Department of Pediatrics' Oncology, IOP-GRAACC-Federal University of Sao Paulo, Sao Paulo, Brazil.
Department of Pediatrics' Allergy, Immunology, and Rheumatology- Federal University of Sao Paulo, Sao Paulo, Brazil.
Childs Nerv Syst. 2024 Dec;40(12):4259-4264. doi: 10.1007/s00381-024-06564-9. Epub 2024 Aug 7.
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.
Prospective analysis of children with LGG submitted to carboplatin desensitization from December 2017 to June 2020 with follow-up until April 2024.
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.
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.
无法切除的儿科低级别胶质瘤（LGG）通常需要辅助治疗，而卡铂过敏反应（HR）通常导致标准化疗方案过早停止。在分子时代，对肿瘤遗传特征的认识的进步使得针对这组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。本研究旨在描述在一个中等收入国家的单一中心中卡铂脱敏方案的结果。
对 2017 年 12 月至 2020 年 6 月期间接受卡铂脱敏的 LGG 患儿进行前瞻性分析，并随访至 2024 年 4 月。
共纳入 9 例患儿。平均年龄为 11 岁。5 例为男性。7 例为视路肿瘤，2 例为颈髓肿瘤。6 例有组织学诊断，4 例有分子分析。研究期间卡铂反应发生率为 39.1%。6 例患儿进行了皮肤点刺试验，其中 3 例结果阳性。首次 HR 平均发生在治疗的第 9 个周期左右。所有患儿均有皮肤症状，9 例中有 5 例出现过敏反应作为首发反应。77.7%的患儿完成了方案，临床获益率（疾病稳定和部分缓解）为 88.8%。6 例患儿进一步需要其他治疗线。每月卡铂的总费用为 409.09 美元，而靶向治疗（dabrafenib 加 trametinib）的费用为 4929.28 至 5548.57 美元。
我们的研究提供了一个有趣且具有成本效益的选择，脱敏使 HR 患儿能够接受一线治疗，避免了有效治疗的中断。
