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@ARTICLE{Tller:305632,
author = {P. Tüller and T. Kiefer and L. Jabbarli and K. Papaioannou
and K. Schramm$^*$ and N. Barwinski$^*$ and S. Schönberger
and I. B. Brecht and D. T. Schneider and E. Livingstone and
L. Zimmer and K. Fischhuber and A. Junker and S. Ting and C.
Kiewert and S. Göricke and N. Bechrakis and E. Biewald and
P. Ketteler},
title = {{M}elanoma of the {C}horoid and {C}iliary {B}ody in
{C}hildren: {R}emission of {M}etastatic {M}elanoma of the
{C}horoid {A}fter {T}reatment {W}ith {C}hemotherapy and
{I}mmune {C}heckpoint {I}nhibition.},
journal = {Pediatric blood $\&$ cancer},
volume = {nn},
issn = {1545-5009},
address = {New York, NY},
publisher = {Wiley},
reportid = {DKFZ-2025-02274},
pages = {nn},
year = {2025},
note = {epub},
abstract = {Uveal melanoma is the most common malignant primary
intraocular tumor in adults, associated with high mortality.
Pediatric uveal melanoma generally has a more favorable
course. However, when metastasis occurs, therapeutic options
are limited.Clinical presentation, treatment, and outcome of
children with uveal melanoma diagnosed in Germany and
Austria between 2013 and 2024 were analyzed.Since 2013, 12
children were diagnosed with uveal melanoma in Germany and
Austria-nine in the choroid, three in the ciliary body, and
iris. Treatment comprised enucleation (five patients),
external beam radiation with protons (two patients), ocular
brachytherapy (four patients), and endoresection (one
patient). Two patients developed metastasis. A 17-year-old
male with liver metastasis died 30 months after diagnosis. A
3-year-old male with metastatic choroidal melanoma presented
with metastasis 6 months after enucleation. Chemotherapy
followed by nivolumab and ipilimumab led to a complete
response. However, immunotherapy caused insulin-dependent
diabetes mellitus. Genetic testing revealed a pathogenic
constitutional TP53 variant, confirming Li-Fraumeni syndrome
(LFS). At 24 months after diagnosis of metastatic disease,
he remains in complete response.This report underscores the
rarity and diverse presentation of pediatric choroidal
melanoma. Diagnosis of LFS in one patient highlights the
importance of genetic testing for tumor predisposition and
personalized approaches in managing rare pediatric tumors.},
keywords = {Li–Fraumeni syndrome (Other) / diabetes mellitus (Other)
/ ipilimumab (Other) / nivolumab (Other) / uveal melanoma
(Other)},
cin = {B360 / HD01 / ED01},
ddc = {610},
cid = {I:(DE-He78)B360-20160331 / I:(DE-He78)HD01-20160331 /
I:(DE-He78)ED01-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41189077},
doi = {10.1002/pbc.32147},
url = {https://inrepo02.dkfz.de/record/305632},
}