Abstract
Simple SummaryMouth cancer is the most common malignancy in the head-and-neck region. Usually, these tumors develop from white lesions in the mouth that appear long before cancer diagnosis. However, platforms that can estimate the time-factored risk of cancer occurring from these diseases and guide treatment and monitoring approaches are elusive. To this end, our study presents time-to-event models that are based on machine learning for prediction of the risk of malignancy from oral white lesions following pathological diagnosis as a function of time. These models displayed very satisfactory discrimination and calibration after multiple tests. To facilitate their preliminary use in clinical practice and further validation, we created a website supporting the use of these models to aid decision making.Machine-intelligence platforms for the prediction of the probability of malignant transformation of oral potentially malignant disorders are required as adjunctive decision-making platforms in contemporary clinical practice. This study utilized time-to-event learning models to predict malignant transformation in oral leukoplakia and oral lichenoid lesions. A total of 1098 patients with oral white lesions from two institutions were included in this study. In all, 26 features available from electronic health records were used to train four learning algorithms—Cox-Time, DeepHit, DeepSurv, random survival forest (RSF)—and one standard statistical method—Cox proportional hazards model. Discriminatory performance, calibration of survival estimates, and model stability were assessed using a concordance index (c-index), integrated Brier score (IBS), and standard deviation of the averaged c-index and IBS following training cross-validation. This study found that DeepSurv (c-index: 0.95, IBS: 0.04) and RSF (c-index: 0.91, IBS: 0.03) were the two outperforming models based on discrimination and calibration following internal validation. However, DeepSurv was more stable than RSF upon cross-validation. External validation confirmed the utility of DeepSurv for discrimination (c-index—0.82 vs. 0.73) and RSF for individual survival estimates (0.18 vs. 0.03). We deployed the DeepSurv model to encourage incipient application in clinical practice. Overall, time-to-event models are successful in predicting the malignant transformation of oral leukoplakia and oral lichenoid lesions.
Highlights
Oral cavity cancer is the 18th most common malignancy worldwide and accounts for many head and neck cancers in contemporary clinical practice [1]
Seven hundred and sixteen patients with oral leukoplakia and lichenoid lesions were utilized for model training and internal validation
Due to the highly variable malignant-transformation potentials reported for oral leukoplakia and oral lichenoid lesions, an effective platform would help clinicians rationalize the choice of treatment intervention and deliver appropriate patient follow-up and long-term monitoring arrangements [15]
Summary
Oral cavity cancer is the 18th most common malignancy worldwide and accounts for many head and neck cancers in contemporary clinical practice [1]. Oral carcinogenesis may be associated with a lengthy pre-pathologic phase (between initial risk-factor exposure and overt disease onset), which features the occurrence of diseases with increased risk of malignancy, known as oral potentially malignant disorders (OPMDs). These include discreet, lesions such as leukoplakia (including proliferative verrucous leukoplakia), erythroplakia, erythroleukoplakia, and oral lichenoid lesions, together with more widespread conditions, such as oral submucous fibrosis, PlummerVinson syndrome, chronic discoid lupus erythematosus, and dyskeratosis congenita [4]. Platforms that encourage accurate prediction of transformation risk for such lesions on an individual basis remain elusive
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