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Prednisone therapy in Becker's muscular dystrophy.
1Blue Bird Clinic, Department of Pediatrics, Baylor College of Medicine, Houston, Texas 77030, USA. sdjohnse@texaschildrenshospital.org
Journal of Child Neurology
|December 6, 2001
Summary
Therapeutic prednisone offers significant strength improvement for some boys with Becker's muscular dystrophy. This corticosteroid treatment may benefit a subset of patients with this genetic muscle disorder.
Area of Science:
- Neurology
- Genetics
- Biochemistry
Background:
- Becker's muscular dystrophy is a genetic disorder characterized by progressive muscle weakness.
- Xp-21 gene defects are associated with Becker's muscular dystrophy.
- Current treatments for Becker's muscular dystrophy are limited.
Observation:
- Two boys diagnosed with Becker's muscular dystrophy showed remarkable strength gains with prednisone therapy.
- Serum creatine kinase levels decreased concurrently with strength improvement.
- Muscle biopsies revealed changes in dystrophin quantity and histology consistent with treatment efficacy.
Findings:
- Prednisone treatment led to dramatic and sustained improvement in muscle strength in these patients.
- A decrease in serum creatine kinase levels correlated with clinical improvement.
- Histological and immunoblot analysis of muscle biopsies indicated a positive response to prednisone.
Implications:
- Therapeutic corticosteroids, specifically prednisone, may be beneficial for a subset of Becker's muscular dystrophy patients.
- All patients with Becker's muscular dystrophy should consider a trial of prednisone to assess potential benefits.
- This finding could lead to revised treatment guidelines for Becker's muscular dystrophy.