Improved clinical outcome after PK-Guided Personalised Prophylaxis with my-PKFIT® in patients with hemophilia A without inhibitors

Main Article Content

Emine Türkkan
Gül Nihal Özdemir
Öykü Arslan
Serap Karaman
Zeynep Karakaş
Ayşegül Ünüvar


Objective: Prophylaxis is the gold standard in patients with severe hemophilia. In recent years, personalisation of prophylaxis treatment according to pharmacokinetic properties has been used in treatment. In this study, personalisation treatment experience based on the pharmacokinetic dosing tool my-PKfit results in pediatric and adult patients from three centers is shared.

Material and Methods: myPKfit ( was used to evaluate pharmacokinetic parameters in hemophilia A patients receiving recombinant Factor VIII (Takeda Advate ®) prophylaxis. 75 samples in 34 patients (3 samples in 7 patients, 2 samples in 27 patients) were analysed for pharmacokinetic evaluation. Age, weight and baseline FVIII level of the patients were recorded. Pharmacokinetic curves were obtained after entering sampling times, factor dose and sample results. The annual bleeding rate (ABR) of the patients were evaluated before and after the changes made after the pharmacokinetic evaluation.

Results: The median age of 34 patients with severe hemophilia A without inhibitors was 12.3±8.7 (1.5-37) years, and the mean weight was 40.0±22.0 (10-83) kg. All patients had a baseline FVIII level of less than or equal to 2 IU/dl. All patients were receiving primary or secondary/tertiary prophylaxis. The mean half-life of the factors of the patients was 9.6±1.4 (7.0-13.4) hours, and the mean time reached below 1 IU/dl was 48.9±11.2 (16.0-77.0) hours. Prophylactic factor therapy was changed in 17 patients after myPKfit, dose increased in 9 patients, the frequency increased in 6 patients, and both dose and frequency increased in 2 patients. With a mean follow-up period of 23.7 +16 (2-49) months, in 17 patients whose prophylaxis regimen was changed after the PK evaluation by myPkyfit, ABR was found to be significantly lower in the post-change period, compared to the last one year before the change of regimen (2.94 + 2.19 and 0.58 + 1.00 respectively) P: 0.028.

Discussion: A pharmacokinetic study by the Bayesian method is an increasingly used method for personalised prophylaxis regimen. We believe that myPKfit is beneficial in providing effective and appropriate prophylaxis.


Download data is not yet available.

Article Details

How to Cite
Türkkan, E., Özdemir, G. N., Arslan, Öykü, Karaman, S., Karakaş, Z., & Ünüvar, A. . (2022). Improved clinical outcome after PK-Guided Personalised Prophylaxis with my-PKFIT® in patients with hemophilia A without inhibitors. Medical Science and Discovery, 9(2), 90–95.
Research Article
Received 2022-01-17
Accepted 2022-02-08
Published 2022-02-18


Nilsson I, Berntrop E, Lofqvist T, Pettersson H. Twenty-five years experience of prophylactic treatment in severe hemophilia A and B. J Intern Med. 1992;232:25-32.

Nilsson IM, Hedner U, Ahlberg A. Haemophilia prophylaxis in Sweden. Acta Paediatr Scand. 1976;65:129–135.

Stoffman J, Andersson NG, Branchford B, Batt K, D'Oiron R, Escuriola Ettingshausen C, et al. Common themes and challenges in hemophilia care: a multinational perspective. Hematology. 2019;24(1):39-48.

Skinner MW. WFH--the cornerstone of global development: 45 years of progress. Haemophilia. 2008;14 Suppl 3:1-9.

Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357:535–544.

Oldenburg J. Optimal treatment strategies for hemophilia : achievements and limitations of current prophylactic regimens. Blood. 2015;125(13):2038–45.

Ar MC, Vaide I, Berntorp E. Methods for individualising factor VIII dosing in prophylaxis. Eur J Haematol Suppl. 2014;76:16–20.

Petrini P, Valentino LA, Gringeri A, Re WM, Ewenstein B. Individualizing prophylaxis in hemophilia: a review. Expert Rev Hematol. 2015;8(2):237-46.

Ar MC, Baslar Z, Soysal T. Personalized prophylaxis in people with hemophilia A: challenges and achievements. Expert Rev Hematol. 2016;9(12):1203-1208.

Hart DP, Kessler CM, Aledort L. Re-personalization and stratification of hemophilia care in an evolving treatment landscape. Hematology. 2019;24(1):737-741.

Reiniger A, Spotts G, Low-Baselli A et al. Optimizing prophylaxis: development of an advate PK calculator and dosing medical device based on a bayesian population PK model [Abstract OR07]. Haemophilia. 2014;20:15.

Álvarez-Román MT, Fernandez-Bello I, de la Corte-Rodríguez H, Hernández-Moreno AL, Martín-Salces M, Butta-Coll N, et al. Experience of tailoring prophylaxis using factor VIII pharmacokinetic parameters estimated with myPKFiT® in patients with severe haemophilia A without inhibitors. Haemophilia. 2017;23(1):50-54.

Nagao A, Yeung CHT, Germini F, Suzuki T. Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing. Thromb Res. 2019;173:79-84.

Arvanitakis A, Berntorp E, Astermark J. A comparison of MyPKFiT and WAPPS-Hemo as dosing tools for optimizing prophylaxis in patients with severe haemophilia A treated with Octocog alfa. Haemophilia. 2021;27(3):417-424.

Mingot-Castellano ME, Parra R, Núñez R, Martorell M. Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic-guided prophylaxis based on Bayesian models with myPKFiT®. Haemophilia. 2018;24(5):338-343.

Lock J, de Bekker-Grob EW, Urhan G, Peters M, Meijer K, Brons P, et al. Facilitating the implementation of pharmacokinetic-guided dosing of prophylaxis in haemophilia care by discrete choice experiment. Haemophilia 2016; 22: e1–10.

Björkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of hemophilia A. Haemophilia. 2010;16(4):597-605.

Inc B. MyPKFiT User Manual, DHF-000951 Revision 4.0. 2020. 2020. Accessed October 2020.

Blanchette VS, Zunino L, Grassmann V, Barnes C, Carcao MD, Curtin J, Jackson S, Khoo L, Komrska V, Lillicrap D, Morfini M, Romanova G, Stephens D, Zapotocka E, Rand ML, Blatny J. A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects. Thromb Haemost. 2021;121(10):1326-1336.

Pasca S, Zanon E. Savings without changing: How to use the MyPKfit® device to improve treatment strategies in a cohort of patients with haemophilia A. Thromb Res. 2019;183:1-3.