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同型半胱氨酸血症不伴甲基丙二酸尿症

Homocystinuria without methylmalonic aciduria

定义 英文原文(暂无中文)

Homocystinuria without methylmalonic aciduria is an inborn error of vitamin B12 (cobalamin) metabolism characterized by megaloblastic anemia, encephalopathy and, sometimes, developmental delay, and associated with homocystinuria and hyperhomocysteinemia. There are three types of homocystinuria without methylmalonic aciduria; cblE, cblG and cblD-variant 1 (cblDv1).

别名

功能性甲硫氨酸合成缺乏症

基本事实

遗传方式
常染色体隐性
发病年龄
各年龄段
患病率
<1 / 1 000 000

相关基因 3来自下位疾病

Orphanet 未在本条目上直接标注致病基因。下表由本组所属的下位疾病汇总而来,「来源条目」列给出基因实际标注在哪一个 ORPHA 条目上。

基因名称来源条目
MMADHCmetabolism of cobalamin associated DORPHA:308380
MTR5-methyltetrahydrofolate-homocysteine methyltransferaseORPHA:2170
MTRR5-methyltetrahydrofolate-homocysteine methyltransferase reductaseORPHA:2169

临床表型 31

极常见 99–80%1

  • 高胱氨酸尿症 HP:0002156

常见 79–30%15

  • 大脑皮层萎缩 HP:0002120
  • 困倦 HP:0002329
  • 脑病 HP:0001298
  • 发育迟滞 HP:0001508
  • 喂养困难 HP:0011968
  • 全面发育迟缓 HP:0001263
  • 高同型半胱氨酸血症 HP:0002160
  • 肌张力减退 HP:0001252
  • 智力障碍 HP:0001249
  • 大细胞性贫血 HP:0001972
  • 眼球震颤 HP:0000639
  • 精神病 HP:0000709
  • 视网膜病变 HP:0000488
  • 癫痫发作 HP:0001250
  • 斜视 HP:0000486

偶见 29–5%11

  • 共济失调 HP:0001251
  • 非典型行为 HP:0000708
  • 脑萎缩 HP:0012444
  • 痴呆 HP:0000726
  • 生长延迟 HP:0001510
  • 低甲硫氨酸血症 HP:0003658
  • 昏睡 HP:0001254
  • 意识下降 HP:0004372
  • 视力下降 HP:0007663
  • 视觉障碍 HP:0000505
  • 呕吐 HP:0002013

罕见 <4–1%3

  • 溶血性尿毒症综合征 HP:0005575
  • 婴儿痉挛 HP:0012469
  • 视神经萎缩 HP:0000648

排除 0%1

  • 甲基丙二酸血症 HP:0002912

近两年的全球研究 183L2

2024/10 起在 Europe PMC 检索所得,按发表时间倒序显示最近 20 篇。本病的检索词较宽泛,命中数可能偏高,请以标题为准。标题未译成中文——自动翻译需要接入 LLM 服务,尚未引入。

  • 2026-09综述开放获取
    Folate: An Emerging Target in Type 2 Diabetes Mellitus and Its Complications
    Metabolites · DOI · Europe PMC
  • 2026-09开放获取
    Global DNA methylation and MTHFR gene polymorphism status of ghanaians with early-onset intracerebral hemorrhage: implications for future interventions
    Equity neuroscience · DOI · Europe PMC
  • 2026-08开放获取
    Plasma Homocysteine as a Biomarker for Cardiovascular Risk Stratification in Children and Adolescents With Type 1 Diabetes Mellitus: A Cross-Sectional Study From Western Algeria
    Cureus · DOI · Europe PMC
  • 2026-08开放获取
    Imerslund-Gräsbeck Syndrome Caused by Compound Heterozygous Mutations in the &lt;i&gt;AMN&lt;/i&gt; Gene: A Case Report
    Clinical case reports · DOI · Europe PMC
  • 2026-08综述开放获取
    Revisiting the hematological manifestations of vitamin B12 deficiency
    Frontiers in nutrition · DOI · Europe PMC
  • 2026-08综述开放获取
    Pediatric Stroke in Asia and Africa: Epidemiology, Etiology, and Resource-Stratified Care Across Geographic and System-Level Disparities
    Journal of central nervous system disease · DOI · Europe PMC
  • 2026-07综述开放获取
    Betaine as a regulator of metabolism, epigenetics, and cellular osmoprotection: therapeutic implications in cardiometabolic and renal diseases
    Frontiers in pharmacology · DOI · Europe PMC
  • 2026-07病例报告开放获取
    Neonatal-onset cblC-type methylmalonic acidemia combined with homocysteinemia: case report
    Frontiers in pediatrics · DOI · Europe PMC
  • 2026-07综述开放获取
    Biomarkers in Clinical Medicine Research: A Literature Survey in the PubMed Database and a Critical Evaluation
    Journal of clinical medicine · DOI · Europe PMC
  • 2026-07荟萃分析系统综述开放获取
    Hyperhomocysteinemia, a risk factor for various health conditions: an umbrella review of systematic reviews with meta-analysis
    Aging clinical and experimental research · DOI · Europe PMC
  • 2026-07开放获取
    Association of coronary artery disease related single nucleotide-polymorphisms with extreme Prakriti types: Insights from a case control study
    Journal of Ayurveda and integrative medicine · DOI · Europe PMC
  • 2026-07开放获取
    Benzbromarone as a Novel Candidate for Preventing Alzheimer's Disease: Evidence From Real-World Data Screening and in Vitro Validation
    Clinical and translational science · DOI · Europe PMC
  • 2026-07系统综述
    First Revision of the Guidelines for the Diagnosis and Management of Remethylation Disorders
    Journal of inherited metabolic disease · 被引 2 · DOI · Europe PMC
  • 2026-07开放获取
    Higher serum gastric parietal cell antibody titers are associated with increased frequencies of macrocytosis, vitamin B12 deficiency, and hyperhomocysteinemia in patients with oral lichen planus
    Journal of dental sciences · DOI · Europe PMC
  • 2026-06开放获取
    Nationwide trends and forecasts in Alzheimer's and cerebrovascular disease-related mortality in the United States, 1999-2023: A CDC WONDER analysis
    The Journal of international medical research · DOI · Europe PMC
  • 2026-06综述开放获取
    Metabolic Reprogramming-Driven Cardiovascular Immune Damage: From Glyco-Lipotoxicity and Epigenetic Memory to Multidimensional Cross-Organ Communication Networks
    International journal of molecular sciences · 被引 1 · DOI · Europe PMC
  • 2026-06开放获取
    Methotrexate Toxicity in a Patient With Renal Failure Despite a Normal Methotrexate Concentration: A Case Report and Literature Review
    Clinical case reports · DOI · Europe PMC
  • 2026-06病例报告开放获取
    Middle cerebral artery fenestration presenting with cerebral ischemia: a case report and review of the literature
    Frontiers in medicine · DOI · Europe PMC
  • 2026-06开放获取
    CIITA/PRMT5 promote CD4&lt;sup&gt;+&lt;/sup&gt;Gzma&lt;sup&gt;+&lt;/sup&gt; T cell activation via H3R2me2-mediated endothelial expression of MHC class II in smoking-induced atherosclerosis
    BMC medicine · DOI · Europe PMC
  • 2026-05
    Dietary sulfur amino acids enhance anti-tumor immunity in colon cancer via an NKT cell-XCL1-cDC1 circuit
    Immunity · 被引 5 · DOI · Europe PMC

外部标识与链接

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本页数据来源

  • 疾病定义、同义词、基因、表型、流行病学:Orphanet(CC BY 4.0)
  • 中文病名:Orphanet 中文包,冻结于 2020-06-01
  • 表型中文标签:HPO 简体中文翻译(CHPO 上游成果)