Development, validation, and clinical utility of electronic patient-reported outcome measure-enhanced prediction models for overall survival in patients with advanced non-small cell lung cancer receiving immunotherapy

Abstract

PURPOSE: Electronic patient-reported outcome measures (ePROMs) are increasingly collected routinely in clinical practice and may be prognostic for survival in adults with advanced non-small cell lung cancer (NSCLC) in addition to clinical data. This study developed ePROM-enhanced models for predicting 1-year overall survival in patients with advanced NSCLC at the start of immunotherapy. METHODS: This is a single-center study using consecutive patients from a tertiary cancer hospital in England. Using Cox proportional hazards models, we developed one clinical factor-only model and three ePROM-enhanced models, each including one of the following factors: quality of life (as measured by EuroQoL five-dimension five-level utility score) and overall symptom burden and number of moderate-to-severe symptoms (as measured by patient-reported version of Common Terminology Criteria for Adverse Events). Predictive performance was evaluated and compared through bootstrapping internal validation, and clinical utility was determined via decision curve analysis. RESULTS: The clinical factor-only model contained age, histology, performance status, and neutrophile-to-lymphocyte ratio. While calibration was similar between the clinical factor-only and ePROM-enhanced models, the latter showed improved discrimination by 0.020 (95% CI, 0.011 to 0.024), 0.024 (95% CI, 0.016 to 0.031), and 0.024 (95% CI, 0.014 to 0.029) when enhanced with ePROMs on quality of life, overall symptom burden, and number of moderate-to-severe symptoms, respectively. If care decisions are to be made at risk thresholds between 25% and 75%, the ePROM-enhanced models led to higher net benefit than the clinical factor-only model and the default strategies of intervention for all and intervention for none. CONCLUSION: The ePROM-enhanced models outperformed the clinical factor-only model in predicting 1-year overall survival for patients with advanced NSCLC receiving immunotherapy and showed potential clinical utility for informing decisions in this population. Future studies should focus on validating the models in external data sets.