Epidermal growth factor receptor positive lung cancer: The nontrial scenario

The aim of this study was to report the median overall survival (OS) in epidermal growth factor receptor (EGFR) mutation-positive patients who were managed out of a clinical trial.

Nonsmall cell lung cancer patients harboring activating EGFR mutations who were either ineligible or refused participation in a clinical trial were selected for this analysis. The reason for not participating in trial, staging, treatment, and outcome details were obtained from a prospective lung cancer database. The Kaplan-Meier method was used to estimate OS. Log-rank test and Cox proportion hazard model were used for univariate and multivariate analysis, respectively.

We included 225 patients in this analysis. The median age of the cohort was 56 years (range 29-85 years). A compromised Eastern Cooperative Oncology Group performance status (PS) of >2 was the major reason (83 patients, 36.9%) for ineligibility of patients in a clinical trial. The major reason provided by eligible patients for refusal to participate in a clinical trial was long distance of travel and inability to comply with the study-mandated follow-up visits (65 patients, 28.9%). The median OS in patients with PS 0-2 was 18.17 months (95% confidence interval [CI]: 15.6-20.8 months) and it was 12.1 months (95% CI: 9.0-15.2 months) in patients with PS 3-4 (hazard ratio - 0.579 [95% CI: 0.398-0.843] P = 0.004).

EGFR positive patients who were ineligible for a clinical trial due to poor PS had lower survival; however, patients with good PS treated off-trial had similar OS to that reported in multiple clinical trials.