EFFECT OF GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR ON ORAL MUCOSITIS IN HEAD AND NECK-CANCER PATIENTS AFTER CISPLATIN, FLUOROURACIL, AND LEUCOVORIN CHEMOTHERAPY

Purpose: To evaluate prospectively the efficacy of granulocyte-macrophage colony-stimulating factor (GM-CSF) in the reduction of chemotherapy-induced oral mucositis. Patients and Methods: Twenty patients with stage IV squamous cell carcinoma of head and neck were studied, Two-cycles (periods) of identical doses of cisplatin, fluorouracil (5-FU), and leucovorin (PFL) chemotherapy with cisplatin 20 mg/m(2)/d, 5-FU 800 mg/m(2)/d, leucovorin 90 mg/m(2)/d by 96-hour continuous intravenous infusion every 3 weeks were given to each patient. After PFL chemotherapy, GM-CSF 4 mu g/kg subcutaneously from days 5 to 14 or no therapy was given by a randomized self-controlled crossover study design, Oral mucositis was graded with modified Radiation Therapy Oncology Group criteria. Results: In the first cycle of PFL chemotherapy, GM-CSF significantly reduced the incidence, mean duration, and mean area under the curve (AUG) of severe oral gross mucositis (grade greater than or equal to 3) compared with no therapy, These beneficial effects continued into the second cycle of PFL chemotherapy after crossover to no GM-CSF. The incidence of severe mucositis was reduced when GM-CSF was given in the second cycle of PFL, Analysis of variance indicated significant direct GM-CSF treatment effects on the mean AUC of gross/functional scores and duration of moderate gross mucositis (grade greater than or equal to 2) over both periods, There was a significant period effect in favor of giving GM-CSF in the first cycle of chemotherapy. Conclusion: GM-CSF can significantly reduce the severity and duration of chemotherapy-induced oral mucositis after PFL chemotherapy. (C) 1995 by American Society of Clinical Oncology.