Department of Gastroenterology, Cumhuriyet University Faculty of Medicine, Sivas, Turkey.
Department of Biochemistry, Cumhuriyet University Faculty of Medicine, Sivas, Turkey.
Liver Int. 2021 May;41(5):905-914. doi: 10.1111/liv.14794. Epub 2021 Mar 18.
The Chanarin-Dorfman syndrome (CDS) is a rare, autosomal recessively inherited genetic disease. This syndrome is associated with a decrease in the lipolysis activity in multiple tissue cells because of recessive mutations in the abhydrolase domain containing 5 (ABHD5) gene, which leads to the accumulation of lipid droplets in multiple types of cells. Major clinical symptoms in patients with CDS include ichthyosis and intracytoplasmic lipid droplets. The variability of clinical symptoms in patients with CDS depends on a large number of mutations involved. In this syndrome, liver involvement is an important cause of mortality and morbidity. This review aims to summarize the demographic characteristic, clinical symptoms, liver involvement and mutations in CDS patients in the literature to date.
Chanarin-Dorfman 综合征(CDS)是一种罕见的常染色体隐性遗传疾病。该综合征与多个组织细胞中的脂肪分解活性降低有关,这是由于 ABHD5 基因的隐性突变所致,导致多种类型细胞中的脂滴积累。CDS 患者的主要临床症状包括鱼鳞癣和细胞内脂滴。CDS 患者的临床症状的可变性取决于涉及的大量突变。在该综合征中,肝脏受累是死亡和发病的重要原因。本综述旨在总结迄今为止文献中 CDS 患者的人口统计学特征、临床症状、肝脏受累和突变。