Thrombotic thrombocytopenic purpura (TTP) is a rare disease, especially in childhood, and has a high mortality rate in the absence of appropriate treatment. It is characterised by microangiopathic haemolytic anaemia and consumptive thrombocytopenia. TTP may be difficult to distinguish from haemolytic uraemic syndrome (HUS) because of similar clinical manifestations and laboratory findings. In the past, TTP and HUS have often been considered to represent variable expressions of a single entity. Our increased understanding of the pathogenesis of TTP has in turn resulted in significant improvements in its treatment and outcomes. Several immunomodulating agents are currently being used with variable outcomes. Pediatr Blood Cancer 2009;53:537–542. © 2009 Wiley-Liss, Inc.