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@ARTICLE{Schlenk:282751,
author = {R. Schlenk$^*$ and D. Weber and J. Krzykalla$^*$ and T.
Kindler and G. Wulf and B. Hertenstein and H. R. Salih and
T. Südhoff and J. Krauter and U. Martens and S. Wessendorf
and V. Runde and H. J. Tischler and M. Bentz and E. Koller
and M. Heuser and F. Thol and A. Benner$^*$ and A. Ganser
and K. Döhner and H. Döhner},
title = {{R}andomized phase-{III} study of low-dose cytarabine and
etoposide + /- all-trans retinoic acid in older unfit
patients with {NPM}1-mutated acute myeloid leukemia.},
journal = {Scientific reports},
volume = {13},
number = {1},
issn = {2045-2322},
address = {[London]},
publisher = {Macmillan Publishers Limited, part of Springer Nature},
reportid = {DKFZ-2023-01870},
pages = {14809},
year = {2023},
note = {#LA:W010#},
abstract = {The aim of this randomized clinical trial was to evaluate
the impact of all-trans retinoic acid (ATRA) in combination
with non-intensive chemotherapy in older unfit patients (>
60 years) with newly diagnosed NPM1-mutated acute myeloid
leukemia. Patients were randomized (1:1) to low-dose
chemotherapy with or without open-label ATRA 45 mg/m2, days
8-28; the dose of ATRA was reduced to 45 mg/m2, days 8-10
and 15 mg/m2, days 11-28 after 75 patients due to toxicity.
Up to 6 cycles of cytarabine 20 mg/day s.c., bid, days 1-7
and etoposide 100 mg/day, p.o. or i.v., days 1-3 with (ATRA)
or without ATRA (CONTROL) were intended. The primary
endpoint was overall survival (OS). Between May 2011 and
September 2016, 144 patients (median age, 77 years; range,
64-92 years) were randomized (72, CONTROL; 72, ATRA).
Baseline characteristics were balanced between the two study
arms. The median number of treatment cycles was 2 in ATRA
and 2.5 in CONTROL. OS was significantly shorter in the ATRA
compared to the CONTROL arm (p = 0.023; median OS: 5 months
versus 9.2 months, 2-years OS rate: $7\%$ versus $10\%,$
respectively). Rates of CR/CRi were not different between
treatment arms; infections were more common in ATRA beyond
treatment cycle one. The addition of ATRA to low-dose
cytarabine plus etoposide in an older, unfit patient
population was not beneficial, but rather led to an inferior
outcome.The clinical trial is registered at
clinicaltrialsregister.eu (EudraCT Number: 2010-023409-37,
first posted 14/12/2010).},
keywords = {Humans / Aged / Etoposide: adverse effects / Leukemia,
Myeloid, Acute: drug therapy / Leukemia, Myeloid, Acute:
genetics / Cytarabine: adverse effects / Tretinoin:
therapeutic use / Nuclear Proteins / Etoposide (NLM
Chemicals) / Cytarabine (NLM Chemicals) / Tretinoin (NLM
Chemicals) / Nuclear Proteins (NLM Chemicals)},
cin = {W010 / C060},
ddc = {600},
cid = {I:(DE-He78)W010-20160331 / I:(DE-He78)C060-20160331},
pnm = {313 - Krebsrisikofaktoren und Prävention (POF4-313)},
pid = {G:(DE-HGF)POF4-313},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:37684299},
pmc = {pmc:PMC10491626},
doi = {10.1038/s41598-023-41964-y},
url = {https://inrepo02.dkfz.de/record/282751},
}