Carboplatin desensitization in the era of target therapies: still worthwhile?

IF 1.3 4区 医学 Q4 CLINICAL NEUROLOGY
Child's Nervous System Pub Date : 2024-12-01 Epub Date: 2024-08-07 DOI:10.1007/s00381-024-06564-9
Natália Dassi, Fernanda Sales da Cunha, Andrea Maria Cappellano, Chayanne Andrade de Araujo, Denise Neiva Santos de Aquino, Daniela Barbosa de Almeida, Nasjla Saba da Silva, Luis Felipe Chiaverini Ensina
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引用次数: 0

Abstract

Purpose: 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.

Method: 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.

Conclusion: 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.

靶向疗法时代的卡铂脱敏:还值得吗?
目的:无法切除的小儿低级别胶质瘤(LGG)通常需要辅助治疗,而卡铂超敏反应(HR)通常会导致标准化疗方案过早停止治疗。在分子时代,人们对肿瘤遗传特征的认识不断进步,从而开发出了针对这类肿瘤的靶向疗法;然而,对治疗的成本效益进行评估至关重要,尤其是在低收入国家。本研究旨在描述中等收入国家的一个中心采用卡铂脱敏方案的结果:方法:对2017年12月至2020年6月期间接受卡铂脱敏治疗的LGG患儿进行前瞻性分析,随访至2024年4月:共纳入九名患者。平均年龄为 11 岁。五名患者为男性。7例为视路癌,2例为颈髓癌。6例进行了组织学诊断,4例进行了分子分析。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,其中三人结果呈阳性。首次HR平均发生在第9个治疗周期左右。所有患者都出现了皮肤症状,9 人中有 5 人的首次反应为过敏性休克。77.7%的患者完成了治疗方案,临床获益率(疾病稳定和部分反应)为88.8%。有 6 名患者还需要接受其他治疗。每月,卡铂的总费用为409.09美元,靶向疗法(达拉菲尼加曲美替尼)的总费用为4929.28美元至5548.57美元:我们的研究提出了一种有趣且具有成本效益的方案,即通过脱敏治疗,HR患儿可接受一线治疗,避免中断有效治疗。
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来源期刊
Child's Nervous System
Child's Nervous System 医学-临床神经学
CiteScore
3.00
自引率
7.10%
发文量
322
审稿时长
3 months
期刊介绍: The journal has been expanded to encompass all aspects of pediatric neurosciences concerning the developmental and acquired abnormalities of the nervous system and its coverings, functional disorders, epilepsy, spasticity, basic and clinical neuro-oncology, rehabilitation and trauma. Global pediatric neurosurgery is an additional field of interest that will be considered for publication in the journal.
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