SINGLE CENTER PHARMACOECONOMIC ANALYSIS OF PALIFERMIN IN AUTOLOGOUS STEM CELL TRANSPLANTATION 公开
Johnson, Heather Renfroe (2013)
Abstract
BACKGROUND: Randomized clinical trials have shown palifermin reduces the incidence and severity of oral mucositis and its subsequent outcomes in patients with hematological malignancies receiving TBI-based conditioning regimens and autologous hematopoietic stem cell transplantation (HSCT). However, similar outcomes data for patients receiving non-TBI based conditioning is inconclusive. Our objective was to determine whether clinical and health care resource outcomes were different between patients who received palifermin and those who did not in the setting of HSCT following non-TBI based conditioning.
METHODS: Patient data was retrospectively obtained on 524 consecutive patients with multiple myeloma (MM) or lymphoma who received autologous HSCT with melphalan 200 mg/m² or high-dose busulfan, cyclophosphamide, and etoposide conditioning between January 2002 and December 2010. Patients were stratified by diagnosis and multivariate analysis using generalized linear models was conducted for each outcome to compare treatment groups. Models were adjusted for differences in baseline characteristics.
RESULTS: The analyses included 254 MM patients (162 palifermin, 92 control) and 270 lymphoma patients (167 palifermin, 103 control). PCA incidence was significantly lower in the palifermin-treated groups (MM: 13% vs. 53%, P<0.001; lymphoma: 46% vs. 68%, P<0.001). Similarly, the median duration of PCA use was significantly shorter among the palifermin group compared to the control group (MM: 0 days vs. 3 days, lymphoma: 0 days vs. 5 days). Palifermin treatment was not associated with a difference in overall survival (OS), days to neutrophil engraftment, or length of stay (LOS). The mean total transplant charges were significantly higher in the palifermin-treated group, after controlling for inflation (MM: $175K vs. $158K, P<0.001; lymphoma: $188K vs. $159K, P<0.001).
CONCLUSION: In patients receiving non-TBI based conditioning regimens and autologous HSCT, palifermin significantly decreases PCA use, but significantly increases total charges associated with autologous HSCT. Palifermin administration was associated with an additional cost (as charges) of $11K (MM) and $15K (lymphoma) per day of PCA use (severe pain) avoided. Future research is suggested to evaluate the cost-effectiveness of palifermin use compared with other symptomatic treatments that reduce suffering without a direct effect on survival using validated measures for quality of life and pain symptoms.
Table of Contents
ACKNOWLEDGEMENTS....................................................................... 6 INTRODUCTION................................................................................ 7 METHODS ...................................................................................... 9 RESULTS....................................................................................... 11 DISCUSSION.................................................................................. 13 FINANCIAL DISCLOSURE STATEMENT.................................................. 16 REFERENCES.................................................................................. 17 TABLES......................................................................................... 19 FIGURES........................................................................................ 23
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