Tamoxifen Improves Final Height Prediction in Girls with McCune-Albright Syndrome: A Long Follow-Up

Background: McCune-Albright syndrome (MAS) is a genetic disorder characterized by the triad of fibrous dysplasia, skin hyperpigmentation, and autonomous hyperfunction of various endocrine organs. MAS frequently presents in females as precocious puberty (PP). Although many treatments have been proposed, the preservation of final height (FH) in these patients remains a challenge. Objectives: To evaluate the efficacy of tamoxifen in improving the FH prediction (FHP) in patients with MAS. Method: We retrospectively analyzed 8 female patients with MAS who presented with cafe-au-lait spots and gonadotropin-independent PP. The patients were followed for a mean period of 8.3 years (range: 3-16). Results: All patients were treated with tamoxifen (10-20 mg/day) for 3-8 years (mean +/- SD: 5.75 +/- 2.05), which resulted in the cessation of vaginal bleeding and the stabilization of bone age maturation. There was a significant difference between the FHP at the beginning of treatment and at the end of treatment (145.1 +/- 8.6 cm; Z score -2.84 +/- 1.44 cm) and at the last evaluation (157.0 +/- 9.2 cm; Z score -0.85 +/- 0.54 cm; p < 0.001). Conclusion: Our results support a role for tamoxifen in improving the FHP in patients with MAS. (C) 2015 S. Karger AG, Basel