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@ARTICLE{Unglaub:293338,
author = {J. M. Unglaub and R. Schlenk$^*$ and J. M. Middeke and S.
W. Krause and S. Kraus and H. Einsele and M. Kramer and S.
Zukunft and J. Kauer and S. Renders and E. Katelari and C.
Schliemann and C. Pabst and T. Luft and P. Dreger and C.
Röllig and M. Bornhäuser and C. Müller-Tidow and T.
Sauer},
title = {{V}enetoclax-based salvage therapy as bridge-to-transplant
is feasible and effective in patients with
relapsed/refractory {AML}.},
journal = {Blood advances},
volume = {9},
number = {2},
issn = {2473-9529},
address = {Washington, DC},
publisher = {American Society of Hematology},
reportid = {DKFZ-2024-01886},
pages = {375-385},
year = {2025},
note = {2025 Jan 28;9(2):375-385},
abstract = {The BCL2-inhibitor Venetoclax (VEN) in combination with
hypomethylating agents (HMA) has been approved for
first-line treatment of acute myeloid leukemai (AML)
patients ineligible for intensive treatment. Emerging Data
suggest that VEN containing treatment strategies may also be
effective in relapsed/refractory (R/R) AML, however,
comparative studies with conventional treatment strategies
for medically fit patients as a bridge-to transplant
strategy are limited. Using propensity score matching (PSM)
analysis, we compared 37 R/R AML patients, who received
VEN-based salvage therapy as bridge to allogeneic
hematopoietic cell transplantation (allo-HCT) with 90
patients from the German Study Alliance Leukemia (SAL) AML
registry, who were treated with non-VEN-containing salvage
therapy according to their treating physician's choice (TPC)
including intensive and non-intensive protocols. The overall
response rate (ORR=CR+CRi) among all VEN patients was
significantly higher compared to the TPC control cohort
$(62\%$ vs. $42\%;$ p=0.049). Overall, $73\%$ of VEN-treated
patients vs. $63\%$ of TPC patients were successfully
bridged to allo-HCT (p =0.41). After a median follow-up of
34.3 months for the VEN cohort and 21.0 months for the TPC
cohort, the median overall-survival (OS) was 15.8 months
$(95\%-CI,$ 10.6-NE) and 10.5 months $(95\%-CI,$ 6.8-19.6)
(p=0.15), respectively. PSM revealed a trend towards
improved OS for VEN patients (HR 0.70; $95\%-CI,$ 0.41-1.22;
p=0.20). Median event free survival (EFS) was significantly
longer in the VEN cohort (8.0 months) compared to the TPC
cohort (3.7 months) (p=0.006). In summary, our data suggests
that VEN-based salvage therapy is a safe and effective
bridge to allo-HCT for this difficult-to-treat AML patient
population.},
cin = {W010},
ddc = {610},
cid = {I:(DE-He78)W010-20160331},
pnm = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
pid = {G:(DE-HGF)POF4-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39293081},
doi = {10.1182/bloodadvances.2024013086},
url = {https://inrepo02.dkfz.de/record/293338},
}