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儿童多学科护理及生长激素治疗对普拉德-威利综合征成年患者健康问题的影响

Effects of Childhood Multidisciplinary Care and Growth Hormone Treatment on Health Problems in Adults with Prader-Willi Syndrome.

作者信息

Pellikaan Karlijn, Rosenberg Anna G W, Davidse Kirsten, Kattentidt-Mouravieva Anja A, Kersseboom Rogier, Bos-Roubos Anja G, Grootjen Lionne N, Damen Layla, van den Berg Sjoerd A A, van der Lely Aart J, Hokken-Koelega Anita C S, de Graaff Laura C G

机构信息

Department of Internal Medicine, Division of Endocrinology, Erasmus Medical Center, University Medical Centre Rotterdam, 3015 GD Rotterdam, The Netherlands.

Department of Internal Medicine, Division of Endocrinology, Center for Adults with Rare Genetic Syndromes, Erasmus Medical Center, University Medical Center Rotterdam, 3015 GD Rotterdam, The Netherlands.

出版信息

J Clin Med. 2021 Jul 23;10(15):3250. doi: 10.3390/jcm10153250.

Abstract

Prader-Willi syndrome (PWS) is a complex hypothalamic disorder. Features of PWS include hyperphagia, hypotonia, intellectual disability, and pituitary hormone deficiencies. The combination of growth hormone treatment and multidisciplinary care (GHMDc) has greatly improved the health of children with PWS. Little is known about the effects of childhood GHMDc on health outcomes in adulthood. We retrospectively collected clinical data of 109 adults with PWS. Thirty-nine had received GHMDc during childhood and adolescence (GHMDc+ group) and sixty-three had never received growth hormone treatment (GHt) nor multidisciplinary care (GHMDc- group). Our systematic screening revealed fewer undetected health problems in the GHMDc+ group (10%) than in the GHMDc- group (84%). All health problems revealed in the GHMDc+ group had developed between the last visit to the paediatric and the first visit to the adult clinic and/or did not require treatment. Mean BMI and the prevalence of diabetes mellitus type 2 were significantly lower in the GHMDc+ group compared to the GHMDc- group. As all patients who received GHt were treated in a multidisciplinary setting, it is unknown which effects are the result of GHt and which are the result of multidisciplinary care. However, our data clearly show that the combination of both has beneficial effects. Therefore, we recommend continuing GHMDc after patients with PWS have reached adult age.

摘要

普拉德-威利综合征(PWS)是一种复杂的下丘脑疾病。PWS的特征包括食欲亢进、肌张力减退、智力残疾和垂体激素缺乏。生长激素治疗与多学科护理相结合(GHMDc)极大地改善了PWS患儿的健康状况。关于儿童期GHMDc对成年期健康结局的影响,人们知之甚少。我们回顾性收集了109例成年PWS患者的临床数据。39例在儿童期和青春期接受过GHMDc(GHMDc+组),63例从未接受过生长激素治疗(GHt)和多学科护理(GHMDc-组)。我们的系统筛查显示,GHMDc+组未被发现的健康问题(10%)少于GHMDc-组(84%)。GHMDc+组发现的所有健康问题均在最后一次儿科就诊至首次成人门诊就诊期间出现和/或不需要治疗。与GHMDc-组相比,GHMDc+组的平均体重指数和2型糖尿病患病率显著更低。由于所有接受GHt治疗的患者均在多学科环境中接受治疗,因此不清楚哪些影响是GHt的结果,哪些是多学科护理的结果。然而,我们的数据清楚地表明,两者结合具有有益效果。因此,我们建议PWS患者成年后继续接受GHMDc治疗。

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