Costs, quality of life, treatment satisfaction and compliance in patients with β-thalassemia major undergoing iron chelation therapy: The ITHACA study

Luciana Scalone, Lorenzo G. Mantovani, Marieke Krol, Diana Rofail, Simona Ravera, Maria Grazia Bisconte, Caterina Borgna-Pignatti, Zelia Borsellino, Paolo Cianciulli, Domenico Gallisai, Luciano Prossomariti, Ippazio Stefàno, Maria D. Cappellini

Research output: Contribution to journalArticlepeer-review


Objectives: Iron chelation treatment (ICT) in β-thalassemia major (β-TM) patients undergoing blood transfusions can cause low satisfaction, low compliance, with possible negative consequences on treatment success, patients' wellbeing, and costs. The purpose was to estimate the societal burden attributable to β-TM in terms of direct and indirect costs, health-related quality-of-life (HRQoL), satisfaction and compliance with ICT in patients undergoing transfusions and ICT. Research design and methods: The naturalistic, multicenter, longitudinal Italian-THAIassemia-Cost-&-Outcomes-Assessment (ITHACA) cost-of-illness study was conducted involving patients of any age, on ICT for at least 3 years, who were enrolled at 8 Italian Thalassemia Care Centers. Costs were estimated from the societal perspective, quantified with tariffs, prices, or net earnings valid in 2006. Results: One-hundred and thirty-seven patients were enrolled (median age = 28.3, 3-48 years, 49.6% male) and retrospectively observed for a median of 11.6 months. Mean direct costs were €1242/patient/month, 55.5% attributable to ICT, 33.2% attributable to transfusions. Relevant quantity and quality of productivity was lost. Both physical and mental components of HRQoL were compromised. Little difficulties remembering to take ICT and positive satisfaction with the perceived effectiveness of therapy were declared, but not good levels of satisfaction with acceptance, perception of side effects and burden of ICT. Conclusions: The management of β-TM patients undergoing transfusions and ICT is efficacious, although costly, but overall benefits were not always perceived as optimal by patients. Efforts must be focused to improve patients' acceptance and satisfaction with their therapy; this would contribute to a better compliance and hence an increase in treatment effectiveness and patients' overall wellbeing, with expected improved allocation of human and economic resources.

Original languageEnglish
Pages (from-to)1905-1917
Number of pages13
JournalCurrent Medical Research and Opinion
Issue number7
Publication statusPublished - Jul 2008


  • β-thalassemia
  • Compliance
  • Cost analysis
  • HRQoL
  • Iron chelation therapy
  • Satisfaction

ASJC Scopus subject areas

  • Medicine(all)


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