Proton radiotherapy outperforms medulloblastoma irradiation with photons in economic savings in Brazil (PROMISE).
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OBJECTIVE: This study aimed to evaluate the cost-effectiveness of proton therapy compared to photon therapy for pediatric medulloblastoma treatment from the payer's perspective in Brazil, assessing its economic viability and potential to improve long-term outcomes by reducing late complications. MATERIALS AND METHODS: A Markov chain model simulated outcomes for a cohort of 5-year-old children with medulloblastoma over a 30-year horizon. The base case involved craniospinal irradiation (36 Gy) with a 54 Gy posterior fossa boost, tracking 11 health states, including hearing loss, cognitive deficit, and coronary artery disease (CAD), with CAD and congestive heart failure (CHF) onset after 10 years. Annual transition probabilities were derived from systematic reviews. Costs (photons: US$3000; protons: US$50,000) reflected Brazilian and international data, with a willingness-to-pay (WTP) threshold of US$50,000/QALY. Incremental cost-effectiveness ratio (ICER), net monetary benefit (NMB), and probabilistic sensitivity analysis (PSA) via Monte Carlo simulations (1000 iterations, 25,000 patients) were calculated using Python 3.11. RESULTS: Proton therapy yielded 20.45 QALYs at US$102,933, versus 16.87 QALYs at US$141,971 for photons, with an ICER of -US$22,857/QALY, indicating dominance. PSA showed a mean NMB of US$70,290 (95 % CI: US$42,377-US$98,662), with protons cost-effective in >95 % of simulations. Survival at 30 years was 40.5 % (protons) versus 27.1 % (photons), driven by reduced late effects (e.g., 68 % vs. 45 % cumulative events). CONCLUSION: Proton therapy is a cost-effective, dominant strategy for pediatric medulloblastoma in Brazil, offering superior outcomes and cost savings. These findings support its adoption through innovative funding models like public-private partnerships.