Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura.

Background: Acquired thrombotic thrombocytopenic purpura (TTP) is caused by aggregation of platelets on ultralarge von Willebrand factor multimers. This microvascular thrombosis causes multiorgan ischemia with potentially life-threatening complications. Daily plasma exchange and immunosuppressive therapies induce remission, but mortality and morbidity due to microthrombosis remain high.

von Willebrand factor inhibition improves endothelial function in patients with stable angina.

ALX-0081 is a novel nano-antibody inhibiting von Willebrand factor (vWF). We evaluated whether direct inhibition of vWF by ALX-0081 improves endothelial function. Stable patients (pts, n = 55) with single vessel disease undergoing percutaneous coronary intervention (PCI) were randomized to ALX-0081 (n = 38) or placebo (n = 17).