Effects of rhuGM-CSF on prevention and treatment of oral mucositis in patients receiving radiotherapy for head and neck cancer: A randomized controlled trial.

PURPOSE: Severe oral mucositis (SOM) is a serious complication of radiotherapy for head and neck cancer (HNC) with limited prevention options. This study evaluated the efficacy of prophylactic recombinant human granulocyte-macrophage colony-stimulating factor (rhuGM-CSF) mouthwash in reducing SOM incidence and explored correlative immune biomarkers.

MATERIALS AND

METHODS

In this randomized study, 100 HNC patients undergoing radiotherapy were assigned to a prevention group (n = 50), starting rhuGM-CSF mouthwash at radiotherapy initiation, or a treatment group (n = 50), initiating mouthwash only after onset of grade 1 OM. The primary endpoint was SOM incidence. Secondary endpoints included SOM duration, onset time, and quality of life (QoL). Exploratory immune biomarker analysis was performed.

RESULTS

In the per-protocol analysis, the incidence of SOM in patients receiving prophylactic rhuGM-CSF treatment was significantly reduced (33.3% vs. 66.7%; HR = 0.31, 95% CI 0.17-0.57, p < 0.001), and the median duration was shorter (0 vs. 14 days, p < 0.001) compared to the treatment group. Below the 39.97% threshold, each unit increase in baseline percentage of activated CD4 + CD38+ / CD4 + T cells (%) was associated with a 9% reduction in the risk of SOM (HR = 0.91, 95% CI 0.87-0.95, p < 0.001).

CONCLUSIONS

The prophylactic application of rhuGM-CSF mouthwash significantly decreases both the incidence and duration of SOM in patients with HNC receiving radiotherapy. An exploratory analysis suggested that the baseline percentage of activated CD4 + CD38+ / CD4 + T cells (%) could be a potential predictive biomarker for mucosal vulnerability and this hypothesis remains to be further verified.