Palifermin In High Dose Chemotherapy and Autologous Stem Cell Transplantation: Reduction of Infections and of Resources Utilization with No Increase in Overall Cost

Abstract 4568

Forty patients affected by various haematological malignancies who underwent high dose chemotherapy and autologous stem cell transplant (ASCT) were treated with Palifermin, this group was compared to 80 control subjects not assuming this drug. Controls were selected on the basis of being matched for length of neutropenia after ASCT and for Diagnosis. Primary end point was occurrence of “infections not CVC-related” (FUO/pneumonia/gram-negative bacteremia) and secondary end point was the “severe oral mucositis”.

Patients and controls resulted to be comparable for many pre-transplant and transplant features. Patients treated with Palifermin displayed a lower rate of “infections not CVC-related” when compared to controls (25% versus 50%, P=0.01) and the protective effect of Palifermin on this outcome remained significant also in a multiple logistic regression model [odds ratio: 0.34, 95% CI: 0.12–0.92, P=0.03] adjusting for a series of potential confounders. The odds ratio of severe mucositis was 30% lower in Palifermin treated patients than in controls (odds ratio: 0.70, 95% CI: 0.33–1.51) but this difference did not attain the statistical significance (P=NS). A stratified analysis by conditioning therapy showed that in patients who received Palifermin after BEAM/BU-CY conditioning, the proportion of patients experiencing severe mucositis was significantly lower than that observed in controls (14% versus 56%, P=0.008) while no such difference was found in those who underwent HD-PAM conditioning (65% versus 56%, P=NS). This result indicates that the conditioning therapy modifies the effect of Palifermin on severe mucositis, a finding fully confirmed in a multiple logistic regression model (P for the effect modification=0.018). Palifermin treated patients had also lower severe gastrointestinal toxicity (12% versus 65%, P<0.001), lower morphine utilization (12% versus 40%, P<0.001), lower total parental nutrition (10% versus 71%, P<0.001) and lower PLT and RBC transfusions (P=0.04) when compared to controls. Average economical costs related to the sum of some resources (inpatient stay, TPN, systemic antifungal treatment and blood products transfusions) were lower in Palifermin treated patients than in controls (11.985 EUROs versus 15.717 EUROs, P=0.002) so that the economical saving in Palifermin treated patients (about 3.700 EUROs) fully compensated for the cost of this drug.

Remarkably, in patients treated by “BEAM/BU-CY” conditioning therapy and receiving Palifermin, the overall costs (sum of the above mentioned resources and including also cost of the study drug) was lower in Palifermin treated group in respect to group of patients not treated. In fact, in BEAM/BU-CY stratum overall cost in group treated by Palifermin was 15990±2789 EUROs versus 19276±10946 EUROs in group treated by the same conditioning but not receiving Palifermin (P=0.11).

In conclusion this controlled study shows that Palifermin, after ASCT using conditioning not containing TBI, significantly reduces rate of “infections not related to CVC” and ameliorates several indicators of resource consumption, without economical overburden.