Immune tolerance induction (ITI) eradicates inhibitors in patients with hemophilia A. This study was designed to investigate the success rate of ITI in high-responding inhibitor patients with severe hemophilia A using recombinant factor VIII (rFVIII). Twenty-six patients received different ITI regimens until a normal recovery (&rt; 66%) and half-life (&rt; 6 h) of infused FVIII was achieved. In order to maximize the chance of success, the initiation of ITI was deferred in the majority of patients until the inhibitor declined to < 10 BU. Twenty-two patients (85%) had baseline inhibitor levels < 10 BU (median 2.3 BU) when ITI began. Within a median of 6 months, immune tolerance was achieved in 19 of 26 patients (73%) including 12/17 (70%) with intron 22 inversion, 5/7 (71%) with other null mutations and two with small deletion/insertions in the F8 gene. In conclusion, recombinant FVIII induces a high rate of immune tolerance even in carriers of null F8 mutations.

Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors / A. Rocino, E. Santagostino, M.E. Mancuso, P.M. Mannucci. - In: HAEMATOLOGICA. - ISSN 0390-6078. - 91:4(2006 Apr), pp. 558-561.

Immune tolerance induction with recombinant factor VIII in hemophilia A patients with high responding inhibitors

M.E. Mancuso
Penultimo
;
P.M. Mannucci
Ultimo
2006

Abstract

Immune tolerance induction (ITI) eradicates inhibitors in patients with hemophilia A. This study was designed to investigate the success rate of ITI in high-responding inhibitor patients with severe hemophilia A using recombinant factor VIII (rFVIII). Twenty-six patients received different ITI regimens until a normal recovery (&rt; 66%) and half-life (&rt; 6 h) of infused FVIII was achieved. In order to maximize the chance of success, the initiation of ITI was deferred in the majority of patients until the inhibitor declined to < 10 BU. Twenty-two patients (85%) had baseline inhibitor levels < 10 BU (median 2.3 BU) when ITI began. Within a median of 6 months, immune tolerance was achieved in 19 of 26 patients (73%) including 12/17 (70%) with intron 22 inversion, 5/7 (71%) with other null mutations and two with small deletion/insertions in the F8 gene. In conclusion, recombinant FVIII induces a high rate of immune tolerance even in carriers of null F8 mutations.
FVIII inhibitors; Hemophilia A; Immune tolerance induction
apr-2006
http://www.haematologica.org/cgi/reprint/91/4/558
Article (author)
File in questo prodotto:
Non ci sono file associati a questo prodotto.
Pubblicazioni consigliate

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/50643
Citazioni
  • ???jsp.display-item.citation.pmc??? 6
  • Scopus 44
  • ???jsp.display-item.citation.isi??? 39
social impact