First Real-World Experience Administering Etranacogene Dezaparvovec Gene Therapy for People with Hemophilia B

Research in gene therapy for hemophilia B has been ongoing for decades. 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) and the European Medicines Agency (EMA) (2023). The gene therapy treatment journey is a multistep process and requires coordination between multiple stakeholders within a hemophilia treatment center (HTC). The first 2 patients to receive etranacogene dezaparvovec-drlb since approval were successfully infused at the Hemophilia Outreach Center in Green Bay, Wisconsin. Here we describe the process by which the Hemophilia Outreach Center prepared their center and patients for the administration of etranacogene dezaparvovec-drlb.

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 management. The education was iterative, involved multiple stakeholders (physician, pharmacist, nurse, social worker, and patient) and required an open channel of communication among the HTC stakeholders, the patients, and manufacturer when appropriate. Although no certification process was required, the manufacturer provided education to the HTC stakeholders in the format of ongoing trainings and a detailed handbook describing the gene therapy process as it relates to etranacogene dezaparvovec-drlb. This education provided a foundation for the HTC stakeholders to develop 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 for different scenarios (e.g., infusion reactions and transaminitis), ensuring the center's preparedness for administration.

By implementing early and iterative education and developing center-specific protocols, the Hemophilia Outreach Center was able to successfully administer etranacogene dezaparvovec-drlb. These elements are crucial to ensure smooth execution and optimal patient outcomes.