title: 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.
mesh_terms: D000293:Adolescent; D000941:Antigens / Q000097:blood; D005169:Factor VIII / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007770:L-Lactate Dehydrogenase / Q000097:blood; D010976:Platelet Count; D011697:Purpura, Thrombotic Thrombocytopenic / Q000151:congenital* / Q000628:therapy; D016896:Treatment Outcome; D014841:von Willebrand Factor / Q000276:immunology
chemical_list: D000941:Antigens; C029417:Von Willebrand antigen; D014841:von Willebrand Factor; D005169:Factor VIII; D007770:L-Lactate Dehydrogenase