Price Thomas H, Boeckh Michael, Harrison Ryan W, McCullough Jeffrey, Ness Paul M, Strauss Ronald G, Nichols W Garrett, Hamza Taye H, Cushing Melissa M, King Karen E, Young Jo-Anne H, Williams Eliot, McFarland Janice, Holter Chakrabarty Jennifer, Sloan Steven R, Friedman David, Parekh Samir, Sachais Bruce S, Kiss Joseph E, Assmann Susan F
Department of Medicine, University of Washington, Seattle, WA; Bloodworks Northwest, Seattle, WA;
Department of Medicine, University of Washington, Seattle, WA; Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, Seattle, WA;
Blood. 2015 Oct 29;126(18):2153-61. doi: 10.1182/blood-2015-05-645986. Epub 2015 Sep 2.
High-dose granulocyte transfusion therapy has been available for 20 years, yet its clinical efficacy has never been conclusively demonstrated. We report here the results of RING (Resolving Infection in Neutropenia with Granulocytes), a multicenter randomized controlled trial designed to address this question. Eligible subjects were those with neutropenia (absolute neutrophil count <500/μL) and proven/probable/presumed infection. Subjects were randomized to receive either (1) standard antimicrobial therapy or (2) standard antimicrobial therapy plus daily granulocyte transfusions from donors stimulated with granulocyte colony-stimulating factor (G-CSF) and dexamethasone. The primary end point was a composite of survival plus microbial response, at 42 days after randomization. Microbial response was determined by a blinded adjudication panel. Fifty-six subjects were randomized to the granulocyte arm and 58 to the control arm. Transfused subjects received a median of 5 transfusions. Mean transfusion dose was 54.9 × 10(9) granulocytes. Overall success rates were 42% and 43% for the granulocyte and control groups, respectively (P > .99), and 49% and 41%, respectively, for subjects who received their assigned treatments (P = .64). Success rates for granulocyte and control arms did not differ within any infection type. In a post hoc analysis, subjects who received an average dose per transfusion of ≥0.6 × 10(9) granulocytes per kilogram tended to have better outcomes than those receiving a lower dose. In conclusion, there was no overall effect of granulocyte transfusion on the primary outcome, but because enrollment was half that planned, power to detect a true beneficial effect was low. RING was registered at www.clinicaltrials.gov as #NCT00627393.
高剂量粒细胞输注疗法已应用20年,但从未有确凿证据证明其临床疗效。我们在此报告RING(粒细胞治疗中性粒细胞减少症感染)研究结果,这是一项旨在解决此问题的多中心随机对照试验。符合条件的受试者为中性粒细胞减少症患者(绝对中性粒细胞计数<500/μL)且已证实/可能/疑似感染。受试者被随机分为两组,一组接受(1)标准抗菌治疗,另一组接受(2)标准抗菌治疗加每日输注来自用粒细胞集落刺激因子(G-CSF)和地塞米松刺激的供体的粒细胞。主要终点是随机分组后42天的生存加微生物反应的综合指标。微生物反应由一个盲法判定小组确定。56名受试者被随机分配到粒细胞组,58名被分配到对照组。接受输注的受试者中位数接受了5次输注。平均输注剂量为54.9×10⁹个粒细胞。粒细胞组和对照组的总体成功率分别为42%和43%(P>.99),接受指定治疗的受试者分别为49%和41%(P=.64)。在任何感染类型中,粒细胞组和对照组的成功率均无差异。在一项事后分析中,每次输注平均剂量≥0.6×10⁹个粒细胞/千克的受试者往往比接受较低剂量的受试者预后更好。总之,粒细胞输注对主要结局没有总体影响,但由于入组人数仅为计划的一半,检测真正有益效果的效能较低。RING在www.clinicaltrials.gov上注册为#NCT00627393。