Caplacizumab in acquired thrombotic thrombocytopenic purpura: a profile of its use

Abstract

Caplacizumab (Cablivi™), an anti-von Willebrand factor Nanobody®, is indicated for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression. When combining the results of phase 2 and 3 trials in patients with aTTP, caplacizumab reduced the time to normalization of platelet count compared with placebo, and was associated with reductions in the rate of aTTP-related death, aTTP recurrence or a major thromboembolic event. Although caplacizumab was associated with more bleeding-related adverse events than placebo, most of these events were of mild or moderate severity and did not require therapeutic intervention.