New Delhi: Scientists have discovered important blood clotting factors in the patients suffering with the inherited bleeding disorder known as hemophilia B.
The research was presented at one of the ASH Annual Meeting.
After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing them to stop clotting factor infusions and to pursue normal activities of daily life without disabling bleeding episodes.
Lindsey A. George, the lead author of the study said, "Our goal in this trial was to evaluate the safety of the gene therapy product and secondarily, to determine if we could achieve levels of factor IX that could decrease bleeding events in patients.
"Adding "These patients have a severe or moderate level of hemophilia, with baseline clotting factor level less than or equal to 2 percent of levels in healthy people.
In current treatment, patients with hemophilia give themselves intravenous doses of factor IX up to a couple times a week.
While generally effective, factor levels fluctuate, and patients may suffer painful, disabling joint bleeds when their clotting factor levels drop.
Such a regimen requires significant planning of daily activities."In the current trial, the patients maintained factor levels of approximately 30 percent, enough to lift them out of the severe category.
"At these new levels, hemophilia patients do not typically need to self-treat with factor to avoid bleeding events," she said.Adding, "This represents a potential dramatic improvement in their quality of life and a shift in the way we think about treating hemophilia."
(With ANI inputs)