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

par Yu-Wai-Man, Patrick;Carelli, Valerio;Newman, Nancy N.J.;Silva, Magda Joana;Linden, Aki;Van Stavern, Gregory G.P.;Szaflik, Jacek Paweł;Banik, Rudrani;Lubiński, Wojciech;Pemp, Berthold;Liao, Yaping Joyce;Subramanian, Prem P.S.;Misiuk-Hojło, Marta;Newman, Steven;Castillo, Lorena;Kocięcki, Jarosław;Levin, Marc M.H.;Muñoz-Negrete, Francisco Jose;Yagan, Ali;Cherninkova, Sylvia;Katz, David;Meunier, Audrey ;Votruba, Marcela;Korwin, Magdalena;Dziedziak, Jacek;Jurkute, Neringa;Harvey, Joshua Paul;La Morgia, Chiara;Priglinger, Claudia;Llòria, Xavier;Tomasso, Livia;Klopstock, Thomas
Référence Cell Reports Medicine, 5, 3, 101437
Publication Publié, 2024-03

Article révisé par les pairs

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Résumé :

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.