Griggs R C, Moxley R T, Mendell J R, Fenichel G M, Brooke M H, Pestronk A, Miller J P, Cwik V A, Pandya S, Robison J
Department of Neurology, University of Rochester, NY.
Neurology. 1993 Mar;43(3 Pt 1):520-7. doi: 10.1212/wnl.43.3_part_1.520.
Prednisone has been shown to improve strength in Duchenne dystrophy. Azathioprine often benefits corticosteroid-responsive diseases and can reduce the dose of prednisone needed. The present study reports a randomized, controlled trial of prednisone and azathioprine designed to assess the longer-term effects of prednisone and to determine whether azathioprine alone, or in combination with prednisone, improves strength. Ninety-nine boys (aged five to 15 years) with Duchenne dystrophy were randomized to one of three groups: (I) placebo; (II) prednisone 0.3 mg/kg/d; or (III) prednisone 0.75 mg/kg/d. After 6 months, azathioprine 2 to 2.5 mg/kg/d was added in groups I and II and placebo added in group III. The study showed that the beneficial effect of prednisone (0.75 mg/kg/d) is maintained for at least 18 months and is associated with a 36% increase in muscle mass. There was weight gain, growth retardation, and other side effects. Azathioprine did not have a beneficial effect. This study suggests that prednisone's beneficial effect is not due to immunosuppression.
泼尼松已被证明可改善杜氏肌营养不良症患者的肌力。硫唑嘌呤通常对皮质类固醇反应性疾病有益，并且可以减少所需的泼尼松剂量。本研究报告了一项泼尼松和硫唑嘌呤的随机对照试验，旨在评估泼尼松的长期效果，并确定单独使用硫唑嘌呤或与泼尼松联合使用是否能改善肌力。99名年龄在5至15岁的杜氏肌营养不良症男孩被随机分为三组之一：（I）安慰剂组；（II）泼尼松0.3毫克/千克/天；或（III）泼尼松0.75毫克/千克/天。6个月后，在I组和II组中添加硫唑嘌呤2至2.5毫克/千克/天，在III组中添加安慰剂。研究表明，泼尼松（0.75毫克/千克/天）的有益效果至少维持18个月，并且与肌肉量增加36%相关。出现了体重增加、生长迟缓及其他副作用。硫唑嘌呤没有产生有益效果。这项研究表明，泼尼松的有益效果并非由于免疫抑制作用。
