Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy

Abstract

Methods: We systematically reviewed the literature from January 2004 to July 2014 using the AAN classification scheme for therapeutic articles and predicated recommendations on the strength of the evidence.Results: Thirty-four studies met inclusion criteria.Recommendations: In children with DMD, prednisone should be offered for improving strength (Level B) and pulmonary function (Level B). Prednisone may be offered for improving timed motor function (Level C), reducing the need for scoliosis surgery (Level C), and delaying cardiomyopathy onset by 18 years of age (Level C). Deflazacort may be offered for improving strength and timed motor function and delaying age at loss of ambulation by 1.4-2.5 years (Level C). Deflazacort may be offered for improving pulmonary function, reducing the need for scoliosis surgery, delaying cardiomyopathy onset, and increasing survival at 5-15 years of follow-up (Level C for each). Deflazacort and prednisone may be equivalent in improving motor function (Level C). Prednisone may be associated with greater weight gain in the first years of treatment than deflazacort (Level C). Deflazacort may be associated with a greater risk of cataracts than prednisone (Level C). The preferred dosing regimen of prednisone is 0.75 mg/kg/d (Level B). Over 12 months, prednisone 10 mg/kg/weekend is equally effective (Level B), with no long-term data available. Prednisone 0.75 mg/kg/d is associated with significant risk of weight gain, hirsutism, and cushingoid appearance (Level B). This document summarizes extensive information provided in the complete guideline, available as a data supplement on the Neurology ® Web site at Neurology.org. Appendices e-1 through e-5, cited in this summary, are presented in the full guideline (data supplement). Tables e-1 through e-5 as well as references e1-e8, cited in this summary, are available at Neurology.org.A 2005 American Academy of Neurology (AAN) guideline on the use of corticosteroids in patients with Duchenne muscular dystrophy (DMD) recommended prednisone or deflazacort in the treatment of DMD for short-term benefit in muscle strength and function.1 There are currently variations in practice in corticosteroid use. 2 We address the following questions with regard to patients with DMD: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?

Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

“…The first studies focusing on glucocorticoid therapy for DMD boys have demonstrated that prednisone, in a dose of 0.75/mg/Kg/daily, can achieve substantial effects on motor function in a six-month period 4,6,43 . Later studies have demonstrated that different drugs with an equivalent dose show similar effects: prednisone or prednisolone 0.75mg/Kg daily (Level of evidence: 1A, Class of Recommendation: A) 35 or deflazacort 0.9 to 1mg/Kg daily (Level of evidence: 3C, Class of Recommendation: C) 44,45 .…”

“…Since DMD patients have, in general, a lifelong prospect of glucocorticoid usage, an optimal follow-up schedule is necessary for monitoring possible side effects 6 . An ideal outline of routine consultations takes into account three relevant factors: the patient' s age, the type of glucocorticoid prescribed and the drug regimen adopted.…”

“…Although this is one question that remains unanswered 6 , indirect evidence suggests it should be continued throughout life (Level of evidence: 5D, Class of Recommendation: D, Expert opinion). Significant side effects should be properly managed during regular clinical reassessments and a regimen shift is recommended in those patients with significant side effects 6 . There are some special situations that require a dose adjustment considering the metabolic modifications during chronic glucocorticoid therapy.…”

“…Improvements in general care, steroid treatment, noninvasive ventilatory support, cardiomyopathy and scoliosis management may significantly change the course of DMD. Therefore, a review of the previous guidelines is necessary, while some new specific guidelines are underway, or have been recently published 6,7 . Evidence-based practice has been heralded as the most appropriate way of ensuring that patients receive the most effective care possible.…”

Upper arm and cardiac magnetic resonance imaging in Duchenne muscular dystrophy

“…Whereas corticosteroids and supportive measures have improved overall prognosis and lifespan, cardiomyopathy continues to be relentlessly progressive and has become a major cause of death 8. Recent guidelines emphasize the need for early diagnosis and aggressive treatment for cardiomyopathy in DMD 9.…”