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: | 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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