Sebban, C., Lefranc, A., Perrier, L., Moreau, P., Espinouse, D., Schmidt, A., . . . Quittet, P. (2012). A randomised phase II study of the efficacy, safety and cost-effectiveness of pegfilgrastim and filgrastim after autologous stem cell transplant for lymphoma and myeloma (PALM study). European Journal of Cancer (Oxford, England: 1990), 48, 713–720.

To compare the efficacy and cost-effectiveness of a single dose of pegfilgrastim to daily injections of filgrastim after peripheral blood stem cell transplant (PBSCT).

Patients were randomized to receive either a single 6-mg dose of pegfilgrastim five days after PBSCT reinfusion or daily 5-µg/kg doses of filgrastim from day 5 posttransplantation until the resolution of neutropenia.  Patients were followed for 100 days after transplantation.  Complete blood cell counts were obtained daily during hospitalization and every two weeks thereafter.

• One hundred fifty patients (57.3% male, 42.7% female) were included.
• Median age was 55 to 59 years (range 19–75) across groups.
• All patients had either lymphoma or multiple myeloma; 50% underwent transplantation as a first-line therapy and 98% were either in complete or partial remission. 
• Patient conditioning was done without any full body radiation.

• Multi-site  
• Multiple settings 
• France

Patients were undergoing the active antitumor treatment phase of care.

This was a randomized, unblinded, open-label study.

• Duration of febrile neutropenia, absolute neutrophil count (ANC) <0.5 G/L, and temperature >38°C
• National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 3.0
• Mean hospital day costs and drug costs across participating centers were used for cost-effectiveness analysis.  Differences in cost and effectiveness were plotted per patient to determine incremental cost ratios.

Patients on pegfilgrastim had a shorter duration of ANC <1 g/l, fewer days with fever, shorter hospital stay, and fewer days of antibiotic therapy.  Significant differences for individual outcomes were not analyzed but were incorporated in the cost-effectiveness analysis.  Severe mucositis was observed in 25% of patients taking pegfilgrastim versus 20% of those taking filgrastim.  Mean total cost with pegfilgrastim was $32,326 compared to $35,373 with filgrastim.  Cost-effectiveness analysis showed that the probability that pegfilgrastim is less costly and more effective than filgrastim is 62%.

Findings suggested that there is no substantial cost difference between a single dose of pegfilgrastim versus daily filgrastim in patients undergoing PBSCT and that the pegfilgrastim regimen may be more cost-effective.

• Risk of bias (no blinding)
• Unintended interventions or applicable interventions not described that would influence results*

* Although the costs of antifungals and antibiotics were provided, no information was provided regarding whether routine prophylaxis was also provided to patients in the sample.

Both of these colony-stimulating factor preparations have been shown to be effective.  This study provided some initial information that the single dose regimen of pegfilgrastim is more cost-effective.  The need to give fewer injections to achieve the same result can be of benefit to patients.  Further evidence to confirm the findings of this study would be beneficial.  Additional study regarding the most cost-effective formulations and schedules of these agents is warranted.