SynthesisBlood advances2024
Adeno-associated virus-based gene therapy for hemophilia A and B: a systematic review and meta-analysis.
Synthesis in Blood advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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.
Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Adeno-associated virus-based gene therapy for hemophilia A and B: a systematic review and meta-analysis.Blood advances · 2024Pooled it
- Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials.Nature medicine · 2026Trial
- Personalized Treatment of Hemophilia: Matching Therapies to Patient Needs in a Rapidly Evolving Landscape.Drugs · 2026Review
- The Global Gap in the Hemophilia Paradigm Shift: Disparities in Research, Care, and Musculoskeletal Health.Hematology reports · 2026Review
- Foods for special medical purposes for the dietary therapy of rare diseases: Current status and future prospects.Intractable & rare diseases research · 2026Review
- Etranacogene dezaparvovec in people with hemophilia B and without adeno-associated virus serotype 5 neutralizing antibodies: a 4-year subgroup analysis of the Health Outcomes with Padua Gene; Evaluation in Hemophilia B (HOPE-B) trial.Research and practice in thrombosis and haemostasis · 2026Article
- Balancing Promise and Peril: Hemophilia Gene Therapy Insights.IUBMB life · 2026Review
- Emerging Technologies Tackling Adeno-Associated Viruses (AAV) Immunogenicity in Gene Therapy Applications.Pharmaceutics · 2025Review
- Therapeutic innovations in hemophilia: the essential role of a positive reinvestment cycle.Blood advances · 2025Review
- Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context.Biomedicines · 2025Review
- RNAi targeting heparin cofactor II promotes hemostasis in a canine model of acquired hemophilia A.Gene therapy · 2025Article
- [Gene therapy marks the beginning of a potential "clinical cure" for hemophilia B patients].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- AAV vector development, back to the future.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Therapeutic advances in hemophilia: from molecular innovation to patient-centered global care.Frontiers in medicine · 2025Review
Corrections and comments
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractAdeno-associated virus (AAV)-based gene therapy is an emerging treatment for hemophilia A (HA) and hemophilia B (HB). In this systematic review and meta-analysis, we searched for studies of adult males with severe or moderately severe HA or HB who received AAV-based gene therapy. Annualized bleeding rate (ABR), annualized infusion rate (AIR), total factor use, factor levels, and adverse events (AEs) were extracted. Eight HA trials representing 7 gene therapies and 211 patients and 12 HB trials representing 9 gene therapies and 184 patients were included. For HA, gene therapy resulted in an annualized decrease of 7.58 bleeding events (95% confidence interval [CI], -11.50 to -3.67) and 117.2 factor infusions (95% CI, -151.86 to -82.53) compared with before gene therapy. Factor VIII level at 12 months ranged from 10.4 to 70.31 IU/mL by 1-stage assay. HB gene therapies were associated with an annualized decrease of 5.64 bleeding events (95% CI, -8.61 to -2.68) and 58.92 factor infusions (95% CI, -68.19 to -49.65). Mean factor IX level at 12 months was 28.72 IU/mL (95% CI, 18.78-38.66). Factor expression was more durable for HB than HA; factor IX levels remained at 95.7% of their peak whereas factor VIII levels fell to 55.8% of their peak at 24 months. The pooled percentage of patients experiencing a serious AE was 19% (10%-31%) and 21% (10%-37%) for HA and HB gene therapies, respectively. No thrombosis or inhibitor formation was reported. AAV-based gene therapies for both HA and HB demonstrated significant reductions in ABR, AIR, and factor use.
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Registered trials
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