Lucchese, A., Matarese, G., Ghislanzoni, L.H., Gastaldi, G., Manuelli, M., & Gherlone, E. (2016). Efficacy and effects of palifermin for the treatment of oral mucositis in patients affected by acute lymphoblastic leukemia. Leukemia and Lymphoma, 57, 820–827. 

To study the efficacy of palifermin for prophylaxis of OM in pediatric patients undergoing hematopoetic cell transplant (HCT)

Patients were randomly assigned to receive either IV palifermin 60 mcg/kg per day starting three days prior to transplant conditioning chemotherapy or placebo. All patients received basic oral hygiene and invasive nutrition as needed. All other aspects of care were essentially the same between groups. OM was assessed daily from 7 days prior to transplantation to 28 days after transplantation. Assessment was conducted by the same clinician.

• N = 54
• MEDIAN AGE = 11 years
• AGE RANGE = 7–16 years
• MALES: 48%, FEMALES: 52%
• KEY DISEASE CHARACTERISTICS: All had lymphoblastic leukemia and were undergoing HCT.

• SITE: Single site  
• SETTING TYPE: Inpatient  
• LOCATION: Italy

• PHASE OF CARE: Active antitumor treatment

• Placebo-controlled randomized controlled trial

• Oral Mucositis Daily Questionnaire (OMDQ)
• World Health Organization (WHO) Oral Mucositis Toxicity Scale

Significantly fewer patients in the palifermin group developed grade 2 (p = 0.038) and grade 4 (p = 0.006) mucositis compared to the placebo group. Grade 4 was seen in 11% of patients in the palifermin group and 59% of patients in the control group. Fewer patients in the palifermin group had any grade mucositis, but not all differences in grades were statistically significant. The duration of grade 3 and 4 OM in patients in the palifermin group was three days compared to eight days in the control group (p < 0.001). The only adverse reactions reported were rashes and altered taste, lasting for 48–72 hours. The cumulative morphine daily dose per body weight was lower in the palifermin group (p = 0.04).

Palifermin was shown to effectively reduce the severity, prevalence, and duration of OM among pediatric patients undergoing HCT and was not associated with any significant adverse effects.

• Small sample (< 100)
• Risk of bias (no blinding)

Palifermin was shown to be beneficial to pediatric patients undergoing HCT for primary prophylaxis for OM. OM is one of the most painful and debilitating effects of high-dose myeloablative chemotherapy. Nurses can advocate for the consideration of palifermin in these patients. Additional research in support of these findings is warranted, and research regarding the potential role of palifermin in other types of chemotherapy regimens associated with OM is needed.