Nguyen, D.T., Shayani, S., Palmer, J., Dagis, A., Forman, S.J., Epstein, J., & Spielberger, R. (2015). Palifermin for prevention of oral mucositis in allogeneic hematopoietic stem cell transplantation: A single-institution retrospective evaluation. Supportive Care in Cancer, 23, 3141–3147. 

To evaluate possible differences between the severity and duration of oral mucositis (OM) and other clinical end points previously found to be associated with OM in patients who received palifermin compared to those who did not

A retrospective chart review of 99 consecutive patients who received an allogeneic hematopoietic stem cell transplantation (AHSCT) with palifermin from December 2006 to December 2009 was conducted, and 30 patients who received AHSCT from January to December 2005 served as control group (this group was treated before palifermin became standard of practice). Palifermin was administered for three consecutive days with the third dose 24 hours before the initiation of fractionated total body irradiation and three additional doses after the completion of the conditioning regimen (days 0–2). All patients received a comprehensive oral care regimen, and oral assessments were conducted every three days by a certified respiratory therapist.

• N = 129  
• AGE RANGE = 18–59.2 years
• MALES: 51%, FEMALES: 49%
• KEY DISEASE CHARACTERISTICS: Hematologic malignancies (acute lymphoblastic leukemia and acute myeloid leukemia)
• OTHER KEY SAMPLE CHARACTERISTICS: Conditioned with fractionated total body irradiation and etoposide

• SITE: Single site    
• SETTING TYPE: Not specified    
• LOCATION: Cancer center

• PHASE OF CARE: Active antitumor treatment
• APPLICATIONS: Elder care  

• Retrospective chart review

• National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v2.0
• OM grades 1 and 2 were categorized as mild, and grades 3 and 4 were categorized as severe.

OM developed in 95% of patients in the palifermin group and all 30 patients in the control group. Severe OM developed in 34% of the palifermin group and 80% of the control group (p < 0.0001) with the median duration of OM 13 days with palifermin and 18 days with the control. The median duration of sever OM was not found to be different between groups. The median duration patient-controlled analgesia use was shorter in the palifermin group as was the use of opioids. No significant difference in the duration of total parenteral nutrition use was found.

Palifermin reduced the incidence of severe OM and the overall duration of OM in this study. This supported the use of palifermin.

• Risk of bias (no blinding)
• Risk of bias (no random assignment)
• Risk of bias (sample characteristics)

This study supported palifermin use in this population. However, additional studies providing evidence on quality of life and patient-reported outcomes are needed.