Long-Term Steroid Therapy in Duchenne Muscular Dystrophy-Positive Results versus Side Effects

Area of Science:

• Neurology
• Pediatrics
• Pharmacology

Background:

• Duchenne muscular dystrophy (DMD) is a progressive genetic disorder characterized by muscle degeneration.
• Long-term management strategies for DMD aim to slow disease progression and improve quality of life.

Purpose of the Study:

• To evaluate the efficacy and safety of long-term steroid treatment in patients with Duchenne muscular dystrophy.
• To compare outcomes in steroid-treated DMD patients with natural history data and an untreated control group.

Main Methods:

• Retrospective study of 19 male DMD patients receiving deflazacort (0.9 mg/kg).
• Comparison with literature-based natural history of DMD.
• Comparison of 13 treated patients with 13 age-matched untreated DMD patients.

Main Results:

• Steroid-treated patients showed significantly better muscle strength, functional grade (Vignos scale), and timed functional tests.
• Vital capacity was significantly improved in the treated group; no significant cardiologic differences were observed.
• Common side effects included obesity, cataracts, and short stature; infectious diseases and fractures were not increased.

Conclusions:

• Long-term deflazacort treatment (average 65 months) demonstrated significant benefits in muscle strength and function for DMD patients.
• The observed side effects were generally tolerable, supporting the use of steroids in DMD management compared to untreated cases.