DEFLAZACORT IN DUCHENNE DYSTROPHY - STUDY OF LONG-TERM EFFECT
DEFLAZACORT IN DUCHENNE DYSTROPHY - STUDY OF LONG-TERM EFFECT
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DOI：
10.1002/mus.880170405
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发表时间：
1994-04-01
期刊：
影响因子：
3.4
通讯作者：
COSTA, C
中科院分区：
文献类型：
--
作者：
ANGELINI, C;PEGORARO, E;COSTA, C
在28例Duchenne型肌营养不良症患者中进行了一项地夫可特随机双盲对照试验，患者接受地夫可特2.0 mg/kg隔日治疗或安慰剂治疗。治疗6个月后，地夫可特组在爬楼梯（P < 0.01）、从椅子上起身、Gower动作和行走（P < 0.0025）方面均有显著改善。治疗1年后，治疗组上述各项指标均明显改善，MRC指数明显优于对照组（P < 0.05）。2年后，MRC指数发生了显著变化：行走，椅子上升得分较高（P < 0.02），Gower's手法评分及时间试验开始后，治疗组的平均脱发时间为20.5 ± 11个月;安慰剂组为33.2 +/-9个月（安慰剂对地夫可特组，P < 0.05）。我们的治疗组患者的中位年龄为11.8岁，而安慰剂组为10.5岁。副作用轻微，包括中度体重增加和轻微的行为变化。(C)1994年，John Wiley and Sons，Inc.
A randomized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated with deflazacort 2.0 mg/kg alternate-day therapy or placebo. The deflazacort group showed significant improvement in climbing stairs (P < 0.01), in rising from a chair, Gower's maneuver, and walking (P < 0.0025) after 6 months of treatment. After 1 year, all the above changes remained significantly improved and the MRC index was significantly better (P < 0.05) in the treated group. After 2 years, a significant change was found in the MRC index: higher scores in walking, chair rising (P < 0.02), and grade and time of Gower's maneuver (P < 0.05) were found. The mean time for loss of ambulation for the treated group after we started the trial was 20.5 +/- 11 months; for the placebo group it was 33.2 +/- 9 months (placebo vs. deflazacort group, P < 0.05). Our treated patients lost their ambulation at a median age of 11.8 years vs. 10.5 years in the placebo group. Side effects were mild, consisting of moderate weight gain and slight behavioral changes. (C) 1994 John Wiley and Sons, Inc.