Therapeutic Effects of Idebenone on Leber Hereditary Optic Neuropathy.

: To characterize the potential therapeutic effects of idebenone on Leber hereditary optic neuropathy (LHON) in terms of visual acuity (VA), visual field (VF) defects, visual evoked potential (VEP) and retinal nerve fibre layer (RNFL) thickness using optical coherence tomography (OCT) measurements. : This was a retrospective case-controlled study of the effect of idebenone (900 mg/d) on 30 patients with LHON due to m.3460 G > A, m.11778 G > A and m.14484 T > C mutations. The primary end-point was the recovery in VA after 3 and 6 mon. The main secondary end-point was the change in VF, VEP and RNFL thickness. The other secondary end-point was the correlation between visual changes after 6 mon and the VF, VEP and RNFL thickness at baseline of the groups. : Idebenone was shown to be safe and well tolerated. The primary end-point reached statistical significance. The VA in the idebenone group improved in both the best eye and worst eye. The mean defect of VF decreased and amplitude of VEP increased. There was no significant difference in latency and RNFL thickness between the groups. The treatment, together with the VA and amplitude at baseline, had a significant effect on the improvement in VA at 6 mon. : This case-controlled study of LHON provides evidence that idebenone treatment may be beneficial in cases of LHON and that the influential factors governing outcomes are the VA and amplitude of the VEP at baseline.