Webert K E, Arnold D M, Carruthers J, Molnar L, Almonte T, Decker K, Seroski W, Reed J, Chan A K, Pai M, Walker I R
Division of Hematology, Department of Medicine, McMaster University, 1200 Main Street West, Hamilton, Ontario, Canada.
Haemophilia. 2007 Sep;13(5):518-26. doi: 10.1111/j.1365-2516.2007.01490.x.
Recombinant activated factor VII (rFVIIa) is licensed for the treatment of bleeding in individuals with haemophilia and inhibitors. The use of rFVIIa appears to be increasing, and an increase in unlicensed use is suspected. There are currently few data about the specific indications for its use. The aim of this study was to describe the patterns of utilization of rFVIIa. We performed a retrospective cohort study using rFVIIa infusion data collected prospectively and clinical data collected retrospectively. Patients were identified using a tracking system designed to account for use of all coagulation factor concentrates issued in southern Ontario. Between 1 January 2001 and 31 December 2005, 85 patients received rFVIIa. 1164 infusions were given (8246.4 mg). Haemophilia patients with inhibitors accounted for 82.9% of rFVIIa infused and represented 8.2% of patients. The total amount of rFVIIa used increased each year from 2001 to 2004 and then decreased in 2005. The total number of infusions of rFVIIa administered annually increased. Both on-label and off-label use of rFVIIa increased. The number of patients with haemophilia receiving rFVIIa remained small and constant. The number of patients receiving rFVIIa for off-label indications increased markedly. Most rFVIIa infusions were given for licensed indications; however, these infusions represented <10% of patients treated. Overall, the utilization of rFVIIa is increasing, mostly for approved indications; however, the number of patients being prescribed rFVIIa for off-label indications has increased. The tracking system used in this study is a valuable tool to describe ongoing utilization patterns of rFVIIa.
重组活化凝血因子 VII（rFVIIa）已获许可用于治疗患有血友病且产生抑制剂的个体的出血情况。rFVIIa 的使用似乎在增加，并且怀疑其未获许可的使用也在增多。目前关于其具体使用指征的数据很少。本研究的目的是描述 rFVIIa 的使用模式。我们进行了一项回顾性队列研究，使用前瞻性收集的 rFVIIa 输注数据和回顾性收集的临床数据。通过一个追踪系统来识别患者，该系统旨在记录安大略省南部发放的所有凝血因子浓缩剂的使用情况。在 2001 年 1 月 1 日至 2005 年 12 月 31 日期间，85 名患者接受了 rFVIIa 治疗。共进行了 1164 次输注（8246.4 毫克）。患有抑制剂的血友病患者占 rFVIIa 输注量的 82.9%，占患者总数的 8.2%。从 2001 年到 2004 年，rFVIIa 的使用总量逐年增加，然后在 2005 年有所下降。每年给予的 rFVIIa 输注总数有所增加。rFVIIa 的标签内和标签外使用均有所增加。接受 rFVIIa 治疗的血友病患者数量仍然很少且保持稳定。因标签外指征接受 rFVIIa 治疗的患者数量显著增加。大多数 rFVIIa 输注是用于已获许可的指征；然而，这些输注的患者占接受治疗患者的比例不到 10%。总体而言，rFVIIa 的使用正在增加，主要用于获批指征；然而，因标签外指征开具 rFVIIa 处方的患者数量有所增加。本研究中使用的追踪系统是描述 rFVIIa 持续使用模式的宝贵工具。
