在为原发性线粒体疾病患者开具药物时,通常会寻求临床指导。有关线粒体疾病患者药物安全性的理论考虑可能导致在临床需要的情况下不必要地扣留药物。这项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。一个由16名线粒体医学专家组成的小组,药理学,建立了来自六个不同国家的基础科学。使用了改进的Delphi技术,使小组成员可以在具有预定协议级别的两轮Delphi中匿名考虑建议草案。这一进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。就所有46种审查药物的安全性达成了高度共识,知道不良事件的风险受个体患者风险因素和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,并提供了先前建立的原发性线粒体疾病患者安全用药指南的重要更新。特定药物,药物组,和临床或遗传条件分开描述,因为它们需要特别注意。重要的是要强调,基于共识的信息有助于提供指导,但是,与药物处方相关的决定应始终针对每个患者的特定需求和风险。我们的目标是介绍当前的知识,并计划定期更新,以包括新药和审查目前包括的药物。
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.