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Cost-Utility Analysis of Three Iron Chelators Used in Monotherapy for the Treatment of Chronic Iron Overload in β-Thalassaemia Major Patients: An Italian Perspective

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Abstract

Purpose

Deferiprone (DFP), deferasirox (DFX) and deferoxamine (DFO) are used in thalassaemia major (TM) patients to treat chronic iron overload. We evaluated the cost-effectiveness of DFP, compared with DFX and DFO monotherapy, from an Italian healthcare system perspective.

Methods

A Markov model was used over a time horizon of 5 years. Italian-specific cost data were combined with Italian efficacy data. Costs and quality-adjusted life years (QALYs) were calculated for each treatment, with cost-effectiveness expressed as cost per QALY.

Results

In all scenarios modelled, DFP was the dominant treatment strategy. Sensitivity analyses showed that DFP dominated the other treatments with a >99% likelihood of being cost-effective against DFX and DFO at a willingness to pay threshold of €20,000 per QALY.

Conclusions

DFP was the dominant and most cost-effective treatment for managing chronic iron overload in TM patients. Its use can result in substantial cost savings for the Italian healthcare system.

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Authors and Affiliations

  1. Magnetic Resonance Imaging Unit, Fondazione G. Monasterio CNR-Regione Toscana, Area della Ricerca S. Cataldo, Via Moruzzi, 56124, Pisa, Italy

    Alessia Pepe & Antonella Meloni

  2. Epidemiology and Biostatistics Unit, Institute of Clinical Physiology, CNR, Pisa, Italy

    Giuseppe Rossi

  3. Decision Resources Group Abacus, Bicester, Oxfordshire, UK

    Anthony Bentley & Aishah Hanif

  4. Dipartimento di Pediatria, Clinica di Emato-Oncologia Pediatrica, Università di Padova/Azienda Ospedaliera, Padua, Italy

    Maria Caterina Putti & Ludovica Frizziero

  5. U.O. Microcitemie, A.O. “Bianchi-Melacrino-Morelli”, Reggio Calabria, Italy

    Domenico Giuseppe D’Ascola

  6. U.O.C. Ematologia con Talassemia, ARNAS Ospedale Civico, Palermo, Italy

    Liana Cuccia

  7. U.O.S.D. Centro per le Microcitemie, AORN Cardarelli, Naples, Italy

    Anna Spasiano & Aldo Filosa

  8. U.O.D. Talassemia, ARNAS “Garibaldi”, Catania, Italy

    Vincenzo Caruso

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  1. Alessia Pepe
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Corresponding author

Correspondence to Alessia Pepe.

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Funding

This manuscript was partially supported by Apopharma Inc. who provided financial support to Decision Resources Group Abacus for economic analysis and writing assistance.

Conflict of interest

Alessia Pepe is the PI of the MIOT project, which received “no profit support” from Chiesi Farmaceutici S.p.A. and ApoPharma Inc. Anthony Bentley and Aishah Hanif were employed by a consultancy (Decision Resources Group) that was commissioned by Apopharma Inc. to prepare cost utility analysis and manuscript. All other authors have no conflicts of interest to declare.

Ethical statement

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.

Informed consent was obtained from all individual participants included in the study.

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Pepe, A., Rossi, G., Bentley, A. et al. Cost-Utility Analysis of Three Iron Chelators Used in Monotherapy for the Treatment of Chronic Iron Overload in β-Thalassaemia Major Patients: An Italian Perspective. Clin Drug Investig 37, 453–464 (2017). https://doi.org/10.1007/s40261-017-0496-1

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  • DOI: https://doi.org/10.1007/s40261-017-0496-1

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