Characteristics of Japanese patients with Leber's hereditary optic neuropathy and idebenone trial: a prospective, interventional, non-comparative study.

Autor: Ishikawa H; Department of Ophthalmology, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan. ohmyeye@gmail.com., Masuda Y; Department of Ophthalmology, The Jikei University School of Medicine, Nishi-Shinbashi, Minato-ku, Tokyo, Japan., Ishikawa H; Department of Ophthalmology, Kitasato University School of Medicine, Sagamihara, Kanagawa, Japan., Shikisima K; Department of Ophthalmology, The Jikei University School of Medicine, Nishi-Shinbashi, Minato-ku, Tokyo, Japan., Goseki T; Department of Ophthalmology, Kitasato University School of Medicine, Sagamihara, Kanagawa, Japan., Kezuka T; Department of Ophthalmology, Tokyo Medical University, Nishi-Shinjuku, Shinjuku-ku, Tokyo, Japan., Terao M; Research Institute for Time Studies, Yamaguchi University, Yamaguchi, Japan., Miyazaki A; Brain Science Institute, Tamagawa University, Machida, Japan., Matsumoto K; Brain Science Institute, Tamagawa University, Machida, Japan., Nishikawa H; Center for Clinical Research and Education, Hyogo College of Medicine, Nishinomiya, Hyogo, Japan., Gomi F; Department of Ophthalmology, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan., Mimura O; Department of Ophthalmology, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan.
Jazyk: angličtina
Zdroj: Japanese journal of ophthalmology [Jpn J Ophthalmol] 2021 Jan; Vol. 65 (1), pp. 133-142. Date of Electronic Publication: 2020 Nov 13.
DOI: 10.1007/s10384-020-00789-2
Abstrakt: Purpose: Leber's hereditary optic neuropathy (LHON) is a mitochondrial neuropathy that causes acute vision loss. Idebenone, a short-chain ubiquinone analog that preserves mitochondrial function is thought to suppress disease progression in early-onset LHON patients. We investigated the effects of idebenone in Japanese LHON patients.
Study Design: Prospective, interventional, non-comparative study in patients with definite LHON diagnosis, under trial registration number UMIN000017939.
Methods: Fifty-seven patients received 900 mg/day idebenone for 24 weeks. We measured baseline best-corrected visual acuity, visual fields, critical fusion frequency and retinal ganglion cell layer complex thickness; we assessed efficacy at 24 and 48 weeks, and safety throughout.
Results: Patients were predominantly male (91.2%) and most had an mt.11778G>A mutation (94.7%). All patients tolerated idebenone therapy well. Data from the 51 mt.11778 patients were compared with their baseline data. At 48 weeks, significant improvement in best-corrected visual acuity was observed in 17 patients (33.3%). Furthermore, 25.5% of patients showed improvements in visual fields and 33.3% in critical fusion frequency. However, retinal ganglion cell layer complex thickness was significantly reduced. Among patients who started idebenone >1 year after disease onset, visual improvement was found in 12 (38.7%). Among patients who developed LHON before 19 years of age, visual improvement was found in 11 (42.3%).
Conclusion: Idebenone's potential and favorable safety profile were confirmed in Japanese LHON patients. However, this study had no placebo group; therefore, we need to undertake a prospective intervention study to further investigate the therapeutic effects of Idebenone in Japanese LHON patients.
Databáze: MEDLINE
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