Novel therapies in thrombotic thrombocytopenic purpura

Thrombotic thrombocytopenic purpura (TTP) is characterized by microangiopathic hemolytic anemia and a consumptive thrombocytopenia, as a result of severe deficiency of ADAMTS13. The standard of care of the acute episode is treatment with plasma exchange and immunosuppression. After the acute episode is resolved, patients face a significant risk of relapse and long-term complications associated with significant morbidity and even mortality. Novel treatments have been under development and will be discussed in this review. Caplacizumab, a nanobody that blocks the interaction between VWF and platelets, has shown promising results in decreasing the time to recover from the acute events that will hopefully translate into long-term clinical benefit for patients. In addition, identifying biomarkers to allow us to better predict the risk for relapse and the development of these long-term complications in patients with TTP are a few of the challenges that require our attention moving forward.