In a post-hoc analysis of the HERCULES trial, caplacizumab, a nanobody that inhibits von Willebrand factor–platelet interaction, demonstrated efficacy regardless of iTTP history, disease severity, or initial immunosuppression regimen, but questions remain about long-term remission.
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening condition characterized by microvascular thrombosis due to ADAMTS13 deficiency. Standard treatment includes therapeutic plasma exchange (TPE) and immunosuppression, but outcomes remain suboptimal. Caplacizumab, a nanobody that inhibits von Willebrand factor–platelet interaction, demonstrated benefit in the phase 3, randomized HERCULES trial, in which 145 patients with acute iTTP received caplacizumab or placebo alongside standard therapy with steroids with or without rituximab (NEJM JW Oncol Hematol Jan 17 2019 and N Engl J Med 2019; 380:335).
Now, in a post hoc analysis, researchers evaluated the efficacy and safety of caplacizumab across clinically relev…
Reviewing Author
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute