背景:重组人生长激素(rhGH)治疗对Prader-Willi综合征(PWS)儿童在改善身材矮小和新陈代谢方面有益,但是早期rhGH治疗对3岁以下PWS儿童的呼吸和睡眠参数的影响仍然难以捉摸。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。
方法:在2018年10月至2023年1月期间,共招募了17名年龄匹配的接受rhGH治疗的PWS患者(rhGH组)和17名未接受rhGH治疗的对照个体(非rhGH组)。收集与多导睡眠图(PSG)相关的数据以及胰岛素样生长因子(IGF-1)和胰岛素样生长因子结合蛋白3(IGFBP-3)的血清水平。
结果: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参数相似。对幼儿施用rhGH不会对阻塞性呼吸暂停低通气指数(OAHI)产生不利影响,中枢呼吸暂停指数(CAI),氧饱和度指数(ODI),平均经皮氧饱和度(SpO2),最低SpO2,当SpO2低于90%时的持续时间,或SpO2低于90%的患者比例。此外,IGF-1z评分和IGFBP-3水平的升高并未使SRBD恶化.
结论:用rhGH治疗52周对患有PWS的幼儿没有显示出对SRBD的有害影响。这进一步阐明了PWS患者早期开始rhGH治疗的重要性。
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 (SpO2), lowest SpO2, duration when SpO2 is lower than 90%, or proportion of the patients with SpO2 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.