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Presentation Details
| Non-joint bleeds in patients with hemophilia A or B with inhibitors: Concizumab explorer7 study Amy Shapiro1, Ana Boban2, Renée Brown Frandsen3, Giancarlo Castaman4, Kingsley Hampton5, Keiji Nogami6, Jameela Sathar7, Senthil Vel3, Jerzy Windyga8, Emily Waters9, Victor Jimenez Yuste10, Allison Duchman9. 1Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA.2Haemophilia Centre, Department of Haematology, University Hospital Centre Zagreb, Zagreb, Croatia and School of Medicine, University of Zagreb, Zagreb, Croatia.3Novo Nordisk A/S, Søborg, Denmark.4Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy.5Department of Cardiovascular Science, University of Sheffield, Sheffield, United Kingdom.6Department of Pediatrics, Nara Medical University, Kashihara, Nara, Japan.7Department of Haematology, Ampang Hospital, Kuala Lumpur, Malaysia.8Department of Haemostasis Disorders and Internal Medicine, Laboratory of Haemostasis and Metabolic Diseases, Institute of Haematology and Transfusion Medicine, 02-776, Warsaw, Poland.9Novo Nordisk Inc., Plainsboro, NJ, USA.10Department of Haematology, La Paz University Hospital Coagulopathies and Disorders of Haemostasis Group IdiPaz, Autónoma University, Madrid, Spain |
Abstract
Background: Concizumab is an anti-tissue factor pathway inhibitor monoclonal antibody intended for hemophilia A/B with (HAwI/HBwI) and without inhibitors, approved in US for once-daily, subcutaneous prophylaxis in HAwI/HBwI. Objectives: To assess non-joint bleeds in the prospective, multicenter, open-label phase 3 explorer7 study (NCT04083781) in HAwI/HBwI during concizumab prophylaxis vs on-demand treatment as an exploratory analysis. Methods: Patients were randomized 1:2 to on-demand treatment or concizumab prophylaxis. On-demand patients switched to concizumab after ≥24 weeks. After a 1.0 mg/kg concizumab loading dose (Day 1), patients received a 0.20 mg/kg daily dose (Day 2+), with potential dose adjustment (5–8 weeks) to 0.15/0.25 mg/kg daily based on concizumab plasma concentration measured after week 4. Here we describe the location and number of non-joint bleeds during on-demand or concizumab treatment at the 32- and 56-week cut-offs in explorer7, and annualized bleeding rates (ABRs) for treated spontaneous and traumatic muscle bleeding episodes (includes re-bleeds and bleeds occurring contemporaneously) at the 32-week cut-off. Informed consent/ethics committee approval were obtained. Results: This exploratory analysis included 19 patients on-demand and 33 patients on concizumab. Amongst non-joint bleeds, muscle bleeds were most frequently reported, with 32 treated bleeds (17.6% of all treated bleeds) in 47.4% of on-demand patients and 4 bleeds (6.1% of all treated bleeds) in 12.1% of patients on concizumab (Table 1). The estimated ABR [95%-confidence interval] for treated spontaneous and traumatic muscle bleeding episodes in patients on-demand was 1.5 [0.78;2.99] vs 0.1 [0.02;0.31] on concizumab (ABR ratio: 0.05 [0.01;0.22]; p<0.001). Origin and severity of treated muscle bleeds are described in Table 2. At the 56-week cut-off (Table 1), low numbers of non-joint bleeds were maintained with concizumab. Conclusions: Few non-joint bleeds were reported with concizumab prophylaxis in explorer7 at the 32- and 56-week cut-offs. The ABR for muscle bleeding episodes was reduced in patients on concizumab compared with on-demand treatment. Previously presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress, Washington, DC, USA, 21-25 June 2025.
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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.