A Multicenter Pharmacosurveillance Study for the Evaluation of the Efficacy and Safety of Recombinant Factor VIII in the Treatment of Patients with Hemophilia A
A Multicenter Pharmacosurveillance Study for the Evaluation of the Efficacy and Safety of Recombinant Factor VIII in the Treatment of Patients with Hemophilia A
复制标题
重组因子VIII治疗甲型血友病疗效和安全性的多中心药物监测研究
DOI：
10.1055/s-0038-1665410
复制
发表时间：
1997
影响因子：
6.7
通讯作者：
I. Scharrer
中科院分区：
文献类型：
--
作者：
E. Aygören‐Pürsün;I. Scharrer
在这项开放性多中心研究中，在39例既往接受过治疗的血友病A患者（凝血因子VIII基础活性≤15%）中评估了重组凝血因子VIII（rFVIII）的安全性和疗效。给予重组FVIII用于预防和治疗出血事件以及用于外科手术。总共输注了3679次rFVIII。通过对输注反应和rFVIII平均年消耗量的主观评价评估，rFVIII的疗效与血浆来源的FVIII浓缩物相当。FVIII的增量回收率（分别为2.4 ± 0.83%/IU/kg、2.12 ± 0.61%/IU/kg）在预期范围内。本试验中未检测到具有临床意义的FVIII抑制物。16例易感患者中有5例出现细小病毒B19血清转化。然而，在两种情况下，结果是模糊的，并可能在另一种情况下，以其他方式解释。因此，在两名患者中观察到细小病毒B19的可靠血清转化。
Summary In this open multicenter study the safety and efficacy of recombinant factor VIII (rFVIII) was assessed in 39 previously treated patients with hemophilia A (factor VIII basal activity ≤15%). Recombinant FVIII was administered for prophylaxis and treatment of bleeding episodes and for surgical procedures. A total of 3679 infusions of rFVIII were given. Efficacy of rFVIII as assessed by subjective evaluation of response to infusion and mean annual consumption of rFVIII was comparable to that of plasma derived FVIII concentrates. The incremental recovery of FVIII (2.4 ± 0,83%/IU/kg, 2.12 ± 0.61%/IU/kg, resp.) was within the expected range. No clinical significant FVIII inhibitor was detected in this trial. Five of 16 susceptible patients showed a seroconversion for parvovirus B19. However, the results are ambiguous in two cases and might be explained otherwise in one further case. Thus, in two patients a reliable seroconversion for parvovirus B19 was observed.