Corticosteroid treatment of DMD: summary of practice guideline for clinicians

This paper provides a summary of the 2005 American Academy of Neurology (AAN) guideline update, “Corticosteroid treatment of Duchenne muscular dystrophy.” With the current variations in practice in corticosteroid use, the following questions regarding patients with DMD were addressed:

  1. What is the efficacy of corticosteroids, specifically their effect on survival, quality of life (QoL), motor function, scoliosis, pulmonary function, and cardiac function?
    2. What are the side effects of corticosteroid treatment?
    3. How do prednisone and deflazacort compare in efficacy or side effect profile? 4.What is the optimal dosing regimen for corticosteroids?
    5. Are there any useful interventions for maximizing bone health?

Clinically, prednisone 0.75 mg/kg/d has significant benefit in DMD management and should be considered the optimal prednisone dose. Prednisone 10 mg/kg/ weekend is equally effective over a 12-month period, although long-term outcomes of this alternate regimen remain to be seen. Because of the expectation of significant adverse events with corticosteroids, proper informed consent is required, and adverse events should be discussed with patients and their families prior to therapy initiation and should be managed proactively.

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Gloss D, Moxley RT 3rd, Ashwal S, Oskoui M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline  Development Subcommittee of the American Academy of Neurology. Neurology. 2016 Feb 2;86(5):465-72.