DETERMINATION OF OPTIMAL PROPHYLACTIC PLATELET DOSE STRATEGY TO PRVNT BLEEDING

确定预防 VNT 出血的最佳预防性血小板剂量策略

• 批准号: 7378121
• 负责人: JAMES W GEORGE
• 金额: $ 2.59万
• 依托单位: UNIVERSITY OF OKLAHOMA HLTH SCIENCES CTR
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-03-01 至 2007-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7378121
• 关键词: DETERMINATION; OPTIMAL; PROPHYLACTIC; PLATELET; DOSE

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. PROTOCOL SYNOPSIS As platelet use continues to increase at a rate disproportionately higher than that of red cells, it is important to identify the most cost-effective strategies for providing platelet support. Identification and implementation of the most safe and cost effective strategies for providing platelet support is crucial for effective disease management without depleting platelet supplies. The two most important factors within the control of the ordering physician that will significantly influence the total amount of platelets transfused are: 1) the prophylactic platelet transfusion trigger selected for transfusion; and 2) the number of platelets given per transfusion. Informative clinical data have been provided in the last fifteen years concerning the platelet transfusion trigger. The optimal quantity of platelets to be used per transfusion remains a highly controversial subject. To date, no prospective platelet transfusion trials have been performed in which patients are randomized to an assigned platelet dose throughout their period of thrombocytopenia to evaluate the effects of different doses on transfusion outcomes. There may be safety issues associated with different dosing strategies for platelet therapy. Maintaining a higher platelet count for a greater percentage of the time with higher dose platelet transfusion therapy might provide better hemostasis than lower dose therapy. On the other hand, trigger study data suggest that there may be no hemostatistically-related safety issues based on the dose of platelets transfused as long as a baseline level of > 5,000 platelets is maintained. This study will investigate the safety of three different dosing strategies for inpatients with thrombocytopenia related to stem cell transplants or chemotherapy. The primary endpoint is the percentage of patients in each treatment arm who have at least one day with Grade 2 or higher bleeding. The most important secondary endpoints, capturing key data on costs and safety, are the total number of platelets dispensed, the total number of transfusion events, the highest grade of bleeding, and the bleeding severity score (if such a score has been validated and published by the end of the Platelet Dose trial, and the necessary information to calculate the score was collected). The results of this trial could have a major effect on standard medical practice. The Platelet Dose Trial is a multi-site trial that will enroll 1350 patients in the United States. Patients will be randomized with equal allocation to three platelet transfusion therapy groups based on body surface area (BSA): Lower dose: 1.1 x 1011/m2 (¿¿¿¿ of the medium dose) Medium dose: 2.2 x 1011/m2 Higher dose: 4.4 x 1011/m2 (twice the medium dose) An acceptable dose will be a dose that is within a range of 25% either above or below the target dose. Patients will be prophylactically transfused at their assigned dose for morning platelet counts of <10,000/ul. If the post-transfusion platelet count is not <10,000/ul the physician will be allowed to order another platelet transfusion but is not required to do so. The protocol allows for additional platelets in the case of invasive procedures or active bleeding. Assessment of bleeding by study personnel will be performed daily by means of physical assessment of the patient, patient interview and review of patient chart and laboratory data. The actual assignment of the bleeding grades will occur at the Data Coordinating Center by a computerized algorithm programmed to evaluate the data from the case report forms, plus adjudication of death due to bleeding. Transfusion Medicine/Hemostasis Network New England Research Institutes, Inc.

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。随着血小板使用率持续以不成比例高于红细胞的速度增长，确定提供血小板支持的最具成本效益的策略是很重要的。确定和实施提供血小板支持的最安全和最具成本效益的战略，对于在不耗尽血小板供应的情况下有效地管理疾病至关重要。在订购医生的控制范围内，将显著影响血小板总量的两个最重要的因素是：1)选择用于输血的预防性血小板输注触发器；以及2)每次输注的血小板数量。在过去的15年里，关于血小板输注触发器的临床数据已经被提供。每次输血使用的最佳血小板数量仍然是一个极具争议的话题。到目前为止，还没有进行前瞻性的血小板输注试验，在这些试验中，患者在整个血小板减少期被随机分配到指定的血小板剂量，以评估不同剂量对输注结果的影响。可能存在与不同的血小板治疗剂量策略相关的安全问题。大剂量的血小板输注治疗在较长时间内维持较高的血小板计数可能比小剂量的治疗提供更好的止血效果。另一方面，Trigger研究数据表明，只要维持5,000个血小板的基线水平，就可能不存在基于输注血小板剂量的血液统计学相关的安全问题。这项研究将调查三种不同给药策略对因干细胞移植或化疗而导致的血小板减少症住院患者的安全性。主要终点是每个治疗组中至少有一天出血2级或更高的患者的百分比。获取关于成本和安全性的关键数据的最重要的次级终点是分配的血小板总数、输血事件总数、出血的最高等级和出血严重程度评分(如果在血小板剂量试验结束时验证并公布了这样的评分，并且收集了计算评分所需的信息)。这项试验的结果可能会对标准的医疗实践产生重大影响。血小板剂量试验是一项多点试验，将在美国招募1,350名患者。患者将根据身体表面积(BSA)被随机分配到三个血小板输注治疗组：较低剂量：1.1×1011/m2(中等剂量的)中剂量：2.2×1011/m2较高剂量：4.4×1011/m2(中剂量的两倍)可接受的剂量将是高于或低于目标剂量25%的范围内。患者将按照分配的剂量预防性输血，使其早晨的血小板计数达到10,000个/微升。如果输血后的血小板计数低于10,000个/微升，医生将被允许再次输注血小板，但不是必须这样做。该方案允许在侵入性手术或活动性出血的情况下增加血小板。研究人员每天将通过对患者的身体评估、患者访谈以及审查患者病历和实验室数据来评估出血情况。出血等级的实际分配将在数据协调中心通过编程的计算机化算法进行，以评估病例报告表中的数据，以及因出血而死亡的裁决。输血医学/止血网络新英格兰研究院，Inc.