Artigo Revisado por pares

Corticosteroids in Duchenne muscular dystrophy: Major variations in practice

2013; Wiley; Volume: 48; Issue: 1 Linguagem: Inglês

10.1002/mus.23831

ISSN

1097-4598

Autores

Robert C. Griggs, Barbara E. Herr, A. Reha, Gary Elfring, Leone Atkinson, Valerie Cwik, Elaine McColl, Rabi Tawil, Shree Pandya, Michael McDermott, Kate Bushby,

Tópico(s)

Exercise and Physiological Responses

Resumo

In 2004, a Cochrane Review and AAN practice parameter concluded that prednisone 0.75 mg/kg/day is of short-term efficacy in Duchenne muscular dystrophy (DMD). Subsequent efforts to standardize care for DMD indicated wide variation in corticosteroid use.We surveyed physicians who follow patients with DMD, including: (1) clinics in the TREAT-NMD (Translational Research in Europe-Assessment and Treatment of Neuromuscular Diseases) network (predominantly Europe) and (2) U.S. MDA clinic directors. We also documented the co-administered corticosteroids in a trial of a putative treatment (ataluren) for DMD.Of 105 Treat-NMD clinicians, corticosteroids were not used in 10 clinics, and 29 different regimens were used--the most frequent 0.75 mg/kg/day prednisone (61 centers); 10 days on/10 days off (36 centers); 0.9 mg/kg/day deflazacort (32 centers); and 5 mg/kg/day on weekends (10 centers). Similar diversity was identified in MDA clinics and in the ataluren trial.Variability in corticosteroid use suggests uncertainty about risks/benefits of corticosteroid regimens for DMD.

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