Pharmacoeconomic analysis of palifermin to prevent mucositis among patients undergoing autologous hematopoietic stem cell transplantation

Ajay K. Nooka, Heather R. Johnson, Jonathan L. Kaufman, Christopher R. Flowers, Amelia Langston, Conor Steuer, Michael Graiser, Zahir Ali, Nishi N. Shah, Sravanti Rangaraju, Dana Nickleach, Jingjing Gao, Sagar Lonial, Edmund K. Waller

Research output: Contribution to journalArticlepeer-review

23 Scopus citations


Trials have shown benefits of palifermin in reducing the incidence and severity of oral mucositis in patients with hematological malignancies undergoing autologous hematopoietic stem cell transplantation (HSCT) with total body irradiation (TBI)-based conditioning regimens. Similar outcome data are lacking for patients receiving non-TBI-based regimens. We performed a retrospective evaluation on the pharmacoeconomic benefit of palifermin in the setting of non-TBI-based conditioning and autologous HSCT. Between January 2002 and December 2010, 524 patients undergoing autologous HSCT for myeloma (melphalan 200 mg/m) and lymphoma (high-dose busulfan, cyclophosphamide, and etoposide) as preparative regimen were analyzed. Use of patient-controlled analgesia (PCA) was significantly lower in the palifermin-treated groups (myeloma: 13% versus 53%, P < .001; lymphoma: 46% versus 68%, P < .001). Median total transplant charges were significantly higher in the palifermin-treated group, after controlling for inflation (myeloma: $167,820 versus $143,200, P < .001; lymphoma: $168,570 versus $148,590, P < .001). Palifermin treatment was not associated with a difference in days to neutrophil engraftment, length of stay, and overall survival and was associated with an additional cost of $5.5K (myeloma) and $14K (lymphoma) per day of PCA avoided. Future studies are suggested to evaluate the cost-effectiveness of palifermin compared with other symptomatic treatments to reduce transplant toxicity using validated measures for pain and quality of life.

Original languageEnglish (US)
Pages (from-to)852-857
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Issue number6
StatePublished - Jun 2014
Externally publishedYes


  • Mucositis
  • Palifermin
  • Pharmacoeconomic analysis
  • Recombinant human keratinocyte growth factor

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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