Dominant optic atrophy (DOA) is a disease of the retinal ganglion cells, with no current treatment options. In most cases, DOA is caused by a mutation in the OPA1 gene. The aim of this study was to evaluate the effect of idebenone treatment in patients with OPA1 DOA. Sixteen patients with OPA1-DOA were treated with 900mg idebenone daily for one year. The primary endpoint was the best recovery / least deterioration of visual acuity. Secondary endpoints were the changes of visual acuity, colour vision, contrast sensitivity, visual field, peripapillary retinal nerve fibre layer thickness (pRNFLT), and visual-related quality of life. For the primary endpoint, a significant increase was observed for the right eye (p=0.0027), the left eye (p=0.0111) and for the better-seeing eye (p=0.0152). For visual fields, a significant improvement was observed for the left eye between baseline and nine months (p=0.0038). Regarding pRNFLT, a significant decrease was found for the left eye between baseline and three months (p=0.0413) and between baseline and six months (p=0.0448). In the visual function questionnaire, a significant improvement was observed in the subscale general vision (p=0.0156) and in the composite score (p=0.0256). In conclusion, best recovery of visual acuity improved, although the amount of improvement was minimal. Furthermore, a maintenance of visual function after 12 months of idebenone intake could be observed as well as a significant improvement in vision-related quality of life. Further research with a control group is required to determine whether this effect is due to idebenone treatment, the placebo effect, or is explainable by the natural progression of DOA. The authors recommend a randomised, placebo-controlled, and double-blind study over a longer time period to explore further evidence.
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