@article{c8a584ddb2334eb08da9bc47b3b68f3c,
title = "Impact of TP53 Mutation Status in Elderly AML Patients When Adding All-Trans Retinoic Acid or Valproic Acid to Decitabine",
abstract = "In a randomized phase II trial (AMLSG 14-09, NCT00867672) of elderly, newly diagnosed AML patients, ATRA combined with decitabine (DEC) significantly improved the overall response rate (ORR) and survival also in patients with adverse-risk genetics, without adding toxicity. We performed a post hoc analysis to determine the predictive impact of TP53 status. Despite a nominally higher ORR, the clinically meaningful survival benefit when adding ATRA to DEC was diminished, but not completely negated, in TP53-mutated patients. Indeed, 2 out of 14 TP53-mutated patients (14%) randomized to a DEC + ATRA-containing regimen lived for > 36 months. Further studies of ATRA combined with hypomethylating agents appear warranted in non-M3 AML patients ineligible for HMA/venetoclax therapy. Trial Registration: ClinicalTrials.gov identifier: NCT00867672.",
keywords = "AML, ATRA, HMA, TP53, epigenetics",
author = "Helena Bresser and Claudia Schmoor and Olga Grishina and Dietmar Pfeifer and Johanna Thomas and Rehman, {Usama Ur} and Martina Crysandt and Edgar Jost and Felicitas Thol and Michael Heuser and Katharina S G{\"o}tze and Richard F Schlenk and Helmut R Salih and Marcus M Schittenhelm and Gerhard Heil and Carsten Schwaenen and Carsten M{\"u}ller-Tidow and Wolfram Brugger and Andrea K{\"u}ndgen and {de Wit}, Maike and Aristoteles Giagounidis and Sebastian Scholl and Andreas Neubauer and J{\"u}rgen Krauter and Gesine Bug and Annette M May and Ralph W{\"a}sch and Justus Duyster and Konstanze D{\"o}hner and Arnold Ganser and Hartmut D{\"o}hner and Bj{\"o}rn Hackanson and Heiko Becker and Michael L{\"u}bbert",
note = "Publisher Copyright: {\textcopyright} 2024 The Author(s). European Journal of Haematology published by John Wiley & Sons Ltd.",
year = "2024",
doi = "10.1111/ejh.14304",
language = "English",
journal = "European Journal of Haematology",
issn = "0902-4441",
publisher = "John Wiley and Sons Inc.",
}