贝特类药物治疗骨骼肌脂肪酸氧化障碍：一项随机临床试验。
Bezafibrate in skeletal muscle fatty acid oxidation disorders: a randomized clinical trial.
机构信息
From the Neuromuscular Clinic and Research Unit (M.C.Ø, K.L.M., N.P., G.A., J.V.), Department of Neurology, Rigshospitalet, University of Copenhagen, Denmark; and Centre de Référence de pathologie neuromusculaire Paris-Est (P.L.), Groupe Hospitalier Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris, France.
出版信息
Neurology. 2014 Feb 18;82(7):607-13. doi: 10.1212/WNL.0000000000000118. Epub 2014 Jan 22.
OBJECTIVE
To assess whether bezafibrate increases fatty acid oxidation (FAO) and lowers heart rate (HR) during exercise in patients with carnitine palmitoyltransferase (CPT) II and very long-chain acyl-CoA dehydrogenase (VLCAD) deficiencies.
METHODS
This was a 3-month, randomized, double-blind, crossover study of bezafibrate in patients with CPT II (n = 5) and VLCAD (n = 5) deficiencies. Primary outcome measures were changes in FAO, measured with stable-isotope methodology and indirect calorimetry, and changes in HR during exercise.
RESULTS
Bezafibrate lowered low-density lipoprotein, triglyceride, and free fatty acid concentrations; however, there were no changes in palmitate oxidation, FAO, or HR during exercise.
CONCLUSION
Bezafibrate does not improve clinical symptoms or FAO during exercise in patients with CPT II and VLCAD deficiencies. These findings indicate that previous in vitro studies suggesting a therapeutic potential for fibrates in disorders of FAO do not translate into clinically meaningful effects in vivo.
CLASSIFICATION OF EVIDENCE
This study provides Class I evidence that bezafibrate 200 mg 3 times daily is ineffective in improving changes in FAO and HR during exercise in adults with CPT II and VLCAD deficiencies.
目的
评估贝特类药物是否能增加肉碱棕榈酰基转移酶（CPT）Ⅱ和极长链酰基辅酶 A 脱氢酶（VLCAD）缺乏症患者运动时的脂肪酸氧化（FAO）并降低心率（HR）。
方法
这是一项为期 3 个月、随机、双盲、交叉的贝特类药物治疗 CPT II（n = 5）和 VLCAD（n = 5）缺乏症患者的研究。主要观察指标是使用稳定同位素法和间接测热法测量的 FAO 变化以及运动时 HR 的变化。
结果
贝特类药物降低了低密度脂蛋白、甘油三酯和游离脂肪酸浓度；然而，在运动期间，棕榈酸氧化、FAO 或 HR 没有变化。
结论
贝特类药物不能改善 CPT II 和 VLCAD 缺乏症患者运动时的临床症状或 FAO。这些发现表明，先前体外研究表明，贝特类药物在 FAO 紊乱中具有治疗潜力，但并不能转化为体内有临床意义的效果。
证据分类
本研究提供了 I 级证据，表明每日 3 次服用 200 毫克贝特类药物不能有效改善 CPT II 和 VLCAD 缺乏症成人运动时 FAO 和 HR 的变化。
Zotero 插件