Understanding the Causes of Duchenne Muscular Dystrophy

Clinical Brief: Steroid Therapy Benefits and Limitations in DMD

Main Discussion Topics

• Prednisone and deflazacort demonstrate significant delay in disease progression
• Potential side effects including mood disorders, bone weakness, weight gain, and stunted growth
• Individualized risk-benefit assessment for continued steroid therapy
• Long-term impact on respiratory and cardiac function less clearly established

Key Points for Physicians

• Regular steroid use can add several years of maintained mobility and independence
• Monitor for behavioral changes and hyperactivity which may limit tolerability
• Risk of fractures increases with long-term use, particularly in older boys
• Dosing regimens (daily vs. weekly) should be individualized based on response and side effects

Notable Insights

While steroid therapy remains a cornerstone of treatment, the balance between functional benefits and side effects requires ongoing assessment. Tolerability varies significantly between patients, necessitating personalized approaches.

Clinical Significance

Despite potential adverse effects, corticosteroid therapy provides substantial benefit in delaying disease progression, with the greatest evidence for preserved ambulation but likely additional benefits for respiratory and cardiac function that require further study.