On outcomes for hemophilia

Balen, E.C. van (2022)

Doctoral Thesis

The high standard of Dutch hemophilia care and the availability of prophylaxis provide an opportunity to focus on health outcomes beyond mortality. Using both qualitative and quantitative methods, this thesis defined, measured and quantified relevant health outcomes for persons with hemophilia. In the first part of this thesis we showed that communication and information provision about treatment options and prophylaxis regimes may support persons with hemophilia in their decisions about current and future treatment products. This will likely result in improved bleeding outcomes. In the second part, we took the first steps towards value-based health care for hemophilia by defining a standard set of ten relevant health outcomes, including instruments to measure these outcomes. Routine measurement of the standard set may be implemented in clinical practice in order to further improve hemophilia care that adds value for patients. Already, the high standard of care has resulted in near-normal socio-economic participation of Dutch persons with hemophilia. Development of more sophisticated data collection tools will help to monitor relevant health outcomes over time.

• Hemophilia A
• Hemophilia B
• Decision-making shared
• Qualitative research
• Outcome assessment, health care
• Delivery of Health Care
• Patient Reported Outcome Measures
• Surveys and Questionnaires
• Validation study
• Patient participation

• All authors: Balen, E.C. van
• Supervisor: Bom, J.G. van der; Rosendaal, F.R.
• Co-supervisor: Gouw, S.C.
• Committee: Dekker, F.W.; Eikenboom, H.C.J.; Vries, M.C. de; Lingsma, H.F.; Terwee, C.B.
• Qualification: Doctor (dr.)
• Awarding Institution: Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University
• Date: 2022-11-30
• ISBN (print): 9789464585520
• Sponsorship: CSL Behring; Chipsoft