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 trials (RCT) 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.

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 RCTs of traditional Chinese medicine (TCM) for treating refractory Mycoplasma pneumoniae pneumonia in children.

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.

The results included a total of 38 relevant studies, covering 50 outcome measures, with a total of 282 observations. Among the 38 studies, 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.

Our findings indicate that current outcome indicators for refractory Mycoplasma pneumoniae pneumonia 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.