Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy

Efficacy and Safety of Intermittent vs. Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy: A Randomized, Open-Label Trial

The alternate-day regimen is hypothesized to reduce hypothalamic-pituitary-adrenal (HPA) axis suppression and decrease steroid-related toxicity. Similarly, intermittent regimens aim to provide periodic steroid exposure sufficient for muscle preservation while allowing recovery periods to limit side effects. There is a critical need for localized, prospective data to determine which protocol offers the optimal balance of efficacy and safety for pediatric patients in Pakistan.

The study will provide evidence to support the standardization of corticosteroid therapy in the local population. By directly comparing alternate-day versus intermittent dosing at The University of Child Health Sciences & Children's Hospital, Lahore, the study aims to identify the regimen that best preserves motor function while minimizing debilitating side effects, thereby improving overall quality of life and treatment compliance among local patients with Duchenne Muscular Dystrophy (DMD).

Inclusion Criteria:

• Male patients
• Aged 4-10 years
• 2. Confirmed diagnosis of DMD (via genetic testing and significantly elevated creatine kinase (CK) levels >1000 U/L with typical clinical presentation)

Exclusion Criteria:

• Patients with pre-existing cardiopulmonary failure
• Patients entirely unable to perform baseline ambulatory assessments (non-ambulatory)
• Patients with clinically suspected muscular dystrophy with normal genetic study for DMD