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@ARTICLE{Flaadt:299483,
author = {T. Flaadt and L. Lemelle and M. Abele and C. Virgone and T.
Ben-Ami and D. Kachanov and A. Pourtsidis and A. Ferrari and
G. Bisogno and E. Bien and N. J. Dos Reis Farinha and J.
Godzinski and Y. Reguerre and J. Roganovic and L. D.
Kloker$^*$ and U. M. Lauer$^*$ and D. T. Schneider and I. B.
Brecht and D. Orbach},
title = {{NUT} carcinoma in children and adolescents: {A}n analysis
of the {E}uropean {C}ooperative {S}tudy {G}roup on pediatric
rare tumors ({EXP}e{RT}).},
journal = {Lung cancer},
volume = {201},
issn = {0169-5002},
address = {Amsterdam [u.a.]},
publisher = {Elsevier},
reportid = {DKFZ-2025-00443},
pages = {108449},
year = {2025},
abstract = {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.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.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.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.},
keywords = {Children (Other) / EXPeRT group (Other) / NUT carcinoma
(Other) / Rare tumors (Other) / Targeted therapy (Other)},
cin = {TU01},
ddc = {610},
cid = {I:(DE-He78)TU01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39999637},
doi = {10.1016/j.lungcan.2025.108449},
url = {https://inrepo02.dkfz.de/record/299483},
}