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Safety and efficacy of ReFacto, the B domain-deleted recombinant factor VIII , in home therapy of previously treated patients with severe hemophilia A An Italian multicenter trial



Safety and efficacy of ReFacto, the B domain-deleted recombinant factor VIII , in home therapy of previously treated patients with severe hemophilia A An Italian multicenter trial



Blood 96(11 Part 1): 262a, November 16



In order to evaluate, on a national basis, efficacy and safety of the BDDrFVIII, ReFacto, 24 patients (age: gtoreq12 years) affected by severe (FVIII:C <2%) hemophilia A with at least 50 previous exposure days to other FVIII concentrates have been enrolled in a 6-month, on demand treatment protocol. After a baseline evaluation which included genotyping, patients have received the product to treat bleedings episodes at home. After a screening visit at month 3, they have been re-evaluated at the end of study (month 6). In these occasions inhibitors, serology, and blood chemistry have been tested. Hemostatic efficacy has been evaluated after each infusion by reporting number of infusions, dose and response to infusion on patients' diary cards. The 24 patients (5 completed, 19 on going) have experienced 139 bleeding events (mean: 5.8 events per patient; range: 2-15) and received 188 infusions. The mean number of infusions required to recover from a bleeding was 1.3 infusion/episode. The median dose was 21.1 IU/kg per infusion. The majority of events were spontaneous hemartroses. Hemostatic efficacy was rated excellent or good in 90% of cases. No significant side effects have been reported. One patient (aged 60 years), however, developed an anti-FVIII inhibitor after 5 exposure days 3 months after the study entry. The peak level was 30 BU/ml, at which time the patient has been withdrawn. This patient had been treated with plasma-derived (pd) concentrates since decades before shifting to BDDrFVIII and had no previous history of inhibitor development. The genetic analysis did not find intron 22 inversion, and further evaluations of the gene defect are on going. After stopping study drug inhibitor titer has rapidly decreased to <.5 BU/ml within 3 months and the patient has then been treated with the previous pdFVIII without anamnestic response. In conclusion, BDDrFVIII results safe and efficacious in the home treatment of bleeding episodes in PTPs. Transient inhibitor has developed in one out of 24 enrolled patients (4%). Moreover, these findings indicate that an accurate monitoring of inhibitor development is required when patients are switched to any new brand concentrate.

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Accession: 035691749

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