Effectiveness Not Established


for Mucositis

Repifermin is a keratinocyte growth factor that stimulates the production and differentiation of keratinocytes. Keratinocytes are primarily found in the skin and are involved in epithelialization of wounds. Repifermin was evaluated in patients with cancer in the treatment of mucositis.

Research Evidence Summaries

Freytes, C.O., Ratanatharathorn, V., Taylor, C., Abboud, C., Chesser, N., Restrepoo, A. et al. (2004). Phase I/II randomized trial evaluating the safety and clinical effects of repifermin administered to reduce mucositis in patients undergoing autologous hematopoietic stem cell transplantation. Clinical Cancer Research, 10, 8318-8324. PubMed Abstract

Intervention Characteristics/Basic Study Process

Repifermin (keratinocyte growth factor-2)
25 or 50 micrograms/kg
IV for 3 days prior to conditioning regimen and continued after transplant for up to 10 days.

Assessments before HSCT regimen, day of transplant, and 3x/wk until mucositis resolved.

Sample Characteristics

The study was comprised of  42 patients (8 sites), with a mean age of 50.
21 each dose level – 14 study agent, 7 placebo



Study Design

Randomized, double blind, placebo-controlled, phase I/II study

Measurement Instruments/Methods

  • NCI-CTC mucositis toxicity scale for BMT OMAS
  • Severity of pain (0-10)
  • Severity of pain while swallowing
  • Duration of pain
  • Ability to eat score (1 = normal, 2 = soft solids, 3 = liquids, 4 = nothing)
  • Narcotic use for pain
  • Frequency, severity, and duration of diarrhea


  • Grade 2 – 4 mucositis
  • Placebo 100%, 64.2%
  • 25 mcg  p = 0.041
  • Placebo vs 50 mcg  (50%) p = 0.006



Effectiveness not established; study was not designed to have sufficient statistical power in identifying differences in efficacy between groups.

Number of end points suggest better outcome for 50 mcg group (mean duration, pain on swallowing, days on narcotic pain medication).


  • Study predominantly designed for “safety” not “efficacy”.
  • Small sample group used (n = 42)
  • Multiple conditioning regimens were used; this may affect final outcome as far as efficacy.
  • Auto HSCT population only
  • Multiple doses were used in study.

Nursing Implications

Number of post-transplant doses required was unclear.

Larger trial is needed.