Sonis, S.T. (2009). Efficacy of palifermin (keratinocyte growth factor-1) in the amelioration of oral mucositis. Core Evidence, 4(1), 199-205.

To evaluate the evidence supporting the use of palifermin for its approved indication in patients undergoing hematopoietic stem cell transplantation (HSCT) for hematologic maligancies, and for other populations at risk of oral mucositis

• Databases searched were MEDLINEand PubMed. Also, meeting abstracts for American Society of Clinical Oncology (ASCO) and American Society for Therapeutic Radiology and Oncology (ASTRO) were searched using the keywords mucositis and palifermin.
• Search keywords were oral mucositis, palifermin, and keratinocyte growth factor (KGF).
• Studies were included if they involved only human clinical trials.
• Studies were excluded if they were reviews or preclinical studies.

A total of 100 papers and four abstracts were retrieved. Full papers were used. Studies with level 2 or higher clinical evidence, studies that were not randomized clinical trials (RCTs), case reports, and economic evidence also were used.

• A total of 12 papers and three abstracts were included in the final review.
• The review did not discuss the samples.
• Patients had hematologic malignancies and planned to receive conditioning regimen for an autologous or allogenic HSCT, cycled chemotherapy for the treatment of solid tumors, or radiation therapy with concomitant chemotherapy for the treatment of cancers of the head and neck.

The evidence review supported palefermin use in the amelioration of oral mucositis. Palifermin was associated with a reduced need for opioid analgesics, reduced risk of febrile neutropenia, reduced need for total parenteral nutrition (TPN), improved patient‐reported function, and reduced hospital stay. In addition, palifermin was associated with a decrease in the costs of mucositis-associated complications in autologus HSCT recepients. It must be noted that the this was not a decrease in costs associated with mucositis‐associated complications, but a decrease in costs because of the lower incidence of adverse outcomes in patients treated with palifermin.

Palefermin is effective in the treatment of mucositis and the need to receive mucotoxic conditioning regimens in preparation for HSCT.

RCTs are needed to investigate the benefits of palifermin in patients who are not diagnosed with head and neck cancer, as the risk of significant mucositis is unpredictable in these patients. Also, concerns exist that palifermin has the potential to stimulate primary or secondary tumor growth.