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

Caplacizumab (CabliviT), an anti-von Willebrand factor Nanobody (R), 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.