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Review
. 2023 Sep:61:101098.
doi: 10.1016/j.blre.2023.101098. Epub 2023 May 9.

The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators

Tine M H J Goedhart  1 , A Janssen  2 , Ron A A Mathôt  3 , Marjon H Cnossen  4 ; OPTI-CLOT Study Group and SYMPHONY Consortium
Affiliations
Free article
Review

The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators

Tine M H J Goedhart et al. Blood Rev. 2023 Sep.
Free article

Abstract

Clinical guidelines and expert groups recommend the use of pharmacokinetic (PK)-guided dosing of factor replacement therapy for the treatment of bleeding disorders, especially for patients with hemophilia. Although PK-guided dosing is increasingly applied, it is generally not considered standard clinical practice. The aim of this scoping review is to map barriers and facilitators for the implementation of PK-guided dosing in clinical practice and to identify knowledge gaps. A literature search was performed and 110 articles were included that describe PK-guided dosing in patients with bleeding disorders, mostly hemophilia A. We defined two overarching themes, efficacy and feasibility, and discuss five topics within each theme. For each topic, barriers, facilitators and knowledge gaps were described. Although consensus was found with regard to some topics, contradicting reports were found for others, especially with respect to the efficacy of PK-guided dosing. These contradictions highlight the need for future research to elucidate current ambiguities.

Keywords: Factor IX; Factor VIII; Hemophilia A; Hemophilia B; Pharmacokinetics; Review.

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Conflict of interest statement

Declaration of Competing Interest M.H.C.'s institution has received investigator-initiated research and travel grants as well as speaker fees over the years from the Netherlands Organization for Scientific Research (NWO) and Netherlands National Research Agenda (NWA), the Netherlands Organization for Health Research and Development (ZonMw), the Dutch Innovatiefonds Zorgverzekeraars, Baxter/Baxalta/Shire/ Takeda, Pfizer, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis and Nordic Pharma, and for serving as a steering board member for Roche, Bayer and Novartis for which fees go to the Erasmus MC as an institution. M.H.C. is coordinator of the European Expert Centers for rare hematological diseases in Health Care Provider (HCP) Erasmus MC, University Medical Center Rotterdam. R.A.A.M. has received grants from governmental and societal research institutes such as NWO, ZonMW, Dutch Kidney Foundation and Innovation Fund and Unrestricted Investigator Research grants from Baxter/ Baxalta/ Shire/Takeda, Bayer, CSL Behring, Sobi and CelltrionHC. He has served as advisor for Bayer, CSL Behring, Merck Sharp & Dohme, Baxter/ Baxalta/ Shire/Takeda. All grants and fees paid to the institution. Other authors have no conflict of interest to declare for this paper.

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