title: Prophylactic therapy with Fibrogammin P is associated with a decreased incidence of bleeding episodes: a retrospective study.
abstract: Congenital factor XIII (FXIII) deficiency is an extremely rare, yet potentially life-threatening, bleeding disorder, with a 30% rate of spontaneous intracranial haemorrhage. Routine prophylactic management is recommended for all individuals with clinically relevant (FXIII) deficiency and for all symptomatic individuals with congenital factor deficiency. Fibrogammin P is a purified, pasteurized concentrate of FXIII that appears to carry negligible risk of viral transmission, unlike other unprocessed products containing FXIII. An ongoing Phase II/III study of Fibrogammin P in patients with congenital FXIII deficiency is being conducted to evaluate the prophylactic efficacy and long-term safety of this product. Using retrospective chart review data from subjects enrolled in the Phase II/III study, the current analysis was designed to compare spontaneous bleed-event rates prior to and after the initiation of Fibrogammin P prophylaxis. Seven subjects were evaluable for comparison, having received no other prophylactic FXIII-containing product during the 24 months prior to study entry. The mean annual number of spontaneous bleeds was 2.5 events per year prior to Fibrogammin P prophylaxis and 0.2 events per year during Fibrogammin P prophylaxis (P = 0.01). Patients reported no severe bleeds during Fibrogammin P therapy. This small sample supports a consistent and clinically meaningful reduction in spontaneous bleeding with prophylactic use of Fibrogammin P.
authors.affiliation: Children's Hospital of Michigan, Detroit, MI 48201, USA. jlusher@med.wayne.edu
authors.forename: J
authors.initials: J
authors.lastname: Lusher
authors.forename: S W
authors.initials: SW
authors.lastname: Pipe
authors.forename: S
authors.initials: S
authors.lastname: Alexander
authors.forename: D
authors.initials: D
authors.lastname: Nugent
mesh_terms: D000293:Adolescent; D000328:Adult; D001779:Blood Coagulation Factors; D002648:Child; D002675:Child, Preschool; D005260:Female; D005341:Fibrinolysin / Q000627:therapeutic use*; D005343:Fibrinolytic Agents / Q000627:therapeutic use*; D006470:Hemorrhage / Q000453:epidemiology* / Q000517:prevention & control*; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D011292:Premedication; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D001779:Blood Coagulation Factors; D005343:Fibrinolytic Agents; C025667:prothrombin complex concentrates; D005341:Fibrinolysin