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神经性厌食症患儿生长障碍应用生长激素治疗 1 年:一项随机安慰剂对照试验。

One Year of GH Treatment for Growth Failure in Children With Anorexia Nervosa: A Randomized Placebo-Controlled Trial.

机构信息

Assistance Publique-Hôpitaux de Paris, Robert Debré University Hospital, Endocrinology-Diabetology Department, Reference Center for Growth and Development Endocrine Diseases, Paris, France.

Université de Paris; NeuroDiderot, Institut National de la Santé et de la Recherche Médicale (INSERM) UMR 1141, Paris, France.

出版信息

J Clin Endocrinol Metab. 2021 Jun 16;106(7):e2535-e2546. doi: 10.1210/clinem/dgab203.

Abstract

CONTEXT

Children with anorexia nervosa (AN) are at risk of adult height deficit due to prolonged low height velocity (HV).

OBJECTIVE

To investigate the effects of human growth hormone (GH) injections on HV in children with AN and severe growth impairment.

DESIGN AND PARTICIPANTS

In this prospective, randomized, double-blind, single-center, proof-of-concept trial, children with AN and low HV (≤2 cm/year) for at least 18 months, and a bone age ≤12 years for girls and ≤14 years for boys, were randomized to receive daily subcutaneous injections of human GH (0.050 mg/kg/day) or placebo for 12 months.

MAIN OUTCOME MEASURES

Change in HV after 12 months.

RESULTS

In total, 8 patients were assigned to the GH group and 6 to the placebo group. Patients had a median (25th-75th percentile) HV of 1.0 (0.5;1.5) cm/year. The effect of GH treatment increased strongly after 6 months, with a height gain after 12 months of 9.65 (8.0;11.6) cm for the GH group vs 3.85 (1.7;7.3) cm for the placebo group, with an absolute median (2.5th-97.5th percentile) difference between the groups of 5.8 (-1.85;9.68) cm after bootstrapping. The percentage of patients with a HV > 5 cm/year during the study period was higher in the GH group than in the placebo group (100% vs 50%, P = 0.05). Adverse events occurred in similar numbers in the 2 groups, were mild or nonfatal, and did not lead to treatment being stopped.

CONCLUSION

GH administration to improve HV is a potentially valid option for increasing HV in children with AN and prolonged severe growth failure.

摘要

背景

由于身高增长速度(HV)长期较低,神经性厌食症(AN)患儿存在成人身高不足的风险。

目的

研究生长激素(GH)注射对 AN 且严重生长障碍儿童 HV 的影响。

设计和参与者

这是一项前瞻性、随机、双盲、单中心、概念验证试验,纳入 HV 持续至少 18 个月且骨龄≤12 岁(女孩)和≤14 岁(男孩)的 AN 且严重生长迟缓儿童,随机分组接受每日皮下注射人 GH(0.050mg/kg/天)或安慰剂治疗 12 个月。

主要结局测量指标

12 个月后 HV 的变化。

结果

共 8 例患儿被分配至 GH 组,6 例患儿被分配至安慰剂组。患儿 HV 中位数(25 分位;75 分位)为 1.0(0.5;1.5)cm/年。GH 治疗的效果在 6 个月后明显增强,GH 组治疗 12 个月后身高增长 9.65(8.0;11.6)cm,而安慰剂组为 3.85(1.7;7.3)cm,经 bootstrap 法校正后两组间绝对中位数(2.5 分位;97.5 分位)差值为 5.8(-1.85;9.68)cm。GH 组在研究期间 HV>5cm/年的患儿比例高于安慰剂组(100%比 50%,P=0.05)。两组不良反应数量相似,均为轻度或非致命性,且未导致治疗停止。

结论

GH 治疗以改善 HV 是增加 AN 且长期严重生长迟缓儿童 HV 的潜在有效方法。

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