Abstract
BackgroundHematopoietic stem cell transplantation (HSCT) is the only curative treatment available to severe thalassemic patients. The treatment, however, is very costly, particularly in the context of low and middle income countries, and no studies have been carried out to explore its economic justifiability. This study aimed to estimate the cost-utility of HSCT compared with blood transfusions combined with iron chelating therapy (BT-ICT) for severe thalassemia in Thailand, and to investigate the affordability of HSCT using a budget impact analysis.MethodsA Markov model was used to estimate the relevant costs and health outcomes over the patients' lifetimes taking a societal perspective as recommended by Thailand's health technology assessment guidelines. All future costs and outcomes were discounted at a rate of 3% per annum. Primary outcomes of interest were lifetime costs, quality adjusted life years (QALYs) gained, and the incremental cost-effectiveness ratio (ICER) in Thai baht (THB) per QALY gained.ResultsCompared to BT-ICT, the incremental cost-effectiveness ratio increased with patient age from 80,700 to 183,000 THB per QALY gained for related HSCT and 209,000 to 953,000 THB per QALY gained for unrelated HSCT among patients aged 1 to 15 years (US$1= 34 THB). The governmental budget impact analysis showed that providing 200 related HSCT to patients aged 1 to 10 years, in accordance with the current infrastructure limitations, would initially require approximately 90 million additional THB per year.ConclusionsAt a societal willingness to pay of 100,000 THB per QALY gained, related HSCT was likely to be a cost-effective and affordable treatment for young children with severe thalassemia in Thailand.
Highlights
Hematopoietic stem cell transplantation (HSCT) is the only curative treatment available to severe thalassemic patients
The lifetime costs were the highest for unrelated HSCT, followed by related HSCT, while blood transfusions combined with iron chelating therapy (BT-iron chelating therapy (ICT)) incurred the lowest cost across all age groups
When the costs and quality adjusted life years (QALYs) of related HSCT and unrelated HSCT were compared, it was confirmed that related HSCT always dominated unrelated HSCT due primarily to an increase in transplant-related complications including early and late toxicity, mortality and rejection [6,7,11,12]
Summary
Hematopoietic stem cell transplantation (HSCT) is the only curative treatment available to severe thalassemic patients. This study aimed to estimate the cost-utility of HSCT compared with blood transfusions combined with iron chelating therapy (BT-ICT) for severe thalassemia in Thailand, and to investigate the affordability of HSCT using a budget impact analysis. The incidence of severe thalassemia (i.e. Hb Bart’s hydrops fetalis, b-thalassemia, and b-thalassemia/Hb E) is estimated at 4,253 patients per year [1]. Patients with severe thalassemia present with anemia at the first year of life. The provision of regular blood transfusion (BT) is standard practice for the treatment of severe thalassemia. Provision of BT is hampered by a shortage of blood donations, as well as the high cost of blood screening in order to reduce the residual risk of transmission of blood-borne viruses, including hepatitis and human immunodeficiency virus/acquired immunodeficiency syndrome (HIV/AIDS) [2]. Effective provision of ICT is often compromised by poor compliance as the process itself can have a detrimental effect on quality of life (QoL), especially amongst children [3,4]
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