Clinical Experience with Human Plasma-Derived Factor VIla in Patients with Hemophilia A and High Titer Inhibitors

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Abstract
FVIIa purified from human plasma and spontaneously activated during the purification procedure was given to 4 patients with hemophilia A and inhibitors against FVIIl:C in association with joint bleeds. A dose of 9–20 μg/kg b.w. (700–1,000 U/kg b.w.) seemed to be hemostatically active in moderate to severe joint bleeds. Plasma levels of FVΠ of 5–7 U/ml were achieved and recoveries varied between 17 and 66%. The lower recovery rates of 17–39% were all found in 1 of the patients. No immediate side effects were seen. Neither were any signs of a systemic activation of the coagulation system observed. It is concluded that highly purified FVIIa may be useful in the treatment of hemophilia A patients with inhibitors against FVIII:C.