title: 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.
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.
  
  
  CONCLUSIONS
  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.
authors: Center of Pharmaceutical Outcomes Research, Department of Pharmacy Practice, Faculty of Pharmaceutical Sciences, Naresuan University, Phitsanulok, Thailand.
authors: Rosarin
authors: R
authors: Sruamsiri
authors: Nathorn
authors: N
authors: Chaiyakunapruk
authors: Samart
authors: S
authors: Pakakasama
authors: Somtawin
authors: S
authors: Sirireung
authors: Nintita
authors: N
authors: Sripaiboonkij
authors: Udomsak
authors: U
authors: Bunworasate
authors: Suradej
authors: S
authors: Hongeng
mesh_terms: D000293:Adolescent; D001803:Blood Transfusion / Q000191:economics* / Q000379:methods*; D002648:Child; D003362:Cost-Benefit Analysis; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000191:economics*; D006801:Humans; D007502:Iron Chelating Agents / Q000191:economics / Q000627:therapeutic use*; D008297:Male; D008390:Markov Chains; D018803:Models, Economic; D012720:Severity of Illness Index; D013785:Thailand; D013789:Thalassemia / Q000628:therapy*
chemical_list: D007502:Iron Chelating Agents
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