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印度患者的伍德豪斯-萨卡蒂综合征伴新型致病性变异。

Woodhouse-Sakati syndrome in an Indian patient with a novel pathogenic variant.

机构信息

Department of Medicine, Jawaharlal Institute of Postgraduate Medical Education & Research (JIPMER), India.

Department of Laboratory Medicine & Pathology, University of Washington, Seattle, Washington, USA.

出版信息

Am J Med Genet A. 2024 Jan;194(1):100-102. doi: 10.1002/ajmg.a.63405. Epub 2023 Sep 14.

Abstract

Woodhouse-Sakati syndrome consists of hypogonadism, diabetes mellitus, alopecia, ECG abnormalities, and dystonia. This condition is caused by the loss of function of the DCAF17 gene. Most of the patients have been reported from Greater Middle Eastern countries. We report a 38 male from southern India who presented with syncope and massive hemoptysis due to ruptured bronchopulmonary collaterals. He also had alopecia, cataracts, recently diagnosed diabetes and hypogonadism. Whole exome sequencing showed a novel homozygous truncating variant in the DCAF17 gene. Despite embolization of the aortopulmonary collaterals, the patient died of recurrent hemoptysis.

摘要

伍德豪斯-萨卡蒂综合征的特征为性腺功能减退、糖尿病、脱发、心电图异常和肌张力障碍。该病症是由 DCAF17 基因突变导致的功能丧失引起的。大多数患者都来自大中东国家。我们报告了一位来自印度南部的 38 岁男性,他因支气管肺侧支循环破裂而出现晕厥和大量咯血。他还患有脱发、白内障、最近诊断出的糖尿病和性腺功能减退。外显子组测序显示 DCAF17 基因中存在一种新的纯合截断变异。尽管进行了主动脉肺侧支栓塞,患者仍因反复咯血而死亡。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cf1f/10842022/fa245208d33a/nihms-1942750-f0001.jpg

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