Abstract
BackgroundHematopoieticic 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.MethodsA 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.ResultsIn 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.ConclusionAt 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.
Highlights
Hematopoieticic stem cell transplantation is the only therapeutic option that can cure thalassemia disease
This study examines the potential cost-utility of Reduced intensity hematopoietic stem cell transplantation (RI-hematopoietic stem cell transplantation (HSCT)) compared to blood transfusions combined with iron chelating therapy (BT-ICT) in the treatment of patients with severe adolescent and young adult thalassemia
Overall description of cost-utility analysis methodology We used a markov model to simulate severe thalassemia patients receiving RI-HSCT compared to BT-ICT, which is a standard practice of thalassemia treatment
Summary
Hematopoieticic stem cell transplantation is the only therapeutic option that can cure thalassemia disease. 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. The standard treatment of severe thalassemia is regular blood transfusion combined with iron chelating therapy (BT-ICT) to prevent iron overload. BT-ICT treatment has to be given subcutaneously for 8 to 12 hours per days, 5 to 7 days per week This arduous treatment regimen and the high cost can lead to poor compliance and difficulty in patients’ life which could affect the effectiveness of the treatment. Adult thalassemia patients (age 17 or older) have more advanced disease and treatment related organ complications mainly due to prolonged exposure to iron overload [2]
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