Marie Sébert
A5048099534- Acute Myeloid Leukemia Research
- Histone Deacetylase Inhibitors Research
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Chronic Myeloid Leukemia Treatments
- Hematopoietic Stem Cell Transplantation
- Multiple Myeloma Research and Treatments
- Otitis Media and Relapsing Polychondritis
- HIV/AIDS drug development and treatment
- Chronic Lymphocytic Leukemia Research
- Cancer therapeutics and mechanisms
- Protein Degradation and Inhibitors
- Acute Lymphoblastic Leukemia research
- Pneumocystis jirovecii pneumonia detection and treatment
- Immunodeficiency and Autoimmune Disorders
- Epigenetics and DNA Methylation
- Retinoids in leukemia and cellular processes
- Cancer Genomics and Diagnostics
- Neutropenia and Cancer Infections
- Hemoglobinopathies and Related Disorders
- Eosinophilic Disorders and Syndromes
- Hematological disorders and diagnostics
- Vascular Anomalies and Treatments
- Lymphoma Diagnosis and Treatment
- CAR-T cell therapy research
- Blood disorders and treatments
Assistance Publique – Hôpitaux de Paris
2014-2025
Université Paris Cité
2015-2025
Hôpital Saint-Louis
2015-2024
Inserm
2010-2024
Délégation Paris 7
2017-2024
Centre National de la Recherche Scientifique
2019-2024
Sorbonne Paris Cité
2023-2024
Institut de recherche Saint-Louis
2019-2023
Groupe Francophone des Myélodysplasies
2010-2023
Hôpital Tenon
2022
A new autoinflammatory syndrome related to somatic mutations of UBA1 was recently described and called VEXAS ('Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome').To describe clinical characteristics, laboratory findings outcomes syndrome.One hundred sixteen patients with were referred a French multicentre registry between November 2020 May 2021. The frequency median parameters vital status, from diagnosis the end follow-up, recorded.The main features found be skin lesions...
TP53-mutated (TP53m) myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) have very poor outcome irrespective of the treatment received, including 40% responses (20% complete remission [CR]) with azacitidine (AZA) alone, short response duration, a median overall survival (OS) approximately 6 months. Eprenetapopt (APR-246), novel first-in-class drug, leads to p53 protein reconformation reactivates its proapoptotic cell-cycle arrest functions.This phase II study assessed safety...
<h3>Importance</h3> VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) is a recently described severe adult-onset autoinflammatory disease that associated with myeloid lineage-restricted ubiquitin-activating enzyme 1 (<i>UBA1</i>) somatic variations primarily affect the skin (Sweet syndrome), cartilage, and bone marrow. Skin symptoms have been poorly described. <h3>Objective</h3> To better describe clinical pathological manifestations their pathophysiology in syndrome....
PURPOSE Secondary myeloid neoplasms (sMNs) remain the most serious long-term complications in patients with aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH). However, sMNs lack specific predictors, dedicated surveillance measures, early therapeutic interventions. PATIENTS AND METHODS We studied a multicenter, retrospective cohort of 1,008 (median follow-up 8.6 years) AA PNH to assess clinical molecular determinants clonal evolution. RESULTS Although none transplanted...
Abstract Acute myeloid leukemia (AML) pathogenesis often involves a mutation in the NPM1 nucleolar chaperone, but bases for its transforming properties and overall association with favorable therapeutic responses remain incompletely understood. Here we demonstrate that an oncogenic mutant form of (NPM1c) impairs mitochondrial function. NPM1c also hampers formation promyelocytic (PML) nuclear bodies (NB), which are regulators fitness key senescence effectors. Actinomycin D (ActD), antibiotic...
Abstract Myelodysplastic syndromes (MDS) are clonal hematopoietic disorders, representing high risk of progression to acute myeloid leukaemia, and frequently associated somatic mutations, notably in the epigenetic regulator TET2 . Natural Killer (NK) cells play a role anti-leukemic immune response via their cytolytic activity. Here we show that patients with MDS clones harbouring mutations gene characterised by phenotypic defects circulating NK cells. Remarkably, from same patient share...
High-risk myelodysplastic syndrome/acute myeloid leukemia patients have a very poor survival after azacitidine failure. Guadecitabine (SGI-110) is novel subcutaneous hypomethylating agent which results in extended decitabine exposure. This multicenter phase II study evaluated the efficacy and safety of guadecitabine high-risk syndrome low blast count acute refractory or relapsing azacitidine. We included 56 with median age 75 years [Interquartile Range (IQR) 69-76]. Fifty-five received at...
Erlotinib was originally developed as an epidermal growth factor receptor (EGFR)-specific inhibitor for the treatment of solid malignancies, yet also exerts significant EGFR-independent antileukemic effects in vitro and vivo. The molecular mechanisms underlying clinical activity erlotinib a standalone agent have not been precisely elucidated. Conversely, preclinical settings, has shown to inhibit constitutive activation SRC kinases mTOR, well synergize with DNA methyltransferase azacytidine...