OBJECTIVE: To assess the efficacy, toxicity, and potential cost benefit of palifermin in the prevention of chemotherapy- and radiation-induced mucositis.

DATA SOURCES: MEDLINE and PubMed database searches were conducted (1966–May 2006) using the following search terms: palifermin, human keratinocyte growth factor, fibroblast growth factor, mucositis, and stomatitis.

STUDY SELECTION AND DATA EXTRACTION: All published clinical trials and abstracts examining the use of palifermin, as well as information from the manufacturer, were included.

DATA SYNTHESIS: Severe mucositis resulting from anticancer therapies increases healthcare expenditures and negatively impacts patients' quality-of-life. Radiation therapy to the head and neck, as well as stem cell transplant conditioning regimens, have the highest incidence of severe mucositis. Consequences include prolonged hospitalization, need for parenteral nutrition, increased risk of infection, and severe pain. Palifermin is a recombinant human keratinacyte growth factor indicated in patients with hematologic malignancies who are undergoing stem cell transplant. In a randomized, placebo-controlled, Phase III trial, palifermin significantly reduced the incidence and duration of severe mucositis and days of parenteral nutrition and opioid analgesics in patients undergoing autologous stem cell transplant. The most common adverse effects of palifermin were rash, pruritus, cough, and taste alterations. Data in patients with solid tumors are limited, and there is a theoretical risk of stimulating tumor growth.

CONCLUSIONS: Treatment with palifermin appears to decrease the severity and duration of severe mucositis following autologous stem cell transplant. Use in these patients appears justified; however, use in non-stem cell transplant patients should be discouraged until more efficacy and toxicity data are available.

Key Words: fibroblast growth factors, human keratinocyte growth factor, mucositis, palifermin, stem cell transplantation, stomatitis

Published Online, December 26, 2006. www.theannals.com, DOI 10.1345/aph.1G473

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