Replacement therapy with a monoclonal antibody purified protein C concentrate in newborns with severe congenital protein C deficiency

Semin Thromb Hemost. 1995;21(4):371-81. doi: 10.1055/s-2007-1000658.


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.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Antibodies, Monoclonal / immunology*
  • Blindness / etiology
  • Consanguinity
  • Disseminated Intravascular Coagulation / congenital
  • Disseminated Intravascular Coagulation / etiology
  • Disseminated Intravascular Coagulation / therapy
  • Female
  • Half-Life
  • Hemorrhage / congenital
  • Hemorrhage / etiology
  • Hemorrhage / therapy
  • Home Nursing
  • Humans
  • Infant, Newborn
  • Intracranial Embolism and Thrombosis / congenital
  • Intracranial Embolism and Thrombosis / etiology
  • Intracranial Embolism and Thrombosis / therapy
  • Male
  • Plasma
  • Point Mutation
  • Protein C / genetics
  • Protein C / immunology
  • Protein C / isolation & purification
  • Protein C / pharmacokinetics
  • Protein C / therapeutic use*
  • Protein C Deficiency*
  • Purpura / congenital
  • Purpura / etiology
  • Purpura / therapy
  • Recurrence
  • Retinal Hemorrhage / congenital
  • Retinal Hemorrhage / etiology
  • Retinal Hemorrhage / therapy


  • Antibodies, Monoclonal
  • Protein C