ArticleHaemophilia : the official journal of the World Federation of Hemophilia2025
UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
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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.
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Who cites it
11 citing papers in PubMed.
- Continuum of Care for Hemophilia: The Story of India.Indian journal of pediatrics · 2026Review
- Real-world provider experiences with hemophilia A gene therapy: administration of valoctocogene roxaparvovec.Research and practice in thrombosis and haemostasis · 2026Article
- Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice.Research and practice in thrombosis and haemostasis · 2026Article
- Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025).Blood science (Baltimore, Md.) · 2025Article
- Therapeutic innovations in hemophilia: the essential role of a positive reinvestment cycle.Blood advances · 2025Review
- Qualification Criteria of Gene Therapy for Haemophilia-Opinion of the EAHAD Gene Therapy Working Group.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- [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
- 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
- A Comprehensive Approach: Addressing Psychological Needs in Gene Therapy for Haemophilia.Haemophilia : the official journal of the World Federation of HemophiliaReview
- Defining the Role of Nurses in Gene Therapy for Haemophilia.Haemophilia : the official journal of the World Federation of HemophiliaArticle
- The emerging need to manage patient expectations of haemophilia gene therapy amidst media hype in the UK.Therapeutic advances in rare diseaseArticle
Corrections and comments
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Authors and funding
16 authors.
Funding
Abstract
introduction2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons with severe haemophilia A and severe to moderately severe haemophilia B and a third in 2024. Gene therapy is a transformative, irreversible treatment with long-lasting effects, necessitating development of new clinical pathways to ensure optimal outcomes.
aimTo develop a consensus framework and service specification for delivery of AAV gene therapy for haemophilia in adults within the UK using the hub-and-spoke model proposed by the European Association of Haemophilia and Allied Disorders and the European Haemophilia Consortium.
methodsThe UK Haemophilia Centre Doctors Organisation (UKHCDO) set up a working party to develop expert consensus guidance, working with NHS England to ensure alignment with NHS England commissioning and the national service specification.
resultsThese guidelines detail the patient pathway, counselling and governance requirements for the hub-and-spoke model. The national service specification requires the hub site to manage governance for AAV-based gene therapy. Proposed regional and national multidisciplinary teams will harmonize clinical practices incorporating expertise from various specialities and professional groups. Key requirements identified include standardized documentation and multidisciplinary collaboration. Nationally agreed patient information and counselling checklists will streamline the informed consent process and facilitate data collection for long-term safety and efficacy monitoring.
conclusionThese guidelines provide a structured framework for the delivery of liver-directed gene therapy. Whilst specific to the United Kingdom they provide a framework for the implementation of gene therapy in other countries for haemophilia and other monogenic disorders.
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