Can activated recombinant factor VII be used to postpone the exposure of infants to factor VIII until after 2 years of age?

Authors


Georges E. Rivard, Division of Hematology Hôpital Sainte-Justine (Room 2617) 3175 Côte Ste-Catherine, Montréal, QC, Canada H3T 1C5.
Tel.: +1 345 4931, ext. 4639; fax: +1 345 4884;
e-mail: rivardge@medclin.umontreal.ca

Abstract

Summary.  Two retrospective studies have suggested that exposure to factor VIII (FVIII) in early infancy is associated with an increased risk of FVIII inhibitor development. We prospectively studied 11 infants who needed replacement therapy for bleeding episodes before the age of 2 years. They received activated recombinant factor VII (rFVIIa) concentrate on demand, with the intention of postponing their first exposure to FVIII after 2 years of age. Thirty-three bleeding episodes were treated with 154 doses of rFVIIa with no evidence of adverse effect. Bleeding was controlled in 27 of 33 episodes. Mouth bleeds were most difficult to treat. The use of rFVIIa allowed postponement of the use of FVIII for a mean of 5.5 months (median 4, range 0–12) but in only three of 11 children could be the first exposure to factor postponed after the age of 2 years. With this modest effect of rFVIIa in postponing the first exposure to FVIII, more convincing evidence for the benefit of such a postponement will have to be demonstrated before rFVIIa could be recommended for this indication.

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