Whether treating a condition involves infusion therapy or is as simple as an aspirin, it's much more bearable if it doesn't have to be administered as often. For adults with severe or moderately severe hemophilia A, a rare, hereditary bleeding disorder that affects mostly males, a new option can make managing their condition a lot easier.
An international team led by researchers at Rush University Medical Center recently found that ADVATE, a treatment that replaces a clotting protein that is missing in patients with hemophilia A, is as effective for adults as it is for children. The study was published in the online version of the Journal of Thrombosis and Haemostasis in January.
Thanks to the study, ADVATE is now the only treatment that replaces the clotting protein in the blood that’s approved in the United States for preventive use in both adults and children with hemophilia A. Unlike other treatments in its class that must be given every other day, ADVATE can be administered every third day. Using ADVATE as a preventive treatment, which involves infusion on a regular schedule to prevent bleeding episodes, is considered superior to using it on an on-demand basis — treating bleeding episodes as they arise.
"The preventive therapy will optimize care for hemophilia patients of all ages by stopping unexpected bleeding that can have a detrimental impact on the lives of patients," says Leonard Valentino, MD, director of the Rush Hemophilia and Thrombophilia Center and principal investigator in the study. "An option that only needs to be administered every third day could increase compliance to a long-term medical regimen that is especially challenging."
Preventive Care Trumps On-Demand Treatment
Compliance with the use of preventive treatments such as ADVATE is particularly important because patients with severe hemophilia A are at extremely high risk for spontaneous bleeding into the joints, muscles and internal organs, as well as bleeding after surgery. In the absence of ADVATE or similar treatments, joint bleeding may occur as many as 20 to 30 times a year, resulting in hemophilia-related arthritis.
While on-demand treatment can slow the progression of hemophilia-related arthritis, it does not seem to prevent the condition because bleeding often causes damage before on-demand therapy can be administered. "A higher compliance rate, which is the hope with ADVATE, would mean fewer complications resulting from bleeding episodes," Valentino says.
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Please note: All physicians featured in Discover Rush Online are on the medical faculty of Rush University Medical Center. Some of the physicians featured are in private practice and, as independent practitioners, are not agents or employees of Rush University Medical Center.
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