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 mu cositis may be dose-limiting. Here we investigate the effect of granul ocyte-macrophage colony-stimulating factor (GM-CSF), which significant ly shortens duration of neutropenia after hematopoietic stem-cell tran splantation (HSCT) on oral mucositis. Patients and Methods: Thirteen c hildren undergoing HSCT were prepared with etoposide (VP-16), thiotepa (TT), and total-body irradiation (TBl), and 13 with VP-16, TT, and cy clophosphamide (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: Muc ositis was more severe and persisted longer in patients prepared with the TBl-containing regimen. For this regimen, the duration of severe o ral 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, a nd CPM. The incidence of positive fungal oral or blood cultures did no t appear different whether patients received GM-CSF or not. Conclusion : For patients undergoing stomatotoxic HSCT regiments, GM-CSF may redu ce the duration of oral mucositis, but is unlikely to effect the sever ity of oral mucositis or risk of airway compromise, and the severity o f mucositis is likely to remain dose-limiting. (C) 1994 by American So ciety of Clinical Oncology.