Abstract

Objective: Factor VIII (FVIII) prophylaxis is now considered a gold standard for the treatment of hemophilia A patients to prevent bleeding and maintain musculoskeletal function. FVIII prophylaxis, tailored with pharmacokinetic data, is the most successful approach in preventing bleeding and reducing the cost of treatment.

Material and Methods: In this study, 17 patients with severe hemophilia A (median age 11.5 years, median factor 25 U / kg) whose FVIII pharmacokinetic data were obtained through the myPKFiT web access program, were presented.

Results: The mean FVIII half-life of the patients was 9.1 hours (min-max: 6.8-11.4); the mean FVIII clearance was 4.7 ml/hour/kg (min-max: 2.6-6.8); and the mean duration of FVIII levels under 1% was 47 hours (min-max: 33-61). The half-life of FVIII was increased with the age of the patient. When a weekly dose schedule simulated with the target FVIII trough level above 1%, it was observed that the dose of FVIII was sufficient in three, it was high in five, and it was insufficient in nine patients.

Conclusion: In our study, prophylaxis doses and frequencies were adjusted according to the FVIII pharmacokinetic data.

Keywords: Hemophilia A, Pharmacokinetic data, Prophylaxis, The Bayesian method

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Copyright and license

Copyright © 2020 The Author(s). This is an open access article distributed under the Creative Commons Attribution License (CC BY), which permits unrestricted use, distribution, and reproduction in any medium or format, provided the original work is properly cited.

How to cite

1.
Aksu T, Koşan V, Kaya Z, Guzelkucuk Z, Gürlek Gökçebay D, Yaralı HN, et al. Pharmacokinetic Studies of Recombinant FVIII in Hereditary Factor VIII Deficiency Patients. Turk J Pediatr Dis [Internet]. 2020 Sep. 29 [cited 2025 Aug. 23];14(5):408-12. Available from: https://turkjpediatrdis.org/article/view/661