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DMD – Corticosteroid Rationale — SCE Neurology MCQ

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ModerateNeurogeneticsDMD – Corticosteroid RationaleSCE Neurology

A 30-year-old man with known Duchenne muscular dystrophy (diagnosed at age 5, wheelchair-dependent since age 12) presents with progressive respiratory insufficiency and dilated cardiomyopathy. He is on deflazacort. What is the rationale for long-term corticosteroid use in DMD?

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Correct answer: E — Deflazacort (or prednisolone) slows the decline in muscle strength and function, prolongs ambulation, preserves respiratory function, and reduces scoliosis — it is the most effective pharmacological intervention in DMD

Long-term corticosteroid therapy (deflazacort or prednisolone) is the most important pharmacological intervention in DMD. Benefits include: prolonged ambulation (by 1–3 years), preserved respiratory function, reduced scoliosis, delayed cardiomyopathy, and improved overall survival. Deflazacort may have fewer side effects (weight gain) than prednisolone. Steroids should be started when motor function plateaus or begins to decline (typically age 4–6), not after ambulation is lost. Gene therapy (exon skipping, micro-dystrophin) is emerging but not yet standard. A: Steroids have well-established benefits. C: They slow decline but do not cure. D: They are used continuously, not acutely. E: They should be started while still ambulant.

Reference: NICE HST3 Ataluren; DMD Care Considerations Working Group Guidelines