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.

Reproducibility of retinal nerve fiber layer thickness measurements using the eye tracker and the retest function of Spectralis SD-OCT in glaucomatous and healthy control eyes.

PURPOSE: To evaluate the impact of Spectralis self-acting eye-tracking (eye tracker) and retest software on the reproducibility of retinal nerve fiber layer (RNFL) thickness measurements in glaucomatous and healthy control eyes by SD-OCT. METHODS: RNFL thickness was measured in 56 normal and 47 glaucomatous eyes by one operator within one session with a brief rest between measurements. Three measurements were taken with the eye tracker and the retest function engaged (method A), and three measurements were taken without the eye tracker and without the retest function (method B). Method A and B measurements were taken alternately. RESULTS: Reliability, measured by intraclass correlation coefficient (ICC) for absolute agreement and coefficient of variation (COV), was calculated for the global mean RNFL thickness (G), for each sector and for the peripapillary bundle. The ICC (and lower 95% confidence interval [CI]) for the global mean RNFL thickness (G) for method A measurements in both normal and glaucomatous eyes was 0.99 (0.98 CI). In glaucomatous eyes, the COV for method B measurements was between 2.7% and 10.5%, and between 1.3% and 3.5% for method A measurements. CONCLUSIONS: The reproducibility of RNFL measurements with Spectralis SD-OCT is excellent in both normal and glaucomatous eyes and can be significantly improved by using the eye tracker and retest software. The gain of reproducibility by using the software is significantly higher in glaucomatous eyes than in normal eyes. These findings suggest that software applications are capable of significantly improving the reproducibility of RNFL thickness measurements.