Inflammatory myofibroblastic tumors in children: clinical characteristics and treatment outcomes with a focus on targeted therapies.

Ülkü Miray Yıldırım, Rejin Kebudi, Bülent Zülfikar, Bilge Bilgiç
{"title":"Inflammatory myofibroblastic tumors in children: clinical characteristics and treatment outcomes with a focus on targeted therapies.","authors":"Ülkü Miray Yıldırım, Rejin Kebudi, Bülent Zülfikar, Bilge Bilgiç","doi":"10.24953/turkjpediatr.2025.5463","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Inflammatory myofibroblastic tumors (IMTs) are rare neoplasms in children. Traditionally, surgical resection has been the primary treatment modality with limited efficacy reported for conventional chemotherapy and radiation therapy. Recently, targeted therapies have emerged as potential options for selected cases. This study aimed to evaluate the demographic, clinical, laboratory, and radiological characteristics, as well as treatment outcomes, in children diagnosed with IMTs.</p><p><strong>Methods: </strong>This study involved a retrospective review of medical records for eight children diagnosed with IMTs between 1990 and 2022. We collected demographic, clinical, laboratory, and radiological data, as well as treatment outcomes. Data on tumor characteristics, surgical procedures, and chemotherapy or targeted therapy treatments were extracted.</p><p><strong>Results: </strong>The mean age at diagnosis was 9 years. None presented with metastatic disease at the time of diagnosis. Anaplastic lymphoma kinase (ALK) positivity was identified in tumor tissue from five patients. Among the six patients who underwent surgical resection, three achieved negative surgical margins. Of the three patients with positive surgical margins, one underwent re-resection, local and metastatic recurrences were noted in another, and one was started on crizotinib. A patient with an inoperable tumor at diagnosis was initiated on crizotinib and achieved complete remission. Ceritinib was administered to a patient with YWHAE-ROS fusion, resulting in more than 90% reduction in tumor volume. The median follow-up time was 67.5 months. The five-year overall survival and event-free survival rates for the cohort were 85.7% and 72.9%, respectively.</p><p><strong>Conclusions: </strong>While surgical resection remains the cornerstone of treatment for IMTs, favorable outcomes can be achieved with chemotherapy and targeted therapies in selected cases. Increasing the utilization of targeted therapies may be beneficial, particularly through molecular studies aimed at minimizing the side effects associated with conventional chemotherapy.</p>","PeriodicalId":101314,"journal":{"name":"The Turkish journal of pediatrics","volume":"67 1","pages":"51-60"},"PeriodicalIF":0.0000,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"The Turkish journal of pediatrics","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.24953/turkjpediatr.2025.5463","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0

Abstract

Background: Inflammatory myofibroblastic tumors (IMTs) are rare neoplasms in children. Traditionally, surgical resection has been the primary treatment modality with limited efficacy reported for conventional chemotherapy and radiation therapy. Recently, targeted therapies have emerged as potential options for selected cases. This study aimed to evaluate the demographic, clinical, laboratory, and radiological characteristics, as well as treatment outcomes, in children diagnosed with IMTs.

Methods: This study involved a retrospective review of medical records for eight children diagnosed with IMTs between 1990 and 2022. We collected demographic, clinical, laboratory, and radiological data, as well as treatment outcomes. Data on tumor characteristics, surgical procedures, and chemotherapy or targeted therapy treatments were extracted.

Results: The mean age at diagnosis was 9 years. None presented with metastatic disease at the time of diagnosis. Anaplastic lymphoma kinase (ALK) positivity was identified in tumor tissue from five patients. Among the six patients who underwent surgical resection, three achieved negative surgical margins. Of the three patients with positive surgical margins, one underwent re-resection, local and metastatic recurrences were noted in another, and one was started on crizotinib. A patient with an inoperable tumor at diagnosis was initiated on crizotinib and achieved complete remission. Ceritinib was administered to a patient with YWHAE-ROS fusion, resulting in more than 90% reduction in tumor volume. The median follow-up time was 67.5 months. The five-year overall survival and event-free survival rates for the cohort were 85.7% and 72.9%, respectively.

Conclusions: While surgical resection remains the cornerstone of treatment for IMTs, favorable outcomes can be achieved with chemotherapy and targeted therapies in selected cases. Increasing the utilization of targeted therapies may be beneficial, particularly through molecular studies aimed at minimizing the side effects associated with conventional chemotherapy.

儿童炎性肌纤维母细胞瘤:临床特征和治疗效果,重点关注靶向疗法。
背景:炎症性肌纤维母细胞瘤(IMTs)是一种罕见的儿童肿瘤。传统上,手术切除一直是主要的治疗方式,但传统化疗和放疗的疗效有限。最近,靶向治疗已成为选定病例的潜在选择。本研究旨在评估诊断为imt的儿童的人口学、临床、实验室和放射学特征以及治疗结果。方法:本研究对1990年至2022年间诊断为imt的8名儿童的医疗记录进行了回顾性分析。我们收集了人口统计学、临床、实验室和放射学数据以及治疗结果。提取肿瘤特征、外科手术、化疗或靶向治疗的数据。结果:平均诊断年龄为9岁。在诊断时没有出现转移性疾病。5例肿瘤组织中发现间变性淋巴瘤激酶(ALK)阳性。在接受手术切除的6例患者中,3例手术切缘为阴性。在3例手术切缘阳性的患者中,1例接受了再次切除,另1例发现局部和转移性复发,1例开始服用克唑替尼。在诊断时无法手术的肿瘤患者开始使用克唑替尼并获得完全缓解。对YWHAE-ROS融合的患者给予Ceritinib,导致肿瘤体积减少90%以上。中位随访时间为67.5个月。该队列的5年总生存率和无事件生存率分别为85.7%和72.9%。结论:虽然手术切除仍然是治疗imt的基石,但在选定的病例中,化疗和靶向治疗可以获得良好的结果。增加靶向治疗的使用可能是有益的,特别是通过旨在减少与传统化疗相关的副作用的分子研究。
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
自引率
0.00%
发文量
0
×
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