{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T23:35:30Z","timestamp":1793316930637,"version":"4.5.0"},"reference-count":22,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2022,4,8]],"date-time":"2022-04-08T00:00:00Z","timestamp":1649376000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007560","name":"Chiesi Farmaceutici","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007560","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Acta Ophthalmologica"],"published-print":{"date-parts":[[2022,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Purpose<\/jats:title><jats:p>The purpose of the study was to present results from a national Dutch cohort of patients with Leber's Hereditary Optic Neuropathy (LHON) treated with idebenone.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The multicentre, open\u2010label, retrospective evaluation of the long\u2010term outcome of idebenone treatment of Dutch LHON patients on visual function and on thickness of the retinal ganglion cell layer. Patients included in the analysis had a confirmed mutation in their mitochondrial DNA encoding either of the seven subunits of complex I, had a reported loss of vision in at least one eye and had a follow\u2010up of more than 6\u2009months after their treatment was started. Control visits involved routine clinical examinations of visual function and retinal structure at (1) the start of treatment, (2) nadir (time of lowest visual acuity), (3) the time of recovery (if any), (4) the time of termination of treatment and (5) more than 6\u2009months after termination of the treatment.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Data from 72 patients were analysed. Treatment duration was 23.8\u2009\u00b1\u200914.4 (mean\u2009\u00b1\u2009SD) months. A positive response, <jats:italic>that is<\/jats:italic> either a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS), occurred in 53% and 11% of the patients, respectively. The magnitude of CRR was 0.41\u2009\u00b1\u20091.54 logMAR. CRR of visual acuity is associated with recovery of colour discrimination. The thickness of both the ganglion cell complex (GCC) and the retinal nerve fibre layer (RNFL) is irreversibly reduced.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Our results confirm that idebenone may help to restore or maintain visual function. Whether this effect will persist is still unknown. Thinning of retinal neural tissue appears to be permanent.<\/jats:p><\/jats:sec>","DOI":"10.1111\/aos.15153","type":"journal-article","created":{"date-parts":[[2022,4,8]],"date-time":"2022-04-08T11:07:27Z","timestamp":1649416047000},"page":"700-706","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":28,"title":["Clinical outcomes of treatment with idebenone in Leber's hereditary optic neuropathy in the Netherlands: A national cohort study"],"prefix":"10.1111","volume":"100","author":[{"given":"Judith A. M.","family":"van Everdingen","sequence":"first","affiliation":[{"name":"Rotterdam Eye Hospital  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan Willem R.","family":"Pott","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, University Medical Center Groningen University of Groningen  Groningen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"No\u00ebl J. C.","family":"Bauer","sequence":"additional","affiliation":[{"name":"University Eye Clinic, Maastricht University Medical Center+  Maastricht The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna M.","family":"Krijnen","sequence":"additional","affiliation":[{"name":"Rotterdam Ophthalmic Institute  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tanya","family":"Lushchyk","sequence":"additional","affiliation":[{"name":"Rotterdam Eye Hospital  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0058-3643","authenticated-orcid":false,"given":"Ren\u00e9 J.","family":"Wubbels","sequence":"additional","affiliation":[{"name":"Rotterdam Ophthalmic Institute  Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,4,8]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2020.628014"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.preteyeres.2003.10.003"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1097\/WNO.0000000000000570"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1097\/WNO.0000000000001023"},{"key":"e_1_2_6_6_1","article-title":"Nieuwe therapie voor hereditaire opticusneuropathie van Leber. Dat vereist wel een snellere diagnose","volume":"165","author":"Everdingen JAM","year":"2021","journal-title":"Ned Tijdschr Geneeskd"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajo.2006.11.059"},{"key":"e_1_2_6_8_1","first-page":"1","article-title":"Optic neurodegeneration: Time to act","volume":"6","author":"Gueven N","year":"2014","journal-title":"Biol Med"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.3109\/01658107.2016.1173709"},{"key":"e_1_2_6_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0042-6989(97)00444-6"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ophtha.2017.01.002"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10384-020-00789-2"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awr170"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.mito.2019.10.002"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-016-0574-3"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1097\/WNO.0000000000001045"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2021.662838"},{"key":"e_1_2_6_18_1","unstructured":"Premarket notification: Canon OCT\u2010A1 Ophthalmoscope U.S. Food & Drug Administration July 24th 2019."},{"key":"e_1_2_6_19_1","first-page":"S261","article-title":"Natural history findings from a large cohort of patients with Lebers hereditary optic neuropathy (LHON): New insights into the natural disease course","volume":"96","author":"Silva M","year":"2018","journal-title":"Acta Ophthalmol"},{"key":"e_1_2_6_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajo.2005.11.007"},{"issue":"4","key":"e_1_2_6_21_1","first-page":"703","article-title":"Genetic and biochemical impairment of mitochondrial complex I activity in a family with Leber hereditary optic neuropathy and hereditary spastic dystonia","volume":"58","author":"Vries DD","year":"1996","journal-title":"Am J Hum Genet"},{"key":"e_1_2_6_22_1","doi-asserted-by":"publisher","DOI":"10.1136\/bjophthalmol-2020-316573"},{"key":"e_1_2_6_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41433-021-01535-9"}],"container-title":["Acta Ophthalmologica"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/aos.15153","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/aos.15153","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/aos.15153","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,23]],"date-time":"2023-08-23T00:24:54Z","timestamp":1692750294000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/aos.15153"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,4,8]]},"references-count":22,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2022,9]]}},"alternative-id":["10.1111\/aos.15153"],"URL":"https:\/\/doi.org\/10.1111\/aos.15153","archive":["Portico"],"relation":{},"ISSN":["1755-375X","1755-3768"],"issn-type":[{"value":"1755-375X","type":"print"},{"value":"1755-3768","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,4,8]]},"assertion":[{"value":"2021-12-03","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-03-30","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-04-08","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}