Since 2014, there have been a significant number of breakthroughs changing the face of treatment for persons with hemophilia.
“It means potentially less infusions, possibly higher factor levels, which can translate into less bleeding, less burden of treatment and a better quality of life,” explains Michelle Witkop, DNP, Head of Research/National Hemophilia Foundation.
It started with extended half-life clotting factors.
“The hemophilia B products have more prolongation than hemophilia A products. Regardless, one less infusion per week means 52 less infusions per year, and 52 less times that a parent must struggle with a child to give them an infusion. For the Hem B population, that extension can be up to two weeks, and sometimes longer.”
Researchers are looking at even newer products with unique mechanisms of action being introduced.
“These are referred to as non-factor replacement therapies. Instead of intravenous infusions several times a week, now we’re talking about subcutaneous injections similar to insulin injections anywhere from weekly to monthly. Clinical trials are showing that the bleed rates for the most severe complications in hemophilia are down tremendously. This is revolutionary.”
Dr. Witkop says gene therapy trials continue, and are showing strong success.
“They’re now in both Hemophilia A and B with sustained expression, as well as sustained duration.
“You hear of gene therapy being a cure, and it’s potentially a cure for the person being treated. But many may still need factor at points in their lives, and hemophilia may still be genetically transmitted to the person’s offspring.”
Through research, education and advocacy, the push continues for better treatments and prevention of complications. But much work remains.
“Because these advances are on the horizon doesn’t necessarily mean everyone will have access to them, will understand them or want to utilize them. This isn’t the end. The end is when we’ve really cured this disorder. When it’s totally eradicated.”