Toxic medications in Leber's hereditary optic neuropathy

Mitochondrion. 2019 May;46:270-277. doi: 10.1016/j.mito.2018.07.007. Epub 2018 Aug 4.


Leber's hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disorder characterized by acute bilateral vision loss. The pathophysiology involves reactive oxygen species (ROS), which can be affected by medications. This article reviews the evidence for medications with demonstrated and theoretical effects on mitochondrial function, specifically in relation to increased ROS production. The data reviewed provides guidance when selecting medications for individuals with LHON mutations (carriers) and are susceptible to conversion to affected. However, as with all medications, the proven benefits of these therapies must be weighed against, in some cases, purely theoretical risks for this unique patient population.

Keywords: Antimicrobials; Leber's hereditary optic neuropathy; Medications; Mitochondrial toxicity.

Publication types

  • Review

MeSH terms

  • Drug-Related Side Effects and Adverse Reactions*
  • Humans
  • Optic Atrophy, Hereditary, Leber / pathology*
  • Reactive Oxygen Species / metabolism*
  • Reactive Oxygen Species / toxicity*


  • Reactive Oxygen Species