title: EQOFIX: a combined economic and quality-of-life study of hemophilia B treatments in France.
abstract: BACKGROUND
  EQOFIX is a medicoeconomic study that analyzed the health-related quality of life (HRQoL) and costs of care of the moderate and severe forms of hemophilia B, treated on demand or by prophylaxis with either plasma-derived Factor IX (pdFIX) or recombinant FIX (rFIX).
  
  
  METHODS
  The primary objectives were evaluations of the impact of hemophilia B on HRQoL and of the costs associated with its management. The secondary objectives were evaluations of the clinical efficacy and costs of care of pdFIX and rFIX. In this observational study we included and followed for 1 year severe and moderate hemophilia B patients without inhibitor. HRQoL was evaluated through generic and disease-specific questionnaires. Information on the health resources consumed was collected every 3 months.
  
  
  RESULTS
  The EQOFIX cohort was composed of 155 patients, including 51 children and 104 adults, with 114 having severe disease and 41 having moderate disease. The regimens were prophylactic for 61 and on demand for 94. Altogether, 78 were treated with rFIX and 77 with pdFIX. There was no difference in the QoL between the pdFIX and rFIX treatments. The extra cost of prophylaxis was €22,605 per bleeding event prevented. The consumption of FIX was 1.4-fold higher for the patients treated with rFIX than for the patients treated with pdFIX.
  
  
  CONCLUSIONS
  Our findings in a cohort composed of 25% of the French population of moderate and severe hemophilia B patients show, with similar clinical and HRQoL results, that treatment with rFIX is more expensive than treatment with pdFIX.
authors: Department of Hematology, University Hospital, and CNRS UMR5525, Grenoble-Alpes University, Grenoble, France.
authors: Benoît
authors: B
authors: Polack
authors: Sorbonne University, UPMC University of Paris 06, INSERM, UMR-S 1136, Pierre Louis Institute of Epidemiology and Public Health, Paris, France.
authors: Thierry
authors: T
authors: Calvez
authors: APHM, Department of Pediatric Hematology, Children Hospital La Timone, and INSERM, UMR 1062, Aix-Marseille University, Marseille, France.
authors: Hervé
authors: H
authors: Chambost
authors: Regional Hemophilia Center, Department of Hematology, University Hospital Necker Enfants Malades, Paris, France.
authors: Chantal
authors: C
authors: Rothschild
authors: Department of Hematology-Transfusion, University Hospital, Lille, France.
authors: Jenny
authors: J
authors: Goudemand
authors: Regional Hemophilia Center, Purpan Hospital, Toulouse, France.
authors: Ségolène
authors: S
authors: Claeyssens
authors: Regional Hemophilia and VWD Center, University Hospital, and INSERM U919, Caen, France.
authors: Annie
authors: A
authors: Borel-Derlon
authors: Department of Health Economics and Outcome Research, IMS Health, La Défense, France.
authors: Isabelle
authors: I
authors: Bardoulat
authors: Department of Health Economics and Outcome Research, IMS Health, La Défense, France.
authors: Frédérique
authors: F
authors: Maurel
authors: Pharmacy, University Hospital, and INSERM UMR1098, Franche-Comté University, Besançon, France.
authors: Marie-Christine
authors: MC
authors: Woronoff-Lemsi
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D015331:Cohort Studies; D003365:Costs and Cost Analysis; D005164:Factor IX / Q000008:administration & dosage / Q000191:economics; D005260:Female; D005602:France; D002836:Hemophilia B / Q000188:drug therapy / Q000191:economics; D006470:Hemorrhage / Q000191:economics / Q000517:prevention & control; D006801:Humans; D008297:Male; D008875:Middle Aged; D011788:Quality of Life
chemical_list: D005164:Factor IX