Pharmacokinetics in haemophilia treatment
Authors:
J. Máchal
Authors‘ workplace:
Oddělení dětské hematologie FN Brno a LF MU Brno
Published in:
Transfuze Hematol. dnes,25, 2019, No. 3, p. 230-240.
Category:
Review/Educational Papers
Overview
Haemophilia is an inborn X-linked bleeding disorder, which is caused by a deficiency of coagulation factor VIII in haemophilia A and factor IX in haemophilia B. Currently, the standard treatment regimen for severe forms of the disease is the prophylactic intravenous administration of the deficient factor. The initial dosage is assessed identically for all persons with haemophilia and subsequently modified empirically depending on the individual bleeding phenotype. Due to the large inter-individual variability in the response, to the administered factor, this „one-size-fits-all “approach is now deemed obsolete. Treatment individualization and personalization is gaining in importance. One of the most important ways to individualize treatment is using a pharmacokinetic approach. Pharmacokinetics of the administered concentrate provide to detailed and individually specific information regarding treatment efficacy. Based on pharmacokinetics, it is possible to adjust treatment – dose, frequency of infusions and even the choice of factor concentrate. It is complicated to perform a classic pharmacokinetic assessment, which is why a population pharmacokinetic approach is becoming more and more attractive. Using sophisticated population models, it is possible to assess the individual pharmacokinetics from the limited number of samples obtained in different situations. Population pharmacokinetic models are easily available for clinicians as internet-based applications. Apart from assessing the individual pharmacokinetics, they can be used to evaluate the efficacy of the current dosing regimen and generate a new one based on the required frequency of application, trough level or dose. Mobile applications, which will allow each patient to gain access, to his/her current factor level are being developed. With this knowledge, a haemophiliac will be able to assess the suitability of a planned activity at any given moment and schedule their activities in advance according to the estimated protection level.
Keywords:
haemophilia – individualization – pharmacokinetics – population pharmacokinetics
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Labels
Haematology Internal medicine Clinical oncologyArticle was published in
Transfusion and Haematology Today
2019 Issue 3
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