 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, 2022. Propensity score matching identified STRIDE and CINRG patient cohorts (N=260) 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 <30%. Results: The mean (standard deviation) ages at onset of first symptoms (STRIDE vs CINRG; N=260 per cohort) were 2.8 (1.7) and 2.8 (1.5) years, respectively. Most patients (STRIDE vs CINRG) received corticosteroids for ≥12 months (85.0% vs 83.8%), with a similar proportion receiving deflazacort (47.7% vs 44.2%) or other corticosteroids (41.9% vs 43.5%). Median (95% confidence interval [CI]) ages at %-predicted FVC <60% (STRIDE vs CINRG) were 17.7 (16.8, not estimable) and 15.3 (14.9, 16.5) years, respectively (p=0.0053). Median (95% CI) ages at %-predicted FVC <30% (STRIDE vs CINRG) were not estimable and 22.5 (20.3, 25.4) years, respectively (p=0.0008). Conclusions: These interim registry data suggest that treatment with ataluren and SoC in routine clinical practice slows disease progression in pulmonary function in nmDMD patients.