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Daily prednisone treatment in duchenne muscular dystrophy in southwest china
Author(s) -
Hu Jun,
Ye Yuanzhen,
Kong Min,
Hong Siqi,
Cheng Li,
Wang Qiao,
Qin Jiaqiang,
Zou Lin,
Jiang Li
Publication year - 2015
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.24665
Subject(s) - prednisone , medicine , duchenne muscular dystrophy , placebo , muscular dystrophy , quality of life (healthcare) , physical therapy , surgery , alternative medicine , nursing , pathology
In this study we aimed to determine the influence of daily prednisone treatment in Duchenne muscular dystrophy (DMD) by performing a prospective, randomized, placebo‐controlled trial in southwestern China. Methods Sixty‐six children with DMD (4–12 years of age) were divided randomly into prednisone and placebo groups. Efficacy and safety of daily prednisone at 0.75 mg/kg/day were evaluated over 12 months by muscle strength and function, quality of life (QoL), quantitative muscle ultrasound (QMUS), and side effects. Results Significant improvements in muscle strength and function, QoL, and QMUS were observed in the prednisone group compared with the placebo‐treated group ( P < 0.05). Changes in body weight, height, body mass index, and diastolic blood pressure were similar in both groups ( P > 0.05). Conclusions This pilot study in southwestern China found that daily prednisone at 0.75 mg/kg/day is suitable for children with DMD. It slowed disease progression and improved QoL and QMUS. Moderate side effects were generally well tolerated. Muscle Nerve 52 : 1001–1007, 2015