J Lock1, E W de Bekker-Grob2, G Urhan1, M Peters3, K Meijer4, P Brons5, F J M van der Meer6, M H E Driessens7, P W Collins8, K Fijnvandraat3, F W G Leebeek9, M H Cnossen1. 1. Department of Paediatric Haematology, Erasmus University Medical Centre - Sophia Children's Hospital, Rotterdam, The Netherlands. 2. Department of Public Health, Erasmus University Medical Centre, Rotterdam, The Netherlands. 3. Department of Paediatric Haematology, Academic Medical Centre, Amsterdam, The Netherlands. 4. Department of Haematology, University Medical Centre Groningen, University of Groningen, Groningen, The Netherlands. 5. Department of Paediatric Haematology, Radboud university medical centre, Nijmegen, The Netherlands. 6. Department of Thrombosis and Haemostasis, Leiden University Medical Centre, Leiden, The Netherlands. 7. Netherlands Haemophilia Patient Society (NVHP), Nijkerk, The Netherlands. 8. Arthur Bloom Haemophilia Centre, School of Medicine, Cardiff University, Cardiff, UK. 9. Department of Haematology, Erasmus University Medical Centre, Rotterdam, The Netherlands.
Abstract
INTRODUCTION: Patients', parents' and providers' preferences with regard to medical innovations may have a major impact on their implementation. AIM: To evaluate barriers and facilitators for individualized pharmacokinetic (PK)-guided dosing of prophylaxis in haemophilia patients, parents of young patients, and treating professionals by discrete choice experiment (DCE) questionnaire. PATIENTS/ METHODS: The study population consisted of patients with haemophilia currently or previously on prophylactic treatment with factor concentrate (n = 114), parents of patients aged 12-18 years (n = 19) and haemophilia professionals (n = 91). DCE data analysis was performed, taking preference heterogeneity into account. RESULTS: Overall, patients and parents, and especially professionals were inclined to opt for PK-guided dosing of prophylaxis. In addition, if bleeding was consequently reduced, more frequent infusions were acceptable. However, daily dosing remained an important barrier for all involved. 'Reduction of costs for society' was a facilitator for implementation in all groups. CONCLUSIONS: To achieve implementation of individualized PK-guided dosing of prophylaxis in haemophilia, reduction of bleeding risk and reduction of costs for society should be actively discussed as they are motivating for implementation; daily dosing is still reported to be a barrier for all groups. The knowledge of these preferences will enlarge support for this innovation, and aid in the drafting of implementable guidelines and information brochures for patients, parents and professionals.
INTRODUCTION:Patients', parents' and providers' preferences with regard to medical innovations may have a major impact on their implementation. AIM: To evaluate barriers and facilitators for individualized pharmacokinetic (PK)-guided dosing of prophylaxis in haemophiliapatients, parents of young patients, and treating professionals by discrete choice experiment (DCE) questionnaire. PATIENTS/ METHODS: The study population consisted of patients with haemophilia currently or previously on prophylactic treatment with factor concentrate (n = 114), parents of patients aged 12-18 years (n = 19) and haemophilia professionals (n = 91). DCE data analysis was performed, taking preference heterogeneity into account. RESULTS: Overall, patients and parents, and especially professionals were inclined to opt for PK-guided dosing of prophylaxis. In addition, if bleeding was consequently reduced, more frequent infusions were acceptable. However, daily dosing remained an important barrier for all involved. 'Reduction of costs for society' was a facilitator for implementation in all groups. CONCLUSIONS: To achieve implementation of individualized PK-guided dosing of prophylaxis in haemophilia, reduction of bleeding risk and reduction of costs for society should be actively discussed as they are motivating for implementation; daily dosing is still reported to be a barrier for all groups. The knowledge of these preferences will enlarge support for this innovation, and aid in the drafting of implementable guidelines and information brochures for patients, parents and professionals.
Authors: Erna C van Balen; Marjolein L Wesselo; Bridget L Baker; Marjan J Westerman; Michiel Coppens; Cees Smit; Mariëtte H E Driessens; Frank W G Leebeek; Johanna G van der Bom; Samantha C Gouw Journal: Patient Date: 2020-04 Impact factor: 3.883
Authors: Alfonso Iorio; Andrea N Edginton; Victor Blanchette; Jan Blatny; Ana Boban; Marjon Cnossen; Peter Collins; Stacy E Croteau; Katheljin Fischer; Daniel P Hart; Shinya Ito; Joan Korth-Bradley; Stefan Lethagen; David Lillicrap; Mike Makris; Ron Mathôt; Massimo Morfini; Ellis J Neufeld; Jeffrey Spears Journal: Res Pract Thromb Haemost Date: 2018-05-20