PK指导下血友病A患者个体化预防治疗
Journal: Basic Medical Theory Research DOI: 10.12238/bmtr.v3i4.4467
Abstract
血友病作为常见的性联遗传性疾病广泛存在于世界的每个角落,其对病人的威胁在于反复关节肌肉出血致残及严重出血致死。血友病患者由于凝血因子缺乏需要终身进行因子替代治疗。现代血友病治疗理念已从按需替代治疗模式转变为预防治疗模式。预防治疗被公认为重型血友病患者的标准治疗方法,提倡在有条件的地方尽早开始预防治疗。其目的在于使病人有维持健康生活状态的机会,预防严重出血事件和骨关节病变的发生。但昂贵的治疗费用和频繁的静脉穿刺严重阻碍了其临床应用。近期研究发现,凝血因子在不同患者体内的药代动力学(PK)以及不同患者的出血类型存在明显差异,提出了个体化预防治疗的概念。PK指导的个体化预防治疗综合评估了血友病A患者的出血表型、凝血因子的PK、活动水平、依从性及治疗成本等,为患者制定了相对完善的个体化治疗方案,从而减少出血次数,提高生活质量。
Keywords
血友病A；预防治疗；药物代谢动力学
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[6] Collins PW, BjÖrkman S, Fischer K,BlanchetteV, et al. Factor VIII requ irement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of vari ance in pharmacokinetics and treatment regimens.J Thromb Haemost2010;8:269-75.
[7] Den Uijl I, Biesma D, Grobbee D, Fischer K. Turning severe into modera te haemophilia by prophylaxis: are we reaching our goal? Blood Transfus 2013:11:364-9.
[8] Collins PW,Fischer K,Morfini M,et al. Implications of coagulation factor Ⅷ and Ⅸ pharmacokinetics in the pr ophylactic treatment of haemophilia[J].Haemophilia,2011,17(1):2-10．
[9] Fijnvandraat K,Peters M,ten Cate JW.Inter-individual variation in halflife of infusedrecombinant factor Ⅷ is related to pre-infusion von Willeb rand factor antigen levels[J].Br J Hae matol,1995,91(2):474-476.
[10] Kepa S,Horvath B,Ｒeitter-Pf oertner S,et al.Parameters influencing FⅧ pharmacokinetics in patients with severe and moderate haemophilia A[J]. Haemophilia,2015,21(3):343-350．
[11] Chen ZP,Li PJ,Li G,et al.Pharma cokinetic studies of factor Ⅷ in Chin ese boys with severe hemophilia A: a single-center study[J].Chin Med J (Eng l),2018,131(15):1780-1785．
[12] Tiede A,Cid R,Goldmann G, et al.Body mass index best predicts rec overy of recombinant factor Ⅷ in und erweight to obese patients with severe haemophilia A[J].Thromb Haemost,2020, 120(2):277．
[13] Bjrkman S,Folkesson A,Berntor p E.In vivo recovery of factor Ⅷand factor Ⅸ: intra-and interindividual variance in a clinical setting[J].Haem ophilia,2007,13(1):2-8．
[14] Carolyn R Broderick,Robert D Herbert,Jane Latimer,Chris Barnes,Simon A Brown. Association between physical activity and risk of bleeding in child ren with hemophilia. JAMA, October 10,2012—Vol308,No.14145-9.
[15] Geraghty S,Dunkley T, Harring ton C, et al. Practice patterns in haem ophilia A therapy–global progress tow ards optimal care.Haemophilia 2006;12:75-81.
[16] Khair K, Gibson F, Meerabeau L. The benefits of prophylaxis: views of adolescents with severe haemophilia. Haemophilia 2012;18:e286-9.
[17] M.CARLSSON,E.BERNTORP,S. BJÖRKMAN,S.LETHAGEN,R.LJUNG.Improvedcost-effectiveness by pharmacokinetic do sing of factor VIII in prophylactic tre atment of haemophilia A.Haemophilia (1997),96-101.
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