Health-related quality of life (HRQOL) assessment in randomized clinical trials (RCTs) in small cell lung cancer (SCLC) provides relevant added information in studies where the treatment arms do not differ in terms of efficacy. More importantly, it provides valuable information for those treatments where better HRQOL is associated with overall survival benefit. Also, the study found that, even though the overall standard for reporting HRQOL was acceptable, improvement is still needed regarding its reporting in RCTs to optimize its value.
Small cell lung cancer accounts for approximately 15% of all lung cancers, and most patients with SCLC present with advanced disease. Consequently, disease management and treatment focuses on symptom control and HRQOL. In randomized clinical trials, HRQOL of small cell lung cancer is now being evaluated more frequently.
The objective of this study by the European Organization for Research and Treatment of Cancer (EORTC) was to evaluate the adequacy of HRQOL methodology reporting in small cell lung cancer RCTs and the possible impact of this on clinical decision making. The study was published in The Lancet Oncology (2014;15:e78-e89).
The study concerned randomized clinical trials that implemented patient-reported HRQOL assessments and oncology treatments for adult patients (age 18 years or older) with small cell lung cancer that were completed between January 1991 and December 2012, included 100 or more patients, and were published in English. More than 10,000 patients from 30 RCTs were classified as eligible for this study, from an initial pool of 79 studies found.
The EORTC study found that HRQOL was a secondary end point in 29 RCTs of which 53% reported no significant difference in overall survival. A difference of HRQOL was reported in 77% of the positive-outcome trials, and in 50% of the negative-outcome trials. A priori hypothesis on the expected overall HRQOL outcome was defined in 27% of the RCTs. Baseline HRQOL assessment was stated as mandatory in 14% of the RCTs. Tests of statistical significance were applied in 90% of the RCTs, and missing data were discussed in detail in 30% of the trials.
“The EORTC has been a leader in establishing standards for conducting systematic reviews of patient-reported outcomes in oncology randomized clinical trials. Arguably, the most important point about science is if results are reproducible. We have seen, time and again in over a decade of systematic literature research, that our results are consistent. We hope our quality-of-life research program has been useful in obtaining robust clinical research results, that these can be interpreted with confidence, and that decision makers can accept these findings from clinical trials in oncology. We hope our research has led others to use the EORTC Patient-Reported Outcomes Checklist to design and evaluate other clinical trials and ensure high-quality HRQOL,” said Andrew Bottomley, PhD, EORTC assistant director and an author of this study.