title: Experience with recombinant-activated factor VII in 30 patients with congenital factor VII deficiency.
abstract: Recombinant-activated factor VII (rFVIIa) represents a therapeutic advance for the treatment and prevention of haemorrhage in patients with the rare bleeding disorder, congenital FVII deficiency. Thirty-nine cases of the use of rFVIIa in 30 patients with congenital FVII deficiency were identified from the international, internet-based registry haemostasis.com, which is a repository of case reports on the investigational use of rFVIIa that have been voluntarily submitted by physicians worldwide. These registry data have limitations compared with clinical-trial data but give valuable insights into a treatment for a rare disease that is virtually impossible to assess in conventional clinical trials. rFVIIa was used in: elective surgery (13 cases); haematoma (9 cases); emergency surgery (6 cases); epistaxis (4 cases); menorrhagia (2 cases); cover during childbirth (2 cases); disseminated intravascular coagulation (1 case; premature infant); removal of intradermal stitches (1 case); and haematuria (1 case). In 22/39 cases, rFVIIa was used prophylactically. Total dose and dosing schedules varied; median individual dose was 13.3 mug/kg body weight (bw) (range 1.2-223.8 mug/kg bw), median total dose was 38 microg/kg bw (range 1.2-758 microg/kg bw) and median number of doses was 3 (range 1-55). rFVIIa was generally associated with bleeding cessation or markedly reduced bleeding. Two adverse events were reported, but neither was regarded as being related to rFVIIa. These 39 cases support data confirming the safety and efficacy of rFVIIa in its EU-licensed indications, including that for preventing and/or controlling haemorrhage in patients with congenital FVII deficiency.
authors: Thrombosis and Hemostasis Unit, Department of Hematology and Bone Marrow Transplantation, Rambam Medical Center, Haifa, Israel. b_brenner@rambam.health.gov.il
authors: Benjamin
authors: B
authors: Brenner
authors: Jørgen
authors: J
authors: Wiis
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000933:Antifibrinolytic Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D005167:Factor VII / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D005168:Factor VII Deficiency / Q000188:drug therapy*; D015942:Factor VIIa; D005260:Female; D006395:Hemarthrosis / Q000188:drug therapy; D006406:Hematoma / Q000188:drug therapy; D006470:Hemorrhage / Q000188:drug therapy* / Q000209:etiology / Q000517:prevention & control; D006490:Hemostatics / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007234:Infant, Premature; D007235:Infant, Premature, Diseases / Q000188:drug therapy; D008297:Male; D008875:Middle Aged; D019106:Postoperative Hemorrhage / Q000517:prevention & control; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D012042:Registries / Q000706:statistics & numerical data; D012189:Retrospective Studies; D013927:Thrombosis / Q000139:chemically induced / Q000517:prevention & control; D016896:Treatment Outcome; D014947:Wounds and Injuries / Q000150:complications
chemical_list: D000933:Antifibrinolytic Agents; D006490:Hemostatics; D011994:Recombinant Proteins; D005167:Factor VII; C103587:recombinant FVIIa; D015942:Factor VIIa