Raphael, M.F., den Boer, A.M., Kollen, W.J., Mekelenkamp, H., Abbink, F.C., Kaspers, G.J., . . . Tissing, W.J. (2014). Caphosol, a therapeutic option in case of cancer therapy-induced oral mucositis in children?: Results from a prospective multicenter double blind randomized controlled trial. Supportive Care in Cancer, 22, 3–6.

To evaluate if Caphosol™ is effective to treat oral mucositis (OM) in pediatric patients who received chemotherapy or hematopoietic stem cell transplant (HSCT)

A sample of 33 patients between 4–18 years old was assigned to Caphosol or placebo. All patients received standard local supportive care, in addition to Caphosol study or 0.9% sodium chloride (NaCl) placebo mouth rinse. All patients were instructed to use the study mouthwash four times daily during their OM period. Primary study outcome was defined as the number of days with OM greater than grade 1. Secondary outcomes were pain and analgesic use.

• N = 29
• AGE RANGE: 4–18 years
• MALES: 66%, FEMALES: 34%
• KEY DISEASE CHARACTERISTICS: Patients had been diagnosed with hematologic malignancies, solid tumor, and benign hematologic disorders and were receiving chemotherapy or HSCT.

• SITE: Multi-site
• SETTING TYPE: Inpatient
• LOCATION: Four university hospitals in the Netherlands

• PHASE OF CARE: Active antitumor treatment
• APPLICATIONS: Pediatrics

• Double-blinded, placebo-controlled trial

• The National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 3.0 mucositis scoring system was used to evaluate mucositis.
  • Days of mucositis greater than grade 1 and peak level of mucositis
  • Days of pain and peak of level pain
  • Days of analgesic use, morphine use, peak dose (mg/kg)
  • Need for tube feeding and number of days
  • Need for parenteral feeding and number of days
  • Blood cultures taken

Chi-square or t-test was used for analysis. The number of days with mucositis greater than grade 1 did not differ significantly between the two study groups (p = 0.154). No significant differences were found between Caphosol and placebo for all the outcome measures except days of pain and tube feeding requirement. Placebo was associated with significantly fewer days of pain (p = 0.035) . The need for tube feeding was significantly higher in the Caphosol group.

Therapeutic use of Caphosol was not beneficial in the treatment of pediatric patients with cancer therapy-induced OM.

• Small sample (< 30)
• Risk of bias (sample characteristics)
• Key sample group differences could influence results.
• Findings not generalizable
• High grades of mucositis were found at the start of the study with 64.3 % of the placebo group versus 33.3 % of the patients with Caphosol having greater than grade 1 mucositis at the start of the study (p = 0.143). The placebo group also showed a trend of shorter mucositis duration, which may suggest that mucositis was more advanced at baseline and resolved earlier. This, in turn, may have contributed to the finding that the placebo group had fewer days with mucositis greater than grade 1.
• A problem was seen with a power calculation, and many analyses failed to demonstrate significant results. This study has significant risk for type II errors. In addition, this study included various types of cancer and cancer treatments, which may have contributed to the lack of significance in findings.

Although this study was a double-blind, randomized, controlled trial, the significantly small sample size was problematic. The authors concluded that Caphosol was not effective in practice; however, it is not conclusive because of the significantly high risk for type II errors. A study with a larger sample size is required to assess the efficacy of Caphosol.