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.
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)
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
Parallel
Alternate-day prednisolone
DrugPatients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day.
Intermittent prednisolone
DrugPatients 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.
Treatment efficacy
Increase in the post-treatment North Star Ambulatory Assessment (NSAA) scores will be noted.
Weight gain
The frequency of patients who will gain weight as an adverse effect, will be noted
Hyperglycemia
The frequency of patients who will who will develop hyperglycemia as an adverse effect will be noted.
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Muhammad Aamir Latif
Lead sponsor
RESnTEC, Institute of Research
Sponsor institution
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