The impact of caplacizumab in the treatment of immune thrombotic thrombocytopenic purpura. A retrospective monocentric cohort study
Gaetano Giuffrida
BackgroundImmune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder caused by severe ADAMTS13 deficiency, leading to microvascular platelet-rich thrombi, hemolysis, and organ damage, mainly involving brain, heart, kidneys, and intestines. Standard treatment includes plasma exchange (PEX) and immunosuppressants. Caplacizumab (CAPLA), an anti-von Willebrand factor nanobody, was recently introduced into clinical practice.MethodsWe retrospectively analyzed 81 pa