Rajpurkar M, Chitlur M, Recht M, Cooper D L
Division of Hematology/Oncology, Carman and Ann Adams Department of Pediatrics, Children's Hospital of Michigan, Detroit, MI, USA; Wayne State University, Detroit, MI, USA.
Haemophilia. 2014 Jul;20(4):464-71. doi: 10.1111/hae.12473.
Glanzmann's thrombasthenia (GT) is a rare bleeding disorder characterized by a quantitative or qualitative defect of glycoprotein IIb/IIIa on the platelet membrane. Managing bleeding episodes is often difficult, and a variety of modalities have been used, including platelet transfusions, recombinant factor VIIa (rFVIIa), and other supportive care. The aim of this review was to present the clinical experience with rFVIIa bolus infusion (rFVIIa BI) for treatment of bleeding episodes and prevention of bleeding during surgical procedures in patients with GT. A literature search was performed to identify rFVIIa-treated patients with GT. Overall, one international survey, one open-label study, and 40 case reports identified 172 bleeding episodes treated with rFVIIa and 62 procedures covered with rFVIIa. In the international survey, rFVIIa BI was used for 96 bleeding episodes in 59 patients. Recombinant FVIIa was effective in 76 bleeding episodes (79%). Of 34 surgical procedures, 25 procedures received rFVIIa BI with 92% bleeding-prevention efficacy. The open-label study reported 28 patients with 28 rFVIIa BI-treated bleeds, and 26 (93%) bleeding episodes responded to rFVIIa. Published case reports revealed that 25 (69%) of 36 bleeds and 27 (96%) of 28 surgeries responded to rFVIIa BI treatment. Overall, 26 adverse events were reported in 19 patients, including five thromboembolic events in two patients where a possible relationship with rFVIIa could not be excluded. Two large studies and 40 case reports provide a literature base to support the efficacy and safety of rFVIIa BI in patients with GT.
Glanzmann血小板无力症（GT）是一种罕见的出血性疾病，其特征是血小板膜上糖蛋白IIb/IIIa存在数量或质量缺陷。处理出血发作通常很困难，已采用了多种治疗方式，包括血小板输注、重组凝血因子VIIa（rFVIIa）及其他支持性治疗。本综述的目的是介绍rFVIIa大剂量输注（rFVIIa BI）治疗GT患者出血发作及预防手术过程中出血的临床经验。进行文献检索以确定接受rFVIIa治疗的GT患者。总体而言，一项国际调查、一项开放标签研究和40例病例报告共确定了172次接受rFVIIa治疗的出血发作以及62例采用rFVIIa覆盖的手术。在国际调查中，rFVIIa BI用于59例患者的96次出血发作。重组凝血因子VIIa在76次出血发作中有效（79%）。在34例手术中，25例接受了rFVIIa BI，预防出血的有效率为92%。开放标签研究报告了28例接受28次rFVIIa BI治疗出血的患者，26次（93%）出血发作对rFVIIa有反应。已发表的病例报告显示，36次出血中的25次（69%）以及28例手术中的27次（96%）对rFVIIa BI治疗有反应。总体而言，19例患者报告了26起不良事件，包括2例患者发生5次血栓栓塞事件，无法排除其与rFVIIa存在可能的关联。两项大型研究和40例病例报告为支持rFVIIa BI在GT患者中的疗效和安全性提供了文献依据。
