BACKGROUND: The aim of this study is to evaluate the cost-effectiveness of 
proton beam therapy with cochlear dose reduction compared with conventional 
X-ray radiotherapy for medulloblastoma in childhood.
METHODS: We developed a Markov model to describe health states of 6-year-old 
children with medulloblastoma after treatment with proton or X-ray radiotherapy. 
The risks of hearing loss were calculated on cochlear dose for each treatment. 
Three types of health-related quality of life (HRQOL) of EQ-5D, HUI3 and SF-6D 
were used for estimation of quality-adjusted life years (QALYs). The incremental 
cost-effectiveness ratio (ICER) for proton beam therapy compared with X-ray 
radiotherapy was calculated for each HRQOL. Sensitivity analyses were performed 
to model uncertainty in these parameters.
RESULTS: The ICER for EQ-5D, HUI3 and SF-6D were $21 716/QALY, $11 773/QALY, and 
$20 150/QALY, respectively. One-way sensitivity analyses found that the results 
were sensitive to discount rate, the risk of hearing loss after proton therapy, 
and costs of proton irradiation. Cost-effectiveness acceptability curve analysis 
revealed a 99% probability of proton therapy being cost effective at a societal 
willingness-to-pay value.
CONCLUSIONS: Proton beam therapy with cochlear dose reduction improves health 
outcomes at a cost that is within the acceptable cost-effectiveness range from 
the payer's standpoint.