Persistence of treatment effect of idebenone in Leber's Hereditary Optic Neuropathy

Abstract

AbstractPurpose To establish the long term benefit of oral idebenone 900mg/day in the treatment of Leber's Hereditary Optic Neuropathy (LHON).Methods Patients who participated in a 24‐week, multi‐centre (3 sites), double‐masked, randomized, placebo controlled trial (RHODOS) were re‐assessed at a single visit by means of Visual Acuity (VA) using ETDRS charts.Results Eighty‐five patients were enrolled in the RHODOS study: 55 treated with idebenone (900mg/day) and 30 with placebo. At the end of the 24 week treatment period, the VA for patients randomized to placebo deteriorated. In contrast, in patients treated with 900mg/day idebenone, VA was preserved. In addition, in severely affected patients with off‐chart vision at Baseline, only idebenone treated patients improved sufficiently to read at least 1 full line on the ETDRS chart (Klopstock et al., 2011). VA was repeated at a follow‐up visit conducted 2.5 years (median) after treatment discontinuation. The difference in VA between placebo and idebenone treated patients was maintained. Specifically, in patients who during RHODOS received idebenone and who on average were protected from vision loss, VA did not deteriorate upon discontinuation of treatment.Conclusion These findings support the original conclusion that in selected patients with LHON, idebenone has significant therapeutic potential in preventing further vision loss and facilitating vision recovery. Commercial interest