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 (ITIR), 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.

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

Pasca S.
2018

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 (ITIR), 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.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11577/3385394
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