Five years of caplacizumab - Lessons learned and remaining controversies in immune-mediated thrombotic thrombocytopenic purpura

J Thromb Haemost. 2023 Aug 8:S1538-7836(23)00591-3. doi: 10.1016/j.jtha.2023.07.027. Online ahead of print.ABSTRACTImmune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare hematologic disease caused by autoantibodies against ADAMTS13 that trigger a microangiopathic hemolytic anemia. Therapeutic plasma exchange and glucocorticoids have been the mainstay of treatment for the past 30 years. In 2019, caplacizumab was approved as an addition to this regimen for the acute treatment of iTTP. Randomized controlled trials and real-world evidence have shown that caplacizumab reduces the time to platelet count normalization, refractoriness, and exacerbations of the disease, with an acceptable safety profile. In the past five years, there have been arguments against the upfront use of caplacizumab in all patients with iTTP, particularly related to the perceived lack of clinical benefit, safety concerns related to bleeding risk, and high costs. This perspective aims to address these concerns in the context of the experience of expert centers that have used the drug for more than five years.PMID:37562668 | DOI:10.1016/j.jtha.2023.07.027

Source: Thrombosis and Haemostasis - August 10, 2023 Category: Hematology Authors: Linus Alexander Volker Paul Thomas Brinkkoetter Spero R Cataland Camila Masias Source Type: research