EFFECT OF GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR ON ORAL MUCOSITIS AFTER HEMATOPOIETIC STEM-CELL TRANSPLANTATION

Purpose: Oral mucositis following high-dose chemotherapy may result in systemic infection and airway compromise, and the severity of oral mucositis may be dose-limiting. Here we investigate the effect of granulocyte-macrophage colony-stimulating factor (GM-CSF), which significantly shortens duration of neutropenia after hematopoietic stem-cell transplantation (HSCT) on oral mucositis. Patients and Methods: Thirteen children undergoing HSCT were prepared with etoposide (VP-16), thiotepa (TT), and total-body irradiation (TBl), and 13 with VP-16, TT, and cyclophosphamide (CPM). Following transplantation, 14 patients received GM-CSF at a dose of 125 mu g/m(2)/d by continuous intravenous infusion (six prepared with VP-16, TT, and TBl, and eight prepared with VP-16, TT, and CPM), and 12 patients received no growth factor. Results: Mucositis was more severe and persisted longer in patients prepared with the TBl-containing regimen. For this regimen, the duration of severe oral mucositis was shortened by the administration of GM-CSF, although the severity of mucositis was unaffected. No statistically significant effect of GM-CSF could be shown in patients who received VP-16, TT, and CPM. The incidence of positive fungal oral or blood cultures did not appear different whether patients received GM-CSF or not. Conclusion: For patients undergoing stomatotoxic HSCT regiments, GM-CSF may reduce the duration of oral mucositis, but is unlikely to effect the severity of oral mucositis or risk of airway compromise, and the severity of mucositis is likely to remain dose-limiting. (C) 1994 by American Society of Clinical Oncology.