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Real World Research: Bridging the Gap Between Clinical Practice Guidelines and Clinical Decision Making
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摘要: 临床实践指南是制订临床决策的重要依据，但仍存在诸多因素导致医生无法遵循指南，其中一个重要因素为指南的外部有效性受限，最终使得指南无法为临床决策的制订提供足够依据，此为指南与临床决策之间的距离。真实世界研究可增强指南的外部有效性，为临床决策的制订提供依据，为指南提供本土化证据、成本效益及其他方面相关证据，同时可监测指南的实施，弥补传统指南的证据不足，有助于弥合指南与临床决策之间的距离。在指南制订过程中，可考虑纳入高质量的真实世界研究和真实世界证据，以促进指南优化，助力指南质量提升。Abstract: Clinical practice guidelines are an important basis for clinical decision-making, but there are still many factors that prevent physicians from following the guidelines. One of the problems, the limited external validity of the guidelines, ultimately prevents the guidelines from providing an adequate basis for clinical decision-making, thus forming the gap between the guidelines and clinical decisions. Real-world research can enhance the external validity of the guidelines, provide a basis for clinical decision-making, and offer localized evidence for the guidelines, evidence of cost-effectiveness and other evidence for the guidelines. It can also monitor the implementation of the guidelines and help to make up for the lack of evidence in the formulation of traditional guidelines. Therefore, high-quality real-world studies and real-world evidence may be considered in the guideline development process to facilitate guideline optimization and serve as an aid to guideline quality improvement.
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Key words:
- real-world study /
- real-world evidence /
- clinical practice guidelines /
- external validity
作者贡献：施秀青负责起草及撰写论文；阎思宇负责确定论文大纲并修订论文；黄桥负责提供修改意见并制作图片；李绪辉、王永博负责提供修改意见；马文昊负责提供写作素材；商洪才负责提出观点并统筹论文；靳英辉负责提出观点并统筹、修订论文。利益冲突：所有作者均声明不存在利益冲突 -
表 1 RWS弥补传统RCT不足、提供临床决策支持的实例
类别 传统RCT不足之处 RWS提供临床决策支持，显示良好的外部有效性 性别 哌柏西利的RCT未纳入男性乳腺癌患者 辉瑞公司利用上市后报告和电子健康记录的额外支持性数据，使得美国FDA批准哌柏西利用于联合芳香酶抑制剂或氟维司群治疗男性激素受体阳性、人表皮生长因子受体2阴性晚期或转移性乳腺癌患者[31] 种族 多发性硬化的RCT普遍缺少黑色人种/非裔美国人的参与，因此缺乏此类人群的最佳治疗证据[32] 基于ESTEEM的RWS研究发现，富马酸二甲酯治疗多发性硬化的疗效在黑色人种/非裔美国人与非黑色人种/非非裔美国人一致[33] 年龄 苯达莫司汀单药或联合利妥昔单抗治疗慢性淋巴细胞白血病老年患者的疗效和耐受性尚不明确 德国的一项大型RWS显示，苯达莫司汀单药或联合利妥昔单抗对慢性淋巴细胞白血病老年患者具有良好的疗效和耐受性[34] 既往史/个人史/依从性 RCT研究NovelSTART[35]旨在探讨短效β2受体激动剂作为哮喘的唯一治疗疗效，其排除标准包括：进入研究前12个月因哮喘入院的患者、每年吸烟20包以上者、40岁以后出现呼吸道症状的现吸烟者或有≥10包烟史的前吸烟者；不愿或不能从目前的哮喘治疗方案中转换的患者等 Salford肺部研究[11]纳入大量真实世界环境下的哮喘或COPD患者，不通过既往史、个人史、合并症及依从性排除患者。该研究结果与RCT一致，每日1次应用吸入性皮质类固醇糠酸氟替卡松与长效β2受体激动剂维兰特罗相结合的干粉吸入剂常规治疗相比，在减少COPD病情恶化方面更具优势[36] 个人史、合并症 RCT研究Wisdom[37]的入组人群为COPD的患者，其排除标准包括：过去几年有严重酗酒或药物滥用史；患有重大疾病(如恶性肿瘤等)；有肺切除的开胸手术史；有哮喘史且需口服或吸入皮质类固醇治疗等10余项内容 BRONCHIOLE trial(Clinical Trials.gov, No.NCT03566667)旨在研究β受体阻断剂治疗对于无明显合并心血管疾病的COPD患者益处的实效性，其排除标准仅包括：现有的心血管疾病、基线时使用β受体阻滞剂治疗、合并β受体阻滞剂的禁忌证，如未经治疗的房室传导阻滞或严重哮喘 RWS：真实世界研究；RCT：随机对照试验；FDA：食品药品监督管理局；COPD：慢性阻塞性肺疾病 
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