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Severe Congenital Protein C Deficiency: Practical Aspects of Management

Ravi Shah1, Patrick Ferreira2, Shelina Karmali1

  • 1Pediatric Hematology, Alberta Children's Hospital, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada.

Pediatric Blood & Cancer
|May 4, 2016
PubMed

Insights

Subcutaneous protein C (PC) therapy effectively managed purpura fulminans in a child with congenital PC deficiency for over three years. Home-based SC PC replacement proved safe and successful, monitored by PC activity and D-dimer levels.

Area of Science:

  • Biochemistry
  • Hematology
  • Pediatric Thrombosis

Background:

  • Severe congenital protein C (PC) deficiency can lead to life-threatening purpura fulminans.
  • Effective long-term management strategies for pediatric PC deficiency are crucial.

Observation:

  • A child with purpura fulminans secondary to severe congenital PC deficiency was treated with subcutaneous (SC) protein C (PC).
  • Home therapy involved SC PC administration (80-120 IU/kg over 60-90 min every 48 hours) for over three years.
  • Treatment monitoring included trough PC chromogenic activity (target ≥15%) and D-dimer levels.

Findings:

  • SC PC home therapy was successful for over three years in maintaining clinical stability.
  • Discontinuation of enoxaparin did not alter the clinical course, suggesting PC replacement alone was sufficient.
  • A notable discrepancy was observed between clotting-based and chromogenic-based PC activity measurements.

Implications:

  • Subcutaneous protein C replacement is a viable and effective long-term home therapy for severe congenital PC deficiency in children.
  • This approach offers a practical alternative to intravenous administration, improving patient quality of life.
  • Further investigation into the discrepancy between different PC activity assays is warranted for accurate therapeutic monitoring.

Keywords:
congenital protein C deficiency; PROC geneprotein C activitysubcutaneous protein C

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