Fallata Ebtehal, Alamri Aisha M, Alrabee Hadeel A, Alghamdi Abdulhadi A, Alsaearei Ameera
Department of Pediatrics, East Jeddah General Hospital, Jeddah, SAU.
Department of Pediatrics, Division of Gastroenterology, East Jeddah General Hospital, Jeddah, SAU.
Cureus. 2023 Feb 18;15(2):e35150. doi: 10.7759/cureus.35150. eCollection 2023 Feb.
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.
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。