Abstract: Background Mycoplasma pneumoniae pneumonia is a common type of community-acquired pneumonia in children. Among these patients, 2.3% to 6.8% continue to show clinical deterioration despite treatment with macrolide antibiotics for 7 days or longer, progressing to refractory pneumonia. In recent years, given the increasing number of pediatric cases, randomized controlled clinical trials on the treatment of this condition using traditional Chinese medicine have made some progress; however, the corresponding set of outcome measures still needs to be refined. Objective To provide references for the improvement of treatment indicators for this disease and the design of clinical trials in TCM，this study systematically analyzing the current status of outcome indicators in randomized controlled trials (RCTs) of traditional Chinese medicine (TCM) for treating refractory Mycoplasma pneumoniae pneumonia in children. Methods This study systematically searched five Chinese and English databases for published research on the treatment of refractory Mycoplasma pneumoniae pneumonia in children using traditional Chinese medicine over the past 10 years. Literature was screened, data were extracted, and the risk of bias was assessed according to established criteria, and the outcome data were processed and analyzed. Results The results included a total of 38 relevant studies, covering 50 outcome measures, with a total of 282 observations. The top 5 outcome indicators were clinical efficacy (32 times, 84.21%), incidence of adverse reactions (25 times, 65.79%), time to defervescence (24 times, 63.16%), time to disappearance of cough (23 times, 60.53%), and interleukin (19 times, 50.00%). 30 studies reported Western medical diagnostic criteria, 23 studies reported reference criteria for TCM syndrome differentiation, and no study clearly distinguished primary and secondary outcome indicators. 8 studies reported the evaluation criteria for TCM syndrome efficacy. Conclusion Our findings indicate that current outcome indicators for refractory Mycoplasma pneumoniae pneumonia (RMPP) in children are predominantly based on clinical symptoms, signs, and laboratory parameters. This field is burdened by longstanding unresolved issues, including the unclear delineation between primary and secondary outcomes and the lack of standardized evaluation criteria. In addition, emerging challenges have been identified, such as a progressive decline in health economic evaluation reports and a notable absence of long-term prognostic indicators.In the future, clinicians need to further explore these issues, establish a core indicator system, and, at the appropriate time, reintroduce health economic evaluations and long-term prognostic indicators. This study contributes to providing an objective basis for conducting more rigorous clinical research in the future.

Key words: Pneumonia, Mycoplasma, Refractory Mycoplasma pneumoniae pneumonia, Child, Traditional Chinese medicine, Outcome indicators, Randomized controlled trial

摘要： 背景 肺炎支原体肺炎是儿童社区获得性肺炎的常见类型，其中 2.3%~6.8% 的患儿经大环内酯类抗生素治疗 7 d 以上病情持续进展，演变为难治性肺炎。近年来鉴于该病患儿数量呈增多趋势，中医药治疗该病的随机对照临床研究取得一定进展，但对应的结局指标体系仍有待完善。目的 通过系统分析中医药治疗儿童难治性肺炎支原体肺炎随机对照试验（RCT）结局指标的现状，旨在为该疾病治疗指标的完善及中医临床试验设计提供参考。方法 系统检索近 10 年 5 个中英文数据库公开发表的中医药治疗儿童难治性肺炎支原体肺炎相关研究。按照标准筛选文献、提取资料和偏倚风险评估，并对结局指标数据进行处理分析。结果 共纳入相关研究 38 篇，涉及结局指标 50 种，总计数为 282 次。前 5 位结局指标依次为临床疗效（32 次，84.21%）、不良反应发生率（25 次，65.79%）、退热时间（24 次，63.16%）、咳嗽消失时间（23 次，60.53%）和白介素（19 次，50.00%）。30 篇报告了西医诊断标准，23 项研究报告了中医辨证参考标准，未见有研究明确区分主次结局指标，8 项报告了中医证候疗效评价标准。结论 目前关于儿童难治性肺炎支原体肺炎的临床结局指标以临床症状和体征以及理化指标为主，既存在主次不清、标准不一等长期未解决的共性问题，也出现经济学评估报告逐渐减少及长期预后指标缺乏等问题。未来需要临床工作者进一步探索，建立核心指标体系，并适时重新纳入卫生经济学评价和远期预后指标，为今后开展更为严谨的临床研究工作提供客观依据。

关键词: 肺炎, 支原体；难治性肺炎支原体肺炎；儿童；中医药；结局指标；随机对照试验