Therapeutic effects of Idebenone in Japanese patients with Leber hereditary optic neuropathy: A multicenter prospective study

Abstract

Purpose : To verify the therapeutic effects of Idebenone in Japanese LHON patients.

Methods : This was a multicenter, prospective, single arm, non-randomized and open-label confirmatory study. Between October 2013 and May 2016, a total of 57 LHON patients (52 males, 5 females; mean age: 28.9 ± 17.7 years) were enrolled (4 patients dropped out during the follow-up period). Study participants received a supplement containing Idebenone at 900mg/day for 24 weeks. Ophthalmological examinations including best collected visual acuity (BCVA), visual field (VF), critical fusion frequency (CFF), and retinal nerve fiber layer thickness (RNFLT), were performed every 2 months, and blood analysis was performed every 6 months. In 4 patients, we performed exploratory examinations by fMRI to assess brain activities before and after Idebenone therapy.

Results : Mitochondrial mutation 11778 G>A was found in 94.7% of the patients. No patients showed any clinically significant adverse events. Among the patients who showed improvements in BCVA at 12M, significant improvement was observed in 32.1%. Also, for VF and CFF, significant improvements were observed in 24.5% and 34.0% respectively. In all eyes, RNFLT showed significant decreases at 12M. In fMRI, 1 of 4 patients who had clinical efficacy for Idebenone showed improvements.

Conclusions : The therapeutic effects of Idebenone with a favorable safety profile were confirmed in Japanese LHON patients in this study.

This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.