Caplacizumab As New Paradigm-Changing Therapy For Patients With Autoimmune Thrombotic Thrombocytopenic Purpura (Attp): Real-World Data From Ttp Spanish Registry

Introduction: Autoimmune thrombotic thrombocytopenic purpura (aTTP) is a severe disease caused by the production of autoantibodies against von Willebrand factor (vWF)-cleaving ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin-1 motifs; 13th member of the family). Without functional ADAMTS13, endothelial cells-derived unusually large vWF (ULVWF) multimers are present and are responsible for platelet clumping in the microvasculature. Plasma exchange (PE) and immunosuppressive therapy with steroids and rituximab are the milestones of the treatment of this disease. Moreover, in 2018, nanobody caplacizumab was approved for the treatment of adult patients with an acute episode of aTTP. Our objective was to retrospectively review the efficacy and safety of the current use of caplacizumab in Spain.