Novel therapies in thrombotic thrombocytopenic purpura

ESSENTIALS: The standard of care for patients with TTP remains daily plasma exchange in addition to immune suppressive therapy. Despite the improved treatment options for TTP, the acute mortality of TTP remains between 15‐20%. Caplacizumab reduces the time to platelet recovery and the exacerbation rate in acute TTP. A better understanding of the cause and treatments of long‐term complications of TTP are needed. . 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.