Individualized prognosis in childhood immune thrombocytopenia

Schmidt, D.E. (2022)

Doctoral Thesis

Children with immune thrombocytopenia (ITP) show a primary hemostasis defect due to reduced number of platelets (thrombocytopenia). This leads to bleeding. In children, ITP is often transient and self-limiting (transient ITP), but some children show persistent thrombocytopenia or even chronic ITP. Treatment with immune-modulating medication like intravenous immunoglobulins (IVIg) leads to early recovery from thrombocytopenia and prevents bleeding. Unfortunately, treatment is only effective in a part of the patients. 
This dissertation aims to better understand, explain, and predict spontaneous recovery and favorable treatment outcomes after IVIg in a specific child with ITP. To that end, molecular disease mechanisms are being evaluated and analyzed together with clinical data. 
The dissertation provides novel data working towards individualized care for children with ITP. This is relevant for communication with the child and caregivers over the expected prognosis, treatment decisions (IVIg), and indications for early additional diagnostic testing for other causes of thrombocytopenia (such as genetic tests). 

• Immune Thrombocytopenia
• Children
• Molecular Epidemiology
• Prognosis

• All authors: Schmidt, D.E.
• Supervisor: Haas, M. de
• Co-supervisor: Bruin, M.C.A.; Vidarsson, G.
• Committee: Zwaginga, J.J.; Groenwold, R.H.H.; Huizinga, T.W.J.; Fischer, K.; Kuijpers, T.W.
• Qualification: Doctor (dr.)
• Awarding Institution: Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University
• Date: 2022-04-07
• Sponsorship: Landsteiner Foundation for Blood Transfusion Research (LSBR); Studienstiftung des Deutschen Volkes.