ATLANTA, Dec. 11 /PRNewswire/ -- Joints are the most common site of bleeding in hemophilia patients, with recurrent episodes leading to arthritis, chronic pain, and permanent loss of function. New research to be presented today during the 47th Annual Meeting of the American Society of Hematology (ASH) demonstrates the efficacy of a proactive regimen of infused Factor VIII, the blood-clotting protein hemophiliacs are unable to produce naturally.
“The availability of purified, safe Factor VIII has revolutionized the care of patients with hemophilia,” said James N. George, M.D., professor of medicine at the University of Oklahoma Health Sciences Center and President of ASH. “This study documents that Factor VIII is not only effective for treatment, but also effective for prevention of bleeding complications.”
Traditionally, children with hemophilia have been treated with infusions of Factor VIII during episodes of joint hemorrhaging, but researchers speculated that routine infusions of Factor VIII every other day may actually prevent this debilitating condition, helping children with the disorder stay healthier rather than simply treating joint hemorrhages when they occur.
Sixty-five boys with hemophilia were randomized into two study groups. Half received prophylactic infusions of Factor VIII, and the other group received Factor VIII treatment following an incident of hemorrhaging as an enhancement of standard care to minimize damage to the joint. The patients were studied for three-and-a-half to more than five years, until they were six years old.
The researchers found that those receiving prophylaxis had an 84 percent reduction in the risk of joint damage by age six. Children on prophylaxis also had improved joint function and fewer joint hemorrhages per year -- less than one on average -- compared to five per year in the other treatment arm. However, in this study joint damage showed only modest correlation with the number of joint hemorrhages, and children not receiving prophylaxis were at risk for joint damage even if they had no or few recognizable joint hemorrhages. One consideration is that the children on prophylaxis used more Factor VIII; on average, each boy had 163 infusions per year compared to 47 in the treatment-on-incidence group. Infusions of Factor VIII are painful to young children, stressful to parents, and very expensive.
“In this study, we saw a significant improvement in the physical well-being of the boys who were provided with prophylaxis,” said Marilyn Manco-Johnson, M.D., professor of pediatrics and director, Mountain States Regional Hemophilia and Thrombosis Center, University of Colorado at Denver Health Sciences Center. “We hope these results will lead to a change in hemophilia care that will help other children with the disorder have a better quality of life.”
The American Society of Hematology (http://www.hematology.org) is the world’s largest professional society concerned with the causes and treatment of blood disorders. Its mission is to further the understanding, diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems, by promoting research, clinical care, education, training, and advocacy in hematology.
The American Society of Hematology
CONTACT: Leslie Priest of Spectrum Science Communications, +1-202-955-6222, lpriest@spectrumscience.com; or Aislinn Raedy of the American Societyof Hematology, +1-202-776-0544, araedy@hematology.org, On-site(12/9-12/13): +1- 404-222-5705
Web site: http://www.hematology.org/