Fernandes Ricardo Ribeiro Alves, Vianna Cid Manso de Mello, Guerra Renata Leborato, Cancela Marianna de Camargo, Almeida Liz Maria de, Pereira Adail José da Conceição, Viégas Célia Maria Pais, Ferman Sima Esther, Corrêa Flávia de
Health Technology Assessment Unit, National Cancer Institute, Rio de Janeiro, Brazil.
Social Medicine Institute, Rio de Janeiro State University, Rio de Janeiro, Brazil.
Value Health Reg Issues. 2019 Dec;20:122-128. doi: 10.1016/j.vhri.2019.03.006. Epub 2019 Jul 15.
Proton radiation therapy offers advantages over photon therapy, assisting with severe side effect avoidance. Pediatric patients with medulloblastoma have demonstrated benefit from this technology in recently published cohort studies.
To compare the costs and benefits between proton and photon therapy in treating pediatric medulloblastoma.
The model was built with a lifetime horizon from the Brazilian health system perspective using a 3% discount rate. A microsimulation model was developed after a literature search, comparing scenarios of equipment life span and number of patients treated per year (50, 100, and 150 patients with 10, 25, and 20 years of equipment life span). The baseline parameters were 50 patients treated annually and 20 years of equipment life span.
The quality-adjusted life-year gain was 2.71, and the average incremental cost-effectiveness ratio was $34 590.54 per quality-adjusted life-year. For the willingness-to-pay threshold of 1 gross domestic product per capita, it was observed that the incorporation of the technology would be cost-effective if more than 150 patients were treated per year. The weight of the equipment life span and other variables was limited when it varied in the sensitivity analysis, without significant changes to the model results.
Proton therapy is not cost-effective for pediatric medulloblastoma treatment from the Brazilian health system perspective. The investment is not worth when considering the number of potential patients and the country dimensions.
质子放射治疗比光子治疗具有优势，有助于避免严重的副作用。在最近发表的队列研究中，患有髓母细胞瘤的儿科患者已证明从这项技术中获益。
比较质子治疗和光子治疗在治疗儿科髓母细胞瘤方面的成本和效益。
从巴西卫生系统的角度，以3%的贴现率构建了一个终身模型。在文献检索后开发了一个微观模拟模型，比较了设备使用寿命和每年治疗患者数量的不同情景（每年治疗50、100和150名患者，设备使用寿命分别为10、25和20年）。基线参数为每年治疗50名患者和设备使用寿命20年。
质量调整生命年增益为2.71，平均增量成本效益比为每质量调整生命年34590.54美元。对于人均国内生产总值1倍的支付意愿阈值，观察到如果每年治疗超过150名患者，采用该技术将具有成本效益。在敏感性分析中，当设备使用寿命和其他变量发生变化时，其影响有限，模型结果无显著变化。
从巴西卫生系统的角度来看，质子治疗在儿科髓母细胞瘤治疗中不具有成本效益。考虑到潜在患者数量和国家规模时，这种投资不值得。
