Treatment of Pediatric Primary Immune Thrombocytopenia With Thrombopoietin Receptor Agonists

Chronic immune thrombocytopenia (ITP) occurs in approximately one fifth of children with primary ITP and is characterized by a significant lack of clinical data. A minority of these children exhibit bleeding and need treatment. Often standard therapy used for patients with newly diagnosed ITP is administered to stop bleeding and to increase the platelet count. These drugs are associated with adverse effects, which is particularly evident when used during long time. In adult patients with chronic ITP, thrombopoietin receptor agonists (TPO-RAs) demonstrated efficacy in approximately 80% of patients. These drugs have been studied intensely for registration purposes; however, for children and adolescents they are not yet approved and studies are ongoing. First experiences with these drugs show similar effects and safety as in adults, though based on very small numbers of children. These drugs have the potential to be used during long time, in order to increase platelets, to stop or prevent bleeding and to augment quality of life, making long-term safety an important issue.