Clinical Predictors of Good/Poor Response to Growth Hormone Treatment in Children with Idiopathic Short Stature.

IF 2.6 3区医学 Q3 ENDOCRINOLOGY & METABOLISM Hormone Research in Paediatrics Pub Date : 2024-11-21 DOI:10.1159/000542579

Andrew Dauber, Moshe Phillip, Jean-Marc Ferran, Nicky Kelepouris, Navid Nedjatian, Anne Helene Olsen, Alexander Augusto de Lima Jorge

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Abstract

Introduction: Children with idiopathic short stature (ISS) are known to have varying responses to growth hormone (GH) treatment (GHT).

Methods: We conducted a post hoc analysis to identify clinical characteristics predictive of good and poor response during year 1 of GHT. Data from the NordiNet® IOS (NCT00960128) and the ANSWER Program (NCT01009905) were used. Patients were grouped according to their response to GHT; good, middle, and poor responders had a change in HSDS of >1.0, 0.4-1.0, and <0.4, respectively. Patients were also grouped according to their responsiveness to GH dose. Logistic regression modelling was performed to identify clinical characteristics predictive of response to GHT.

Results: The response analysis set included 207 patients. Patients were 3-11 years old (males) or 3-10 years old (females) at treatment start and were prepubertal throughout year 1 of treatment. Age at treatment start (odds ratio [OR] 0.69, 95% confidence interval [CI] 0.5;0.9, p = 0.0169) and distance from target HSDS (OR 2.05, 95% CI 1.1;3.9, p = 0.0259) were found to be significant independent predictors of being in the good- versus poor-response groups. When patients were grouped according to their responsiveness to GH dose, a positive correlation between GH dose and change in HSDS was observed.

Conclusion: We identified younger age and further distance from target HSDS as clinical predictors of response to GHT in a heterogenous population of children with ISS. Future studies, designed to identify the genetic determinants of response to GHT could further facilitate individualisation of GHT. .

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特发性矮身材儿童对生长激素治疗良好/不良反应的临床预测因素。

简介：特发性矮身材（ISS）儿童对生长激素治疗（GHT）的反应各不相同：特发性矮身材（ISS）患儿对生长激素（GHT）治疗的反应各不相同：我们进行了一项事后分析，以确定可预测生长激素治疗第一年期间良好和不良反应的临床特征。我们使用了来自 NordiNet® IOS (NCT00960128) 和 ANSWER Program (NCT01009905) 的数据。根据患者对 GHT 的反应进行分组；良好、中等和差反应者的 HSDS 变化分别大于 1.0、0.4-1.0 和结果：反应分析组包括 207 名患者。患者开始治疗时的年龄为 3-11 岁（男性）或 3-10 岁（女性），在治疗的第 1 年一直处于青春期前。治疗开始时的年龄（比值比 [OR] 0.69，95% 置信区间 [CI]0.5;0.9，p = 0.0169）和与目标 HSDS 的距离（比值比 2.05，95% 置信区间 [CI]1.1;3.9，p = 0.0259）是预测反应良好组和反应不佳组的重要独立因素。根据患者对 GH 剂量的反应程度进行分组后，发现 GH 剂量与 HSDS 的变化呈正相关：结论：我们发现，年龄较小和距离目标 HSDS 较远是 ISS 儿童中对 GHT 反应的临床预测因素。未来的研究旨在确定对 GHT 反应的遗传决定因素，这将进一步促进 GHT 的个体化。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Hormone Research in Paediatrics ENDOCRINOLOGY & METABOLISM-PEDIATRICS

CiteScore

4.90

自引率

6.20%

发文量

审稿时长

4-8 weeks

期刊介绍： The mission of ''Hormone Research in Paediatrics'' is to improve the care of children with endocrine disorders by promoting basic and clinical knowledge. The journal facilitates the dissemination of information through original papers, mini reviews, clinical guidelines and papers on novel insights from clinical practice. Periodic editorials from outstanding paediatric endocrinologists address the main published novelties by critically reviewing the major strengths and weaknesses of the studies.