一名枫糖尿病患者肝移植后出现急性代谢失调

IF 1.8 Q2 Biochemistry, Genetics and Molecular Biology
JIMD reports Pub Date : 2024-11-25 DOI:10.1002/jmd2.12460
Shao Ching Tu, Marium Khan, Katie Wolfe, Sakil S. Kulkarni, Elizabeth Toolan, Dorothy K. Grange
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引用次数: 0

摘要

枫糖浆尿病(MSUD)是一种以支链氨基酸(亮氨酸、异亮氨酸和缬氨酸)积累为特征的先天性代谢错误,由支链α -酮酸脱氢酶复合物缺陷引起。肝移植是MSUD的有效治疗方法,移植后患者通常可以耐受正常饮食,无症状性代谢失代偿。大多数移植后患者不遵循病日饮食。我们报告一例7岁男性MSUD IA型,2岁肝移植后状态,在口服摄入不良和呕吐3天后出现深度脑病。广泛的实验室检查对高白细胞增多症和未揭示的感染性检查具有重要意义。我们对圣路易斯华盛顿大学随访的8例肝移植后MSUD患者进行了回顾。该综述显示,在该患者队列中,在并发疾病期间通常未检查血浆氨基酸。虽然我们的大多数患者没有脑病记录,但其中一名癫痫患者在胃肠道疾病期间发作。根据文献回顾和本中心的经验,肝移植后MSUD患者并发疾病的急性代谢失代偿似乎很少见。本病例报告提高了人们对MSUD患者在肝移植后仍有发生代谢危象风险的认识,并对该患者队列中与代谢失代偿相关的危险因素提供了额外的见解。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Acute metabolic decompensation after liver transplant in a patient with maple syrup urine disease

Maple syrup urine disease (MSUD) is an inborn error of metabolism characterized by the accumulation of branched-chain amino acids (leucine, isoleucine, and valine) caused by a defect in the branched-chain alpha-keto acid dehydrogenase complex. Liver transplant is an effective therapy for MSUD, and patients can usually tolerate a regular diet after transplant without symptomatic metabolic decompensation. Most post-transplant patients do not follow a sick-day diet. We report a case of a 7-year-old male with MSUD Type IA, status post-liver transplant at 2 years of age, who presented with profound encephalopathy following poor oral intake and vomiting for 3 days. Broad laboratory workup was significant for hyperleucinosis and an unrevealing infectious workup. We conducted a review of eight post-liver transplant MSUD patients followed at Washington University in St. Louis. The review revealed that plasma amino acids were generally not checked during intercurrent illnesses in this patient cohort. While most of our patients have not had documented encephalopathy, one of the patients with epilepsy had a seizure during a gastrointestinal illness. Based on the review of the literature and from our center's experience, acute metabolic decompensation with intercurrent illnesses in MSUD patients after liver transplant appears to be rare. This case report raises awareness that patients with MSUD are still at risk of developing metabolic crisis post-liver transplant and provides additional insight into the risk factors associated with metabolic decompensation in this patient cohort.

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来源期刊
JIMD reports
JIMD reports Biochemistry, Genetics and Molecular Biology-Biochemistry, Genetics and Molecular Biology (miscellaneous)
CiteScore
3.30
自引率
0.00%
发文量
84
审稿时长
12 weeks
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