Department of Pediatrics, University of Alberta, Edmonton, Alberta, Canada.
Department of Agricultural, Food and Nutritional Science, University of Alberta, Edmonton, Alberta, Canada.
Obes Rev. 2020 May;21(5):e12992. doi: 10.1111/obr.12992. Epub 2019 Dec 30.
In early childhood, individuals with Prader-Willi syndrome (PWS) experience excess weight gain and severe hyperphagia with food compulsivity, which often leads to early onset morbid obesity. Effective treatments for appetite suppression and weight control are currently unavailable for PWS. Our aim to further understand the pathogenesis of PWS led us to carry out a comprehensive search of the current and emerging therapies for managing hyperphagia and extreme weight gain in PWS. A literature search was performed using PubMed and the following keywords: "PWS" AND "therapy" OR "[drug name]"; reference lists, pharmaceutical websites, and the ClinicalTrials.gov registry were also reviewed. Articles presenting data from current standard treatments in PWS and also clinical trials of pharmacological agents in the pipeline were selected. Current standard treatments include dietary restriction/modifications, exercise, and growth hormone replacement, which appear to have limited efficacy for appetite and weight control in patients with PWS. The long-term safety and effectiveness of bariatric surgery in PWS remains unknown. However, many promising pharmacotherapies are in development and, if approved, will bring much needed choices into the PWS pharmacological armamentarium. With the progress that is currently being made in our understanding of PWS, an effective treatment may not be far off.
在儿童早期,普拉德-威利综合征(PWS)患者会出现体重过度增加和严重的暴食症伴食物强迫,这通常会导致早期发病的病态肥胖。目前尚无有效的食欲抑制和体重控制方法可用于治疗 PWS。我们旨在进一步了解 PWS 的发病机制,因此对目前和新兴的治疗 PWS 患者暴食症和体重过度增加的方法进行了全面搜索。使用 PubMed 进行了文献检索,并使用了以下关键字:“PWS”和“治疗”或“[药物名称]”;还查阅了参考文献列表、制药网站和 ClinicalTrials.gov 注册处。选择了介绍 PWS 现行标准治疗方法的数据以及正在研究中的药物的临床试验的文章。目前的标准治疗包括饮食限制/调整、运动和生长激素替代治疗,但对于 PWS 患者的食欲和体重控制似乎效果有限。减肥手术在 PWS 中的长期安全性和有效性尚不清楚。然而,许多有前途的药物治疗正在开发中,如果获得批准,将为 PWS 的药物治疗带来急需的选择。随着我们对 PWS 理解的不断进步,有效的治疗方法可能指日可待。