title: Iron chelation therapy: clinical effectiveness, economic burden and quality of life in patients with iron overload.
abstract: BACKGROUND
  This study of UK patients examines clinical, health-related quality of life (HRQOL) and economic outcomes associated with iron chelation therapy (ICT). Desferrioxamine (DFO) (Desferal; Novartis, Switzerland) and Deferiprone (Ferriprox; Apotex, Canada) are ICTs used to treat iron overload. DFO requires 8-to 12-hour infusions a minimum of five times per week. Deferiprone is administered in an oral daily regimen. Although pharmacologically efficacious, clinical effectiveness of ICT within the real-world setting is yet to be fully elucidated.
  
  
  METHODS
  A naturalistic cohort study of 60 patients (beta-thalassaemia, n=40; sickle cell disease, n=14; myelodysplastic syndromes, n=6; 63% female) receiving ICT in four UK treatment centres was conducted. Serum ferritin level data were abstracted from medical charts. Compliance, HRQOL, satisfaction and resource utilisation data were collected from interviews. Maximum ICT costs were estimated using the resource utilisation data associated with DFO.
  
  
  RESULTS
  Mean serum ferritin levels, generally, remained elevated despite ICT. Compliance was suboptimal and HRQOL scores were lower than population norms. The total estimated mean weighted annual per-patient cost of DFO treatment was approximately pound19,000. DFO-related equipment, DFO drug, and home healthcare were estimated to account for 43%, 19% and 24% of costs, respectively. Other more minor components of total annual costs were for in-patient infusions, ICT home delivery services and monitoring costs.
  
  
  CONCLUSIONS
  Generally, patients are not achieving target serum ferritin thresholds despite chronic treatment for iron overload. ICT appears to negatively impact HRQOL; compliance with ICT is poor; and, in the case of DFO, treatment costs well exceed the cost of DFO alone. These results suggest that current ICT in the real-world setting is suboptimal with respect to various clinical, HRQOL and economic outcomes.
authors.affiliation: United BioSource Corporation, Quebec, Canada. krista.payne@unitedbiosource.com
authors.forename: Krista A
authors.initials: KA
authors.lastname: Payne
authors.forename: Diana
authors.initials: D
authors.lastname: Rofail
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Baladi
authors.forename: Muriel
authors.initials: M
authors.lastname: Viala
authors.forename: Linda
authors.initials: L
authors.lastname: Abetz
authors.forename: Marie-Pierre
authors.initials: MP
authors.lastname: Desrosiers
authors.forename: Noreen
authors.initials: N
authors.lastname: Lordan
authors.forename: Khajak
authors.initials: K
authors.lastname: Ishak
authors.forename: Irina
authors.initials: I
authors.lastname: Proskorovsky
mesh_terms: D000293:Adolescent; D000328:Adult; D015913:Chelation Therapy / Q000009:adverse effects / Q000191:economics; D002648:Child; D003365:Costs and Cost Analysis; D000077543:Deferiprone; D003676:Deferoxamine / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy* / Q000191:economics; D008297:Male; D011728:Pyridones / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D011788:Quality of Life; D055815:Young Adult
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine