Predicting Outcomes and Optimizing Therapy in Thrombotic Thrombocytopenic Purpura: Insights on Caplacizumab Use from a Romanian Hematology Center

Background: Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy requiring prompt diagnosis and treatment. This retrospective single-center study analyzed 31 adult patients diagnosed between 2013 and 2024 (PLASMIC score ≥ 5, ADAMTS13 activity < 10%), aiming to characterize their clinical profiles and assess the impact of caplacizumab. Methods: Baseline laboratory parameters (platelet count, LDH, creatinine, hemoglobin, number of plasmapheresis sessions, number of hospitalization days, number of days in intensive care, and days required to recover platelet count) were included in statistical analysis to predict diverse outcomes, such as respiratory distress, infection, major neurological manifestations, gastrointestinal involvement, or refractoriness/exacerbation. Sixteen patients underwent treatment with caplacizumab in addition to plasmapheresis (PEX) and corticosteroids, while the remainder received PEX and corticosteroids alone. Results: Our predictive models proved noteworthy, providing results with ROC values ranging from 0.80 to 0.90 (p < 0.01). Caplacizumab was associated with faster platelet recovery (4 days vs. 7 days), fewer PEX sessions, shorter hospital stays, and a significantly lower incidence of refractoriness or exacerbation (p < 0.05). Inter-group analysis confirmed a significant reduction of overall resource use (p < 0.05). Conclusions: Early caplacizumab use improved outcomes and optimized resource utilization. This real-world study suggests that routinely available laboratory markers at presentation can help predict outcomes and guide early clinical decisions in centers without rapid ADAMTS13 testing.