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Treatment of homozygous protein C deficiency with subcutaneous protein C concentrate
A M Minford1, L A Parapia, C Stainforth
1St Luke's Hospital, Bradford.
Insights
Subcutaneous protein C concentrate offers a safe and effective alternative for managing homozygous protein C deficiency. This method maintains therapeutic levels for 48 hours, reducing risks associated with central venous lines.
Area of Science:
- Hematology
- Pharmacology
Background:
- Homozygous protein C deficiency requires lifelong anticoagulation therapy.
- Traditional treatment involves intravenous protein C concentrate, necessitating central venous access.
Observation:
- A child with homozygous protein C deficiency was switched from intravenous to subcutaneous protein C concentrate.
- The subcutaneous route was administered every third day.
Findings:
- Subcutaneous protein C concentrate (250 iu/kg) maintained protective levels for 48 hours post-infusion.
- Peak protein C levels were observed at 12 hours.
- The treatment was well-tolerated, with no thrombotic events reported.
Implications:
- Subcutaneous protein C concentrate is a viable long-term therapeutic option for homozygous protein C deficiency.
- This administration route potentially mitigates risks associated with long-term central venous catheter use.
- Home-based subcutaneous infusions may improve patient quality of life.
Abstract:
Subcutaneous protein C concentrate (Immuno, Vienna) was used to treat a child with homozygous protein C deficiency who was formerly treated with intravenous protein C concentrate. After 3000 units subcutaneous protein C concentrate (250 iu/kg), protective protein C levels were maintained for 48 h after infusion, with peak levels at 12 h. Subcutaneous protein C concentrate is given every third day and is well tolerated by the patient. No thrombotic events have occurred. We conclude that subcutaneous administration of protein C concentrate is a valuable therapeutic option in the long-term management of homozygous protein C deficiency and avoids the potential hazards of long-term central venous lines.
