Presentation Details
| First Real-World Experience Administering Etranacogene Dezaparvovec Gene Therapy for People with Hemophilia B Matthew Ryan1, Andrea B.Miller1, Patrick O'Hearn1, Vidhi Desai2, Nathan Visweshwar3. 1Hemophilia Outreach Center, Green Bay, WI, USA.2CSL Behring, King of Prussia, PA, USA.3University of South Florida, Tampa, FL, USA |
Abstract
Background: Etranacogene dezaparvovec-drlb (CSL Behring) is the first gene therapy for hemophilia B to be approved by the United States Food and Drug Administration (2022), the European Medicines Agency (2023), and Health Canada (2023). The gene therapy treatment journey is a multistep process and requires coordination between multiple stakeholders within a hemophilia treatment center (HTC). The first patients to receive etranacogene dezaparvovec-drlb were successfully infused at the Hemophilia Outreach Center in Green Bay, Wisconsin and the University of South Florida in Tampa, Florida. Objective: To describe the process by which HTCs prepared their centers and patients for administration of etranacogene dezaparvovec-drlb. Results: Early education, both for HTC stakeholders and patients, was critical in addressing key steps in the treatment journey, including patient eligibility, preparation, administration, and post-administration monitoring, and long-term follow-up. The education was iterative, involved multiple stakeholders (physician, pharmacist, nurse, social worker, and patient) and required open communication among the HTC, patients, and manufacturer when appropriate. The manufacturer provided education in the format of ongoing trainings and a detailed handbook describing the gene therapy process as it relates to etranacogene dezaparvovec-drlb. From this education, HTCs developed their own center-specific protocols, checklists, and worksheets. These protocols provided clear guidance on how to manage every step of the process in addition to providing a contingency plan (e.g., for infusion reactions or transaminitis), ensuring the center’s preparedness for administration. Practical elements to site preparation included establishing the care team (including the hematologist, pharmacist, and nursing staff, among others), assigning roles for administration day, and rehearsing administration day in advance. The HTCs also ensured infusion reaction supplies were available if needed and demonstrated the financial viability of gene therapy for people with hemophilia to the institution’s finance department. Initial discussions between the hematologist and advanced practitioner to consult on patient eligibility occurred 4 to 8 weeks before infusion and covered liver health, prophylaxis compliance, likelihood of patient follow through, and insurance. After assessing eligibility and obtaining payer approval, details of the administration were discussed with the patient, including date, transportation, arrival time, duration of infusion and monitoring, and the possibility of admission. A handling and preparation policy was developed to ensure the gene therapy product was available for administration. During administration and for 3 hours post administration, patients were closely monitored for signs and symptoms of an infusion reaction. A post-treatment calendar was shared with the patient as a guide to follow-up visits for monitoring liver function and factor IX levels. Before patients left the infusion center, they were also given a corticosteroid prescription to use in the case of elevated liver function tests and a contact number at the HTC. Long-term follow-up care teams have maintained open communication with the patients. HTCs were encouraged to enroll their patients in the American Thrombosis & Hemostasis Network TRANSCENDS study for long-term data collection to characterize the safety and efficacy of etranacogene dezaparvovec-drlb. Conclusion: By implementing early and iterative education, developing center-specific protocols, and handling practical considerations, HTCs were able to successfully administer etranacogene dezaparvovec-drlb.
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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.