Advances in chemical pharmacotherapy for the treatment of pediatric immune thrombocytopenia.

INTRODUCTION: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder of heterogeneous pathophysiological mechanisms. Treatment endpoints include elevation of platelets and reduction of bleeding risk, elevation of quality of life, reduction of concomitant therapies and prevention from bleeding. Persistent and chronic ITP is more common in adults but occurs in children. Standard therapies include corticosteroids and immunoglobulins, both associated with side effects. There are new treatments, such as thrombopoietin-receptor agonists and promising investigational drugs.

AREAS COVERED: Experience from the management of adults is valuable for children with persistent and chronic symptomatic ITP. In this review first- and second-line therapies, but also investigational drugs for children with ITP are discussed.

EXPERT OPINION: Although time-consuming and based on experience, children with no or mild bleeding can be safely managed with a watch and wait strategy. Chronic symptomatic immune thrombocytopenia is an area of second-line treatments based on a highly individualized approach. Furthermore, there are investigational drugs, which may also be of benefit for children with chronic symptomatic ITP.