Schnedl Wolfgang J, Schenk Michael, Enko Dietmar, Mangge Harald
Practice for General Internal Medicine, Dr.-Theodor-Körner-Str. 19b, 8600 Bruck/Mur, Austria.
Das Kinderwunsch Institut Schenk GmbH, Am Sendergrund 11, 8143 Dobl, Austria.
EXCLI J. 2020 Sep 11;19:1309-1313. doi: 10.17179/excli2020-2866. eCollection 2020.
Carnitine palmitoyltransferase II (CPT II) deficiency represents an inherited defect in mitochondrial long-chain fatty acid oxidation. Rhabdomyolysis with necrosis of muscle is caused by the destruction of skeletal muscle and leads to systemic, multiorgan complications due to the release of intracellular muscle components. Severe rhabdomyolysis may be triggered by combination of a genetic predisposition, including CPT II deficiency, with additionally acting causes. Generally, patients with CPT II deficiency are rarely clinical recognized and reported. We describe a patient presenting severe rhabdomyolysis due to urosepsis, who, in genetic testing, demonstrated the homozygous CPT II deficiency (c.338C>T, p.Ser113Leu) mutation. The diagnosis of CPT II deficiency helped this patient to put the symptoms into context, and this reduced myopathy and the risk of recurring rhabdomyolysis. We report on this patient to increase awareness of diagnostic and medical management in CPT II deficiency.
肉碱棕榈酰转移酶II(CPT II)缺乏症是一种线粒体长链脂肪酸氧化的遗传性缺陷。横纹肌溶解伴肌肉坏死是由骨骼肌破坏引起的,由于细胞内肌肉成分的释放,会导致全身多器官并发症。严重的横纹肌溶解可能由包括CPT II缺乏症在内的遗传易感性与其他诱发因素共同作用引发。一般来说,CPT II缺乏症患者很少得到临床诊断和报告。我们描述了一名因泌尿道感染引发严重横纹肌溶解的患者,该患者在基因检测中显示出纯合子CPT II缺乏症(c.338C>T,p.Ser113Leu)突变。CPT II缺乏症的诊断有助于该患者理解症状,从而减轻肌病并降低复发性横纹肌溶解的风险。我们报告该病例以提高对CPT II缺乏症诊断和医疗管理的认识。