Related Experiment Video
Updated: May 31, 2026
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy
D M Escolar1, L P Hache, P R Clemens
1Children's National Medical Center, Washington, DC, USA. diana.escolar@gmail.com
Insights
Weekend prednisone dosing is as effective and safe as daily dosing for boys with Duchenne muscular dystrophy (DMD). This approach preserves muscle strength and is well-tolerated over 12 months.
Area of Science:
- Neurology
- Pediatrics
- Pharmacology
Background:
- Duchenne muscular dystrophy (DMD) is a progressive genetic disorder.
- Corticosteroids, like prednisone, are a standard treatment for DMD.
- Optimizing prednisone dosing regimens is crucial for managing DMD.
Purpose of the Study:
- To compare the efficacy and safety of daily versus weekend prednisone dosing in boys with DMD.
- To evaluate muscle strength and side effect profiles of different prednisone schedules.
- To provide evidence-based recommendations for DMD treatment.
Main Methods:
- A double-blind, randomized study involving 64 boys aged 4-10 years with DMD.
- Comparison of daily prednisone (0.75 mg/kg/day) versus weekend prednisone (10 mg/kg/wk) over 12 months.
- Assessment of primary outcomes including quantitative muscle testing (QMT) arm and leg scores.
Main Results:
- Equivalence in efficacy was demonstrated for both daily and weekend prednisone regimens.
- No significant differences in secondary strength scores or side effect profiles were observed.
- Key safety indicators such as height, weight, bone density, and behavior remained comparable.
Conclusions:
- Weekend prednisone dosing is a viable and equally effective alternative to daily dosing for DMD.
- Both dosing schedules exhibit favorable tolerability and safety profiles.
- This study supports the use of less frequent prednisone administration in managing DMD.
Objective:
To perform a double-blind, randomized study comparing efficacy and safety of daily and weekend prednisone in boys with Duchenne muscular dystrophy (DMD).
Methods:
A total of 64 boys with DMD who were between 4 and 10 years of age were randomized at 1 of 12 centers of the Cooperative International Neuromuscular Research Group. Efficacy and safety of 2 prednisone schedules (daily 0.75 mg/kg/day and weekend 10 mg/kg/wk) were evaluated over 12 months.
Results:
Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score. Secondary strength scores for QMT elbow flexors also showed equivalence between the 2 treatment groups. Overall side effect profiles of height and weight, bone density, cataract formation, blood pressure, and behavior, analyzed at 12 months, did not differ between weekend and daily dosing of prednisone.
Conclusions:
Weekend dosing of prednisone is equally beneficial to the standard daily dosing of prednisone. Analysis of side effect profiles demonstrated overall tolerability of both dosing regimens.
Classification Of Evidence:
This study provides Class I evidence that weekend prednisone dosing is as safe and effective as daily prednisone in preserving muscle strength and preventing body mass index increases in boys with DMD over a 12-month period.
