How we treat a hemophilia A patient with a factor VIII inhibitor

• Published in: Blood 2009-01, Vol.113 (1), p.11-17
• Main Authors: Kempton, Christine L., White, Gilbert C.
• Format: Article
• Language: English
• Subjects: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Biological and medical sciences; Blood. Blood and plasma substitutes. Blood products. Blood cells. Blood typing. Plasmapheresis. Apheresis; Factor VIII - administration & dosage; Factor VIII - antagonists & inhibitors; Factor VIII - immunology; Hematologic and hematopoietic diseases; Hemophilia A - drug therapy; Hemophilia A - epidemiology; Hemophilia A - immunology; Humans; Immune Tolerance; Isoantibodies - immunology; Medical sciences; Platelet diseases and coagulopathies; Risk Factors; Transfusions. Complications. Transfusion reactions. Cell and gene therapy
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2008-06-160432

The most significant complication of treatment in patients with hemophilia A is the development of alloantibodies that inhibit factor VIII activity. In the presence of inhibitory antibodies, replacement of the missing clotting factor by infusion of factor VIII becomes less effective. Once replacement therapy is ineffective, acute management of bleeding requires agents that bypass factor VIII activity. Long-term management consists of eradicating the inhibitor through immune tolerance. Despite success in the treatment of acute bleeding and inhibitor eradication, there remains an inability to predict or prevent inhibitor formation. Ideally, prediction and ultimately prevention will come with an improved understanding of how patient-specific and treatment-related factors work together to influence anti–factor VIII antibody production.