Evaluating Emerging Gene Therapies to Further Treatment for Patients with Hemophilia A

Target Audience

This initiative is intended for hemophilia specialists, hematologists, emergency department clinicians, nurses, medical geneticists, genetic counselors, and other healthcare providers who care for patients with hemophilia A.

Program Overview

Hemophilia A is an inherited bleeding disorders due to the partial or total deficiency of coagulation factor (F) VIII. Progress in hemophilia therapy has been remarkable over the last decades with the development of efficacious therapies and extended half-life products based on prophylactic replacement of FVIII and improved understanding of the management of inhibitory antibodies to infused factor and the potential for gene therapy as a therapeutic option.

These activities will review the management of hemophilia A and examine the role of current and emerging therapeutic recommendations. Case vignettes will be presented to highlight the hematology care team’s role in the management of hemophilia A along with the importance of using shared decision-making strategies to help with patient and/or caregiver adherence to treatment plans.

Learning Objectives

Upon completion of this activity, participants should be better able to:

Identify emerging gene therapies for hemophilia A
Describe the potential implications of implementing gene therapies in practice
Recognize limitations of gene therapy