Between 1979 and 1983, 129 children (95 girls) with
precocious puberty were referred to the National Institutes of Health and received treatment for at least 6 months with the long-acting
LHRH analogue D-Trp6-Pro9-NEt-LHRH. The majority (107 of 129) of the children had
central precocious puberty mediated by activation of the hypothalamic-pituitary-gonadal axis in association with
hypothalamic hamartomas (24 of 107) or other central nervous system lesions (21 of 107), or idiopathic
precocious puberty (62 of 107).
Hypothalamic hamartomas or other central nervous system lesions were a frequent cause of
central precocious puberty in girls (27 of 87), but idiopathic
precocious puberty was still the most frequent diagnosis (63%). Idiopathic
precocious puberty was uncommon in boys (6%). The patients with peripheral
precocious puberty included six girls with
McCune-Albright syndrome and six boys with familial male
precocious puberty. These children had peripheral sex
steroid secretion in the absence of hypothalamic-pituitary-gonadal axis maturation. The children with combined peripheral and
central precocious puberty included nine children with
congenital adrenal hyperplasia and one girl with a virilizing adrenal
tumor. In the patients with
central precocious puberty or combined peripheral and
central precocious puberty, LHRHa
therapy caused suppression of
gonadotropin and sex
steroid levels (P less than 0.001), stabilization or regression of secondary sexual characteristics, and decreases in growth rate and in the rate of bone age maturation (P less than 0.005). Patients with peripheral
precocious puberty, however, had no significant change in
gonadotropin or sex
steroid levels, growth rate, or the rate of bone age maturation, and no improvement in secondary sexual characteristics. Thus, LHRHa is an effective treatment of
central precocious puberty and combined peripheral and
central precocious puberty, but is ineffective in the
therapy of peripheral
precocious puberty.