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摘要: 近年来，作为随机对照试验（RCT）的有力补充手段，真实世界研究（RWS）得到越来越多的关注。但是在慢性伤口领域，大多数患者的病情复杂，RCT的严格纳入和排除标准缺乏实际应用价值。RWS在临床医疗实践、药物与医疗器械监管和卫生技术评估等方面提供了更接近真实医疗环境的数据，但RWS也存在一些待解决的问题，如诊断标准未统一、研究终点不清等。此外，我国慢性伤口领域的RWS似乎还未真正开始，各级机构需要共同努力以推进RWS的发展。Abstract: In recent years, as a powerful supplement to the randomized controlled trial (RCT), real world study (RWS) has received more and more attention. However, in the field of chronic wounds, most of the patients have complex condition, and the strict inclusion and exclusion criteria of RCT were lack of practical application values. The RWS provides data closer to the real medical environment in clinical medical practice, drug and medical device supervision, and health technology evaluation. However, RWS has some problems that need to be resolved, such as inconsistent diagnostic criteria and unclear research endpoints. In addition, RWS in chronic wound research in China seems to have not really started yet, and institutions at all levels need to work together to promote the development of RWS.
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Key words:
- Wound healing /
- Chronic wounds /
- Real world study /
- Real world data /
- Real world evidence
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参考文献
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表1 真实世界研究的主要类型
研究类型 特点 优点 不足 应用场景举例 前瞻性队列研究 根据研究对象暴露状态决定分组，前瞻性观察一定时间后得到感兴趣结果 单个研究可得到多个结果，清楚表明暴露于结果的先后顺序，促进罕见暴露研究，确定发病率的最佳方法 成本高昂而耗时，不适合罕见疾病及长潜伏期疾病的研究 收集全国吸烟与不吸烟的糖尿病患者，跟踪数年后，确定他们是否以及何时发生糖尿病足 回顾性队列研究 根据过去某一段时间研究对象的暴露状况对其进行分组，根据已有的数据追溯感兴趣结果 相较于前瞻性队列研究更加省时省力 存在选择偏倚，数据缺失 观察伤口中心在过去10年间使用超声清创术与外科器械清创处理下肢慢性伤口后，伤口愈合率及医疗花费的差异 病例对照研究 根据感兴趣的结果选择研究对象，回顾每个研究对象的情况 成本低而高效，适用于罕见疾病及长潜伏期疾病研究，生成假设 存在选择偏倚、回忆偏倚，难以做出因果关系推断 收集下肢静脉溃疡患者住院病历，分为截肢组与非截肢组，分析确定截肢相关危险因素 横断面研究 仅在某一个时间点或很短时间进行的研究，不考虑感兴趣的结果 快速且便宜，确定患病率的最佳方法，可研究多个结局 不能有效地研究罕见病症，不能区分因果关系 调查2019年全国慢性伤口的患病率 病例报告 报告单个患者接受某种诊疗措施产生的某种结果 识别新疾病或新症状，检测新药的不良反应或潜在用途，产生假设 不能做因果推断，不具有可推广性，严重的发表偏倚 手部烧伤瘢痕引起恶性黑色素瘤的病例报告 病例系列研究 一段时间内对多个病例报告的集合描述分析 优点同病例报告 不足同病例报告 回顾性分析A型肉毒毒素治疗3例慢性伤口病例，评估20周伤口愈合率及伤口愈合时间 实用性RCT 在日常卫生保健环境中纳入研究对象，随机分组后测量干预效果 可同时比较多种干预措施的优点和不足，允许研究对象的异质性，增加了实验结果的外推性，相对较低的研究成本；可用于罕见疾病的研究 不能评价某项干预措施对各组患者的效果 纳入真实世界伤口门诊的糖尿病溃疡患者，不排除重症或合并症患者，使用新型敷料或常规换药处理，12周后确定伤口愈合率差异 基于注册登记研究的RCT 根据注册登记处的患者信息，纳入研究对象，随机分组后测量干预效果 优点同实用性RCT 不足同实用性RCT 借助美国慢性伤口登记处，筛选慢性下肢溃疡患者，利用在线工具将其随机分为负压伤口疗法组和标准治疗组，分析一段时间后2组患者治疗满意度及伤口愈合率的差别 非随机同期对照试验 医师根据病情或有关因素人为纳入试验组或对照组，并进行同期对照试验 可行性好，患者依从性佳，不存在伦理道德问题 实验组与对照组一定程度上缺乏可比性，难以应用盲法，存在选择偏倚与测量偏倚 根据重症下肢溃疡患者病情和意愿选择膝上截肢或膝下截肢，随访5年内病死率差异 注：RCT为随机对照试验 
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