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Comparison Pharmacokinetic Dosing Tools in Hemophilia A Children

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Abstract

Prophylaxis is the gold standard for the management of hemophilia A patients. It has been shown that prophylaxis regulated with pharmacokinetic (PK) data reduces frequency of bleeding and cost of treatment. To determine the best prophylaxis regimen, PK dosing tools using the Bayesian method have been developed. We aimed to compare two PK dosing tools. Blood samples were drawn before, 4, 24, and 48 h after FVIII infusions from patients with severe hemophilia A and inhibitor negative. FVIII levels were measured by PTT-based one-stage assay method. PK parameters obtained using WAPPS and myPKFiT, which are web-accessible PK dosing tools using Bayesian algorithm, and daily prophylaxis dose estimated by the programs were compared. Forty-two hemophilia A patients [median age 13 years (IQR 8.9–16.4)] included in the study. There was no difference between the daily dose of FVIII given for prophylaxis and the dose recommended by the myPKFiT for the 1% trough level; whereas, a significant difference was found with the WAPPS. The half-lives of FVIII did not differ between the two dosing tools; however, significant differences were found in the estimated dose, clearances, and times to 1% trough level. There was no significant difference between PK data of patients who received Advate® and those who received non-Advate® factor concentrates. Choice of PK dosing tool can affect recommended FVIII dose. However, target trough levels should be individualized according to bleeding phenotype and daily activity of patient.

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This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.

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Authors and Affiliations

  1. Department of Pediatrics, Ankara City Hospital, University of Health Sciences, Ankara, Turkey

    Can Alp Genç

  2. Department of Pediatric Hematology and Oncology, Ankara City Hospital, University of Health Sciences, Ankara, Turkey

    Dilek Gürlek Gökçebay, Vildan Koşan Çulha & Namık Yaşar Özbek

  3. Department of Pediatric Hematology, Faculty of Medicine, Gazi University, Ankara, Turkey

    Zühre Kaya

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  1. Can Alp Genç
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  2. Dilek Gürlek Gökçebay
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  5. Namık Yaşar Özbek
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Correspondence to Can Alp Genç.

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Genç, C.A., Gürlek Gökçebay, D., Koşan Çulha, V. et al. Comparison Pharmacokinetic Dosing Tools in Hemophilia A Children. Indian J Hematol Blood Transfus 40, 108–115 (2024). https://doi.org/10.1007/s12288-023-01671-0

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  • DOI: https://doi.org/10.1007/s12288-023-01671-0

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