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@ARTICLE{Schlenk:143062,
author = {R. Schlenk and D. Weber and W. Fiedler and H. R. Salih and
G. Wulf and H. Salwender and T. Schroeder and T. Kindler and
M. Lübbert and D. Wolf and J. Westermann and D. Kraemer and
K. S. Götze and H.-A. Horst and J. Krauter and M.
Girschikofsky and M. Ringhoffer and T. Südhoff and G. Held
and H.-G. Derigs and R. Schroers and R. Greil and M.
Grießhammer and E. Lange and A. Burchardt and U. Martens
and B. Hertenstein and L. Marretta and M. Heuser and F. Thol
and V. I. Gaidzik and W. Herr and J. Krzykalla$^*$ and A.
Benner$^*$ and K. Döhner and A. Ganser and P. Paschka and
H. Döhner},
collaboration = {G. A. S. Group},
title = {{M}idostaurin added to chemotherapy and continued
single-agent maintenance therapy in acute myeloid leukemia
with {FLT}3-{ITD}.},
journal = {Blood},
volume = {133},
number = {8},
issn = {0006-4971},
address = {Stanford, Calif.},
publisher = {HighWire Press},
reportid = {DKFZ-2019-00681},
pages = {840 - 851},
year = {2019},
abstract = {Patients with acute myeloid leukemia (AML) and a FLT3
internal tandem duplication (ITD) have poor outcomes to
current treatment. A phase 2 hypothesis-generating trial was
conducted to determine whether the addition of the
multitargeted kinase inhibitor midostaurin to intensive
chemotherapy followed by allogeneic hematopoietic cell
transplantation (alloHCT) and single-agent maintenance
therapy of 12 months is feasible and favorably influences
event-free survival (EFS) compared with historical controls.
Patients 18 to 70 years of age with newly diagnosed AML and
centrally confirmed FLT3-ITD were eligible: 284 patients
were treated, including 198 younger (18-60 years) and 86
older (61-70 years) patients. Complete remission (CR) rate,
including CR with incomplete hematological recovery (CRi)
after induction therapy, was $76.4\%$ (younger, $75.8\%;$
older, $77.9\%).$ The majority of patients in CR/CRi
proceeded to alloHCT $(72.4\%).$ Maintenance therapy was
started in 97 patients $(34\%):$ 75 after alloHCT and 22
after consolidation with high-dose cytarabine (HiDAC).
Median time receiving maintenance therapy was 9 months after
alloHCT and 10.5 months after HiDAC; premature termination
was mainly a result of nonrelapse causes (gastrointestinal
toxicity and infections). EFS and overall survival at 2
years were $39\%$ $(95\%$ confidence interval [CI],
$33\%-47\%)$ and $34\%$ $(95\%$ CI, $24\%-47\%)$ and $53\%$
$(95\%$ CI, $46\%-61\%)$ and $46\%$ $(95\%$ CI, $35\%-59\%)$
in younger and older patients, respectively. EFS was
evaluated in comparison with 415 historical controls treated
within 5 prospective trials. Propensity score-weighted
analysis revealed a significant improvement of EFS by
midostaurin (hazard ratio [HR], 0.58; $95\%$ CI, 0.48-0.70;
P < .001) overall and in older patients (HR, 0.42; $95\%$
CI, 0.29-0.61). The study was registered at
www.clinicaltrials.gov as #NCT01477606.},
cin = {C060},
ddc = {610},
cid = {I:(DE-He78)C060-20160331},
pnm = {312 - Functional and structural genomics (POF3-312)},
pid = {G:(DE-HGF)POF3-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:30563875},
doi = {10.1182/blood-2018-08-869453},
url = {https://inrepo02.dkfz.de/record/143062},
}