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

• Type of Material: Article
• Authors: Ajay Nooka, Emory University; Heather R. Johnson, Emory University; Jonathan Kaufman, Emory University; Christopher Flowers, Emory University; Amelia Langston, Emory University; Conor Steuer, Emory University; Michael Graiser, Emory University; Zahir Ali, Emory University; Nisha Shah, Emory University; Sravanti Rangaraju, Emory University; Dana Nickleach, Emory University; Jingjing Gao, Emory University; Sagar Lonial, Emory University; Edmund Waller, Emory University
• Language: English
• Date: 2014-06-01
• Publisher: Elsevier: 12 months
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2014 American Society for Blood and Marrow Transplantation.
• License: Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1016/j.bbmt.2014.02.025
• Title of Journal or Parent Work: Biology of Blood and Marrow Transplantation
• ISSN: 1083-8791
• Volume: 20
• Issue: 6
• Start Page: 852
• End Page: 857
• Grant/Funding Information: 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.; SOBI provided the funding for this study.
• Keywords: Hematology; Palifermin; Recombinant human keratinocyte growth factor; Science & Technology; THERAPY; DOUBLE-BLIND; Immunology; SEVERITY; IMPACT; RECIPIENTS; TRIAL; ECONOMIC OUTCOMES; Pharmacoeconomic analysis; ORAL MUCOSITIS; Transplantation; Mucositis; Life Sciences & Biomedicine; MULTIPLE-MYELOMA
• Research Categories: Health Sciences, Oncology; Biology, Biostatistics

Abstract

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.