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


METHODS
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.
Eugster
Erica A
EA
James Whitcomb Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, Indiana 46202, USA. eeugster@iupui.edu
Rubin
Stephen D
SD
Reiter
Edward O
EO
Plourde
Paul
P
Jou
Hann-Chang
HC
Pescovitz
Ora H
OH
D019080:Cafe-au-Lait Spots / Q000453:epidemiology; D004958:Estradiol / Q000097:blood; D004965:Estrogen Antagonists / Q000627:therapeutic use*; D004970:Estrone / Q000097:blood; D005260:Female; D005357:Fibrous Dysplasia of Bone / Q000453:epidemiology; D005359:Fibrous Dysplasia, Polyostotic / Q000188:drug therapy*; D005640:Follicle Stimulating Hormone / Q000097:blood; D006801:Humans; D006980:Hyperthyroidism / Q000453:epidemiology; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D007986:Luteinizing Hormone / Q000097:blood; D011446:Prospective Studies; D011629:Puberty, Precocious / Q000188:drug therapy*; D013629:Tamoxifen / Q000627:therapeutic use*; D013997:Time Factors
D004965:Estrogen Antagonists; D013629:Tamoxifen; D004970:Estrone; D004958:Estradiol; D007334:Insulin-Like Growth Factor I; D007986:Luteinizing Hormone; D005640:Follicle Stimulating Hormone