ArticleHaemophilia : the official journal of the World Federation of Hemophilia2021
Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 2 of them syntheses that pooled it.
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Who cites it
12 citing papers in PubMed, 2 syntheses or guidelines pooled it, 27 citations in OpenAlex.
- The Evolving Landscape of Discrete Choice Experiments in Health Economics: A Systematic Review.PharmacoEconomics · 2025Pooled it
- 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
- Estimating the Impact of Hemophilia A Treatment Administration on Patient Health Utility: Combining a Discrete Choice Experiment with Time Trade-Off Method.Advances in therapy · 2026Article
- Parental perception of treatment options for mucopolysaccharidosis: a survey to bridge the gap for personalized medicine.Orphanet journal of rare diseases · 2025Article
- Preferences for genetic interventions for SCA and Huntington's disease: results of a discrete choice experiment among patients.Orphanet journal of rare diseases · 2024Article
- Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey.Orphanet journal of rare diseases · 2024Article
- Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study.JMIR public health and surveillance · 2023Article
- Health literacy correlates with abbreviated full-scale IQ in adolescent and young adults with sickle cell disease.Pediatric blood & cancer · 2023Article
- Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making.Patient preference and adherence · 2023Article
- Gene Therapy and Hemophilia: Where Do We Go from Here?Journal of blood medicine · 2022Review
- Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- Perceptions and Recommendations Regarding Haemophilia B Gene Therapy: A Multistakeholder View From Patients, Caregivers and Healthcare Professionals.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
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Authors and funding
14 authors at 10 institutions in 7 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionGene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments.
aimWe conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes.
methodsWe surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21. Participants indicated preferences for gene therapy attributes including dosing frequency/durability, effect on annual bleeding, uncertainty related to side effects, impact on daily activities, impact on mental health, and post-treatment requirements. The relative importance of each attribute was analysed overall and for subgroups based on haemophilia type and severity.
resultsA total of 183 males with haemophilia A (n = 120) or B (n = 63) were included. Half (47%) had severe haemophilia; most (75%) were White. Overall, participants gave effect on bleeding rate the greatest relative importance (31%), followed by dose frequency/durability (26%), uncertainty regarding safety issues (17%), and impact on daily activities (11%). Dose frequency/durability had the greatest importance for those with haemophilia B (35%).
conclusionPeople with haemophilia prioritised reduced bleeding and treatment burden; the former was more important in haemophilia A and the latter in haemophilia B, followed by safety and impact on daily life in this DCE of gene therapy attributes. These findings and differences can inform clinical and health policy decisions to improve health equity for people with haemophilia.
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