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钴胺素相关疾病的临床表现:从后天缺乏到吸收和细胞内途径的先天错误。

The clinical presentation of cobalamin-related disorders: From acquired deficiencies to inborn errors of absorption and intracellular pathways.

机构信息

Division of Metabolism and Children's Research Center, University Children's Hospital Zürich, Zürich, Switzerland.

Department of Paediatrics, Landeskrankenhaus Bregenz, Bregenz, Austria.

出版信息

J Inherit Metab Dis. 2019 Jul;42(4):686-705. doi: 10.1002/jimd.12012. Epub 2019 Feb 13.


DOI:10.1002/jimd.12012
PMID:30761552
Abstract

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.

摘要

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。

相似文献

[1]
The clinical presentation of cobalamin-related disorders: From acquired deficiencies to inborn errors of absorption and intracellular pathways.

J Inherit Metab Dis. 2019-2-13

[2]
Acquired and inherited disorders of cobalamin and folate in children.

Br J Haematol. 2006-7

[3]
[Remethylation disorders: about two cases].

Ann Biol Clin (Paris). 2020-12-1

[4]
Phenotype, treatment practice and outcome in the cobalamin-dependent remethylation disorders and MTHFR deficiency: Data from the E-HOD registry.

J Inherit Metab Dis. 2019-2-17

[5]
Clinical pattern, mutations and in vitro residual activity in 33 patients with severe 5, 10 methylenetetrahydrofolate reductase (MTHFR) deficiency.

J Inherit Metab Dis. 2016-1

[6]
Outcomes after newborn screening for propionic and methylmalonic acidemia and homocystinurias.

J Inherit Metab Dis. 2024-7

[7]
Cobalamin and folate deficiency: acquired and hereditary disorders in children.

Semin Hematol. 1999-1

[8]
Genetic defects of folate and cobalamin metabolism.

Eur J Pediatr. 1998-4

[9]
Update on cobalamin, folate, and homocysteine.

Hematology Am Soc Hematol Educ Program. 2003

[10]
High folic acid consumption leads to pseudo-MTHFR deficiency, altered lipid metabolism, and liver injury in mice.

Am J Clin Nutr. 2015-3

引用本文的文献

[1]
Disorder of intracellular cobalamin metabolism: Importance of rapid diagnostic illustrated by a case report of early-onset methylmalonic aciduria and homocystinuria, cobalamin C type.

Heliyon. 2025-1-23

[2]
Laboratory evolution of with a natural vitamin B analog reveals roles for cobamide uptake and adenosylation in methionine synthase-dependent growth.

J Bacteriol. 2025-2-20

[3]
Late-onset renal TMA and tubular injury in cobalamin C disease: a report of three cases and literature review.

BMC Nephrol. 2024-10-10

[4]
Three-generation female cohort with macrocytic anemia and iron overload.

Am J Hematol. 2025-1

[5]
The Benefit of Detecting Reduced Intracellular B12 Activity through Newborn Screening Remains Unclear.

Int J Neonatal Screen. 2024-6-18

[6]
Late-onset refractory hemolytic anemia in siblings treated for methionine synthase reductase deficiency: A rare complication possibly prevented by hydroxocobalamin dose escalation?

JIMD Rep. 2024-4-15

[7]
Laboratory evolution of with a natural vitamin B analog reveals roles for cobamide uptake and adenosylation in methionine synthase-dependent growth.

bioRxiv. 2024-8-7

[8]
Interaction of Glutathione with MMACHC Arginine-Rich Pocket Variants Associated with Cobalamin C Disease: Insights from Molecular Modeling.

Biomedicines. 2023-12-4

[9]
Biochemical, Nutritional, and Clinical Parameters of Vitamin B12 Deficiency in Infants: A Systematic Review and Analysis of 292 Cases Published between 1962 and 2022.

Nutrients. 2023-11-29

[10]
Late-onset cblC defect: clinical, biochemical and molecular analysis.

Orphanet J Rare Dis. 2023-9-28

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