Therapeutic Benefit of Idebenone in Leber Hereditary Optic Neuropathy: A Systematic Review and Meta-Analysis
Source
Ophthalmic Genet., 2025 Jul 13, DOI: 10.1080/13816810.2025.2521647
Leber hereditary optic neuropathy (LHON) is a rare mitochondrial disorder that causes rapid, severe bilateral vision loss. Current treatment options remain limited. Idebenone, a synthetic analogue of coenzyme Q10 with neuroprotective properties, has emerged as a potential therapy. This systematic review and meta-analysis evaluated its efficacy in improving visual acuity in patients with LHON.
Study Overview and Methods
Researchers included five studies—three clinical trials and two retrospective cohorts—comprising a total of 375 patients. The analysis focused on changes in visual acuity measured by the LogMAR scale, comparing idebenone treatment with outcomes in patients who did not receive the drug.
Key Efficacy Findings
The pooled analysis demonstrated a statistically significant improvement with idebenone. The overall mean LogMAR difference was -0.32 (95% CI: -0.50 to -0.15) in favor of idebenone. This corresponds to a clinically meaningful gain of approximately 1.5 to 5 lines of better visual performance on the LogMAR chart.
Clinical Significance
These findings indicate that idebenone provides substantial improvement in visual acuity for patients with LHON. The consistent benefit across the included studies supports its role as a disease-modifying option in this challenging condition.
Conclusion and Implications
This systematic review and meta-analysis confirms a clear therapeutic benefit of idebenone for visual outcomes in LHON. The results strengthen the evidence base for its clinical use and may help guide treatment decisions for patients affected by this mitochondrial optic neuropathy.