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Six-Year Results of Spironolactone and Testolactone Treatment of Familial Male-Limited Precocious Puberty with Addition of Deslorelin after Central Puberty Onset

Developmental Endocrinology Branch, National Institute of Child Health and Human Development (E.W.L., J.J., K.M.B., G.B.C.), and the Department of Radiology (S.C.H.), National Institutes of Health, Bethesda, Maryland 20892

Address all correspondence and requests for reprints to: Ellen Leschek, M.D., Developmental Endocrinology Branch, National Institute of Child Health and Human Development, National Institutes of Health, Building 10, Room 10N262, 10 Center Drive, MSC 1862, Bethesda, Maryland 20892-1862. E-mail: ellen_leschek{at}nih.gov

Short term treatment with spironolactone, testolactone, and, after the onset of central puberty, deslorelin can normalize the rate of growth and bone maturation in boys with familial male-limited precocious puberty. To test the hypothesis that this treatment can achieve long term normalization of the growth and development of these children, we examined the growth rate, bone maturation rate (change in bone age/change in chronological age), and predicted adult height of 10 boys who were treated with spironolactone (5.7 mg/kg·day) and testolactone (40 mg/kg·day) for at least 6 yr. Deslorelin (4 µg/kg·day) treatment was initiated 2.6 ± 1.3 yr after beginning spironolactone and testolactone treatment.

The growth rate normalized within 1 yr of starting treatment and remained normal during the next 5 yr of treatment (P < 0.001). The rate of bone maturation normalized during the second year of treatment and remained normal thereafter (P < 0.001). Predicted height increased from 160.7 ± 14.7 centimeters at baseline to 173.6 ± 10.1 centimeters after 6 yr of treatment (P < 0.05 during the fourth through the sixth year of treatment compared to baseline).

We conclude that long term treatment with spironolactone, testolactone, and, after central puberty, deslorelin normalizes the growth rate and bone maturation and improves the predicted height in boys with familial male-limited precocious puberty. The ultimate effect of this approach on adult height will require further study.

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