Use of recombinant, activated factor VII in the treatment of congenital factor VII deficiencies.
OBJECTIVE
Factor VII (FVII) deficiency is a rare coagulation disorder, historically treated with prothrombin complex concentrates or plasma-derived FVII concentrates. We treated such patients (n = 17) with a recombinant, activated FVII preparation.


METHODS
Twenty-seven spontaneous bleeding episodes were treated and 7 major and 13 minor surgical interventions were carried out. The dosages employed ranged from 8.08 to 70.5 Ig/kg body weight.


RESULTS
A mean dose between 22 and 26 Ig/kg was sufficient to normalise the prothrombin time. Fifteen haemarthroses were treated with single doses and results were excellent in 13 cases. In 5/6 bleeding episodes of other types, the treatment gave either excellent or at least effective results. Haemostasis was secured in the 7 major and 13 minor surgical interventions. One patient developed antibodies 4-5 weeks after an extremely high dose. Otherwise, there were no side effects and no evidence of a thrombotic tendency.


CONCLUSIONS
This recombinant concentrate is efficacious in FVII-deficient patients. It is safe since any risk of transmission of blood-borne viruses is eliminated.
Mariani
G
G
University of Palermo, Italy. mariangu@tin.it
Testa
M G
MG
Di Paolantonio
T
T
Molskov Bech
R
R
Hedner
U
U
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D005168:Factor VII Deficiency / Q000151:congenital* / Q000188:drug therapy* / Q000601:surgery; D015942:Factor VIIa / Q000008:administration & dosage* / Q000037:antagonists & inhibitors / Q000276:immunology; D006395:Hemarthrosis / Q000188:drug therapy; D006487:Hemostasis / Q000187:drug effects; D006801:Humans; D007223:Infant; D007518:Isoantibodies / Q000097:blood; D008297:Male; D008875:Middle Aged; D011516:Prothrombin / Q000187:drug effects; D011994:Recombinant Proteins / Q000008:administration & dosage; D013917:Thrombin / Q000187:drug effects; D013997:Time Factors
D007518:Isoantibodies; D011994:Recombinant Proteins; D011516:Prothrombin; D015942:Factor VIIa; D013917:Thrombin