A beneficial effect of oxandrolone in the treatment of Duchenne muscular dystrophy: A pilot study

被引:31
作者
Fenichel, G [1 ]
Pestronk, A [1 ]
Florence, J [1 ]
Robison, V [1 ]
Hemelt, V [1 ]
机构
[1] WASHINGTON UNIV, SCH MED, DEPT NEUROL, ST LOUIS, MO 63110 USA
关键词
D O I
10.1212/WNL.48.5.1225
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
We treated 10 boys with Duchenne muscular dystrophy with oxandrolone, an anabolic steroid, for 3 months. The mean of the changes in the average muscle score was an improvement of 0.315 +/- 0.097. The expected mean change in muscle score after 3 months from natural history data is a loss of 0.1. The difference of 0.415 between the actual and expected values is significant at p < 0.01. This preliminary evidence suggests that oxandrolone treatment may provide a degree of functional benefit for children with Duchenne muscular dystrophy that is similar to that after prednisone administration.
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页码:1225 / 1226
页数:2
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