Composite patient-reported outcomes and risk prediction for overall survival in advanced non-small cell lung cancer...

Patient-reported outcomes (PROs) are associated with overall survival (OS) in advanced cancer. Risk modeling of OS based on a single PRO scale was previously evaluated in patients with advanced non-small cell lung cancer (NSCLC) in two pivotal cemiplimab ± chemotherapy phase III trials. Here we report evaluation of predictive performance for a composite of two PRO scales.

Data from two previously published phase III clinical trials (EMPOWER-Lung 1 and EMPOWER-Lung 3) were used to develop a Cox proportional hazards model evaluating the association between baseline PRO burden and OS, stratified by treatment, histology, and programmed cell death-ligand 1 (PD-L1) level. PRO data were collected using the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 (QLQ-C30) and Lung Cancer 13 (QLQ-LC13) modules. Single-scale PROs and composite PROs based on a combination of one functioning and one symptom scale were evaluated for prognostic value for OS using hazard ratios (HRs; a higher HR indicates a higher risk of death).

The top 10 composite PROs that predicted the highest risk for death (nominal P values <.05 and HRs ≥2) included combinations of functioning scales (ie, social, role, and physical) and select symptom scales (ie, dyspnea, appetite loss, and pain from the EORTC QLQ-C30; dyspnea and coughing from the EORTC QLQ-LC13). Patients with composite PROs of low functioning and high symptom burden had worse OS than those with high functioning and low symptom burden. A clear separation in Kaplan-Meier survival curves was observed between high-risk and low-risk groups based on the composite PRO measure of role functioning and dyspnea.

In patients with advanced NSCLC receiving first-line cemiplimab-based therapy, composite PROs consisting of one functioning and one symptom scale had greater prognostic value than single PRO scales. Further development and analysis of composite PROs for clinical trials is warranted.