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Randomized phase-III study of low-dose cytarabine and etoposide + /− all-trans retinoic acid in older unfit patients with NPM1-mutated acute myeloid leukemia

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 chemother...

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Main Authors: Schlenk, Richard Friedrich (Author) , Weber, D. (Author) , Krzykalla, J. (Author) , Kindler, T. (Author) , Wulf, G. (Author) , Hertenstein, B. (Author) , Salih, H. R. (Author) , Südhoff, T. (Author) , Krauter, J. (Author) , Martens, U. (Author) , Wessendorf, S. (Author) , Runde, V. (Author) , Tischler, H. J. (Author) , Bentz, M. (Author) , Koller, E. (Author) , Heuser, M. (Author) , Thol, F. (Author) , Benner, Axel (Author) , Ganser, A. (Author) , Döhner, K. (Author) , Döhner, H. (Author)
Format: Article (Journal)
Language:English
Published: 08 September 2023
In: Scientific reports
Year: 2023, Volume: 13, Pages: 1-9
ISSN:2045-2322
DOI:10.1038/s41598-023-41964-y
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1038/s41598-023-41964-y
Verlag, kostenfrei, Volltext: https://www.nature.com/articles/s41598-023-41964-y
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Author Notes:R.F. Schlenk, D. Weber, J. Krzykalla, T. Kindler, G. Wulf, B. Hertenstein, H.R. Salih, T. Südhoff, J. Krauter, U. Martens, S. Wessendorf, V. Runde, H.J. Tischler, M. Bentz, E. Koller, M. Heuser, F. Thol, A. Benner, A. Ganser, K. Döhner & H. Döhner
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Summary: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.
Item Description:Online veröffentlicht: 08. September 2023
Gesehen am 08.12.2023
Physical Description:Online Resource
ISSN:2045-2322
DOI:10.1038/s41598-023-41964-y

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