title: Hemophilia treatment. Immune tolerance induction: prospective clinical trials.
abstract: Successful immune tolerance induction (ITI) relates significantly to the starting inhibitor titer but not the peak historical inhibitor titer. Below a starting titer of 10 BU/mL success appears to be unrelated to the dose of factor VIII (FVIII) used for ITI. Above this starting titer, larger doses of FVIII may be associated with a better outcome from ITI. Opinion on the importance of the dose of FVIII used for ITI is polarized and there is no agreement on the optimal regimen for ITI. A prospective randomized clinical trial is proposed in which patients will initially be treated on demand with bypass therapy until the inhibitor titer has fallen below 10 BU/mL. Patients will then be randomized to receive a high or low-dose regime. Efficacy, cost-effectiveness and morbidity will be compared.
authors.affiliation: University Dept. of Hematology, Manchester Royal Infirmary, UK. haemophilia@man.ac.uk
authors.forename: C R
authors.initials: CR
authors.lastname: Hay
mesh_terms: D003362:Cost-Benefit Analysis; D005169:Factor VIII / Q000008:administration & dosage / Q000276:immunology; D006467:Hemophilia A / Q000188:drug therapy* / Q000191:economics / Q000276:immunology; D006801:Humans; D007108:Immune Tolerance; D011446:Prospective Studies; D016032:Randomized Controlled Trials as Topic / Q000379:methods
chemical_list: D005169:Factor VIII