Bleeding Disorders Awareness

Healio spoke with Glaivy Batsuli, MD, about advances in the bleeding disorders sphere, including treatment options for patients with hemophilia A and the possibilities of gene therapy.

“I think one of the things that gene therapy provides is that it is another option for patients with hemophilia, particularly for those with severe and moderate disease where they have to do some sort of infusion to prevent bleeding,” Batsuli, assistant professor of pediatrics at Emory University and pediatric oncologist at Aflac Cancer and Blood Disorders Center and Children's Healthcare of Atlanta, said.

She also discussed:

• recent approvals for hemophilia A, highlighting the expansion of the initial 2017 FDA approval of a bispecific antibody therapy;
• an exciting treatment option for hemophilia B;
• new factor VIII, as well as non-factor, agents in the pipeline for both hemophilia A and B;
• areas of unmet need in bleeding disorders, including treatment options for patients with hemophilia B and focused research for von Willebrand disease, Glanzmann thrombasthenia and Bernard-Soulier syndrome;
• the role of gene-based therapies for treating hemophilia; and
• the meaning of awareness in the broader bleeding disorders community and its impact on where research into diagnostics and treatments are focused.

Disclosure:

Batsuli reports receiving honoraria for serving on advisory boards for Bayer, Genentech, Kedrion Biopharma, and Sanofi.