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Prophylaxis in rare coagulation disorders -- factor XIII deficiency.
1Division of Hematology, Hemostasis/Thrombosis Research, Children's Hospital of Orange County, Orange, CA 92868, USA. djn0@choc.org
Thrombosis Research
|April 18, 2006
Summary
Factor XIII (FXIII) deficiency, a rare bleeding disorder, is effectively managed with FXIII concentrate, leading to normal clotting and good bleeding control. Monthly prophylaxis shows excellent patient response without inhibitor development or viral infections.
Area of Science:
- Hematology
- Coagulation Disorders
- Rare Diseases
Background:
- Factor XIII (FXIII) deficiency is a rare inherited bleeding disorder.
- It is characterized by a predisposition to bleeding, notably umbilical stump bleeding in 80% of cases.
- FXIII's role extends beyond the terminal clotting cascade, influencing the entire coagulation process.
Purpose of the Study:
- To evaluate the efficacy of FXIII concentrate (Fibrogammin P) in treating FXIII deficiency.
- To assess the effectiveness of monthly prophylaxis with plasma-derived, pasteurized FXIII concentrate.
- To examine patient outcomes, including bleeding control, inhibitor development, and viral seroconversion.
Main Methods:
- Treatment involved administration of FXIII concentrate (Fibrogammin P).
- Prophylaxis studies were conducted in France and the USA.
- Monthly prophylaxis regimens were employed using a plasma-derived, pasteurized concentrate.
Main Results:
- FXIII concentrate treatment re-established normal clotting patterns.
- Monthly prophylaxis demonstrated an excellent response in patients.
- Patients achieved good bleeding control with no inhibitor development or viral seroconversion.
Conclusions:
- FXIII concentrate is an effective treatment for FXIII deficiency, restoring normal hemostasis.
- Monthly prophylaxis with this concentrate provides excellent disease control and safety.
- Disease registries are crucial for further exploration of FXIII deficiency manifestations and outcomes.