%0 Journal Article %A Schlenk, Richard %A Weber, D. %A Krzykalla, J. %A Kindler, T. %A Wulf, G. %A Hertenstein, B. %A Salih, H. R. %A Südhoff, T. %A Krauter, J. %A Martens, U. %A Wessendorf, S. %A Runde, V. %A Tischler, H. J. %A Bentz, M. %A Koller, E. %A Heuser, M. %A Thol, F. %A Benner, A. %A Ganser, A. %A Döhner, K. %A Döhner, H. %T Randomized phase-III study of low-dose cytarabine and etoposide + /- all-trans retinoic acid in older unfit patients with NPM1-mutated acute myeloid leukemia. %J Scientific reports %V 13 %N 1 %@ 2045-2322 %C [London] %I Macmillan Publishers Limited, part of Springer Nature %M DKFZ-2023-01870 %P 14809 %D 2023 %Z #LA:W010# %X 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 %K Humans %K Aged %K Etoposide: adverse effects %K Leukemia, Myeloid, Acute: drug therapy %K Leukemia, Myeloid, Acute: genetics %K Cytarabine: adverse effects %K Tretinoin: therapeutic use %K Nuclear Proteins %K Etoposide (NLM Chemicals) %K Cytarabine (NLM Chemicals) %K Tretinoin (NLM Chemicals) %K Nuclear Proteins (NLM Chemicals) %F PUB:(DE-HGF)16 %9 Journal Article %$ pmid:37684299 %2 pmc:PMC10491626 %R 10.1038/s41598-023-41964-y %U https://inrepo02.dkfz.de/record/282751