Mitochondrial diseases are predominantly caused by mutations of mitochondrial or nuclear DNA, resulting in multisystem defects. Current treatments are largely supportive, and the disorders progress relentlessly. Nutritional supplements, pharmacological agents and physical therapies have been used in different clinical trials, but the efficacy of these interventions need to be further evaluated. Several recent reviews discussed some of the interventions but ignored bias in those trials. This review was conducted to discover new studies and grade the original studies for potential bias with revised Cochrane Collaboration guidelines. We focused on seven published studies and three unpublished studies; eight of these studies showed improvement in outcome measurements. In particular, two of the interventions have been tested in studies with strict design, which we believe deserve further clinical trials with a large sample. Additionally, allotopic expression of the ND4 subunit seemed to be an effective new treatment for patients with Leber hereditary optic neuropathy.

中文翻译：

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线粒体疾病的治疗。


线粒体疾病主要由线粒体或核 DNA 突变引起，导致多系统缺陷。目前的治疗主要是支持性的，并且疾病不断进展。营养补充剂、药物制剂和物理疗法已用于不同的临床试验，但这些干预措施的疗效需要进一步评估。最近的几篇评论讨论了一些干预措施，但忽略了这些试验中的偏见。进行本次审查的目的是发现新的研究，并根据修订后的 Cochrane 协作指南对原始研究的潜在偏差进行评级。我们重点关注七项已发表的研究和三项未发表的研究；其中八项研究显示结果测量有所改善。特别是，其中两种干预措施已经在严格设计的研究中进行了测试，我们认为值得进一步进行大样本的临床试验。此外，ND4亚基的同位素表达似乎是治疗莱伯遗传性视神经病患者的有效新疗法。