The potential role of emicizumab prophylaxis in severe von Willebrand disease
| Autor: | Assaf Arie Barg, Ilia Tamarin, Tami Livnat, Shadan Lalezari, Ivan Budnik, Mudi Misgav, Rima Dardik, Einat Avishai, Gili Kenet, Aharon Lubetsky, D. Bashari, Tamar Barazani Brutman |
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| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
Adult Male congenital hereditary and neonatal diseases and abnormalities medicine.medical_specialty Adolescent Antibodies Monoclonal Humanized Thrombin generation Gastroenterology 03 medical and health sciences Young Adult 0302 clinical medicine hemic and lymphatic diseases Internal medicine Arthropathy Antibodies Bispecific Hemarthrosis medicine Von Willebrand disease Humans Adverse effect Child Molecular Biology Blood Coagulation Emicizumab Hemostasis business.industry Thrombin Cell Biology Hematology medicine.disease von Willebrand Diseases 030104 developmental biology Coagulation Molecular Medicine Female business Ex vivo 030215 immunology |
| Zdroj: | Blood cells, moleculesdiseases. 87 |
| ISSN: | 1096-0961 |
| Popis: | Background Severe von Willebrand disease (VWD) may be associated with chronic joint damage and may require prophylactic therapy. Emicizumab is a humanized bispecific antibody, which mimics the function of coagulation factor VIII (FVIII), and it has been approved for prophylaxis in hemophilia A. Methods This is the first study assessing the potential future role of emicizumab as an alternative prophylactic treatment in patients with severe VWD, based upon a thrombin generation (TG) ex vivo analysis. We report 51 weeks of successful off label emicizumab prophylaxis in a child with severe VWD and recurrent hemarthroses and progressive arthropathy despite adherence to previous prophylaxis with replacement therapy. Results and conclusions Our work demonstrated that ex vivo spiking with emicizumab increased TG in plasma from patients with type 3 VWD. Similar TG results were observed in our treated patient, whose therapy was well tolerated without any adverse events. Both in vitro and ex vivo TG data support sufficient hemostasis without exceeding the range seen in healthy volunteers. Further collaborative studies on the efficacy and safety of emicizumab prophylaxis in severe VWD is warranted. |
| Databáze: | OpenAIRE |
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