Department of Pediatric Hematology, Erasmus University Medical Center, Sophia Children's Hospital, Rotterdam, The Netherlands.
Hospital Pharmacy - Clinical Pharmacology, Academic Medical Center, Amsterdam, The Netherlands.
Blood Rev. 2018 Jul;32(4):265-271. doi: 10.1016/j.blre.2018.01.001. Epub 2018 Jan 31.
Replacement therapy with clotting factor concentrates (CFC) is the mainstay of treatment in hemophilia. Its widespread application has led to a dramatic decrease in morbidity and mortality in patients, with concomitant improvement of quality of life. However, dosing is challenging and costs are high. This review discusses benefits and limitations of pharmacokinetic (PK)-guided dosing of replacement therapy as an alternative for current dosing regimens. Dosing of CFC is now primarily based on body weight and based on its in vivo recovery (IVR). Benefits of PK-guided dosing include individualization of treatment with better targeting, more flexible blood sampling, increased insight into association of coagulation factor levels and bleeding, and potential overall lowering of overall costs. Limitations include a slight burden for the patient, and availability of closely collaborating, experienced clinical pharmacologists.
凝血因子浓缩物（CFC）的替代治疗是血友病治疗的主要手段。它的广泛应用显著降低了患者的发病率和死亡率，同时提高了生活质量。然而，剂量的确定具有挑战性，且成本高昂。本文讨论了基于药代动力学（PK）指导的替代治疗剂量作为当前剂量方案的替代方案的优缺点。目前，CFC 的剂量主要基于体重和体内恢复（IVR）。PK 指导剂量的优点包括个体化治疗，更好的靶向性，更灵活的采血，增加对凝血因子水平与出血之间关联的了解，以及潜在的总体降低总费用。局限性包括患者的负担略重，以及需要有密切合作、经验丰富的临床药理学家。
