TY  - JOUR
AU  - Schlenk, Richard
AU  - Weber, D.
AU  - Krzykalla, J.
AU  - Kindler, T.
AU  - Wulf, G.
AU  - Hertenstein, B.
AU  - Salih, H. R.
AU  - Südhoff, T.
AU  - Krauter, J.
AU  - Martens, U.
AU  - Wessendorf, S.
AU  - Runde, V.
AU  - Tischler, H. J.
AU  - Bentz, M.
AU  - Koller, E.
AU  - Heuser, M.
AU  - Thol, F.
AU  - Benner, A.
AU  - Ganser, A.
AU  - Döhner, K.
AU  - Döhner, H.
TI  - Randomized phase-III study of low-dose cytarabine and etoposide + /- all-trans retinoic acid in older unfit patients with NPM1-mutated acute myeloid leukemia.
JO  - Scientific reports
VL  - 13
IS  - 1
SN  - 2045-2322
CY  - [London]
PB  - Macmillan Publishers Limited, part of Springer Nature
M1  - DKFZ-2023-01870
SP  - 14809
PY  - 2023
N1  - #LA:W010#
AB  - 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
KW  - Humans
KW  - Aged
KW  - Etoposide: adverse effects
KW  - Leukemia, Myeloid, Acute: drug therapy
KW  - Leukemia, Myeloid, Acute: genetics
KW  - Cytarabine: adverse effects
KW  - Tretinoin: therapeutic use
KW  - Nuclear Proteins
KW  - Etoposide (NLM Chemicals)
KW  - Cytarabine (NLM Chemicals)
KW  - Tretinoin (NLM Chemicals)
KW  - Nuclear Proteins (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:37684299
C2  - pmc:PMC10491626
DO  - DOI:10.1038/s41598-023-41964-y
UR  - https://inrepo02.dkfz.de/record/282751
ER  -