Factor VIII Treatment Video Perspectives

In these videos, Glaivy M. Batsuli, MD, assistant professor of pediatrics at Emory University School of Medicine and pediatric hematologist/oncologist at Aflac Cancer & Blood Disorders Center at Children’s Healthcare of Atlanta, discussed:

• Advances in the prevention and reduction of bleeding in patients with hemophilia A, such as the approval of more factor VIII replacement therapies and emicizumab (Hemlibra, Genentech);
• New clinical guidelines from leading hematology organizations on the diagnosis, management, treatment and future research priorities in von Willebrand disease – “I think there’s more to come now that there’s more focus on the disease;”
• How clinicians approach choosing plasma-derived or recombinant factor VIII products for patients;
• The importance of involving the families of the pediatric patient in the treatment selection and the clinician’s role in “guiding that decision and making sure that they have all of the necessary information;”
• The hemophilia community, which encompasses patients, families, scientists and physicians who “work together;”
• Social and developmental aspects of caring for infants and children with factor VIII deficiency;
• Ongoing clinical trials to determine whether gene therapy can “restore” the ability to make factor VIII; and
• The greatest areas of unmet need, such as reducing the risk of inhibitor inflammation, predicting who will develop an inhibitor and interventions for those who do in hemophilia A, and the need to increase understanding of prophylaxis in von Willebrand disease.