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

Br J Haematol. 2002 Oct;119(1):176-9. doi: 10.1046/j.1365-2141.2002.03809.x.

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.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Antigens / blood
  • Factor VIII / therapeutic use*
  • Female
  • Humans
  • L-Lactate Dehydrogenase / blood
  • Platelet Count
  • Purpura, Thrombotic Thrombocytopenic / congenital*
  • Purpura, Thrombotic Thrombocytopenic / therapy
  • Treatment Outcome
  • von Willebrand Factor / immunology

Substances

  • Antigens
  • Von Willebrand antigen
  • von Willebrand Factor
  • Factor VIII
  • L-Lactate Dehydrogenase