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A therapeutic, highly purified factor XI concentrate from human plasma
M Burnouf-Radosevich1, T Burnouf
1Centre Régional de Transfusion Sanguine de Lille, France.
Transfusion
|November 1, 1992
Summary
A novel, highly purified factor XI (FXI) concentrate was developed for treating FXI deficiency. This preparation demonstrates high stability, potency, and safety, offering an alternative to fresh-frozen plasma.
Area of Science:
- Biochemistry
- Hematology
- Biotechnology
Background:
- Factor XI (FXI) deficiency can lead to bleeding disorders.
- Current treatment options, like fresh-frozen plasma, carry risks and limitations.
- A purified FXI concentrate could offer a safer and more effective therapeutic alternative.
Purpose of the Study:
- To develop and characterize a highly purified factor XI concentrate from human plasma.
- To assess the safety and efficacy of the FXI concentrate for potential therapeutic use.
Main Methods:
- Plasma-derived FXI concentrate prepared using filter adsorption and cation exchange chromatography.
- Solvent-detergent treatment for viral inactivation.
- Characterization of purity, potency, stability, and contaminants.
- In vitro and in vivo safety assessments (thrombogenicity, hypotensive, and toxic effects).
Main Results:
- High specific activity (130-150 U/mg protein) and potency (approx. 100 U/mL) achieved.
- Excellent stability demonstrated for at least 24 hours at room temperature.
- Minimal protein contaminants and undetectable levels of fibrinogen, other coagulation factors, and activated factors.
- No thrombogenic, hypotensive, or toxic effects observed in animal studies.
Conclusions:
- A highly purified and stable factor XI concentrate can be successfully prepared from human plasma.
- The concentrate exhibits a favorable safety profile, with no observed thrombogenic or toxic effects.
- This FXI preparation holds promise for substitution therapy in factor XI deficiency, potentially replacing fresh-frozen plasma.