Semin Thromb Hemost 2000; Volume 26(Number 4): 0401-0406
DOI: 10.1055/s-2000-8459
Copyright © 2000 by Thieme Medical Publishers, Inc., 333 Seventh Avenue, New York, NY 10001, USA. Tel. +1(212)584-4662.

Recombinant Factor VIIa for the Treatment of Congenital Factor VII Deficiency

Mathilde Hunault, Kenneth A. Bauer
  • Hematology-Oncology Section, Department of Medicine, VA Boston Healthcare System and Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USA (MH, KAB) and Service des Maladies du Sang, CHU, Angers cedex, France (MH).
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Publikationsverlauf

Publikationsdatum:
31. Dezember 2000 (online)

ABSTRACT

-Factor VII deficiency is a rare autosomal bleeding disorder with a highly variable hemorrhagic predisposition. Severe bleeding, including hemarthroses, may be encountered when plasma factor VII levels are below 1%. Patients have prolonged prothrombin times, and the final diagnosis is established by quantitative factor VII assays. Some patients have true deficiencies, that is, very low factor VII activity and low factor VII antigen (cross-reacting material) levels (CRM-); others have normal antigen levels but low activity (CRM+). Still others have reduced antigen levels (CRM®). There is a rather poor correlation between clinical symptoms and factor VII activity levels in plasma. Treatment of these patients consists of fresh frozen plasma, prothrombin complex concentrates, or factor VII concentrates. Recombinant activated factor VII (rFVIIa) is a very useful alternative, and several patients have been treated successfully. Because of the short half-life of factor VIIa, repeated doses have to be administered, and continuous infusion may be even better. Antibodies to factor VII have been reported but seem to be rather rare. From the available data it appears that rFVIIa is a safe and effective treatment modality for patients with congenital factor VII deficiency.