Spielberger, R., Stiff, P., Bensinger, W., Gentile, T., Weisdorf, D., Kewalramani, T., … Emmanouilides, C. (2004). Palifermin for oral mucositis after intensive therapy for hematologic cancers. New England Journal of Medicine, 351(25), 2590–2598.

IV palifermin (recombinant human keratinocyte growth factor) 60 mg/kg was given for three consecutive days immediately before a conditioning regimen (total body irradiation [TBI] plus high-dose [HD] chemo). Additional doses administered on days 0, 1, and 2.

• The study reported on 212 patients with a median age of 48.5 years and an age range of 18–69 years.
• Patients were stratified according to the center and type of cancer; 106 were given palifermin and 106 received placebo.
• All patients had hematologic cancers and were receiving autologous stem cell transplant (SCT).

The study was conducted in a multisite setting.

This was a placebo-controlled, double-blind, phase 3, randomized trial.

• The World Health Organization (WHO) oral toxicity scale was used.
• The Radiation Therapy Oncology Group (RTOG) acute radiation-morbidity scoring criteria for mucous membranes was used.
• The Western Consortium for Cancer Nursing Research (WCCNR) revised staging system for oral mucositis was used.
• Use of analgesia and total parenteral nutrition (TPN) was recorded.
• Duration of mucositis was recorded.
• Incidence of mucositis was recorded.
• Mucositis was evaluated for 28 days.

• Fewer patients in the palifermin group had WHO grades 3 or 4 (63%) than in the control group (98%) (p < 0.001).
• Fewer patients in the palifermin group had WHO grades 4 (20%) than in the control group (62%) (p < 0.001).
• Patients in the palifermin group had a lower median duration of mucositis at 6 days versus 9 days in the control group (p < 0.001).
• Fewer patient in the palifermin group reported soreness of mouth and throat (29%) than in the control group (46.8%) (p < 0.001).
• Patients in the palifermin group reported lower use of opioids (212 mg morphine equivalents) compared to the control group (535 mg morphine equivalents) (p < 0.001).
• Incidence of TPN was lower in the palifermin group (31%) than the control group (55%) (p < 0.001).
• Adverse events were rash, pruritus, erythema, mouth and tongue disorders, and taste alteration.

Patients in the palifermin group experienced statistically significant decreases in incidence and duration of mucositis.

• One or more of the study researchers had a relationship with AMGEN, which manufactures palifermin.
• The study only looked at a hemotologic autologous SCT population.
• The trial only studied the IV formulation.
• Palifermin costs may be a limitation.