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儿童部分生长激素缺乏症的生长激素治疗。我们是否治疗了正确的患者?

Growth hormone therapy in children with partial growth hormone deficiency. Are we treating the right patients?

机构信息

Division of Pediatrics and Neurology of Developmental Age, The Independent Public Clinical Hospital No. 6 of the Medical University of Silesia in Katowice, John Paul II Upper Silesian Child Health Centre, Poland.

Department of Paediatrics and Paediatric Endocrinology, School of Medicine in Katowice, Medical University of Silesia, Poland.

出版信息

Pediatr Endocrinol Diabetes Metab. 2020;26(2):65-72. doi: 10.5114/pedm.2020.95624.

DOI:10.5114/pedm.2020.95624
PMID:32564582
Abstract

INTRODUCTION

Diagnosis of growth hormone deficiency (GHD) in children with short stature, whose height is below -2SD for the population norm, is based on the assessment of growth hormone (GH) peaks in stimulation tests. However, cut-off values for GH secretion are arbitrary and vary in different centres. Indications for recombinant GH therapy remain disputable in children with GH concentrations between 5 and 10 ng/ml (pGHD).

AIM OF THE STUDY

The aim of our study was to assess the effects of rhGH therapy in children with transient pGHD deficiency compared to untreated children with idiopathic short stature (ISS).

MATERIAL AND METHODS

The study group comprised 54 patients at the mean age of 13.5 (SD 2.36) years, who were diagnosed as pGHD and treated with rhGH. The control group comprised 32 subjects with ISS matched for sex and age, untreated with rhGH.

RESULTS

Mean final height was within the normal range for population norms in both groups. The average height gain was statistically significant at -1.3 SD (p < 0.001) for the study group and -1.02 SD (p ≤ 0.001) for the control group. However after exclusion of children with familial short stature (FSS) the height gains were, respectively, 1.41 SD ±0.67 for the study group and 1.22 SD ±0.77 for the control group, without statistical significance.

CONCLUSIONS

The results of our study did not show beneficial effects of rhGH treatment in children with pGHD as compared to untreated ISS subjects. Therefore, it is necessary to determine criteria other than arbitrarily established GH concentration for starting rhGH treatment in children with pGHD.

摘要

简介

对于身高低于人群正常值-2SD 的矮小儿童,生长激素缺乏症(GHD)的诊断基于生长激素(GH)刺激试验中 GH 峰值的评估。然而,GH 分泌的截断值是任意的,并且在不同中心有所不同。在 GH 浓度在 5 至 10ng/ml 之间的儿童中,重组 GH 治疗的适应症仍然存在争议(pGHD)。

目的

我们的研究旨在评估与未经治疗的特发性矮小症(ISS)儿童相比,rhGH 治疗对暂时性 pGHD 缺乏儿童的影响。

材料和方法

研究组包括 54 名平均年龄为 13.5(SD 2.36)岁的患者,他们被诊断为 pGHD 并接受 rhGH 治疗。对照组包括 32 名性别和年龄匹配的 ISS 患者,未接受 rhGH 治疗。

结果

两组的最终身高均在人群正常值范围内。研究组的平均身高增长在统计学上有显著差异(p<0.001),为-1.3SD,对照组为-1.02SD(p≤0.001)。然而,在排除家族性矮小症(FSS)儿童后,研究组的身高增长分别为 1.41SD±0.67,对照组为 1.22SD±0.77,无统计学意义。

结论

我们的研究结果并未显示 rhGH 治疗对 pGHD 儿童与未经治疗的 ISS 患者相比有有益的影响。因此,有必要确定其他标准,而不仅仅是任意设定的 GH 浓度,以开始对 pGHD 儿童进行 rhGH 治疗。

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