Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial

Cell Rep Med. 2024 Mar 19;5(3):101437. doi: 10.1016/j.xcrm.2024.101437. Epub 2024 Feb 29.

Abstract

Leber hereditary optic neuropathy (LHON) is a mitochondrial disease leading to rapid and severe bilateral vision loss. Idebenone has been shown to be effective in stabilizing and restoring vision in patients treated within 1 year of onset of vision loss. The open-label, international, multicenter, natural history-controlled LEROS study (ClinicalTrials.gov NCT02774005) assesses the efficacy and safety of idebenone treatment (900 mg/day) in patients with LHON up to 5 years after symptom onset (N = 199) and over a treatment period of 24 months, compared to an external natural history control cohort (N = 372), matched by time since symptom onset. LEROS meets its primary endpoint and confirms the long-term efficacy of idebenone in the subacute/dynamic and chronic phases; the treatment effect varies depending on disease phase and the causative mtDNA mutation. The findings of the LEROS study will help guide the clinical management of patients with LHON.

Keywords: LHON; Leber hereditary optic neuropathy; idebenone; mitochondrial disease; mtDNA; neuro-ophthalmology; optic atrophy; optic neuropathy; retinal ganglion cells.

Publication types

  • Controlled Clinical Trial
  • Multicenter Study

MeSH terms

  • Antioxidants / therapeutic use
  • Humans
  • Mutation
  • Optic Atrophy, Hereditary, Leber* / diagnosis
  • Optic Atrophy, Hereditary, Leber* / drug therapy
  • Optic Atrophy, Hereditary, Leber* / genetics
  • Ubiquinone / analogs & derivatives*
  • Ubiquinone / genetics
  • Ubiquinone / therapeutic use

Substances

  • idebenone
  • Antioxidants
  • Ubiquinone

Associated data

  • ClinicalTrials.gov/NCT02774005