Successful treatment of congenital thrombotic thrombocytopenic purpura using the intermediate purity factor VIII concentrate BPL 8Y

Abstract

There is increasing evidence that congenital thrombotic thrombocytopenic purpura (TTP) is caused by an absolute deficiency of von Willebrand factor-cleaving protease. The recent identification of this protease and the development of assays for its detection have enabled its quantification in a number of plasma products, including some commercial intermediate-purity plasma-derived factor VIII preparations. We report the successful, weekly prophylactic use of a commercial intermediate-purity plasma-derived factor VIII concentrate in the treatment of a 14-year-old girl with severe congenital TTP who had previously required transfusions of fresh-frozen plasma every 2 weeks from the age of 4 months.