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

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A Correction to this article was published on 06 January 2021

This article has been updated

Abstract

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.

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Change history

  • 06 January 2021

    The original article can be found online.

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Acknowledgements

The authors thank Yoshiko Nakano for data acquisition. The authors thank Ryan Chastain-Gross, Ph.D. and Claire Barnes, Ph.D., from Edanz Group (https://en-author-services.edanzgroup.com/ac) for editing a draft of this manuscript.

Funding

This study received no financial support.

Author information

Authors and Affiliations

  1. Department of Ophthalmology, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, 663-8501, Japan

    Hiroto Ishikawa, Fumi Gomi & Osamu Mimura

  2. Department of Ophthalmology, The Jikei University School of Medicine, Nishi-Shinbashi, Minato-ku, Tokyo, Japan

    Yoichiro Masuda & Keigo Shikisima

  3. Department of Ophthalmology, Kitasato University School of Medicine, Sagamihara, Kanagawa, Japan

    Hitoshi Ishikawa & Toshiaki Goseki

  4. Department of Ophthalmology, Tokyo Medical University, Nishi-Shinjuku, Shinjuku-ku, Tokyo, Japan

    Takeshi Kezuka

  5. Research Institute for Time Studies, Yamaguchi University, Yamaguchi, Japan

    Masahiko Terao

  6. Brain Science Institute, Tamagawa University, Machida, Japan

    Atsushi Miyazaki & Kenji Matsumoto

  7. Center for Clinical Research and Education, Hyogo College of Medicine, Nishinomiya, Hyogo, Japan

    Hiroki Nishikawa

Authors
  1. Hiroto Ishikawa
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  2. Yoichiro Masuda
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  12. Osamu Mimura
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Corresponding author

Correspondence to Hiroto Ishikawa.

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Corresponding Author: Hiroto Ishikawa

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Cite this article

Ishikawa, H., Masuda, Y., Ishikawa, H. et al. Characteristics of Japanese patients with Leber’s hereditary optic neuropathy and idebenone trial: a prospective, interventional, non-comparative study. Jpn J Ophthalmol 65, 133–142 (2021). https://doi.org/10.1007/s10384-020-00789-2

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  • DOI: https://doi.org/10.1007/s10384-020-00789-2

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