中医药治疗肺动脉高压随机对照试验结局指标的选择及评价

Selection and evaluation of outcome measures in randomized controlled trials of traditional Chinese medicine for the treatment of pulmonary hypertension

关键词:  肺动脉高压  中医药  随机对照试验  结局指标

Key words:  pulmonary hypertension  traditional Chinese medicine  randomized controlled trial  outcome measures

摘要:

[目的] 对中医药治疗肺动脉高压（PH）随机对照试验（RCT）选用结局指标进行评价。[方法] 系统检索中国知网（CNKI）、万方数据库（Wanfang Database）、维普数据库（VIP）、中国生物医学文献服务系统（SinoMed）、PubMed、Cochrane Library、Web of Science 7个数据库，检索时限为各数据库建库至2024年12月31日，纳入中医药治疗PH的RCT研究，由2位研究者进行文献筛选和数据收集，对纳入文献的偏倚风险及结局指标进行分析评价。[结果] 最终纳入文献107篇，涉及患者9 454例，提取结局指标总报告频次730次，可分为以下5类：终点事件（3次，0.41%）、症状/体征（临床症状疗效90次、中医症状/证候69次，共占比21.78%）、运动耐量与生活质量（运动耐量50次、生活质量26次，共占比10.41%）、理化检测（440次，60.28%）及安全性指标（52次，7.12%）。在全部文献中，仅1篇明确区分了主/次要结局指标。从具体指标使用频次来看，排名前3的依次为：临床症状有效率、超声心动图估测肺动脉收缩压和中医证候评分。在测量时点方面，多数研究于治疗4周进行评价；中医症状/证候的评价主要依据《中药新药临床研究指导原则（试行）》。[结论] 近年来中医药治疗PH的RCT研究数量逐渐增多，但结局指标的选择与应用尚不规范，有必要构建体现中医药特色与优势的核心指标集，以提升临床研究的质量和规范性。

Abstract:

[Objective] To evaluate the selection and reporting of outcome measures used in randomized controlled trials（RCTs） of traditional Chinese medicine（TCM） for pulmonary hypertension（PH）. [Methods] Seven databases（China National Knowledge Infrastructure，Wanfang Data，VIP Database，SinoMed，PubMed，Cochrane Library，and Web of Science） were systematically searched from inception to December 31，2024. RCTs investigating TCM interventions for PH were included. Two reviewers independently screened studies and extracted data. The risk of bias and the characteristics of outcome measures were analyzed and evaluated. [Results] A total of 107 RCTs involving 9 454 participants were included. Overall，730 reporting instances of outcome measures were extracted and categorized into five domains：endpoint events（3 times，0.41%），symptoms/signs（clinical symptom efficacy 90 times，TCM symptoms/syndromes 69 times，total 21.78%），exercise capacity and quality of life（exercise capacity 50 times，quality of life 26 times，total 10.41%），laboratory/instrumental assessments（440 times，60.28%），and safety outcomes（52 times，7.12%）. Only one trial explicitly distinguished primary and secondary outcomes. The three most frequently reported outcomes were clinical symptom effective rate，echocardiography-estimated pulmonary artery systolic pressure，and TCM syndrome score. Most trials assessed outcomes at 4 weeks after treatment initiation. The evaluation of TCM symptoms/syndromes was mainly based on the Guiding Principles for Clinical Research of New Chinese Herbal Medicine（Trial Implementation）. [Conclusion] In recent years，the number of RCTs on TCM for PH has increased；however，the selection and application of outcome measures remain heterogeneous and insufficiently standardized. Developing a core outcome set（COS） that reflects the characteristics and advantages of TCM is warranted to improve the quality and standardization of future clinical research.