ArticleHaemophilia : the official journal of the World Federation of Hemophilia2021
Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey.
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 14 papers, 1 of them a synthesis that pooled it.
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Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled 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
- The Impact of Video-Based Educational Materials with Voiceovers on Preferences for Glucose Monitoring Technology in Patients with Diabetes: A Randomised Study.The patient · 2023Trial
- Acceptance of Cell and Gene Therapy Among Patients with Hematological Diseases in China: A Cross-Sectional Study.Patient preference and adherence · 2026Article
- Public perceptions and willingness to accept somatic gene therapy: A Belgian survey study.Open research Europe · 2025Article
- CCR5 gene editing and HIV immunotherapy: current understandings, challenges, and future directions.Frontiers in immunology · 2025Article
- Patient Perceptions of Emerging Gene Therapies for Arrhythmogenic Right Ventricular Cardiomyopathy.Circulation. Genomic and precision medicine · 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
- Decision threshold models in medical decision making: a scoping literature review.BMC medical informatics and decision making · 2024Article
- Research Priorities to Increase Confidence in and Acceptance of Health Preference Research: What Questions Should be Prioritized Now?The patient · 2024Article
- How can patient preferences be used and communicated in the regulatory evaluation of medicinal products? Findings and recommendations from IMI PREFER and call to action.Frontiers in pharmacology · 2023Article
- How to translate and implement the current science of gene therapy into haemophilia care?Therapeutic advances in hematology · 2023Review
- Haemophilia gene therapy: experiences and lessons from treated patients.Orphanet journal of rare diseases · 2022Article
- Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Article
- 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
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesThe aim of the Patient preferences to Assess Value IN Gene therapies (PAVING) study was to investigate trade-offs that adult Belgian people with haemophilia (PWH) A and B are willing to make when choosing between prophylactic factor replacement therapy (PFRT) and gene therapy.
methodsThe threshold technique was used to quantify the minimum acceptable benefit (MAB) of a switch from PFRT to gene therapy in terms of 'Annual bleeding rate' (ABR), 'Chance to stop prophylaxis' (STOP), and 'Quality of life' (QOL). The design was supported by stakeholder involvement and included an educational tool on gene therapy. Threshold intervals were analysed using interval regression models in Stata 16.
resultsA total of 117 PWH completed the survey. Mean thresholds were identified for all benefits, but substantial preference heterogeneity was observed; especially for the STOP thresholds, where the distribution of preferences was bimodal. Time spent on the educational tool and residence were found to impact MAB thresholds. The most accepted (88% of PWH) gene therapy profile investigated in this study comprised of zero bleeds per year (vs. six for PFRT), 90% chance to stop prophylaxis, no impact on QoL, and 10 years of follow-up on side effects (vs. 30 for PFRT).
conclusionsResults from this study proved the value of educating patients on novel treatments. Moreover, preference heterogeneity for novel treatments was confirmed in this study. In gene therapy decision-making, preference heterogeneity and the impact of patient education on acceptance should be considered.
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