Prophylaxis with activated prothrombin complex concentrate in patients with hemophilia A with high response inhibitors

An alternative approach to standard treatment

Abstract

The most serious complication of hemophilia patients is the development of inhibitory antibodies; up to 30% of patients with severe hemophilia A develop them. To eradicate these antibodies, induction of immune tolerance is the treatment of choice; when they persist, prophylactic treatment with bridge agents as activated prothrombin complex concentrate aPCC (FEIBA®) or rFVIIa (Novoseven®) offer a therapeutic alternative for reducing bleeding and hemophilic arthropathy. To evaluate the efficacy of prophylaxis with aPCC, bleeds were compared before and after receiving prophylaxis (11-12 months) in 8 hemophilia patients with high response inhibitors.

Material and methods: a multicenter study was conducted in children and adults with a diagnosis of hemophilia A with high titer inhibitors in 4 attention centers in two cities. Patients with acquired haemophilia were excluded.

Results: six patients had severe hemophilia A and 2 moderate; 7/8 patients had hemophilic arthropathy. The mean age was 19 years (range 7-38) and mean inhibitor titer was 80 UB (range 15- 1178). aPCC dose ranged from 40-75 U / kg, 2-3 times a week. The overall annual rates of bleeding and hemarthrosis pre-prophylaxis were (8 / year and 3.1 / yr) and after prophylaxis during a period of 11- 12 months were (1.08 / year and 1 / year); a reduction of 86% and 68% respectively was found. There were no thrombotic events. Compliance scheme of aPCC treatment was higher than 80%.

Conclusions: this is the first case report in Colombia on the use of aPCC in haemophilia patients with high responding inhibitors to factor VIII. Questions remain about the length or adjustments to the treatment schedule