Rosen, L.S., Abdi, E., Davis, I.D., Gutheil, J., Schnell, F.M., Zalcberg, J., … Clarke, S. (2006). Palifermin reduces the incidence of oral mucositis in patients with metastatic colorectal cancer treated with fluorouracil-based chemotherapy. Journal of Clinical Oncology, 24(433), 5194–5200.

DOI Link

Intervention Characteristics/Basic Study Process

Palifermin was administered at 40 mcg/kg IV for three consecutive days before each of two chemotherapy cycles with fluorouracil (5-FU) or leucovorin (LV).

Sample Characteristics

The study reported on patients with metastatic colorectal cancer receiving 5-FU or LV. The group receiving palifermin had 28 patients, and the group receiving placebo had 36 patients.

Study Design

This was a phase I and II randomized double-blind, placebo-controlled study.

Measurement Instruments/Methods

  • The World Health Organization (WHO) Oral Toxicity scale was used.
  • Patients were assessed on days 1, 4, 8,12, 15, and at the end of both cycles on day 28.
  • Patients also were assessed with the Oral Mucositis Daily Questionnaire, which consists of 10 questions on overall health, mouth and throat soreness, and diarrhea.
  • Incidence and severity of mucositis and diarrhea were recorded.

Results

  • Statistically significant differences in grade 2 or higher mucositis were observed during the first cycle (p = 0.002).
  • Lower incidence of mucositis was observed during the second cycle (still statistically significant for palifermin group) (p = 0.003).
  • Fewer patients receiving palifermin required a chemotherapy dose reduction of 10% or more.
  • Disease outcomes were similar for the two groups; the authors concluded that palifermin had no effect on the disease in this clinical setting.

Limitations

  • A researcher had relationships with Amgen.
  • The study involved the IV formulation.
  • Expense was not addressed.
  • Palifermin currently is not U.S. Food and Drug Administration (FDA) approved for nonhematologic malignancies.