Use of recombinant, activated factor VII in the treatment of congenital factor VII deficiencies

Background and Objectives: factor VII (FVII) deficiency is a rare coagulati on disorder, historically treated with prothrombin complex concentrates or plasma-derived FVII concentrates. We treated such patients (n = 17) with a recombinant, activated FVII preparation. Materials and Methods: Twenty-seve n spontaneous bleeding episodes were treated and 7 major and 13 minor surgi cal interventions were carried out. The dosages employed ranged from 8.08 t o 70.5 mu g/kg body weight. Results: A mean dose between 22 and 26 mu g/kg was sufficient to normalise the prothrombin time. Fifteen haemarthroses wer e treated with single doses and results were excellent in 13 cases. In 516 bleeding episodes of other types, the treatment gave either excellent or at least effective results. Haemostasis was secured in the 7 major and 13 min or surgical interventions. One patient developed antibodies 4-5 weeks after an extremely high dose. Otherwise, there were no side effects and no evide nce of a thrombotic tendency. Conclusion: This recombinant concentrate is e fficacious in FVII-deficient patients. It is safe since any risk of transmi ssion of blood-borne viruses is eliminated.