Med Clin (Barc). 2026 Aug 22;166(10):107588. doi: 10.1016/j.medcli.2026.107588. Online ahead of print.
ABSTRACT
BACKGROUND AND OBJECTIVE: Acquired or immune thrombotic thrombocytopenic purpura (iTTP) is a rare thrombotic microangiopathy caused by autoantibodies against ADAMTS13. The aim of this study was to evaluate the efficacy and safety of caplacizumab (CPZ) and rituximab (RTX) added to standard therapy in a real-world cohort.
METHODS: A single-center retrospective observational study of all adult patients with iTTP treated at Hospital Universitari de Bellvitge (2010-2024). Efficacy and safety variables were compared between patients treated with CPZ added to standard therapy versus those who were not, between patients who received RTX versus those who did not, and finally between quadruple therapy (corticosteroids [CS] + plasma exchange [PEX] + CPZ + RTX) and dual therapy (CS + PEX).
RESULTS: Thirty patients were included (46 acute episodes). CPZ was significantly associated with a shorter time to clinical response (5 vs. 9days; P=.023), shorter time to remission (24.8 vs. 40.6days; P<.001), and fewer plasma exchange sessions (5.5 vs. 9.5 sessions; P<.001). Early administration (<72hours) was associated with a shorter hospital stay (11 vs. 19.5days; P=.012). RTX showed a reduction in the number of relapses (13.3% vs. 50%; P=.085), without reaching statistical significance. Both drugs showed favorable safety profiles. Quadruple therapy resulted in a shorter time to clinical remission (26.1±9.9 vs. 40.3±7days; P<.001) and a lower number of PEX sessions (6 [4-7] vs. 8 [7-15.8]; P=.034).
CONCLUSIONS: In iTTP, CPZ improves short-term outcomes, particularly when administered early. RTX is associated with a trend toward greater relapse-free survival. Quadruple therapy reduces the time to clinical remission and the number of plasma exchange sessions. In iTTP, the incorporation of targeted and personalized therapeutic advances into clinical practice is associated with an improvement in clinical outcomes.
PMID:42632382 | DOI:10.1016/j.medcli.2026.107588
