The impact of individualized prophylactic treatment based on pharmacokinetic parameters on the prognosis of patients with severe hemophilia A.

OBJECTIVES

To evaluate the effectiveness of PK-guided prophylaxis in severe hemophilia A.

METHODS

25 patients received PK-guided prophylaxis using a population PK tool (MyPKFiT), 30 received conventional fixed-dose prophylaxis. Outcomes over six months included bleeding frequency, joint health (HJHS), quality of life (SF-36), factor use, infusion frequency, costs, and adverse events.

RESULTS

PK-guided group had fewer bleeds, improved HJHS and SF-36 scores, lower FVIII use, reduced infusions, and lower costs (all p<0.001).

CONCLUSION

PK-guided prophylaxis improves clinical outcomes and reduces costs, but the retrospective design and small sample size limit generalizability; further studies are needed for confirmation.