Caplacizumab and immunosuppression without plasma exchange in thrombotic thrombocytopenic purpura

Paul Coppo, M.D., Ph.D.
Saint-Antoine Hospital
Sorbonne University
Paris, France

Therapeutic plasma exchange (TPE) has long been central to the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP), but it remains an invasive intervention with considerable burden. In this presentation, Paul Coppo shares results from MAYARI, a phase 3, open-label, single-arm study evaluating caplacizumab in combination with immunosuppressive therapy (IST) as first-line treatment without TPE. Among patients in the modified intention-to-treat population, 93.5% achieved remission without requiring TPE. The median time to platelet count response was four days, and 97.8% of patients reached clinical response. Only two patients required TPE, and no recurrences or deaths were reported. Safety outcomes showed treatment-emergent adverse events in 90.9% of patients, with caplacizumab discontinued in four due to adverse events. These findings suggest that a non–TPE-based approach using caplacizumab and IST may be effective for most patients with acute iTTP.