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Thank you for attending THSNA 2026. The virtual meeting is now closed.
Thank you for attending THSNA 2026. The virtual meeting is now closed.
Presentation Details
| Emicizumab for Severe von Willebrand Disease (VWD): the EmiVWD Study Enrollment 2026 Jonathan C.Roberts1, 2, May Chien3, Sweta Gupta4, Angela Weyand5, Rebecca Kruse-Jarres6, Erin Cockrell7, Fernando F.Corrales-Medina8, Vanessa Salinas9, Shannon L.Carpenter10, Erin Espinoza11, Abdulraheem Yacoub12, Dayna Lenski1, Sara Malik1, Sarah Kreitzer1, Michael D.Tarantino1, 2. 1Bleeding & Clotting Disorders Institute, Dills Family Foundation Center for Research at BCDI, Peoria, IL, USA.2Departments of Pediatrics and Medicine, University of Illinois College of Medicine at Peoria, Peoria, IL, USA.3Stanford University, Stanford, CA, USA.4Innovative Hematology, Indiana Hemophilia and Thrombosis Center, Indianapoils, IN, USA.5University of Michigan Medicine, Ann Arbor, MI, USA.6Washington Center for Bleeding Disorders, Seattle, WA, USA.7St.Joseph’s Hospital Center for Bleeding and Clotting Disorders, Tampa, FL, USA.8Division of Pediatric Hematology-Oncology, University of Miami Miller School of Medicine, Miami, FL, USA.9Center for Inherited Blood Disorders, Orange, CA, USA.10Children’s Mercy, Kansas City, MO, USA.11Tri-Health Cancer Institute, Cincinnati, OH, USA.12University of Kansas Cancer Center, Westwood Kansas, KS, USA |
Abstract
Background and Significance: Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, typically characterized by mucocutaneous bleeding. Prophylaxis for VWD with severe bleeding phenotypes currently has limited treatment options, and non-intravenous therapeutics are desired to tailor therapy. Emicizumab is a monoclonal, bispecific antibody that demonstrates factor VIII-like activity enhancing thrombin generation, transforming prophylactic therapy for many with hemophilia A. Emicizumab is administered subcutaneously less frequently than other currently available VWD therapeutics. Based on available literature emicizumab has been utilized successfully for VWD prophylaxis, and further investigation is warranted. Our objective is to evaluate the safety and efficacy of emicizumab for prophylaxis in severe VWD compared to the preceding 12-month bleed history. Study Design and Methods: We initiated a pilot multicenter, prospective open-label study (NCT05500807) to evaluate emicizumab prophylaxis in severe VWD type 3, or VWD with VWF antigen (VWF:Ag), VWF activity (VWF:RCo or VWF:GPIbM), or VWF collagen binding (VWF:CB) ≤20 IU/dl or variant VWD confirmed by genetic mutation or additional VWF activity assays (ie. VWF platelet binding, VWF:FVIII binding, VWF propeptide), or VWD with concomitant hemophilia A defined as VWF:Ag, VWF activity, or VWF:CB <50 U/dl, and mild, moderate or severe hemophilia A based upon historical medical records, with indication for hemostatic prophylaxis. Targeted enrollment is 40 patients of any age (≥3 kg). Exclusion criteria include patients with non-severe VWD, other bleeding disorders, renal and/or hepatic impairment, emicizumab treatment in the previous 18 months, or previously treated thromboembolic disease in the past 12 months. Pre-investigation annualized bleed rate and hemostatic therapies are evaluated with one-year retrospective chart review, collected at enrollment. Patients then receive Emicizumab, 3mg/kg weekly for 4 consecutive weeks, followed by once weekly dosing of 1.5mg/kg for 52 weeks. Dose up-titration to 3 mg/kg once weekly will be allowed if suboptimal efficacy. Treatment records, safety and tolerability are monitored along with bleeding events. Breakthrough bleeds may be treated with the patient’s usual on-demand products per investigator’s discretion. Central clinical laboratory testing will be completed, in addition to genetic testing. Patient-reported outcomes (PROMIS-29 PROs, SF-36) will be gathered to gain understanding on treatment impact. Analysis will be performed through descriptive statistics to determine proof of principle, with bleeding evaluated prior to and after emicizumab prophylaxis. End of study is expected to occur 18 months after the last patient’s first dose of study drug, to include a 6-month post-emicizumab prophylaxis follow-up. The primary hypothesis is that emicizumab is safe and efficacious for prophylaxis in VWD. Secondary objectives include evaluation of treatment burden vs VWF concentrate prophylaxis, bleed rate and severity, VWF qualitative defects or genetic mutations that may have impact on emicizumab clinical response. Nine centers in the United States have currently been enrolling patients, with 19 patients enrolled to date. Enrollment is currently ongoing. Conclusions: This ongoing pilot study is the first prospective investigation of emicizumab in patients with severe VWD. This study will shed light on feasibility, safety and potential efficacy of emicizumab prophylaxis in this patient population.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.