NUT carcinoma in children and adolescents: An analysis of the European Cooperative Study Group on pediatric rare tumors (EXPeRT)

IF 4.4 2区医学 Q1 ONCOLOGY Lung Cancer Pub Date : 2025-03-01 Epub Date: 2025-02-19 DOI:10.1016/j.lungcan.2025.108449

Tim Flaadt , Lauriane Lemelle , Michael Abele , Calogero Virgone , Tal Ben-Ami , Denis Kachanov , Apostolos Pourtsidis , Andrea Ferrari , Gianni Bisogno , Ewa Bien , Nuno Jorge Dos Reis Farinha , Jan Godzinski , Yves Reguerre , Jelena Roganovic , Linus D. Kloker , Ulrich M. Lauer , Dominik T. Schneider , Ines B. Brecht , Daniel Orbach

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Abstract

Background and Aims

NUT carcinoma (NC) is a sporadic, highly aggressive tumor that primarily affects children, adolescents, and young adults and is characterized by the presence of somatic NUTM1 rearrangements. This analysis by the European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) aims to fill the knowledge gap regarding the clinical characteristics of children with NC.

Methods

A retrospective case series of NC-patients aged 0–18 years treated between 2011 and 2023 was conducted using the EXPeRT database. Relevant clinical characteristics, including treatment and outcome were recorded.

Results

Twenty-seven patients with a median age of 13 years (range 7–18) were analyzed. Thirteen patients were initially misdiagnosed. Sixteen patients had thoracic and 11 extra-thoracic tumors, including three in the nasal/sinus region and two in the submandibular glands. Despite intense multimodal treatment, median event-free and overall survivals were 1.5 and 6.5 months, respectively.

Conclusions

Early diagnosis of NC by examination of the NUTM1 rearrangement in undifferentiated or poorly differentiated carcinomas is crucial in order to initiate specific and intensive therapy as quickly as possible. Similar to adult patients, only a minority of pediatric patients achieved prolonged survival. Therefore, the development of novel therapeutic strategies in future joint clinical trials is essential.

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儿童和青少年NUT癌：欧洲儿科罕见肿瘤合作研究小组分析（专家）

背景和AimsNUT癌（NC）是一种散发的、高度侵袭性的肿瘤，主要影响儿童、青少年和年轻人，其特征是存在体细胞NUTM1重排。这项由欧洲儿科罕见肿瘤合作研究小组（EXPeRT）进行的分析旨在填补关于儿童NC临床特征的知识空白。方法采用EXPeRT数据库对2011 ~ 2023年间0 ~ 18岁的nc患者进行回顾性病例分析。记录相关临床特征，包括治疗和结果。结果共分析27例患者，年龄7 ~ 18岁，中位年龄13岁。13名患者最初被误诊。16例患者有胸部和11例胸外肿瘤，其中3例在鼻/窦区，2例在下颌腺。尽管进行了密集的多模式治疗，中位无事件生存期和总生存期分别为1.5个月和6.5个月。结论在未分化或低分化的肿瘤中，通过检测NUTM1重排及早诊断NC，以尽早启动特异性和强化治疗是至关重要的。与成人患者相似，只有少数儿科患者延长了生存期。因此，在未来的联合临床试验中开发新的治疗策略至关重要。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Lung Cancer 医学-呼吸系统

CiteScore

9.40

自引率

3.80%

发文量

407

审稿时长

25 days

期刊介绍： Lung Cancer is an international publication covering the clinical, translational and basic science of malignancies of the lung and chest region.Original research articles, early reports, review articles, editorials and correspondence covering the prevention, epidemiology and etiology, basic biology, pathology, clinical assessment, surgery, chemotherapy, radiotherapy, combined treatment modalities, other treatment modalities and outcomes of lung cancer are welcome.