Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine

Abstract: The prognostic impact of monocytic differentiation in patients with acute myeloid leukemia (AML) receiving venetoclax (Ven) and azacitidine (Aza) remains unclear. In a prospective cohort of 86 newly diagnosed patients with AML treated with Ven-Aza, we used multiparametric flow cytometry (M...

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Main Authors: Lin-Pierre Zhao, Typhaine Dumas-Rivero, Lauren Barette, Lorea Aguinaga, Arij Cheffai, Clémentine Chauvel, Reinaldo Dal Bello, Emmanuel Raffoux, Emmanuelle Clappier, Matthieu Duchmann, Pierre Fenaux, Pierre Lemaire, Stephanie Mathis, Marie Sébert, Lionel Adès, Raphaël Itzykson
Format: Article
Language:English
Published: Elsevier 2025-07-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925002332
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author Lin-Pierre Zhao
Typhaine Dumas-Rivero
Lauren Barette
Lorea Aguinaga
Arij Cheffai
Clémentine Chauvel
Reinaldo Dal Bello
Emmanuel Raffoux
Emmanuelle Clappier
Matthieu Duchmann
Pierre Fenaux
Pierre Lemaire
Stephanie Mathis
Marie Sébert
Lionel Adès
Raphaël Itzykson
author_facet Lin-Pierre Zhao
Typhaine Dumas-Rivero
Lauren Barette
Lorea Aguinaga
Arij Cheffai
Clémentine Chauvel
Reinaldo Dal Bello
Emmanuel Raffoux
Emmanuelle Clappier
Matthieu Duchmann
Pierre Fenaux
Pierre Lemaire
Stephanie Mathis
Marie Sébert
Lionel Adès
Raphaël Itzykson
author_sort Lin-Pierre Zhao
collection DOAJ
description Abstract: The prognostic impact of monocytic differentiation in patients with acute myeloid leukemia (AML) receiving venetoclax (Ven) and azacitidine (Aza) remains unclear. In a prospective cohort of 86 newly diagnosed patients with AML treated with Ven-Aza, we used multiparametric flow cytometry (MFC) to define monoblasts as AML blasts coexpressing ≥2 monocytic markers (CD4, CD36, and CD64) per European LeukemiaNet (ELN) guidelines. Patients with higher monoblasts/CD45+ proportions had lower complete response rates (odds ratio, 0.24; P = .005) and significantly shorter overall survival (OS; 4.0 vs 14.9 months; P = .003). A ≥10% monoblasts/CD45+ threshold, identified via maximally selected rank statistics, stratified patients into monoblasthigh (≥10%) and monoblastlow (<10%) groups. MFC reclassified 20% of French-American-British (FAB) non-M4/5 and 15% of FAB M4/5 cases into monoblasthigh and monoblastlow groups, respectively. Multivariable analysis confirmed monoblasthigh status as an independent adverse prognostic factor for OS (hazard ratio [HR], 1.95; P = .023), with a particularly strong impact in ELN 2024 favorable-risk patients (HR, 2.81; P = .024). Our findings highlight monocytic differentiation, assessed via MFC, as a key predictor of Ven-Aza resistance and poor survival, independent of genetic classification. Given its availability in routine diagnostics, MFC-based monocytic assessment could improve AML risk stratification and treatment decisions in patients eligible for less intensive therapies. This trial was registered at www.clinicaltrials.gov as #NCT05326919.
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spelling doaj-art-d421be660d114bcfab4ccbc7eb48292c2025-08-20T03:51:29ZengElsevierBlood Advances2473-95292025-07-019143556356510.1182/bloodadvances.2024015734Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidineLin-Pierre Zhao0Typhaine Dumas-Rivero1Lauren Barette2Lorea Aguinaga3Arij Cheffai4Clémentine Chauvel5Reinaldo Dal Bello6Emmanuel Raffoux7Emmanuelle Clappier8Matthieu Duchmann9Pierre Fenaux10Pierre Lemaire11Stephanie Mathis12Marie Sébert13Lionel Adès14Raphaël Itzykson15Université Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Seniors, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceUniversité Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Laboratoire d’Hématologie, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Seniors, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Seniors, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Seniors, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Laboratoire d’Hématologie, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Adulte, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Adulte, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceUniversité Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Laboratoire d’Hématologie, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceUniversité Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Laboratoire d’Hématologie, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceUniversité Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Seniors, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Laboratoire d’Hématologie, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceLeukemia Institute Paris Saint-Louis, Paris, France; Laboratoire d’Hématologie, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceUniversité Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Seniors, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceUniversité Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Seniors, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, FranceUniversité Paris Cité, INSERM UMR 1342, Institut de Recherche Saint-Louis, Paris, France; Leukemia Institute Paris Saint-Louis, Paris, France; Service Hématologie Adulte, Assistance Publique des Hôpitaux de Paris, Université Paris Cité, Paris, France; Correspondence: Raphaël Itzykson, Service Hématologie Adultes, Hôpital Saint-Louis, 1 Ave Claude Vellefaux, F-75010 Paris, France;Abstract: The prognostic impact of monocytic differentiation in patients with acute myeloid leukemia (AML) receiving venetoclax (Ven) and azacitidine (Aza) remains unclear. In a prospective cohort of 86 newly diagnosed patients with AML treated with Ven-Aza, we used multiparametric flow cytometry (MFC) to define monoblasts as AML blasts coexpressing ≥2 monocytic markers (CD4, CD36, and CD64) per European LeukemiaNet (ELN) guidelines. Patients with higher monoblasts/CD45+ proportions had lower complete response rates (odds ratio, 0.24; P = .005) and significantly shorter overall survival (OS; 4.0 vs 14.9 months; P = .003). A ≥10% monoblasts/CD45+ threshold, identified via maximally selected rank statistics, stratified patients into monoblasthigh (≥10%) and monoblastlow (<10%) groups. MFC reclassified 20% of French-American-British (FAB) non-M4/5 and 15% of FAB M4/5 cases into monoblasthigh and monoblastlow groups, respectively. Multivariable analysis confirmed monoblasthigh status as an independent adverse prognostic factor for OS (hazard ratio [HR], 1.95; P = .023), with a particularly strong impact in ELN 2024 favorable-risk patients (HR, 2.81; P = .024). Our findings highlight monocytic differentiation, assessed via MFC, as a key predictor of Ven-Aza resistance and poor survival, independent of genetic classification. Given its availability in routine diagnostics, MFC-based monocytic assessment could improve AML risk stratification and treatment decisions in patients eligible for less intensive therapies. This trial was registered at www.clinicaltrials.gov as #NCT05326919.http://www.sciencedirect.com/science/article/pii/S2473952925002332
spellingShingle Lin-Pierre Zhao
Typhaine Dumas-Rivero
Lauren Barette
Lorea Aguinaga
Arij Cheffai
Clémentine Chauvel
Reinaldo Dal Bello
Emmanuel Raffoux
Emmanuelle Clappier
Matthieu Duchmann
Pierre Fenaux
Pierre Lemaire
Stephanie Mathis
Marie Sébert
Lionel Adès
Raphaël Itzykson
Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine
Blood Advances
title Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine
title_full Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine
title_fullStr Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine
title_full_unstemmed Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine
title_short Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine
title_sort prognostic significance of monocytic like phenotype in patients with aml treated with venetoclax and azacytidine
url http://www.sciencedirect.com/science/article/pii/S2473952925002332
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