The addition of sargramostim to ipilimumab therapy for unresectable stage III or IV melanoma may improve overall survival and toxicity, but it does not appear to affect progression-free survival, results from a phase II randomized trial suggest.
Patients randomized to receive ipilimumab plus sargramostim showed significantly lower mortality at 1 year, compared with those treated with ipilimumab alone (hazard ratio, 0.64), and they experienced significantly fewer grade 3 or above treatment-related toxicities, particularly gastrointestinal toxicities such as colonic perforation.
“The improved toxicity profile must be considered as contributing to the improved survival even in light of the survival advantage remaining when patients who discontinued therapy due to toxicity are excluded,” wrote Dr. F. Stephen Hodi of the Dana-Farber Cancer Institute, Boston, and colleagues.
Researchers stressed that the study, which enrolled a total of 245 patients, is ongoing, and that the findings need to be confirmed in larger studies with longer follow-up, according to a paper published online Nov. 4 (JAMA 2014 [doi:10.1001/jama.2014.13943].
The study was supported by Public Health Service grants, the Eastern Cooperative Oncology Group, the U.S. National Cancer Institute, the U.S. National Institutes of Health, the U.S. Department of Health & Human Services, Bristol-Myers Squibb, and Genzyme. Some authors reported grants, personal fees, and support from pharmaceutical companies, including Bristol-Myers Squibb and Genzyme.