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Real-world outcomes of venetoclax plus hypomethylating agents in unfit acute myeloid leukaemia: Results of the GIMEMA AML2320 trial.

Created on 18 Aug 2026

Authors

Raffaele Palmieri, Alfonso Piciocchi, Stefano Soddu, Marco Frigeni, Erika Borlenghi, Nicola Fracchiolla, Maria Paola Martelli, Ernesta Audisio, Alessandro Cignetti, Giacomo Gianfaldoni, Calogero Vetro, Nicola di Rienzo, Daniela Esposito, Ambra Di Veroli, Carmine Selleri, Federica Gigli, Antonio Curti, Antonino Mulè, Catello Califano, Sara Bigliardi, Massimo Breccia, Claudio Fozza, Monica Bocchia, Albana Lico, Federico Simonetti, Elisabetta Pierdomenico, Monia Marchetti, Ilaria Cerroni, Francesco Buccisano, Maria Ilaria Del Principe, Luca Maurillo, Giovangiacinto Paterno, Paola Fazi, Marco Vignetti, Giuseppe Rossi, Alessandro Rambaldi, Adriano Venditti

Published in

British journal of haematology. Aug 17, 2026. Epub Aug 17, 2026.

Abstract

Venetoclax (VEN) plus hypomethylating agents (HMAs) represents the standard of care for newly diagnosed acute myeloid leukaemia (AML) patients unfit for intensive chemotherapy, but prospective real-world observational data outside randomized clinical trials remain limited. The Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA) AML2320 is a prospective, multicentre, observational study (NCT04589728) including 193 newly diagnosed unfit AML patients receiving VEN plus azacitidine or decitabine between November 2020 and December 2021. Primary end-point was overall survival (OS); secondary end-points included composite complete remission (cCR), disease-free survival and safety. Median age was 74 years with 42% of patients being ≥75 years. After completing cycle 4, cCR was achieved in 73% of the patients, with 54% responding before cycle 2. After a median follow-up of 23 months, median OS was 13.0 months. cCR achievement within cycle 4 correlated with longer OS (19.1 vs. 9.1 months, p = 0.001). Patients receiving 400 mg VEN without azoles had improved OS compared with those on reduced doses with azoles (18.2 vs. 11.4 months, p = 0.015). An anchored matching-adjusted indirect comparison with the phase III VIALE-A trial (NCT02993523) showed comparable median OS (14.8 months vs. 14.9 months; p = 0.6). The GIMEMA AML2320 trial confirmed the efficacy of VEN/HMAs in a prospective, real-world unfit population. Early remission was associated with improved outcomes.

PMID:
42608172
Bibliographic data and abstract were imported from PubMed on 18 Aug 2026.

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