左旋肉碱治疗原发性肉碱缺乏症的临床研究进展  

Clinical Research Progress of L-carnitine in the Treatment of Primary Carnitine Deficiency

作　　者：李娴 于飞[2] LI Xian;YU Fei(Training Base of Maternal and Child Health Hospital of Hubei Province,Hubei University of Medicine,Wuhan 430070,China;Department of Child Endocrinology and Genetic Metabolism,Maternal and Child Health Hospital of Hubei Province,Wuhan 430070,China)

机构地区：[1]湖北医药学院湖北省妇幼保健院培养基地,武汉430070 [2]湖北省妇幼保健院儿童内分泌与遗传代谢科,武汉430070

出　　处：《医药导报》2024年第3期402-407,共6页Herald of Medicine

摘　　要：左旋肉碱是协助长链脂肪酸转运至线粒体内的载体,是治疗原发性肉碱缺乏症(PCD)的有效药物,在发生不可逆转的器官损害之前,尽早开始补充能可逆性改善患者脏器损害,降低猝死风险,提高生活质量。然而,近年来,有研究提出长期口服左旋肉碱有潜在风险,左旋肉碱经肠道菌群代谢产生氧化三甲胺有增加心血管疾病的风险。因此,该文对左旋肉碱治疗PCD的临床研究进展进行总结,为临床应用左旋肉碱治疗PCD提供参考。L-carnitine is a carrier that assist in transport of long-chain fatty acids into mitochondria and an effective drug for treating primary carnitine deficiency(PCD).Starting supplementation as early as possible before irreversible organ damage occured can alleviate organ damage,reduce the risk of sudden death,and improve the quality of life.However,in recent years,some studies have suggested that long-term oral L-carnitine has potential risks,the metabolism of L-carnitine through intestinal flora results in the production of trimethylamine oxide(TMAO),which can increase the risk of cardiovascular disease(CVD).Therefore,this paper summarizes the clinical research progress of L-carnitine in treating PCD to provide a theoretical basis for the clinical application of L-carnitine in PCD.

关 键 词：左旋肉碱 原发性肉碱缺乏症 氧化三甲胺 

分 类 号：R985[医药卫生—药品] R725.8[医药卫生—药学]