Hejna, M., Kostler, W.J., Raderer, M., Steger, G.G., Brodowicz, T., Scheithauer, W., … Zielinski, C.C. (2001). Decrease of duration and symptoms in chemotherapy-induced oral mucositis by topical GM-CSF: Results of a prospective randomized trial. European Journal of Cancer, 37, 1971–1975.

Patients were randomized to one of two arms.

• Arm A was given topical granulocyte-macrophage colony-stimulating factor (GM-CSF) (Leucomax) mouthwash three times per day. The mouthwash consisted of 400 mcg GM-CSF in 250 mL water. Patients were instructed to rinse with 25 mL for 3 minutes and repeat 10 times within 30 minutes. They were to repeat this process at three identical times each day.
• Arm B was given a solution of 4 mL povidone-iodine in 125 ml water with amphotericin B. They were to rinse with 10 mg four times per day according to the same instructions as Arm A.

Both groups were instructed to continue using the respective mouthwashes until complete response (CR). A third, independent investigator randomized patients without knowing individual mucositis ratings.

• The study reported on 31 patients, with 15 in Arm A and 16 in Arm B.
• Patients' ages ranged from 39–77 years, with a median age of 58 years.
• All patients had solid tumor diagnoses.
• The World Health Organization (WHO) oral mucositis scale was used to grade patients after they had received fluorouracil (5FU)-based chemotherapy. Two investigators independently rated mucositis severity.

The study was conducted between March 1998 and June 1999.

This was a prospective, randomized, controlled study.

Every three days, objective and subjective evaluations were conducted.

• Patients who received the GM-CSF treatment experienced shorter duration (5.3 versus 8.1 [p = 0.0008]) and quicker resolution (2.8 versus 4.1 [p = 0.0011]) of mucositis.
• No side effects were reported, and the treatment was well tolerated.

• The sample size was small.
• Application of mouthwash findings is complicated.
• The treatment has a high cost.