Institute of Endocrinology and Diabetes, The Children's Hospital at Westmead, Westmead, NSW, Australia.
Pediatric Endocrinology, Lady Ridge Way Hospital, Colombo, Sri Lanka.
J Pediatr Endocrinol Metab. 2024 Aug 1;37(8):680-685. doi: 10.1515/jpem-2024-0059. Print 2024 Aug 27.
Clinical benefits of growth hormone (GH) in Prader-Willi syndrome (PWS) are proven and scoliosis is a known association of both PWS and GH therapy. The aims of this study were to assess GH prescribing practices and growth outcomes over time, the prevalence and predictors of scoliosis in GH-treated PWS children, and the near-final height of GH-treated PWS patients.
This is a retrospective, descriptive study evaluating data from all clinic visits of patients aged 0-18 years with PWS, seen through the Children's Hospital at Westmead between March 1992 and May 2022 (n=75).
A total of 64 patients were treated with GH (visits = 1,414). In the recent decade, the diagnosis of PWS and GH commencement were made significantly earlier in life. The prevalence of scoliosis was 41 %, in which age was the only significant predictor for scoliosis (odds ratio 1.19: 95 % CI [1.08-1.31; p=0.001]) adjusted for other predictors. In patients with data available at the age 16 years (23/28 treated with GH), those who were GH treated had significantly higher height SDS vs. nontreated group (SDS -0.67 vs. -2.58; p=0.0001) and lower BMI SDS (1.18 vs. 2.37; p<0.001).
Significant improvements in growth and body composition were seen in the GH-treated group vs. non-treated group of children with PWS. There were no significant modifiable clinical predictors of scoliosis in children with PWS, but our findings confirm the high prevalence of scoliosis in GH-treated children with PWS reinforcing the need for close surveillance.
生长激素（GH）治疗普拉德-威利综合征（PWS）的临床益处已得到证实，而脊柱侧弯是 PWS 和 GH 治疗的已知并发症。本研究的目的是评估随着时间的推移 GH 处方实践和生长结果，GH 治疗的 PWS 儿童脊柱侧弯的患病率和预测因素，以及 GH 治疗的 PWS 患者的最终身高。
这是一项回顾性描述性研究，评估了 1992 年 3 月至 2022 年 5 月期间在西悉尼儿童医院就诊的所有 0-18 岁 PWS 患者的所有就诊数据（n=75）。
共有 64 名患者接受了 GH 治疗（就诊=1414 次）。在最近十年中，PWS 的诊断和 GH 的开始治疗在生命早期进行得更早。脊柱侧弯的患病率为 41%，其中年龄是唯一显著的脊柱侧弯预测因素（优势比 1.19：95%CI [1.08-1.31;p=0.001]），调整了其他预测因素。在 16 岁时可获得数据的患者中（23/28 例接受 GH 治疗），与未接受 GH 治疗的患者相比，接受 GH 治疗的患者身高 SDS 显著更高（SDS-0.67 对-2.58;p=0.0001），而 BMI SDS 较低（1.18 对 2.37;p<0.001）。
与未接受 GH 治疗的 PWS 儿童相比，GH 治疗组的生长和身体成分有显著改善。在 PWS 儿童中，脊柱侧弯没有明显的可改变的临床预测因素，但我们的研究结果证实了 GH 治疗的 PWS 儿童中脊柱侧弯的高患病率，这强调了密切监测的必要性。
