Hemophilia treatment. Immune tolerance induction: prospective clinical trials

Haematologica. 2000 Oct;85(10 Suppl):52-5; discussion 55-6.

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.

MeSH terms

  • Cost-Benefit Analysis
  • Factor VIII / administration & dosage
  • Factor VIII / immunology
  • Hemophilia A / drug therapy*
  • Hemophilia A / economics
  • Hemophilia A / immunology
  • Humans
  • Immune Tolerance*
  • Prospective Studies
  • Randomized Controlled Trials as Topic / methods

Substances

  • Factor VIII