药代动力学指导下1例重型血友病A合并急性髓系白血病的个体化治疗  

Pharmacokinetic⁃guided individualized treatment for a case of severe hemophilia A complicated with acute myeloid leukemia

作　　者：林淑华[1] 李华晖 Lin Shuhua;Li Huahui(Department of Hematology,Zhongshan City People’s Hospital,Zhongshan 528400,China)

机构地区：[1]中山市人民医院血液内科,中山528400

出　　处：《血栓与止血学》2025年第1期20-25,共6页Chinese Journal of Thrombosis and Hemostasis

摘　　要：重型血友病A合并急性髓系白血病时,治疗方案需兼顾出血风险控制与白血病的治疗,本研究旨在探索药代动力学(pharmacokinetics,PK)指导下的人凝血因子Ⅷ预防治疗方案在此类患者中的应用。通过回顾性分析病例,发现在PK指导下,将FⅧ目标谷浓度设定为5%的预防治疗方案能有效控制出血,患者顺利完成4个化疗疗程,仅发生1次可疑出血,未影响白血病治疗,随访显示骨髓持续完全缓解(complete remission,CR),且未增加患者经济负担。结果表明,PK指导的预防治疗在血友病A合并白血病患者中具有可行性,为个体化治疗提供了新策略。When severe hemophilia A is combined with acute myeloid leukemia,the treatment plan needs to take into account both bleeding risk control and leukemia treatment.This study aims to explore the application of human coagulation factorⅧpreventive treatment under pharmacokinetics(PK)guidance in the treatment of such patients.Through retrospective analysis of cases,it was found that under the guidance of PK,the preventive treatment plan with the target trough concentration of FⅧset at 5%can effectively control bleeding.The patient successfully completed 4 courses of chemotherapy,with only one suspected bleeding,which did not affect the treatment of leukemia.Follow⁃up showed that the bone marrow continued to be completely remitted(CR),and the economic burden of the patient was not increased.The results showed that PK⁃guided preventive treatment was feasible in patients with hemophilia A and leukemia,providing a new strategy for individualized treatment.

关 键 词：血友病A 白血病 药代动力学(PK) 

分 类 号：R554.1[医药卫生—血液循环系统疾病]