Cost-effectiveness analysis of cochlear dose reduction by proton beam therapy for medulloblastoma in childhood

Area of Science:

• Pediatric Oncology
• Radiation Oncology
• Health Economics

Background:

• Childhood medulloblastoma treatment involves radiotherapy, posing risks of hearing loss.
• Conventional X-ray radiotherapy can lead to significant cochlear dose exposure.
• Proton beam therapy (PBT) offers potential for dose reduction to critical structures like the cochlea.

Purpose of the Study:

• To evaluate the cost-effectiveness of PBT with cochlear dose reduction versus conventional X-ray radiotherapy for pediatric medulloblastoma.
• To compare health outcomes, specifically hearing preservation and quality of life, between the two treatment modalities.

Main Methods:

• A Markov model was developed to simulate health states for 6-year-old medulloblastoma patients.
• Cochlear dose was calculated for both PBT and X-ray radiotherapy to estimate hearing loss risks.
• Quality-adjusted life years (QALYs) were estimated using EQ-5D, HUI3, and SF-6D health-related quality of life measures.
• Incremental cost-effectiveness ratios (ICERs) and sensitivity analyses were performed.

Main Results:

• The ICERs for PBT compared to X-ray radiotherapy were $21,716/QALY (EQ-5D), $11,773/QALY (HUI3), and $20,150/QALY (SF-6D).
• Sensitivity analyses indicated results were sensitive to discount rate, hearing loss risk with PBT, and PBT costs.
• Cost-effectiveness acceptability curve analysis showed a 99% probability of PBT being cost-effective at a societal willingness-to-pay threshold.

Conclusions:

• Proton beam therapy with cochlear dose reduction demonstrates improved health outcomes, including better hearing preservation.
• The evaluated cost-effectiveness of PBT falls within an acceptable range for payers.
• PBT represents a valuable therapeutic option for childhood medulloblastoma, balancing efficacy, toxicity reduction, and economic considerations.