White G, Shapiro A, Ragni M, Garzone P, Goodfellow J, Tubridy K, Courter S
Genetics Institute, Inc, Cambridge, MA 02140, USA.
Semin Hematol. 1998 Apr;35(2 Suppl 2):33-8.
The pharmacokinetics, safety, and efficacy of recombinant factor IX (rFIX) have been evaluated in previously treated and untreated patients with hemophilia B. In a study of 56 previously treated patients, there was a total of 1,070 hemorrhages, which required 1,514 infusions of rFIX. Of the 1,070 episodes, 80% resolved after a single rFIX infusion. The majority of the remaining episodes requiring more than 1 infusion were either major or complicated hemorrhages. Eighty-seven percent of the 1,514 infusions were rated by the physicians or patients as providing an excellent or good response. These results are consistent with the efficacy data from other hemophilia product clinical trials. rFIX has also been administered as bolus doses or by continuous infusion in conjunction with 13 surgical procedures. Estimated blood loss during and after surgery was as expected when compared with blood loss for similar procedures in patients without hemophilia, and 97% of the clinical responses during and after surgery were rated by physicians or patients as excellent or good. Pharmacokinetic comparison of rFIX with one plasma-derived product showed a statistically significant lower recovery (28% less) of rFIX. The half-lives between the two products were comparable. Common adverse events associated with rFIX treatment have been comparable to other factor IX products. As expected, there has been no evidence of viral transmission by rFIX.
重组凝血因子IX（rFIX）的药代动力学、安全性和疗效已在既往接受治疗和未接受治疗的B型血友病患者中进行了评估。在一项针对56例既往接受治疗患者的研究中，共发生1070次出血事件，需要输注1514次rFIX。在这1070次事件中，80%在单次输注rFIX后得到缓解。其余需要多次输注的事件大多为严重或复杂出血。在1514次输注中，87%被医生或患者评为反应极佳或良好。这些结果与其他血友病产品临床试验的疗效数据一致。rFIX还曾在13例外科手术中作为大剂量推注或持续输注给药。与无血友病患者进行类似手术时的失血量相比，手术期间及术后的估计失血量在预期范围内，并且手术期间及术后97%的临床反应被医生或患者评为极佳或良好。rFIX与一种血浆源性产品的药代动力学比较显示，rFIX的回收率在统计学上显著较低（低28%）。两种产品的半衰期相当。与rFIX治疗相关的常见不良事件与其他凝血因子IX产品相当。正如预期的那样，没有证据表明rFIX会传播病毒。
