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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
40
Patients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day.
Patients will be administered prednisolone at a dose of 0.75 mg/kg/day for 10 consecutive days, followed by a 10-day steroid-free period.
Children's Hospital/University of Child Health Sciences
Lahore, Punjab Province, Pakistan
Treatment efficacy
Increase in the post-treatment North Star Ambulatory Assessment (NSAA) scores will be noted.
Time frame: 6 month after start of treatment
Weight gain
The frequency of patients who will gain weight as an adverse effect, will be noted
Time frame: 6 month after start of treatment
Hyperglycemia
The frequency of patients who will who will develop hyperglycemia as an adverse effect will be noted.
Time frame: 6 month after start of treatment
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