Therapeutic benefit of idebenone in Leber hereditary optic neuropathy: a systematic review and meta-analysis

source extracted confidence: high 2026-04-09
#humans #optic-atrophy-hereditary-leber #ubiquinone #visual-acuity #antioxidants

Therapeutic benefit of idebenone in Leber hereditary optic neuropathy: a systematic review and meta-analysis

Ribeiro et al., 2025 | Ophthalmic Genet | Meta Analysis

Citation

Ribeiro Paulo Victor Zattar, Pari Mitre Lucas, ... Gomy Israel. Therapeutic benefit of idebenone in Leber hereditary optic neuropathy: a systematic review and meta-analysis. Ophthalmic Genet. 2025-Dec;46(6):517-522. doi:10.1080/13816810.2025.2521647

Abstract

INTRODUCTION: Leber hereditary optic neuropathy (LHON) is a mitochondrial disorder characterized by the rapid loss of vision due to the degeneration of retinal ganglion cells. Current therapeutic options are limited. However, idebenone, a synthetic analog of coenzyme Q10, has shown promising neuroprotective properties. This systematic review and meta-analysis aim to evaluate the efficacy of idebenone in improving visual acuity in patients with LHON. METHODS: We performed a systematic review on PubMed, Cochrane, and Embase databases on 7 July 2024, for randomized and non-randomized studies analyzing the effect of idebenone on visual acuity in patients with LHON. Analyses were conducted using random-effects models. The main outcome of interest was visual acuity measured by the Logarithm of the Minimum Angle of Resolution (LogMAR). All statistical analyses were performed in R software version 4.3.2, using the "meta" and "metafor" packages. We registered the study on PROSPERO under protocol number CRD42024617308. RESULTS: Five studies (3 clinical trials and 2 retrospective cohorts) with 375 patients were included. The overall mean LogMAR difference was -0.32 (95% CI: -0.50 to -0.15), with a favorable effect of idebenone as compared to treatment without idebenone. This indicates a meaningful improvement in visual acuity with idebenone therapy, with changes translating to approximately 1.5-5 lines of better visual performance on the LogMAR scale. CONCLUSION: This systematic review and meta-analysis found a substantial clinical improvement in visual acuity with idebenone therapy among patients with LHON.

Key Findings

Five studies (3 clinical trials and 2 retrospective cohorts) with 375 patients were included. The overall mean LogMAR difference was -0.32 (95% CI: -0.50 to -0.15), with a favorable effect of idebenone as compared to treatment without idebenone. This indicates a meaningful improvement in visual acuity with idebenone therapy, with changes translating to approximately 1.5-5 lines of better visual performance on the LogMAR scale.

Outcomes Measured

  • Requires manual extraction

Population

Field Value
Population lhon
Sample Size 375
Age Range See abstract
Condition See abstract

MeSH Terms

  • Humans
  • Optic Atrophy, Hereditary, Leber
  • Ubiquinone
  • Visual Acuity
  • Antioxidants

Evidence Classification

  • Level: Meta Analysis
  • Publication Types: Journal Article, Systematic Review, Meta-Analysis
  • Vertical: coq10

Provenance

Source extracted via PubMed E-utilities API on 2026-04-09