Dabrafenib and Trametinib for the Treatment of Pediatric and Adolescent Melanoma: Single Center Experience Data From Italian Compassionate Use

IF 2.4 3区 医学 Q2 HEMATOLOGY
Stefano Chiaravalli, Alice Indini, Michele Del Vecchio, Michela Casanova, Maura Massimino, Luca Bergamaschi, Andrea Ferrari
{"title":"Dabrafenib and Trametinib for the Treatment of Pediatric and Adolescent Melanoma: Single Center Experience Data From Italian Compassionate Use","authors":"Stefano Chiaravalli,&nbsp;Alice Indini,&nbsp;Michele Del Vecchio,&nbsp;Michela Casanova,&nbsp;Maura Massimino,&nbsp;Luca Bergamaschi,&nbsp;Andrea Ferrari","doi":"10.1002/pbc.31644","DOIUrl":null,"url":null,"abstract":"<p>Data on BRAF/MEK inhibitors in pediatric and adolescent melanoma patients are limited. We report data of patients treated with dabrafenib/trametinib for compassionate use at our institution. From January 2020, four patients with Stage III and two patients with Stage IV disease were treated. Our report demonstrates that targeted therapy in children and adolescents is safe and achieves survival rates comparable to those observed in adults. These findings support granting young patients with adult-onset tumors access to compassionate use, as well as increasing their inclusion in clinical trials. Such measures would ensure equitable access to treatments available for adult patients and contribute to improved treatment outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 5","pages":""},"PeriodicalIF":2.4000,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31644","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Blood & Cancer","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/pbc.31644","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

Data on BRAF/MEK inhibitors in pediatric and adolescent melanoma patients are limited. We report data of patients treated with dabrafenib/trametinib for compassionate use at our institution. From January 2020, four patients with Stage III and two patients with Stage IV disease were treated. Our report demonstrates that targeted therapy in children and adolescents is safe and achieves survival rates comparable to those observed in adults. These findings support granting young patients with adult-onset tumors access to compassionate use, as well as increasing their inclusion in clinical trials. Such measures would ensure equitable access to treatments available for adult patients and contribute to improved treatment outcomes.

达非尼和曲美替尼治疗儿童和青少年黑色素瘤:来自意大利同情心使用的单中心经验数据
BRAF/MEK抑制剂在儿童和青少年黑色素瘤患者中的应用数据有限。我们报告了在我们机构接受达非尼/曲美替尼治疗的患者的数据。从2020年1月起,4名III期患者和2名IV期患者接受了治疗。我们的报告表明,儿童和青少年的靶向治疗是安全的,其生存率与成人相当。这些发现支持给予患有成人发病肿瘤的年轻患者获得同情使用的机会,并增加他们在临床试验中的纳入。这些措施将确保成人患者公平获得现有治疗,并有助于改善治疗结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
Pediatric Blood & Cancer
Pediatric Blood & Cancer 医学-小儿科
CiteScore
4.90
自引率
9.40%
发文量
546
审稿时长
1.5 months
期刊介绍: Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信