Journal Article

DZNE-2024-00303

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.

Yu-Wai-Man, P. ; Carelli, V. ; Newman, N. J. ; Silva, M. J. ; Linden, A. ; Van Stavern, G. ; Szaflik, J. P. ; Banik, R. ; Lubiński, W. ; Pemp, B. ; Liao, Y. J. ; Subramanian, P. S. ; Misiuk-Hojło, M. ; Newman, S. ; Castillo, L. ; Kocięcki, J. ; Levin, M. H. ; Muñoz-Negrete, F. J. ; Yagan, A. ; Cherninkova, S. ; Katz, D. ; Meunier, A. ; Votruba, M. ; Korwin, M. ; Dziedziak, J. ; Jurkutė, N. ; Harvey, J. P. ; La Morgia, C. ; Priglinger, C. ; Llòria, X. ; Tomasso, L. ; Klopstock, T. (Last author)DZNE* ; Group, L. S. (Collaboration Author)

2024
Elsevier Maryland Heights, MO

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Please use a persistent id in citations: doi:10.1016/j.xcrm.2024.101437

Abstract: Leber hereditary optic neuropathy (LHON) is a mitochondrial disease leading to rapid and severe bilateral vision loss. Idebenone has been shown to be effective in stabilizing and restoring vision in patients treated within 1 year of onset of vision loss. The open-label, international, multicenter, natural history-controlled LEROS study (ClinicalTrials.gov NCT02774005) assesses the efficacy and safety of idebenone treatment (900 mg/day) in patients with LHON up to 5 years after symptom onset (N = 199) and over a treatment period of 24 months, compared to an external natural history control cohort (N = 372), matched by time since symptom onset. LEROS meets its primary endpoint and confirms the long-term efficacy of idebenone in the subacute/dynamic and chronic phases; the treatment effect varies depending on disease phase and the causative mtDNA mutation. The findings of the LEROS study will help guide the clinical management of patients with LHON.

Keyword(s): Humans (MeSH) ; Optic Atrophy, Hereditary, Leber: drug therapy (MeSH) ; Optic Atrophy, Hereditary, Leber: genetics (MeSH) ; Optic Atrophy, Hereditary, Leber: diagnosis (MeSH) ; Antioxidants: therapeutic use (MeSH) ; Ubiquinone: therapeutic use (MeSH) ; Ubiquinone: genetics (MeSH) ; Ubiquinone: analogs & derivatives (MeSH) ; Mutation (MeSH) ; LHON ; Leber hereditary optic neuropathy ; idebenone ; mitochondrial disease ; mtDNA ; neuro-ophthalmology ; optic atrophy ; optic neuropathy ; retinal ganglion cells ; idebenone ; Antioxidants ; Ubiquinone

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Contributing Institute(s):

1. Clinical Research (Munich) (Clinical Research (Munich))

Research Program(s):

1. 353 - Clinical and Health Care Research (POF4-353) (POF4-353)

Appears in the scientific report 2024

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Linked articles:

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Journal Article (Editorial) Yu-Wai-Man, P. ; Newman, N. J. ; Poincenot, L. ; Ferguson, J. ; Klopstock, T. (Last author)DZNE*
Idebenone as a treatment for Leber hereditary optic neuropathy: A plain language summary of the LEROS study publication
Expert review of ophthalmology 20(6), 333 - 350 (2025) [10.1080/17469899.2025.2563196]2025   Files BibTeX | EndNote: XML, Text | RIS

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