Immune tolerance induction rescue with turoctocog-alfa in a poor risk haemophilia A inhibitor young child: the history of a success

Blood Coagul Fibrinolysis. 2018 Jul;29(5):465-468. doi: 10.1097/MBC.0000000000000736.

Abstract

: The development of alloantibodies against the replacement of Factor VIII (FVIII) is the major complication in haemophilia A treatment. The gold standard to eradicate inhibitors is the immune tolerance induction (ITI), but in some cases it fails requiring another immune tolerance, defined ITI rescue (ITI-R), using a different concentrate, even though it is still debated. We report a successful case of a poor risk (titre of inhibitor at start of ITI > 10 BU/ml, peak titre on ITI > 200 BU/ml, >2 years since the inhibitor diagnosis) haemophilia A child treated with a high-dose regimen (200 UI/kg/day) turoctocog-alfa after a failed first-line ITI with octocog-alfa lasting 29 months. At 22 months of ITI-R, the inhibitor titre was undetectable, the FVIII recovery was 74%, of the expected level and the FVIII half-life more than 7 h. A complete successful ITI-R was then achieved with turoctocog-alfa.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Factor VIII / pharmacology
  • Factor VIII / therapeutic use*
  • Hemophilia A / drug therapy*
  • Humans
  • Risk Factors

Substances

  • recombinant factor VIII N8
  • Factor VIII