Diagnosis and management of immune thrombocytopenia in childhood

Evidence-based medicine is growing in immune thrombocytopenia (ITP), but solid clinical data are still lacking in many areas. A majority of children has self-limited ITP, but chronic symptomatic ITP exists also in pediatrics. Management includes a watch-and-wait strategy for children with newly diagnosed ITP and no or mild bleeding, and immunoglobulins and corticosteroids, if more bleeding and mucous membrane involvement is present. Treatment endpoints differ in clinical research and in clinical practice. The requirement of platelet enhancing drugs needs to be better defined in guidelines. Second-line therapies for children are rarely required and include thrombopoietin-receptor agonists, rituximab, dexamethasone and immunosuppressants. Thrombopoietin-receptor agonists are successful in adult but also in pediatric ITP. The strategical position of splenectomy differs from that in adults. Although effective in children it is less frequently used because of its life-long cumulative risk of infectious diseases and a higher potential of spontaneous remission in ITP, providing a strong argument to defer splenectomy. The rarity of ITP makes clinical research expensive.