Singh Prapti, Amaro Deirdre, Obi Olugbemisola, Kiran Fnu, Hediger Erin, Toler Tomi L, Dickson Patricia I, Grange Dorothy K
Division of Genetics and Genomic Medicine, Department of Pediatrics Washington University School of Medicine in St. Louis Saint Louis Missouri USA.
Division of Reproductive Endocrinology and Infertility, Department of Obstetrics and Gynecology University of Iowa, Roy J. and Lucille A. Carver College of Medicine Iowa City Iowa USA.
JIMD Rep. 2023 Apr 30;64(4):261-264. doi: 10.1002/jmd2.12365. eCollection 2023 Jul.
Very long chain acyl-CoA dehydrogenase (VLCAD) deficiency is an autosomal recessive long chain fatty acid β-oxidation disorder with a variable clinical spectrum, ranging from an acute neonatal presentation with cardiac and hepatic failure to childhood or adult onset of symptoms with hepatomegaly or rhabdomyolysis provoked by illness or exertion. Neonatal cardiac arrest or sudden unexpected death can be the presenting phenotype in some patients, emphasizing the importance of early clinical suspicion and intervention. We report a patient who had a cardiac arrest and died at one day of age. Following her death, the newborn screen reported biochemical evidence of VLCAD deficiency, which was confirmed with pathologic findings at autopsy and by molecular genetic testing.
极长链酰基辅酶A脱氢酶(VLCAD)缺乏症是一种常染色体隐性遗传的长链脂肪酸β氧化障碍疾病,临床谱表现多样,从伴有心脏和肝功能衰竭的急性新生儿表现,到儿童期或成年期发病,出现因疾病或劳累诱发的肝肿大或横纹肌溶解症状。在一些患者中,新生儿心脏骤停或意外猝死可能是首发表型,这凸显了早期临床怀疑和干预的重要性。我们报告了一名出生一天时发生心脏骤停并死亡的患者。她死后,新生儿筛查报告了VLCAD缺乏症的生化证据,尸检病理结果及分子遗传学检测均证实了这一诊断。