Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz
Gewählte Publikation:
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Valentin, K; Georgi, T; Riedl, R; Aminfar, H; Singer, C; Klopstock, T; Wedrich, A; Schneider, M.
Idebenone Treatment in Patients with OPA1-Dominant Optic Atrophy: A Prospective Phase 2 Trial
NEURO-OPHTHALMOLOGY. 2023;
Doi: 10.1080/01658107.2023.2251575
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- Führende Autor*innen der Med Uni Graz
- Valentin Katharina
- Co-Autor*innen der Med Uni Graz
- Aminfar Haleh
- Georgi Thomas Patrick
- Riedl Regina
- Schneider Mona Regina
- Singer Christoph
- Wedrich Andreas
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- Abstract:
- The aim of this study was to evaluate the therapeutic effect of idebenone in patients with OPA1-dominant optic atrophy (DOA). Sixteen patients with genetically confirmed OPA1-DOA were treated with 900 mg idebenone daily for 12 months. The primary endpoint was the best recovery/least deterioration of visual acuity. Secondary endpoints were the changes of visual acuity, colour vision, contrast sensitivity, visual field, peripapillary retinal nerve fibre layer thickness (pRNFLT), and visual-related quality of life. For the primary endpoint, a significant increase was observed for the right eye (p = .0027), for the left eye (p = .0111) and for the better-seeing eye (p = .0152). For visual fields, a significant improvement was observed for the left eye between baseline and 9 months (p = .0038). Regarding pRNFLT, a significant decrease was found for the left eye between baseline and 3 months (p = .0413) and between baseline and 6 months (p = .0448). In the visual function questionnaire, a significant improvement was observed in the subscale general vision (p = .0156) and in the composite score (p = .0256). In conclusion, best recovery of visual acuity improved, even though the amount of improvement was small. Furthermore, a maintenance of visual function after 12 months of idebenone intake could be observed as well as a significant improvement in vision-related quality of life.Whether this effect is due to idebenone treatment, the placebo effect, or is explainable by the natural progression of DOA, remains unclear. Trial registration: EU Clinical Trials Register, EudraCT Number: 2019-001493-28.
- Find related publications in this database (Keywords)
- Dominant optic atrophy
- OPA1
- idebenone
- optic neuropathy
- visual function