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

Hazendonk, H.C.A.M., van Moort, I., Mathôt, R.A.A., Fijnvandraat, K., Leebeek, F.W.G., Collins, P.W. and Cnossen, M.H. 2018. Setting the stage for individualized therapy in hemophilia: what role can pharmacokinetics play? Blood Reviews 32 (4) , pp. 265-271. 10.1016/j.blre.2018.01.001

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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.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Publisher: Elsevier
ISSN: 0268-960X
Date of First Compliant Deposit: 24 April 2018
Date of Acceptance: 31 January 2018
Last Modified: 25 Jul 2018 12:30

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