成人重型血友病A患者药代动力学参数测定及个体化预防治疗的疗效

Determination of Pharmacokinetic Parameters and Individualized Prevention Effect in Adult Patients with Severe Hemophilia A Therapeutic

目的:了解成人重型血友病A(SHA)患者的FⅧ药代动力学参数(PK参数),制定个体化预防治疗方案,观察其临床疗效及经济效益。方法:收集2017年7月~2018年6月在某院治疗的35例SHA患者,检测其PK参数,制定个体化预防治疗,随访1年,对比治疗前后患者的年总出血次数(ARB)、年关节出血次数(AJRB)、年FⅧ用量、HJHS评分等变化情况。结果:35例患者半衰期最短者6.59h,最长者15.04h,平均10.55h;在随访1年后,患者ARB和AJRB分别减少89.5%、89.7%;HJHS评分改善者有77%,年FⅧ用量增加62.3%,但其中4例患者FⅧ用量较预防前减少,这4例患者平均半衰期13.89h。结论:成人SHA患者的PK参数波动范围大,个体化预防治疗可明显减少出血次数,改善关节评分,对于半衰期长者,还能减少年FⅧ总用量,达到生存和经济双重获益。

Objective:To understand the FVIII pharmacokinetic parameters(PK parameters)in adult patients with severe hemophilia A(SHA),and develop individual preventive treatment plan and observe its clinical effect and economic benefit.Methods:A total of 35 SHA patients treated in a hospital from July 2017 to June 2018 were collected,their PK parameters were tested,individualized preventive treatments were developed,and followed-up for 1 year,the annual total number of bleeding(ARB),annual joint bleeding times(AJRB),dosage of annual FVIII,HJHS score changes were compared before and after treatments in patients.Results:The 35 patients had the shortest half-life of 6.59 h and the oldest of 15.04 h,with an average of 10.55 h.After 1 year of following-up,ARB and AJRB decreased by 89.5%and 89.7%,respectively.77%of HJHS scores improved,the annual FVIII dosage increased by 62.3%,but the dosage of FVIII in four patients fell,with the average half-life of 13.89 h in 4 patients.Conclusion:PK parameters in adult SHA patients fluctuate widely,and individualized preventive treatment can significantly reduce the number of bleeding,improve joint score,for the long half-life patients,it can reduce the annual FVIII dosage to achieve survival and economic benefit.