Presentation Details
| Surgical and procedural outcomes for patients with factor VIII deficiency taking Emicizumab prophylactically: A regional approach to data collection and analysis Janice Kuhn1, Butler Regina2, Mailie-Howell Lisa2, Druzgal Colleen3, Sennett Margy3. 1Virginia Commonwealth University, Richmond, VA, USA.2Children's Hospital of Philadelphia, Philadelphila, PA, USA.3University of Virginia, Charlottesville, VA, USA |
Abstract
Background
Emicizumab (Emi) is a recombinant, humanized bi-specific antibody directed against activated factor IX and factor X. Emi is used to bypass the need for Factor VIII (FVIII) to prevent bleeding in persons with hemophilia A (PWHA) with and without inhibitors. While Emi improves hemostasis and is effective in preventing bleeding, it does not normalize hemostasis and interferes with standard coagulation tests. Experience to date with surgeries is limited.
Objectives
Primary objective: To evaluate bleeding and clotting outcomes of patients with Hemophilia A (PWHA), with and without inhibitors on Emi, who underwent major and minor surgery.
Secondary objectives:
1) To examine the incidence of excessive bleeding or clotting in PWHA with and without inhibitors undergoing major and minor surgery with and without FVIII prophylaxis and/or bypassing agents.
2) To determine the amount of FVIII and/or bypassing agents’ utilization in these patients and to compare the utilization to bleeding or clotting outcomes.
Methods:
In 2021, the Mid-Atlantic Region III Hemophilia Treatment Centers (MAR) contracted through its core center with the American Thrombosis and Hemostasis Network (ATHN) to develop two data validation forms and data entry guides. The forms use the ATHN dataset (ADS), a de-identified data set derived from clinical data entered in ATHN Clinical Manager (CM) by hemophilia treatment centers, reducing the need for human subject research requirements and data re-entry. All data fields for this project were previously available in CM. The MAR Community of Practice Forum supported site recruitment and project implementation.
The data elements from the ADS that were analyzed in the project objectives include:
1) Demographic Data: Year of birth, race, ethnicity, sex, education level, employment status, first 3 numbers of zip code.
2) Clinical Data: Diagnoses, inhibitor status, medication usage, and surgery/procedure details including month and year, type, anatomical location, and outcome.
An Emi Surgery Project Committee from MAR received retrospective de-identified monthly data cuts for their review and analysis. Data analysis was descriptive in nature. The type of surgeries was classified into major and minor surgeries according to criteria established by Santagostino (Haemophilia 2015, 21, 24-40).
Results:
Sixty-two surgeries/procedures have been analyzed as of December 1, 2023. All sixteen of the major surgeries/procedures used factor prophylactically with an average of 4.47 days of factor (0-15 range). Two cases also used an antifibrinolytic. Two surgeries (prostatectomy and tonsillectomy) had unexpected bleeding, but neither required the administration of blood products. No clotting was reported.
Of the 32/46 minor surgeries/procedures, fourteen (10 ENT and 4 scopes) did not use any factor concentrate, and 22/46 used an antifibrinolytic. Of the 18 using factor concentrate, the average days of use was 2.9 days (range 1-7) with a mode of 1 day. No expected bleeding or clotting was reported.
Nineteen dental surgeries were reported. Eight did not use factor concentrate. Seventy-five percent of those used an antifibrinolytic (Figure 1).
Conclusion
The MAR Emi Surgery and Procedure Project provides real world data on the hemostasis management of patients taking Emi. This abstract presents preliminary results in an ongoing project.
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.
Emicizumab (Emi) is a recombinant, humanized bi-specific antibody directed against activated factor IX and factor X. Emi is used to bypass the need for Factor VIII (FVIII) to prevent bleeding in persons with hemophilia A (PWHA) with and without inhibitors. While Emi improves hemostasis and is effective in preventing bleeding, it does not normalize hemostasis and interferes with standard coagulation tests. Experience to date with surgeries is limited.
Objectives
Primary objective: To evaluate bleeding and clotting outcomes of patients with Hemophilia A (PWHA), with and without inhibitors on Emi, who underwent major and minor surgery.
Secondary objectives:
1) To examine the incidence of excessive bleeding or clotting in PWHA with and without inhibitors undergoing major and minor surgery with and without FVIII prophylaxis and/or bypassing agents.
2) To determine the amount of FVIII and/or bypassing agents’ utilization in these patients and to compare the utilization to bleeding or clotting outcomes.
Methods:
In 2021, the Mid-Atlantic Region III Hemophilia Treatment Centers (MAR) contracted through its core center with the American Thrombosis and Hemostasis Network (ATHN) to develop two data validation forms and data entry guides. The forms use the ATHN dataset (ADS), a de-identified data set derived from clinical data entered in ATHN Clinical Manager (CM) by hemophilia treatment centers, reducing the need for human subject research requirements and data re-entry. All data fields for this project were previously available in CM. The MAR Community of Practice Forum supported site recruitment and project implementation.
The data elements from the ADS that were analyzed in the project objectives include:
1) Demographic Data: Year of birth, race, ethnicity, sex, education level, employment status, first 3 numbers of zip code.
2) Clinical Data: Diagnoses, inhibitor status, medication usage, and surgery/procedure details including month and year, type, anatomical location, and outcome.
An Emi Surgery Project Committee from MAR received retrospective de-identified monthly data cuts for their review and analysis. Data analysis was descriptive in nature. The type of surgeries was classified into major and minor surgeries according to criteria established by Santagostino (Haemophilia 2015, 21, 24-40).
Results:
Sixty-two surgeries/procedures have been analyzed as of December 1, 2023. All sixteen of the major surgeries/procedures used factor prophylactically with an average of 4.47 days of factor (0-15 range). Two cases also used an antifibrinolytic. Two surgeries (prostatectomy and tonsillectomy) had unexpected bleeding, but neither required the administration of blood products. No clotting was reported.
Of the 32/46 minor surgeries/procedures, fourteen (10 ENT and 4 scopes) did not use any factor concentrate, and 22/46 used an antifibrinolytic. Of the 18 using factor concentrate, the average days of use was 2.9 days (range 1-7) with a mode of 1 day. No expected bleeding or clotting was reported.
Nineteen dental surgeries were reported. Eight did not use factor concentrate. Seventy-five percent of those used an antifibrinolytic (Figure 1).
Conclusion
The MAR Emi Surgery and Procedure Project provides real world data on the hemostasis management of patients taking Emi. This abstract presents preliminary results in an ongoing project.
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.