Presentation Details
| CHIEF: A retrospective self-control study of Children <12 with severe Hemophilia a without Inhibitors comparing Emicizumab to FVIII Daniel Mashiach1, 2, Patrice Mead1, Kendall Carneiro1, Jemily Malvar1, Guy Young1, 3. 1Children’s Hospital Los Angeles, Los Angeles, CA, USA.2WesternU College of Osteopathic Medicine of the Pacific, Pomona, CA, USA.3Keck School of Medicine, University of Southern California, Los Angeles, CA, USA |
Abstract
Background: Hemophilia A (HA) is an X-linked bleeding disorder caused by a deficiency in Factor VIII (FVIII). For severe HA (SHA), prophylaxis has become the standard of care, however it imparts a high treatment burden and typically still results in an annualized bleeding rate (ABR) of 2-6. Emicizumab, a subcutaneously administered FVIII substitute, has become the de facto standard of care prophylaxis for children with SHA. Previous clinical trials have done ABR analyses with emicizumab in patients greater than 12 years old and in children with inhibitors to FVIII who were previously on bypassing agents, but their is limited data on emicizumab in non-inhibitor SHA children. This study aims to examine the ABR in pediatric patients who switched from FVIII to emicizumab prophylaxis. Objectives: To compare ABR, safety, costs, joint health, and physical activity of individuals <12 years old with SHA without inhibitors on FVIII prophylaxis therapy after they switch to emicizumab prophylaxis. Methods: Using a retrospective electronic medical record chart review, we conducted a self-control analysis of 15 patients less than 12 years of age during equivalent periods of factor versus emicizumab prophylaxis collecting the data stated in the objectives above. Results: The mean ABR on FVIII and emicizumab was 1.79 and 1.13, respectively, however substantially lower joint (FVIII, 0.94; emicizumab, 0.33) and spontaneous bleeds (FVIII, 0.79; emicizumab, 0.23). On FVIII, there were 74 bleeds in total for all subjects, of which 53% were mild and 47% were moderate bleeds, while on emicizumab there were 51 total bleeds, of which 51% were mild, 45% moderate, and 4% were severe bleeds. Nine administration safety events occurred to five patients while on FVIII, of which eight were due to port complications. No administration safety events were recorded for patients while administering emicizumab. The mean annual cost (based on wholesale acquisition cost) of FVIII prophylaxis was $515,340 (S.D. $199,540), compared to $328,410 (S.D. $137,230) for emicizumab prophylaxis. No notable changes in Hemophilia Joint Health Scores or physical activity were seen in patients during the duration of the study. Conclusions: Emicizumab resulted in an improved ABR, especially for joint and spontaneous bleeds, had fewer administration complications, and was substantially less expensive compared to FVIII prophylaxis.
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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.