Impact of personalized pharmacokinetics-guided prophylaxis using standard half-life factor VIII in patients with hemophilia A

Introduction: Patients with Hemophilia A (HA) are at risk of severe bleeding and joint damage, which can lead to disability. Nonpharmacokinetic (PK)-guided prophylaxis treatment may not be suitable for all patients, as it does not involve personalized prophylaxis tailored to their activity levels. Objective: This study aimed to evaluate the clinical impact of PK-guided prophylaxis using standard half-life factor VIII (SHL FVIII) based on activity risk levels in patients with severe phenotype HA. Material and method: This retrospective and prospective cohort study compared data from the same group of patients receiving prophylactic treatment at Naradhiwasrajanagarindra Hospital between September 2023 and August 2024. Data from 6 months before and 6 months after the implementation of personalized PK-guided prophylaxis, combined with activity risk level design and digital diary application monitoring, were compared. Collected data included the annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), Hemophilia Joint Health Score (HJHS), Functional Independence Score in Hemophilia (FISH) and weekly consumption of SHL FVIII. Result: The study included 12 participants (mean age ± standard deviation [SD]: 16 ± 6.95 years). The results showed statistically significant reductions in ABR, AJBR, and HJHS (P = 0.005, 0.007, and 0.002, respectively). The median (interquartile range [IQR])FISH score increased from 26.50 (24 – 28.5) to 28 (26 – 29.75) (P = 0.002), while weekly SHL FVIII consumption did not significantly increase. Conclusion: Personalized treatment adjustments using PK data SHL FVIII and digital tools effectively improved treatment outcomes and supported patient activities.