ArticleHaemophilia : the official journal of the World Federation of Hemophilia2022
Examining patient and professional perspectives in the UK for gene therapy in haemophilia.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 13 citations in OpenAlex.
- Preferences for Advanced Therapy Medicinal Products: Understanding the Published Literature on the Value of Innovative Health Interventions.Inquiry : a journal of medical care organization, provision and financingPooled it
- Acceptance of Cell and Gene Therapy Among Patients with Hematological Diseases in China: A Cross-Sectional Study.Patient preference and adherence · 2026Article
- Parental perception of treatment options for mucopolysaccharidosis: a survey to bridge the gap for personalized medicine.Orphanet journal of rare diseases · 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
- Patient Perceptions of Emerging Gene Therapies for Arrhythmogenic Right Ventricular Cardiomyopathy.Circulation. Genomic and precision medicine · 2024Article
- 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
- Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2024Article
- Infrastructural considerations of implementing gene therapy for hemophilia in the Nordic context.Therapeutic advances in hematology · 2023Article
- Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.Patient preference and adherence · 2023Article
- Examining patient and professional perspectives in the UK for gene therapy in haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
14 authors at 6 institutions in 2 countries.
Funding
Abstract
introductionWith the development of gene therapy for people with haemophilia (PWH), it is important to understand how people impacted by haemophilia (PIH) and clinicians prioritise haemophilia treatment attributes to support informed treatment decisions.
objectiveTo examine the treatment attribute preferences of PIH and clinical experts in the United Kingdom (UK) and to develop a profile of gene therapy characteristics fit for use in future discrete choice experiments (DCEs).
methodsSemi-structured interviews were conducted with PIH (n = 14) and clinical experts (n = 6) who ranked pre-defined treatment attributes by importance. Framework analysis was conducted to identify key themes and treatment attributes; points were allocated based on the rankings. Synthesis of results by a multidisciplinary group informed development of a profile of gene therapy characteristics for use in future research.
resultsKey themes identified by PIH and clinical experts included patient relevant features and the importance of 'informed decision making'. The six top-ranked treatment attributes were 'effect on factor level' (79 points), 'uncertainty regarding long-term risks' (57 points), 'impact on daily life' (41 points), 'frequency of monitoring' (33 points), 'impact on ability to participate in physical activity' (29 points), and 'uncertainty regarding long-term benefits' (28 points). The final treatment characteristics were categorised as therapeutic option, treatment effectiveness, safety concerns, impact on self-management and quality of life (role limitations).
conclusionWe identified several gene therapy characteristics important to PIH and clinicians in the UK. These characteristics will be used in a future DCE to further investigate patient preferences for gene therapy.
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What OpenQuestion holds
Registered trials
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.