Replacement therapy for bleeding episodes in factor VII deficiency. A prospective evaluation

Thromb Haemost. 2013 Feb;109(2):238-47. doi: 10.1160/TH12-07-0476. Epub 2012 Dec 13.

Abstract

Patients with inherited factor VII (FVII) deficiency display different clinical phenotypes requiring ad hoc management. This study evaluated treatments for spontaneous and traumatic bleeding using data from the Seven Treatment Evaluation Registry (STER). One-hundred one bleeds were analysed in 75 patients (41 females; FVII coagulant activity <1-20%). Bleeds were grouped as haemarthroses (n=30), muscle/subcutaneous haematomas (n=16), epistaxis (n=12), gum bleeding (n=13), menorrhagia (n=16), central nervous system (CNS; n=9), gastrointestinal (GI; n=2) and other (n=3). Of 93 evaluable episodes, 76 were treated with recombinant, activated FVII (rFVIIa), eight with fresh frozen plasma (FFP), seven with plasma-derived FVII (pdFVII) and two with prothrombin-complex concentrates. One-day replacement therapy resulted in very favourable outcomes in haemarthroses, and was successful in muscle/subcutaneous haematomas, epistaxis and gum bleeding. For menorrhagia, single- or multiple-dose schedules led to favourable outcomes. No thrombosis occurred; two inhibitors were detected in two repeatedly treated patients (one post-rFVIIa, one post-pdFVII). In FVII deficiency, most bleeds were successfully treated with single 'intermediate' doses (median 60 µg/kg) of rFVIIa. For the most severe bleeds (CNS, GI) short- or long-term prophylaxis may be optimal.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Blood Coagulation Factors / administration & dosage*
  • Blood Coagulation Factors / adverse effects
  • Blood Component Transfusion* / adverse effects
  • Child
  • Child, Preschool
  • Coagulants / administration & dosage*
  • Coagulants / adverse effects
  • Drug Administration Schedule
  • Factor VII Deficiency / complications
  • Factor VII Deficiency / diagnosis
  • Factor VII Deficiency / genetics
  • Factor VII Deficiency / therapy*
  • Factor VIIa / administration & dosage*
  • Factor VIIa / adverse effects
  • Female
  • Hemorrhage / drug therapy*
  • Hemorrhage / genetics
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Middle Aged
  • Prospective Studies
  • Recombinant Proteins / administration & dosage
  • Registries
  • Time Factors
  • Treatment Outcome
  • Young Adult

Substances

  • Blood Coagulation Factors
  • Coagulants
  • Recombinant Proteins
  • prothrombin complex concentrates
  • Factor VIIa