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中国女孩中枢性性早熟或早发性快速进展性青春期治疗的长期结局。

Long-Term Outcomes of Treatments for Central Precocious Puberty or Early and Fast Puberty in Chinese Girls.

机构信息

Children's Hospital of Zhejiang University School of Medicine, Hangzhou, China.

Shaoxing Women and Children's Hospital, Shaoxing, China.

出版信息

J Clin Endocrinol Metab. 2020 Mar 1;105(3). doi: 10.1210/clinem/dgz027.

DOI:10.1210/clinem/dgz027
PMID:31702013
Abstract

CONTEXT

Gonadotropin-releasing hormone analogues (GnRHa) and recombinant human growth hormone (rhGH) have been widely used to treat idiopathic central precocious puberty (CPP) or early and fast puberty (EFP). However, large-scale studies to evaluate the treatment effects on final adult height (FAH) are still lacking.

OBJECTIVE

To assess the effects of long-term treatment for CPP/EFP on FAH and its main influencing factors.

DESIGN AND SETTING

Retrospective, multicenter observational study from 1998 to 2017.

PARTICIPANTS

Four hundred forty-eight Chinese girls with CPP/EFP received GnRHa and rhGH treatment (n = 118), GnRHa alone (n = 276), or no treatment (n = 54).

MAIN OUTCOME MEASURES

FAH, target height (Tht), and predictive adult height (PAH).

RESULTS

The height gain (FAH-PAH) was significantly different among the GnRHa and rhGH treatment, GnRHa alone, and no treatment groups (P < 0.05; 9.51 ± 0.53, 8.07 ± 0.37, and 6.44 ± 0.91 cm, respectively). The genetic height gain (FAH-Tht) was 4.0 ± 0.5 cm for the GnRHa + rhGH group and 2.0 ± 0.27 cm for the GnRHa group, while the control group reached their Tht. In addition, 5 critical parameters derived from PAH, bone age, and Tht, showed excellent performance in predicting which patients could gain ≥5 cm (FAH-PAH), and this was further validated using an independent study.

CONCLUSIONS

The overall beneficial effect of GnRHa + rhGH or GnRHa on FAH was significant. The control group also reached their genetic target height. Clinicians are recommended to consider both the potential gains in height and the cost of medication.

摘要

背景

促性腺激素释放激素类似物(GnRHa)和重组人生长激素(rhGH)已广泛用于治疗特发性中枢性性早熟(CPP)或早发性和快速性青春期(EFP)。然而,评估其对最终成年身高(FAH)治疗效果的大规模研究仍缺乏。

目的

评估 CPP/EFP 长期治疗对 FAH 的影响及其主要影响因素。

设计和设置

1998 年至 2017 年的回顾性、多中心观察性研究。

参与者

448 例中国 CPP/EFP 女孩接受 GnRHa 和 rhGH 治疗(n=118)、GnRHa 单药治疗(n=276)或未治疗(n=54)。

主要观察指标

FAH、靶身高(Tht)和预测成年身高(PAH)。

结果

GnRHa 和 rhGH 治疗组、GnRHa 单药治疗组和未治疗组的身高增长(FAH-PAH)差异有统计学意义(P<0.05;分别为 9.51±0.53、8.07±0.37 和 6.44±0.91cm)。GnRHa+rhGH 组遗传身高增长(FAH-Tht)为 4.0±0.5cm,GnRHa 组为 2.0±0.27cm,而对照组达到了 Tht。此外,从 PAH、骨龄和 Tht 得出的 5 个关键参数在预测哪些患者可获得≥5cm(FAH-PAH)方面表现出优异的性能,这在使用独立研究进一步验证后得到证实。

结论

GnRHa+rhGH 或 GnRHa 对 FAH 的整体有益效果显著。对照组也达到了他们的遗传靶身高。建议临床医生既要考虑身高增长的潜力,也要考虑药物的费用。

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