title: Experience with a third generation recombinant factor VIII concentrate (Advate) for immune tolerance induction in patients with haemophilia A.
abstract: The development of an inhibitor represents one of the most challenging complications in patients with haemophilia A. Optimal management is immune tolerance induction (ITI), typically through the administration of high doses of factor VIII (FVIII) concentrate. Among 12 patients who underwent ITI using Advate, a third-generation recombinant FVIII product that is free of animal and human protein additives, tolerance was achieved in nine (75%), including seven of 10 patients (70%) with high-titre inhibitors. ITI is ongoing in two patients and not yet successful; immune tolerance failed in the third patient. The median time to success was 4.0 months for group as a whole and for patients with high-titre inhibitors. Treatment was well tolerated, and no adverse events were observed. Advate was found to be equivalent to other FVIII products with regard to both ITI success rates and the incidence of adverse effects when used in these immune tolerance regimens.
authors.affiliation: Department of Pediatrics, Rush University Medical Center, Chicago, IL 60612-3833, USA. lvalentino@rush.edu
authors.forename: L A
authors.initials: LA
authors.lastname: Valentino
authors.forename: M
authors.initials: M
authors.lastname: Recht
authors.forename: J
authors.initials: J
authors.lastname: Dipaola
authors.forename: A D
authors.initials: AD
authors.lastname: Shapiro
authors.forename: S W
authors.initials: SW
authors.lastname: Pipe
authors.forename: N
authors.initials: N
authors.lastname: Ewing
authors.forename: J
authors.initials: J
authors.lastname: Urgo
authors.forename: T
authors.initials: T
authors.lastname: Bullock
authors.forename: M
authors.initials: M
authors.lastname: Simmons
authors.forename: C
authors.initials: C
authors.lastname: Deguzman
mesh_terms: D019774:Blood Coagulation Factor Inhibitors / Q000276:immunology*; D005169:Factor VIII / Q000008:administration & dosage / Q000276:immunology*; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy / Q000276:immunology*; D006801:Humans; D007108:Immune Tolerance / Q000187:drug effects*; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000276:immunology*; D016896:Treatment Outcome
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D011994:Recombinant Proteins; D005169:Factor VIII