Treatment of progressive
precocious puberty in patients with
McCune-Albright syndrome (MAS) has traditionally been with
aromatase inhibitors, such as
testolactone. However, the use of these agents has been characterized by problems with both efficacy and compliance. We report a case of MAS in which
tamoxifen proved to be a successful alternative in the treatment of progressive
precocious puberty. An African-American female presented with MAS at 2-5/12 years. Frequent menses, skeletal maturation and growth acceleration prompted initiation of
therapy with
testolactone at 22 mg/kg/d. Over the next 13 months, the patient's puberty advanced unchecked, despite progressive increases in the dose of
testolactone. At age 4 years, medication was discontinued due to treatment failure. At 4-6/12 years, bone age was 10 years, predicted adult height was 137 cm, and monthly
bleeding continued.
Tamoxifen was then begun on an experimental basis. In response, the patient experienced immediate cessation of menses, and had an abrupt decrease in the rates of pubertal progression and linear growth. This patient has now been maintained on
tamoxifen for over three years with no apparent adverse effects.
GnRH analogue
therapy was begun when the onset of
central precocious puberty was noted. Predicted adult height has improved to 154 cm and growth velocity and skeletal maturation remain stable. Our results suggest that
tamoxifen may have a valuable role in the treatment of
precocious puberty in patients with MAS and may lead to superior results compared with those achieved with
aromatase inhibitors.