Severe congenital protein C deficiency: the use of protein C concentrates (human) as replacement therapy for life-threatening blood-clotting complications

Abstract

The protein C pathway has an important function in regulating and modulating blood coagulation and ensuring patency of the microcirculation. Protein C deficiency leads to macro- and microvascular thrombosis. Congenital severe protein C deficiency is a life-threatening state with neonatal purpura fulminans and pronounced coagulopathy. Patients with heterozygous protein C deficiency have an increased risk for thromboembolic events or experience coumarin-induced skin necrosis during initiation of coumarin therapy. Replacement with protein C concentrates is an established therapy of congenital protein C deficiency, resulting in rapid resolving of coagulopathy and thrombosis without reasonable side effects. This article summarizes the current knowledge on protein C replacement therapy in congenital protein C deficiency.

Keywords:

• protein C
• deficiency
• replacement therapy
• purpura fulminans
• coagulopathy

Acknowledgements

I gratefully acknowledge the assistance and positive input from Dr. Alex Veldman, Newborn Services, Monash Medical Centre, Monash University, Melbourne, Australia and Bruno Eberspaecher, Baxter Germany, Bioscience.