The potential role of emicizumab prophylaxis in severe von Willebrand disease.

Abstract

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.

• Authors: Assaf A Barg, Einat Avishai, Ivan Budnik, Tamar Barazani Brutman, Ilia Tamarin, Rima Dardik, Dalia Bashari, Mudi Misgav, Aharon Lubetsky, Shadan Lalezari, Tami Livnat, Gili Kenet
• Journal: Blood cells, molecules & diseases (Blood Cells Mol Dis) Vol. 87 Pg. 102530 (03 2021) ISSN: 1096-0961 [Electronic] United States
• PMID: 33341070 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2020 Elsevier Inc. All rights reserved.

Chemical References

• Antibodies, Bispecific
• Antibodies, Monoclonal, Humanized
• emicizumab
• Thrombin

Topics

• Adolescent
• Adult
• Antibodies, Bispecific (administration & dosage, therapeutic use)
• Antibodies, Monoclonal, Humanized (administration & dosage, therapeutic use)
• Blood Coagulation (drug effects)
• Child
• Female
• Hemarthrosis (blood, drug therapy)
• Hemostasis (drug effects)
• Humans
• Male
• Thrombin (analysis)
• Young Adult
• von Willebrand Diseases (blood, drug therapy)