Prognostic impact of DDX41 germline mutations in intensively treated acute myeloid leukemia patients: an ALFA-FILO study.


Journal

Blood
ISSN: 1528-0020
Titre abrégé: Blood
Pays: United States
ID NLM: 7603509

Informations de publication

Date de publication:
18 08 2022
Historique:
received: 07 01 2022
accepted: 01 04 2022
pubmed: 21 4 2022
medline: 23 8 2022
entrez: 20 4 2022
Statut: ppublish

Résumé

DDX41 germline mutations (DDX41MutGL) are the most common genetic predisposition to myelodysplastic syndrome and acute myeloid leukemia (AML). Recent reports suggest that DDX41MutGL myeloid malignancies could be considered as a distinct entity, even if their specific presentation and outcome remain to be defined. We describe here the clinical and biological features of 191 patients with DDX41MutGL AML. Baseline characteristics and outcome of 86 of these patients, treated with intensive chemotherapy in 5 prospective Acute Leukemia French Association/French Innovative Leukemia Organization trials, were compared with those of 1604 patients with DDX41 wild-type (DDX41WT) AML, representing a prevalence of 5%. Patients with DDX41MutGL AML were mostly male (75%), in their seventh decade, and with low leukocyte count (median, 2 × 109/L), low bone marrow blast infiltration (median, 33%), normal cytogenetics (75%), and few additional somatic mutations (median, 2). A second somatic DDX41 mutation (DDX41MutSom) was found in 82% of patients, and clonal architecture inference suggested that it could be the main driver for AML progression. DDX41MutGL patients displayed higher complete remission rates (94% vs 69%; P < .0001) and longer restricted mean overall survival censored at hematopoietic stem cell transplantation (HSCT) than 2017 European LeukemiaNet intermediate/adverse (Int/Adv) DDX41WT patients (5-year difference in restricted mean survival times, 13.6 months; P < .001). Relapse rates censored at HSCT were lower at 1 year in DDX41MutGL patients (15% vs 44%) but later increased to be similar to Int/Adv DDX41WT patients at 3 years (82% vs 75%). HSCT in first complete remission was associated with prolonged relapse-free survival (hazard ratio, 0.43; 95% confidence interval, 0.21-0.88; P = .02) but not with longer overall survival (hazard ratio, 0.77; 95% confidence interval, 0.35-1.68; P = .5).

Identifiants

pubmed: 35443031
pii: S0006-4971(22)00549-3
doi: 10.1182/blood.2021015328
pmc: PMC9389637
doi:

Substances chimiques

DDX41 protein, human EC 3.6.1.-
DEAD-box RNA Helicases EC 3.6.4.13

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

756-768

Commentaires et corrections

Type : CommentIn

Informations de copyright

© 2022 by The American Society of Hematology.

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Auteurs

Nicolas Duployez (N)

Hematology Laboratory, Unité 1277-Cancer Heterogeneity Plasticity and Resistance to Therapies (CANTHER), Centre Hospitalier Universitaire (CHU) de Lille, University of Lille, INSERM, Lille, France.

Laëtitia Largeaud (L)

Hematology Laboratory, CHU de Toulouse-Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse, France.

Matthieu Duchmann (M)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.

Rathana Kim (R)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Laboratory, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.

Julie Rieunier (J)

Hematology Laboratory, CHU de Toulouse-Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse, France.

Juliette Lambert (J)

Hematology Department, Versailles Hospital, Le Chesnay, France.

Audrey Bidet (A)

Hematology Laboratory, CHU de Bordeaux, Bordeaux, France.

Lise Larcher (L)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Laboratory, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.

Jean Lemoine (J)

Hematology Department, Saint Louis Hospital, AP-HP, Paris, France.

François Delhommeau (F)

Sorbonne Université, INSERM, Centre de Recherche Saint-Antoine, CRSA, AP-HP, Laboratoire d'hématologie biologique, Hôpital Saint-Antoine, Paris, France.

Pierre Hirsch (P)

Sorbonne Université, INSERM, Centre de Recherche Saint-Antoine, CRSA, AP-HP, Laboratoire d'hématologie biologique, Hôpital Saint-Antoine, Paris, France.

Laurène Fenwarth (L)

Hematology Laboratory, Unité 1277-Cancer Heterogeneity Plasticity and Resistance to Therapies (CANTHER), Centre Hospitalier Universitaire (CHU) de Lille, University of Lille, INSERM, Lille, France.

Olivier Kosmider (O)

Hematology Laboratory, Cochin Hospital, AP-HP, Paris, France.

Justine Decroocq (J)

Hematology Department, Cochin Hospital, AP-HP, Paris, France.

Anne Bouvier (A)

Hematology Laboratory, CHU Angers, Angers, France.

Yannick Le Bris (Y)

Hematology Biology, Nantes University Hospital, Nantes, France.
CRCINA, INSERM, CNRS, Université de Nantes, Université d'Angers, Nantes, France.

Marlène Ochmann (M)

Hematology Department, CHU Orléans, Orléans, France.

Alberto Santagostino (A)

Hematology Department, CHU Troyes, Troyes, France.

Lionel Adès (L)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Department, Saint Louis Hospital, AP-HP, Paris, France.

Pierre Fenaux (P)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Department, Saint Louis Hospital, AP-HP, Paris, France.

Xavier Thomas (X)

Hematology Department, Hospices Civils de Lyon, Lyon-Sud Hospital, Lyon, France.

Jean-Baptiste Micol (JB)

Hematology Department, Gustave Roussy Institute, University of Paris-Saclay, Villejuif, France.

Claude Gardin (C)

Hematology Department, Avicenne Hospital, AP-HP, Bobigny, France.
Unité 3518, Saint-Louis Institute for Research, Université de Paris, Paris, France.

Raphael Itzykson (R)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Department, Saint Louis Hospital, AP-HP, Paris, France.

Jean Soulier (J)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Laboratory, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.

Emmanuelle Clappier (E)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Laboratory, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.

Christian Recher (C)

Service d'Hématologie, Centre Hospitalier Universitaire de Toulouse, Institut Universitaire du Cancer de Toulouse Oncopole, Université Toulouse III Paul Sabatier, Toulouse, France; and.

Claude Preudhomme (C)

Hematology Laboratory, Unité 1277-Cancer Heterogeneity Plasticity and Resistance to Therapies (CANTHER), Centre Hospitalier Universitaire (CHU) de Lille, University of Lille, INSERM, Lille, France.

Arnaud Pigneux (A)

Hematology Department, CHU de Bordeaux, Bordeaux, France.

Hervé Dombret (H)

Hematology Department, Saint Louis Hospital, AP-HP, Paris, France.
Unité 3518, Saint-Louis Institute for Research, Université de Paris, Paris, France.

Eric Delabesse (E)

Hematology Laboratory, CHU de Toulouse-Institut Universitaire du Cancer de Toulouse-Oncopole, Toulouse, France.

Marie Sébert (M)

Université de Paris, Unité 944/7212-GenCellDi, INSERM and Centre National de la Recherche Scientifique (CNRS), Paris, France.
Hematology Department, Saint Louis Hospital, AP-HP, Paris, France.

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