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Human protein C: new preparations. Effective replacement therapy for some clotting disorders
Insights
Congenital protein C deficiency can lead to severe thrombosis. Human protein C concentrates like Ceprotin and Protexel are effective treatments, particularly for severe cases and prophylaxis in high-risk situations.
Area of Science:
- Hematology
- Pharmacology
- Genetics
Background:
- Congenital protein C deficiency presents risks including venous thrombosis, skin necrosis, and neonatal thrombotic events.
- Fresh frozen plasma was the primary treatment, carrying risks like hypervolemia.
- Two human protein C concentrates, Ceprotin and Protexel, are approved for intravenous therapy.
Purpose of the Study:
- To evaluate the efficacy and safety of human protein C concentrates for congenital protein C deficiency.
- To compare Ceprotin and Protexel in treating and preventing thrombotic events.
Main Methods:
- Review of retrospective case series for Ceprotin (22 children) and Protexel (10 patients).
- Analysis of clinical data on treatment effectiveness and adverse events.
- No direct comparative studies between the two preparations were identified.
Main Results:
- Human protein C replacement therapy is effective in severe deficiency, including neonates, for cutaneous thrombosis and prophylaxis.
- Prophylaxis with human protein C reduces thrombosis frequency in moderate deficiency during high-risk periods.
- Long-term prophylaxis may reduce recurrent venous thrombosis when added to vitamin K antagonists, despite limitations.
Conclusions:
- Ceprotin and Protexel are key treatments for constitutional protein C deficiency.
- Adverse effects are not well-documented but include allergic reactions, bleeding, and infection risk.
- Further documentation on indications and adverse effects is needed for these protein C concentrates.
Abstract:
(1) Depending on its severity, congenital protein C deficiency can cause a variety of problems, such as increasing the frequency of venous thrombosis in high risk situations; recurrent venous thrombosis; skin necrosis at the start of treatment with a vitamin K antagonist; and severe thrombotic events in neonates. For many years the only available replacement treatment consisted of fresh frozen plasma which, among other adverse effects, carries a risk of hypervolemia. (2) Two human protein C concentrates prepared from donated blood have been given marketing authorisation in Europe for intravenous replacement therapy (Ceprotin from Baxter, and Protexel from LFB). (3) Their clinical files contain only retrospective case series (22 children with severe deficiency treated with Ceprotin; and 10 patients of various ages and with different degrees of severity treated with Protexel). The two preparations have not been compared with each other. (4) In patients with severe protein C deficiency, including neonates, replacement therapy with human protein C is effective, especially for treating cutaneous thrombosis and preventing thrombosis in high risk situations. (5) In patients with moderate deficiency, a short-course of human protein C prophylaxis reduces the frequency of thrombosis in high risk situations. (6) In long-term prophylaxis, human protein C replacement therapy, added to ongoing (but inadequately effective) vitamin K antagonist therapy, seems to reduce the risk of recurrent venous thrombosis even though it has some constraints. (7) The adverse effects of the two preparations are poorly documented. Allergic reactions and bleeding have been reported. Human protein C is a blood product, and therefore carries a risk of infection. (8) Ceprotin offers a small advantage, being available in two dose strengths: for a given dose the volume injected is halved. (9) In practice, Ceprotin and Protexel are the reference drugs for replacement therapy of constitutional protein C deficiency, although their indications and adverse effects should be better documented.