The clinical presentation of cobalamin‐related disorders: from acquired deficiencies to inborn errors of absorption and intracellular pathways

M Huemer, MR Baumgartner - Journal of inherited metabolic …, 2019 - Wiley Online Library
M Huemer, MR Baumgartner
Journal of inherited metabolic disease, 2019Wiley Online Library
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 …
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.
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