Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies
Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual parameters over the first year after vision loss. Methods: RESCU...
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| Veröffentlicht in: | Journal of neuro-ophthalmology 2021-09, Vol.41 (3), p.298-308 |
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| creator | Moster, Mark L. Sergott, Robert C. Newman, Nancy J. Yu-Wai-Man, Patrick Carelli, Valerio Bryan, Molly Scannell Smits, Gerard Biousse, Valérie Vignal-Clermont, Catherine Klopstock, Thomas Sadun, Alfredo A. DeBusk, Adam A. Carbonelli, Michele Hage, Rabih Priglinger, Siegfried Karanjia, Rustum Blouin, Laure Taiel, Magali Katz, Barrett Sahel, José Alain |
| description | Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual parameters over the first year after vision loss. Methods: RESCUE and REVERSE were 2 phase III clinical trials designed to assess the efficacy of rAAV2/2-ND4 gene therapy in ND4-LHON subjects. At enrollment, subjects had vision loss for |
| doi_str_mv | 10.1097/WNO.0000000000001316 |
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| fullrecord | <record><control><sourceid>proquest_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8366757</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2555637517</sourcerecordid><originalsourceid>FETCH-LOGICAL-c4534-112f589d0858391872a97e5dc95bed2c27ed302dd60088bd9cca2984dfc503e53</originalsourceid><addsrcrecordid>eNqNUt1u0zAYjRCIjcEbIORLJJThnzhObpC2ULZJVYvabVxarv2FeqRxsR2mvgDPjaOOMrjCF_Znfecc-_g4y14TfEpwLd5_mc1P8aNBGCmfZMeEszLnpCyfphoLnFeY0qPsRQh3CcQwrZ9nR6xgBBdlcZz9bLwLIV-Cjtb1qkNnadoFG5Br0bkK0Nke0K0NQ-p9Vl5tIIIPyPZoOazuEi2gBWg_2AgGXfXRobgGtJgsm5sJUr1J5e1ksZyg2ccin17OZ-gCkuL1Grza7tAyDsZCeJk9a1UX4NXDepLdfJpcN5f5dH5x1ZxNc11wVuSE0JZXtcEVr1hNKkFVLYAbXfMVGKqpAJMsGlNiXFUrU2utaF0VptUcM-DsJPuw190Oqw0YDX30qpNbbzfK76RTVv7d6e1afnU_ZMXKUnCRBN4-CHj3fYAQ5cYGDV2nenBDkJRzXjLByQgt9lA9PrGH9nAMwXKMUKYI5b8RJtqbx1c8kH5nlgDv9oB7WLk2aAu9hgMsyQhCKiz4KEgTuvp_dGOjGv9B44Y-_jFw77ox9G_dcA9erkF1cT1SC8KoyCmmyU3a5qOHgv0C-TvHEw</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2555637517</pqid></control><display><type>article</type><title>Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies</title><source>MEDLINE</source><source>Journals@Ovid LWW Legacy Archive</source><source>Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals</source><source>Web of Science - Science Citation Index Expanded - 2021<img src="https://exlibris-pub.s3.amazonaws.com/fromwos-v2.jpg" /></source><creator>Moster, Mark L. ; Sergott, Robert C. ; Newman, Nancy J. ; Yu-Wai-Man, Patrick ; Carelli, Valerio ; Bryan, Molly Scannell ; Smits, Gerard ; Biousse, Valérie ; Vignal-Clermont, Catherine ; Klopstock, Thomas ; Sadun, Alfredo A. ; DeBusk, Adam A. ; Carbonelli, Michele ; Hage, Rabih ; Priglinger, Siegfried ; Karanjia, Rustum ; Blouin, Laure ; Taiel, Magali ; Katz, Barrett ; Sahel, José Alain</creator><creatorcontrib>Moster, Mark L. ; Sergott, Robert C. ; Newman, Nancy J. ; Yu-Wai-Man, Patrick ; Carelli, Valerio ; Bryan, Molly Scannell ; Smits, Gerard ; Biousse, Valérie ; Vignal-Clermont, Catherine ; Klopstock, Thomas ; Sadun, Alfredo A. ; DeBusk, Adam A. ; Carbonelli, Michele ; Hage, Rabih ; Priglinger, Siegfried ; Karanjia, Rustum ; Blouin, Laure ; Taiel, Magali ; Katz, Barrett ; Sahel, José Alain ; LHON Study Grp ; LHON study group ; for the LHON study group</creatorcontrib><description>Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual parameters over the first year after vision loss. Methods: RESCUE and REVERSE were 2 phase III clinical trials designed to assess the efficacy of rAAV2/2-ND4 gene therapy in ND4-LHON subjects. At enrollment, subjects had vision loss for <= 6 months in RESCUE, and between 6 and 12 months in REVERSE. Functional visual parameters (best-corrected visual acuity [BCVA], contrast sensitivity [CS], and Humphrey Visual Field [HVF]) and structural parameters assessed by spectral-domain optical coherence tomography were analyzed in both cohorts before treatment. The cross-sectional analysis of functional and anatomic parameters included the baseline values collected in all eyes at 2 different visits (Screening and Inclusion). Results: Seventy-six subjects were included in total, 39 in RESCUE and 37 in REVERSE. Mean BCVA was significantly worse in RESCUE subjects compared with REVERSE subjects (1.29 and 1.61 LogMAR respectively, P = 0.0029). Similarly, mean CS and HVF were significantly more impaired in REVERSE vs RESCUE subjects (P < 0.005). The cross-sectional analysis showed that the monthly decrease in BCVA, ganglion cell layer macular volume, and retinal nerve fiber layer thickness was much more pronounced in the first 6 months after onset (+0.24 LogMAR, -0.06 mm(3), and -6.00 mu m respectively) than between 6 and 12 months after onset (+0.02 LogMAR, -0.01 mm(3), and -0.43 mu m respectively). Conclusion: LHON progresses rapidly in the first months following onset during the subacute phase, followed by relative stabilization during the dynamic phase.</description><identifier>ISSN: 1070-8022</identifier><identifier>EISSN: 1536-5166</identifier><identifier>DOI: 10.1097/WNO.0000000000001316</identifier><identifier>PMID: 34310464</identifier><language>eng</language><publisher>PHILADELPHIA: Journal of Neuro-Ophthalmology</publisher><subject>Adolescent ; Adult ; Aged ; Clinical Neurology ; Clinical Research: Epidemiology Meets Neuro-Ophthalmology ; Cross-Sectional Studies ; DNA, Mitochondrial - genetics ; Double-Blind Method ; Female ; Genetic Therapy - methods ; Humans ; Life Sciences & Biomedicine ; Male ; Middle Aged ; Neurosciences & Neurology ; Ophthalmology ; Optic Atrophy, Hereditary, Leber - genetics ; Optic Atrophy, Hereditary, Leber - physiopathology ; Optic Atrophy, Hereditary, Leber - therapy ; Retinal Ganglion Cells - pathology ; Science & Technology ; Tomography, Optical Coherence - methods ; Visual Acuity ; Visual Fields - physiology ; Young Adult</subject><ispartof>Journal of neuro-ophthalmology, 2021-09, Vol.41 (3), p.298-308</ispartof><rights>Journal of Neuro-Ophthalmology</rights><rights>Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the North American Neuro-Opthalmology Society.</rights><rights>Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the North American Neuro-Opthalmology Society. 2021</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>true</woscitedreferencessubscribed><woscitedreferencescount>5</woscitedreferencescount><woscitedreferencesoriginalsourcerecordid>wos000711807500012</woscitedreferencesoriginalsourcerecordid><citedby>FETCH-LOGICAL-c4534-112f589d0858391872a97e5dc95bed2c27ed302dd60088bd9cca2984dfc503e53</citedby><cites>FETCH-LOGICAL-c4534-112f589d0858391872a97e5dc95bed2c27ed302dd60088bd9cca2984dfc503e53</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf><![CDATA[$$Uhttp://ovidsp.ovid.com/ovidweb.cgi?T=JS&CSC=Y&NEWS=N&PAGE=fulltext&PDF=y&D=ovft&AN=00041327-202109000-00004$$EPDF$$P50$$Gwolterskluwer$$H]]></linktopdf><linktohtml>$$Uhttp://ovidsp.ovid.com/ovidweb.cgi?T=JS&NEWS=n&CSC=Y&PAGE=fulltext&D=ovft&AN=00041327-202109000-00004$$EHTML$$P50$$Gwolterskluwer$$H</linktohtml><link.rule.ids>230,315,781,785,886,4610,27929,27930,39263,64671,65466</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/34310464$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Moster, Mark L.</creatorcontrib><creatorcontrib>Sergott, Robert C.</creatorcontrib><creatorcontrib>Newman, Nancy J.</creatorcontrib><creatorcontrib>Yu-Wai-Man, Patrick</creatorcontrib><creatorcontrib>Carelli, Valerio</creatorcontrib><creatorcontrib>Bryan, Molly Scannell</creatorcontrib><creatorcontrib>Smits, Gerard</creatorcontrib><creatorcontrib>Biousse, Valérie</creatorcontrib><creatorcontrib>Vignal-Clermont, Catherine</creatorcontrib><creatorcontrib>Klopstock, Thomas</creatorcontrib><creatorcontrib>Sadun, Alfredo A.</creatorcontrib><creatorcontrib>DeBusk, Adam A.</creatorcontrib><creatorcontrib>Carbonelli, Michele</creatorcontrib><creatorcontrib>Hage, Rabih</creatorcontrib><creatorcontrib>Priglinger, Siegfried</creatorcontrib><creatorcontrib>Karanjia, Rustum</creatorcontrib><creatorcontrib>Blouin, Laure</creatorcontrib><creatorcontrib>Taiel, Magali</creatorcontrib><creatorcontrib>Katz, Barrett</creatorcontrib><creatorcontrib>Sahel, José Alain</creatorcontrib><creatorcontrib>LHON Study Grp</creatorcontrib><creatorcontrib>LHON study group</creatorcontrib><creatorcontrib>for the LHON study group</creatorcontrib><title>Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies</title><title>Journal of neuro-ophthalmology</title><addtitle>J NEURO-OPHTHALMOL</addtitle><addtitle>J Neuroophthalmol</addtitle><description>Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual parameters over the first year after vision loss. Methods: RESCUE and REVERSE were 2 phase III clinical trials designed to assess the efficacy of rAAV2/2-ND4 gene therapy in ND4-LHON subjects. At enrollment, subjects had vision loss for <= 6 months in RESCUE, and between 6 and 12 months in REVERSE. Functional visual parameters (best-corrected visual acuity [BCVA], contrast sensitivity [CS], and Humphrey Visual Field [HVF]) and structural parameters assessed by spectral-domain optical coherence tomography were analyzed in both cohorts before treatment. The cross-sectional analysis of functional and anatomic parameters included the baseline values collected in all eyes at 2 different visits (Screening and Inclusion). Results: Seventy-six subjects were included in total, 39 in RESCUE and 37 in REVERSE. Mean BCVA was significantly worse in RESCUE subjects compared with REVERSE subjects (1.29 and 1.61 LogMAR respectively, P = 0.0029). Similarly, mean CS and HVF were significantly more impaired in REVERSE vs RESCUE subjects (P < 0.005). The cross-sectional analysis showed that the monthly decrease in BCVA, ganglion cell layer macular volume, and retinal nerve fiber layer thickness was much more pronounced in the first 6 months after onset (+0.24 LogMAR, -0.06 mm(3), and -6.00 mu m respectively) than between 6 and 12 months after onset (+0.02 LogMAR, -0.01 mm(3), and -0.43 mu m respectively). Conclusion: LHON progresses rapidly in the first months following onset during the subacute phase, followed by relative stabilization during the dynamic phase.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Aged</subject><subject>Clinical Neurology</subject><subject>Clinical Research: Epidemiology Meets Neuro-Ophthalmology</subject><subject>Cross-Sectional Studies</subject><subject>DNA, Mitochondrial - genetics</subject><subject>Double-Blind Method</subject><subject>Female</subject><subject>Genetic Therapy - methods</subject><subject>Humans</subject><subject>Life Sciences & Biomedicine</subject><subject>Male</subject><subject>Middle Aged</subject><subject>Neurosciences & Neurology</subject><subject>Ophthalmology</subject><subject>Optic Atrophy, Hereditary, Leber - genetics</subject><subject>Optic Atrophy, Hereditary, Leber - physiopathology</subject><subject>Optic Atrophy, Hereditary, Leber - therapy</subject><subject>Retinal Ganglion Cells - pathology</subject><subject>Science & Technology</subject><subject>Tomography, Optical Coherence - methods</subject><subject>Visual Acuity</subject><subject>Visual Fields - physiology</subject><subject>Young Adult</subject><issn>1070-8022</issn><issn>1536-5166</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>HGBXW</sourceid><sourceid>EIF</sourceid><recordid>eNqNUt1u0zAYjRCIjcEbIORLJJThnzhObpC2ULZJVYvabVxarv2FeqRxsR2mvgDPjaOOMrjCF_Znfecc-_g4y14TfEpwLd5_mc1P8aNBGCmfZMeEszLnpCyfphoLnFeY0qPsRQh3CcQwrZ9nR6xgBBdlcZz9bLwLIV-Cjtb1qkNnadoFG5Br0bkK0Nke0K0NQ-p9Vl5tIIIPyPZoOazuEi2gBWg_2AgGXfXRobgGtJgsm5sJUr1J5e1ksZyg2ccin17OZ-gCkuL1Grza7tAyDsZCeJk9a1UX4NXDepLdfJpcN5f5dH5x1ZxNc11wVuSE0JZXtcEVr1hNKkFVLYAbXfMVGKqpAJMsGlNiXFUrU2utaF0VptUcM-DsJPuw190Oqw0YDX30qpNbbzfK76RTVv7d6e1afnU_ZMXKUnCRBN4-CHj3fYAQ5cYGDV2nenBDkJRzXjLByQgt9lA9PrGH9nAMwXKMUKYI5b8RJtqbx1c8kH5nlgDv9oB7WLk2aAu9hgMsyQhCKiz4KEgTuvp_dGOjGv9B44Y-_jFw77ox9G_dcA9erkF1cT1SC8KoyCmmyU3a5qOHgv0C-TvHEw</recordid><startdate>20210901</startdate><enddate>20210901</enddate><creator>Moster, Mark L.</creator><creator>Sergott, Robert C.</creator><creator>Newman, Nancy J.</creator><creator>Yu-Wai-Man, Patrick</creator><creator>Carelli, Valerio</creator><creator>Bryan, Molly Scannell</creator><creator>Smits, Gerard</creator><creator>Biousse, Valérie</creator><creator>Vignal-Clermont, Catherine</creator><creator>Klopstock, Thomas</creator><creator>Sadun, Alfredo A.</creator><creator>DeBusk, Adam A.</creator><creator>Carbonelli, Michele</creator><creator>Hage, Rabih</creator><creator>Priglinger, Siegfried</creator><creator>Karanjia, Rustum</creator><creator>Blouin, Laure</creator><creator>Taiel, Magali</creator><creator>Katz, Barrett</creator><creator>Sahel, José Alain</creator><general>Journal of Neuro-Ophthalmology</general><general>Lippincott Williams & Wilkins</general><scope>BLEPL</scope><scope>DTL</scope><scope>HGBXW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope></search><sort><creationdate>20210901</creationdate><title>Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies</title><author>Moster, Mark L. ; Sergott, Robert C. ; Newman, Nancy J. ; Yu-Wai-Man, Patrick ; Carelli, Valerio ; Bryan, Molly Scannell ; Smits, Gerard ; Biousse, Valérie ; Vignal-Clermont, Catherine ; Klopstock, Thomas ; Sadun, Alfredo A. ; DeBusk, Adam A. ; Carbonelli, Michele ; Hage, Rabih ; Priglinger, Siegfried ; Karanjia, Rustum ; Blouin, Laure ; Taiel, Magali ; Katz, Barrett ; Sahel, José Alain</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4534-112f589d0858391872a97e5dc95bed2c27ed302dd60088bd9cca2984dfc503e53</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Aged</topic><topic>Clinical Neurology</topic><topic>Clinical Research: Epidemiology Meets Neuro-Ophthalmology</topic><topic>Cross-Sectional Studies</topic><topic>DNA, Mitochondrial - genetics</topic><topic>Double-Blind Method</topic><topic>Female</topic><topic>Genetic Therapy - methods</topic><topic>Humans</topic><topic>Life Sciences & Biomedicine</topic><topic>Male</topic><topic>Middle Aged</topic><topic>Neurosciences & Neurology</topic><topic>Ophthalmology</topic><topic>Optic Atrophy, Hereditary, Leber - genetics</topic><topic>Optic Atrophy, Hereditary, Leber - physiopathology</topic><topic>Optic Atrophy, Hereditary, Leber - therapy</topic><topic>Retinal Ganglion Cells - pathology</topic><topic>Science & Technology</topic><topic>Tomography, Optical Coherence - methods</topic><topic>Visual Acuity</topic><topic>Visual Fields - physiology</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Moster, Mark L.</creatorcontrib><creatorcontrib>Sergott, Robert C.</creatorcontrib><creatorcontrib>Newman, Nancy J.</creatorcontrib><creatorcontrib>Yu-Wai-Man, Patrick</creatorcontrib><creatorcontrib>Carelli, Valerio</creatorcontrib><creatorcontrib>Bryan, Molly Scannell</creatorcontrib><creatorcontrib>Smits, Gerard</creatorcontrib><creatorcontrib>Biousse, Valérie</creatorcontrib><creatorcontrib>Vignal-Clermont, Catherine</creatorcontrib><creatorcontrib>Klopstock, Thomas</creatorcontrib><creatorcontrib>Sadun, Alfredo A.</creatorcontrib><creatorcontrib>DeBusk, Adam A.</creatorcontrib><creatorcontrib>Carbonelli, Michele</creatorcontrib><creatorcontrib>Hage, Rabih</creatorcontrib><creatorcontrib>Priglinger, Siegfried</creatorcontrib><creatorcontrib>Karanjia, Rustum</creatorcontrib><creatorcontrib>Blouin, Laure</creatorcontrib><creatorcontrib>Taiel, Magali</creatorcontrib><creatorcontrib>Katz, Barrett</creatorcontrib><creatorcontrib>Sahel, José Alain</creatorcontrib><creatorcontrib>LHON Study Grp</creatorcontrib><creatorcontrib>LHON study group</creatorcontrib><creatorcontrib>for the LHON study group</creatorcontrib><collection>Web of Science Core Collection</collection><collection>Science Citation Index Expanded</collection><collection>Web of Science - Science Citation Index Expanded - 2021</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Journal of neuro-ophthalmology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Moster, Mark L.</au><au>Sergott, Robert C.</au><au>Newman, Nancy J.</au><au>Yu-Wai-Man, Patrick</au><au>Carelli, Valerio</au><au>Bryan, Molly Scannell</au><au>Smits, Gerard</au><au>Biousse, Valérie</au><au>Vignal-Clermont, Catherine</au><au>Klopstock, Thomas</au><au>Sadun, Alfredo A.</au><au>DeBusk, Adam A.</au><au>Carbonelli, Michele</au><au>Hage, Rabih</au><au>Priglinger, Siegfried</au><au>Karanjia, Rustum</au><au>Blouin, Laure</au><au>Taiel, Magali</au><au>Katz, Barrett</au><au>Sahel, José Alain</au><aucorp>LHON Study Grp</aucorp><aucorp>LHON study group</aucorp><aucorp>for the LHON study group</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies</atitle><jtitle>Journal of neuro-ophthalmology</jtitle><stitle>J NEURO-OPHTHALMOL</stitle><addtitle>J Neuroophthalmol</addtitle><date>2021-09-01</date><risdate>2021</risdate><volume>41</volume><issue>3</issue><spage>298</spage><epage>308</epage><pages>298-308</pages><issn>1070-8022</issn><eissn>1536-5166</eissn><abstract>Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual parameters over the first year after vision loss. Methods: RESCUE and REVERSE were 2 phase III clinical trials designed to assess the efficacy of rAAV2/2-ND4 gene therapy in ND4-LHON subjects. At enrollment, subjects had vision loss for <= 6 months in RESCUE, and between 6 and 12 months in REVERSE. Functional visual parameters (best-corrected visual acuity [BCVA], contrast sensitivity [CS], and Humphrey Visual Field [HVF]) and structural parameters assessed by spectral-domain optical coherence tomography were analyzed in both cohorts before treatment. The cross-sectional analysis of functional and anatomic parameters included the baseline values collected in all eyes at 2 different visits (Screening and Inclusion). Results: Seventy-six subjects were included in total, 39 in RESCUE and 37 in REVERSE. Mean BCVA was significantly worse in RESCUE subjects compared with REVERSE subjects (1.29 and 1.61 LogMAR respectively, P = 0.0029). Similarly, mean CS and HVF were significantly more impaired in REVERSE vs RESCUE subjects (P < 0.005). The cross-sectional analysis showed that the monthly decrease in BCVA, ganglion cell layer macular volume, and retinal nerve fiber layer thickness was much more pronounced in the first 6 months after onset (+0.24 LogMAR, -0.06 mm(3), and -6.00 mu m respectively) than between 6 and 12 months after onset (+0.02 LogMAR, -0.01 mm(3), and -0.43 mu m respectively). Conclusion: LHON progresses rapidly in the first months following onset during the subacute phase, followed by relative stabilization during the dynamic phase.</abstract><cop>PHILADELPHIA</cop><pub>Journal of Neuro-Ophthalmology</pub><pmid>34310464</pmid><doi>10.1097/WNO.0000000000001316</doi><tpages>11</tpages><oa>free_for_read</oa></addata></record> |
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| subjects | Adolescent Adult Aged Clinical Neurology Clinical Research: Epidemiology Meets Neuro-Ophthalmology Cross-Sectional Studies DNA, Mitochondrial - genetics Double-Blind Method Female Genetic Therapy - methods Humans Life Sciences & Biomedicine Male Middle Aged Neurosciences & Neurology Ophthalmology Optic Atrophy, Hereditary, Leber - genetics Optic Atrophy, Hereditary, Leber - physiopathology Optic Atrophy, Hereditary, Leber - therapy Retinal Ganglion Cells - pathology Science & Technology Tomography, Optical Coherence - methods Visual Acuity Visual Fields - physiology Young Adult |
| title | Cross-Sectional Analysis of Baseline Visual Parameters in Subjects Recruited Into the RESCUE and REVERSE ND4-LHON Gene Therapy Studies |
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