Redefining outcomes in immune TTP: an international working group consensus report
Abstract Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a potentially fatal thrombotic microangiopathy caused by autoantibody-mediated severe deficiency of ADAMTS13. Standardized definitions of response, exacerbation, remission, and relapse were initially proposed in 2003 and modified by the International Working Group for TTP in 2017. These definitions, which have been widely used in clinical practice and research, are based primarily on the platelet count and are benchmarked against the timing of discontinuation of therapeutic plasma exchange (TPE). They do not incorporate ADAMTS13 activity or the temporizing effects on the platelet count of caplacizumab, a novel anti–von W…
Long-Term Renal Outcomes in Hereditary TTP Patients: Data from the International Hereditary TTP Registry
Abstract Introduction Hereditary thrombotic thrombocytopenic purpura (hTTP) is an ultra-rare thrombotic microangiopathy caused by autosomal recessively inherited severe ADAMTS13 deficiency. In respect to organ damage, we recently reported a heterogeneous clinical course with some patients having various degrees of organ damage while others are almost asymptomatic*. The long-term consequences of hTTP are still not fully known. Methods We analyzed the prevalence and development of renal disease in confirmed hTTP patients in the International Hereditary TTP Registry until July 2021. We studied the onset of kidney disease, the presence of additional co-morbidities and the possibility of a speci…