Clinical effectiveness of palifermin in prevention and treatment of oral mucositis in children with acute lymphoblastic leukaemia: a case-control study

Int J Oral Sci. 2014 Mar;6(1):27-30. doi: 10.1038/ijos.2013.93. Epub 2013 Dec 20.


The aim of this study was to evaluate the efficacy of palifermin, an N-terminal truncated version of endogenous keratinocyte growth factor, in the control of oral mucositis during antiblastic therapy. Twenty patients undergoing allogeneic stem-cell transplantation for acute lymphoblastic leukaemia were treated with palifermin, and compared to a control group with the same number of subjects and similar inclusion criteria. Statistical analysis were performed to compare the outcomes in the treatment vs. control groups. In the treatment group, we found a statistically significant reduction in the duration of parenteral nutrition (P=0.002), duration of mucositis (P=0.003) and the average grade of mucositis (P=0.03). The statistical analysis showed that the drug was able to decrease the severity of mucositis. These data, although preliminary, suggest that palifermin could be a valid therapeutic adjuvant to improve the quality of life of patients suffering from leukaemia.

Publication types

  • Comparative Study

MeSH terms

  • Adolescent
  • Allografts / transplantation
  • Anti-Inflammatory Agents / therapeutic use*
  • Case-Control Studies
  • Child
  • Cohort Studies
  • Cyclophosphamide / therapeutic use
  • Female
  • Fibroblast Growth Factor 7 / therapeutic use*
  • Hematopoietic Stem Cell Transplantation / methods
  • Humans
  • Male
  • Myeloablative Agonists / therapeutic use
  • Parenteral Nutrition
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy*
  • Radiotherapy Dosage
  • Stomatitis / classification
  • Stomatitis / drug therapy
  • Stomatitis / prevention & control*
  • Time Factors
  • Transplantation Conditioning / methods
  • Treatment Outcome
  • Whole-Body Irradiation


  • Anti-Inflammatory Agents
  • Myeloablative Agonists
  • Fibroblast Growth Factor 7
  • Cyclophosphamide