Nasilowska-Adamska, B., Rzepecki, P., Manko, J., Czyz, A., Markiewicz, M., Federowicz, I., … Marianska, B. (2007). The influence of palifermin (Kepivance) on oral mucositis and acute graft versus host disease in patients with hematological diseases undergoing hematopoietic stem cell transplant. Bone Marrow Transplantation, 40, 983–988.

To assess the use of palifermin in the prevention of oral mucositis (OM) and acute graft-versus-host disease (GVHD) after hematopoietic stem cell transplant (HSCT)

IV palifermin was administered at 60 mcg/kg for three consecutive days before and after conditioning therapy. These patients were compared to a retrospective control group.

• The study reported on a total of 106 patients, with 53 in the palifermin group and 53 in the control group.
• All patients received autologous or allogeneic transplantation with hematologic and nonhematologic diseases and had a Karnofsky score of 80 or higher.
• The palifermin group received transplantations between June 2005 and March 2006; the control group received transplantations between December 2000 and December 2005.
• Protocols were not indicated for either group.

This was a multicenter study conducted in Poland.

This was a retrospective control trial.

• The World Health Organization (WHO) Oral Toxicity Scale was used.
• Researchers recorded mucositis onset and incidence, use of analgesics, duration of total parenteral nutrition (TPN), incidence of febrile neutropenia, severe infections, and incidence of acute GVHD.

Incidence of all grades of mucositis was lower in the palifermin group (p < 0.001). Incidence of grades 3–4 was 13% in the palifermin group and 43% in the control group (p < 0.001). Mean duration was significantly lower (p < 0.001). No statistically significant differences in the onset of OM, duration of TPN, opioid use, incidence of febrile neutropenia, or severe infection were observed. No statistical significance in acute GVHD measures were observed, although the authors suggested that a decrease in acute GVHD may occur. Additional studies are necessary.

Adverse events (e.g., rash, pruritis, erythema, generalized edema, taste alteration, mouth or tongue thickness and discoloration, proteinuria) were mild in 15 patients, moderate in 15 patients, and severe but not life-threatening in 4 patients. No events caused discontinuation of palifermin.

• This was a retrospective trial.
• Expense was not addressed.
• The study used an IV formulation of palifermin.
• Ensuring all treatments were equal is not possible because of the study’s historical nature.