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@ARTICLE{Stelmach:277279,
author = {P. Stelmach$^*$ and S. Richter and S. Sauer and M. A. Fabre
and M. Gu and C. Rohde and M. Janssen and N. Liebers and R.
Proynova and N. Weinhold and M. S. Raab and H. Goldschmidt
and B. Besenbeck and P. Pavel and S. Laier and A. Trumpp$^*$
and S. Dietrich and G. S. Vassiliou and C. Müller-Tidow},
title = {{C}lonal hematopoiesis with {DNMT}3{A} and {PPM}1{D}
mutations impairs regeneration in autologous stem cell
transplant recipients.},
journal = {Haematologica},
volume = {108},
number = {12},
issn = {0390-6078},
address = {Pavia},
publisher = {Ferrata Storti Foundation},
reportid = {DKFZ-2023-01313},
pages = {3308-3320},
year = {2023},
note = {DKFZ-ZMBH Alliance / 2023 Dec 1;108(12):3308-3320 /
#EA:A010#},
abstract = {Clonal hematopoiesis (CH) is an age-related condition
driven by stem- and progenitor cells harboring recurrent
mutations linked to myeloid neoplasms. Currently, potential
effects on hematopoiesis, stem cell function and
regenerative potential under stress conditions are unknown.
We performed targeted DNA sequencing of 457 hematopoietic
stem cell grafts collected for autologous stem cell
transplantation (ASCT) in myeloma patients and correlated
our findings with high-dimensional longitudinal clinical and
laboratory data (26,510 data points for blood cell
counts/serum values in 25 days around transplantation). We
detected CH-related mutations in 152 patients $(33.3\%).$
Since many patients (n = 54) harbored multiple CH mutations
in one or more genes, we applied a non-negative matrix
factorization (NMF) clustering algorithm to identify genes
that are commonly co-mutated in an unbiased approach.
Patients with CH were assigned to one of three clusters
(C1-C3) and compared to patients without CH (C0) in a gene
specific manner. To study the dynamics of blood cell
regeneration following ASCT, we developed a time-dependent
linear mixed effect model to validate differences in blood
cell count trajectories amongst different clusters. The
results demonstrated that C2, composed of patients with
DNMT3A and PPM1D single and comutated CH, correlated with
reduced stem cell yields and delayed platelet count recovery
following ASCT. Also, the benefit of maintenance therapy was
particularly strong in C2 patients. Taken together, these
data indicate an impaired regenerative potential of
hematopoietic stem cell grafts harboring CH with DNMT3A and
PPM1D mutations.},
cin = {A010 / HD01},
ddc = {610},
cid = {I:(DE-He78)A010-20160331 / I:(DE-He78)HD01-20160331},
pnm = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
pid = {G:(DE-HGF)POF4-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:37381752},
doi = {10.3324/haematol.2023.282992},
url = {https://inrepo02.dkfz.de/record/277279},
}