Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany.
J Clin Endocrinol Metab. 2011 Dec;96(12):E2097-105. doi: 10.1210/jc.2011-0399. Epub 2011 Oct 12.
Children with X-linked hypophosphatemic rickets (XLH) are prone to progressive disproportionate stunting despite oral phosphate and vitamin D treatment.
Our objective was to analyze the effects of GH treatment on stature and lengths of linear body segments in short children with XLH.
DESIGN, SETTINGS, AND PATIENTS: A 3-yr randomized controlled open-label GH study in short prepubertal children with XLH (n = 16) on phosphate and calcitriol treatment was conducted. A cohort of XLH patients (n = 76) on conservative treatment served as an XLH reference population.
Changes in SD scores (SDS) of stature and linear body segments, i.e. sitting height, leg and arm length, and sitting height index (i.e. ratio between sitting height and stature) were the main outcome measures.
XLH patients presented at time of enrollment with significant impairments of stature (-3.3 SDS) and linear body segments compared with healthy children. Leg length (-3.8 SDS) was most impaired, whereas sitting height (-1.7 SDS) was best preserved. The markedly elevated mean sitting height index (+3.3 SDS) reflected severe body disproportion. GH resulted in a sustained increase in linear growth (stature, +1.1 SDS; sitting height, +1.3 SDS; leg length, +0.8 SDS; arm length, +1.1 SDS; each P < 0.05 vs. baseline), whereas no significant changes were observed in controls. Mean height SDS at 3 yr did not significantly differ between groups. Sitting height index remained stable in both the GH-treated patients and in study controls but increased further in the XLH-reference population.
The 3-yr GH treatment improved linear growth without progression of body disproportion in short children with XLH.
尽管进行了口服磷酸盐和维生素 D 治疗，患有 X 连锁低磷血症性佝偻病（XLH）的儿童仍容易出现进行性不成比例的生长迟缓。
我们的目的是分析 GH 治疗对 XLH 矮小儿童身高和线性身体部位长度的影响。
设计、地点和患者：对接受磷酸盐和骨化三醇治疗的 XLH 矮小儿童进行了为期 3 年的随机对照、开放标签 GH 研究（n=16）。作为 XLH 参考人群的一组 XLH 患者（n=76）接受保守治疗。
身高和线性身体部位（即坐高、腿长和臂长）的 SD 评分（SDS）变化是主要观察指标。
与健康儿童相比，XLH 患者在入组时身高（-3.3 SDS）和线性身体部位明显受损。腿长（-3.8 SDS）受损最严重，而坐高（-1.7 SDS）保存最好。显著升高的平均坐高指数（+3.3 SDS）反映了严重的身体不成比例。GH 导致线性生长持续增加（身高，+1.1 SDS；坐高，+1.3 SDS；腿长，+0.8 SDS；臂长，+1.1 SDS；每项 P<0.05 与基线相比），而对照组无明显变化。3 年时的平均身高 SDS 两组间无显著差异。GH 治疗组和研究对照组的坐高指数保持稳定，但 XLH 参考人群进一步增加。
3 年 GH 治疗可改善 XLH 矮小儿童的线性生长，而不会导致身体比例进一步失调。
