TY  - JOUR
AU  - Yu-Wai-Man, Patrick
AU  - Carelli, Valerio
AU  - Newman, Nancy J
AU  - Silva, Magda Joana
AU  - Linden, Aki
AU  - Van Stavern, Gregory
AU  - Szaflik, Jacek P
AU  - Banik, Rudrani
AU  - Lubiński, Wojciech
AU  - Pemp, Berthold
AU  - Liao, Yaping Joyce
AU  - Subramanian, Prem S
AU  - Misiuk-Hojło, Marta
AU  - Newman, Steven
AU  - Castillo, Lorena
AU  - Kocięcki, Jarosław
AU  - Levin, Marc H
AU  - Muñoz-Negrete, Francisco Jose
AU  - Yagan, Ali
AU  - Cherninkova, Sylvia
AU  - Katz, David
AU  - Meunier, Audrey
AU  - Votruba, Marcela
AU  - Korwin, Magdalena
AU  - Dziedziak, Jacek
AU  - Jurkutė, Neringa
AU  - Harvey, Joshua P
AU  - La Morgia, Chiara
AU  - Priglinger, Claudia
AU  - Llòria, Xavier
AU  - Tomasso, Livia
AU  - Klopstock, Thomas
TI  - Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.
JO  - Cell reports / Medicine
VL  - 5
IS  - 3
SN  - 2666-3791
CY  - Maryland Heights, MO
PB  - Elsevier
M1  - DZNE-2024-00303
SP  - 101437
PY  - 2024
AB  - 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.
KW  - Humans
KW  - Optic Atrophy, Hereditary, Leber: drug therapy
KW  - Optic Atrophy, Hereditary, Leber: genetics
KW  - Optic Atrophy, Hereditary, Leber: diagnosis
KW  - Antioxidants: therapeutic use
KW  - Ubiquinone: therapeutic use
KW  - Ubiquinone: genetics
KW  - Ubiquinone: analogs & derivatives
KW  - Mutation
KW  - LHON (Other)
KW  - Leber hereditary optic neuropathy (Other)
KW  - idebenone (Other)
KW  - mitochondrial disease (Other)
KW  - mtDNA (Other)
KW  - neuro-ophthalmology (Other)
KW  - optic atrophy (Other)
KW  - optic neuropathy (Other)
KW  - retinal ganglion cells (Other)
KW  - idebenone (NLM Chemicals)
KW  - Antioxidants (NLM Chemicals)
KW  - Ubiquinone (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C2  - pmc:PMC10982982
C6  - pmid:38428428
DO  - DOI:10.1016/j.xcrm.2024.101437
UR  - https://pub.dzne.de/record/268724
ER  -