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

Abstract

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 café-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.

• Authors: Caroline de G Buff Passone, Hilton Kuperman, Hamilton Cabral de Menezes-Filho, Lygia Spassapan Oliveira Esteves, Rachel Lana Obata Giroto, Durval Damiani
• Journal: Hormone research in paediatrics (Horm Res Paediatr) Vol. 84 Issue 3 Pg. 184-9 ( 2015) ISSN: 1663-2826 [Electronic] Switzerland
• PMID: 26227563 (Publication Type: Journal Article)
• Copyright: © 2015 S. Karger AG, Basel.

Chemical References

• Estrogen Antagonists
• Gonadotropins
• Tamoxifen
• Estradiol

Topics

• Adolescent
• Body Height (drug effects)
• Bone Development (drug effects)
• Cafe-au-Lait Spots (complications)
• Child
• Child, Preschool
• Estradiol (blood)
• Estrogen Antagonists (adverse effects, therapeutic use)
• Female
• Fibrous Dysplasia, Polyostotic (drug therapy, pathology)
• Follow-Up Studies
• Gonadotropins (blood)
• Humans
• Ovary (pathology)
• Puberty, Precocious (prevention & control)
• Retrospective Studies
• Tamoxifen (adverse effects, therapeutic use)
• Treatment Outcome