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@ARTICLE{Schoot:284824,
author = {R. A. Schoot and D. Orbach and V. Minard Colin and R.
Alaggio and D. Di Carlo and N. Corradini and F. Mercolini
and G. M. Milano and M. M. van Noesel and A. Rome and P.
Dall'Igna and K. Pajtler$^*$ and M. Sparber-Sauer and A.
Ferrari and M. Casanova},
title = {{I}nflammatory {M}yofibroblastic {T}umor {W}ith {ROS}1
{G}ene {F}usions in {C}hildren and {Y}oung {A}dolescents.},
journal = {JCO precision oncology},
volume = {7},
number = {7},
issn = {2473-4284},
address = {Alexandria, VA},
publisher = {American Society of Clinical Oncology},
reportid = {DKFZ-2023-02120},
pages = {e2300323},
year = {2023},
abstract = {Inflammatory myofibroblastic tumors (IMTs) are often driven
by anaplastic lymphoma kinase fusions and less frequently by
alternative fusions such as ROS1. We describe the clinical
characteristics, treatment approach, and outcome for a
series of young patients with IMTs and ROS1 alterations.This
was a retrospective, international, multicenter study
analyzing young patients (younger than 21 years) with
ROS1-altered IMTs treated in 10 European referral centers
between 2014 and 2022. Patients were included in the
European pediatric Soft tissue sarcoma Study Group
NRSTS-2005 protocol or registered in the Soft Tissue Sarcoma
Registry. Primary surgery was recommended if a microscopic
radical resection was feasible without mutilation. No
standard systemic treatment protocol was available, but
several medical options were recommended.A total of 19
patients (median age 8.3 years) were included. Most patients
had a biopsy at diagnosis (Intergroup Rhabdomyosarcoma Study
[IRS] I; n = 2, IRS II; n = 1, IRS III biopsy; n = 11, IRS
III resection; n = 3, IRS IV; n = 2). Twelve patients
received neoadjuvant systemic therapy in first line (four
received multiple treatments): high-dose steroids (n = 2),
vinorelbine/vinblastine with methotrexate (n = 6), or ROS1
inhibitors (n = 8). After a median follow-up of 2.8 years
(range, 0.2-13.4), seven patients developed an event. The
3-year event-free survival was $41\%$ $(95\%$ CI, 11 to 71),
and the 3-year overall survival was $100\%.Outcome$ for
ROS1-altered IMTs appears excellent. A complete resection at
diagnosis was often not feasible, and most patients needed
neoadjuvant therapy. Patients who developed a tumor event
could be cured with reinitiation of systemic therapy and/or
surgery. This approach illustrates a switch in treatment
philosophy moving from immediate, often mutilating, surgery
to systemic (targeted) therapy as a bridge to more
conservative surgery later in the treatment course.},
cin = {B062 / HD01},
ddc = {610},
cid = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:37856763},
doi = {10.1200/PO.23.00323},
url = {https://inrepo02.dkfz.de/record/284824},
}