title: The impact of concurrent granulocyte macrophage-colony stimulating factor on radiation-induced mucositis in head and neck cancer patients: a double-blind placebo-controlled prospective phase III study by Radiation Therapy Oncology Group 9901.
abstract: OBJECTIVE
  Based on early clinical evidence of potential mucosal protection by granulocyte-macrophage colony stimulating factor (GM-CSF), the Radiation Therapy Oncology Group conducted a double-blind, placebo-controlled, randomized study to test the efficacy and safety of GM-CSF in reducing the severity and duration of mucosal injury and pain (mucositis) associated with curative radiotherapy (RT) in head-and-neck cancer patients.
  
  
  METHODS
  Eligible patients included those with head-and-neck cancer with radiation ports encompassing >50% of oral cavity and/or oropharynx. Standard RT ports were used to cover the primary tumor and regional lymphatics at risk in standard fractionation to 60-70 Gy. Concurrent cisplatin chemotherapy was allowed. Patients were randomized to receive subcutaneous injection of GM-CSF 250 microg/m2 or placebo 3 times a week. Mucosal reaction was assessed during the course of RT using the National Cancer Institute Common Toxicity Criteria and the protocol-specific scoring system.
  
  
  RESULTS
  Between October 2000 and September 2002, 130 patients from 36 institutions were accrued. Nine patients (7%) were excluded from the analysis, 3 as a result of drug unavailability. More than 80% of the patients participated in the quality-of-life endpoint of this study. The GM-CSF did not cause any increase in toxicity compared with placebo. There was no statistically significant difference in the average mean mucositis score in the GM-CSF and placebo arms by a t test (p = 0.4006).
  
  
  CONCLUSIONS
  This placebo-controlled, randomized study demonstrated no significant effect of GM-CSF given concurrently compared with placebo in reducing the severity or duration of RT-induced mucositis in patients undergoing definitive RT for head-and-neck cancer.
authors.affiliation: Department of Radiation Oncology, University of California, Davis Medical Center, Sacramento, CA 95817, USA. janice.ryu@ucdmc.ucdavis.edu
authors.forename: Janice K
authors.initials: JK
authors.lastname: Ryu
authors.forename: Suzanne
authors.initials: S
authors.lastname: Swann
authors.forename: Francis
authors.initials: F
authors.lastname: LeVeque
authors.forename: Charles W
authors.initials: CW
authors.lastname: Scarantino
authors.forename: Darlene
authors.initials: D
authors.lastname: Johnson
authors.forename: Allan
authors.initials: A
authors.lastname: Chen
authors.forename: Andre
authors.initials: A
authors.lastname: Fortin
authors.forename: JonDavid
authors.initials: J
authors.lastname: Pollock
authors.forename: Harold
authors.initials: H
authors.lastname: Kim
authors.forename: Kian K
authors.initials: KK
authors.lastname: Ang
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D004311:Double-Blind Method; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000009:adverse effects / Q000627:therapeutic use*; D006258:Head and Neck Neoplasms / Q000532:radiotherapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009061:Mouth Mucosa / Q000187:drug effects / Q000528:radiation effects; D011446:Prospective Studies; D011832:Radiation Injuries / Q000517:prevention & control*; D011837:Radiation-Protective Agents / Q000009:adverse effects / Q000627:therapeutic use*; D013280:Stomatitis / Q000209:etiology / Q000517:prevention & control*
chemical_list: D011837:Radiation-Protective Agents; D016178:Granulocyte-Macrophage Colony-Stimulating Factor