Blenkiron Tom, Ferri Grazzi Enrico, Burke Tom, Watt Maureen, Davis Kimberly H
HCD Economics Ltd, Daresbury, UK.
Faculty of Social Care, University of Chester, Chester, UK.
J Blood Med. 2025 Aug 6;16:359-372. doi: 10.2147/JBM.S521079. eCollection 2025.
To describe the demographic and clinical characteristics of patients with hemophilia A receiving different levels of treatment personalization (TP), and to assess the relationship between TP and sport active time (SAT).
This post hoc analysis of the CHESS II study used data from physician-completed patient record forms and patient self-completion forms for adult males receiving prophylaxis for severe hemophilia A in Europe between November 2018 and October 2020. SAT was assessed using propensity score matching (PSM) across levels of TP, including pharmacokinetic (PK)-guided and non-PK-guided.
Of 54 patients, 32 (59.3%) received TP. Of these, 22 (68.8%) and 10 (31.3%) received non-PK-guided and PK-guided treatment, respectively. Median age varied between the TP and no-TP groups (29.5 and 34.0 years, respectively). Median (IQR) annual bleeding incidence was higher with non-PK-guided vs PK-guided TP (4.0 [3.0-8.0] vs 3.5 [2.0-4.0]). Median (IQR) problem joints were similar with non-PK-guided and PK-guided TP (1.0 [0.0-1.0] and 1.0 [0.0-2.0]). Patients in the TP vs no-TP group had higher median (IQR) SAT per month (3.3 [1.8-6.2] vs 1.8 [0.7-5.0] hours). Median (IQR) SAT per month was higher with PK-guided vs non-PK-guided TP (4.0 [3.0-20.0] vs 3.0 [1.3-5.3] hours). After controlling for confounding in the PSM model, SAT remained higher with TP vs no-TP and with PK-guided vs non-PK-guided TP. In both PSM models, P values were <0.05 for the average treatment effect and <0.01 for the average treatment effect on the treated. Sensitivity analyses confirmed the robustness of the PSM.
Patients receiving TP vs no-TP had higher median SAT. Of those with TP, those receiving PK-guided vs non-PK-guided TP experienced lower bleeding rates and higher SAT. PK-guided TP may help patients to be more active, potentially gaining the clinical and psychosocial benefits of exercise.
描述接受不同程度治疗个性化(TP)的甲型血友病患者的人口统计学和临床特征,并评估TP与运动活跃时间(SAT)之间的关系。
这项对CHESS II研究的事后分析使用了2018年11月至2020年10月期间欧洲成年男性接受重度甲型血友病预防治疗的医生填写的患者记录表和患者自填表格中的数据。通过倾向得分匹配(PSM)评估不同TP水平(包括药代动力学(PK)指导和非PK指导)下的SAT。
54例患者中,32例(59.3%)接受了TP。其中,22例(68.8%)接受非PK指导治疗,10例(31.3%)接受PK指导治疗。TP组和非TP组的中位年龄不同(分别为29.5岁和34.0岁)。非PK指导的TP组与PK指导的TP组相比,年出血发生率中位数(IQR)更高(4.0 [3.0 - 8.0] 对比 3.5 [2.0 - 4.0])。非PK指导的TP组与PK指导的TP组的问题关节中位数(IQR)相似(均为1.0 [0.0 - 1.0] 和1. [0.0 - 2.0])。TP组患者每月的SAT中位数(IQR)高于非TP组(3.3 [1.8 - 6.2] 小时对比1.8 [0.7 - 5.0] 小时)。PK指导的TP组每月的SAT中位数(IQR)高于非PK指导的TP组(4.0 [3.0 - 20.0] 小时对比3.0 [1.3 - 5.3] 小时)。在PSM模型中控制混杂因素后,TP组与非TP组以及PK指导的TP组与非PK指导的TP组相比,SAT仍然更高。在两个PSM模型中,平均治疗效果的P值<0.05,对治疗对象的平均治疗效果的P值<0.01。敏感性分析证实了PSM的稳健性。
接受TP的患者与未接受TP的患者相比,SAT中位数更高。在接受TP的患者中,接受PK指导的TP组与非PK指导的TP组相比,出血率更低,SAT更高。PK指导的TP可能有助于患者更积极活动,潜在地获得运动带来的临床和心理社会益处。