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Journal of Clinical Endocrinology & Metabolism Vol. 61, No. 3 536-540
doi:10.1210/jcem-61-3-536
Copyright © 1985 by the Endocrine Society.
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Successful Treatment of Cushing’s Syndrome with the Glucocorticoid Antagonist RU 486*

LYNNETTE K. NIEMAN, GEORGE P. CHROUSOS, CHARLES KELLNER, IRVING M. SPITZ, BRUCE C. NISULA, GORDON B. CUTLER, GEORGE R. MERRIAM, C. WAYNE BARDIN and D. LYNN LORIAUX

Developmental Endocrinology Branch, National Institute of Child Health and Human Developmen, Bethesda, Maryland 20205;
he Biological Psychiatry Branch, National Institute of Mental Health (C.K.), National Institutes of Health, Bethesda, Maryland 20205;
Population Council, New York, New York 00000

Address requests for reprints to: Dr. Nieman, National Institutes of Health, Building 10, Room 10N262, Bethesda, Maryland 20205.

A patient with Cushing’s syndrome due to ectopic ACTH secretion was treated successfully with the new glucocorticoid antagonist RU 486 [17β3-hydroxy-llβ-(4-dimethylamino phenyl)17{alpha}-(l-propynyl)estra-4,9-dien-3-one]. This compound is a 19-nor steroid with substitutions at positions Cll and G17 which antagonizes cortisol action competitively at the receptor level. Oral RU 486 was given in increasing doses of 5, 10,15, and 20 mg/kg- day for a 9-week period. Treatment eficacy was monitored by assessment of clinical status and by measuring several glucocorticoid-sensitive variables, including fasting blood sugar, blood sugar 120 min after oral glucose administration, and plasma concentrations of TSH, corticosteroid-binding globulin, LH, testosterone-estradiol-binding globulin, and total and free testosterone. With therapy, the somatic features of Cushing’s syndrome (buffalo hump, central obesity, and moon facies) ameliorated, mean arterial blood pressure normalized, suicidal depression resolved, and libido returned. All biochemical glucocorticoid- sensitive parameters normalized. No side-effects of drug toxicity were observed. We conclude that RU 486 may provide a safe, well tolerated, and effective medical treatment for hypercortisolism.

* Presented in part at the Seventh International Congress of Endocrinology, Quebec, Canada, July 1984.

Received March 15, 1985.




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