目的:不可切除的小儿低度胶质瘤(LGG)通常需要辅助治疗,卡铂超敏反应(HR)通常导致标准化疗方案的过早治疗停止。在分子时代,在了解肿瘤遗传特征方面取得的进展使得针对这类肿瘤的靶向治疗得以发展;然而,治疗的成本效益评估,特别是在低收入国家,至关重要。目的是描述中等收入国家单个中心的卡铂脱敏方案的结果。
方法:对2017年12月至2020年6月接受卡铂脱敏的LGG患儿进行前瞻性分析,随访至2024年4月。
结果:纳入9例患者。平均年龄为11岁。5名患者为男性。七个有视神经通路和两个宫颈髓腔位置。六个进行了组织学诊断和四个分子分析。研究期间卡铂反应的发生率为39.1%。六名患者接受了皮肤点刺试验,三是积极成果。第一个HR发生了,平均而言,在第9周期的治疗。所有病人都有皮肤症状,九分之五的人有过敏反应作为第一反应。77.7%的患者完成了协议,临床获益率(疾病稳定和部分缓解)为88.8%。六名患者进一步需要其他治疗路线。每月,卡铂的总费用为409.09美元,目标疗法(达拉非尼加曲美替尼),4929.28美元至5548.57美元。
结论:我们的研究提出了一种有趣且具有成本效益的选择,其中脱敏允许HR患儿接受一线治疗,避免停止有效的治疗。
OBJECTIVE: Unresectable pediatric low-grade gliomas (LGG) usually need adjuvant therapy, and carboplatin hypersensitivity reaction (HR) commonly leads to premature treatment cessation of a standard chemotherapy regimen. In the molecular era, advances in understanding tumor genetic characteristics allowed the development of targeted therapies for this group of tumors; however, cost-effectiveness assessment of treatments, especially in low-income countries, is crucial. The aim is to describe the results of carboplatin desensitization protocol in a single center in a middle-income country.
METHODS: Prospective analysis of children with LGG submitted to carboplatin desensitization from December 2017 to June 2020 with follow-up until April 2024.
RESULTS: Nine patients were included. The mean age was 11 years. Five patients were male. Seven had optic pathway and two cervicomedullary location. Six had histologic diagnosis and four molecular analyses. The incidence of carboplatin reactions during the study period was 39.1%. Six patients underwent skin prick test, three with positive results. The first HR occurred, on average, around the 9th cycle of treatment. All patients had cutaneous symptoms, and five out of nine had anaphylaxis as the first reaction. 77.7% of the patients completed the protocol, and the clinical benefit rate (stable disease and partial response) was 88.8%. Six patients further required other lines of therapy. Monthly, the total cost for carboplatin was $409.09, and for target therapies (dabrafenib plus trametinib), $4929.28 to $5548.57.
CONCLUSIONS: Our study presented an interesting and cost-effective option where desensitization allowed children with HR to be treated with first-line therapy, avoiding the discontinuation of an effective treatment.