granulocyte/monocyte-colony Therapeutic efficacy by recombinant humanstimulating factor on mucositis occurring in patients with oral and oropharynx tumors treated with curative radiotherapy - A multicenter open randomized phase III study

Background. Previous studies suggested granulocyte-macrophage-colony stimulating factor (GM-CSF) might be beneficial for radiotherapy-induced mucositis. This trial examined the efficacy of GM-CSF in reducing mucositis of the oral cavity and/or oropharynx compared with conventional treatment. Methods. Mucositis, documented by a five-grade scale, was defined in patients with tumors of the head-neck. Centers were allowed to use their own preferred fractionation regimen. Randomization to treatment was decided before radiotherapy. Treatment with GM-CSF 4 mu g/kg/d subcutaneous, started when patients displayed a mucositis score greater than= 1.5. Results. Ninety-two patients entered the study according to intention-to-treat principle. Twenty did not reach a mucositis index of 1.5. Sixty-one patients were included in the statistical analysis. Forty-five percent of the patients randomized to receive GM-CSF had a significant reduction of the mucositis more than one grade compared to 9% of the conventional treated. Conclusions. In severe mucositis, GM-CSF is more effective than conventional treatment.