他莫昔芬改善McCune-Albright综合征女孩的最终身高预测：长期随访

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

作者信息

de G Buff Passone Caroline, Kuperman Hilton, Cabral de Menezes-Filho Hamilton, Spassapan Oliveira Esteves Lygia, Lana Obata Giroto Rachel, Damiani Durval

机构信息

Pediatric Endocrinology Unit, Instituto da Crianx00E7;a do Hospital das Clx00ED;nicas da Faculdade de Medicina da Universidade de Sx00E3;o Paulo, Sx00E3;o Paulo, Brazil.

出版信息

Horm Res Paediatr. 2015;84(3):184-9. doi: 10.1159/000435881. Epub 2015 Jul 23.

DOI:10.1159/000435881

PMID:26227563

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.

摘要

背景

McCune - Albright综合征（MAS）是一种遗传性疾病，其特征为纤维性发育异常、皮肤色素沉着以及各种内分泌器官的自主性功能亢进。MAS在女性中常表现为性早熟（PP）。尽管已经提出了许多治疗方法，但这些患者最终身高（FH）的保留仍然是一个挑战。

目的

评估他莫昔芬在改善MAS患者最终身高预测（FHP）方面的疗效。

方法

我们回顾性分析了8例患有MAS且出现咖啡斑和促性腺激素非依赖性PP的女性患者。患者平均随访8.3年（范围：3 - 16年）。

结果

所有患者均接受他莫昔芬治疗（10 - 20毫克/天）3 - 8年（平均±标准差：5.75±2.05），这导致阴道出血停止且骨龄成熟稳定。治疗开始时的FHP与治疗结束时（145.1±8.6厘米；Z评分 - 2.84±1.44厘米）以及最后一次评估时（157.0±9.2厘米；Z评分 - 0.85±0.54厘米；p < 0.001）存在显著差异。