Predicting pathologic complete response in locally advanced rectal cancer patients after neoadjuvant therapy: a machine learning model using XGBoost

PURPOSE: Watch and wait strategy is a safe and effective alternative to surgery in patients with locally advanced rectal cancer (LARC) who have achieved pathological complete response (pCR) after neoadjuvant therapy (NAT); present restaging methods do not meet clinical needs. This study aimed to construct a machine learning (ML) model to predict pCR preoperatively. METHODS: LARC patients who received NAT were included to generate an extreme gradient boosting-based ML model to predict pCR. The group was divided into a training set and a tuning set at a 7:3 ratio. The SHapley Additive exPlanations value was used to quantify feature importance. The ML model was compared with a nomogram model developed using independent risk factors identified by conventional multivariate logistic regression analysis. RESULTS: Compared with the nomogram model, our ML model improved the area under the receiver operating characteristics from 0.72 to 0.95, sensitivity from 43 to 82.2%, and specificity from 87.1 to 91.6% in the training set, the same trend applied to the tuning set. Neoadjuvant radiotherapy, preoperative carbohydrate antigen 125 (CA125), CA199, carcinoembryonic antigen level, and depth of tumor invasion were significant in predicting pCR in both models. CONCLUSION: Our ML model is a potential alternative to the existing assessment tools to conduct triage treatment for patients and provides reference for clinicians in tailoring individual treatment: the watch and wait strategy is used to avoid surgical trauma in pCR patients, and non-pCR patients receive surgical treatment to avoid missing the optimal operation time window. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s00384-022-04157-z.