Partsch Carl-Joachim, Land Christof, Pfäffle Roland Werner, Schwab Karl Otfried, Sommer Heide
Department of Pediatrics, Endokrinologikum Hamburg, Hamburg, Germany.
Zentrum für Kinder- und Jugendendokrinologie, Gauting, Germany.
Horm Res Paediatr. 2024 Apr 25:1-11. doi: 10.1159/000539068.
Growth hormone (GH) treatment in children with growth hormone deficiency (GHD), short children born small for gestational age (SGA), and Turner syndrome (TS) is well established. However, a variety of parameters are still under discussion to achieve optimal growth results and efficiency of GH use in real-world treatment.
German GH-treatment naïve patients of the PATRO Children database were grouped according to their start of treatment into groups of 3 years from 2007 to 2018. Time trends in age, gender, GH dose, height standard deviation score (SDS), first-year growth response, and Index of Responsiveness (IoR) were investigated in children with GHD, short children born SGA, and TS starting GH treatment in the German patient population of the PATRO Children database from 2007 to 2018 to determine specific parameters for GH treatment optimization.
All patient groups started GH treatment at a relatively high chronological age (2007-2009: GHD 8.33 ± 3.19, SGA 7.32 ± 2.52, TS 8.65 ± 4.39) with a slight but not significant trend towards younger therapy start up to 2016-2018 (GHD 8.04 ± 3.36, SGA 6.67 ± 2.65, TS 7.85 ± 3.38). In the GHD and SGA groups, female patients were underrepresented compared to male patients (GHD 32.3%, SGA 43.6%) with no significant change over the 4 time periods. Patients with GHD started GH treatment at a low dose (0.026 mg/kg/day). In SGA and TS patients, GH therapy was started below the registered dose recommendation (30.0 μg/kg/day and 33.7 μg/kg/day, respectively). In the first year of treatment, the mean GH dose was increased moderately (GHD: 30.7, SGA: 35.7, TS: 40.8 μg/kg/day). There was no significant change of GH dosing over time from 2007 to 2018. The IoR was comparable between time-groups for all 3 diagnoses.
This study shows potential for improvement of GH treatment results in GHD, SGA, and TS patients in terms of early dose adjustment and younger age at the start of treatment. This is in accordance with important parameters used in prediction models.
生长激素（GH）治疗生长激素缺乏症（GHD）儿童、小于胎龄儿（SGA）出生的矮小儿童以及特纳综合征（TS）已得到广泛认可。然而，在实际治疗中，为实现最佳生长效果和生长激素使用效率，仍有多种参数有待探讨。
将PATRO儿童数据库中未接受过生长激素治疗的德国患者，根据其治疗开始时间分为2007年至2018年的3年一组。对2007年至2018年在PATRO儿童数据库的德国患者群体中开始生长激素治疗的GHD儿童、SGA出生的矮小儿童和TS儿童的年龄、性别、生长激素剂量、身高标准差评分（SDS）、第一年生长反应和反应指数（IoR）的时间趋势进行研究，以确定优化生长激素治疗的具体参数。
所有患者组开始生长激素治疗时的实足年龄相对较高（2007 - 2009年：GHD为8.33±3.19，SGA为7.32±2.52，TS为8.65±4.39），到2016 - 2018年有轻微但不显著的趋势表明治疗开始年龄更年轻（GHD为8.04±3.36，SGA为6.67±2.65，TS为7.85±3.38）。在GHD和SGA组中，女性患者的比例低于男性患者（GHD为32.3%，SGA为43.6%），在4个时间段内无显著变化。GHD患者开始生长激素治疗时剂量较低（0.026mg/kg/天）。在SGA和TS患者中，生长激素治疗开始时低于注册剂量推荐（分别为30.0μg/kg/天和33.7μg/kg/天）。在治疗的第一年，平均生长激素剂量适度增加（GHD：30.7，SGA：35.7，TS：40.8μg/kg/天）。从2007年到2018年，生长激素剂量随时间无显著变化。所有3种诊断的时间组之间的IoR相当。
本研究表明，在早期剂量调整和治疗开始时年龄更小方面，GHD、SGA和TS患者的生长激素治疗结果有改善的潜力。这与预测模型中使用的重要参数一致。
