Changes in Visual Function and Correlations with Inner Retinal Structure in Acute and Chronic Leber's Hereditary Optic Neuropathy Patients after Treatment with Idebenone.

Progressive impairment and degeneration of retinal ganglion cells (RGC) and nerve fibers in Leber's hereditary optic neuropathy (LHON) usually cause permanent visual loss. Idebenone is currently the only approved treatment. However, its therapeutic potential in different stages of LHON has not been definitely clarified. We aimed to investigate the changes in visual function and correlations with retinal structure in acute and in chronic LHON patients after treatment with idebenone. Twenty-three genetically confirmed LHON patients were followed during treatment using logMAR charts, automated perimetry and optical coherence tomography (OCT). Mean visual acuity improved significantly in acute patients treated within 1 year from onset (-0.52 ± 0.46 logMAR from nadir), in early chronic patients who started after 1-5 years (-0.39 ± 0.27 logMAR from baseline), and in late chronic patients with treatment initiation after >5 years (-0.33 ± 0.28 logMAR from baseline, < 0.001 all groups). In acute and in chronic patients, strong correlations between OCT and visual function parameters were present only after treatment. This and the sustained visual recovery after treatment may indicate a reactivated signal transduction in dysfunctional RGC that survive the acute phase. Our results support previous evidence that idebenone has therapeutic potential in promoting visual recovery in LHON.