Exploring the Relationship Between Personalization of Care and Participation in Sport Activities Among People with Severe Hemophilia A Across Europe: Post Hoc Analysis of the CHESS II Study.

Abstract

Purpose: 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).

Patients and methods: 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.

Results: 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.

Conclusion: 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.