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Helical Organization of Blood Coagulation Factor VIII on Lipid Nanotubes
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Issues complicating precision dosing for factor VIII prophylaxis.

Leonard A Valentino1, Peter L Turecek2, Herbert Gritsch3

  • 1Rush University, Chicago, IL, USA.

Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis
|August 25, 2018
PubMed
Summary
This summary is machine-generated.

Personalizing factor VIII (FVIII) prophylaxis using pharmacokinetic (PK) response improves outcomes for hemophilia A patients. However, assay, product, and patient factors complicate precise dosing, necessitating improved quality assurance and research.

Keywords:
Chromogenic substrate assayFVIII concentratesHemophiliaOne-stage clotting assayPharmacokinetic dosingProphylaxis

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Area of Science:

  • Hematology
  • Pharmacokinetics
  • Clinical Pharmacy

Background:

  • Personalized prophylaxis based on factor VIII (FVIII) pharmacokinetic (PK) response has been shown to reduce bleeding events in hemophilia A.
  • The accuracy of FVIII assays, choice of FVIII product, and interpatient variability can impact PK assessment and precise prophylaxis dosing.

Purpose of the Study:

  • To identify variables affecting FVIII assay, product selection, and interpatient differences in PK assessment for hemophilia A prophylaxis.
  • To explore obstacles to precision prophylactic dosing in hemophilia A.

Main Methods:

  • A comprehensive literature search was conducted for articles published between January 2004 and September 2017.
  • The search focused on identifying variables related to FVIII assay, FVIII product, and interpatient differences impacting PK assessment.

Main Results:

  • Discrepancies related to FVIII products and patient-specific factors were identified as significant issues.
  • Variability among different types of FVIII products (plasma-derived, recombinant) and interpatient response differences hinder precise dosing.
  • Assay inaccuracies and interpatient PK variability present obstacles to optimal FVIII prophylaxis.

Conclusions:

  • Improving FVIII measurement accuracy through stringent laboratory quality assurance and proficiency testing is crucial.
  • Widespread adoption of PK assessment is essential for fine-tuning FVIII dosing strategies.
  • Further research into patient characteristics and other bleeding risk factors is necessary for optimizing hemophilia A prophylaxis outcomes.