Shea, T.C., Kewalramani, T., Mun, Y., Jayne, G., & Dreiling, L.K. (2007). Evaluation of single-dose palifermin to reduce oral mucositis in fractionated total-body irradiation and high-dose chemotherapy with autologous peripheral blood progenitor cell transplantation. Journal of Supportive Oncology, 5(4, Suppl. 2), 60–61.

To evaluate the safety and efficacy of single-dose palifermin in reducing oral mucositis incidence in patients with hematologic malignancies undergoing peripheral blood progenitor cell transplant receiving total-body irradiation and high-dose chemotherapy 

Patients were randomized to receive 60 mcg/kg palifermin once daily for three days before the start of fractionated total-body irradiation (TBI) (Arm A), 180 mcg/kg once only on day -1 (Arm B), day -2 (Arm C), or day -3 (Arm D) before starting fractionated TBI and stratified by etoposide use and number of days of TBI.  All patients received 60 mcg/kg for three days post-transplantation.

The study reported on 47 patients receiving high-dose chemotherapy with an age range of 18–74 years. Patients had multiple cancer diagnoses.

The World Health Organization (WHO) Oral Toxicity Scale was used.

• All arms had similar adverse effects.
• Overall incidence of severe mucositis was 61% with a mean of 4.3 days.
  • Arm A: 82% (70% actual)
  • Arm B: 60% (73% actual)
  • Arm C: 31% (38% actual)
  • Arm D: 75% (67% actual)

• The four study arm sample sizes were small.
• The total sample was small with only 47 patients total.
• The authors collapsed Arms B, C, and D for severe mucositis.
• A large percentage (n = 20) of patients had significant protocol deviations.
• The study was stopped early; participant accrual was difficult.
• The report did not include a discussion of costs.