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.

Morphological and Clinical Characterization of Foveal Bulge Sign Three Years After Retinal Detachment Repair: A Longitudinal Prospective Evaluation.

Purpose: To evaluate optical-coherence-tomography (OCT)-morphological and clinical parameters three years after primary rhegmatogenous retinal detachment (RRD) repair surgery and the role of postoperative foveal bulge sign. Methods: Of the 32 initially enrolled patients with primary RRD 20 (14 fovea-on, 6 fovea-off) completed this prospective study. Preoperatively and 3 years after surgery best corrected visual acuity (BCVA) and OCT including macula status, central retinal thickness (CRT), central choroidal thickness (CCT), thickness of each single retinal layer, integrity of cone-interdigitation-zone (CIZ) and ellipsoid zone (EZ), and presence of foveal bulge were evaluated. Results: Preoperatively fovea-off RRD patients show significantly thinner CCT, inner nuclear layer (INL) and inner plexiform layer (IPL) compared to fovea-on RRD patients, whereas only IPL and INL were significantly thicker compared to the fellow eye. Three years after surgery this thickening recovered. Final BCVA did not differ statistically significantly between fovea-off and fovea-on patients, no difference in CIZ-integrity or presence of foveal bulge was observed. Presence of foveal bulge at 3 years showed significantly better final BCVA and was associated with intact preoperative CIZ-integrity and postoperative EZ- and CIZ-integrity. The preoperative fovea status showed no correlation to the postoperative presence of foveal bulge. Conclusion: Three years after RRD repair retinal layers show similar thickness. The presence of foveal bulge is associated with better final BCVA. Sufficient pre- and postoperative CIZ-integrity as well as postoperative CIZ-integrity seem to be strongly associated with the restoration of foveal bulge. No correlation was found with the preoperative macular status or BCVA.

We analyzed long-term optical-coherence-tomography changes after rhegmatogenous retinal detachment repair. Three years after rhegmatogenous retinal detachment repair retinal layers show similar thickness and visual acuity did not differ between fovea off and fovea on patients. The restoration of foveal bulge sign was the most important factor for good postoperative visual acuity. Integrity of specific retinal layers, such as the ellipsoid zone and cone-interdigitation-zone, seems to be strongly associated with the restoration of the foveal bulge.