Prophylactic Therapy with Fibrogammin Is Associated with a Decreased Incidence of Bleeding Episodes: A Retrospective Case Control Study.

In an ongoing Phase III study we are evaluating the prophylactic efficacy and long-term safety of Fibrogammin® P, a purified, pasteurized concentrate of Factor XIII, in patients (pts) with congenital Factor XIII deficiency (an extremely rare, hereditary, life-threatening bleeding disorder). The study is being conducted at 37 sites and has enrolled 61 pts; these 61 represent approximately 2/3 of US patients with FXIII deficiency. For this analysis, a retrospective chart review was undertaken to compare spontaneous bleed-event rates 24 months prior to (when possible) and after the initiation of therapy in a subset of 7 study subjects who did not receive any prophylactic therapy prior to entering the study. The table below presents data for each of these pts on dosing, bleeding episodes and treatment (Rx) during both observation periods (Obs Pd). An additional 4 pts had been treated prophylactically with regular infusions of FFP or cryoprecipitate and transitioned successfully to FXIII therapy; there were no seroconversions to HIV, Hepatitis B/C in any study pts.

As shown in the table, these children had 2.5 bleeds per year in the period before Fibrogammin prophylaxis, and only 0.2 per year while on Fibrogammin. These results demonstrate a consistent and clinically significant reduction in spontaneous bleeding with prophylactic use of Fibrogammin® P in pts with symptomatic congenital Factor XIII deficiency.