Institute of Pediatrics, Children's Hospital of Fudan University, National Children's Medical Center, and the Shanghai Key Laboratory of Medical Epigenetics, International Co-Laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Institutes of Biomedical Sciences, Fudan University, Shanghai, 200032, China.
National Health Commission (NHC) Key Laboratory of Neonatal Diseases, Fudan University, Shanghai, 201102, China.
BMC Pediatr. 2024 Jan 10;24(1):32. doi: 10.1186/s12887-023-04513-0.
Recombinant human growth hormone (rhGH) therapy is beneficial for children with Prader-Willi syndrome (PWS) in improving short stature and metabolism, but the effect of early rhGH treatment on respiratory and sleep parameters for PWS children under three years old remains elusive. Thus, this study aimed to investigate the impact of rhGH treatment on sleep-related breathing disorders (SRBDs) for toddlers with PWS.
A total of 17 age-matched PWS patients receiving rhGH treatment (rhGH group) and 17 control individuals not receiving rhGH treatment (non-rhGH group) were recruited for this study between October 2018 and January 2023. Data related to polysomnography-polygraphy (PSG) and serum levels of insulin-like growth factor (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3) were collected.
The mean age in the rhGH group was 20.76 ± 9.22 months, which was comparable to that of the non-rhGH group (25.23 ± 13.81 months). The demographic and anthropometric parameters were similar across the two groups after 52 weeks of treatment. Administration of rhGH to toddlers did not exert adverse effects on the obstructive apnea-hypopnea index (OAHI), central apnea index (CAI), oxygen desaturation index (ODI), mean percutaneous oxygen saturation (SpO), lowest SpO, duration when SpO is lower than 90%, or proportion of the patients with SpO lower than 90%. Furthermore, the increased IGF-1 z-score and IGFBP-3 level did not worsen SRBDs.
Treatment with rhGH for 52 weeks on young toddlers with PWS showed no deleterious effects on SRBDs. This shed more light on the importance of initiating rhGH therapy early in PWS patients.
重组人生长激素（rhGH）治疗有益于改善 Prader-Willi 综合征（PWS）患儿的身材矮小和代谢，但对于三岁以下 PWS 儿童 rhGH 治疗对呼吸和睡眠参数的影响仍不清楚。因此，本研究旨在探讨 rhGH 治疗对 PWS 幼儿睡眠相关呼吸障碍（SRBDs）的影响。
本研究于 2018 年 10 月至 2023 年 1 月期间共招募了 17 名接受 rhGH 治疗的年龄匹配的 PWS 患者（rhGH 组）和 17 名未接受 rhGH 治疗的对照个体（非 rhGH 组）。收集了与多导睡眠描记术-多导仪（PSG）和胰岛素样生长因子（IGF-1）和胰岛素样生长因子结合蛋白 3（IGFBP-3）血清水平相关的数据。
rhGH 组的平均年龄为 20.76±9.22 个月，与非 rhGH 组（25.23±13.81 个月）相当。治疗 52 周后，两组的人口统计学和人体测量学参数相似。rhGH 治疗对幼儿的阻塞性呼吸暂停-低通气指数（OAHI）、中枢性呼吸暂停指数（CAI）、氧减饱和度指数（ODI）、平均经皮血氧饱和度（SpO）、最低 SpO、SpO 低于 90%的持续时间或 SpO 低于 90%的患者比例均无不良影响。此外，IGF-1 z 评分和 IGFBP-3 水平的增加并没有加重 SRBDs。
rhGH 治疗 52 周对 PWS 幼儿无明显不良影响。这进一步强调了在 PWS 患者中尽早开始 rhGH 治疗的重要性。
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