Long-term Outcomes of Patients Treated With Caplacizumab for Immune-mediated Thrombotic Thrombocytopenic Purpura (ittp): the Post-hercules Study
Year: 2022
Type: article
Abstract: The efficacy and safety of caplacizumab (CPLZ) for patients (pts) with immune-mediated thrombotic thrombocytopenic purpura (iTTP; also known as acquired TTP) were demonstrated in the Phase 3 HERCULES trial, with a 28-day follow-up period after end of treatment. Post-HERCULES (NCT02878603) evaluated the long-term outcomes of pts with iTTP treated with CPLZ during HERCULES, and the safety and efficacy of repeated CPLZ use for iTTP recurrence. Over 3 years’ follow-up, pts could receive CPLZ with therapeutic plasma exchange (TPE) and immunosuppressive therapy (IST) for iTTP recurrence. Safety was assessed during the overall study period in the intention-to-observe (ITO) population; TTP-related events (TTP-related mortality, recurrence, or major thromboembolic events) were assessed in pts without recurrence in HERCULES or prior to post-HERCULES (efficacy ITO population). Safety and efficacy were also evaluated during recurrences. Of 104 pts enrolled, incidences of adverse events (AEs) were similar between pts treated with CPLZ+TPE+IST during HERCULES (n=75) and pts treated with TPE+IST only (n=29). TTP-related events occurred in 4/49 pts (8%) randomized to CPLZ vs 11/29 pts (38%) randomized to placebo. The first recurrence episode was resolved/resolving for all 13 pts treated with CPLZ for recurrence, including 9 pts with repeat CPLZ. The safety profile of CPLZ for recurrence was consistent with HERCULES. Over long-term follow-up, the safety profile of patients treated with CPLZ in combination with TPE+IST was generally similar to those who received IST+TPE only, with no observed increases in iTTP recurrence. Repeat use of CPLZ was efficacious, with no new safety concerns.
Institutions: Sanofi (Turkey), University College Hospital, Hospital Universitario Doctor Peset, St. Michael's Hospital, University of Toronto, University of Colorado Anschutz, University of Colorado Denver, Medical University of Vienna, University of Milan, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, The Ohio State University, Assistance Publique – Hôpitaux de Paris, Hôpital Saint-Antoine, University of Bern, University Hospital of Bern, Sanofi (Belgium), Sanofi (Portugal)
Language: English
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: diamond
OpenAlex ID: W4301397756