دورية أكاديمية
Journal of Thrombosis and Haemostasis / Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study
| العنوان: | Journal of Thrombosis and Haemostasis / Efficacy and safety of open-label caplacizumab in patients with exacerbations of acquired thrombotic thrombocytopenic purpura in the HERCULES study |
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| المؤلفون: | Callewaert, Filip, Knöbl, Paul, Cataland, Spero, Peyvandi, Flora, Coppo, Paul, Scully, Marie, Kremer Hovinga, Johanna A., Metjian, Ara, De la Rubia, Javier, Pavenski, Katerina, Minkue Mi Edou, Jessica, De Winter, Hilde |
| بيانات النشر: | Wiley |
| سنة النشر: | 2020 |
| المجموعة: | MedUni Vienna ePub (Medzinische Universität Wien) |
| مصطلحات موضوعية: | ADAMTS13 protein, caplacizumab, plasma exchange, purpura, thrombotic thrombocytopenic, von Willebrand factor |
| جغرافية الموضوع: | UMW:14557 |
| الوصف: | Background Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare, life-threatening autoimmune thrombotic microangiopathy. Caplacizumab, an anti-von Willebrand Factor Nanobody®, is effective for treating aTTP episodes and is well tolerated. Objectives and methods In the phase 3 HERCULES trial (NCT02553317), patients with aTTP received double-blind caplacizumab or placebo during daily therapeutic plasma exchange (TPE) and for ≥30 days thereafter. Patients who experienced an exacerbation while on blinded study drug treatment switched to receive open-label caplacizumab plus re-initiation of daily TPE. Exacerbations were defined as recurrence of disease occurring within 30 days after cessation of daily TPE. Results Thirty-one patients (placebo, n = 28; caplacizumab, n = 3) had an exacerbation during double-blind treatment. Twenty-eight patients switched to open-label caplacizumab (placebo, n = 26; caplacizumab, n = 2); the three others discontinued upon exacerbation. Median time to platelet count response (≥150 × 109/L) was 3.49 days upon receiving caplacizumab. There were no deaths. During open-label treatment, further exacerbation or a major thromboembolic event (vena cava thrombosis) was experienced by one patient (3.6%) each. Consistent with the double-blind phase, the most frequent treatment-emergent adverse events were catheter site hemorrhage (28.6%), headache (21.4%), and epistaxis (17.9%). Conclusions These results suggest that caplacizumab was efficacious and well tolerated in patients with aTTP who experienced a disease exacerbation during double-blind treatment in HERCULES. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | text/html |
| اللغة: | English |
| تدمد: | 1538-7836 |
| العلاقة: | vignette : https://repositorium.meduniwien.ac.at/titlepage/urn/urn:nbn:at:at-ubmuw:3-59949/128Test; urn:nbn:at:at-ubmuw:3-59949; https://resolver.obvsg.at/urn:nbn:at:at-ubmuw:3-59949Test; local:99146883357903331; system:AC16575811 |
| DOI: | 10.1111/jth.14679 |
| الإتاحة: | https://doi.org/10.1111/jth.14679Test https://resolver.obvsg.at/urn:nbn:at:at-ubmuw:3-59949Test |
| حقوق: | cc-by-nc_4 |
| رقم الانضمام: | edsbas.CC0A6CD1 |
| قاعدة البيانات: | BASE |
Full Text Finder | تدمد: | 15387836 |
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| DOI: | 10.1111/jth.14679 |