Oral mucositis - palifermin

Oral mucositis is a painful and debilitating condition in which patients develop severe mouth ulcerations that can make normal activities such as eating, swallowing and talking difficult, if not impossible. The pain can prevent the patient eating and drinking properly, which can ultimately lead to malnutrition and dehydration, so in severe cases sufferers often need to be fed intravenously. It is a common side-effect of in-patients with haemotologic malignancies who are undergoing high dose chemotherapy and radiotherapy.

Amgen has been developing palifermin as a potential solution. It is a recombinant human keratinocyte growth factor (rHuKGF) that appears to protect the epithelial cells lining the mouth and gut from this damage caused by radio-therapy or chemotherapy. Natural keratinocyte growth factor stimulates the growth and development of epithelial cells, including those that line the mouth and gastrointestinal tract.

In a Phase III study, 212 patients were randomised to receive either 60µg/kg a day of palifermin or placebo for three days prior to high dose chemotherapy and total body irradiation.1,2 All patients then received peripheral blood progenitor cell transplantation, followed by an additional three days of either palifermin or placebo. The group given the active had a significantly lower incidence of severe oral mucositis, with three times fewer of those given palifermin developing the most severe form of oral mucositis. It was also well tolerated, with adverse events including mild to moderate skin and oral erythema, with or without oedema.

Those given palifermin who did develop severe oral mucositis had a shorter duration of the condition - 3.7 days compared with 10.4 days. Active treated patients also reported 60% less soreness of the mouth and throat, required lower doses of painkillers, and less total parenteral nutrition use.