Vadhan-Raj, S., Trent, J., Patel, S., Zhou, X., Johnson, M.M., Araujo, D., … Benjamin, R.S. (2010). Single-dose palifermin prevents severe oral mucositis during multicycle chemotherapy in patients with cancer: a randomized trial. Annals of Internal Medicine, 153, 358–367.

To evaluate the efficacy and safety of palifermin given as a single dose before each cycle in patients receiving doxorubicin-based multicycle chemotherapy

Patients were randomly assigned in a 2:1 ratio to receive palifermin or placebo.

• The study reported on 48 patients with an age range of 15–65 years.
• The sample was 53% male and 47% female.
• All patients had sarcoma with Karnofsky performance status greater than or equal to 80% and adequate bone marrow, renal, and hepatic function.

This study was conducted at a single-site at the University of Texas M.D. Anderson Cancer Center.

Patients were undergoing the active treatment phase of care.

This was a randomized, double-blind, placebo-controlled trial.

• The World Health Organization (WHO) oral toxicity scale was used.
• Common Terminology Criteria for Adverse Events were used.
• A patient-reported outcome questionnaire was used; no data was provided about the reliability or validity of this instrument.
• A daily record symptom record diary was used; no data was provided about the reliability or validity of this instrument but it was referenced.
• Optical imaging studies and oral punch biopsies were performed prior to and 48–72 hours after the first dose of study drug to evaluate biologic effects of treatment on mucosa.

Palifermin significantly reduced the incidence of moderate to severe (grade 2 or higher) mucositis (44% versus 88%; p <0.001) and severe mucositis (13% versus 51%; p < 0.002).

A single dose of palifermin before each cycle reduced the incidence and severity of mucositis. It also demonstrated effectiveness as secondary prophylaxis in a few patients with severe mucositis.

• The sample size was small with fewer than 100 patients.
• A perceived unblinding of the treatment may have occurred because of notable differences between the biologic effects of palifermin and placebo.
• The study only looked at treatment of one disease.
• The follow-up period was short.

Mucositis and the pain it causes can significantly impact patients with cancer during treatment. Further research is needed to establish the alleviation of pain and the improved ability to drink, eat, and talk. If the mucosal lining is maintained, it would be important to establish if there are fewer infections, use of total parenteral nutrition (TPN) to maintain nutrition, and use of opiod patient-controlled analgesia (PCA) for pain control.