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

• Published in: Blood reviews 2023-09, Vol.61, p.101098-101098, Article 101098
• Main Authors: Goedhart, Tine M.H.J., Janssen, A., Mathôt, Ron A.A., Cnossen, Marjon H.
• Format: Article
• Language: English
• Subjects: Factor IX; Factor VIII; Hemophilia A; Hemophilia B; Pharmacokinetics; Review
• ISSN: 0268-960X; 1532-1681
• DOI: 10.1016/j.blre.2023.101098

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.