L eber hereditary optic neuropathy (LHON) is a form of blindness due to retinal ganglion cell dysfunction (1), caused by mutations in mitochondrial DNA (mtDNA), which affect complex I (NADH-ubiquinone oxidoreductase) of the mitochondrial respiratory chain (2,3). Although rare (estimated prevalence of 1 in 27,000-45,000), it affects all ages and gender, causing rapid and severe, bilateral (usually sequential), painless loss of central vision (4-7). Spon
Due to the high comparability of the patient collectives an increase of RVO during and 4 weeks after the 2014 football World Cup could be identified, compared to the same period in 2013. It can be assumed that emotional strain caused by a World Cup is a risk factor. Data from other centers have to be assessed in order to be able to make a more general statement.
Purpose LHON is an orphan mitochondrial disorder affecting the retinal ganglion cells leading to permanent blindness from which recovery is rare. Here we report visual acuity outcomes for patients with recent onset who received Raxone® (idebenone) under an ongoing global Expanded Access Program (EAP). Methods Visual acuity was measured in 3‐monthly intervals. Clinically relevant recovery was defined as (i) improvement from nadir by at least 10 letters on the ETDRS chart or (ii) improvement from “off‐chart” at nadir to being able to read at least 5 letters on‐chart. Results Currently there are 61 LHON patients enrolled, of which 48 patients have been treated for an average of 11 months. So far, 24 of 48 patients (50%) have experienced a clinically relevant and stable recovery in VA (89% for T14484C, 70% for G3460A and 31% for G11778A). The average treatment effect size in patients with recovery was 29 letters and 84 % recovered within 12 months of the initiation of the EAP. Conclusion A high proportion of LHON patients treated with idebenone under a global EAP experienced a clinically meaningful recovery of vision, further demonstrating the therapeutic potential of idebenone in the treatment of LHON. Commercial interest
PurposeLHON is an orphan mitochondrial disorder affecting the retinal ganglion cells leading to permanent blindness from which recovery is rare. An increasing body of evidence indicates that idebenone has therapeutic potential for the treatment of LHON. Data from a randomized placebo‐controlled study (RHODOS), from a number of case reports and retrospective cohort studies demonstrate that patients with established vision loss may benefit from idebenone treatment and recover visual acuity (VA).This study reports VA outcomes for patients with recent onset of vision loss who received idebenone treatment under an ongoing global Expanded Access Program (EAP) where currently 82 patients are enrolled and have been treated for an average of 15 months. The outcomes will be compared with findings of the RHODOS study and a Case Record Survey collecting VA data from untreated patients.MethodsAnalyses are performed to assess recovery from the VA nadir. Clinically relevant recovery was defined as (i) improvement from nadir by at least 10 letters on the ETDRS chart or (ii) improvement from “off‐chart” at nadir to being able to read at least 5 letters on‐chart. Furthermore, the prevention of vision loss for patients with residual vision below 1.0 logMAR (20/200) at start of therapy was analysed.ResultsA high proportion of patients (about 50% at submission of the abstract) treated with idebenone under this global EAP experienced a clinically meaningful recovery of vision. In addition, in patients with residual vision below 1.0 logMAR at start of therapy, loss of VA to above this level could be prevented in a large number of patients (about 60% at submission of the abstract).ConclusionsThe therapeutic potential of idebenone in the treatment of LHON is further demonstrated by the clinical experience in a large cohort of patients under a global EAP.
PurposeLHON is a mitochondrial disorder leading to progressive, irreversible loss of visual acuity (VA). Here we report on the long term treatment outcomes in real world clinical practice in which we evaluated the efficacy and safety of idebenone.MethodsPatients were treated with idebenone as part of an Expanded Access Program. Efficacy was assessed as a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS). CRR defined as improvement from off-chart to reading 5 letters on the ETDRS chart, or improvement of 10 letters. CRS is defined as maintenance of VA <1.0 logMAR (20/200).ResultsData from 111 patients was collected.The majority of patients (72.4%) had severe vision loss (>1.0 logMAR). A subset of 24 patients presented with VA <1.0 logMAR, and 50% of these experienced a CRS. CRR at last observation was achieved in 47.1% of patients after a mean of 10 months. For patients achieving CRR, 90.2% (37/41) had done so by 24 months. Mean of magnitude of CRR at last observation was more than seven lines on the ETDRS chart. The safety profile of idebenone in this longer term analysis was consistent with previous reports.ConclusionsIdebenone is well tolerated and effective in a large proportion of patients. Maximal patient benefit may be achieved by early initiation of treatment, and by prolonged treatment.
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