Long-term follow-up of patients treated with caplacizumab and safety and efficacy of repeat caplacizumab use: Post-HERCULES study
| العنوان: | Long-term follow-up of patients treated with caplacizumab and safety and efficacy of repeat caplacizumab use: Post-HERCULES study |
|---|---|
| المؤلفون: | Scully, Marie, de la Rubia, Javier, Pavenski, Katerina, Metjian, Ara, Knöbl, Paul, Peyvandi, Flora, Cataland, Spero, Coppo, Paul, Kremer Hovinga, Johanna A, Minkue Mi Edou, Jessica, De Passos Sousa, Rui, Callewaert, Filip, Gunawardena, Sriya, Lin, Julie |
| المصدر: | Journal of thrombosis and haemostasis : JTH r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe instname |
| بيانات النشر: | WILEY-BLACKWELL, 2022. |
| سنة النشر: | 2022 |
| مصطلحات موضوعية: | 340 Recht, 610 Medizin und Gesundheit |
| الوصف: | INTRODUCTION Caplacizumab demonstrated efficacy and safety in patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in the Phase 3 HERCULES trial. However, data on long-term outcomes following caplacizumab treatment are limited. OBJECTIVES The post-HERCULES trial (NCT02878603) evaluated long-term outcomes of patients with iTTP treated with caplacizumab in HERCULES and safety and efficacy of repeated caplacizumab use. PATIENTS/METHODS Over 3 years' follow-up, patients could receive open-label caplacizumab with therapeutic plasma exchange (TPE) and immunosuppressive therapy (IST) in case of recurrence. Adverse events (AEs) were assessed during the overall study period (intention-to-observe [ITO] population) and during recurrences (recurrence population). TTP-related events (TTP-related death, recurrence, major thromboembolic events) were assessed in the efficacy ITO population (patients without recurrence during HERCULES or before post-HERCULES). RESULTS Among 104 enrolled patients, incidences of AEs and serious AEs were similar between patients who had received caplacizumab+TPE+IST during HERCULES (n=75) and those treated with placebo+TPE+IST (placebo; n=29). TTP-related events occurred in 8% of patients (4/49) randomized to caplacizumab during HERCULES versus 38% (11/29) randomized to placebo. Nineteen patients had ≥1 recurrence; 13 of these were treated with caplacizumab. First recurrence episode was resolved or resolving for all patients treated with caplacizumab, including 9 patients with repeat caplacizumab use. All second recurrences (6/6) were resolved. Safety profile of caplacizumab for treatment of recurrence was consistent with HERCULES; most bleeding events were non-serious. No major cases of organ dysfunction were observed. CONCLUSIONS Long-term follow-up supports the safety and efficacy of caplacizumab for iTTP and its repeated use for recurrences. |
| تدمد: | 1538-7933 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f9853292b7eb3490045c28b8a3a04a5cTest https://fundanet.iislafe.san.gva.es/publicaciones/ProdCientif/PublicacionFrw.aspx?id=17101Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....f9853292b7eb3490045c28b8a3a04a5c |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 15387933 |
|---|