Outcome of patients with accelerated and blast‐phase myeloproliferative neoplasms not eligible for intensive chemotherapy or allogeneic hematopoietic cell transplantation treated by azacitidine alone or in combination—A FIM study

Accelerated-phase (AP) or blast-phase (BP) myeloproliferative neoplasms (MPNs) are associated with dismal prognosis, with non-curative therapies such as hypomethylating agents (HMAs) considered in patients not eligible for intensive therapy, while some studies advocate for combination therapy with either ruxolitinib (RUXO) or venetoclax (VEN). To assess the relationship between treatment modalities and outcome, herein, we report a multicentric cohort of 149 patients (median age, 75 years) with AP/BP MPN not eligible for intensive therapy and/or allogeneic hematopoietic cell transplantation who received azacitidine (AZA) alone (<i>n</i> = 60) or in combination (<i>n</i> = 89; VEN [<i>n</i> = 51], RUXO [<i>n</i> = 27], or both [<i>n</i> = 9], isocitrate dehydrogenase inhibitors [<i>n</i> = 2]) between January 2019 and October 2023. With a median follow-up of 15 months, the median overall survival of the full cohort was 8.04 months, with a 3-year overall survival (OS) of 13%. Among disease characteristics, OS was lower in patients with BP (6.24 vs. 18.00 months in patients with AP disease, P = 0.03), complex karyotype (6.00 vs. 13.08 months, P = 0.005), and <i>TP53</i> mutations (8.04 vs. 11.04 months, P = 0.009). OS was nonsignificantly higher in patients receiving AZA combinations (10.08 vs. 6.96 months in patients receiving AZA monotherapy, P = 0.12). When analyzing AZA combinations separately, patients who were treated with AZA-RUXO had higher OS (18.00 vs. 9.00 vs. 10.08 months in patients receiving AZA-VEN and AZA-VEN-RUXO, P = 0.015). The improved survival with AZA-RUXO in the absence of complex karyotype and/or <i>TP53</i> mutations warrants further prospective validation. New therapeutic options are urgently needed, especially in patients with complex karyotype and/or <i>TP53</i> mutations.

Authors

Institutions

Publication Details

Journal
HemaSphere
Published
2025-09-01
DOI
https://doi.org/10.1002/hem3.70202
Citations
1
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
1.77
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Outcome of patients with accelerated and blast‐phase myeloproliferative neoplasms not eligible for intensive chemotherapy or allogeneic hematopoietic cell transplantation treated by azacitidine alone or in combination—A FIM study

Guillaume Denis, Anaïse Blouet, Lise Willems, Lydia Roy et al.
1 citations
HemaSphere
Acute Myeloid Leukemia Research
1.77
article

Outcome of patients with accelerated and blast‐phase myeloproliferative neoplasms not eligible for intensive chemotherapy or allogeneic hematopoietic cell transplantation treated by azacitidine alone or in combination—A FIM study

Guillaume Denis, Anaïse Blouet, Lise Willems, Lydia Roy, Jean‐Jacques Kiladjian, Jean‐Christophe Ianotto, Diane Lara, Clémence Mediavilla, Pascal Turlure, Kamel Laribi, Hélène Pasquer, Christophe Nicol, Katell Le Dû, Lina Benajiba, Jean‐Baptiste Robin, Mathieu Wémeau, Elsa Lestang, Thomas Systchenko, Samia Madene, Laurence Legros, Antoine Machet, Geoffroy Venton, Éric Lippert, Damien Luque Paz, Lénaïg Le Clech, Tony Marchand, Corentin Orvain, Suzanne Tavitian, Franck‐Emmanuel Nicolini, Mélanie Mercier, Françoise Boyer, Alberto Santagostino, Clémentine Salvado
article en
1 citations

Abstract

Accelerated-phase (AP) or blast-phase (BP) myeloproliferative neoplasms (MPNs) are associated with dismal prognosis, with non-curative therapies such as hypomethylating agents (HMAs) considered in patients not eligible for intensive therapy, while some studies advocate for combination therapy with either ruxolitinib (RUXO) or venetoclax (VEN). To assess the relationship between treatment modalities and outcome, herein, we report a multicentric cohort of 149 patients (median age, 75 years) with AP/BP MPN not eligible for intensive therapy and/or allogeneic hematopoietic cell transplantation who received azacitidine (AZA) alone (<i>n</i> = 60) or in combination (<i>n</i> = 89; VEN [<i>n</i> = 51], RUXO [<i>n</i> = 27], or both [<i>n</i> = 9], isocitrate dehydrogenase inhibitors [<i>n</i> = 2]) between January 2019 and October 2023. With a median follow-up of 15 months, the median overall survival of the full cohort was 8.04 months, with a 3-year overall survival (OS) of 13%. Among disease characteristics, OS was lower in patients with BP (6.24 vs. 18.00 months in patients with AP disease, P = 0.03), complex karyotype (6.00 vs. 13.08 months, P = 0.005), and <i>TP53</i> mutations (8.04 vs. 11.04 months, P = 0.009). OS was nonsignificantly higher in patients receiving AZA combinations (10.08 vs. 6.96 months in patients receiving AZA monotherapy, P = 0.12). When analyzing AZA combinations separately, patients who were treated with AZA-RUXO had higher OS (18.00 vs. 9.00 vs. 10.08 months in patients receiving AZA-VEN and AZA-VEN-RUXO, P = 0.015). The improved survival with AZA-RUXO in the absence of complex karyotype and/or <i>TP53</i> mutations warrants further prospective validation. New therapeutic options are urgently needed, especially in patients with complex karyotype and/or <i>TP53</i> mutations.

HemaSphereVol. 9(9)
Centre National de la Recherche Scientifique (FR), Inserm (FR), Robert Morris University (US), Université Paris-Est Créteil (FR), Université Paris Cité (FR), Université Nantes Angers Le Mans (FR), Université de Poitiers (FR), Centre Hospitalier de Roubaix (FR), Assistance Publique Hôpitaux de Marseille (FR), Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes Angers (FR), Hôpital Cochin (FR), Centre Hospitalier Universitaire de Tours (FR), Assistance Publique – Hôpitaux de Paris (FR), Institut universitaire du cancer de Toulouse Oncopole (FR), Hôpital Bretonneau (FR), Centre d'Énergétique et de Thermique de Lyon (FR), Fédération Hospitalo-Universitaire, Paris Center for Microbiome Medicine (FR), Centre hospitalier Bretagne Atlantique (FR), Centre Hospitalier Universitaire de Poitiers (FR), Centre Hospitalier Universitaire d'Angers (FR), CIC Rennes (FR), Strasbourg Oncologie Libérale (FR), Centre Hospitalier de Cornouaille (FR), Centre Hospitalier Universitaire de Limoges (FR), Hôpital Cardiologique du Haut-Lévêque (FR), Centre Hospitalier de Troyes (FR), Centre Hospitalier du Mans (FR), Centre Hospitalier de la Côte Basque (FR), Hôpitaux Universitaires Henri-Mondor (FR), Centre Hospitalier Régional Universitaire de Brest (FR), Centre Hospitalier de Rochefort (FR), Hôpital privé du Confluent (FR), Clinic of Hope (BE), Centre Hospitalier de Dax –Côte d’Argent (FR), Hôpital Saint-Louis (FR), Centre Hospitalier des Pays de Morlaix (FR), Centre Hospitalier Universitaire de Rennes (FR), Centre Hospitalier Saint-Nazaire (FR), Bicêtre Hospital (FR), Université d'Angers (FR)
Openalex Percentile: Top 13%
Acute Myeloid Leukemia Research
1.77
DOIOpen Access PDFOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.