The authors present a meta-analysis of studies reporting the effects of idebenone treatment on visual acuity (VA) in patients with Leber hereditary optic neuropathy (LHON), the most common inherited mitochondrial disorder. They included randomised controlled trials, non-randomised controlled trials and cohort studies of idebenone with or without multivitamin treatment. Included studies were quality assessed by 2 authors using published quality assessment tools. 5 studies met the inclusion criteria for this meta-analysis. Of these, 3 were considered to have moderate risk of bias and 2 considered to have low risk of bias. Pooled mean difference in VA between idebenone treated and comparator groups was -0.32 (p<0.001), indicating a beneficial effect of idebenone therapy. The limitations of this analysis were the relatively small sample size of 375 patients across the 5 included studies, and the variations in treatment protocol across these studies. In August of 2025, idebenone was recommended by NICE for the treatment of LHON in patients 12 years and older. This was despite limited evidence of efficacy in clinical trials. This meta-analysis goes some way towards demonstrating an improvement in visual acuity from this treatment.
