Biggar W D, Gingras M, Fehlings D L, Harris V A, Steele C A
Bloorview MacMillan Centre and Department of Paediatrics, University of Toronto, Toronto, Ontario, Canada.
J Pediatr. 2001 Jan;138(1):45-50. doi: 10.1067/mpd.2001.109601.
We report the long-term effects on muscle strength and side effects with deflazacort in Duchenne muscular dystrophy (DMD).
Boys with DMD between the ages of 7 and 15 years were reviewed retrospectively; 30 had been treated with deflazacort, and 24 had not. Muscle function, pulmonary function, and side effects were compared.
The boys not treated with deflazacort stopped walking at 9.8 +/-1.8 years. Seven of 30 treated boys had stopped walking at 12.3+/-2.7 years (P<.05), and of the 23 boys who were still walking, 21 were older than 10 years. Pulmonary function (percent predicted functional vital capacity) was significantly greater in treated boys at 15 years (88% +/- 18%) than in boys not treated (39%+/-20%) (P<.001). Between 9 and 15 years, treated boys were shorter. Between 9 and 13 years, treated boys weighed less. After 13 years the treated boys maintained their weight, whereas boys not treated lost weight. Asymptomatic cataracts developed in 10 of 30 boys who received deflazacort. Other potential side effects of deflazacort such as hypertension, glucosuria, acne, infection, or bruising were not more common.
We conclude that deflazacort can preserve gross motor and pulmonary function in boys with DMD with limited side effects.
我们报告了地夫可特对杜氏肌营养不良症（DMD）患者肌肉力量的长期影响及副作用。
回顾性分析7至15岁的DMD男孩；30名接受了地夫可特治疗，24名未接受治疗。比较了肌肉功能、肺功能及副作用。
未接受地夫可特治疗的男孩在9.8±1.8岁时停止行走。30名接受治疗的男孩中有7名在12.3±2.7岁时停止行走（P<0.05），在仍能行走的23名男孩中，21名年龄超过10岁。15岁时接受治疗的男孩肺功能（预计功能性肺活量百分比）显著高于未接受治疗的男孩（88%±18% 比 39%±20%）（P<0.001）。9至15岁期间，接受治疗的男孩身高较矮。9至13岁期间，接受治疗的男孩体重较轻。13岁后，接受治疗的男孩体重维持不变，而未接受治疗的男孩体重下降。30名接受地夫可特治疗的男孩中有10名出现无症状性白内障。地夫可特的其他潜在副作用如高血压、糖尿、痤疮、感染或瘀伤并不常见。
我们得出结论，地夫可特可保留DMD男孩的总体运动和肺功能，且副作用有限。
