Division of Metabolism and Children's Research Center, University Children's Hospital Zürich, Zürich, Switzerland.
Department of Paediatrics, Landeskrankenhaus Bregenz, Bregenz, Austria.
J Inherit Metab Dis. 2019 Jul;42(4):686-705. doi: 10.1002/jimd.12012. Epub 2019 Feb 13.
This review gives an overview of clinical characteristics, treatment and outcome of nutritional and acquired cobalamin (Cbl; synonym: vitamin B12) deficiencies, inborn errors of Cbl absorption and intracellular trafficking, as well as methylenetetrahydrofolate dehydrogenase (MTHFD1) and methylene tetrahydrofolate reductase (MTHFR) deficiencies, which impair Cbl-dependent remethylation. Acquired and inborn Cbl-related disorders and MTHFR deficiency cause multisystem, often severe disease. Failure to thrive, neurocognitive or psychiatric symptoms, eye disease, bone marrow alterations, microangiopathy and thromboembolic events are characteristic. The recently identified MTHFD1 defect additionally presents with severe immune deficiency. Deficient Cbl-dependent enzymes cause reduced methylation capacity and metabolite toxicity. Further net-effects of perturbed Cbl function or reduced Cbl supply causing oxidative stress, altered cytokine regulation or immune functions are discussed.
这篇综述概述了营养性和获得性钴胺素(Cbl;同义词:维生素 B12)缺乏症、Cbl 吸收和细胞内转运的先天性错误以及亚甲基四氢叶酸脱氢酶(MTHFD1)和亚甲基四氢叶酸还原酶(MTHFR)缺乏症的临床特征、治疗和转归,这些疾病会损害 Cbl 依赖性再甲基化。获得性和先天性 Cbl 相关疾病以及 MTHFR 缺乏症可导致多系统疾病,通常病情严重。生长不良、神经认知或精神症状、眼病、骨髓改变、微血管病和血栓栓塞事件是其特征。最近发现的 MTHFD1 缺陷还伴有严重的免疫缺陷。缺乏 Cbl 依赖性酶会导致甲基化能力降低和代谢物毒性增加。此外,Cbl 功能紊乱或 Cbl 供应减少导致氧化应激、细胞因子调节或免疫功能改变的净效应也将进行讨论。
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