Outcome differences by sex in oncology clinical trials

Abstract

Identifying sex differences in outcomes and toxicity between males and females in oncology clinical trials is important and has also been mandated by National Institutes of Health policies. Here we analyze the Trialtrove database, finding that, strikingly, only 472/89,221 oncology clinical trials (0.5%) had curated post-treatment sex comparisons. Among 288 trials with comparisons of survival, outcome, or response, 16% report males having statistically significant better survival outcome or response, while 42% reported significantly better survival outcome or response for females. The strongest differences are in trials of EGFR inhibitors in lung cancer and rituximab in non-Hodgkin’s lymphoma (both favoring females). Among 44 trials with side effect comparisons, more trials report significantly lesser side effects in males (N = 22) than in females (N = 13). Thus, while statistical comparisons between sexes in oncology trials are rarely reported, important differences in outcome and toxicity exist. These considerable outcome and toxicity differences highlight the need for reporting sex differences more thoroughly going forward.

Reporting outcome comparisons by sex in oncology clinical trials

“…A recent data analysis article reported by Kammula et al in Nature Communications 11 represents a significant response to this urgent research need, which will be studied and applied by oncology field for years to come. This study comprehensively characterised sex outcome comparisons in all oncology interventional clinical trials and identified comparisons that found a significant difference between males and females.…”