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@ARTICLE{Jahn:282341,
author = {E. Jahn and M. Saadati and P. Fenaux and M. Gobbi and G. J.
Roboz and L. Bullinger and P. Lutsik and A. Riedel$^*$ and
C. Plass$^*$ and N. Jahn and C. Walter and K. Holzmann and
Y. Hao and S. Naim and N. Schreck$^*$ and J. Krzykalla$^*$
and A. Benner$^*$ and H. N. Keer and M. Azab and K. Döhner
and H. Döhner},
title = {{C}linical impact of the genomic landscape and leukemogenic
trajectories in non-intensively treated elderly acute
myeloid leukemia patients.},
journal = {Leukemia},
volume = {37},
number = {11},
issn = {0887-6924},
address = {London},
publisher = {Springer Nature},
reportid = {DKFZ-2023-01725},
pages = {2187-2196},
year = {2023},
note = {2023 Nov;37(11):2187-2196},
abstract = {To characterize the genomic landscape and leukemogenic
pathways of older, newly diagnosed, non-intensively treated
patients with AML and to study the clinical implications,
comprehensive genetics analyses were performed including
targeted DNA sequencing of 263 genes in 604 patients treated
in a prospective Phase III clinical trial. Leukemic
trajectories were delineated using oncogenetic tree modeling
and hierarchical clustering, and prognostic groups were
derived from multivariable Cox regression models. Clonal
hematopoiesis-related genes (ASXL1, TET2, SRSF2, DNMT3A)
were most frequently mutated. The oncogenetic modeling
algorithm produced a tree with five branches with ASXL1,
DDX41, DNMT3A, TET2, and TP53 emanating from the root
suggesting leukemia-initiating events which gave rise to
further subbranches with distinct subclones. Unsupervised
clustering mirrored the genetic groups identified by the
tree model. Multivariable analysis identified FLT3 internal
tandem duplications (ITD), SRSF2, and TP53 mutations as poor
prognostic factors, while DDX41 mutations exerted an
exceptionally favorable effect. Subsequent backwards
elimination based on the Akaike information criterion
delineated three genetic risk groups: DDX41 mutations
(favorable-risk), DDX41wildtype/FLT3-ITDneg/TP53wildtype
(intermediate-risk), and FLT3-ITD or TP53 mutations
(high-risk). Our data identified distinct trajectories of
leukemia development in older AML patients and provide a
basis for a clinically meaningful genetic outcome
stratification for patients receiving less intensive
therapies.},
cin = {B370 / C060},
ddc = {610},
cid = {I:(DE-He78)B370-20160331 / I:(DE-He78)C060-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:37591941},
doi = {10.1038/s41375-023-01999-6},
url = {https://inrepo02.dkfz.de/record/282341},
}