ReviewHaemophilia : the official journal of the World Federation of Hemophilia2019
How to discuss gene therapy for haemophilia? A patient and physician perspective.
Review in Haemophilia : the official journal of the World Federation of Hemophilia, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 papers.
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
52 citing papers in PubMed.
- Overview of Patient-Reported Outcomes in Haemophilia Gene Therapy.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- Psychometric properties and outcomes of the PROMIS Sexual Function and Satisfaction measures in individuals with congenital bleeding disorders.Research and practice in thrombosis and haemostasis · 2026Article
- Gene Therapy: Knowledge, Attitudes, and Preferences Among Individuals with Alpha-1 Antitrypsin Deficiency.Chronic obstructive pulmonary diseases (Miami, Fla.) · 2026Article
- Building a gene editing lexicon: a model for rare and inherited disorders.Gene therapy · 2026Article
- Transforming Hemophilia Management: Lessons from Gene Therapy Clinical Trials.Molecular biotechnology · 2026Review
- An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study.Therapeutic advances in hematology · 2026Article
- Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025).Blood science (Baltimore, Md.) · 2025Article
- A qualitative study on the impact of hemophilia on sexual functioning and well-being in Dutch men.Journal of patient-reported outcomes · 2025Article
- Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- [Chinese guidance for the clinical application of Adeno-associated virus vector-based gene therapy for hemophilia B (2025)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors.Journal of clinical medicine · 2025Review
- Development of a novel gene editing lexicon for hemophilia: methodology and results.Research and practice in thrombosis and haemostasis · 2025Article
- UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Role of Pharmacy Professionals in Gene Therapy Based on Adeno-Associated Viruses: Treatment of Hemophilia as a Template of Care.The Canadian journal of hospital pharmacy · 2025Review
- A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient.Orphanet journal of rare diseases · 2024Review
- Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy.Blood advances · 2023Article
- AAV induces hepatic necroptosis and carcinoma in diabetic and obese mice dependent on Pebp1 pathway.EMBO molecular medicine · 2023Article
- [Gene therapy of Hemophilia: Recommendations from the German, Austrian, and Swiss Society for Thrombosis and Haemostasis Research (GTH)].Hamostaseologie · 2023Article
- The evolving role of medical geneticists in the era of gene therapy: An urgency to prepare.Genetics in medicine : official journal of the American College of Medical Genetics · 2023Article
- Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia.Gene therapy · 2023Article
Corrections and comments
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Authors and funding
4 authors.
Funding
Abstract
Gene therapy has the potential to revolutionise treatment for patients with haemophilia and is close to entering clinical practice. While factor concentrates have improved outcomes, individuals still face a lifetime of injections, pain, progressive joint damage, the potential for inhibitor development and impaired quality of life. Recently published studies in adeno-associated viral (AAV) vector-mediated gene therapy have demonstrated improvement in endogenous factor levels over sustained periods, significant reduction in annualised bleed rates, lower exogenous factor usage and thus far a positive safety profile. In making the shared decision to proceed with gene therapy for haemophilia, physicians should make it clear that research is ongoing and that there are remaining evidence gaps, such as long-term safety profiles and duration of treatment effect. The eligibility criteria for gene therapy trials mean that key patient groups may be excluded, eg children/adolescents, those with liver or kidney dysfunction and those with a prior history of factor inhibitors or pre-existing neutralising AAV antibodies. Gene therapy offers a life-changing opportunity for patients to reduce their bleeding risk while also reducing or abrogating the need for exogenous factor administration. Given the expanding evidence base, both physicians and patients will need sources of clear and reliable information to be able to discuss and judge the risks and benefits of treatment.
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Registered trials
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