Efficacy of bezafibrate for preventing myopathic attacks in patients with very long-chain acyl-CoA dehydrogenase deficiency
Efficacy of bezafibrate for preventing myopathic attacks in patients with very long-chain acyl-CoA dehydrogenase deficiency
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DOI：
10.1016/j.braindev.2020.07.019
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发表时间：
2021-01-07
影响因子：
1.7
通讯作者：
Sato, Norihiro
中科院分区：
文献类型：
--
作者：
Shiraishi, Hideaki;Yamada, Kenji;Sato, Norihiro
背景资料：极长链酰基辅酶A脱氢酶缺乏症（VLCADD）是一种线粒体脂肪酸氧化障碍，可引起发作性发作，如全身疲劳、肌张力减退、肌痛和横纹肌溶解，伴有能量缺乏。到目前为止，还没有预防药物，这些VLCADD相关的代谢attacks.Patients和方法：我们进行了一项开放标签，非随机，多中心研究苯扎贝特对5例VLCADD患者的影响。苯扎贝特管理了4年，我们分析了需要住院和治疗infuses.Results的肌病发作的数量，需要输液的24小时或更长时间的肌病发作的数量显着下降，在研究期间。患者进行日常活动的能力也得到了改善的treatment.Conclusion：我们的研究结果显示潜在的长期疗效苯扎贝特在预防肌病发作VLCADD患者。(C)2020日本儿童神经病学学会。Elsevier B.V.出版，保留所有权利。
Background: Very long-chain acyl-CoA dehydrogenase deficiency (VLCADD) is a mitochondrial fatty acid oxidation disorder that causes episodic attacks, such as general fatigue, hypotonia, myalgia, and rhabdomyolysis accompanied by lack of energy. As yet, there are no preventative drugs for these VLCADD-associated metabolic attacks.Patients and methods: We conducted an open-label, non-randomized, multi-center study into the effects of bezafibrate on five patients with VLCADD. Bezafibrate was administered for 4 years, and we analyzed the number of myopathic attacks requiring hospitalization and treatment infusions.Results: The number of myopathic attacks requiring infusions of 24 h or longer significantly decreased during the study period. The patients' ability to conduct everyday activities was also improved by the treatment.Conclusion: Our findings show the potential long-term efficacy of bezafibrate in preventing myopathic attacks for patients with VLCADD. (C) 2020 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.