April 2009
Volume 50, Issue 13
Free
ARVO Annual Meeting Abstract  |   April 2009
Idebenone Treatment in Patients With Leber Hereditary Optic Neuropathy
Author Affiliations & Notes
  • J. G. Eng
    Department of Ophthalmology, University of South Carolina, Columbia, South Carolina
    Doheny Eye Institute/USC Keck School of Medicine, Los Angeles, California
  • D. Aggarwal
    Doheny Eye Institute/USC Keck School of Medicine, Los Angeles, California
  • A. A. Sadun
    Doheny Eye Institute/USC Keck School of Medicine, Los Angeles, California
  • Footnotes
    Commercial Relationships  J.G. Eng, None; D. Aggarwal, None; A.A. Sadun, None.
  • Footnotes
    Support  International Foundation for Optic Nerve Disease, Research to Prevent Blindness
Investigative Ophthalmology & Visual Science April 2009, Vol.50, 1440. doi:
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    • Get Citation

      J. G. Eng, D. Aggarwal, A. A. Sadun; Idebenone Treatment in Patients With Leber Hereditary Optic Neuropathy. Invest. Ophthalmol. Vis. Sci. 2009;50(13):1440.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose: : To examine whether patients with Leber Hereditary Optic Neuropathy (LHON) may show improvement in vision after starting idebenone, a synthetic analog of Coenzyme Q10.

Methods: : 7 patients (6 males and 1 female) who took idebenone for LHON were selected for this study. Four patients had the 11778 mutation; one patient had the 3460 mutation; one patient had the 14484 mutation; the remaining patient had no mutation identified. These patients had visited the neuro-ophthalmology clinic at Doheny Eye Institute from 2003 to 2008. Charts were reviewed and evaluated for any improvement in visual acuity (VA), color vision, and Humphrey visual field after initiation of idebenone therapy.

Results: : Four patients experienced no improvement in visual acuity, color vision, or visual fields after taking idebenone. Three patients demonstrated a dramatic or marked improvement. A 50 year old female with the 11778 mutation had VA of 20/200 OU when starting idebenone 450 mg/day. VA improved to 20/50 OU in one month. When VA decreased to 20/400 OD and CF 2 ft OS three months later, idebenone was increased to 540 mg/day. At a follow-up visit six months later, VA had improved to 20/30 OD and 20/60 OS and visual fields showed complete clearing of central scotomas. Her color vision also improved from discerning 0/12 color plates OU prior to taking idebenone to 2/12 color plates OU. A 16 year old male with the 14484 mutation had progressive visual loss with VA 4/200 OD and 5/200 OS, bilateral central visual field defects, and color vision of 0/8 OD and 3/8 OS using AO test plates. He was started on idebenone 360 mg/day and over the course of 1.5 years, VA improved to 20/40 OD and 20/60 OS. Visual fields showed improvement in both eyes with a decrease in central depression OD and only a patchy central defect with preserved vision nasally OS. Patient could discern 8/14 plates OD and 6/14 plates OS. A 17 year old male with the 11778 mutation had VA of CF 4 ft OD and 20/400 OS and color vision of 0/8 plates OD and 4/8 plates OS. He was started on idebenone 360 mg/day and after two months, he experienced a modest improvement in VA OD to 20/200 with VA of 20/400 OS. He could also discern 9/20 OD and 8/20 OS H-R-R plates after 2 months of idebenone. This patient also experienced an improvement in his visual field.

Conclusions: : Some patients with LHON treated with idebenone showed marked improvement in visual acuity, color vision, and visual fields. Although prospective studies are needed to determine the role of idebenone in visual recovery, the relatively safe side effect profile of idebenone makes it a treatment option for many patients diagnosed with LHON.

Keywords: optic nerve • antioxidants • mitochondria 
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