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
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.
The study was comprised of 42 patients (8 sites), with a mean age of 50.
21 each dose level – 14 study agent, 7 placebo
Randomized, double blind, placebo-controlled, phase I/II study
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).
Number of post-transplant doses required was unclear.
Larger trial is needed.