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一名儿科患者中由新型LIPC基因变异引起的高甘油三酯血症诱导的急性胰腺炎

Hypertriglyceridemia Induced Acute Pancreatitis Caused by a Novel LIPC Gene Variant in a Pediatric Patient.

作者信息

Balanescu Laura, Cardoneanu Ancuta, Stanciu Gabriel, Balanescu Radu, Minulescu Cristian, Pacurar Daniela, Moga Andreea

机构信息

Pediatric Surgery Department, Grigore Alexandrescu Emergency Hospital for Children, 011743 Bucharest, Romania.

Department of Pediatric Surgery and Orthopedics, Carol Davila University of Medicine and Pharmacy, 050474 Bucharest, Romania.

出版信息

Children (Basel). 2022 Feb 2;9(2):188. doi: 10.3390/children9020188.

Abstract

Hypertriglyceridemia induced acute pancreatitis is a rare cause of pancreatitis in children. Hepatic lipase deficiency is an extremely rare cause of hypertriglyceridemia, reported in only a few families to date. Hepatic lipase is the enzyme involved in the hydrolysis of triglycerides and phospholipids in remnants of triglyceride-rich lipoproteins that have a role in the conversion of very low density lipoprotein remnants to low density lipoproteins. Hepatic lipase deficiency is inherited in an autosomal recessive pattern. Detection of heterozygous carriers of hepatic lipase mutations remains accidental at the population level, as affected persons with a heterozygous state of hepatic lipase mutation do not display specific lipoprotein abnormalities and also patients with complete hepatic lipase deficiency have inconstant phenotype. The proximal promoter of the LIPC gene consists of four polymorphic sites in complete linkage disequilibrium. Five missense mutations in encoding exons have been described and proved to be responsible for hepatic lipase deficiency to date: S267F, T383M, L334F, A174T, and R186H, affecting the activity and secretion of hepatic lipase. We identified a primary disorder of the lipid metabolism as the cause of the acute episode of pancreatitis in a four years old patient, consisting of hepatic lipase deficiency caused by a novel genetic variant of the LIPC gene, a gross deletion of the genomic region encompassing exon 1. This variant was not previously described in the literature in persons with LIPC-related disorders and its significance is currently uncertain, but in the presented clinical and paraclinical context, it has the characteristics of a pathological variant inducing a hepatic lipase deficiency phenotype.

摘要

高甘油三酯血症诱发的急性胰腺炎是儿童胰腺炎的罕见病因。肝脂肪酶缺乏是高甘油三酯血症的极其罕见的病因,迄今为止仅在少数家族中有报道。肝脂肪酶是一种参与富含甘油三酯脂蛋白残粒中甘油三酯和磷脂水解的酶,在极低密度脂蛋白残粒转化为低密度脂蛋白过程中起作用。肝脂肪酶缺乏以常染色体隐性模式遗传。在人群水平上,肝脂肪酶突变杂合携带者的检测仍然是偶然的,因为肝脂肪酶突变杂合状态的患者没有表现出特定的脂蛋白异常,而且完全肝脂肪酶缺乏的患者也有不恒定的表型。LIPC基因的近端启动子由四个处于完全连锁不平衡状态的多态性位点组成。迄今为止,已描述了编码外显子中的五个错义突变,并证明它们是肝脂肪酶缺乏的原因:S267F、T383M、L334F、A174T和R186H,这些突变影响肝脂肪酶的活性和分泌。我们在一名4岁患者中确定了脂质代谢的原发性紊乱是急性胰腺炎发作的原因,该紊乱由LIPC基因的一种新型遗传变异导致肝脂肪酶缺乏引起,即基因组区域包含外显子1的大片段缺失。这种变异在先前关于LIPC相关疾病的文献中未被描述,其意义目前尚不确定,但在本文呈现的临床和辅助临床背景下,它具有诱导肝脂肪酶缺乏表型的病理性变异的特征。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a607/8870319/6ad6b5c9fb0c/children-09-00188-g001.jpg

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