Tamoxifen treatment for precocious puberty in McCune-Albright syndrome: a multicenter trial

J Pediatr. 2003 Jul;143(1):60-6. doi: 10.1016/S0022-3476(03)00128-8.


Objective: We undertook a 1-year multicenter trial of tamoxifen treatment for precocious puberty in girls with McCune-Albright syndrome (MAS).

Study design: Girls < or =10 years with classic or atypical MAS were recruited. Pretreatment history was collected for 6 months. Patients received 20 mg tamoxifen daily. Diaries were used to record bleeding. Evaluations included physical examination, bone age, pelvic ultrasound, hormone levels, and safety assessments.

Results: A total of 28 girls (2.9-10.9 years of age) were enrolled from 20 centers, of whom 25 completed 12 months of tamoxifen treatment. Compared with before the study, vaginal bleeding episodes decreased (3.42+/-3.36/year vs 1.17+/-1.41/year), growth velocity slowed (SDS 1.22+/-2.65 vs -0.59+/-3.06, P=.005), and rate of bone maturation decreased (1.21+/-0.78 vs 0.72+/-0.36, P=.02). Ovarian volumes were enlarged and asymmetric throughout the study, and uterine volumes were increased. No adverse events occurred.

Conclusions: Tamoxifen treatment of precocious puberty in MAS results in a reduction of vaginal bleeding and significant improvements in growth velocity and rate of skeletal maturation.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cafe-au-Lait Spots / epidemiology
  • Estradiol / blood
  • Estrogen Antagonists / therapeutic use*
  • Estrone / blood
  • Female
  • Fibrous Dysplasia of Bone / epidemiology
  • Fibrous Dysplasia, Polyostotic / drug therapy*
  • Follicle Stimulating Hormone / blood
  • Humans
  • Hyperthyroidism / epidemiology
  • Insulin-Like Growth Factor I / metabolism
  • Luteinizing Hormone / blood
  • Prospective Studies
  • Puberty, Precocious / drug therapy*
  • Tamoxifen / therapeutic use*
  • Time Factors


  • Estrogen Antagonists
  • Tamoxifen
  • Estrone
  • Estradiol
  • Insulin-Like Growth Factor I
  • Luteinizing Hormone
  • Follicle Stimulating Hormone