A prospective, observational, post-approval registry of inhibitor patients treated with multiple or single doses of recombinant activated factor VII

In March 2007, the European Medicines Agency (EMEA) approved the use of single-dose recombinant activated factor VII (rFVIIa; NovoSeven (R), Novo Nordisk, Bagsvaerd, Denmark) 270 mu g/kg for the treatment of mild-to-moderate bleeding episodes in patients with hemophilia A or B with inhibitors. The prospective, observational ONE Registry was recently established to describe "real world" use of the standard multiple-dose (3 x 90 mu g/kg) and new single-dose (270 mu g/kg) regimens, and to compare their safety, effectiveness, and effects on both quality of life and patient satisfaction with treatment. Enrollment started at the beginning of 2008, with a planned recruitment period of 18 months, and it is expected that approximately 100 patients will be enrolled from up to 30 European hemophilia centers. The primary efficacy outcome is global treatment response, based on subjective measures of bleeding cessation and pain relief. Secondary outcome measures include treatment satisfaction, time to hemostasis and pain relief, and quality of life assessment using the Haemo-QoL questionnaire; details of all adverse events also will be recorded. Interim results are expected by December 2008. It is hoped that the ONE Registry will raise awareness of the new single-dose rFVIIa treatment schedule, aggregate experience, and accelerate learning, fostering confidence in its use.