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Efficacy of bezafibrate for preventing myopathic attacks in patients with very long-chain acyl-CoA dehydrogenase deficiency
Brain and Development ( IF 1.7 ) Pub Date : 2021-02-01 , DOI: 10.1016/j.braindev.2020.07.019
Hideaki Shiraishi 1 , Kenji Yamada 2 , Kiyoshi Egawa 1 , Mika Ishige 3 , Fumihiro Ochi 4 , Asami Watanabe 4 , Sanae Kawakami 5 , Kazuyo Kuzume 6 , Kenji Watanabe 7 , Koji Sameshima 7 , Kiyotaka Nakamagoe 8 , Akira Tamaoka 8 , Naoko Asahina 1 , Saki Yokoshiki 9 , Keiko Kobayashi 9 , Takashi Miyakoshi 9 , Koji Oba 10 , Toshiyuki Isoe 9 , Hiroshi Hayashi 9 , Seiji Yamaguchi 2 , Norihiro Sato 9
Affiliation  

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.

中文翻译:

苯扎贝特预防极长链酰基辅酶A脱氢酶缺乏症患者肌病发作的功效

背景极长链酰基辅酶A脱氢酶缺乏症(VLCADD)是一种线粒体脂肪酸氧化障碍,会导致间歇性发作,如全身乏力、肌张力减退、肌痛和横纹肌溶解伴随着能量缺乏。迄今为止,还没有针对这些 VLCADD 相关代谢发作的预防药物。患者和方法 我们对苯扎贝特对 5 名 VLCADD 患者的影响进行了一项开放标签、非随机、多中心研究。苯扎贝特给药 4 年,我们分析了需要住院和治疗输注的肌病发作次数。结果 在研究期间,需要输注 24 小时或更长时间的肌病发作次数显着减少。治疗也提高了患者进行日常活动的能力。
更新日期:2021-02-01
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