Replacement therapy for a homozygous protein C deficiency-state using a concentrate of human protein C and S.

A severe congenital deficiency of protein C was diagnosed in a 10-month-old girl who had been suffering from skin necrosis since the age of 7 months. The patient was treated initially with fresh frozen plasma, 10 ml per kg body weight, every 24 h. Following treatment, the mean plasma level of protein C was 0.1 U/ml after 30 min and less than 0.02 U/ml after 24 h. The child was then treated with a concentrate of human protein C and S, 100 U protein C per kg body weight, given every 48 h for a period of 9 months. The mean plasma level of protein C was 0.93 U/ml 30 min after administration of the concentrate and 0.13 and 0.08 U/ml after 24 and 48 h, respectively. The mean post-transfusional in vivo recovery of protein C was 44% and the half life was 8.3 h. The mean plasma level of 'free' protein S increased from 1.1 to 2.2 U/ml after administration of the concentrate. There was no increase in 'bound' protein S. The in vivo recovery of 'free' protein S was 49% and the half life was about 17 h. Since the start of this replacement therapy using a human protein C and S concentrate, the patient has not developed any thromboembolic complications. These results indicate the therapeutic value of human protein C and S concentrate in the treatment of severe protein C deficiency.