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转醛醇酶缺乏症合并肝肺综合征患者的肝移植机会

Chances of Liver Transplantation in a Patient With Transaldolase Deficiency Complicated by Hepatopulmonary Syndrome.

作者信息

Fallata Ebtehal, Alamri Aisha M, Alrabee Hadeel A, Alghamdi Abdulhadi A, Alsaearei Ameera

机构信息

Department of Pediatrics, East Jeddah General Hospital, Jeddah, SAU.

Department of Pediatrics, Division of Gastroenterology, East Jeddah General Hospital, Jeddah, SAU.

出版信息

Cureus. 2023 Feb 18;15(2):e35150. doi: 10.7759/cureus.35150. eCollection 2023 Feb.

Abstract

Eyaid's syndrome or Transaldolase Deficiency (TD) (OMIM 606003) is a rare autosomal recessive inborn error of metabolism. In this report, we describe the case of an eight-year-old Saudi girl with a history of hepatosplenomegaly since infancy, who presented to the emergency department for a short history of cough and worsening cyanosis. She had growth retardation, facial dysmorphia, cardiac defect, neutropenia, and thrombocytopenia, besides hepatosplenomegaly. A thorough investigation led to the diagnosis of hepatopulmonary syndrome and whole exome sequencing showed a homozygous frameshift variant in the TALDO1gene, c.793del, p.Gln265fs. Thus, the patient was diagnosed with TD complicated with hepatopulmonary syndrome, and the indication of liver transplantation was discussed.

摘要

艾亚德综合征或转醛醇酶缺乏症(TD)(OMIM 606003)是一种罕见的常染色体隐性遗传性代谢缺陷病。在本报告中,我们描述了一名8岁沙特女孩的病例,她自婴儿期起就有肝脾肿大病史,因咳嗽病史短且发绀加重而就诊于急诊科。除肝脾肿大外,她还有生长发育迟缓、面部畸形、心脏缺陷、中性粒细胞减少和血小板减少。全面检查后诊断为肝肺综合征,全外显子组测序显示TALDO1基因存在纯合移码变异,即c.793del,p.Gln265fs。因此,该患者被诊断为TD合并肝肺综合征,并讨论了肝移植指征。

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引用本文的文献

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本文引用的文献

1
Prenatal Diagnosis of Fetus With Transaldolase Deficiency Identifies Compound Heterozygous Variants: A Case Report.
Front Genet. 2022 Feb 4;12:752272. doi: 10.3389/fgene.2021.752272. eCollection 2021.
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Sci Rep. 2021 Feb 18;11(1):4134. doi: 10.1038/s41598-021-83785-x.
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Hypergonadotrophic hypogonadism in a patient with transaldolase deficiency: novel mutation in the pentose phosphate pathway.
Hormones (Athens). 2021 Sep;20(3):581-585. doi: 10.1007/s42000-020-00252-4. Epub 2020 Nov 7.
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Apparent Acetaminophen Toxicity in a Patient with Transaldolase Deficiency.
JIMD Rep. 2019;44:9-15. doi: 10.1007/8904_2018_116. Epub 2018 Jun 20.
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