生长激素治疗后的 Prader-Willi 综合征患者在 3 年生长激素治疗期间的骨密度。

Bone mineral density during 3 years of growth hormone in previously GH-treated young adults with PWS.

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

Eur J Endocrinol. 2021 May 4;184(6):773-782. doi: 10.1530/EJE-20-1335.

DOI:10.1530/EJE-20-1335

PMID:33769952

Abstract

OBJECTIVE

In children with Prader-Willi syndrome (PWS), growth hormone (GH) treatment has positive effects on bone mineral density (BMD). Two 1-year studies did not show a difference between GH or placebo on BMD in young adults with PWS. However, there are no studies investigating BMD during longer-term GH treatment in young adults with PWS.

DESIGN

Open-label, a prospective study in 43 young adults with PWS.

METHODS

BMD of the total body (BMDTBSDS) and lumbar spine (BMADLSSDS) measured by DXA.

RESULTS

In the total group, estimated mean (95% CI) of BMDTB remained similar during 3 years of GH, being -0.76 (-1.11 to -0.41) SDS at start and -0.90 (-1.27 to -0.54) SDS after 3 years (P = 0.11), as did BMADLS, being -0.36 (-0.72 to 0.01) SDS and -0.46 (-0.77 to -0.16) SDS, respectively (P = 0.16). In men, there was a significant decrease in BMDTBSDS during 3 years of GH, while BMADLSSDS remained similar. In women, both BMDTBSDS and BMADLSSDS remained similar. BMDTBSDS was associated with female sex, lean body mass and age. The majority of patients received sex steroid replacement therapy (SSRT).

CONCLUSIONS

During 3 years of combined GH and SSRT treatment, BMD remained stable in the normal range in young adults with PWS. However, men showed a decline in BMDTBSDS, probably due to insufficient SSRT. We recommended to continue GH treatment in young adults with PWS and to start SSRT during adolescence unless puberty progresses normally.

摘要

目的

在患有普拉德-威利综合征（PWS）的儿童中，生长激素（GH）治疗对骨密度（BMD）有积极影响。两项为期 1 年的研究并未显示 GH 或安慰剂对 PWS 年轻成人 BMD 的差异。然而，目前尚无研究调查 PWS 年轻成人接受长期 GH 治疗期间的 BMD。

设计

43 名 PWS 年轻成人的开放标签、前瞻性研究。

方法

通过 DXA 测量全身骨密度（BMDTBSDS）和腰椎骨密度（BMADLSSDS）。

结果

在整个组中，GH 治疗 3 年后，估计平均（95%CI）BMDTB 保持相似，开始时为-0.76（-1.11 至-0.41）SDS，3 年后为-0.90（-1.27 至-0.54）SDS（P=0.11），BMADLS 也是如此，分别为-0.36（-0.72 至 0.01）SDS 和-0.46（-0.77 至-0.16）SDS（P=0.16）。在男性中，3 年内 GH 治疗后 BMDTBSDS 显著下降，而 BMADLSSDS 保持相似。在女性中，BMDTBSDS 和 BMADLSSDS 均保持相似。BMDTBSDS 与女性性别、瘦体重和年龄相关。大多数患者接受了性激素替代疗法（SSRT）。