Caplacizumab improves clinical outcomes and is well tolerated across clinically relevant subgroups of patients with immune-mediated thrombotic thrombocytopenic purpura.

Caplacizumab treatment of acute iTTP, in combination with therapeutic plasma exchange and immunosuppression, was safe and effective regardless of prior iTTP history, severity, or initial immunosuppression regimen and improved patient outcomes across clinically diverse subgroups. These findings emphasize the need for treatments with rapid onset of action that can reduce mortality and iTTP-related complications.