Abstract Number: PB0616
Meeting: ISTH 2021 Congress
Background: Individualized treatment can be tailored by requirement of hemophilia patients . Pharmacokinetics (PK)-guided individualized treatment is important to avoid disability .
Aims: The aim of this study was to analyze whether PK-guided individual regimen impacts the cost of treatment.
Methods: 17 patients from our center’s registry were included in this study, which divided into individualized group (n=8) and non-individualized group (n=9) according to different treatment regimens after PK analysis. All patients received a single dose infusion of FVIII after a 72-hour washout period, post-infusion blood sample were collected at 9 h and 15 h, and the concentration of FVIII was detected. WAPPS system was used to calculate individual pharmacokinetic parameters. Amount of recombinant factor VIII usage, health-related quality of life (HRQoL) scores, number of joint bleeds, HJHS scores of joint rehabilitation and 6 major joint ultrasound scale scores (0-14 points) were collected in both groups for 6 months before and after PK test. SPSS 20.0 statistical software was used to analyze the differences in efficacy between and within groups using 2 Independent Samples Tests, and a significant difference was expressed at P<0.05.
Results: 17 patients had a median age of 21 (8-57) years and a median baseline FVIII:C activity of 0.6 (0.1-1.1) %. The number of bleeding (P= 0.020) and joint ultrasound scores (P=0.014) were significantly different in two groups, especially in the right elbow (P= 0.006). Although non-significantly difference was shown in HRQOL scores, the individualized group was significantly improved in social functioning (P= 0.031).Also there was no significant increase in FVIII consumption before and after individualized treatment.
Conclusions: Individualized treatment is expected to improve the quality of life by significantly reducing joint bleeds and improving social skills without significantly increasing the cost of additional FVIII consumption.
To cite this abstract in AMA style:Hong Q, Shu Qin C, Wen Jing Z, Xiao Hua H, Yong Fang C, Yu Hua Y, Xing Yi X. Association of Individual Pharmacokinetics-guided Regimen in Hemophilia A Patients and FVIII Consumption: A Single-center Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/association-of-individual-pharmacokinetics-guided-regimen-in-hemophilia-a-patients-and-fviii-consumption-a-single-center-study/. Accessed December 8, 2021.
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