Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up

ABSTRACT Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6‐month intervals for 2 years. We analyzed all subjects using an intent‐to‐treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty‐six completed 12‐month follow‐up, and 51 completed 24‐month follow‐up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non‐ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016