基于实效性随机对照试验的慢性病热敏灸社区干预模式效果评价关键技术研究

Heat-sensitive moxibustion is expected to be a community intervention measures of traditional Chinese medicine that could benefit the whole population owing to its advantages in manipulation, convenience, efficacy, and cost. However, because many methodological issues in pragmatic randomized controlled trials (pRCTs) remain unresolved, the heat-sensitive moxibustion-based community intervention model for chronic disease lacks support of the best real-world evidence to date. To address the key methodological issues, we will perform key technique studies on pRCTs of heat-sensitive moxibustion-based community intervention model for chronic diseases. First, we will design a two-stage partial randomization method with consideration of patient preference and develop a matched patient preference scale and random/non-random grouping criteria to reduce performance and measurement bias induced by patient preference effects. Second, we will customize a community heat-sensitive moxibustion sensation scale for summarizing the treatment effects and develop a multilevel statistical model for confounder adjustment to reduce confounding bias caused by the complex and dynamic intervention. Third, we will select outcomes suitable for self-reporting and design a standardized follow-up program to enhance quality and compliance of patients/family members self-reporting. We will further conduct a confirmatory pRCT that investigates the effectiveness of heat-sensitive moxibustion in community patients with hypertension to validate and optimize the rationality and feasibility of the key techniques and establish a high-quality research paradigm. This project will ultimately improve the quantity and quality of pRCT evidence and promote the development and popularization of the heat-sensitive moxibustion-based community intervention model for chronic diseases.

热敏灸因简、便、效、廉的优点，有潜力成为惠及全民的慢性病社区中医干预技术。但因实效性随机对照试验（pRCT）方法学不完善，慢性病热敏灸社区干预模式效果至今缺乏最佳真实世界证据支持。为此，本项目将针对慢性病热敏灸社区干预pRCT的方法学问题开展关键技术研究。第一，设计考虑患者偏好的两阶段部分随机分组法，并研制配套的患者偏好量表和随机/非随机分组界定标准，以降低患者偏好效应所致实施和测量偏倚；第二，定制用于归纳疗效的社区热敏灸感量表，建立多水平混杂因素调整模型，以降低复杂动态干预所致混杂偏倚；第三，筛选适合自我报告的结局指标，设计规范化随访方案，以提高患者/家属自我报告的随访质量和依从性；最后将以热敏灸治疗社区高血压患者为例开展pRCT实证研究，验证和优化关键技术的合理性和适用性，形成高质量研究范式。研究成果将最终转化为pRCT证据数量和质量整体提升，推动慢性病热敏灸社区干预模式的发展和普及。

实效性随机对照试验（pRCT）因兼具真实世界环境和主动随机干预的特点，适合成为热敏灸社区慢性病管理效果评价的最佳真实世界证据。然而，在社区环境下开展热敏灸pRCT，对研究设计和管理提出了更大挑战。本项目综合应用文献评价、专家咨询、患者访谈、量表研制和统计建模等方法，建立了考虑患者偏好的两阶段部分随机化技术指南和配套的偏好量表，研制了社区患者热敏灸感自评量表，开发了复杂动态干预的混杂因素调整模型，建立了患者/家属自我报告结局指标集和基于自我报告的实施与随访技术指南。通过开展实证研究，验证了关键技术的可行性、优化了关键技术细节，也证实了为期6月的热敏灸社区干预治疗高血压可有效降低收缩压和提高降压有效率，患者安全性和依从性良好且成本低。本项目的研究成果为热敏灸社区pRCT的随机分组、实施与随访、统计分析中的关键方法学难点提供了有效的解决方案，可促进提升热敏灸社区pRCT研究设计的科学性、研究实施的严谨性和研究结果的可靠性。