Expert review of hematology · 2025
The impact of individualized prophylactic treatment based on pharmacokinetic parameters on the prognosis of patients with severe hemophilia A.
СтатьяПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
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Gao Xinfang, Xu Panpan, Luo Xinguo, Ye Hongwei, Yu Fangquan, Hu Shanshan, Huang Li, Xiang Jingjing
Аннотация
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.
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.