Mantovani, G., Massa, E., Astara, G., Murgia, V., Gramignano, G., Lusso, M.R., … Maccio, A. (2003). Phase II clinical trial of local use of GM-CSF for prevention and treatment of chemotherapy and concomitant chemoradiotherapy-induced severe oral mucositis in advanced head and neck cancer patients: An evaluation of effectiveness, safety and costs. Oncology Reports, 10, 197-206.

DOI Link

Study Purpose

To evaluate a granulocyte-macrophage colony-stimulating factor (GM-CSF) mouthwash in the prophylactic and curative settings of oral mucositis (OM)

Intervention Characteristics/Basic Study Process

All patients were given 300 mcg of granulocyte-macrophage colony-stimulating factor (GM-CSF) in 300 ml of water; patients were instructed to rinse and gargle with the mouthwash for as long as possible, three times daily (morning, midday, and before bedtime). Patients were instructed to not eat, drink, or rinse with another mouthwash for at least one hour afterward. Patients in the prophylactic group also received amifostine (500 mg IV). Patients in the curative group were treated from the appearance of mucositis until two days after clinical resolution.

Sample Characteristics

  • The study reported on 68 patients with 46 in the prophylaxis group and 22 in the curative group.
  • All patients were treated with chemotherapy and radiation therapy. Most patients were diagnosed with advanced head and neck cancer.

Study Design

This was an open, nonrandomized, phase II study.

Measurement Instruments/Methods

  • The National Cancer Institute (NCI) Common Toxicity Criteria for oral mucositis were used.
  • The European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)–Head and Neck (H&N) 35 was used.

Results

  • Duration of mucositis was 8.7 in the prophylaxis group and 11.5 in the curative group (p = 0.089).
  • Patients had a lower mean grade of OM on day 3 (p = 0.012) and day 6 (p = 0.007).
  • Only 50% of patients in the prophylaxis group developed mucositis.

Limitations

  • The QLQ was only administered to those in the prophylactic arm of study.
  • Too many factors could have influenced patient outcomes. No control group was included.
  • The  two groups had different sizes.
  • Determining significance is difficult to based on the study design.