Pulmonary Function in Nonambulatory Patients with nmDMD from the STRIDE Registry and CINRG Duchenne Natural History Study: A Matched Cohort Analysis

Background: STRIDE (Strategic Targeting of Registries and International Database of Excellence) is an ongoing, multicenter, observational registry providing data on ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) in routine clinical practice.

Objective: To examine whether nonambulatory patients with nmDMD receiving ataluren + standard of care experienced a lesser decline in pulmonary function compared with matched patients with DMD receiving SoC alone (Cooperative International Neuromuscular Research Group Duchenne Natural History Study [CINRG DNHS; NCT00468832]). Data cut-off was January 31, 2019.

Propensity score matching was performed to identify non-ambulatory patients from STRIDE and CINRG DNHS with comparable predictors of disease progression: age at first symptoms; age at first corticosteroid use; duration of deflazacort use; and duration of other corticosteroid use. Patients from CINRG DNHS who received any other mutation-specific investigational drug for DMD were excluded. Kaplan–Meier analyses estimated age at loss of ambulation (LOA) and age at pulmonary function decline.

Results: Median age at LOA (95% confidence interval [CI]) for matched STRIDE versus CINRG DNHS cohorts (each n = 22) was 12.4 (10.7, 12.9) versus 11.1 (10.0, 12.5) years.

Mean (95% CI) ataluren exposure for patients in STRIDE up to LOA was 302 (163,440) days. Median (95% CI) age for patients reaching %-predicted forced vital capacity (FVC) < 60% (each n = 22) was delayed for STRIDE versus CINRG DNHS (18.7 [17.7, 18.7] vs. 15.6 [13.2, 16.7] years). Mean (95% CI) ataluren exposure in STRIDE up to %-predicted FVC <60% was 661 (495,826) days.

Conclusion: These data suggest that ataluren + SoC treatment slows down pulmonary disease progression in nonambulatory patients with nmDMD.

Publication History

Article published online:
28 October 2021