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Joint Outcome Study Results Published in the New England Journal of Medicine

A recently published report in the New England Journal of Medicine (NEJM) uses hard clinical data to suggest that when it comes to reducing joint bleeds and life-threatening bleeding in young boys with severe hemophilia A, prophylactic therapy is favorable to on-demand treatment. The principal investigator and lead author of the report is Marilyn J. Manco-Johnson, MD, Professor of Pediatrics and Associate Professor of Pathology in the Department of Pediatrics, University of Colorado at Denver and Health Science Center. She is also Director of the Mountain States Regional Hemophilia & Thrombosis Center at the University of Colorado.

The data was collected and analyzed from a five-year, multi-center study involving 15 U.S. academic and medical institutions. Funding for the study came from the Centers for Disease Control and Prevention and the National Institutes of Health. Study participants were treated with donated recombinant factor VIII (Kogenate® or Kogenate FS®) from Bayer HealthCare.

Enrollment in the study occurred between August 1996 and September 2000. Eligibility criteria included being younger than 30 months old, factor VIII level of 2 Units per deciliter or less, a history of two or fewer joint bleeds into an index joint and undetectable FVIII inhibitor. The 65 boys with hemophilia A were between one and two-and-a-half years old when they were enrolled in the study, and were randomly assigned to one of two groups. While 32 of the patients on prophylactic therapy were given regular, every-other-day infusions of recombinant factor VIII, 33 patients on episodic therapy were treated more intensively with multiple factor infusions only at the onset of a “clinically recognized.” Most participants were in the study for approximately four years, until they turned six years old. 

The study was designed with special emphasis on the “target joints”—the elbow, knee and ankle joints, which are most prone to bleeding in severe hemophilia patients. Joint structural outcomes and functioning were measured by X-ray, magnetic resonance imaging (MRI) and physical exams. The study also tracked the amount of factor used to treat each child. Results indicated that 93% of children in the prophylaxis group showed normal joint function, in contrast to 55% in the on-demand group. Overall, there was an 83% reduction in the risk for joint damage in patients receiving prophylaxis from an early age. Investigators reported that 18 abnormal joints (13 ankles, 3 elbows and 2 knees) were found in 15 boys, with two coming from the prophylaxis group and  a disproportionate 13 from the episodic group.

“Our results show for the first time that prophylaxis, initiated between six and 30 months of age, is effective at preventing joint bleeds and preserving joint function and structure in young boys with hemophilia A,” said Manco-Johnson. “These results provide health care professionals - as well as parents of children with hemophilia A - with solid information to guide optimum treatment.”

Five boys were removed from the study—two in the prophylaxis therapy group who developed an inhibitor to infused factor VIII, and three in the episodic therapy group who experienced life-threatening bleeding.

Manco-Johnson, who has years of extensive experience with pediatric hemophilia and joint disease prevention, is a former member of the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC). In MASAC Recommendation 170 Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding) prophylaxis is considered optimal therapy for individuals with severe hemophilia A and B.

The report, “Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia,” was published in the August 9, 2007, issue of the NEJM.

Source: AScribe Newswire, August 8, 2007


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