Rossi, A., Rosati, G., Colarusso, D., & Manzione, L. (2003). Subcutaneous granulocyte–macrophage colony-stimulating factor in mucositis induced by an adjuvant 5-fluorouracil plus leucovorin regimen. Oncology, 64(4), 353–360.

GMCSF subcutaneously
(4 mcg/kg) from days 6-10 during cycle following the cycle Grade ≥ 2 mucositis was reported.

Also received “standard” anti-mucositis treatment

The study was comprised of 31 patients, age 39-77 years with a median age of 68.
Three stage phase II trial
Colorectal cancer treated with 5FU plus leucovorin and reporting mucositis ≥ Grade 2.
 

Chen design
WHO Mucositis – 3x/wk:

Grade 0 = none

I = painless ulcers, erythema, or mild soreness
2 = painful erythema, edema, or ulcers
3 = can’t eat solids
4 = TPN or enteral support
 

77 GM-CSF cycles were administered (median/pt = 3).

Success claimed based on reduction of at least one grade of mucositis.

9 of 20 responders mucositis disappeared – 64.5%

45% with mucositis responded, 12.9% failure, 3.2% stopped treatment
3.2 % (1 patient) with mucositis plus diarrhea responded, 3.2% treatment failure, 3.2% stopped treatment

Toxicity in 7 of 20 patients

 

Claim results are rationale for well-designed trial

No control group
Small N
No P values stated percentages hard to follow.