Safety of drug use in patients with a primary mitochondrial disease: An international Delphi-based consensu

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 withhold-
ing 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 consen-
sus conference that included the panellists and representatives of patient advo-
cacy 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 use-
ful 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.
K E Y W O R D S
drugs, in vitro studies, in vivo studies, mitochondrial diseases, mitochondrial toxicity, safety