BetaEntity Annotation Prototype
← Back to diseases

Annotated abstract

4CPS-117 Rituximab and caplacizumab in thrombotic thrombocytopenic purpura: 11 years of real-world evidence

ejhpharm · 2026-03-18 · canonical JSON source

15 visible annotations · policy: published · automated confidence ≥ 75.00%

Document resource

Background and Importance Thrombotic thrombocytopenic purpura (TTP) is a rare and serious disease. Initial treatment includes plasma exchange and corticosteroids, with relapses occurring in 20–50% of cases. Rituximab and caplacizumab (available since 2018) are currently used in clinical practice; however, real-world evidence remains limited.Aim and Objectives The aim of this study is to analyse the effectiveness of these treatments in reducing relapses and length of hospitalisation in adult patients with TTP in a tertiary hospital setting.Material and Methods Retrospective and observational study of patients diagnosed with TTP from 2014 to 2025. Collected variables were: demographic data (age, sex), treatment (drug, dosage, start date), effectiveness (relapse, retreatment, length of hospitalisation).Results A total of 30 episodes in 22 patients were included (2014–2017: 8; 2018–2025: 22). The mean age was 52.5 ± 13.8 years, and 54.5% (n=12) were women. All patients received one cycle of rituximab; it was administered a median of 6.3 days after admission in 27 episodes, while in three episodes it was given in the outpatient setting. The regimens used were four doses of: 375 mg/m 2 per week (n=23; 76.6%), 375 mg/m2 every 4 days (n=4; 13.3%), and 100 mg/m2 per week (n=3; 10.0%). Five patients (22.7%) experienced relapse, three of them had two relapse episodes each. Caplacizumab was used in addition to the initial treatment, in seven episodes (23.3%), a median of 4.7 days after admission. The regimen was daily (except for one patient every 48 h) with a mean duration of 19.5 days. The average length of hospitalisation was 20.4 days, with no differences between patients treated with caplacizumab and those who were not, from 2018 onwards (20.7 vs 19.8).Conclusion and Relevance In our cohort, rituximab did not show a clear reduction in relapse rates compared with those reported in the general TTP population and caplacizumab did not demonstrate a reduction in the length of hospitalisation. However, the small number of patients, particularly those treated with caplacizumab, limits the strength of these findings. Larger studies are needed to better define the role of these therapies in TTP management.Conflict of Interest No conflict of interest