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.
Vukovich
T
T
Department of Medical Physiology, University of Vienna, Austria.
Auberger
K
K
Weil
J
J
Engelmann
H
H
Knöbl
P
P
Hadorn
H B
HB
D005260:Female; D006023:Glycoproteins / Q000172:deficiency* / Q000627:therapeutic use; D006720:Homozygote; D006801:Humans; D007223:Infant; D009336:Necrosis; D010949:Plasma; D011486:Protein C / Q000627:therapeutic use; D020151:Protein C Deficiency; D017293:Protein S; D012867:Skin / Q000473:pathology
D006023:Glycoproteins; D011486:Protein C; D017293:Protein S