title: Cost effectiveness of pegfilgrastim versus filgrastim after high-dose chemotherapy and autologous stem cell transplantation in patients with lymphoma and myeloma: an economic evaluation of the PALM Trial.
abstract: BACKGROUND
  Use of the recombinant human granulocyte colony-stimulating factor (rhG-CSF) filgrastim accelerates neutrophil recovery following myelosuppressive chemotherapy. Since filgrastim requires multiple daily administrations, forms of rhG-CSF with a longer half life, including pegfilgrastim, have been developed. Pegfilgrastim is safe and effective in supporting neutrophil recovery and reducing febrile neutropenia after conventional chemotherapy. Pegfilgrastim has also been successfully used to support patients undergoing peripheral blood stem cell (PBSC) transplantation for haematological malignancies. To our knowledge, no cost-effectiveness analysis (CEA) of pegfilgrastim in this setting has been published yet.
  
  
  OBJECTIVE
  We undertook a CEA to compare a single injection of pegfilgrastim versus repeated administrations of filgrastim in patients who had undergone PBSC transplantation for lymphoma or myeloma. The CEA was set in France and covered a period of 100 ± 10 days from transplant.
  
  
  METHODS
  The CEA was designed as part of an open-label, multicentre, randomized phase II trial. Costs were assessed from the hospital's point of view and are expressed in 2009 euros. Costs computation focused on inpatient, outpatient, and home care. Costs in the two arms of the study were compared using the Mann-Whitney test. When differences were statistically significant, multiple regression analyses were performed in order to identify cost drivers. Incremental cost-effectiveness ratios (ICER) were calculated for the major endpoints of the trial; i.e., duration of febrile neutropenia (absolute neutrophil count [ANC] <0.5 × 10(9)/L and temperature ≥38 °C), duration of neutropenia (ANC <1.0 × 10(9)/L and ANC <0.5 × 10(9)/L), duration of thrombopenia (platelets <50 × 10(9)/L and <20 × 10(9)/L), and days with a temperature ≥38 °C). Uncertainty around the ICER was captured by a probabilistic analysis using a non-parametric bootstrap method.
  
  
  RESULTS
  151 patients were enrolled at ten French centres from October 2008 to September 2009. The mean total cost in the pegfilgrastim arm of the study (n = 74) was <euro>25,024 (SD 9,945). That in the filgrastim arm (n = 76) was <euro>28,700 (SD 20,597). Pegfilgrastim strictly dominated filgrastim for days of febrile neutropenia avoided, days of neutropenia (ANC <1.0 × 10(9)/L) avoided, days of thrombopenia (platelets <20 × 10(9)/L) avoided, and days with temperature ≥38 °C) avoided. Pegfilgrastim was less costly and less effective than filgrastim for the number of days with ANC <0.5 × 10(9)/L avoided and the number of days with platelets <50.0 × 10(9)/L avoided. Taking uncertainty into account, the probabilities that pegfilgrastim strictly dominated filgrastim were 67 % for febrile neutropenia, 86 % for neutropenia (ANC <1.0 × 10(9)/L), 59 % for thrombopenia (platelets <20 × 10(9)/L), 86 % for temperature ≥38 °C, 32 % for neutropenia (ANC <0.5 × 10(9)/L), and 43 % for thrombopenia (platelets <50 × 10(9)/L). Conversely, the probability that filgrastim strictly dominated pegfilgrastim for neutropenia (ANC <0.5 × 10(9)/L) is 5 %.
  
  
  CONCLUSIONS
  This study found no evidence that the use of pegfilgrastim is associated with greater cost in lymphoma and myeloma patients after high-dose chemotherapy and PBSC transplantation.
authors: Department Cancer and Environment, Cancer Centre Léon Bérard, University of Lyon, GATE Lyon-St Etienne, UMR-CNRS 5824, 28 rue Laënnec, 69373, Lyon Cedex 08, France. lionel.perrier@lyon.unicancer.fr
authors: Lionel
authors: L
authors: Perrier
authors: Anne
authors: A
authors: Lefranc
authors: David
authors: D
authors: Pérol
authors: Philippe
authors: P
authors: Quittet
authors: Aline
authors: A
authors: Schmidt-Tanguy
authors: Carole
authors: C
authors: Siani
authors: Christian
authors: C
authors: de Peretti
authors: Bertrand
authors: B
authors: Favier
authors: Pierre
authors: P
authors: Biron
authors: Philippe
authors: P
authors: Moreau
authors: Jacques Olivier
authors: JO
authors: Bay
authors: Séverine
authors: S
authors: Lissandre
authors: Fabrice
authors: F
authors: Jardin
authors: Daniel
authors: D
authors: Espinouse
authors: Catherine
authors: C
authors: Sebban
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000191:economics / Q000627:therapeutic use; D003362:Cost-Benefit Analysis; D016527:Drug Costs; D004358:Drug Therapy / Q000191:economics; D005260:Female; D000069585:Filgrastim; D005602:France; D016179:Granulocyte Colony-Stimulating Factor / Q000191:economics* / Q000627:therapeutic use*; D006801:Humans; D008223:Lymphoma / Q000191:economics / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000191:economics / Q000628:therapy*; D011092:Polyethylene Glycols; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000191:economics / Q000627:therapeutic use; D033581:Stem Cell Transplantation / Q000191:economics*
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; C455861:pegfilgrastim; D011092:Polyethylene Glycols; D000069585:Filgrastim