Automatic prediction of treatment outcomes in patients with diabetic macular edema using ensemble machine learning

Background: This study aimed to predict the treatment outcomes in patients with diabetic macular edema (DME) after 3 monthly anti-vascular endothelial growth factor (VEGF) injections using machine learning (ML) based on pretreatment optical coherence tomography (OCT) images and clinical variables. Methods: An ensemble ML system consisting of four deep learning (DL) models and five classical machine learning (CML) models was developed to predict the posttreatment central foveal thickness (CFT) and the best-corrected visual acuity (BCVA). A total of 363 OCT images and 7,587 clinical data records from 363 eyes were included in the training set (304 eyes) and external validation set (59 eyes). The DL models were trained using the OCT images, and the CML models were trained using the OCT images features and clinical variables. The predictive posttreatment CFT and BCVA values were compared with true outcomes obtained from the medical records. Results: For CFT prediction, the mean absolute error (MAE), root mean square error (RMSE), and R2 of the best-performing model in the training set was 66.59, 93.73, and 0.71, respectively, with an area under receiver operating characteristic curve (AUC) of 0.90 for distinguishing the eyes with good anatomical response. The MAE, RMSE, and R2 was 68.08, 97.63, and 0.74, respectively, with an AUC of 0.94 in the external validation set. For BCVA prediction, the MAE, RMSE, and R2 of the best-performing model in the training set was 0.19, 0.29, and 0.60, respectively, with an AUC of 0.80 for distinguishing eyes with a good functional response. The external validation achieved a MAE, RMSE, and R2 of 0.13, 0.20, and 0.68, respectively, with an AUC of 0.81. Conclusions: Our ensemble ML system accurately predicted posttreatment CFT and BCVA after anti-VEGF injections in DME patients, and can be used to prospectively assess the efficacy of anti-VEGF therapy in DME patients.