Medical treatments in patients with mitochondrial disease: an observational cohort study

Abstract

Mitochondrial diseases are common inherited neurometabolic disorders. They can cause symptoms in various organs. In these situations, medication treatment is usually only able to address the symptoms, not the underlying mitochondrial dysfunction. Moreover, many common drugs have harmful effects on mitochondria and patients with mitochondrial disease may be even more sensitive to these side effects. Unfortunately, published data on the medications used in patients with mitochondrial disease is surprisingly scarce. In this retrospective register-based study, a cohort of 44 patients with mitochondrial disease was reviewed for the medications used. Medication data were obtained for the years 2010-2022 and categorized according to the Anatomical Therapeutic Chemical (ATC) classification. In addition, potential drug-drug interactions and potential adverse drug reactions were analyzed from the patients' current medications. The use of clearly harmful medications or those with severe drug-drug interactions was not observed among these patients with mitochondrial disease. However, the use of pain medication and benzodiazepines was considerably high in this patient group and warrants more detailed study. Overall, drug safety in patients with mitochondrial disease requires further research and increased awareness in the future.