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

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.

SUBMITTER: Yu-Wai-Man P 

PROVIDER: S-EPMC10982982 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature

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

Yu-Wai-Man Patrick P   Carelli Valerio V   Newman Nancy J NJ   Silva Magda Joana MJ   Linden Aki A   Van Stavern Gregory G   Szaflik Jacek P JP   Banik Rudrani R   Lubiński Wojciech W   Pemp Berthold B   Liao Yaping Joyce YJ   Subramanian Prem S PS   Misiuk-Hojło Marta M   Newman Steven S   Castillo Lorena L   Kocięcki Jarosław J   Levin Marc H MH   Muñoz-Negrete Francisco Jose FJ   Yagan Ali A   Cherninkova Sylvia S   Katz David D   Meunier Audrey A   Votruba Marcela M   Korwin Magdalena M   Dziedziak Jacek J   Jurkutė Neringa N   Harvey Joshua P JP   La Morgia Chiara C   Priglinger Claudia C   Llòria Xavier X   Tomasso Livia L   Klopstock Thomas T  

Cell reports. Medicine 20240229 3

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 =  ...[more]

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