Related Experiment Video
Updated: Jul 22, 2026
13:08
Measurement of Factor V Activity in Human Plasma Using a Microplate Coagulation Assay
Published on: September 9, 2012
Prophylaxis in factor IX deficiency product and patient variation.
C T Kisker1, A Eisberg, B Schwartz
1University of Iowa Hospitals and Clinics, Iowa City, IA 52242, USA. c-kisker@uiowa.edu
Summary
This study compared plasma-derived factor IX (pd-FIX) and recombinant factor IX (r-FIX) for hemophilia prophylaxis. pd-FIX showed better recovery and a similar half-life, suggesting potentially lower dosing needs for maintaining prophylactic FIX levels.
Area of Science:
- Hematology
- Pharmacokinetics
- Pharmacoeconomics
Background:
- Severe hemophilia A requires factor IX (FIX) replacement therapy for prophylaxis.
- Understanding the pharmacokinetics of different FIX concentrates is crucial for optimizing treatment.
- Plasma-derived (pd-FIX) and recombinant (r-FIX) concentrates have different characteristics that may affect dosing and cost.
Purpose of the Study:
- To determine the optimal dosing of pd-FIX and r-FIX for maintaining a prophylactic FIX level of >/=2% in severe hemophilia patients.
- To compare the pharmacokinetic profiles (recovery and half-life) of pd-FIX and r-FIX.
- To estimate the cost-effectiveness of prophylaxis using both FIX concentrates.
Main Methods:
- A double-blind, two-period crossover study involving 15 non-inhibitor severe hemophilia subjects.
- Assessment of pharmacokinetics, including recovery and half-life, for Mononine (pd-FIX) and BeneFix (r-FIX).
- Computer-simulated multiple-dose activity curves to determine dosing for maintaining prophylactic FIX levels.
Main Results:
- pd-FIX demonstrated significantly higher median recovery (1.67 IU/dL per IU/kg) compared to r-FIX (0.86 IU/dL per IU/kg) (P = 0.0002).
- Median half-lives were similar: 12.9 h for pd-FIX and 13.7 h for r-FIX (P = 0.016).
- Estimated median annual prophylaxis costs for a child were lower with pd-FIX ($19,972) than r-FIX ($34,456).
Conclusions:
- pd-FIX exhibits superior recovery, potentially allowing for lower doses to achieve target prophylactic FIX levels.
- Despite similar half-lives, pharmacokinetic variability necessitates individualized dosing assessments for both pd-FIX and r-FIX.
- The choice of FIX concentrate can significantly impact the cost of long-term prophylaxis.
Related Concept Videos
01:18Anticoagulant Drugs: Vitamin K Antagonists and Direct Oral Anticoagulants
Oral anticoagulants are vital tools in preventing and treating blood clotting disorders. This diverse class of medications can be categorized as vitamin K antagonists, exemplified by warfarin, and direct thrombin inhibitors (DTIs), such as dabigatran, as well as factor Xa inhibitors, including rivaroxaban.
Warfarin, a prominent vitamin K antagonist family member, exerts its effect by inhibiting the enzyme VKORC1 (vitamin K epoxide reductase complex 1). By hindering this enzyme, warfarin...
Warfarin, a prominent vitamin K antagonist family member, exerts its effect by inhibiting the enzyme VKORC1 (vitamin K epoxide reductase complex 1). By hindering this enzyme, warfarin...
01:29Factors Affecting Protein-Drug Binding: Patient-Related Factors
Protein-drug binding, a pivotal aspect of pharmacokinetics, is subject to considerable variability influenced by an array of patient-related factors. The intricate interplay of age, individual differences, and pathological conditions significantly impact the binding dynamics and subsequent pharmacological effects.
Age stands as a key determinant in protein-drug binding. Neonates, characterized by low albumin content, experience heightened concentrations of unbound drugs such as phenytoin and...
Age stands as a key determinant in protein-drug binding. Neonates, characterized by low albumin content, experience heightened concentrations of unbound drugs such as phenytoin and...
01:22Biopharmaceutical Factors Influencing Drug Product Design: Overview
Rational drug product design integrates knowledge of the drug’s physicochemical properties, formulation components, manufacturing techniques, and intended route of administration. Each factor influences the drug’s performance, including how it is released, absorbed, and eliminated in the body.The physicochemical properties of a drug—such as solubility, stability, and particle size—affect its compatibility with excipients and the choice of dosage form. Excipients, though pharmacologically...
01:22Pharmaceutical Alternatives: Stability-Related Therapeutic Nonequivalence
Generic intravenous (IV) drugs are considered bioequivalent to their branded counterparts due to their 100% bioavailability upon administration. However, variations in stability among different drug products can significantly influence their therapeutic performance, even if they are pharmaceutically equivalent.Cefuroxime, a prophylactic antimicrobial, is often used as a single-dose IV injection for patients undergoing coronary artery bypass grafting surgery. A 3 g dose typically provides...
01:29Pharmacogenetic Phenotypes: Alterations in Pharmacokinetics, Drug Targets and Biologic Milieu
Genetic variations significantly influence drug response through pharmacokinetics, receptor interactions, and biologic milieu modifications. Pharmacokinetic alterations impact drug metabolism and clearance, affecting efficacy and toxicity. Variants in drug-metabolizing enzymes, such as CYP2C9 and CYP2C19, alter drug activation and elimination. For example, CYP2C9 loss-of-function variants require lower warfarin doses to prevent excessive bleeding, while CYP2C19 variants reduce clopidogrel...
01:29Venous Thrombosis III: Interprofessional Care
Venous thrombosis requires effective prevention and treatment strategies to improve patient outcomes and reduce potential complications.Prevention StrategiesHealthcare providers must prioritize preventing venous thromboembolism (VTE) for all adult patients upon admission. Interventions depend on bleeding and thrombosis risk, medical history, current medications, diagnoses, planned procedures, and patient preferences. Patients on bed rest should change positions every two hours and, if not...