Abstract:
The objectives of this study were to estimate the cost-utility of hematopoietic
stem cell transplantation (HSCT) compared with blood transfusions and iron
chelating therapy (BT-ICT) for severe thalassemic patients and to estimate the budget
impact of HSCT in instances where it was found to be cost-effective when included
in the benefit package of the Universal Coverage scheme using a governmental
budget impact analysis. The study population was patients with severe thalassemia
receiving BT-ICT or HSCT. Cost-utility and budget impact analyses were performed
to compare BT-ICT and the two types of HSCT (i.e., related HSCT and unrelated
HSCT). A Markov model was used to estimate the costs and health outcome over the
patients lifetimes taking a societal perspective. The health outcome was quality
adjusted life years (QALYs) gained. One-way and probabilistic sensitivity analyses
(PSA) were conducted to investigate the effect of parameter uncertainty.
Among patients aged between 1 and 15 years, the ICERs increased in older
patients ranging 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. For patients
aged greater than 20 years, BT-ICT was dominant since it incurred less cost and
yielded more QALYs compared to HSCT. At a societal willingness to pay (WTP) of
100,000 THB per QALY gained, related HSCT was likely to be cost-effective when
provided to patients aged less than 10 years, while unrelated HSCT was not a costeffective
option. With a higher WTP threshold of 300,000 THB per QALY gained
related and unrelated HSCT were likely to be cost-effective when provided to
patients aged up to 17 and 10, respectively. The governmental budget impact analysis
showed that providing related HSCT to patients aged 1 to 10 years would reduce the
overall budget from the third fiscal year onwards. In the Thai context, related HSCT
appears to be a cost-effective and affordable treatment for young children with severe
thalassemia. Related HSCT for older patients and unrelated HSCT for young patients
could also be considered for use, subject to other criteria and a higher willingness to
pay for each QALY gained.