By Fiona Soltes
This year’s NHF Bleeding Disorders Conference includes a variety of compelling sessions and events. But for those who have or who care for someone with hemophilia B, there’s one “can’t miss” gathering.
Pre-Con: Hemophilia B – Pathways to Empowerment aims to check in on the ever-advancing landscape of treatment options including improved factor products, rebalancing agents and gene therapy. The session features Amy Board, director of patient engagement, Believe Limited and host of the BloodStream podcast; Mark T. Reding, MD, director of the Center for Bleeding and Clotting Disorders, University of Minnesota Medical Center; lived experience expert Bill Maurits, who had a “fantastic” experience participating in a gene therapy trial; National Youth Leadership Institute member Nathan Mermilliod, who is a patient of severe hemophilia B and advocate in the bleeding disorders community; BDC planning committee member Fel Echandi; and Donna Coffin, research and public policy director, World Federation of Hemophilia.
Hemophilia B patients have new choices in how they want to be treated and what they want their lifestyle to be, thanks in part to FDA approval of gene therapy. The session, then, will explore this from a number of angles: not only the view of someone who has had a positive experience with gene therapy, but also from someone who has had other positive experiences and is unsure what to do next, as well as someone who will address how language barriers can impact learning about and/or taking part in these opportunities.
“It’s a completely different landscape than it was before,” Board said. It calls for renewed focus on shared decision making and a thorough understanding the options. As such, the session will be interactive, with opportunities to ask questions and stimulate the brain, she said. In addition, WFH’s Coffin will introduce a decision-making tool.
Overall, the response of the community to the advances has varied greatly, depending on where people are on the spectrum, she said. Some more seasoned patients might be “stuck in the mud,” she said, with the attitude of “I’m fine, and I’ve been doing what I’ve been doing for years. I’m not going to change.”
“Then some of the people in their 30s and 40s, they’re really engaged with the landscape and meticulously going over data, which is wonderful. And the younger guys can be like deer in the headlights; their moms may have taken care of everything. As for the young parents, they’re ferociously asking questions.”
Board encourages attendees to think ahead and come with a solid list of questions. “This session is about you and your continued learning,” she said. “There have been some landmark times over the 75 years that NHF has been around when it comes to the evolution of treatment for bleeding disorders,” she said. “They’ve all been significant. This is probably the most significant. It truly is about a choice of how you’d like to be treated. As much as we’d like to say that has happened before, it hasn’t. Each drug does something different within our own coagulation mechanisms.” ■
Thursday, 2:40 p.m. – 4:50 p.m.
Consumer Track – English
Consumer Track – En Español
Chesapeake D-F | Ballroom Level