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

Erica A. Eugster, Stephen D. Rubin, Edward O. Reiter, Paul Plourde, Hann Chang Jou, Ora H. Pescovitz

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Abstract

Objective: We undertook a 1-year multicenter trial of tamoxifen treatment for precocious puberty in girls with McCune-Albright syndrome (MAS). Study design: Girls ≤ 10 years with classic or atypical MAS were recruited. Pretreatment history was collected for 6 months. Patients received 20 mg tamoxifen daffy. Diaries were used to record bleeding. Evaluations included physical examination, bone age, pelvic ultrasound, hormone levels, and safety assessments. Results: A total of 2S 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 ± 2.6,5 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.

Original languageEnglish (US)
Pages (from-to)60-66
Number of pages7
JournalJournal of Pediatrics
Volume143
Issue number1
DOIs
StatePublished - Jul 1 2003

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ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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