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Prophylaxis Study Results for Boys with Severe Hemophilia A

A group of Canadian researchers recently published findings from a clinical study on the use of prophylaxis with a standard half-life recombinant factor VIII (SHL-rFVIII) therapy to treat boys with severe hemophilia A. They investigated an approach to prophylactic therapy in which patients began by receiving weekly infusions of SHL-rFVIII and ultimately transition to “tailored frequency-escalated prophylaxis.” The lead investigator of the study was Professor Brian M Feldman, MD of the Division of Rheumatology at The Hospital for Sick Children in Toronto.

A total of 56 boys with severe hemophilia from 12 Canadian treatment centers were included in the study. These 56 participants were recruited as part of on an ongoing enrollment between June 26, 1997 and January 30, 2007. The boys were followed for a median of 10.2 years (to a maximum of 16.1 years) once they began with once weekly prophylaxis using SHL-rFVIII. Participants were eventually treated with escalating frequency, including requisite dose adjustments, in response to breakthrough bleeding events.

The study, “Tailored Frequency-Escalated Primary Prophylaxis for Severe Haemophilia A: Results of the 16-year Canadian Hemophilia Prophylaxis Study Longitudinal Cohort,” was published online May 3, 2018 in the journal The Lancet Haematology.

Source: RareDisease Report, May 11, 2018

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