Pediatric Unit, Ospedale Del Ponte, Insubria University, 21100 Varese, Italy.
J Clin Endocrinol Metab. 2013 Sep;98(9):E1516-23. doi: 10.1210/jc.2013-1831. Epub 2013 Jul 26.
Adenotonsillar tissue hypertrophy and obstructive sleep apnea have been reported during short-term GH treatment in children with Prader-Willi syndrome (PWS).
We conducted an observational study to evaluate the effects of long-term GH therapy on sleep-disordered breathing and adenotonsillar hypertrophy in children with PWS.
This was a longitudinal observational study.
We evaluated 75 children with genetically confirmed PWS, of whom 50 fulfilled the criteria and were admitted to our study. The patients were evaluated before treatment (t0), after 6 weeks (t1), after 6 months (t2), after 12 months (t3), and yearly (t4-t6) thereafter, for up to 4 years of GH therapy. The central apnea index, obstructive apnea hypopnea index (OAHI), respiratory disturbance index, and minimal blood oxygen saturation were evaluated overnight using polysomnography. We evaluated the adenotonsillar size using a flexible fiberoptic endoscope.
The percentage of patients with an OAHI of >1 increased from 3 to 22, 36, and 38 at t1, t4, and t6, respectively (χ(2) = 12.2; P < .05). We observed a decrease in the respiratory disturbance index from 1.4 (t0) to 0.8 (t3) (P < .05) and the central apnea index from 1.2 (t0) to 0.1 (t4) (P < .0001). We had to temporarily suspend treatment for 3 patients at t1, t4, and t5 because of severe obstructive sleep apnea. The percentage of patients with severe adenotonsillar hypertrophy was significantly higher at t4 and t5 than at t0. The OAHI directly correlated with the adenoid size (adjusted for age) (P < .01) but not with the tonsil size and IGF-1 levels.
Long-term GH treatment in patients with PWS is safe; however, we recommend annual polysomnography and adenotonsillar evaluation.
在患有普拉德-威利综合征（PWS）的儿童中，短期生长激素（GH）治疗期间已报告出现腺样体扁桃体组织肥大和阻塞性睡眠呼吸暂停。
我们进行了一项观察性研究，以评估长期 GH 治疗对 PWS 儿童睡眠呼吸障碍和腺样体扁桃体肥大的影响。
这是一项纵向观察性研究。
我们评估了 75 名经基因证实患有 PWS 的儿童，其中 50 名符合纳入标准并被纳入我们的研究。患者在治疗前（t0）、治疗 6 周后（t1）、治疗 6 个月后（t2）、治疗 12 个月后（t3）以及此后每年（t4-t6）进行评估，最长达 4 年的 GH 治疗。使用多导睡眠图评估夜间中枢性呼吸暂停指数、阻塞性呼吸暂停低通气指数（OAHI）、呼吸紊乱指数和最小血氧饱和度。我们使用可弯曲纤维内镜评估腺样体扁桃体大小。
OAHI>1 的患者比例从 t1、t4 和 t6 时的 3%、22%和 38%分别增加（χ²=12.2；P<.05）。我们观察到呼吸紊乱指数从 1.4（t0）降至 0.8（t3）（P<.05），以及中枢性呼吸暂停指数从 1.2（t0）降至 0.1（t4）（P<.0001）。我们不得不因严重阻塞性睡眠呼吸暂停在 t1、t4 和 t5 暂时停止治疗 3 名患者。t4 和 t5 时严重腺样体扁桃体肥大的患者比例明显高于 t0 时。OAHI 与腺样体大小（按年龄调整）直接相关（P<.01），但与扁桃体大小和 IGF-1 水平无关。
PWS 患者长期 GH 治疗是安全的；然而，我们建议每年进行多导睡眠图和腺样体扁桃体评估。
