Specialization
Pediatrics, pediatric hematology
Focus of research
My line of research focuses on antidrug antibodies against therapeutic factor VIII (i.e. inhibitors) in persons with hemophilia. Nowadays advanced treatment and even gene therapy are available for this congenital bleeding disorder. However, this is not attainable for children who develop inhibitors. Immune tolerance induction therapy to eradicate inhibitors is only effective in a subset of patients.
I initiated an international multicenter cohort study to identify genetic and non-genetic determinants of achieving immune tolerance after immune tolerance induction therapy. Furthermore, to gain more insight in the pathophysiology of factor VIII specific immune tolerance we study factor VIII specific dendritic and T cell responses in persons who did and who did not achieve immune tolerance.
I have a great interest in research on value based health care and E-health innovations to improve treatment outcomes for persons living with hemophilia. I designed and set up a nationwide study on clinical and psychosocial health outcomes, including a biobank. Now I follow the national hemophilia population in a hemophilia patient registry. To further improve data collection and data exchange for both individuals’ care as well as data driven research, we are developing a personalized health record.