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University of Pittsburgh Recruiting Adults for Prophylaxis Study

Investigators from the University of Pittsburgh (UP) are recruiting patients for The Hemophilia Adult Prophylaxis Study. They will explore whether a prophylaxis regimen in adults with hemophilia that calls for weekly doses of clotting factor is comparable, or even preferable, to the traditional preventive regimen of thrice-weekly infusions, commonly used in children. The lead investigator of the study is Margaret V. Ragni, MD, UP Medical Center, Department of Medicine, Division of Hematology/Oncology and Hemophilia Center of Pittsburgh. Ragni is a member of the National Hemophilia Foundation’s Medical and Scientific Advisory Council.


“A major goal of comprehensive hemophilia care is to prevent occurrence of bleeds by prophylaxis or regular preventive factor, one or more times weekly. Although prophylaxis is effective in reducing bleeding and joint damage in children, whether it is necessary to continue into adulthood is not known,” explained Ragni. The study will compare the two prophylactic dosing regimens in adults with severe hemophilia A.


A report describing the study, “Rationale for a randomized controlled trial comparing two prophylaxis regimens in adults with severe hemophilia A: the Hemophilia Adult Prophylaxis Trial,” was published in October 2011 in the journal Expert Review of Hematology. In it, Ragni theorizes that adults with more mature cartilage and joints are less susceptible to joint bleeds and joint damage. Thus, regular weekly infusions with a recombinant factor VIII product (allowing for up to two “rescue doses” per week when necessary), may be as effective as thrice-weekly prophylaxis. Such a dosing regimen would still decrease bleeding frequency while lowering cost and improving quality of life.


“This is an innovative concept, as it challenges the current paradigm of thrice-weekly prophylaxis in adults, which is based on dosing in children,” added Ragni. To learn more about the study go to


Source: Hematology Week, August 27, 2012


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