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Author Notes:

Address correspondence to: Ajay K. Nooka MD MPH FACP Assistant Professor, Department of Hematology and Medical Oncology Winship Cancer Institute of Emory University 1365 Clifton Road NE, C4010, Atlanta, GA 30322 anooka@emory.edu Phone: 404-778-5530.

AKN, HRJ, EKW conceived the study, and reviewed data and writing of the manuscript; JLK, SL, AKNtreated patients with myeloma, and reviewed data and writing of the manuscript; CRF, ALtreated patients with lymphoma, and reviewed data and writing of the manuscript;CS, MG, ZA, NS, SR collected the data, and reviewed data and writing of the manuscript; DN, JG, HRJ performed statistical analysis.

The authors have no conflict of interest to disclose.

SOBI representatives had no input into the data analysis or conclusions.

The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.


Research Funding:

SOBI provided the funding for this study.

Research reported in this publication was supported in part by the Biostatistics & Bioinformatics Shared Resource of Winship Cancer Institute of Emory University and NIH/NCI under award number P30CA138292.


  • Science & Technology
  • Life Sciences & Biomedicine
  • Hematology
  • Immunology
  • Transplantation
  • Palifermin
  • Recombinant human keratinocyte growth factor
  • Mucositis
  • Pharmacoeconomic analysis

Pharmacoeconomic Analysis of Palifermin to Prevent Mucositis among Patients Undergoing Autologous Hematopoietic Stem Cell Transplantation

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Journal Title:

Biology of Blood and Marrow Transplantation


Volume 20, Number 6


, Pages 852-857

Type of Work:

Article | Post-print: After Peer Review


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.

Copyright information:

© 2014 American Society for Blood and Marrow Transplantation.

This is an Open Access work distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (http://creativecommons.org/licenses/by-nc-nd/4.0/).

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