0000000000739863
AUTHOR
Johanna A. Kremer Hovinga Strebel
Long-Term Safety and Efficacy of Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura (aTTP): The Post-HERCULES Study
Abstract Background: The post-HERCULES trial (NCT02878603) evaluated long-term safety and efficacy of caplacizumab in patients with acquired thrombotic thrombocytopenic purpura (aTTP; also known as immune-mediated TTP), and efficacy of repeated use of caplacizumab for aTTP recurrence. Methods: Patients who completed the HERCULES trial were invited to participate in the post-HERCULES study and attend twice-yearly visits for 3 years. In case of aTTP recurrence, patients could receive open-label (OL) caplacizumab with therapeutic plasma exchange (TPE) and immunosuppression (IST). Safety was assessed during the overall study period (intention-to-observe [ITO] population) and during recurrences.…
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…