Evidence map›Paper›PMID 35156839›Full record

ArticleHuman gene therapy2022

Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A.

Robert Klamroth, Gregory Hayes, Tatiana Andreeva, Keith Gregg, Takashi Suzuki, Ismail Haroon Mitha, Brandon Hardesty, Midori Shima, Toni Pollock, Patricia Slev and 16 more

Open access · hybridAbstract read
In one paragraph

Article in Human gene therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 66 papers.

0numbers the graph read from it
0cells of the map it votes in
66citing papers in PubMed
17.2field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

66 citing papers in PubMed, 98 citations in OpenAlex.

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  17. Deconstructing gene therapy in hemophilia for the clinician.Hematology. American Society of Hematology. Education Program · 2025
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6 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

26 authors at 18 institutions in 9 countries.

Robert KlamrothComprehensive Care Haemophilia Treatment Center, Vivantes Klinikum im Friedrichshain, Berlin, Germany.ORCID 0000-0003-4194-8183
Gregory HayesBioMarin Pharmaceutical, Inc., Novato, California, USA.
Tatiana AndreevaMunicipal Center of Hemophilia Therapy, Saint Petersburg, Russia.
Keith GreggBioMarin Pharmaceutical, Inc., Novato, California, USA.
Takashi SuzukiOgikubo Hospital, Tokyo, Japan.
Ismail Haroon MithaWorthwhile Clinical Trials, Lakeview Hospital, Benoni, South Africa.
Brandon HardestyIndiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA.
Midori ShimaNara Medical University, Kashihara, Japan.
Toni PollockARUP Laboratories, Salt Lake City, Utah, USA.
Patricia SlevARUP Laboratories, Salt Lake City, Utah, USA.
Johannes OldenburgUniversitätsklinikum Bonn, Bonn, Germany.
Margareth C OzeloHemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil.
Natalie StieltjesDepartment of Haematology and Regional Centre of Haemophilia Treatment, Hôpital Cochin, Assistance Publique Hôpitaux de Paris (AP-HP), Sorbonne Paris Cité, Université Paris Descartes, Paris, France.
Sabine-Marie CastetCentre de Ressources et de Compétence des Maladies Hémorragiques Constitutionnelles, CHU de Bordeaux, Bordeaux, France.
Johnny MahlanguHaemophilia Comprehensive Care Centre, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa.
Flora PeyvandiFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
Rashid KazmiDepartment of Haematology, Southampton University Hospital, Southampton, United Kingdom.
Jean-François SchvedCentre Régional de Traitement des Hémophiles, Hôpital Saint-Eloi, CHRU de Montpellier, Montpellier, France.
Andrew D LeavittDepartments of Medicine and Laboratory Medicine, University of California San Francisco, San Francisco, California, USA.
Michael CallaghanDivision of Pediatric Hematology/Oncology, Central Michigan University, Detroit, Michigan, USA.
Brigitte Pan-PeteschCentre Hospitalier Régional Universitaire de Brest, Hôpital A. Morvan, Brest, France.
Doris V QuonOrthopaedic Hemophilia Treatment Center, Orthopaedic Institute for Children, Los Angeles, California, USA.
Jayson AndrewsBioMarin Pharmaceutical, Inc., Novato, California, USA.
Alex TrinhBioMarin Pharmaceutical, Inc., Novato, California, USA.
Mingjin LiBioMarin Pharmaceutical, Inc., Novato, California, USA.
Wing Yen WongBioMarin Pharmaceutical, Inc., Novato, California, USA.
NovAtel (Canada) · CAARUP Laboratories (United States) · USCentral Michigan University · USCentre Hospitalier Régional Universitaire de Brest · FRCentre Hospitalier Universitaire de Bordeaux · FRDélégation Paris 5 · FRHôpital Saint Eloi · FRIndiana Hemophilia and Thrombosis Center · USKlinikum im Friedrichshain · DENara Medical University · JPNational Health Laboratory Service · ZAOgikubo Hospital · JPOrthopaedic Institute for Children · USUniversidade Estadual de Campinas (UNICAMP) · BRUniversity Hospital Bonn · DEUniversity Hospital Southampton NHS Foundation Trust · GBUniversity of California, San Francisco · USUniversity of Milan · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adeno-associated virus (AAV)-mediated gene therapy may provide durable protection from bleeding events and reduce treatment burden for people with hemophilia A (HA). However, pre-existing immunity against AAV may limit transduction efficiency and hence treatment success. Global data on the prevalence of AAV serotypes are limited. In this global, prospective, noninterventional study, we determined the prevalence of pre-existing immunity against AAV2, AAV5, AAV6, AAV8, and AAVrh10 among people ≥12 years of age with HA and residual FVIII levels ≤2 IU/dL. Antibodies against each serotype were detected using validated, electrochemiluminescent-based enzyme-linked immunosorbent assays. To evaluate changes in antibody titers over time, 20% of participants were retested at 3 and 6 months. In total, 546 participants with HA were enrolled at 19 sites in 9 countries. Mean (standard deviation) age at enrollment was 36.0 (14.87) years, including 12.5% younger than 18 years, and 20.0% 50 years of age and older. On day 1, global seroprevalence was 58.5% for AAV2, 34.8% for AAV5, 48.7% for AAV6, 45.6% for AAV8, and 46.0% for AAVrh10. Considerable geographic variability was observed in the prevalence of pre-existing antibodies against each serotype, but AAV5 consistently had the lowest seroprevalence across the countries studied. AAV5 seropositivity rates were 51.8% in South Africa (

Indexed as

DependovirusHemophilia AAntibodies, NeutralizingAntibodies, ViralGenetic VectorsHumansProspective StudiesSeroepidemiologic StudiesSerogroupAntibodies, NeutralizingAntibodies, Viraladeno-associated virusantibodygene therapyhemophilia Aseropositivity

Identifiers

PMID35156839
PMCPMC9063149
OpenAlexW4212868305

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.