GM-CSF mouthrinses in the treatment of severe oral mucositis: a pilot study

The aim of this open trial was to test the efficacy of granulocyte-macrophage colony-stimulating factor (GM-CSF) mouthrinses as a potential treatment in reducing the duration of severe oral mucositis in patients undergoing bone marrow transplantation for hematologic malignancies.

The study group was composed of 10 consecutive patients suffering from severe oral mucositis during bone marrow transplantation procedures. The control group was similar to the study group in age and gender and comprised 29 historical patients with similar clinical characteristics. Freshly prepared GM-CSF mouthwash (0.5 microg/mL) was administered to the study population for 1 minute 3 times per day after oral hygiene procedures, starting from the first day of mucositis until clinical improvement of oral lesions. The study and control populations were compared with respect to duration of severe oral mucositis (1-9 days, 10-19 days, > or =20 days).

There was no statistically (chi2 exact test) significant difference in mean mucositis score between the study group (11.9+/-6.1) and the control group (16.6+/-8.9). However, the duration of severe mucositis appeared to be reduced; 60% of the GM-CSF mouthrinse patients had severe mucositis for less than 9 days, whereas only 28% of the controls had severe mucositis for less than 9 days. In addition, 10% of the GM-CSF mouthrinse patients experienced severe mucositis lasting 20 or more days, whereas 34% of the controls experienced severe mucositis for 20 or more days.

These findings suggest that GM-CSF may reduce the duration of severe mucositis, but controlled, double-blind clinical trials are now required.