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

LEROS Study Group; Patrick Yu-Wai-Man; Valerio Carelli; Nancy J. Newman; Magda Joana Silva; Aki Linden; Gregory Van Stavern; Jacek P. Szaflik; Rudrani Banik; Wojciech Lubiński; Berthold Pemp; Yaping Joyce Liao; Prem S. Subramanian; Marta Misiuk-Hojło; Steven Newman; Lorena Castillo; Jarosław Kocięcki; Marc H. Levin; Francisco Jose Muñoz-Negrete; Ali Yagan; Sylvia Cherninkova; David Katz; Audrey Meunier; Marcela Votruba; Magdalena Korwin; Jacek Dziedziak; Neringa Jurkutė; Joshua P. Harvey; Chiara La Morgia; Claudia Priglinger; Xavier Llòria; Livia Tomasso; Thomas Klopstock

doi:10.1016/j.xcrm.2024.101437

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

LEROS Study Group, Patrick Yu-Wai-Man, Valerio Carelli, Nancy J. Newman, Magda Joana Silva, Aki Linden, Gregory Van Stavern, Jacek P. Szaflik, Rudrani Banik, Wojciech Lubiński, Berthold Pemp, Yaping Joyce Liao, Prem S. Subramanian, Marta Misiuk-Hojło, Steven Newman, Lorena Castillo, Jarosław Kocięcki, Marc H. Levin, Francisco Jose Muñoz-Negrete, Ali YaganSylvia Cherninkova, David Katz, Audrey Meunier, Marcela Votruba, Magdalena Korwin, Jacek Dziedziak, Neringa Jurkutė, Joshua P. Harvey, Chiara La Morgia, Claudia Priglinger, Xavier Llòria, Livia Tomasso, Thomas Klopstock

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18 Scopus citations

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.

Original language	English
Article number	101437
Journal	Cell Reports Medicine
Volume	5
Issue number	3
DOIs	https://doi.org/10.1016/j.xcrm.2024.101437
State	Published - Mar 19 2024

Keywords

LHON
Leber hereditary optic neuropathy
idebenone
mitochondrial disease
mtDNA
neuro-ophthalmology
optic atrophy
optic neuropathy
retinal ganglion cells

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10.1016/j.xcrm.2024.101437

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LEROS Study Group, Yu-Wai-Man, P., Carelli, V., Newman, N. J., Silva, M. J., Linden, A., Van Stavern, G., Szaflik, J. P., Banik, R., Lubiński, W., Pemp, B., Liao, Y. J., Subramanian, P. S., Misiuk-Hojło, M., Newman, S., Castillo, L., Kocięcki, J., Levin, M. H., Muñoz-Negrete, F. J., ... Klopstock, T. (2024). Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial. Cell Reports Medicine, 5(3), Article 101437. https://doi.org/10.1016/j.xcrm.2024.101437

@article{7ca43d8680ae403c9b22a9047c56d06d,

title = "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.",

keywords = "LHON, Leber hereditary optic neuropathy, idebenone, mitochondrial disease, mtDNA, neuro-ophthalmology, optic atrophy, optic neuropathy, retinal ganglion cells",

author = "{LEROS Study Group} and Patrick Yu-Wai-Man and Valerio Carelli and Newman, {Nancy J.} and Silva, {Magda Joana} and Aki Linden and {Van Stavern}, Gregory and Szaflik, {Jacek P.} and Rudrani Banik and Wojciech Lubi{\'n}ski and Berthold Pemp and Liao, {Yaping Joyce} and Subramanian, {Prem S.} and Marta Misiuk-Hoj{\l}o and Steven Newman and Lorena Castillo and Jaros{\l}aw Koci{\c e}cki and Levin, {Marc H.} and Mu{\~n}oz-Negrete, {Francisco Jose} and Ali Yagan and Sylvia Cherninkova and David Katz and Audrey Meunier and Marcela Votruba and Magdalena Korwin and Jacek Dziedziak and Neringa Jurkutė and Harvey, {Joshua P.} and {La Morgia}, Chiara and Claudia Priglinger and Xavier Ll{\`o}ria and Livia Tomasso and Thomas Klopstock",

note = "Publisher Copyright: {\textcopyright} 2024 The Authors",

year = "2024",

month = mar,

day = "19",

doi = "10.1016/j.xcrm.2024.101437",

language = "English",

volume = "5",

journal = "Cell Reports Medicine",

issn = "2666-3791",

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LEROS Study Group, Yu-Wai-Man, P, Carelli, V, Newman, NJ, Silva, MJ, Linden, A, Van Stavern, G, Szaflik, JP, Banik, R, Lubiński, W, Pemp, B, Liao, YJ, Subramanian, PS, Misiuk-Hojło, M, Newman, S, Castillo, L, Kocięcki, J, Levin, MH, Muñoz-Negrete, FJ, Yagan, A, Cherninkova, S, Katz, D, Meunier, A, Votruba, M, Korwin, M, Dziedziak, J, Jurkutė, N, Harvey, JP, La Morgia, C, Priglinger, C, Llòria, X, Tomasso, L & Klopstock, T 2024, 'Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial', Cell Reports Medicine, vol. 5, no. 3, 101437. https://doi.org/10.1016/j.xcrm.2024.101437

TY - JOUR

T1 - Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy

T2 - The LEROS nonrandomized controlled trial

AU - LEROS Study Group

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

PY - 2024/3/19

Y1 - 2024/3/19

N2 - 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.

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 - LHON

KW - Leber hereditary optic neuropathy

KW - idebenone

KW - mitochondrial disease

KW - mtDNA

KW - neuro-ophthalmology

KW - optic atrophy

KW - optic neuropathy

KW - retinal ganglion cells

UR - http://www.scopus.com/inward/record.url?scp=85186340511&partnerID=8YFLogxK

U2 - 10.1016/j.xcrm.2024.101437

DO - 10.1016/j.xcrm.2024.101437

M3 - Article

C2 - 38428428

AN - SCOPUS:85186340511

SN - 2666-3791

VL - 5

JO - Cell Reports Medicine

JF - Cell Reports Medicine

IS - 3

M1 - 101437

ER -

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

Abstract

Keywords

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