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新生儿筛查确诊的五例高甲硫氨酸血症病例分析。

Analysis of five cases of hypermethioninemia diagnosed by neonatal screening.

作者信息

Zhang Zhilei, Wang Yanyun, Ma Dingyuan, Cheng Wei, Sun Yun, Jiang Tao

机构信息

Center of Genetic Medicine, The Affiliated Obstetrics and Gynecology Hospital with Nanjing Medical University, Nanjing Maternity and Child Health Care Hospital, Nanjing, China.

出版信息

J Pediatr Endocrinol Metab. 2020 Jan 28;33(1):47-52. doi: 10.1515/jpem-2019-0285.

Abstract

Background Hypermethioninemia is a group of diseases with elevated plasma methionine (Met) caused by hereditary and non-hereditary factors, although it could also be caused by administration of the amino acid Met. Among these, the disease caused by methionine adenosyltransferase (MAT) I/III deficiency is the most common, and is characterized by persistent, isolated hypermethioninemia as well as slightly elevated homocysteine. S-adenosylmethionine is the product of Met, which can be used as a direct methyl donor of many substances, such as choline and nucleotide, and essential in the development of the body. Among the patients, most have no symptoms, and a small number have central nervous system complications with high levels of plasma Met, including mental retardation, cognitive impairment and special breathing odor. Methods In this study, five cases of MAT I/III deficiency were diagnosed and retrospectively analyzed among 220,000 newborns. Patients with high Met levels received a Met-restricted diet treatment. Results and conclusions MAT I/III deficiency is a common reason for Met elevation in neonatal screening by tandem mass spectrometry (MS/MS), which needs long-term follow-up except for these patients with explicitly benign mutations.

摘要

背景

高甲硫氨酸血症是一组由遗传和非遗传因素导致血浆甲硫氨酸(Met)升高的疾病,不过也可能由给予氨基酸甲硫氨酸引起。其中,由甲硫氨酸腺苷转移酶(MAT)I/III缺乏引起的疾病最为常见,其特征为持续性、孤立性高甲硫氨酸血症以及同型半胱氨酸轻度升高。S-腺苷甲硫氨酸是甲硫氨酸的产物,可作为许多物质(如胆碱和核苷酸)的直接甲基供体,对身体发育至关重要。在这些患者中,大多数无症状,少数血浆Met水平高的患者会出现中枢神经系统并发症,包括智力发育迟缓、认知障碍和特殊呼吸气味。方法:本研究在220,000例新生儿中诊断出5例MAT I/III缺乏症并进行回顾性分析。Met水平高的患者接受限制甲硫氨酸饮食治疗。结果与结论:MAT I/III缺乏是串联质谱(MS/MS)新生儿筛查中Met升高的常见原因,除了那些具有明确良性突变的患者外,都需要长期随访。

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