BACKGROUND: Health-related quality of life (HRQOL) parameters are often used as secondary endpoints in advanced non-small-cell lung cancer (NSCLC). We assessed the frequency and correlates of both usage and gain in HRQOL endpoints in NSCLC phase III trials. METHODS: We searched PubMed for phase III trials on systemic anticancer therapies for NSCLC published between 1/98 and 12/09 in 13 leading journals. RESULTS: The search yielded 122 trials that enrolled a total of 56,031 patients in 273 trial arms. HRQOL was reportedly used as an endpoint in 72 trials (59%). HRQOL parameters were used as primary or co-primary endpoints in nine trials, whereas overall survival (OS) was the primary endpoint or one of the co-primary endpoints in 90 trials. There was no temporal trend for usage of HRQOL parameters as endpoints. Formal statistical comparisons involving HRQOL were reported in 68/72 cases, and a significant difference was found in 37/68 trials (54.4%), 24 of which favored the experimental arm. In many cases, such differences were restricted to specific symptoms or even favored more than one regimen according to symptoms analyzed. We found no significant association between gain in HRQOL and gain in OS or any other trial feature. CONCLUSIONS: HRQOL has been assessed formally in nearly 60% of contemporary phase III trials in advanced NSCLC, and a significant gain in HRQOL has been found in almost one-half of cases. It is questionable, however, whether HRQOL results may help select between treatments with no differential impact on OS.
BACKGROUND: Health-related quality of life (HRQOL) parameters are often used as secondary endpoints in advanced non-small-cell lung cancer (NSCLC). We assessed the frequency and correlates of both usage and gain in HRQOL endpoints in NSCLC phase III trials. METHODS: We searched PubMed for phase III trials on systemic anticancer therapies for NSCLC published between 1/98 and 12/09 in 13 leading journals. RESULTS: The search yielded 122 trials that enrolled a total of 56,031 patients in 273 trial arms. HRQOL was reportedly used as an endpoint in 72 trials (59%). HRQOL parameters were used as primary or co-primary endpoints in nine trials, whereas overall survival (OS) was the primary endpoint or one of the co-primary endpoints in 90 trials. There was no temporal trend for usage of HRQOL parameters as endpoints. Formal statistical comparisons involving HRQOL were reported in 68/72 cases, and a significant difference was found in 37/68 trials (54.4%), 24 of which favored the experimental arm. In many cases, such differences were restricted to specific symptoms or even favored more than one regimen according to symptoms analyzed. We found no significant association between gain in HRQOL and gain in OS or any other trial feature. CONCLUSIONS: HRQOL has been assessed formally in nearly 60% of contemporary phase III trials in advanced NSCLC, and a significant gain in HRQOL has been found in almost one-half of cases. It is questionable, however, whether HRQOL results may help select between treatments with no differential impact on OS.