Redefining outcomes in immune TTP: an international working group consensus report

Abstract Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a potentially fatal microangiopathy caused by autoantibody-mediated severe deficiency of ADAMTS13. Standardized definitions response, exacerbation, remission, and relapse were initially proposed in 2003 modified the International Working Group for TTP 2017. These definitions, which have been widely used clinical practice research, are based primarily on platelet count benchmarked against timing discontinuation therapeutic plasma exchange (TPE). They do not incorporate ADAMTS13 activity or temporizing effects caplacizumab, novel anti–von Willebrand factor (VWF) nanobody. In light these limitations, IWG aimed to develop revised consensus outcome that anti-VWF therapy, using an estimate-talk-estimate approach. The updated distinguish remission (defined count) from activity). exacerbation only TPE but also therapy. Retrospective validation described, although they yet be prospectively validated. Clinical implications discussed example their application provided highlight relevance.