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)

Eur J Cancer. 2012 Mar;48(5):713-20. doi: 10.1016/j.ejca.2011.12.016. Epub 2012 Jan 14.

Abstract

Aim: To evaluate in a multicentre randomised study the effect on duration of febrile neutropenia (FN), the safety and cost-effectiveness of a single subcutaneous pegfilgrastim injection compared with daily injections of filgrastim after peripheral blood stem cell transplantation in patients receiving high dose chemotherapy for myeloma and lymphoma.

Methods: Patients were randomly assigned to a single dose of pegfilgrastim at day 5 (D5) or daily filgrastim from D5 to the recovery of absolute neutrophil count (ANC) to 0.5 G/L. Duration of FN, of neutrophil and platelet recovery, transfusion and antibiotic requirements were the main end-points of the study. Costs were calculated from D0 until transplant unit discharge. The incremental cost-effectiveness ratio was expressed as the cost per day of FN prevented. Probabilistic sensitivity analysis was performed by non-parametric bootstrap methods.

Results: Between October 2008 and September 2009, 10 centres enrolled 151 patients: 80 patients with lymphoma and 71 patients with myeloma. The mean duration of FN was 3.07 days (standard deviation (SD) 1.96) in the pegfilgrastin arm and 3.29 (SD 2.54) in the filgrastim one. Mean total costs were 23,256 and 25,448 euros for pegfilgrastim and filgrastim patients, respectively. There was a 62% probability that pegfilgrastim strictly dominates filgrastim.

Concluding statement: Pegfilgrastim after PBSC transplantation in myeloma and lymphoma is safe, effective when compared with filgrastim and could represent a cost-effective alternative in this setting.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Combined Chemotherapy Protocols / economics
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Combined Modality Therapy
  • Cost-Benefit Analysis
  • Female
  • Fever / drug therapy
  • Fever / economics
  • Fever / etiology
  • Filgrastim
  • Granulocyte Colony-Stimulating Factor / economics
  • Granulocyte Colony-Stimulating Factor / therapeutic use*
  • Humans
  • Lymphoma / drug therapy
  • Lymphoma / surgery
  • Lymphoma / therapy*
  • Male
  • Middle Aged
  • Multiple Myeloma / drug therapy
  • Multiple Myeloma / surgery
  • Multiple Myeloma / therapy*
  • Neutropenia / drug therapy*
  • Neutropenia / economics
  • Neutropenia / etiology
  • Peripheral Blood Stem Cell Transplantation / adverse effects
  • Peripheral Blood Stem Cell Transplantation / economics
  • Peripheral Blood Stem Cell Transplantation / methods*
  • Polyethylene Glycols
  • Recombinant Proteins / economics
  • Recombinant Proteins / therapeutic use
  • Young Adult

Substances

  • Recombinant Proteins
  • Granulocyte Colony-Stimulating Factor
  • pegfilgrastim
  • Polyethylene Glycols
  • Filgrastim