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

ABSTRACT

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-THAlassemia-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.

Key words: :

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

Acknowledgments

Declaration of interest: An unrestricted grant was provided by Novartis Pharmaceutical Corporation, East Hanover, NJ, USA, for this study.

L.G.M. and M.D.C. designed the study. S.R. managed the collection of the data from the participating centers. S.R., L.S., M.K., and D.R. analyzed the data. L.S., M.D.C., L.G.M. wrote the paper. M.B., C.B-P., C/B P.C., D.G., L. P., I.S, S.R., M.K., and D.R. reviewed and integrated the paper by adding further information that helped to interpret the results. The other co-investigators, listed as ITHACA study group members, contributed with the collection of the data. The views expressed in the paper are those of the authors.

We thank the patients and caregivers who took part in this study. Thanks also to Dr. Jean-François Baladi.

Other ITHACA study group members were: M. Capra, L. Cuccia, from U.O.C. Ematologia – Emoglobinopatie, A.R.N.A.S. Civico, Di Cristina, Ascoli – Palermo; F. Sorrentino, from Ospedale S. Eugenio – Centro Microcitemie DH talassemia Roma; M. Viala, from Mapi Valued Questionnaire Design and Validation Unit – Bollington, Cheshire, UK; C. Uyl-de Groot, from Institute for Medical Technology Assessment, Erasmus Medical Centre, Rotterdam, The Netherlands.

The results of this study have been presented at a national and some international Congresses: poster at The 11th EHA Congress, Amsterdam (The Netherlands) June 15–18, 2006; posters at The 9th Annual European Congress of ISPOR, Copenhagen (Denmark), October 29–31, 2006; oral presentation at The IV National Congress of SOSTE (Società per lo Studio delle Talassemie ed Emoglobinemie), Rome (Italy), November 18–19, 2006; posters at The ASH Congress, Orlando (USA), December 9–12, 2006.