Department of Critical Care Medicine, Shanghai General Hospital of Nanjing Medical University, 650# New Songjiang Road, Songjiang, Shanghai, 201620, China.
Clinical Research Institute, Shanghai Jiaotong University School of Medicine, 227# South Chongqing Road, Xuhui, Shanghai, 200025, China.
BMC Infect Dis. 2019 Sep 6;19(1):780. doi: 10.1186/s12879-019-4388-2.
Sepsis is still a common critical disease with high morbidity and mortality in intensive care unit. Despite published guidelines for sepsis, development of antibiotic therapy and advanced organ support technologies, the mortality of sepsis patients is still 25% or more. It is necessary to distinguish the subtypes of sepsis, and the targeted therapy for the patients need to be explored. Platelets have various biological functions in hemostasis and thrombosis, host defense, inflammatory/immune responses and tissue repair/regeneration. Moreover, severe thrombocytopenia or sustained thrombocytopenia was closely associated with multiply organ dysfunction and higher mortality in sepsis patients. The clinical therapies for thrombocytopenia are platelet transfusion and platelet-elevating drugs. However, platelet transfusion has many defects in clinical practice in sepsis patients, and the impact of platelet-elevating drugs for sepsis patients is still unclear. RESCUE trial is aim to explore the effect of a platelet-elevating drug, recombinant human thrombopoietin (rhTPO), as an effective rescue therapy on sepsis patients with acute severe thrombocytopenia.
It is a randomized, open-label, multi-center, controlled trial in 5 tertiary academic hospitals including medical, surgical or general ICUs. In this study, a total of 200 sepsis patients with severe thrombocytopenia will be randomly assigned in a 1:1 ratio to the control and rhTPO group. The patients will be followed up to 28 days after randomization. All patients in two groups receive the same treatment based on the guideline of Surviving Sepsis Campaign. Primary outcome is 28-day mortality. Secondary outcomes are the changes of PCs, blood transfusion, biomarkers of infection and organ function, days free from advanced organ support, drug-related adverse events, the length of ICU and hospital stay.
RESCUE trial is the first randomized controlled trial to explore the impact of rhTPO for severe thrombocytopenia in sepsis patients diagnosed by sepsis-3.0 standard. Furthermore, RESCUE trial results will be of significant clinical value on the targeted therapy and add clinical evidence that rhTPO is an effective rescue therapy for these sepsis patients.
ClinicalTrials.gov : NCT02707497. Registered Date: March 3rd, 2016. Protocol Version 3. Protocol Date: January 25th, 2019.
败血症仍然是重症监护病房中一种常见的高发病率和高死亡率的危急病症。尽管有关于败血症的已发表指南、抗生素治疗和先进的器官支持技术的发展，但败血症患者的死亡率仍在 25%或以上。有必要区分败血症的亚型，并探索针对患者的靶向治疗。血小板在止血和血栓形成、宿主防御、炎症/免疫反应和组织修复/再生中具有多种生物学功能。此外，严重血小板减少或持续血小板减少与败血症患者的多器官功能障碍和更高的死亡率密切相关。血小板减少症的临床治疗方法是血小板输注和血小板升高药物。然而，在败血症患者的临床实践中，血小板输注存在许多缺陷，血小板升高药物对败血症患者的影响仍不清楚。RESCUE 试验旨在探索血小板升高药物重组人血小板生成素（rhTPO）作为急性严重血小板减少症败血症患者有效抢救治疗的效果。
这是一项在包括内科、外科或普通重症监护病房在内的 5 家三级学术医院进行的随机、开放标签、多中心、对照试验。在这项研究中，将 200 名严重血小板减少症的败血症患者按照 1:1 的比例随机分配到对照组和 rhTPO 组。患者将在随机分组后 28 天进行随访。两组患者均根据《拯救脓毒症运动》指南接受相同的治疗。主要结局是 28 天死亡率。次要结局是血小板计数（PCs）的变化、输血、感染和器官功能的生物标志物、无高级器官支持的天数、药物相关不良事件、重症监护病房和住院时间。
RESCUE 试验是第一项探索 rhTPO 对根据败血症-3.0 标准诊断的败血症患者严重血小板减少症影响的随机对照试验。此外，RESCUE 试验结果将对靶向治疗具有重要的临床价值，并为 rhTPO 是这些败血症患者有效抢救治疗的方法提供临床证据。
ClinicalTrials.gov：NCT02707497。注册日期：2016 年 3 月 3 日。方案版本 3. 方案日期：2019 年 1 月 25 日。
