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Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial

Yu-Wai-Man, Patrick, Carelli, Valerio, Newman, Nancy J., Silva, Magda Joana, Linden, Aki, Van Stavern, Gregory, Szaflik, Jacek P., Banik, Rudrani, Lubinski, Wojciech, Pemp, Berthold, Liao, Yaping Joyce, Subramanian, Prem S., Misiuk-Hojto, Marta, Newman, Steven, Castillo, Lorena, Kociecki, Jaroslaw, Levin, Marc H., Muñoz-Negrete, Francisco Jose, Yagan, Ali, Cherninkova, Sylvia, Katz, David, Meunier, Audrey, Votruba, Marcela ORCID: https://orcid.org/0000-0002-7680-9135, Korwin, Magdalena, Dziedziak, Jacek, Jurkute, Neringa, Harvey, Joshua P., La Morgia, Chiara, Priglinger, Claudia, Llòria, Xavier, Tomasso, Livia and Klopstock, Thomas 2024. Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. Cell Reports Medicine 5 (3) , 101437. 10.1016/j.xcrm.2024.101437

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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.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Optometry and Vision Sciences
Publisher: Cell Press
ISSN: 2666-3791
Date of First Compliant Deposit: 5 March 2024
Date of Acceptance: 29 February 2024
Last Modified: 15 Apr 2024 12:52
URI: https://orca.cardiff.ac.uk/id/eprint/166887

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