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Patient preferences...
Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey
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- van Overbeeke, Eline (författare)
- Clinical Pharmacology and Pharmacotherapy,University of Leuven, Leuven, Belgium
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- Hauber, Brett (författare)
- Health Preference Assessment, RTI Health Solutions, Research Triangle Park, North Carolina, USA; Comparative Health Outcomes, Policy and Economics (CHOICE) Institute, University of Washington School of Pharmacy, Seattle, WA, USA
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- Michelsen, Sissel (författare)
- Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium
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- Peerlinck, Kathelijne (författare)
- Haemophilia Centre, UZ Leuven, Leuven, Belgium
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- Lambert, Catherine (författare)
- Haemophilia Clinic, St-Luc University Hospital, Brussels, Belgium
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- Hermans, Cedric (författare)
- Haemophilia Clinic, St-Luc University Hospital, Brussels, Belgium
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- Lê, Phu Quoc (författare)
- Hémato-Oncologie, Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium
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- Goldman, Michel (författare)
- Institute for Interdisciplinary Innovation in healthcare, Université libre de Bruxelles, Brussels, Belgium
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- Simoens, Steven (författare)
- Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium
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- Huys, Isabelle (författare)
- Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium
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(creator_code:org_t)
- 2021-09
- 2021
- Engelska.
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516.
- Relaterad länk:
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https://doi.org/10.1...
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https://onlinelibrar...
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https://urn.kb.se/re...
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https://doi.org/10.1...
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Abstract
Ämnesord
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- Objectives: The 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.Methods: The 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. Results: A 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). Conclusions: Results 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.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Samhällsfarmaci och klinisk farmaci (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Social and Clinical Pharmacy (hsv//eng)
Nyckelord
- gene therapy
- haemophilia
- perspectives
- preference
- questionnaire
- survey
Publikations- och innehållstyp
- ref (ämneskategori)
- art (ämneskategori)
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van Overbeeke, E ...
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Hauber, Brett
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Michelsen, Sisse ...
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Peerlinck, Kathe ...
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Lambert, Catheri ...
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Hermans, Cedric
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Lê, Phu Quoc
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Goldman, Michel
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Simoens, Steven
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Huys, Isabelle
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