Pediatric immune thrombocytopenia: To treat or not to treat

Abstract

With the research described in this thesis we aimed to identify clinical and laboratory parameters that are involved in etiology, bleeding severity, response to therapy and clinical course in children with immune thrombocytopenia (ITP). We have learned that the majority of children with newly diagnosed ITP, even in case of mild mucosal bleeding, do not need immune modulating treatment in order to raise platelet counts and will recover within several weeks to months without bleeding complications and with a good health-related quality of life. However, in children treated with intravenous immunoglobulin (IVIg), bleeding complications occurred significantly less often, so for a subgroup of patient, treatment with IVIg might be beneficial in order to provide adequate hemostasis. Pediatricians treating a child with newly diagnosed ITP should balance the risk of bleeding in the individual patient as well as the availability of 24/7 emergency care against the adverse effects and costs of medication to decide whether a watch and wait policy is justified or whether treatment is indicated. The challenge for the future will be to identify at diagnosis those children who have a higher risk of bleeding complications and who will respond to IVIg treatment, in order to treat only the small subgroup of patients with IVIg that will benefit from it. Based on our studies, this can be accomplished by using clinical characteristics, combined with results of platelet function tests and Fc-gamma receptor genetic profiles. In the end, we hope that our findings provide guidance to clinicians to make an evidence-based decision ‘to treat or not to treat’ their individual ITP patients.