Goldman Victoria E, Naguib Monica N, Vidmar Alaina P
Department of Pediatrics, Children's Hospital Los Angeles, Los Angeles, CA 90027, USA.
Center for Endocrinology, Children's Hospital Los Angeles, Los Angeles, CA 90027, USA.
J Clin Med. 2021 Sep 30;10(19):4540. doi: 10.3390/jcm10194540.
(1) Background: children with Prader-Willi syndrome (PWS) have high obesity rates due to hyperphagia and decreased metabolic rates. Although anti-obesity medications (AOMs) are prescribed to this population, there are no consensus guidelines on acceptability, safety, and efficacy. We present literature review and case series on AOMs in youth with PWS. (2) Methods: we performed PubMed review from January 2000 to April 2021 utilizing keywords: "Prader-Willi syndrome" or "PWS" and "medication" including: topiramate, metformin, phentermine, liraglutide, orlistat, oxytocin, semaglutide, naltrexone-bupropion. For our case series, patients were identified through retrospective chart reviews from a multi-disciplinary PWS clinic. Eligibility criteria: age ≤ 18 years, genetically confirmed PWS, AOM use for at least 16 weeks, and recent anthropometric data. (3) Results: a literature search yielded 14 articles (3 topiramate, 1 metformin, 4 liraglutide, 5 oxytocin, 1 naltrexone-bupropion). All studies reported improved hyperphagia with variable BMI effects. Ten adolescents met case series eligibility (mean age 13.2 ± 2.6 years, 40% female; AOMs: 6 metformin, 5 topiramate, 2 semaglutide, 3 liraglutide). After AOM course, 60% had decreased or stable BMI z-score. No significant side effects. (4) Conclusions: results suggest AOMs may be useful for weight management in youth with PWS. Additional studies are required to validate findings and support AOM treatment guidelines.
（1）背景：普拉德-威利综合征（PWS）患儿因食欲亢进和代谢率降低而肥胖率较高。尽管已为该人群开具了抗肥胖药物（AOM），但在可接受性、安全性和疗效方面尚无共识指南。我们展示了关于PWS青少年使用AOM的文献综述和病例系列。（2）方法：我们在2000年1月至2021年4月期间利用关键词“普拉德-威利综合征”或“PWS”以及“药物”进行了PubMed检索，包括：托吡酯、二甲双胍、芬特明、利拉鲁肽、奥利司他、催产素、司美格鲁肽、纳曲酮-安非他酮。对于我们的病例系列，通过对一家多学科PWS诊所的回顾性病历审查来确定患者。纳入标准：年龄≤18岁，基因确诊为PWS，使用AOM至少16周，以及近期人体测量数据。（3）结果：文献检索产生了14篇文章（3篇关于托吡酯，1篇关于二甲双胍，4篇关于利拉鲁肽，5篇关于催产素，1篇关于纳曲酮-安非他酮）。所有研究均报告食欲亢进有所改善，对体重指数（BMI）有不同影响。10名青少年符合病例系列纳入标准（平均年龄13.2±2.6岁，40%为女性；AOM：6例使用二甲双胍，5例使用托吡酯，2例使用司美格鲁肽，3例使用利拉鲁肽）。在使用AOM疗程后，60%的患者BMI z评分降低或稳定。无显著副作用。（4）结论：结果表明AOM可能对PWS青少年的体重管理有用。需要进一步研究来验证这些发现并支持AOM治疗指南。
