Long-term follow-up of a phase I/II randomized, placebo-controlled trial of palifermin to prevent graft-versus-host disease (GVHD) after related donor allogeneic hematopoietic cell transplantation (HCT)

Biol Blood Marrow Transplant. 2008 Sep;14(9):1017-1021. doi: 10.1016/j.bbmt.2008.06.013.


We previously conducted a randomized, double-blind, placebo-controlled study conducted from 2000 to 2003 of palifermin, a recombinant human keratinocyte growth factor, dosed from 240 microg/kg to 720 microg/kg, in 100 allogeneic hematopoietic stem cell transplantation (HCT) recipients. Treatment with palifermin showed beneficial effects on mucositis, but no significant effect on engraftment, acute graft-versus-host disease (GVHD), or early survival. In addition to the effect of palifermin on mucosa, other pleotrophic effects, including more rapid immune reconstitution, have been seen in experimental transplant models. Therefore, we investigated whether with longer follow-up we could detect additional differences between the palifermin-treated and placebo cohorts. We found no differences in CMV or invasive fungal infections, chronic GVHD, or long-term survival between cohorts. We conclude that the benefits of palifermin appear primarily to be limited to ameliorating mucotoxicity when given to allogeneic HCT recipients.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Child
  • Chronic Disease
  • Cytomegalovirus Infections / mortality
  • Cytomegalovirus Infections / prevention & control
  • Double-Blind Method
  • Female
  • Fibroblast Growth Factor 7 / administration & dosage*
  • Graft vs Host Disease / mortality
  • Graft vs Host Disease / prevention & control*
  • Hematologic Neoplasms / mortality
  • Hematologic Neoplasms / therapy
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Living Donors*
  • Male
  • Middle Aged
  • Mucositis / mortality
  • Mucositis / prevention & control
  • Mycoses / mortality
  • Mycoses / prevention & control
  • Transplantation, Homologous


  • Fibroblast Growth Factor 7