Ngu Hock Lock, Zabedah Md Yunus, Kobayashi Keiko
Genetics Department, Kuala Lumpur Hospital, Jalan Pahang, 50586 Kuala Lumpur, Malaysia.
Malays J Pathol. 2010 Jun;32(1):53-7.
Citrin deficiency is an autosomal recessive disorder caused by mutation in the SLC25AJ3 gene. It has two major phenotypes: adult-onset type II citrullinemia (CTLN2) and neonatal intrahepatic cholestatic caused by citrin deficiency (NICCD). NICCD is characterized by neonatal/infantile-onset cholestatic hepatitis syndrome associated with multiple amino acidemia and hypergalactosemia. NICCD is self-limiting in most patients. However, some patients may develop CTLN2 years later, which manifests as fatal hyperammonemia coma. We report three unrelated Malay children with genetically confirmed NICCD characterised by an insertion mutation IVS16ins3kb in SLC25A13 gene. All 3 patients presented with prolonged neonatal jaundice which resolved without specific treatment between 5 to 10 months. Of note was the manifestation of a peculiar dislike of sweet foods and drinks. Elevated plasma citrulline was an important biochemical marker. NICCD should be considered in the differential diagnosis of cholestatic jaundice in Malaysian infants regardless of ethnic origin.
瓜氨酸缺乏症是一种由SLC25AJ3基因突变引起的常染色体隐性疾病。它有两种主要表型:成人发作的II型瓜氨酸血症(CTLN2)和由瓜氨酸缺乏引起的新生儿肝内胆汁淤积症(NICCD)。NICCD的特征是新生儿/婴儿期发作的胆汁淤积性肝炎综合征,伴有多种氨基酸血症和高半乳糖血症。大多数NICCD患者病情会自我缓解。然而,一些患者可能在数年之后发展为CTLN2,表现为致命的高氨血症昏迷。我们报告了3名无亲缘关系的马来儿童,经基因检测确诊为NICCD,其特征为SLC25A13基因存在IVS16ins3kb插入突变。所有3例患者均出现新生儿黄疸持续时间延长的情况,未经特殊治疗,黄疸在5至10个月内消退。值得注意的是,他们均表现出对甜食和饮料的特殊厌恶。血浆瓜氨酸升高是一项重要的生化指标。在马来西亚婴儿胆汁淤积性黄疸的鉴别诊断中,无论种族出身,均应考虑NICCD。