Setting the stage for individualized therapy in hemophilia: What role can pharmacokinetics play?

Autor: Hazendonk HCAM; Department of Pediatric Hematology, Erasmus University Medical Center, Sophia Children's Hospital, Rotterdam, The Netherlands., van Moort I; Department of Pediatric Hematology, Erasmus University Medical Center, Sophia Children's Hospital, Rotterdam, The Netherlands., Mathôt RAA; Hospital Pharmacy - Clinical Pharmacology, Academic Medical Center, Amsterdam, The Netherlands., Fijnvandraat K; Department of Pediatric Hematology, Academic Medical Center, Amsterdam, The Netherlands; Department of Plasma Proteins, Sanquin Research, Amsterdam, The Netherlands., Leebeek FWG; Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands., Collins PW; Department of Haematology, School of Medicine, Cardiff University, United Kingdom., Cnossen MH; Department of Pediatric Hematology, Erasmus University Medical Center, Sophia Children's Hospital, Rotterdam, The Netherlands. Electronic address: m.cnossen@erasmusmc.nl.
Jazyk: angličtina
Zdroj: Blood reviews [Blood Rev] 2018 Jul; Vol. 32 (4), pp. 265-271. Date of Electronic Publication: 2018 Jan 31.
DOI: 10.1016/j.blre.2018.01.001
Abstrakt: Replacement therapy with clotting factor concentrates (CFC) is the mainstay of treatment in hemophilia. Its widespread application has led to a dramatic decrease in morbidity and mortality in patients, with concomitant improvement of quality of life. However, dosing is challenging and costs are high. This review discusses benefits and limitations of pharmacokinetic (PK)-guided dosing of replacement therapy as an alternative for current dosing regimens. Dosing of CFC is now primarily based on body weight and based on its in vivo recovery (IVR). Benefits of PK-guided dosing include individualization of treatment with better targeting, more flexible blood sampling, increased insight into association of coagulation factor levels and bleeding, and potential overall lowering of overall costs. Limitations include a slight burden for the patient, and availability of closely collaborating, experienced clinical pharmacologists.
(Copyright © 2018 The Authors. Published by Elsevier Ltd.. All rights reserved.)
Databáze: MEDLINE
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