Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence
Spinal muscular atrophy (SMA) is characterized by progressive weakness of skeletal and respiratory muscles. This study aimed to evaluate the prevalence of pre-existing anti adeno-associated virus serotype 9 antibody (AAV9-Ab) titers among infantile-onset SMA diagnosed infants pre-screened for treatm...
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| Veröffentlicht in: | Gene therapy 2023-02, Vol.30 (1-2), p.101-106 |
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| creator | Aharoni, Sharon Bistritzer, Jacob Levine, Hagit Sagi, Liora Fattal-Valevski, Aviva Ginzberg, Mira Noyman, Iris Cohen, Rony Nevo, Yoram |
| description | Spinal muscular atrophy (SMA) is characterized by progressive weakness of skeletal and respiratory muscles. This study aimed to evaluate the prevalence of pre-existing anti adeno-associated virus serotype 9 antibody (AAV9-Ab) titers among infantile-onset SMA diagnosed infants pre-screened for treatment with AAV9-based onasemnogene abeparvovec, and to explore whether clinical and/or demographic characteristics are correlated with AAV9 Ab test results. This is a retrospective multicenter study of children diagnosed with 5q SMA younger than two years of age. The obtained data included demographic data, SMA type,
SMN2
gene copy number, onset date, and results of AAV9-Ab test and of SMA prior treatments. Thirty-four patients were enrolled; six patients had positive results of AAV9-Ab (titer > 1:50) in the initial screening, 15 patients were re-tested for AAV9-Abs, of whom, three patients had seroreverted [1.5–4.5 months] between the two AAV9-Abs tests. One patient had seroconverted (5.5 months after the first AAV9-Abs test). The remaining 11 patients presented matching titer results in the two tests. No demographic/clinical factors were correlated to high AAV9-Abs titers (
P
> 0.05).We recommend AAV9-Ab re-tests to be performed until the age of 8 months, or, if 1.5 months or more have passed after the initial AAV9-Abs test. |
| doi_str_mv | 10.1038/s41434-022-00339-0 |
| format | Article |
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SMN2
gene copy number, onset date, and results of AAV9-Ab test and of SMA prior treatments. Thirty-four patients were enrolled; six patients had positive results of AAV9-Ab (titer > 1:50) in the initial screening, 15 patients were re-tested for AAV9-Abs, of whom, three patients had seroreverted [1.5–4.5 months] between the two AAV9-Abs tests. One patient had seroconverted (5.5 months after the first AAV9-Abs test). The remaining 11 patients presented matching titer results in the two tests. No demographic/clinical factors were correlated to high AAV9-Abs titers (
P
> 0.05).We recommend AAV9-Ab re-tests to be performed until the age of 8 months, or, if 1.5 months or more have passed after the initial AAV9-Abs test.</description><identifier>ISSN: 0969-7128</identifier><identifier>EISSN: 1476-5462</identifier><identifier>DOI: 10.1038/s41434-022-00339-0</identifier><identifier>PMID: 35474244</identifier><language>eng</language><publisher>London: Nature Publishing Group UK</publisher><subject>13 ; 13/1 ; 42 ; 42/44 ; 631/378/1959 ; 692/699/375 ; Age ; Antibodies ; Atrophy ; Biomedical and Life Sciences ; Biomedicine ; Cell Biology ; Child ; Copy number ; Demographics ; Demography ; Dependovirus - genetics ; FDA approval ; Gene Expression ; Gene Therapy ; Genetic engineering ; Genetic Therapy - methods ; Human Genetics ; Humans ; Immunization ; Infant ; Medical records ; Muscles ; Muscular Atrophy, Spinal - therapy ; Nanotechnology ; Neurology ; Neuromuscular diseases ; Patients ; Serogroup ; Skeletal muscle ; SMN protein ; Spinal Muscular Atrophies of Childhood - drug therapy ; Spinal Muscular Atrophies of Childhood - genetics ; Spinal muscular atrophy ; Viral infections ; Viruses</subject><ispartof>Gene therapy, 2023-02, Vol.30 (1-2), p.101-106</ispartof><rights>The Author(s), under exclusive licence to Springer Nature Limited 2022</rights><rights>2022. The Author(s), under exclusive licence to Springer Nature Limited.</rights><rights>The Author(s), under exclusive licence to Springer Nature Limited 2022.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c305t-44a36b74ab445f720df644d6ae7f735cd143e7079d94101862abd3d6e76b6b0e3</citedby><cites>FETCH-LOGICAL-c305t-44a36b74ab445f720df644d6ae7f735cd143e7079d94101862abd3d6e76b6b0e3</cites><orcidid>0000-0001-7255-6276</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>315,782,786,27931,27932</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/35474244$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Aharoni, Sharon</creatorcontrib><creatorcontrib>Bistritzer, Jacob</creatorcontrib><creatorcontrib>Levine, Hagit</creatorcontrib><creatorcontrib>Sagi, Liora</creatorcontrib><creatorcontrib>Fattal-Valevski, Aviva</creatorcontrib><creatorcontrib>Ginzberg, Mira</creatorcontrib><creatorcontrib>Noyman, Iris</creatorcontrib><creatorcontrib>Cohen, Rony</creatorcontrib><creatorcontrib>Nevo, Yoram</creatorcontrib><title>Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence</title><title>Gene therapy</title><addtitle>Gene Ther</addtitle><addtitle>Gene Ther</addtitle><description>Spinal muscular atrophy (SMA) is characterized by progressive weakness of skeletal and respiratory muscles. This study aimed to evaluate the prevalence of pre-existing anti adeno-associated virus serotype 9 antibody (AAV9-Ab) titers among infantile-onset SMA diagnosed infants pre-screened for treatment with AAV9-based onasemnogene abeparvovec, and to explore whether clinical and/or demographic characteristics are correlated with AAV9 Ab test results. This is a retrospective multicenter study of children diagnosed with 5q SMA younger than two years of age. The obtained data included demographic data, SMA type,
SMN2
gene copy number, onset date, and results of AAV9-Ab test and of SMA prior treatments. Thirty-four patients were enrolled; six patients had positive results of AAV9-Ab (titer > 1:50) in the initial screening, 15 patients were re-tested for AAV9-Abs, of whom, three patients had seroreverted [1.5–4.5 months] between the two AAV9-Abs tests. One patient had seroconverted (5.5 months after the first AAV9-Abs test). The remaining 11 patients presented matching titer results in the two tests. No demographic/clinical factors were correlated to high AAV9-Abs titers (
P
> 0.05).We recommend AAV9-Ab re-tests to be performed until the age of 8 months, or, if 1.5 months or more have passed after the initial AAV9-Abs test.</description><subject>13</subject><subject>13/1</subject><subject>42</subject><subject>42/44</subject><subject>631/378/1959</subject><subject>692/699/375</subject><subject>Age</subject><subject>Antibodies</subject><subject>Atrophy</subject><subject>Biomedical and Life Sciences</subject><subject>Biomedicine</subject><subject>Cell Biology</subject><subject>Child</subject><subject>Copy number</subject><subject>Demographics</subject><subject>Demography</subject><subject>Dependovirus - genetics</subject><subject>FDA approval</subject><subject>Gene Expression</subject><subject>Gene Therapy</subject><subject>Genetic engineering</subject><subject>Genetic Therapy - methods</subject><subject>Human Genetics</subject><subject>Humans</subject><subject>Immunization</subject><subject>Infant</subject><subject>Medical records</subject><subject>Muscles</subject><subject>Muscular Atrophy, Spinal - therapy</subject><subject>Nanotechnology</subject><subject>Neurology</subject><subject>Neuromuscular diseases</subject><subject>Patients</subject><subject>Serogroup</subject><subject>Skeletal muscle</subject><subject>SMN protein</subject><subject>Spinal Muscular Atrophies of Childhood - drug therapy</subject><subject>Spinal Muscular Atrophies of Childhood - genetics</subject><subject>Spinal muscular atrophy</subject><subject>Viral infections</subject><subject>Viruses</subject><issn>0969-7128</issn><issn>1476-5462</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>8G5</sourceid><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>AZQEC</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><sourceid>DWQXO</sourceid><sourceid>GNUQQ</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNp9kbuO1TAQhiMEYg8LL0CBLNHQGMaX2El5tOImLaIAasuJJ4tXJ3awnYNOxzvQ8Hw8CYYsIFFQWRp_889ovqZ5yOApA9E9y5JJISlwTgGE6CncanZMakVbqfjtZge96qlmvDtr7uV8DQBSd_xucyZaqSWXctd82zsMkdqc4-htQUeOPq2ZZEyxnBYkPbGh-CG6Eym-YMrEB7LY4jGUTD778pG8e7MnS0Kax4QYasQUEykJbZkrtDEx2IxziFcVIHbAxaZjPOJIvn_5muJafC2PNiHBo68bjXi_uTPZQ8YHN-958-HF8_cXr-jl25evL_aXdBTQFiqlFWrQ0g5StpPm4CYlpVMW9aRFO7p6ItSge9dLBqxT3A5OOIVaDWoAFOfNky13SfHTirmY2ecRDwcbMK7ZcNUqDhxaVtHH_6DXcU2hbme41pr1GtquUnyjxhRzTjiZJfnZppNhYH56M5s3U72ZX94M1KZHN9HrMKP70_JbVAXEBuT6Fa4w_Z39n9gfc0SmaQ</recordid><startdate>20230201</startdate><enddate>20230201</enddate><creator>Aharoni, Sharon</creator><creator>Bistritzer, Jacob</creator><creator>Levine, Hagit</creator><creator>Sagi, Liora</creator><creator>Fattal-Valevski, Aviva</creator><creator>Ginzberg, Mira</creator><creator>Noyman, Iris</creator><creator>Cohen, Rony</creator><creator>Nevo, Yoram</creator><general>Nature Publishing Group UK</general><general>Nature Publishing Group</general><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>3V.</scope><scope>7QP</scope><scope>7TK</scope><scope>7TM</scope><scope>7U9</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8C1</scope><scope>8FD</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>8G5</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>H94</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M2O</scope><scope>M7P</scope><scope>MBDVC</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>Q9U</scope><scope>RC3</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0001-7255-6276</orcidid></search><sort><creationdate>20230201</creationdate><title>Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence</title><author>Aharoni, Sharon ; Bistritzer, Jacob ; Levine, Hagit ; Sagi, Liora ; Fattal-Valevski, Aviva ; Ginzberg, Mira ; Noyman, Iris ; Cohen, Rony ; Nevo, Yoram</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c305t-44a36b74ab445f720df644d6ae7f735cd143e7079d94101862abd3d6e76b6b0e3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>13</topic><topic>13/1</topic><topic>42</topic><topic>42/44</topic><topic>631/378/1959</topic><topic>692/699/375</topic><topic>Age</topic><topic>Antibodies</topic><topic>Atrophy</topic><topic>Biomedical and Life Sciences</topic><topic>Biomedicine</topic><topic>Cell Biology</topic><topic>Child</topic><topic>Copy number</topic><topic>Demographics</topic><topic>Demography</topic><topic>Dependovirus - genetics</topic><topic>FDA approval</topic><topic>Gene Expression</topic><topic>Gene Therapy</topic><topic>Genetic engineering</topic><topic>Genetic Therapy - methods</topic><topic>Human Genetics</topic><topic>Humans</topic><topic>Immunization</topic><topic>Infant</topic><topic>Medical records</topic><topic>Muscles</topic><topic>Muscular Atrophy, Spinal - therapy</topic><topic>Nanotechnology</topic><topic>Neurology</topic><topic>Neuromuscular diseases</topic><topic>Patients</topic><topic>Serogroup</topic><topic>Skeletal muscle</topic><topic>SMN protein</topic><topic>Spinal Muscular Atrophies of Childhood - drug therapy</topic><topic>Spinal Muscular Atrophies of Childhood - genetics</topic><topic>Spinal muscular atrophy</topic><topic>Viral infections</topic><topic>Viruses</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Aharoni, Sharon</creatorcontrib><creatorcontrib>Bistritzer, Jacob</creatorcontrib><creatorcontrib>Levine, Hagit</creatorcontrib><creatorcontrib>Sagi, Liora</creatorcontrib><creatorcontrib>Fattal-Valevski, Aviva</creatorcontrib><creatorcontrib>Ginzberg, Mira</creatorcontrib><creatorcontrib>Noyman, Iris</creatorcontrib><creatorcontrib>Cohen, Rony</creatorcontrib><creatorcontrib>Nevo, Yoram</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Calcium & Calcified Tissue Abstracts</collection><collection>Neurosciences Abstracts</collection><collection>Nucleic Acids Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Public Health Database</collection><collection>Technology Research Database</collection><collection>ProQuest SciTech Collection</collection><collection>ProQuest Natural Science Collection</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>Research Library (Alumni Edition)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>ProQuest Central Essentials</collection><collection>Biological Science Collection</collection><collection>ProQuest Central</collection><collection>Natural Science Collection</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Engineering Research Database</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>Research Library Prep</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>SciTech Premium Collection</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>ProQuest Biological Science Collection</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>Research Library</collection><collection>Biological Science Database</collection><collection>Research Library (Corporate)</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central Basic</collection><collection>Genetics Abstracts</collection><collection>MEDLINE - Academic</collection><jtitle>Gene therapy</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Aharoni, Sharon</au><au>Bistritzer, Jacob</au><au>Levine, Hagit</au><au>Sagi, Liora</au><au>Fattal-Valevski, Aviva</au><au>Ginzberg, Mira</au><au>Noyman, Iris</au><au>Cohen, Rony</au><au>Nevo, Yoram</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence</atitle><jtitle>Gene therapy</jtitle><stitle>Gene Ther</stitle><addtitle>Gene Ther</addtitle><date>2023-02-01</date><risdate>2023</risdate><volume>30</volume><issue>1-2</issue><spage>101</spage><epage>106</epage><pages>101-106</pages><issn>0969-7128</issn><eissn>1476-5462</eissn><abstract>Spinal muscular atrophy (SMA) is characterized by progressive weakness of skeletal and respiratory muscles. This study aimed to evaluate the prevalence of pre-existing anti adeno-associated virus serotype 9 antibody (AAV9-Ab) titers among infantile-onset SMA diagnosed infants pre-screened for treatment with AAV9-based onasemnogene abeparvovec, and to explore whether clinical and/or demographic characteristics are correlated with AAV9 Ab test results. This is a retrospective multicenter study of children diagnosed with 5q SMA younger than two years of age. The obtained data included demographic data, SMA type,
SMN2
gene copy number, onset date, and results of AAV9-Ab test and of SMA prior treatments. Thirty-four patients were enrolled; six patients had positive results of AAV9-Ab (titer > 1:50) in the initial screening, 15 patients were re-tested for AAV9-Abs, of whom, three patients had seroreverted [1.5–4.5 months] between the two AAV9-Abs tests. One patient had seroconverted (5.5 months after the first AAV9-Abs test). The remaining 11 patients presented matching titer results in the two tests. No demographic/clinical factors were correlated to high AAV9-Abs titers (
P
> 0.05).We recommend AAV9-Ab re-tests to be performed until the age of 8 months, or, if 1.5 months or more have passed after the initial AAV9-Abs test.</abstract><cop>London</cop><pub>Nature Publishing Group UK</pub><pmid>35474244</pmid><doi>10.1038/s41434-022-00339-0</doi><tpages>6</tpages><orcidid>https://orcid.org/0000-0001-7255-6276</orcidid></addata></record> |
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| ispartof | Gene therapy, 2023-02, Vol.30 (1-2), p.101-106 |
| issn | 0969-7128 1476-5462 |
| language | eng |
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| source | MEDLINE; Alma/SFX Local Collection |
| subjects | 13 13/1 42 42/44 631/378/1959 692/699/375 Age Antibodies Atrophy Biomedical and Life Sciences Biomedicine Cell Biology Child Copy number Demographics Demography Dependovirus - genetics FDA approval Gene Expression Gene Therapy Genetic engineering Genetic Therapy - methods Human Genetics Humans Immunization Infant Medical records Muscles Muscular Atrophy, Spinal - therapy Nanotechnology Neurology Neuromuscular diseases Patients Serogroup Skeletal muscle SMN protein Spinal Muscular Atrophies of Childhood - drug therapy Spinal Muscular Atrophies of Childhood - genetics Spinal muscular atrophy Viral infections Viruses |
| title | Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence |
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