Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy : The LEROS nonrandomized controlled trial
Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved..
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.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2024 |
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| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:5 |
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| Enthalten in: |
Cell reports. Medicine - 5(2024), 3 vom: 19. März, Seite 101437 |
| Sprache: |
Englisch |
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| Links: |
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| Anmerkungen: |
Date Completed 22.03.2024 Date Revised 03.04.2024 published: Print-Electronic ClinicalTrials.gov: NCT02774005 Citation Status MEDLINE |
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| doi: |
10.1016/j.xcrm.2024.101437 |
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| PPN (Katalog-ID): |
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| 100 | 1 | |a Yu-Wai-Man, Patrick |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy |b The LEROS nonrandomized controlled trial |
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| 500 | |a Date Completed 22.03.2024 | ||
| 500 | |a Date Revised 03.04.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a ClinicalTrials.gov: NCT02774005 | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved. | ||
| 520 | |a 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 | ||
| 650 | 4 | |a Controlled Clinical Trial | |
| 650 | 4 | |a Multicenter Study | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a LHON | |
| 650 | 4 | |a Leber hereditary optic neuropathy | |
| 650 | 4 | |a idebenone | |
| 650 | 4 | |a mitochondrial disease | |
| 650 | 4 | |a mtDNA | |
| 650 | 4 | |a neuro-ophthalmology | |
| 650 | 4 | |a optic atrophy | |
| 650 | 4 | |a optic neuropathy | |
| 650 | 4 | |a retinal ganglion cells | |
| 650 | 7 | |a idebenone |2 NLM | |
| 650 | 7 | |a HB6PN45W4J |2 NLM | |
| 650 | 7 | |a Antioxidants |2 NLM | |
| 650 | 7 | |a Ubiquinone |2 NLM | |
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| 700 | 1 | |a Carelli, Valerio |e verfasserin |4 aut | |
| 700 | 1 | |a Newman, Nancy J |e verfasserin |4 aut | |
| 700 | 1 | |a Silva, Magda Joana |e verfasserin |4 aut | |
| 700 | 1 | |a Linden, Aki |e verfasserin |4 aut | |
| 700 | 1 | |a Van Stavern, Gregory |e verfasserin |4 aut | |
| 700 | 1 | |a Szaflik, Jacek P |e verfasserin |4 aut | |
| 700 | 1 | |a Banik, Rudrani |e verfasserin |4 aut | |
| 700 | 1 | |a Lubiński, Wojciech |e verfasserin |4 aut | |
| 700 | 1 | |a Pemp, Berthold |e verfasserin |4 aut | |
| 700 | 1 | |a Liao, Yaping Joyce |e verfasserin |4 aut | |
| 700 | 1 | |a Subramanian, Prem S |e verfasserin |4 aut | |
| 700 | 1 | |a Misiuk-Hojło, Marta |e verfasserin |4 aut | |
| 700 | 1 | |a Newman, Steven |e verfasserin |4 aut | |
| 700 | 1 | |a Castillo, Lorena |e verfasserin |4 aut | |
| 700 | 1 | |a Kocięcki, Jarosław |e verfasserin |4 aut | |
| 700 | 1 | |a Levin, Marc H |e verfasserin |4 aut | |
| 700 | 1 | |a Muñoz-Negrete, Francisco Jose |e verfasserin |4 aut | |
| 700 | 1 | |a Yagan, Ali |e verfasserin |4 aut | |
| 700 | 1 | |a Cherninkova, Sylvia |e verfasserin |4 aut | |
| 700 | 1 | |a Katz, David |e verfasserin |4 aut | |
| 700 | 1 | |a Meunier, Audrey |e verfasserin |4 aut | |
| 700 | 1 | |a Votruba, Marcela |e verfasserin |4 aut | |
| 700 | 1 | |a Korwin, Magdalena |e verfasserin |4 aut | |
| 700 | 1 | |a Dziedziak, Jacek |e verfasserin |4 aut | |
| 700 | 1 | |a Jurkutė, Neringa |e verfasserin |4 aut | |
| 700 | 1 | |a Harvey, Joshua P |e verfasserin |4 aut | |
| 700 | 1 | |a La Morgia, Chiara |e verfasserin |4 aut | |
| 700 | 1 | |a Priglinger, Claudia |e verfasserin |4 aut | |
| 700 | 1 | |a Llòria, Xavier |e verfasserin |4 aut | |
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| 700 | 0 | |a LEROS Study Group |e verfasserin |4 aut | |
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