McAleese, J.J., Bishop, K.M., A’Hern, R., & Henk, J.M. (2006). Randomized phase II study of GM-CSF to reduce mucositis caused by accelerated radiotherapy of laryngeal cancer. British Journal of Radiology, 79(943), 608–613.

Intervention Characteristics/Basic Study Process

GM-CSF 2 mcg/kg (-1) (150 mcg) daily for 14 days starting on day 14 of a 21-day course of RT.

Note: Placebo injections were not given; observer was blinded to treatment.

Sample Characteristics

The study was comprised of 29 patients (GM-CSF = 14).

Power analysis was completed, but not met (n = 17 in each group).

Patients with proven T1 N0 or T2 N0 glottic carcinoma were being treated with radiotherapy using a 16 fraction 3-week regimen.

Required to have a WHO performance status of grade 0 or higher, no renal or hepatic issues, serious infections requiring antibiotics, or likely need for corticosteroids.
 

Study Design

Prospective, randomized, observer blind phase II trial

Measurement Instruments/Methods

  • RTOG
  • Skin erythema
  • Moist and dry desquamation
  • Pain on swallowing
  • Severity of dysphagia
  • Analgesic usage
  • Evidence of candida infection and laryngeal edema
  • Patients weights before and after treatment
     

Results

Significant difference in the incidence of mucositis p < 0.05, mean time of healing improved but not significant p = 0.25.
No differences in dysphagia, odynophagia, analgesic use, candida infection, of laryngeal edema.
 

Limitations

  • Study sample size was very small.
  • Only used for early treatment of laryngeal cancer with radiotherapy using 16 fraction 3-week regimen.
  • Frequent side effects related to GMCSF
  • No large trials have been conducted.