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 life-long 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® 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™ in the USA. To support the US registration of Corifact™, 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™/Fibrogammin® 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™/Fibrogammin® P successfully prevented postoperative bleeding in two surgeries. Corifact™/Fibrogammin® P was well tolerated during a total exposure of ≈ 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™/Fibrogammin® P is an effective and well-tolerated prophylactic treatment for congenital FXIII deficiency.