Kim, J.W., Kim, M.G., Lee, H.J., Koh, Y., Kwon, J.H., Kim, I., . . . Yoon, S.S. (2017). Topical recombinant human epidermal growth factor for oral mucositis induced by intensive chemotherapy with hematopoietic stem cell transplantation: Final analysis of a randomized, double-blind, placebo-controlled, phase 2 trial. PLOS ONE, 12, e0168854. 

To evaluate the efficacy and safety of recombinant human epidermal growth factor (rhEGF) oral spray for the prevention of oral mucositis

Patients were randomly assigned to rhEGF or placebo oral spray. The spray was applied to the entire oral mucosa twice daily from the first day of conditioning chemotherapy until neutrophil recovery. The spray was used six times for each application. Patients were not to eat or drink for 30 minutes after application. The severity of oral mucositis was recorded daily.

• N = 136   
• MEAN AGE = 52 years
• AGE RANGE = 18–65 years
• MALES: 51.5%, FEMALES: 48.5%
• CURRENT TREATMENT: Combination radiation and chemotherapy
• KEY DISEASE CHARACTERISTICS: Patients undergoing hematopoietic cell transplantation (HCT). Most had lymphoma or multiple myeloma. Ninety-six percent had autologous HCT.

• SITE: Single site   
• SETTING TYPE: Inpatient    
• LOCATION: Republic of Korea

PHASE OF CARE: Active antitumor treatment

Double-blind, placebo-controlled, randomized, controlled trial

• Common Terminology Criteria for Adverse Events (CTCAE), version 3.0
• Oral Mucositis Daily Questionnaire

No significant difference existed between groups in the incidence of mucositis grade 2 or higher. Subgroup analysis by specific chemotherapy used also did not show any difference. Patients in the study group received less cumulative dose of opioids (p = 0.046) and for a shorter duration (p = 0.036) than those in the control group. No differences between groups in adverse events were reported.

rhEGF oral spray did not reduce the incidence of grade 2 or higher mucositis. This might have a beneficial effect in terms of the reduction of pain associated with oral mucositis.

rhEGF was not shown to have a preventive effect for oral mucositis among patients receiving high-dose chemotherapy prior to HCT. The oral spray examined here may have some positive effect for pain reduction. Additional research is needed to evaluate this potential use.