Individualizing Treatment for Hemophilia A: A Key Approach in An Evolving Treatment Landscape
As more therapeutic options become available, individualizing treatment for hemophilia A becomes increasingly important.
Jonathan Roberts, MD: As the hemophilia landscape evolves, I think the individualization of hemophilia therapy becomes all the more important. I think, as I mentioned, the old paradigm of prophylaxis was infusing every other day or 3 times per week. It was a lot more cookie-cutter [approach], I would say. People would ascribe to that and didn’t really look at pharmacokinetic profiles that much because there wasn’t really an onus there. There wasn’t really a need to. And I think the community accepted the fact that breakthrough bleeding was inevitable. So you did your best to try to prevent as much as you could. And then as you’ve had an aging hemophilia population, we see that even a few joint bleeds, even 1 joint bleed has the potential to cause lifelong morbidity, and our mandate should be to prevent bleeds, period, in people who have bleeding disorders. So with all of the new options, as I mentioned, you know, factor VIII therapy and nonfactor therapies, gene therapies, the tools in the toolbox and the choices that patients and clinicians have to make is a lot more than it ever has been. So there’s a lot of education that needs to happen for both patients and clinicians to understand the nuances of each of these therapeutic options. They ultimately need to be comfortable with the knowns and unknowns about whatever option they choose and know how to manage bleeding when it occurs or trauma or surgery when it happens. So, I think, as I said, it’s an exciting time. There are lots of changes and developments, but I think it makes people like myself in the field and our mandate to advance the field through better clinical translational basic science research is certainly what I’m devoting the rest of my career to and I know many other colleagues are as well. But it really puts the onus on us to better understand how these therapies can enhance people’s lives with hemophilia.
Transcript edited for clarity.
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November 8th 2023Briana Contreras, an editor with Managed Healthcare Executive, spoke with Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, in this month's episode of Tuning in to the C-Suite podcast. The conversation was about how the disparity in diversity and ethnicity in rare disease clinical trials in the U.S. has led to gaps in understanding diseases and conditions, jeopardizing universal health, and increasing the economic burden of healthcare.
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October 18th 2024Statins have had an evolving role in the management of cardiovascular risk in people living with HIV, explained Michelle Cespedes, M.D., M.S., professor of medicine, Icahn School of Medicine at Mount Sinai and Mount Sinai Health System.
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