BACKGROUND: Radiation therapy is an important component in the treatment of 
medulloblastoma; however, in many patients, it is associated with risk of late 
adverse events. Proton radiation therapy has potential to reduce the risk of 
adverse events compared with conventional radiation, but it is associated with a 
higher treatment cost. The objective of the current study was to assess the 
cost-effectiveness of proton therapy compared with conventional radiation 
therapy in the treatment of childhood medulloblastoma.
METHODS: The consequences of radiation therapy were evaluated using a Markov 
simulation model. Children age 5 years with medulloblastoma were followed. The 
patients were at risk of several types of adverse events, including hearing 
loss, intelligence quotient (IQ) loss, hypothyroidism, growth hormone deficiency 
(GHD), osteoporosis, cardiac disease, and secondary malignancies. The patients 
also were at risk of death and were divided into risk groups for normal death, 
death due to tumor recurrence, treatment-related cardiac death, 
treatment-related subsequent tumor death, or treatment-related other death. A 
review of the literature was conducted to estimate the parameters in the model.
RESULTS: The base-case results showed that proton therapy was associated with 
23,600 in cost savings and 0.68 additional quality-adjusted life-years per 
patient. The analyses showed that reductions in IQ loss and GHD contributed to 
the greatest part of the cost savings and were the most important parameters for 
cost-effectiveness.
CONCLUSIONS: The results of the current study indicated that proton radiation 
therapy can be cost-effective and cost-saving compared with conventional 
radiation therapy in the treatment of children with medulloblastoma if the 
appropriate patients are selected for the therapy. However, there have been few 
long-term follow-up studies, and more much information on the long-term 
consequences of radiation therapy is needed.