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@ARTICLE{Hein:306174,
author = {S. J. Hein and F. Aus dem Siepen and A. V. Kristen and S.
Schönland and U. Hegenbart and K. Rein$^*$ and H. A. Katus
and N. Frey and J. Furkel$^*$ and M. H. Konstandin and M.
Knoll$^*$},
title = {{P}rofibrotic {B}iomarkers {C}orrelate with {C}linical
{P}resentation and {O}utcome in {C}ardiac {T}ransthyretin
{A}myloidosis.},
journal = {International journal of molecular sciences},
volume = {26},
number = {21},
issn = {1422-0067},
address = {Basel},
publisher = {Molecular Diversity Preservation International},
reportid = {DKFZ-2025-02414},
pages = {10714},
year = {2025},
note = {#LA:E210#},
abstract = {In transthyretin cardiac amyloidosis (ATTR-CA), misfolded
transthyretin accumulates in the myocardium, leading to wall
thickening and interstitial fibrosis. Recently published in
vitro studies revealed direct effects of transthyretin on
the structure, function, and gene expression of cardiac
fibroblasts. Therefore, we hypothesized that biomarkers
known to modulate myocardial remodeling might be clinically
valuable in ATTR-CA and may improve risk stratification in
ATTR-CA. To analyze this hypothesis, we evaluated 14
fibrosis-related biomarkers (EN-RAGE, IGFBP-1, -2, -3, -4,
-6, FGF-23, MMP-2, -7, -9, -13, TIMP-2, -4, and RAGE-AGE) in
125 patients using Luminex multiplex assays. The study
cohort consists of 14 asymptomatic gene carriers
(ATTRv-asymp), 47 symptomatic hereditary (ATTRv-CA), 43
wild-type Transthyretin amyloidosis (ATTRwt) patients, and
21 were healthy controls (ctrl). Associations of fibrotic
biomarkers and clinical routine data with clinical
outcomes-cardiac decompensation (DMP) and
transplantation/death (HTX)-were assessed via hierarchical
cluster analysis, regression, and prediction modeling. We
found that ATTR-CA patients showed distinct biomarker
profiles compared to controls. Several markers (e.g., MMP-7,
RAGE-AGE, IGFBP-1, FGF-23, TIMP-2) were significantly
associated with both endpoints. Cluster analysis identified
a high-risk phenotype (Cluster 2) with worse renal function,
greater myocardial thickening, and elevated NT-proBNP,
hsTNT. Prediction modeling revealed IGFPB-1, -3, -4 and -6
as well as FGF-23, TIMP-2, and RAGE/AGE as the best
predictive parameters for cluster assignment. Taken
together, these findings confirm our hypothesis that
fibrosis-related biomarkers are associated with adverse
outcomes in ATTR-CM. Profibrotic mediators such as IGFBP-1,
FGF-23, and TIMP-2 may, therefore, provide additional
prognostic information beyond established cardiac biomarkers
and may reflect underlying fibrotic remodeling pathways.},
keywords = {Humans / Biomarkers: metabolism / Biomarkers: blood / Male
/ Female / Middle Aged / Amyloid Neuropathies, Familial:
metabolism / Amyloid Neuropathies, Familial: pathology /
Amyloid Neuropathies, Familial: diagnosis / Fibroblast
Growth Factor-23 / Aged / Fibrosis / Prealbumin: metabolism
/ Prealbumin: genetics / Cardiomyopathies: metabolism /
Cardiomyopathies: pathology / Myocardium: pathology /
Myocardium: metabolism / Adult / Prognosis / cardiac
transthyretin amyloidosis (Other) / fibrosis (Other) /
outcome (Other) / remodeling (Other) / risk prediction
(Other) / Biomarkers (NLM Chemicals) / Fibroblast Growth
Factor-23 (NLM Chemicals) / FGF23 protein, human (NLM
Chemicals) / Prealbumin (NLM Chemicals)},
cin = {E210 / HD01},
ddc = {540},
cid = {I:(DE-He78)E210-20160331 / I:(DE-He78)HD01-20160331},
pnm = {315 - Bildgebung und Radioonkologie (POF4-315)},
pid = {G:(DE-HGF)POF4-315},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41226753},
doi = {10.3390/ijms262110714},
url = {https://inrepo02.dkfz.de/record/306174},
}