Immune tolerance: a synopsis of the international experience.
Because of the increased morbidity and cost of care associated with inhibitor development, immune tolerance therapy (ITT) is of crucial value in the care of haemophilia. The 24-year experience with this modality, primarily in the treatment of factor VIII inhibitors, has included the use of both high and low doses of clotting factor, with and without immune modulation. Overall success rates for ITT in haemophilia A have been similar (63-83%), while median time to IT has been variable (1.2-24 months). The role of type and purity of clotting factor used remains unclear. Three immune tolerance registries have suggested the potential importance of treatment parameters such as pre-induction inhibitor titer and daily factor dose in the prediction of successful outcome. Ultimately, prospective randomized studies of ITT are required to definitively compare therapeutic regimens with respect to efficacy, safety, and cost effectiveness.
Di Michele
D M
DM
Department of Paediatrics, New York Hospital-Cornell Medical College, NY 10021, USA. dmdimich@mail.med.cornell.edu
D000818:Animals; D005169:Factor VIII / Q000276:immunology / Q000627:therapeutic use; D006467:Hemophilia A / Q000188:drug therapy / Q000276:immunology; D006801:Humans; D007108:Immune Tolerance; D016032:Randomized Controlled Trials as Topic
D005169:Factor VIII