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Open Access Research article

Cost utility analysis of reduced intensity hematopoietic stem cell transplantation in adolescence and young adult with severe thalassemia compared to hypertransfusion and iron chelation program

Rosarin Sruamsiri1, Nathorn Chaiyakunapruk1234*, Samart Pakakasama5, Somtawin Sirireung5, Nintita Sripaiboonkij6, Udomsak Bunworasate7 and Suradej Hongeng5*

Author Affiliations

1 Center of Pharmaceutical Outcomes Research, Department of Pharmacy Practice, Faculty of Pharmaceutical Sciences, Naresuan University, Phitsanulok, Thailand

2 School of Population Health, University of Queensland, Brisbane, Australia

3 School of Pharmacy, University of Wisconsin-Madison, Madison, WI, USA

4 Jeffrey Cheah School of Medicine and Health Sciences, Monash University Sunway campus, Monash, Malaysia

5 Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

6 Cancer registry unit, health case management services, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand

7 Department of Medicine, Faculty of Medicine, Chulalongkorn Memorial Hospital, Chulalongkorn University, Bangkok, Thailand

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BMC Health Services Research 2013, 13:45  doi:10.1186/1472-6963-13-45

Published: 5 February 2013

Abstract

Background

Hematopoieticic stem cell transplantation is the only therapeutic option that can cure thalassemia disease. Reduced intensity hematopoietic stem cell transplantation (RI-HSCT) has demonstrated a high cure rate with minimal complications compared to other options. Because RI-HSCT is very costly, economic justification for its value is needed. This study aimed to estimate the cost-utility of RI-HSCT compared with blood transfusions combined with iron chelating therapy (BT-ICT) for adolescent and young adult with severe thalassemia in Thailand.

Methods

A Markov model was used to estimate the relevant costs and health outcomes over the patients’ lifetimes using a societal perspective. All future costs and outcomes were discounted at a rate of 3% per annum. The efficacy of RI-HSCT was based a clinical trial including a total of 18 thalassemia patients. Utility values were derived directly from all patients using EQ-5D and SF-6D. Primary outcomes of interest were lifetime costs, quality adjusted life-years (QALYs) gained, and the incremental cost-effectiveness ratio (ICER) in US ($) per QALY gained. One-way and probabilistic sensitivity analyses (PSA) were conducted to investigate the effect of parameter uncertainty.

Results

In base case analysis, the RI-HSCT group had a better clinical outcomes and higher lifetime costs. The incremental cost per QALY gained was US $ 3,236 per QALY. The acceptability curve showed that the probability of RI-HSCT being cost-effective was 71% at the willingness to pay of 1 time of Thai Gross domestic product per capita (GDP per capita), approximately US $ 4,210 per QALY gained. The most sensitive parameter was utility of severe thalassemia patients without cardiac complication patients.

Conclusion

At a societal willingness to pay of 1 GDP per capita, RI-HSCT was a cost-effective treatment for adolescent and young adult with severe thalassemia in Thailand compared to BT-ICT.

Keywords:
Cost-utility analysis; Reduced intensity transplantation; Thalassemia; Adolescence; Adult