Therapeutic Effects of Idebenone on Leber Hereditary Optic Neuropathy

Source
Curr Eye Res, 2020 Oct, DOI: 10.1080/02713683.2020.1736307

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.

Conclusion: This case-controlled study of LHON provides evidence that idebenone treatment may be beneficial

The study included 30 patients with LHON caused by the primary mutations m.3460G>A, m.11778G>A, or m.14484T>C. Patients received oral idebenone at 900 mg/day. Researchers assessed visual acuity (VA), visual field (VF) defects, visual evoked potentials (VEP), and retinal nerve fibre layer (RNFL) thickness measured by optical coherence tomography (OCT).

The primary endpoint was recovery in visual acuity after 3 and 6 months of treatment. Secondary endpoints included changes in VF, VEP parameters, and RNFL thickness, as well as correlations between baseline measures and visual outcomes at 6 months.

Idebenone was safe and well tolerated. The primary endpoint reached statistical significance. Visual acuity improved in both the best-seeing and worst-seeing eyes in the treated group.
Additional findings included:

• A decrease in the mean defect of the visual field
• An increase in VEP amplitude
• No significant differences between groups in VEP latency or RNFL thickness
• Baseline visual acuity and VEP amplitude, together with treatment, significantly influenced the degree of visual acuity improvement at 6 months.

This case-controlled study provides evidence that idebenone treatment can be beneficial for patients with LHON. The results also highlight that patients with better baseline visual acuity and higher VEP amplitude may experience greater gains from therapy. These factors can help clinicians set realistic expectations and identify candidates most likely to respond.

Overall, the findings support the continued use of idebenone as a therapeutic option for LHON while underscoring the value of early functional assessments in predicting treatment response.