A comprehensive review of rFVIIa use in a tertiary care pediatric center

Background. Recombinant activated factor VII (rFVIIa) is a hemostatic agent developed for the treatment of bleeds in patients with hemophilia and inhibitors. Case reports/series document its growing use in patients without hemophilia. Such reports however do not accurately describe the proportion of rFVIIa used for various indications. We sought to document the complete use of rFVIIa at our institution over a 6-year period (2000-2005). Procedure. Using a computerized registry documenting all rFVIIa use in our institution a complete list of patients receiving rFVIIa was generated. Clinical data on these patients was obtained through chart review. Results. 111 patients received 7,016,400 mu g of rFVIIa over the 6 years: 23 patients had congenital bleeding disorders (10 patients with hemophilia and inhibitors; 7 with congenital FVII deficiency; 6 with platelet function disorders). These 23 patients (21% of all patients receiving rFVIIa) accounted for 79.9% of all rFVIIa used; patients with hemophilia alone accounted for 68.6%. The 88 patients without a congenital bleeding disorder (79% of all patients using rFVIIa) accounted for 20.1% of rFVIIa used. However their annual use of rFVIIa increased 10-fold during the 6 years. Conclusions. Patients with hemophilia use massive amounts of rFVIIa repeatedly while patients without hemophilia use rFVIIa infrequently and at smaller doses. The use of rFVIIa in patients without congenital bleeding disorders (all "off-label" use) is rapidly growing in both number of patients and in total use and has likely significant clinical and economic ramifications.