Steroid versus Non-Steroid Regimen in Treating Duchenne Muscular Dystrophy: An Observational Study from South India

Abstract

There are no large studies from Southern India describing the treatment regimen of the Duchenne muscular dystrophy which is the most common muscular dystrophy. The current observational study aimed to compare steroid versus non-steroid treatment outcomes in Duchenne muscular dystrophy diagnosed population of South India. Immunohistochemically/genetically confirmed Duchenne muscular dystrophy patients diagnosed between February 2019 and March 2021 were ambispectively included. The main outcomes assessed in the study were loss of ambulation with respect to age, disease progression of motor milestones, medical research council grade, change in body weight and reported adverse events. We correlated these outcomes comparatively for both intervention groups (steroid and non-steroid). A total of 274 Duchenne muscular dystrophy patients were included in the study. The mean minimal age of onset of disease was 2.0±1.2 y. 32.85 % patients were treated with steroids and 67.15 % patients were on non-steroidal treatment. Comparatively, both regimen using patients demonstrated loss of ambulation at 13 y of age. Gait, stairs, Gower, chair scores reported loss of motor functions at 13 y and 25 y for steroid and non-steroid using patients respectively. Medical research council grade improved in steroid using patients to a greater extent compared to non-steroid using patients. Change in body weight and adverse events were comparatively low in non-steroid using patients to that of steroid using patients. The pattern of primary outcome measures in this large cohort is comparable despite variability in medical care. The contemporary observational study adds to the real-world evidence in guiding clinicians towards better approach in treating Duchenne muscular dystrophy patient populationa.