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Xavier Lloria, Magda Silva, Guenther Rudolph, Felice Lob, Bettina von Livonius, Claudia Catarino, Thomas Klopstock; Maintenance of Idebenone Treatment after an Initial Visual Recovery Results in Continued Improvement in LHON Patients. Invest. Ophthalmol. Vis. Sci. 2018;59(9):3361.
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© ARVO (1962-2015); The Authors (2016-present)
LHON is a mitochondrial (mt) disorder affecting the retinal ganglion cells and leading to progressive, irreversible bilateral loss of visual acuity (VA). Over 90% of European and North American patients harbor one of three primary pathogenic mtDNA mutations. To date, the only treatment approved for patients with LHON is idebenone (150 mg tablets), which at a dose of 900 mg/day, has been shown to be effective and safe. Here we report on the long term treatment outcomes in real world clinical practice in which we evaluated the efficacy and safety of idebenone in LHON using data from a multi-center Expanded Access Program (EAP).
Patients with a confirmed diagnosis of LHON (based on genetic testing) were treated with idebenone under Named Patient Regulations as part of an EAP. Patients were followed up in accordance with routine clinical practice, and safety and VA data was collected at each visit. Efficacy was assessed as a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS). CRR is 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).
Data from 111 patients was collected. 87 patients (82% male) carried one of the three major LHON mutations, were within 1 year of disease onset and provided post-baseline (BL) VA data. Median age onset at BL, mean time since symptoms onset and mean treatment duration at last observation were 31.4 years, 4.6 months and 23.8 months, respectively. 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 with some eyes occurring later than 30 months post-BL. Mean of magnitude of CRR at last observation was more than seven lines on the ETDRS chart, far above the minimum threshold of 10 letters. The safety profile of idebenone in this longer term analysis was consistent with previous reports.
Idebenone is well tolerated and effective in a large proportion of patients, both in preventing VA loss, and in recovering lost VA. Maximal patient benefit may be achieved by early initiation of treatment, and by prolonged treatment.
This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.
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