Purpose LHON is a mitochondrial disorder leading to progressive, irreversible loss of visual acuity (VA). Here we report on the long term treatment outcomes in real world clinical practice in which we evaluated the efficacy and safety of idebenone.
Methods Patients were treated with idebenone as part of an Expanded Access Program. Efficacy was assessed as a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS). CRR defined as improvement from off-chart to reading 5 letters on the ETDRS chart, or improvement of 10 letters. CRS is defined as maintenance of VA <1.0 logMAR (20/200).
Results Data from 111 patients was collected.The majority of patients (72.4%) had severe vision loss (>1.0 logMAR). A subset of 24 patients presented with VA <1.0 logMAR, and 50% of these experienced a CRS. CRR at last observation was achieved in 47.1% of patients after a mean of 10 months. For patients achieving CRR, 90.2% (37/41) had done so by 24 months. Mean of magnitude of CRR at last observation was more than seven lines on the ETDRS chart. The safety profile of idebenone in this longer term analysis was consistent with previous reports.
Conclusions Idebenone is well tolerated and effective in a large proportion of patients. Maximal patient benefit may be achieved by early initiation of treatment, and by prolonged treatment.
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