普拉德-威利综合征年轻成人的骨密度：一项随机、安慰剂对照、交叉生长激素试验。

Bone mineral density in young adults with Prader-Willi syndrome: A randomized, placebo-controlled, crossover GH trial.

机构信息

Dutch Growth Research Foundation, Rotterdam, The Netherlands.

Department of Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center-Sophia Children's Hospital, Rotterdam, The Netherlands.

出版信息

Clin Endocrinol (Oxf). 2018 Jun;88(6):806-812. doi: 10.1111/cen.13567. Epub 2018 Mar 22.

DOI:10.1111/cen.13567

PMID:29418016

Abstract

CONTEXT

The prevalence of osteoporosis is increased in adults with Prader-Willi syndrome (PWS). In children with PWS, growth hormone (GH) treatment has beneficial effects on bone mineral density (BMD). BMD might deteriorate after cessation of GH at adult height (AH), while continuing GH might maintain BMD.

OBJECTIVE

To investigate the effects of GH vs placebo, and furthermore the effects of sex steroid replacement therapy (SSRT), on BMD in GH-treated young adults with PWS who had attained AH.

DESIGN

Two-year, randomized, double-blind, placebo-controlled, crossover GH study.

PATIENTS

Twenty-seven young adults with PWS were stratified for gender and BMI and then randomly and blindly assigned to receive GH (0.67 mg/m /day) or placebo for 1 year, after which they crossed over to the alternative treatment for another year.

MEASUREMENTS

Bone mineral density of the total body (BMD ) and lumbar spine (BMD ) SDS were measured by dual-energy x-ray absorptiometry.

RESULTS

At AH, BMD SDS was significantly lower compared to healthy peers (P < .01), while BMAD SDS was similar. Both BMD SDS and BMAD SDS were similar during 1 year of GH vs 1 year of placebo. In hypogonadal young adults without SSRT, BMD SDS and BMAD SDS decreased during the 2-year study (P = .11 and P = .01), regardless of GH or placebo, while BMD SDS increased in those with SSRT (P < .01).

CONCLUSIONS

Compared to GH treatment, 1 year of placebo after attainment of AH does not deteriorate BMD SDS in young adults with PWS. In addition, our data suggest that GH is not able to prevent the decline in BMD SDS in hypogonadal young adults with PWS, unless it is combined with SSRT.

摘要

背景

普拉德-威利综合征（PWS）患者骨质疏松症的患病率增加。在 PWS 儿童中，生长激素（GH）治疗对骨密度（BMD）有有益的影响。在达到成人身高（AH）后停止 GH 治疗时，BMD 可能会恶化，而继续使用 GH 可能会维持 BMD。

目的

研究 GH 与安慰剂相比，以及性激素替代疗法（SSRT）对达到 AH 的 GH 治疗的 PWS 年轻成人的 BMD 的影响。

设计

为期两年、随机、双盲、安慰剂对照、交叉 GH 研究。

患者

27 名达到 AH 的 PWS 年轻成人按性别和 BMI 分层，然后随机和盲法分配接受 GH（0.67mg/m²/天）或安慰剂治疗 1 年，然后交叉接受另一种治疗 1 年。

测量

通过双能 X 射线吸收法测量全身（BMD）和腰椎（BMD）的骨矿物质密度（BMD）的 SDS。

结果

在 AH 时，BMD SDS 明显低于健康同龄人（P<0.01），而 BMAD SDS 相似。GH 治疗 1 年与安慰剂治疗 1 年期间，BMD SDS 和 BMAD SDS 相似。在没有 SSRT 的性腺功能减退的年轻成人中，无论 GH 或安慰剂，BMD SDS 和 BMAD SDS 在 2 年研究期间均下降（P=0.11 和 P=0.01），而有 SSRT 的 BMD SDS 增加（P<0.01）。