Activated recombinant human coagulation factor VII therapy for intracranial hemorrhage in patients with hemophilia A or B with inhibitors - Results of the NovoSeven emergency-use program

Activated recombinant human coagulation factor VII (rFVIIa) is a promising new therapeutic agent for patients with hemophilia A or B with inhibitors w ho experience serious bleeding episodes or who need coverage during surgica l procedures. This open-label, uncontrolled, emergency-use study evaluated the efficacy and safety of rFVIIa in 11 hemophiliac patients and 1 FVII-def icient patient with life-threatening intracranial hemorrhage previously unr esponsive to one or more alternative therapies. rFVIIa effectively controll ed intracranial hemorrhage in 10 of the 12 patients. Patients with hemophil ia A or B received an average of 96.9 rFVIIa injections over 14.7 days with a mean total administration of 153.3 mg, corresponding to 8.1 mg/kg. Most reported adverse events were considered to be unrelated to rFVIIa therapy. These findings suggest that rFVIIa is an effective and well-tolerated thera peutic option in the management of central nervous system bleeding in patie nts with hemophilia A or B with inhibitors.