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Thesis defense David Schmidt

Individualized Prognosis in Childhood Immune Thrombocytopenia

On 7 April 2022 (13:45) Sanquin researcher David Schmidt defended his thesis 'Individualized Prognosis in Childhood Immune Thrombocytopenia' at Leiden University

Promotor
Prof M de Haas MD PhD
Copromotor
MCA Bruin MD PhD and G Vidarsson PhD

Venue
Academy Building, Leiden University and online

Summary

Childhood immune thrombocytopenia (ITP) is an acquired autoimmune disease with humoral and cellular immune reactions against platelet self-antigens. ITP results in a bleeding tendency of varying severity, and leads to an impaired quality of life. The clinical course ranges from self-limiting (recovery within a few months after diagnosis) to development of chronic disease, lasting for minimally one year. It is a rare disease, which occurs in about 2-6 per 100,000 children annually. Although ITP occurs at any age, it is observed more frequently in children younger than six years. A key challenge is identifying a child’s disease course at the time of diagnosis. This may help to determine optimal clinical management, including whether to start treatment versus watchful waiting, the timing of additional diagnostic tests, and to counsel families appropriately.
In his research, David focused on possibilities to determine and explain prognosis of disease course in childhood ITP, at the time of diagnosis. He investigated if he could identify known or novel molecular markers, such as antibody characteristics and genotypes and immune markers, as prognostic factors in childhood ITP. He used clinical and molecular data from our Dutch large multicenter randomized clinical trial in childhood ITP (TIKI trial) in computational approaches for different prediction models. He initiated an international collaboration to validate a new clinical prediction score and made it available for use for treating physicians. 
David performed his research at Sanquin both in the group of Gestur Vidarsson at the Dept of Experimental Immunohematology in collaboration with Ellen van der Schoot; in the group of Masja de Haas in collaboration with Leendert Porcelijn at Immunohematology Diagnostics and with Anske van der Bom at the Center for Clinical Transfusion Research and Clinical Epidemiology of LUMC; all in close collaboration with Marrie Bruin and Katja Heitink-Pollé, both pediatrician haematologists at the Princess Maxima Center, Utrecht. Since 2020, David continued his training as specialist in pediatrics at the Astrid Lindgren Childrens Hospital, at the Karolinksa University Hospital, In Sweden.