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-Hojlo, Marta; Newman, Steven; Castillo, Lorena; Kociecki, Jaroslaw; Levin, Marc H.; Munoz-Negrete, Francisco Jose; Yagan, Ali; Cherninkova, Sylvia; Katz, David; Meunier, Audrey; Votruba, Marcela; Korwin, Magdalena; Dziedziak, Jacek; Jurkute, Neringa; Harvey, Joshua P.; La Morgia, Chiara; Priglinger, Claudia; Lloria, Xavier; Tomasso, Livia und Klopstock, Thomas (2024): Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. In: Cell Reports Medicine, Bd. 5, Nr. 3, 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.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin > Munich Cluster for Systems Neurology (SyNergy)
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• Sprache: Englisch
• DFG: Gefördert durch die Deutsche Forschungsgemeinschaft (DFG) - 390857198
• DFG: Gefördert durch die Deutsche Forschungsgemeinschaft (DFG) - 491502892