基因重组人凝血因子Ⅷ在中国人血友病A患者中使用的研究  

Recombinant Factor Ⅷ for Treatment of Patients with Hemophilia A in China

作　　者：刘欣[1] 吴竞生 孙萍[1] 朱薇波[1] 蔡晓燕[1] 郑昌成[1] 王兴兵[1] 杨会志[1] 韩永胜[1] 

机构地区：[1]安徽医科大学附属省立医院血液科,合肥230001

出　　处：《血栓与止血学》2007年第2期57-59,共3页Chinese Journal of Thrombosis and Hemostasis

摘　　要：目的探讨第二代rFⅧ(Kogenate FS)在中国人血友病A患者中使用的安全性和有效性。方法采用第二代rFⅧ(Kogenate FS)治疗14例血友病A志愿者患者,用药前进行FⅧ抗体、FⅧ:C活性、血清病毒学(HBV、HCV和HIV)、血常规、尿常规、大便常规以及肝肾功能检查,于第一次用药后10 min和60 min进行FⅧ:C测定,治疗结束后进行FⅧ抗体和肝肾功能检测。结果①采用Kogenate FS治疗的14例血友病A志愿者患者中,Kogenate FS用量12．99～25．00 u／kg(平均18．74 u／kg),总用量3 000～11 000 u(平均6 570 u),自治疗至出血停止或出血症状改善所需的时间为1．5～5．5 d(平均3．29 d),除1例FⅧ抗体滴度较高者外,其余13例患者第一次用药后10 min和60 min FⅧ: C活性均明显升高,用药前后FⅧ:C活性比较差异具有显著性(P<0．01)。显效13例(占92．86％),好转1例(占7．14％),总有效率为100％。②14例血友病A志愿者患者中,用药前FⅧ抗体阳性2例,其中1例FⅧ抗体>5 Bu,第一次用药后10 min和60 min FⅧ:C活性无明显升高,治疗结束后FⅧ抗体仍然>5 Bu,但出血症状改善;另1例FⅧ抗体为4 Bu,第一次用药后10 min和60 min FⅧ:C活性显著升高,治疗结束后FⅧ抗体转为阴性。③14例血友病A志愿者患者在应用Kogenate FS治疗期间和治疗结束后均表现出良好的耐受性,无不良事件或药物不良反应发生。结论第二代rFⅧ(Kogenate FS)治疗中国人血友病A患者耐受性好,安全有效。Objectives To explore the safety and efficacy of recombinant factor Ⅷ （ Kogenate FS） for treatment of patients with hemophilia A in China. Methods 14 patients with hemophilia A were treated with the secondgeneration recombinant factor Ⅷ product （ Kogenate FS）. The factor Ⅷ antibodies, factor Ⅷ activities,HBV,HCV, HIV,blood routine,urine routine,stool routine,liver function and renal function were detected before the treatment. The detection of factor Ⅷ activities were performed at 10 min and 60 min after the first dose. The factor Ⅷ antibodies, liver functions and renal functions were detected after the treatment. Resuits The doses of Kogenate FS which treated for 14 patients with hemophilia A were 12.99 ～ 25.00 u/kg （mean 18.74 u/kg） and the total doses were 3 000 ～ 11 000 u（mean 6 570 u）. The time from the initial treatment to bleeding stoping or improving was 1.5 ～5.5 d（mean 3.29 d）. It was observed that the factor Ⅷ activities at 10 min and 60 rain after the first dose significantly increased（ P 〈0.01 ）. Among 14 patients, 13 showed the effect ,one improved,overall response rate was 100%. There were two patients detected positive for factor Ⅷ antibodies. One＇ s factor Ⅷ antibodie was beyond 5 Bu ,who＇ s factor Ⅷ activitie at 10 rain and 60 rain after the first dose had no significantly increased but the bleeding symptom was improved. The other＇ s factor Ⅷ antibodie was 4BU ,who＇ s factor Ⅷ activitie at 10 min and 60 min after the first dose had significantly increased and the factor Ⅷ antibodie became negative after the treatment. There was no sideeffect in all patients. Conclusion The second-generation recombinant factor Ⅷ product（ Kogenate FS）was safe, efficacious and well-tolerated for treatment of patients with hemophilia A in China.

关 键 词：重组人凝血因子Ⅷ 血友病A FⅧ抑制物 

分 类 号：R554.1[医药卫生—血液循环系统疾病]