Abstract
Original language | English |
---|---|
Article number | 101098 |
Journal | Blood Reviews |
Volume | 61 |
Early online date | 2023 |
DOIs | |
Publication status | Published - Sept 2023 |
Keywords
- Factor IX
- Factor VIII
- Hemophilia A
- Hemophilia B
- Pharmacokinetics
- Review
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In: Blood Reviews, Vol. 61, 101098, 09.2023.
Research output: Contribution to journal › Review article › Academic › peer-review
TY - JOUR
T1 - 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
AU - Goedhart, Tine M. H. J.
AU - OPTI-CLOT study group and SYMPHONY consortium
AU - Janssen, A.
AU - Mathôt, Ron A. A.
AU - Cnossen, Marjon H.
N1 - Funding Information: 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.This study was performed as part of the OPTI-CLOT International Multicenter Study Group, “Patient tailOred PharmacokineTIc-guided dosing and other pharmacometrics of CLOTting factor concentrates, alternative hemostatic medication and desmopressin in bleeding disorders,” which is currently embodied by WP6 within the SYMPHONY consortium. The SYMPHONY NWO-NWA consortium which aims to orchestrate personalized treatment in patients with bleeding disorders, is a unique collaboration between patients, health care professionals and translational & fundamental researchers specialized in inborn bleeding disorders, as well as experts from multiple disciplines. It aims to identify best treatment choice for each individual based on bleeding phenotype. In order to achieve this goal, work packages (WPs) have been organized according to three themes e.g. Diagnostics (WPs 3&4), Treatment (WPs 5-9) and Fundamental Research (WPs 10-12). This research received funding from the Netherlands Organization for Scientific Research (NWO) in the framework of the NWA-ORC Call grant agreement NWA.1160.18.038. Principal investigator: Dr. M.H. Cnossen. Project manager: Dr. S.H. Reitsma. Beneficiaries of the SYMPHONY consortium: Erasmus MC and Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam, project leadership and coordination; Sanquin Diagnostics; Sanquin Research; Amsterdam University Medical Centers; University Medical Center Groningen; University Medical Center Utrecht; Leiden University Medical Center; Radboud University Medical Center. All Hemophilia Treatments Centers are active members of ERN/EuroBloodNet; Netherlands Society of Hemophilia Patients (NVHP); Netherlands Society for Thrombosis and Hemostasis (NVTH); Bayer B.V. CSL Behring B.V. Swedish Orphan Biovitrum (Belgium) BVBA/SPRL. Outside the SYMPHONY consortium, additional beneficiaries specifically of the OPTI-CLOT TARGET study are Novonordisk, as well as Roche (Partitura) and Stichting Hemophilia (WP05). The authors are grateful to Maarten Engel and Wichor Bramer from the Erasmus MC Medical Library for developing and updating literature search strategies and support for reference management. Funding Information: The SYMPHONY NWO-NWA consortium which aims to orchestrate personalized treatment in patients with bleeding disorders, is a unique collaboration between patients, health care professionals and translational & fundamental researchers specialized in inborn bleeding disorders, as well as experts from multiple disciplines. It aims to identify best treatment choice for each individual based on bleeding phenotype. In order to achieve this goal, work packages (WPs) have been organized according to three themes e.g. Diagnostics (WPs 3&4), Treatment (WPs 5-9) and Fundamental Research (WPs 10-12). This research received funding from the Netherlands Organization for Scientific Research (NWO) in the framework of the NWA-ORC Call grant agreement NWA.1160.18.038. Principal investigator: Dr. M.H. Cnossen. Project manager: Dr. S.H. Reitsma. Publisher Copyright: © 2023 The Authors
PY - 2023/9
Y1 - 2023/9
N2 - 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.
AB - 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.
KW - Factor IX
KW - Factor VIII
KW - Hemophilia A
KW - Hemophilia B
KW - Pharmacokinetics
KW - Review
UR - http://www.scopus.com/inward/record.url?scp=85162011768&partnerID=8YFLogxK
U2 - https://doi.org/10.1016/j.blre.2023.101098
DO - https://doi.org/10.1016/j.blre.2023.101098
M3 - Review article
C2 - 37321952
SN - 0268-960X
VL - 61
JO - Blood Reviews
JF - Blood Reviews
M1 - 101098
ER -