普拉德-威利综合症:儿童指导和成年过渡。

IF 2.6 3区 医学 Q3 ENDOCRINOLOGY & METABOLISM
Endocrine Connections Pub Date : 2024-07-10 Print Date: 2024-08-01 DOI:10.1530/EC-24-0091
M Guftar Shaikh, Timothy G Barrett, Nicola Bridges, Robin Chung, Evelien F Gevers, Anthony P Goldstone, Anthony Holland, Shankar Kanumakala, Ruth Krone, Andreas Kyriakou, E Anne Livesey, Angela K Lucas-Herald, Christina Meade, Susan Passmore, Edna Roche, Chris Smith, Sarita Soni
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引用次数: 0

摘要

普拉德-威利综合征(PWS)是一种罕见的孤儿病和复杂的遗传性神经发育障碍,出生率约为万分之一到三万分之一。对普氏综合症患者的管理需要多学科方法,最好是通过多学科团队(MDT)诊所,并在社区的支持下进行。肌张力低下、喂养不良和发育迟缓是新生儿期的特征,随后会出现多食和儿童期体重快速增长的风险。患有PWS的儿童和青少年(CA)通常表现出发育迟缓和轻度学习障碍,并可能发展成内分泌疾病、脊柱侧弯、呼吸困难(中枢性和阻塞性睡眠呼吸暂停)、挑战性行为、皮肤搔痒和心理健康问题,尤其是在成年后。本共识声明旨在为临床医生管理患有脊髓灰质炎的儿童和青少年(18 岁以下)提供一份参考文件。它考虑了儿科环境中的诊断、临床评估和管理,以及过渡到成人服务期间和之后的生物-心理-社会领域。该指南是从同行评议的科学报告中收集的信息,以及英国和爱尔兰参与护理 PWS 患者的一系列经验丰富的临床医生的专业知识编写而成的。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Prader-Willi syndrome: guidance for children and transition into adulthood.

Prader-Willi syndrome (PWS) is a rare orphan disease and complex genetic neurodevelopmental disorder, with a birth incidence of approximately 1 in 10,000-30,000. Management of people with PWS requires a multi-disciplinary approach, ideally through a multi-disciplinary team (MDT) clinic with community support. Hypotonia, poor feeding and faltering growth are characteristic features in the neonatal period, followed by hyperphagia and risk of rapid weight gain later in childhood. Children and adolescents (CA) with PWS usually display developmental delay and mild learning disability and can develop endocrinopathies, scoliosis, respiratory difficulties (both central and obstructive sleep apnoea), challenging behaviours, skin picking, and mental health issues, especially into adulthood. This consensus statement is intended to be a reference document for clinicians managing children and adolescents (up to 18 years of age) with PWS. It considers the bio-psycho-social domains of diagnosis, clinical assessment, and management in the paediatric setting as well as during and after transition to adult services. The guidance has been developed from information gathered from peer-reviewed scientific reports and from the expertise of a range of experienced clinicians in the United Kingdom and Ireland involved in the care of patients with PWS.

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来源期刊
Endocrine Connections
Endocrine Connections Medicine-Internal Medicine
CiteScore
5.00
自引率
3.40%
发文量
361
审稿时长
6 weeks
期刊介绍: Endocrine Connections publishes original quality research and reviews in all areas of endocrinology, including papers that deal with non-classical tissues as source or targets of hormones and endocrine papers that have relevance to endocrine-related and intersecting disciplines and the wider biomedical community.
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