BIOCHEMICAL RATIONALE AND THE CARDIAC RESPONSE OF PATIENTS WITH MUSCLE DISEASE TO THERAPY WITH COENZYME-Q10
BIOCHEMICAL RATIONALE AND THE CARDIAC RESPONSE OF PATIENTS WITH MUSCLE DISEASE TO THERAPY WITH COENZYME-Q10
复制标题
DOI：
10.1073/pnas.82.13.4513
复制
发表时间：
1985-01-01
影响因子：
11.1
通讯作者：
VADHANAVIKIT, S
中科院分区：
文献类型：
--
作者：
FOLKERS, K;WOLANIUK, J;VADHANAVIKIT, S
心脏病通常与几乎所有形式的肌营养不良症和肌病有关。对12例进展性肌营养不良和神经源性萎缩患者进行了口服辅酶Q10(CoQ10)的双盲开放交叉试验。这些疾病包括Duchenne、Becker和肢带营养不良、强直性肌营养不良、Charcot-Marie-Tooth病和Welander病。心阻抗图可无创、广泛监测心功能受损情况。仅根据每搏输出量和心输出量的显著变化或无变化，服用辅酶Q10的8名患者和服用安慰剂的4名患者均被正确分配(P<；0.003)。在有限的3个月之后。试验中，观察到4/8的接受治疗的患者和0/4的安慰剂患者的身体状况有所改善；后者中，3/4的患者在CoQ10的基础上有所改善；2/8的患者在交叉之前辞职；5/6的患者在交叉时保持改善的心功能；1/6的患者从CoQ10交叉到安慰剂复发。这项试验的基本原理是基于已知的线粒体肌病，其中涉及呼吸酶，已知呼吸中存在辅酶Q10，以及辅酶Q10和营养不良的先前临床数据。这类肌肉疾病患者的心肌功能受损可能与骨骼肌功能受损有关，辅酶Q10治疗可改善这两种功能。心脏的改善肯定是积极的。幸福感的改善是主观的，但可能是真实的。很可能，CoQ10不会改变遗传缺陷，但可以受益于这种缺陷造成的线粒体损伤的后遗症。辅酶Q10是唯一已知的物质，可以为这些患有肌肉疾病的患者提供安全和改善的生活质量，它是基于内在的生物能量学。
Cardiac disease is commonly associated with virtually every form of muscular dystrophy and myopathy. A double-blind and open crossover trial on the oral administration of coenzyme Q10 (CoQ10) to 12 patients with progressive muscular dystrophies and neurogenic atrophies was conducted. These diseases included the Duchenne, Becker and limb-girdle dystrophies, myotonic dystrophy, Charcot-Marie-Tooth disease and Welander disease. The impaired cardiac function was noninvasively and extensively monitored by impedance cardiography. Solely by significant change or no change in stroke volume and cardiac output, all 8 patients on blind CoQ10 and all 4 on blind placebo were correctly assigned (P < 0.003). After the limited 3-mo. trial, improved physical well-being was observed for 4/8 treated patients and for 0/4 placebo patients; of the latter, 3/4 improved on CoQ10; 2/8 patients resigned before crossover; 5/6 on CoQ10 in crossover maintained improved cardiac function; 1/6 crossed over from CoQ10 to placebo relapsed. The rationale of this trial was based on known mitochondrial myopathies, which involve respiratory enzymes, the known presence of CoQ10 in respiration, and prior clinical data on CoQ10 and dystrophy. The impaired myocardial function of such patients with muscular disease may have some association with impaired function of skeletal muscle, both of which may be improved by CoQ10 therapy. The cardiac improvement was definitely positive. The improvement in well-being was subjective, but probably real. Likely, CoQ10 does not alter genetic defects but can benefit the sequelae of mitochondrial impairment from such defects. CoQ10 is the only known substance that offers a safe and improved quality of life for such patients having muscle disease, and it is based on intrinsic bioenergetics.