Corifact™/Fibrogammin® P in the prophylactic treatment of hereditary factor XIII deficiency: results of a prospective, multicenter, open-label study

Factor XIII (FXIII) deficiency is an extremely rare congenital condition that is associated with a high risk of potentially life-threatening intracranial hemorrhage, poor wound healing, spontaneous abortion, and a lifelong tendency towards spontaneous bleeding and severe bleeding after trauma or surgery. Routine prophylaxis with FXIII concentrate is recommended in all individuals with FXIII levels <1 IU/dL from the time of diagnosis, and in some severely affected patients with FXIII levels of 1-4 IU/dL. Fibrogammin (R) P is a highly purified, pasteurized, plasma-derived concentrate that has been available in Europe and other countries since 1993 and has recently been approved as Corifact (TM) in the USA. To support the US registration of Corifact (TM), a 52-week, prospective, multicenter, open-label study was conducted in 41 patients (mean age 19 years; range <1-42 years) with congenital FXIII deficiency. Corifact (TM)/Fibrogammin (R) P was administered intravenously at an initial dose of 40 IU/kg every 4 weeks, with dosing adjusted to maintain a trough FXIII activity level of 5-20%. No spontaneous bleeding episodes requiring FXIII treatment were reported during the study (primary endpoint). Preoperative use of Corifact (TM)/Fibrogammin (R) P successfully prevented postoperative bleeding in two surgeries. Corifact (TM)/Fibrogammin (R) P was well tolerated during a total exposure of similar to 455 subject-months. No patient withdrew from treatment, and there were no reports of virus transmission or thromboembolism-related events. This study adds to the wealth of data gained from clinical trials and almost 20 years of clinical use confirming that Corifact (TM)/Fibrogammin (R) P is an effective and well-tolerated prophylactic treatment for congenital FXIII deficiency. (C) 2012 Elsevier Ltd. All rights reserved.