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

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. 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. 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. This systematic review and meta-analysis found a substantial clinical improvement in visual acuity with idebenone therapy among patients with LHON.