Revisão sistemática sobre o papel do caplacizumab no tratamento da púrpura trombocitopénica trombótica

Abstract

Background: Acquired thrombotic thrombocytopenic purpura is a rare disorder in which clots form in small blood vessels, and may even block blood flow to downstream organs. Caplacizumab is a humanised bivalent nanobody that prevents the ultralarge von Willebrand factor-mediated platelet adhesion. The goal of this review is to analyze the effect of the inclusion of caplacizumab in the treatment of acquired thrombotic thrombocytopenic purpura. Methods: The systematic review of the literature was carried out in several databases with the keywords "caplacizumab" and "thrombotic thrombocytopenic purpura". We considered different study designs, in which was reported the clinical follow-up of individuals diagnosed with thrombotic thrombocytopenic purpura and whose therapeutic scheme included caplacizumab. All steps outlined by the PRISMA guidelines were completed and described. Results: We included 8 articles that met the eligibility criteria, published between 2016 and 2020, only 2 of which consisted of original clinical trials and all the others used these as a model for comparison. Discussion: The efficacy and safety of caplacizumab in adults experiencing an episode of aTTP were established in 2 randomised, controlled studies. The therapeutic scheme defined by the phase III study "HERCULES" was replicated in most of the other reported clinical cases, including in the pediatric stratum, except for those that excluded plasmapheresis. Conclusion: There are benefits to including caplacizumab in the therapeutic regimen of acquired thrombotic thrombocytopenic purpura, but further studies are needed to better understand its effects. Keywords: ADAMTS13 Protein (MeSH); Plasma Exchange (MeSH); Purpura, Thrombotic Thrombocytopenic (MeSH); von Willebrand Factor (MeSH).