Diethylstilbestrol: Observations on Its Use in Duchenne's Muscular Dystrophy (DMD)

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Abstract
Summary Three boys with Duchenne's muscular dystrophy were treated with diethylstilbestrol for 0.5 to 3 years. The administration of this medication resulted in each instance in a considerable reduction in the serum enzymes, lactate dehydrogenase and creatine phosphokinase, which are characteristically elevated in this disease. The reduction was reversible when DIES was discontinued and reproducible when it was restarted. Tests of muscle function indicated that during administration of DIES there was no deterioration in muscle strength. These findings suggest that diethylstilbestrol may have a beneficial effect on Duchenne's muscular dystrophy, but a long-term, controlled trial will be required to establish this. We are indebted to Dr. Irwin Siegel of the University of Illinois and Drs. Robert Cutler, Nicholas Lenn, Nicholas Vick, Jack Stevens, Scott Kleiman, and Mr. Robert Babbs for their respective and many contributions. The serum insulin assays were performed by Dr. Ann Lawrence and Mrs. Lydia Kirsten and Mrs. Gladys Rider helped enormously with the manuscript. Dr. John Morgan made possible the computerized statistical analyses reported here. This work was supported by the Muscular Dystrophy Association of America, the Chicago and Illinois Heart Association, the Fay Hunter Research Fund, and Grants RR 55 and 305 from the General Clinical Research Center's Program of the Division of Research Resources, National Institutes of Health. A special word of thanks is due to the families of these children.

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