Clinical guidance is often sought when prescribing drugs for patients with primary mitochondrial disease. Theoretical considerations concerning drug safety in patients with mitochondrial disease may lead to unnecessary withholding of a drug in a situation of clinical need. The aim of this study was to develop consensus on safe medication use in patients with a primary mitochondrial disease. A panel of 16 experts in mitochondrial medicine, pharmacology, and basic science from six different countries was established. A modified Delphi technique was used to allow the panellists to consider draft recommendations anonymously in two Delphi rounds with predetermined levels of agreement. This process was supported by a review of the available literature and a consensus conference that included the panellists and representatives of patient advocacy groups. A high level of consensus was reached regarding the safety of all 46 reviewed drugs, with the knowledge that the risk of adverse events is influenced both by individual patient risk factors and choice of drug or drug class. This paper details the consensus guidelines of an expert panel and provides an important update of previously established guidelines in safe medication use in patients with primary mitochondrial disease. Specific drugs, drug groups, and clinical or genetic conditions are described separately as they require special attention. It is important to emphasise that consensus‐based information is useful to provide guidance, but that decisions related to drug prescribing should always be tailored to the specific needs and risks of each individual patient. We aim to present what is current knowledge and plan to update this regularly both to include new drugs and to review those currently included.

中文翻译：

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原发性线粒体疾病患者用药的安全性：基于德尔菲的国际共识。

在为原发性线粒体疾病患者开药时，通常会寻求临床指导。关于线粒体疾病患者药物安全性的理论考虑可能会导致在临床需要的情况下不必要地扣留药物。本研究的目的是就原发性线粒体疾病患者的安全用药达成共识。成立了一个由来自六个不同国家的 16 名线粒体医学、药理学和基础科学专家组成的专家小组。修改后的德尔菲技术被用来允许小组成员在两轮德尔菲中匿名考虑建议草案，并达到预定的一致程度。这一过程得到了对现有文献的审查和包括小组成员和患者倡导团体代表在内的共识会议的支持。关于所有 46 种审查药物的安全性达成了高度共识，因为不良事件的风险受到患者个体风险因素以及药物或药物类别选择的影响。本文详细介绍了专家小组的共识指南，并对先前制定的原发性线粒体疾病患者安全用药指南进行了重要更新。特定药物、药物组以及临床或遗传状况单独描述，因为它们需要特别注意。需要强调的是，基于共识的信息有助于提供指导，但与药物处方相关的决策应始终根据每个患者的具体需求和风险进行调整。我们的目标是介绍当前的知识，并计划定期更新，以包括新药物并审查当前包括的药物。