Division of Pediatric Endocrinology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH 45229-3039, USA.
Department of Endocrinology, Alder Hey Children's NHS Foundation Trust, Liverpool L14 5AB, UK.
J Clin Endocrinol Metab. 2023 Sep 18;108(10):2653-2665. doi: 10.1210/clinem/dgad159.
Despite having normal growth hormone (GH) secretion, individuals with Turner syndrome (TS) have short stature. Treatment with recombinant human GH is recommended for TS girls with short stature.
This work aimed to evaluate the effectiveness and safety of Norditropin (somatropin, Novo Nordisk) with up to 10 years of follow-up in children with TS.
Secondary analysis was conducted of Norditropin data from 2 non-interventional studies: NordiNet® IOS (NCT00960128) and the ANSWER program (NCT01009905).
A total of 2377 girls with TS were included in the safety analysis set (SAS), with 1513 in the treatment-naive effectiveness analysis set (EAS). At the start of treatment, 1273 (84%) participants were prepubertal (EAS); mean (SD) age was 8.8 (3.9) years. Mean (SD) dose received at the start of GH treatment was 0.045 (0.011) mg/kg/day (EAS). Mean (SD) baseline insulin-like growth factor-1 (IGF-I) SD score (SDS) was -0.86 (1.52), and mean (SD) duration of GH treatment (SAS) was 3.8 (2.8) years.Height SDS (HSDS) increased throughout follow-up, with near-adult HSDS reached by 264 (17%) participants (mean [SD] -1.99 [0.94]; change from baseline +0.90 [0.85]). During the study, 695 (46%) participants (EAS) entered puberty at a mean (SD) age of 12.7 (1.9) years (whether puberty was spontaneous or induced was unknown). Within the SAS, mean IGF-I SDS (SD) at year 10 was 0.91 (1.69); change from baseline +1.48 (1.70). Serious adverse reactions were reported in 10 participants (epiphysiolysis [n = 3]).
GH-treated participants with TS responded well, without new safety concerns. Our real-world data are in agreement with previous studies.
尽管特纳综合征（TS）患者的生长激素（GH）分泌正常，但他们仍存在身材矮小的问题。对于身材矮小的 TS 女孩，推荐使用重组人生长激素进行治疗。
本项工作旨在评估使用诺泽（生长激素，诺和诺德）治疗 TS 儿童长达 10 年的有效性和安全性。
对两项非干预性研究（NordiNet® IOS，NCT00960128；ANSWER 项目，NCT01009905）的诺泽数据进行二次分析。
安全性分析集（SAS）共纳入 2377 例 TS 女孩，治疗初效分析集（EAS）纳入 1513 例女孩。治疗开始时，1273 例（84%）参与者处于青春前期（EAS）；平均（SD）年龄为 8.8（3.9）岁。开始 GH 治疗时，平均（SD）剂量为 0.045（0.011）mg/kg/天（EAS）。平均（SD）基线胰岛素样生长因子-1（IGF-1）标准差评分（SDS）为-0.86（1.52），平均（SD）GH 治疗持续时间（SAS）为 3.8（2.8）年。身高 SDS（HSDS）在随访期间持续增加，264 例（17%）参与者达到接近成人的 HSDS（平均[SD]-1.99[0.94]；与基线相比增加 0.90[0.85]）。研究期间，695 例（46%）参与者（EAS）在平均（SD）年龄为 12.7（1.9）岁时进入青春期（青春期是自发的还是诱导的尚不清楚）。在 SAS 中，第 10 年 IGF-1 SDS（SD）的平均值（SD）为 0.91（1.69）；与基线相比增加 1.48（1.70）。10 例参与者报告了严重不良反应（骺板分离[n=3]）。
接受 GH 治疗的 TS 患者反应良好，无新的安全性问题。我们的真实世界数据与之前的研究一致。
