Health-related quality of life in the Phase III LUME-Colon 1 study: comparison and interpretation of results from EORTC QLQ-C30 analyses

Abstract Introduction We used EORTC QLQ-C30 data from the LUME-Colon 1 study to illustrate different methods of statistical analysis for health-related quality of life (HRQoL), and compared the results. Patients and Methods Patients were randomized 1:1 to receive nintedanib 200 mg twice daily plus best supportive care (BSC) (n = 386) or matched placebo plus BSC (n = 382). Five methods (mean treatment difference averaged over time, using a mixed-effects growth curve model; mixed-effects models for repeated measurements [MMRM]; time-to-deterioration [TTD]; status change; and responder analysis) were used to analyze EORTC QLQ-C30 global health status (GHS)/QoL and scores from functional scales. Results Overall, GHS/QoL and physical functioning deteriorated over time. Mean treatment difference slightly favored nintedanib over placebo for physical functioning (adjusted mean: 2.66; 95% confidence interval [CI]: 0.97‒4.34) and social functioning (adjusted mean: 2.62; 95% CI: 0.66‒4.47). GHS/QoL was numerically better with nintedanib versus placebo (adjusted mean: 1.61; 95% CI: ‒0.004‒3.27). MMRM analysis had similar results, with better physical functioning in the nintedanib group at all timepoints. There was no significant delay in GHS/QoL deterioration (10%) and physical functioning (16%) with nintedanib versus placebo (TTD analysis). Status change analysis showed a higher proportion of patients with markedly improved GHS/QoL and physical functioning in the nintedanib versus placebo groups. Responder analysis showed a similar, less pronounced pattern. Conclusion Analyses of EORTC QLQ-C30 data showed that HRQoL was not impaired by treatment with nintedanib versus placebo. Analysis and interpretation of HRQoL endpoints should consider symptom type and severity and course of disease.