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Guenther Metz, Constanze Gallenmueller, Thomas Meier, Thomas Klopstock, Physicians participating in the Raxone Expanded Access Program; Clinical experience with Idebenone (Raxone®) in the treatment of patients with Leber’s Hereditary Optic Neuropathy (LHON). Invest. Ophthalmol. Vis. Sci. 2014;55(13):6206.
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An increasing body of evidence indicates that idebenone has therapeutic potential for the treatment of LHON. Data from a randomized placebo-controlled study (RHODOS) and from a number of case reports and retrospective cohort studies demonstrate that patients with established vision loss may benefit from idebenone treatment and recover visual acuity (VA). This study reports VA outcomes for LHON patients with recent onset of vision loss who received idebenone (Raxone) treatment under an Expanded Access Program (EAP).
LHON patients with recent onset of vision loss were enrolled in a global EAP between November 2011 and October 2013. Treating physicians who wished to enrol patients under Named Patient Programs (Europe, Australia, New Zealand) or treatment IND (USA) were provided with Raxone (idebenone 150 mg tablets). Physicians were requested to report any safety issues and were encouraged to also report VA outcomes in 3-monthly intervals for patients under treatment. Improvement in VA was defined as (i) improvement in VA by at least 10 letters on ETDRS chart or (ii) improvement from “off-chart” vision to being able to read at least 5 letters on the ETDRS chart.
There were 50 LHON patients enrolled in the ongoing EAP of which 42 patients had provided post treatment VA data at submission of the abstract. The average time to treatment from onset was 7 months and average treatment duration was 8 months (range: 3-18 months); patients had disease-typical demographics with respect to age at symptom onset (mean: 32 years), gender (72 % male), and mtDNA mutations (G11778A: 54%, G3460A: 20%, T14484C: 14%, other: 12%). Across all mutations, 19 of 42 (45 %) of patients experienced clinically meaningful VA recovery from nadir with the majority (74%) of these patients showing VA recovery within 6 months of treatment. Treatment with idebenone was safe and well tolerated
A high proportion of LHON patients treated with idebenone under a global EAP experienced a clinically meaningful recovery of vision, further demonstrating the therapeutic potential of idebenone in the treatment of LHON.
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