Blijlevens, N., de Chateau, M., Krivan, G., Rabitsch, W., Szomor, A., Pytlik, R., … Niederwieser, D. (2013). In a high-dose melphalan setting, palifermin compared with placebo had no effect on oral mucositis or related patient's burden. Bone Marrow Transplantation, 48, 966–971.

DOI Link

Study Purpose

To evaluate efficacy of palifermin versus placebo for prevention of oral mucositis (OM), as well as burden of patients with multiple myeloma (MM) who receive autologous stem cell transplant (SCT)

Intervention Characteristics/Basic Study Process

This randomized study compared three groups: (1) placebo, (2) palifermin on days -6, -5, -4, 0, 1, and 2 (pre-/post-SCT), and (3) palifermin on days 0, 1, and 2 (post-SCT). The palifermin dose was 60 µg/kg per day IV. Patients were assessed daily for OM from day 2 until day 32 or discharge.

Sample Characteristics

  • The study reported on 257 patients with MM.
  • Median age was 57 years old with a range of 32–69 years.
  • The placebo group sample was 58% male and 42% female; the group 2 sample was 55% male and 45% female; and the group 3 sample was 54% male and 46% female.
  • All patients were receiving autologous SCT and high-dose melphalan.

Setting

This was a multisite, inpatient study conducted in the Netherlands.

Phase of Care and Clinical Applications

  • Patients were undergoing the active antitumor treatment phase of care. 
  • This study has clinical applicability for elder care.

Study Design

This was a randomized, placebo-controlled, parallel-group study.

Measurement Instruments/Methods

  • The World Health Organization (WHO) scale for OM, the Oral Mucocitis Daily Questionnaire, and the Quality of Life Utility Scale were used.
  • Investigators recorded the use of opioids, non-opioids, days of severe OM, incidence of infections, time to absolute monocyte count (AMC) recovery, and adverse events.

Results

  • No statistically significant differences were found in maximum OM severity. Severe OM occurred in 37% of patients in the placebo group, 38% in the pre-/post-group (group 2), and 24% in the pre- group (group 3).
  • No significant differences were observed with respect to PRO assessments or medical resource use, but more infections and fever during neutropenia were reported in group 2 versus the placebo group (51% versus 26%).
  • No significant differences were found across groups in incidence of ulcerative OM or duration of OM. Those on palifermin pre-transplant had significantly lower incidence of opioid analgesic use compared to placebo (p = 0.03).

Conclusions

Palifermin was unable to reduce OM or OM-related patient burden in patients with MM undergoing transplant.

Limitations

  • A risk of bias exists because no blinding was done.
  • Unintended interventions or applicable interventions that could have influenced results were not described.
  • Findings are not generalizable.
  • The authors indicated that the short period of intervention time or timing of giving palifermin may have had a suboptimal effect with palifermin. The authors also suggested that hyperkeratosis may have been misinterpreted for mucositis, despite observer training in assessment.

Nursing Implications

Short term of use of palifermin for auto-SCT in patients with MM undergoing transplant was not effective in reducing OM. The fact that authors suggest that hyperkeratosis may have been incorrectly interpreted as OM suggests that correct assessment can be an issue in evaluating this symptom. Findings suggest that the specific timing of use of this agent may be critical. In using palifermin, nurses need to be aware of the appropriate timing in concert with timing of antineoplastic treatment and treatment effects.