Refractory Thrombotic Thrombocytopenic Purpura to Therapeutic Plasma Exchange

Abstract

Thrombotic thrombocytopenic purpura (TTP) is a rare, potentially fatal hematologic disorder characterized by microangiopathic hemolytic anemia, thrombocytopenia, and varying signs of visceral ischemia secondary to microvascular thrombosis. TTP is caused by a severe deficiency of ADAMTS13, a protease enzyme responsible for cleaving von Willebrand-factor (vWF) multimers. First-line therapy with plasmapheresis has increased survival rates immensely; however, there are few reported cases that are refractory to standardized treatment. We describe two cases of refractory TTP successfully managed with the addition of caplacizumab, an anti-von Willebrand factor immunoglobulin fragment that inhibits the interaction of vWF multimers with platelets.