Nantavithya Chonnipa, Prayongrat Anussara, Laohavinij Wasin, Techavichit Piti, Srilanchakon Khomsak, Utoomprurkporn Nattawan, Chantadisai Maythinee, Wanlapakorn Chaisiri, Shotelersuk Kanjana
Division of Radiation Oncology, Department of Radiology, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.
Division of Radiation Oncology, Department of Radiology, King Chulalongkorn Memorial Hospital, Thai Red Cross Society, Bangkok, Thailand.
Int J Part Ther. 2025 Jun 11;17:100754. doi: 10.1016/j.ijpt.2025.100754. eCollection 2025 Sep.
The benefit of proton beam therapy (PBT) is evident in pediatric cancer, as survivors have to face long-term radiation-related side effects and lifetime costs. However, PBT's higher costs cause a challenge especially in middle-income countries. This study analyzed cost-effectiveness of PBT versus photon beam therapy (XRT) for medulloblastoma based on updated clinical outcomes within middle-income country context.
Markov and Monte Carlo models were constructed for simulation analysis. We used annual mortality rates based on actual medulloblastoma patients receiving craniospinal irradiation over the first 5 years, combined with general population mortality rates thereafter. Rates and utilities for side effects, including hormone deficiencies, hearing loss, brain tumors, thyroid cancer, and IQ decline, were derived from previous literature, while the cardiac mortality rate was estimated using a linear model. Medical and nonmedical costs, along with other economic parameters and mortality rates during the first 5 years, were derived within the context of a middle-income country, using Thailand as a representative setting where PBT is operational.
For the base-case model, the total costs for PBT and XRT were 69 349 USD (2 357 874 THB) and 80 217 USD (2 727 366 THB), respectively, with total utilities of 36.12 and 35.23, respectively. The ICER was -12 211 USD (-415 160 THB)/QALY, and the net monetary benefit for PBT and XRT were 100 627 USD (3 421 326 THB) and 85 572 USD (2 909 434 THB), respectively. Including microsimulation and probabilistic sensitivity analysis for side effects rates, mortality rates, inflation, cost reduction, and utility reduction, PBT was still cost-effective. However, XRT was found to be more cost-effective when excluding opportunity loss from IQ decline.
Despite its higher cost, PBT was more cost-effective than XRT for treating medulloblastoma in a middle-income country, based on updated clinical outcomes, particularly when incorporating IQ decline into the analysis.
质子束治疗（PBT）在儿童癌症治疗中的益处显而易见，因为幸存者不得不面对长期的辐射相关副作用和终身成本。然而，PBT成本较高，这在中等收入国家尤其构成挑战。本研究基于中等收入国家背景下更新的临床结果，分析了PBT与光子束治疗（XRT）治疗髓母细胞瘤的成本效益。
构建马尔可夫模型和蒙特卡罗模型进行模拟分析。我们使用了基于最初5年接受全脑全脊髓照射的实际髓母细胞瘤患者的年度死亡率，并结合此后的一般人群死亡率。副作用的发生率和效用，包括激素缺乏、听力丧失、脑肿瘤、甲状腺癌和智商下降，均来自以往文献，而心脏死亡率则使用线性模型进行估计。医疗和非医疗成本，以及最初5年的其他经济参数和死亡率，是在中等收入国家的背景下得出的，以泰国作为PBT可实施的代表性地区。
对于基础案例模型，PBT和XRT的总成本分别为69349美元（2357874泰铢）和80217美元（2727366泰铢），总效用分别为36.12和35.23。增量成本效果比为-12211美元（-415160泰铢）/质量调整生命年，PBT和XRT的净货币效益分别为100627美元（3421326泰铢）和85572美元（2909434泰铢）。包括对副作用发生率、死亡率、通货膨胀、成本降低和效用降低的微观模拟和概率敏感性分析，PBT仍然具有成本效益。然而，当排除智商下降带来的机会损失时，XRT被发现更具成本效益。
尽管成本较高，但基于更新的临床结果，特别是在将智商下降纳入分析时，在中等收入国家治疗髓母细胞瘤，PBT比XRT更具成本效益。
