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Iron chelation therapy: Clinical effectiveness, economic burden and quality of life in patients with iron overload

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Abstract

Introduction

This study of UK patients examines clinical, healthrelated 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 (betathalassaemia, 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 £19,000. 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.

Conclusion

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.

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Authors and Affiliations

  1. United BioSource Corporation, Montreal, Quebec, Canada

    Krista A. Payne, Marie-Pierre Desrosiers, Khajak Ishak & Irina Proskorovsky

  2. Mapi Values, Bollington, Cheshire, UK

    Diana Rofail & Linda Abetz

  3. Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA

    Jean-François Baladi

  4. Mapi Values, Lyon, France

    Muriel Viala

  5. United BioSource Corporation, Concord, Massachusetts, USA

    Noreen Lordan

Authors
  1. Krista A. Payne
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  2. Diana Rofail
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Correspondence to Krista A. Payne.

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Payne, K.A., Rofail, D., Baladi, JF. et al. Iron chelation therapy: Clinical effectiveness, economic burden and quality of life in patients with iron overload. Adv Therapy 25, 725–742 (2008). https://doi.org/10.1007/s12325-008-0085-z

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