Bone Mineral Density in Children and Adolescents With Prader-Willi Syndrome: A Longitudinal Study During Puberty and 9 Years of Growth Hormone Treatment
Bone Mineral Density in Children and Adolescents With Prader-Willi Syndrome: A Longitudinal Study During Puberty and 9 Years of Growth Hormone Treatment
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DOI：
10.1210/jc.2014-4347
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发表时间：
2015-04-01
影响因子：
5.8
通讯作者：
Hokken-Koelega, A. C. S.
中科院分区：
文献类型：
--
作者：
Bakker, N. E.;Kuppens, R. J.;Hokken-Koelega, A. C. S.
目的：本研究旨在探讨长期GH治疗和青春期对儿童和青少年全身(BMDTB)、腰椎(BMDLS)、腰椎(BMDLS)骨密度的影响。设计和地点：这是一项前瞻性的纵向研究。参与者：77名在生长激素治疗期间仍处于青春期前的PWS儿童和接受GH治疗9年的PWS儿童参加研究。干预：这些儿童接受GH治疗，1 mg/m(2)/d(相当于0.035 mg/kg/d)。主要观察指标：BMDTB，BMDLS，BMDLS，结果：青春期前儿童GH治疗4年后，BMDTB标准差分(SDS)和BMDLSSD显著增加，而BMAD(LS)的SDS值基本不变。在青春期，BMDTBSDS和BMAD(LS)SDS显著下降，女孩从11岁开始下降，男孩从14岁和16岁开始下降，但所有骨密度参数都保持在正常范围内。Tanner分期越高，BMDTBSD越低(P=0.083)，BMAD(LS)SD越低(P=0.016)。经过9年的生长激素治疗后，瘦体重抑郁自评量表是预测青春期PWS儿童BMDTB、BMDLS和BMDLSSD的最有效的指标。结论：长期的GH研究显示，在PWS的青春期前儿童，BMDTB、BMDLS和BMAD(LS)保持稳定，但在青春期下降，与青春期发育不完全平行。根据我们的发现，临床医生应该从11岁的女孩和14岁的男孩开始性激素治疗，除非有正常的青春期进展。
Context: Longitudinal data on bone mineral density(BMD) in children and adolescents with Prader-Willi Syndrome (PWS) during long-term GH treatment are not available.Objective: This study aimed to determine effects of long-term GH treatment and puberty on BMD of total body (BMDTB), lumbar spine (BMDLS), and bone mineral apparent density of the lumbar spine (BMAD(LS)) in children with PWS.Design and Setting: This was a prospective longitudinal study of a Dutch PWS cohort.Participants: Seventy-seven children with PWS who remained prepubertal during GH treatment for 4 years and 64 children with PWS who received GH treatment for 9 years participated in the study.Intervention: The children received GH treatment, 1 mg/m(2)/day (congruent to 0.035 mg/kg/d).Main Outcome Measures: BMDTB, BMDLS, and BMAD(LS) was measured by using the same dual-energy x-ray absorptiometry machine for all annual measurements.Results: In the prepubertal group, BMDTB standard deviation score (SDS) and BMDLSSDS significantly increased during 4 years of GH treatment whereas BMAD(LS)SDS remained stable. During adolescence, BMDTBSDS and BMAD(LS)SDS decreased significantly, in girls from the age of 11 years and in boys from the ages of 14 and 16 years, respectively, but all BMD parameters remained within the normal range. Higher Tanner stages tended to be associated with lower BMDTBSDS (P = .083) and a significantly lowerBMAD(LS)SDS (P = .016). After 9 years of GH treatment, lean body mass SDS was the most powerful predictor of BMDTBSDS and BMDLSSDS in adolescents with PWS.Conclusions: This long-term GH study demonstrates that BMDTB, BMDLS, and BMAD(LS) remain stable in prepubertal children with PWS but decreases during adolescence, parallel to incomplete pubertal development. Based on our findings, clinicians should start sex hormone therapy from the age of 11 years in girls and 14 years in boys unless there is a normal progression of puberty.