1/2 A comparison of individualized vs. weight based protocols to treat vaso-occlusive episodes in Sickle Cell Disease (COMPARE VOE)

1/2 治疗镰状细胞病血管闭塞发作的个体化方案与基于体重的方案的比较（COMPARE VOE）

• 批准号: 10021699
• 负责人: Paula Tanabe
• 金额: $ 52.72万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-07 至 2022-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10021699
• 关键词: Accident and Emergency department; Adult; Affect; African American; American; Analgesics; Blinded; Caring; Chronic Disease; Clinical; Clinical Trials; Complication; Computerized Medical Record; Consensus; Consent; Data; Data Collection; Data Coordinating Center; Diagnosis; Disease; Dose; Emergency Department Physician; Emergency Nursing; Emergency department visit; Enrollment; Ensure; Feedback; Future; Genetic Diseases; Goals; Grant; Guidelines; Healthcare; Hemoglobin; Hemoglobinopathies; Hospitalization; Hour; Knowledge; Leadership; Length of Stay; Level of Evidence; Medicaid; Medical; Monitor; National Heart, Lung, and Blood Institute; Opioid; Outcome Study; Pain; Patients; Phase; Physicians; Population; Process; Protocols documentation; Randomized; Randomized Clinical Trials; Recommendation; Reporting; Resources; Respondent; Safety; Sample Size; Sampling; Services; Shapes; Sickle Cell Anemia; Single-Blind Study; Site; Societies; Statistical Data Interpretation; Surveys; Testing; United States; Variant; Veno-Occlusive Disease; Visit; Visual; Visual Analogue Pain Scale; Weight; analog; base; clinically significant; college; data management; design; evidence base; evidence based guidelines; experience; improved; individual patient; medical specialties; pain score; point of care; primary endpoint; primary outcome; prospective; secondary outcome; side effect; standard of care; success; usability

Abstract Sickle cell disease (SCD) is a genetic disorder and the most common hemoglobin disorder in the United States (U.S.). It affects an estimated 90,000-100,000 Americans and was ranked as the fifth most common principal diagnosis for Medicaid’s super-utilizer hospital stays. Painful vaso-occlusive episodes (VOE), a new onset of pain (7/10 or greater) for at least four hours for which there is no other explanation than vaso-occlusion, are the most common manifestation of SCD experienced by patients and the most common reason for emergency department (ED) visits. There is no standard, evidence based approach to treating painful VOE. In 2014 the National Heart, Lung, and Blood Institute (NHLBI) released recommendations for the “Management of Sickle Cell Disease” where recommendations to treat VOE were based on consensus panel expertise due to lack of evidence. Recommendations included the use of a patient specific protocol (specific agents and doses for an individual patient) or a standard VOE analgesic protocol, where the standard VOE protocol was not defined. We implemented a weight-based analgesic protocol as a “standard” VOE protocol and conducted a pilot randomized clinical trial (RCT) at two EDs with 52 patients comparing the patient-specific and weight-based analgesic protocols; results were encouraging, however, limited due to small numbers of patients and sites. We propose a Phase III randomized and single-blinded (with subject blinded) clinical trial to test the hypothesis that the patient-specific analgesic protocol is clinically superior to the weight-based analgesic protocol for treating SCD patients with an ED visit due to VOE. This five-site RCT will randomize 460 adult SCD patients to one of the two protocols in order to have the target of 230 patients with an ED visit for VOE during the 24 month enrollment period. A sample size of 230 patients will provide 90% power for detecting a 14 mm clinically significant difference between the two protocols in the primary endpoint, pain score reduction from arrival in the ED to discharge by using a 0-100 mm visual analog scale. Secondary outcomes include ED length of stay, hospitalization, seven day return ED visits and hospitalizations and we will monitor side effects and safety. A Clinical Coordinating Center (CCC; Paula Tanabe, PI) and a Data Coordinating Center (DCC; Huiman Barnhart, PI) will manage this trial. This experienced multi-specialty team will deliver the integrated services vital to the trial, including clinical and operational leaderships, site management, statistical analysis and data management. High level evidence generated by this trial will shift the paradigm on treating VOE in the ED and decreasing the pain of SCD patients suffering from this devastating complication.

抽象的 镰状细胞疾病（SCD）是一种遗传疾病，是美国最常见的血红蛋白疾病 （我们。）。它影响了估计的90,000-100,000美国人，并被评为第五位最常见的校长 医疗补助超级利润医院的诊断。痛苦的血管熟悉情节（VOE），新的发作 疼痛（7/10或更高）至少四个小时，除了血管c 2之外没有其他解释，是 患者经历的SCD最常见的表现和紧急情况的最常见原因 部门（ED）访问。没有标准的，基于证据的方法来治疗痛苦的VOE。 2014年 国家心脏，肺和血液研究所（NHLBI）发布了“镰刀管理的建议 细胞疾病”，因为缺乏，请根据共识面板的专业知识来治疗VOE的建议 证据。建议包括使用患者特定方案（特定的药物和剂量 单个患者）或标准VOE镇痛协议，该方案未定义标准VOE协议。 我们将基于权重的镇痛协议作为“标准” VOE协议实施，并进行了试点 在两名EDS的随机临床试验（RCT）和52名患者比较患者特异性和基于体重的患者 镇痛协议；然而，由于少数患者和部位，结果受到鼓舞。 我们提出了一项III期随机和单盲（受试者盲）临床试验，以检验假设 患者特异性镇痛方案在临床上优于基于体重的镇痛方案 因VOE而进行ED访问治疗SCD患者。这个五个站点的RCT将将460名成人SCD患者随机 两种方案之一是为了将230名患者的目标造成24期间的ED访问。 月注册期。 230名患者的样本量将提供90％的功率来检测14 mm临床 主要端点中两个方案之间的显着差异，疼痛评分从到达中降低 通过使用0-100 mm的视觉模拟量表进行排出。次要结果包括ED住院时间， 住院，七天的返程访问和住院治疗，我们将监控副作用和安全性。一个 临床协调中心（CCC； Paula Tanabe，PI）和数据协调中心（DCC； Huiman Barnhart，PI）将管理此试验。这个经验丰富的多专科团队将提供集成服务 对试验至关重要，包括临床和运营领导力，现场管理，统计分析和数据 管理。该试验产生的高水平证据将改变有关在ED中处理VOE的范式和 减轻患有这种毁灭性并发症的SCD患者的疼痛。