Granulocyte-macrophage colony-stimulating factor (GM-CSF) with melanoma vaccine combined treatment does not improve survival in advanced melanoma

This study investigated the effect of granulocyte-macrophage colony-stimulating factor (GM-CSF) and peptide vaccination (PV) on survival of patients with advanced melanoma. Researchers suggested that neither GM-CSF nor PV were associated with improved survival.

GM-CSF stimulates the immune system to produce more immune cells, increasing the capacity of the body to combat and eliminate cancer cells. The GM-CSF with PV (melanoma vaccine) combined treatment has shown an increased antitumor response in previous studies. However, other studies showed no significant benefit with this combined therapy. 

The objective of this study was to evaluate the capacity of GM-CSF with PV combined treatment to increase survival in advanced melanoma.

A total of 815 patients with stage 3 or 4 melanoma were enrolled in this study. Patients were grouped by human leukocyte antigen (HLA) status. HLA-positive patients were randomly assigned to receive GM-CSF, PV, both or placebo. HLA-negative patients were randomly assigned to receive GM-CSF or placebo. Treatment was received for 1 year.

There were no significant difference in survival or recurrence rates between the groups. The average overall survival (time from treatment until death from any cause) with GM-CSF was 69.6 months and with placebo was 59.3 months. The average relapse-free survival time (time from treatment until disease return) with GM-CSF was 11.4 months and 8.8 months with placebo. Treatment-related side effects were similar between GM-CSF and placebo groups.

In patients with a visceral metastasis (such as to the liver or lungs), the average overal survival following GM-CSF was 72.4 months. This was significantly longer than the 37.3 months seen with placebo.

This study suggested that neither GM-CSF nor PV significantly improved RFS or OS in patients with advanced melanoma. The question of whether it is beneficial in patients with visceral metastasis needs further study.