Suppr超能文献

普拉德-威利综合征的内分泌特征:一项侧重于基因型-表型相关性的叙述性综述。

Endocrine features of Prader-Willi syndrome: a narrative review focusing on genotype-phenotype correlation.

机构信息

Department of Medical and Surgical Sciences for Mother, Children and Adults, Pediatric Unit, University of Modena and Reggio Emilia, Modena, Italy.

Department of Pediatrics, University of L'Aquila, L'Aquila, Italy.

出版信息

Front Endocrinol (Lausanne). 2024 Apr 26;15:1382583. doi: 10.3389/fendo.2024.1382583. eCollection 2024.

Abstract

Prader-Willi syndrome (PWS) is a complex genetic disorder caused by three different types of molecular genetic abnormalities. The most common defect is a deletion on the paternal 15q11-q13 chromosome, which is seen in about 60% of individuals. The next most common abnormality is maternal disomy 15, found in around 35% of cases, and a defect in the imprinting center that controls the activity of certain genes on chromosome 15, seen in 1-3% of cases. Individuals with PWS typically experience issues with the hypothalamic-pituitary axis, leading to excessive hunger (hyperphagia), severe obesity, various endocrine disorders, and intellectual disability. Differences in physical and behavioral characteristics between patients with PWS due to deletion versus those with maternal disomy are discussed in literature. Patients with maternal disomy tend to have more frequent neurodevelopmental problems, such as autistic traits and behavioral issues, and generally have higher IQ levels compared to those with deletion of the critical PWS region. This has led us to review the pertinent literature to investigate the possibility of establishing connections between the genetic abnormalities and the endocrine disorders experienced by PWS patients, in order to develop more targeted diagnostic and treatment protocols. In this review, we will review the current state of clinical studies focusing on endocrine disorders in individuals with PWS patients, with a specific focus on the various genetic causes. We will look at topics such as neonatal anthropometry, thyroid issues, adrenal problems, hypogonadism, bone metabolism abnormalities, metabolic syndrome resulting from severe obesity caused by hyperphagia, deficiencies in the GH/IGF-1 axis, and the corresponding responses to treatment.

摘要

普拉德-威利综合征(PWS)是一种复杂的遗传疾病,由三种不同类型的分子遗传异常引起。最常见的缺陷是父系 15q11-q13 染色体缺失,约见于 60%的个体。其次常见的异常是母系 15 号染色体三体,见于约 35%的病例,以及控制 15 号染色体上某些基因活性的印迹中心缺陷,见于 1-3%的病例。PWS 患者通常存在下丘脑-垂体轴问题,导致过度饥饿(贪食)、严重肥胖、各种内分泌紊乱和智力障碍。文献中讨论了由于缺失与母系二体性导致的 PWS 患者在身体和行为特征上的差异。母系二体性患者往往更频繁地出现神经发育问题,如自闭症特征和行为问题,并且与缺失关键 PWS 区域的患者相比,一般具有更高的智商水平。这促使我们回顾相关文献,以探讨 PWS 患者遗传异常与内分泌紊乱之间可能存在的联系,从而制定更有针对性的诊断和治疗方案。在本综述中,我们将回顾目前专注于 PWS 患者内分泌紊乱的临床研究现状,特别关注各种遗传病因。我们将探讨新生儿人体测量学、甲状腺问题、肾上腺问题、性腺功能减退、骨代谢异常、贪食引起的严重肥胖导致的代谢综合征、GH/IGF-1 轴缺陷以及相应的治疗反应等主题。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/096f/11082343/b24e3f2d6b2a/fendo-15-1382583-g001.jpg

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验