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Semin Thromb Hemost 1995; 21(4): 371-381
DOI: 10.1055/s-2007-1000658
Copyright © 1995 by Thieme Medical Publishers, Inc.

Replacement Therapy with a Monoclonal Antibody Purified Protein C Concentrate in Newborns with Severe Congenital Protein C Deficiency

Marie Dreyfus* , Margaret Masterson† , Michele David‡ , Georges E. Rivard‡ , Frank-Michael Müller , Wolfhart Kreuz§ , Thomas Beeg§ , Adrian Minford∥ , Jeremy Allgrove# , Jesse D. Cohen** , Jürgen Christoph†† , Frauke Bergmann‡‡ , Vivian E. Mitchell¶¶ , Catherine Haworth¶¶ , Kathryn Nelson§§ , Hans Peter Schwarz§§
  • *From the Hospital Bicêtre, Paris, France (M.D.);
  • †University of Cincinnati, Cincinnati, Ohio (M.M.);
  • ‡Ste. Justine Hospital, Montreal, Quebec, Canada (M.D., G.E.R.);
  • ¶RWTH Children's Hospital, Aachen, Germany (F.M.M.);
  • §University Hospital, Frankfurt, Germany (W.K., T.B.);
  • ∥St. Luke's Hospital, Bradford, United Kingdom (A.M.);
  • #Newham General Hospital, London, United Kingdom (J.A.);
  • **St. Joseph's Hospital, Phoenix, Arizona, (J.D.C.);
  • ††Children's Hospital auf der Bult, Hannover, Germany (J.C.);
  • ‡‡Hannover Medical University, Hannover, Germany (F.B.);
  • ¶¶Leicester Royal Infirmary, Leicester, United Kingdom (V.E.M., C.H.); and
  • §§Immuno AG, Vienna, Austria (K.N., H.P.S.).
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Publication History

Publication Date:
08 February 2008 (online)

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Abstract

Protein C replacement therapy with a monoclonal antibody purified, virus inactivated protein C concentrate was carried out in nine infants (three male, six female) with severe congenital protein C deficiency and life-threatening purpura fulminans and/or thrombosis associated with disseminated intravascular coagulation (DIC). Eight infants were homozygous for protein C deficiency; one was a compound heterozygote. The treatment period varied from 22 days to three years. The half-life of protein C was found to be as short as two to three hours during activation of the coagulation system, increasing to approximately ten hours after stabilization. During the acute phase, protein C levels of 0.10 to 0.25 IU/mL were associated with elevated markers of coagulation activation indicating DIC, while protein C levels greater than 0.25 were associated with normalization of coagulation markers. No product-related side effects were reported. Episodes of bleeding or purpura recurred in all patients who were switched to oral anticoagulant therapy, necessitating reinstatement of protein C replacement therapy, either as needed to control symptoms, or on a long-term prophylactic schedule, alone or in addition to oral anticoagulation. Home treatment with protein C concentrate allowed a near-normal life-style for patients who otherwise would be hospitalized for long periods of time.

Key words:

Protein C deficiency - homozygous - treatment - protein C replacement - monoclonal antibody purified protein C concentrate