National Health Service (NHS) Blood and Transplant, Oxford University Hospitals NHS Trust, John Radcliffe Hospital, and Nuffield Department of Medicine, University of Oxford, Oxford, United Kingdom.
N Engl J Med. 2013 May 9;368(19):1771-80. doi: 10.1056/NEJMoa1212772.
The effectiveness of platelet transfusions to prevent bleeding in patients with hematologic cancers remains unclear. This trial assessed whether a policy of not giving prophylactic platelet transfusions was as effective and safe as a policy of providing prophylaxis.
We conducted this randomized, open-label, noninferiority trial at 14 centers in the United Kingdom and Australia. Patients were randomly assigned to receive, or not to receive, prophylactic platelet transfusions when morning platelet counts were less than 10×10(9) per liter. Eligible patients were persons 16 years of age or older who were receiving chemotherapy or undergoing stem-cell transplantation and who had or were expected to have thrombocytopenia. The primary end point was bleeding of World Health Organization (WHO) grade 2, 3, or 4 up to 30 days after randomization.
A total of 600 patients (301 in the no-prophylaxis group and 299 in the prophylaxis group) underwent randomization between 2006 and 2011. Bleeding of WHO grade 2, 3, or 4 occurred in 151 of 300 patients (50%) in the no-prophylaxis group, as compared with 128 of 298 (43%) in the prophylaxis group (adjusted difference in proportions, 8.4 percentage points; 90% confidence interval, 1.7 to 15.2; P=0.06 for noninferiority). Patients in the no-prophylaxis group had more days with bleeding and a shorter time to the first bleeding episode than did patients in the prophylaxis group. Platelet use was markedly reduced in the no-prophylaxis group. A prespecified subgroup analysis identified similar rates of bleeding in the two study groups among patients undergoing autologous stem-cell transplantation.
The results of our study support the need for the continued use of prophylaxis with platelet transfusion and show the benefit of such prophylaxis for reducing bleeding, as compared with no prophylaxis. A significant number of patients had bleeding despite prophylaxis. (Funded by the National Health Service Blood and Transplant Research and Development Committee and the Australian Red Cross Blood Service; TOPPS Controlled-Trials.com number, ISRCTN08758735.).
血小板输注预防血液系统恶性肿瘤患者出血的效果尚不清楚。本试验评估不给予预防性血小板输注的策略与给予预防性血小板输注的策略相比是否同样有效和安全。
我们在英国和澳大利亚的 14 个中心进行了这项随机、开放标签、非劣效性试验。当清晨血小板计数＜10×10(9)/升时，患者被随机分配接受或不接受预防性血小板输注。合格患者为年龄≥16 岁、正在接受化疗或进行干细胞移植且有或预计有血小板减少症的患者。主要终点是随机分组后 30 天内发生的世界卫生组织（WHO）分级 2、3 或 4 级出血。
共有 600 例患者（无预防性治疗组 301 例，预防性治疗组 299 例）于 2006 年至 2011 年期间进行了随机分组。无预防性治疗组 300 例患者中有 151 例（50%）发生了 WHO 分级 2、3 或 4 级出血，而预防性治疗组 298 例患者中有 128 例（43%）发生了这种出血（调整后比例差异，8.4 个百分点；90%置信区间，1.7 至 15.2；非劣效性检验 P=0.06）。无预防性治疗组患者出血天数更多，且首次出血时间更短。无预防性治疗组血小板用量明显减少。一项预先设定的亚组分析在接受自体干细胞移植的患者中发现两组研究之间出血发生率相似。
我们的研究结果支持继续使用预防性血小板输注，并且表明与不进行预防性治疗相比，预防性治疗可减少出血。尽管进行了预防，但仍有相当数量的患者发生出血。（由英国国民健康服务部血液与移植研究与开发委员会和澳大利亚红十字会血液服务部资助；TOPPS 对照试验。com 编号，ISRCTN08758735。）
