ORIGINAL RESEARCH ARTICLE Cost-Utility Analysis of Three Iron Chelators Used in Monotherapy for the Treatment of Chronic Iron Overload in b-Thalassaemia Major Patients: An Italian Perspective Alessia Pepe 1 • Giuseppe Rossi 2 • Anthony Bentley 3 • Maria Caterina Putti 4 • Ludovica Frizziero 4 • Domenico Giuseppe D’Ascola 5 • Liana Cuccia 6 • Anna Spasiano 7 • Aldo Filosa 7 • Vincenzo Caruso 8 • Aishah Hanif 3 • Antonella Meloni 1 Ó Springer International Publishing Switzerland 2017 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-effec- tive treatment for managing chronic iron overload in TM patients. Its use can result in substantial cost savings for the Italian healthcare system. Key Points Deferiprone is the most cost-effective iron chelator in Italy and its use may result in cost savings for the healthcare system. Deferasirox produced a higher QALY gain than deferoxamine but at a greater total cost. 1 Introduction Thalassaemia is a genetic blood disorder, characterised by decreased or altered synthesis of haemoglobin. With approximately 60,000 severely affected children born each year, it is one of the most common genetic diseases worldwide [1]. The estimated total annual incidence of symptomatic individuals is 1 in 100,000 people worldwide and 1 in 10,000 in Europe [2]. It is commonly found in people of Mediterranean descent, such as Italian nationals, and is a major health problem in Italy [3], with an esti- mated prevalence of approximately 7000 patients [4]. There are three main forms of b-thalassaemia that have been described based on the phenotype—minor, intermedia & Alessia Pepe alessia.pepe@ftgm.it 1 Magnetic Resonance Imaging Unit, Fondazione G. Monasterio CNR-Regione Toscana, Area della Ricerca S. Cataldo, Via Moruzzi, 56124 Pisa, Italy 2 Epidemiology and Biostatistics Unit, Institute of Clinical Physiology, CNR, Pisa, Italy 3 Decision Resources Group Abacus, Bicester, Oxfordshire, UK 4 Dipartimento di Pediatria, Clinica di Emato-Oncologia Pediatrica, Universita ` di Padova/Azienda Ospedaliera, Padua, Italy 5 U.O. Microcitemie, A.O. ‘‘Bianchi-Melacrino-Morelli’’, Reggio Calabria, Italy 6 U.O.C. Ematologia con Talassemia, ARNAS Ospedale Civico, Palermo, Italy 7 U.O.S.D. Centro per le Microcitemie, AORN Cardarelli, Naples, Italy 8 U.O.D. Talassemia, ARNAS ‘‘Garibaldi’’, Catania, Italy Clin Drug Investig DOI 10.1007/s40261-017-0496-1