Dazzi, C., Cariello, A., Giovanis, P., Monti, M., Vertogen, B., Leoni, M. … Marangolo, M. (2003). Prophylaxis with GM-CSF mouthwashes does not reduce frequency and duration of severe oral mucositis in patients with solid tumors undergoing high-dose chemotherapy with autologous peripheral blood stem cell transplantation rescue: A double blind, randomized, placebo-controlled study. Annals of Oncology, 14, 559–563.

Patients were stratified on the basis of their conditioning treatment. Patients in the treatment group were given granulocyte-macrophage colony-stimulating factor (GM-CSF) mouthwash, 150 mcg per day in 100 cm³ of sterile water. Patients in the control group received 100 cm³ of sterile water alone as placebo. Both groups were instructed to perform one-minute mouthwashes, four times per day. Treatment started the day after chemotherapy ended and continued until stomatitis resolution, neutrophil recovery, or both.

• The study reported on 90 patients with lymphoma or solid tumors who were hospitalized and receiving high-dose chemotherapy and autologous stem cell transplant.
• The treatment group had 46 patients, and the control group had 44 patients. 
• Patients' ages ranged from 15–61 years, with a median age of 29 years.

The study was conducted between July 1997 and February 2002.

This was a double-blind, randomized, placebo-controlled study.

The National Cancer Institute (NCI) Common Toxicity Criteria (CTC) for mucositis was used.

• Incidence of mucositis was similar in both groups with 87% in the treatment group and 95% in the control group experiencing mucositis.
• Grade 4 stomatitis was also similar with 33% in the treatment group and 34% in the control group.
• No significant difference was found in duration of symptoms.
• Median days with oral pain, maximum mucositis scores, and mean length of time with a mucositis score greater than 4, were all higher in the GM-CSF group than in the control group.

The intervention was not effective.

• All patients received subcutaneous G-CSF 300 mcg each day until hematopoietic reconstitution.
• Observers graded mucositis differently.
• Because 0.2% chlorhexidine solution and amphotericin B were also being used as a rinse, the effect of specific agents was not clear.
• The overall rate of oral mucositis was lower than expected, making the sample size insufficient.