Real-world clinical experience with Idebenone in the treatment of Leber hereditary optic neuropathy

Background: Leber hereditary optic neuropathy (LHON) leads to bilateral central vision loss. In a clinical trial setting, idebenone has been shown to be safe and to provide a trend toward improved visual acuity, but long-term evidence of effectiveness in real-world clinical practice is sparse. Methods: Open-label, multicenter, retrospective, noncontrolled analysis of long-term visual acuity and safety in 111 LHON patients treated with idebenone (900 mg/day) in an expanded access program. Eligible patients had a confirmed mitochondrial DNA mutation and had experienced the onset of symptoms (most recent eye) within 1 year before enrollment. Data on visual acuity and adverse events were collected as per normal clinical practice. Efficacy was assessed as the proportion of patients with either a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS) of visual acuity. In the case of CRR, time to and magnitude of recovery over the course of time were also assessed. Results: At time of analysis, 87 patients had provided longitudinal efficacy data. Average treatment duration was 25.6 months. CRR was observed in 46.0% of patients. Analysis of treatment effect by duration showed that the proportion of patients with recovery and the magnitude of recovery increased with treatment duration. Average gain in best-corrected visual acuity for responders was 0.72 logarithm of the minimal angle of resolution (logMAR), equivalent to more than 7 lines on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Furthermore, 50% of patients who had a visual acuity below 1.0 logMAR in at least one eye at initiation of treatment successfully maintained their vision below this threshold by last observation. Idebenone was well tolerated, with most adverse events classified as minor. Conclusions: These data demonstrate the benefit of idebenone treatment in recovering lost vision and maintaining good residual vision in a real-world setting. Together, these findings indicate that idebenone treatment should be initiated early and be maintained more than 24 months to maximize efficacy. Safety results were consistent with the known safety profile of idebenone

Expanded Access Program (EAP) in Leber's Hereditary Optic Neuropathy (LHON) Patients Treated for 24 Months (Video)

LHON is a mitochondrial genetic disorder resulting in severe bilateral central VA loss. Three primary mitochondrial DNA mutations cause over 90% of cases. Data from idebenone EAP for patients with LHON receiving treatment for at least 24 months were analyzed to assess rate of clinically-relevant recovery (CRR)

Long-term experience from an Expanded Access Program with idebenone in pediatric LHON patients

LHON results in bilateral, severe central vision loss, and is caused by mitochondrial DNA mutations. LHON is typically diagnosed between 15 - 30 years of age, although it can be detected earlier. Idebenone, the only approved treatment for LHON in Europe, has been shown to be efficacious and safe in a large proportion of adult patients, but pediatric data is limited. The Expanded Access Program (EAP, a named patient program under local regulations) provides insights into the potential of idebenone in pediatric LHON

Long Term Treatment with Idebenone in Leber's Hereditary Optic Neuropathy (LHON): Real World Clinical Practice

LHON is caused by mitochondrial DNA (mtDNA) mutations, resulting in progressive bilateral, severe central blindness. Idebenone (900 mg/day) is approved for use in LHON in the EU . We report long-term treatment outcomes in real-world clinical practice from a multicenter Expanded Access Program