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波兰部分中心小于胎龄(SGA)出生的身材矮小儿童接受重组人生长激素(rhGH)治疗的反应

Response to Treatment with Recombinant Human Growth Hormone (rhGH) of Short Stature Children Born Too Small for Gestational Age (SGA) in Selected Centres in Poland.

作者信息

Glińska Marta, Walczak Mieczysław, Wikiera Beata, Pyrżak Beata, Majcher Anna, Paluchowska Monika, Gawlik Aneta, Antosz Aleksandra, Kusz Marcin, Bossowski Artur, Stożek Karolina, Wędrychowicz Anna, Starzyk Jerzy, Petriczko Elżbieta

机构信息

Department of Pediatrics, Endocrinology, Diabetology, Metabolic Disorders and Cardiology of the Developmental Age, Pomeranian Medical University, 71-252 Szczecin, Poland.

Department of Endocrinology and Diabetology of Children and Adolescents, Wroclaw Medical University, 50-368 Wroclaw, Poland.

出版信息

J Clin Med. 2022 May 30;11(11):3096. doi: 10.3390/jcm11113096.

Abstract

Short stature resulting from SGA is an obligatory indication for treatment with rhGH. The aim of the study was to assess the response to rhGH treatment in patients treated in the years 2016−2020 in six clinical centers in Poland. During the analysis, auxological data were collected, and anthropometrical parameters (Ht, SDS Ht, HV and ΔHV) were reassessed. Subgroups of patients with dysmorphic features (DYSM), fetal alcohol syndrome (FAS) and Silver-Russel syndrome (SRS) were selected. The study group consisted of 235 children (137 boys). The medium initial age was 9.08 years, and 190 patients were in the prepubertal stage. The poor response to treatment was defined as ΔHt SDS < 0.3 and/or ΔHV < 3 cm/year. Seventeen per cent of all patients after the first year and 44% after the second year met the ΔHt SDS < 0.3 criterion, and 56% during the first and 73% during the second year met the ΔHV < 3 cm/year criterion. Our data suggest that patients with SRS may show the best response to treatment, which was sustained throughout the follow-up period. The best response in all subgroups was observed during the first 12 months of therapy. Although the proportion of patients meeting the poor response criteria was high, only a few patients exceeded the 97th percentile for IGF-1 concentration during the first year of treatment. This might suggest that increasing the dose of rhGH in the second treatment year in order to sustain accelerated HV would be safe in these patients.

摘要

小于胎龄儿导致的身材矮小是使用重组人生长激素(rhGH)治疗的必要指征。本研究的目的是评估2016 - 2020年在波兰六个临床中心接受治疗的患者对rhGH治疗的反应。分析过程中,收集了生长学数据,并重新评估了人体测量参数(身高、身高标准差分值、身高增长值和身高增长值变化量)。选取了具有畸形特征的患者亚组(DYSM)、胎儿酒精综合征(FAS)和Silver-Russel综合征(SRS)。研究组由235名儿童组成(137名男孩)。平均初始年龄为9.08岁,190名患者处于青春期前阶段。治疗反应不佳定义为身高标准差分值变化量(ΔHt SDS)< 0.3和/或身高增长值变化量(ΔHV)< 3厘米/年。所有患者中,17%在第一年、44%在第二年达到ΔHt SDS < 0.3标准,56%在第一年、73%在第二年达到ΔHV < 3厘米/年标准。我们的数据表明,SRS患者可能对治疗反应最佳,且在整个随访期内持续存在。在治疗的前12个月观察到所有亚组的最佳反应。尽管达到治疗反应不佳标准的患者比例较高,但在治疗的第一年只有少数患者的胰岛素样生长因子-1(IGF-1)浓度超过第97百分位数。这可能表明,在治疗的第二年增加rhGH剂量以维持身高增长加速对这些患者是安全的。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8747/9181578/d0de8a5e9266/jcm-11-03096-g001.jpg

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