Idiopathic thrombocytopenic purpura in childhood

Idiopathic thrombocytopenic purpura (ITP) is a bleeding disorder characterised by shortened platelet survival. ITP is heterogeneous and is affected by endogenous and acquired factors. The diagnosis is one of exclusion, meaning that differential diagnosis is always important. Because of the unknown aetiology and the lack of clinical data collected from controlled prospective studies, the diagnosis and management of patients with ITP are controversial. The objective in prospective trials has been to alter the velocity of platelet increase. This study goal is frequently extrapolated to clinical situations, but no evidence has been found to show that a fast increase in the platelet count is clinically significant. The treatment of a patient with ITP is generally directed at preventing life-threatening haemorrhages, which are very rare in childhood. The goals of management and the study end-points in clinical trials should be re-evaluated. Important aspects are bleeding, quality of life for the patient and adherents, side effects of drugs and economic matters.