Community Practice Connections™: New Horizons in Hemophilia: Preparing for the Advent of Gene Therapy

Target Audience
This educational program is directed toward hematologists, specialty NPs, PAs, and RNs. Pediatricians, emergency medicine clinicians, and other HCPs involved in the management of patients with hemophilia are invited to participate.

Program Overview
Hemophilia A is caused by a mutation in the gene coding for coagulation factor VIII (FVIII), resulting in the loss of functional FVIII protein. FVIII is a critical cofactor for factor IX (FIX), which is deficient in patients with hemophilia B. Treatment of hemophilia requires lifelong use of clotting factor replacement therapy. Recent and ongoing clinical trials are currently investigating novel therapeutic approaches such as gene therapy to improve management of this condition.

This Community Practice Connections™ program provides an in-depth review of some of the key highlights from the live program New Horizons in Hemophilia: Preparing for the Advent of Gene Therapy held in December 2021. This unique and engaging multimedia activity is ideal for the community-based clinician and focuses on the practical aspects of managing patients with hemophilia, putting recent trial data into a clinical context. The program is designed for those who did not attend the live meeting and to help reinforce learnings for those who did.

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

Describe the role of gene therapy for the treatment of hemophilia A and B
Evaluate clinical trial findings investigating the role of gene therapy in patients with hemophilia A and B
Assess current evidence regarding the relationship between cost-effectiveness data and access barriers for gene therapy
Select appropriate therapies for patients with hemophilia based on patient-centered considerations