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
Hauptverfasser: Aharoni, Sharon, Bistritzer, Jacob, Levine, Hagit, Sagi, Liora, Fattal-Valevski, Aviva, Ginzberg, Mira, Noyman, Iris, Cohen, Rony, Nevo, Yoram
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container_end_page 106
container_issue 1-2
container_start_page 101
container_title Gene therapy
container_volume 30
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
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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 &gt; 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  &gt; 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. 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ispartof Gene therapy, 2023-02, Vol.30 (1-2), p.101-106
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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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