Prior trials have shown benefits ofpalifermin 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 outcomes 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. 524 patients that underwent autologous HSCT for myeloma (melphalan 200 mg/m2) and lymphoma (high-dose busulfan, cyclophosphamide, and etoposide)as preparative regimen between January 2002 and December 2010 were analyzed. Usage of patient controlled analgesia (PCA) was significantly lower in the palifermin-treated groups (myeloma: 13% vs. 53%; p<0.001; lymphoma: 46% vs. 68%; p<0.001).Median total transplant charges were significantly higher in the palifermin-treated group, after controlling for inflation (myeloma: $167,820 vs. $143,200, p<0.001; lymphoma: $168,570 vs. $148,590, p<0.001). 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.
BACKGROUND
One proposed mechanism of extracorporeal photopheresis (ECP) in reducing chronic graft-versus-host disease (cGVHD) is alteration in numbers of circulating dendritic cells (DCs). This hypothesis was tested by correlating numbers of DC precursors and T cells in the blood before and during ECP therapy with response of cGVHD.
STUDY DESIGN AND METHODS
Twenty-five patients with cGVHD were treated with ECP. Data were collected with emphasis on blood cellular markers, clinical response to ECP, and overall survival.
RESULTS
Fourteen patients (56%) responded and had better 2-year survival than nonresponders (88% vs. 18%, p = 0.003). Responders had higher baseline circulating myeloid DC (mDC) and plasmacytoid DC precursors and CD4+ and CD8+ T cells compared with nonresponders. Receiver operating characteristic curve analyses showed that the best baseline cutoff values to predict response to ECP were mDC counts of 3.7 cells/µL (79% sensitivity, 82% specificity) and CD4+ T-cell counts of 104 cells/µL (71% sensitivity, 82% specificity). CD4+ T cells declined in responders over time, but not in nonresponders, and no significant changes were seen in CD8 T-cell or DC numbers over a 12-month period in responder or nonresponder groups.
CONCLUSIONS
Higher baseline numbers of circulating DCs and T cells may predict clinical response to ECP in patients with cGVHD.
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