Targeting the inhibitor of ADAMTS13 in thrombotic thrombocytopenic purpura

There has clearly been an evolution in the treatment of thrombotic thrombocytopenic purpura (TTP) over the past several years based upon a better understanding of the pathophysiology of this disorder. An autoantibody inhibitor of the ADAMTS13 protease and the resulting severe deficiency of ADAMTS13 function seem to be significant risk factors for the development of TTP, a disease increasingly recognized as an autoimmune disorder. Immune-based therapy aimed at suppressing the inhibitor of ADAMTS13 has been applied to the treatment of patients with TTP who are refractory to the standard treatment of plasma exchange and to patients with multiple recurrences of TTP. More recently, immunosuppressive therapy has been used alone as prophylactic therapy to prevent recurrences of acute TTP. This review focuses on the recent developments in the use of rituximab and ciclosporin in the treatment of TTP.