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@ARTICLE{Jansen:299580,
author = {P. Jansen$^*$ and W. Galetzka and C. M. Thielmann$^*$ and
R. Murali and A. Zaremba$^*$ and F. Standl and G. Lodde$^*$
and I. Möller$^*$ and A. Sucker$^*$ and A. Paschen$^*$ and
E. Hadaschik$^*$ and S. Ugurel$^*$ and L. Zimmer$^*$ and E.
Livingstone$^*$ and D. Schadendorf$^*$ and A. Stang and K.
G. Griewank$^*$},
title = {p{TERT} mutational status is associated with survival in
stage {IV} melanoma patients receiving first-line immune
therapy.},
journal = {European journal of cancer},
volume = {220},
issn = {0959-8049},
address = {Amsterdam [u.a.]},
publisher = {Elsevier},
reportid = {DKFZ-2025-00521},
pages = {115337},
year = {2025},
abstract = {TERT promoter mutations are the most prevalent mutations in
melanoma. In this study, we investigated clinical
characteristics and survival after first line therapies in a
cohort of melanoma patients with known TERT promoter (pTERT)
mutation status.Sequencing data from 2013 to 2021 covering
29 genes and the pTERT status was assessed and 774 melanomas
patients with known pTERT status and clinical data were
analyzed. Progression free survival (PFS) and overall
survival (OS) of 374 melanoma patients in AJCC-stage IV who
received first-line immune checkpoint inhibitors (ICI,
anti-CTLA4 /anti-PD1 combination therapy or anti-PD1
monotherapy) or targeted therapy (TT) were assessed applying
Cox uni-/ multivariable analyses and Kaplan-Meier curves.The
cohort included 573 cutaneous, 69 mucosal, 37 acral and 95
MUP (melanomas of unknown primary) melanoma patients with a
median observational time from first diagnosis to patient
death or censoring of 38.5 months. TERT promoter mutations
were identified in 476 melanomas (61.5 $\%).$ Survival
analysis of 374 patients with stage IV disease undergoing
first-line systemic therapy (ICI or TT) suggested prolonged
PFS and OS for patients with pTERT mutation positive tumors
(pTERT(+)). Particularly, pTERT(+) patients receiving
anti-CTLA4/anti-PD1 therapy showed mPFS of 14.8 months (95
$\%$ CI: 7.1-40.3) and mOS of 105.2 months (95 $\%$ CI:
27.6-not reached) compared to pTERT(-) patients with mPFS of
5.5 months (95 $\%$ CI: 2.7-10.0) and mOS of 14.7 months (95
$\%$ CI: 11.7-24.1).Our findings suggest that presence of a
pTERT mutation in melanomas might favor PFS and OS after
first line ICI with the greatest improvement after receiving
anti-CTLA4 / anti-PD1. If validated in larger prospective
studies, pTERT mutation status may be a valuable prognostic
marker for stage IV melanoma patients.},
keywords = {Immune therapy (Other) / Melanoma (Other) / Mutational
profiling (Other) / PTERT (Other)},
cin = {ED01},
ddc = {610},
cid = {I:(DE-He78)ED01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40056560},
doi = {10.1016/j.ejca.2025.115337},
url = {https://inrepo02.dkfz.de/record/299580},
}