Publications by authors named "Odile Beyne Rauzy"

Article Synopsis
  • Systemic inflammatory and autoimmune diseases (SIADs) affect 10-20% of patients with myelodysplastic syndrome (MDS), with VEXAS syndrome being a newly identified condition linked to specific genetic mutations.
  • This study aimed to analyze myeloid immune cell types (dendritic cells and monocytes) in MDS patients with and without SIADs, revealing significant reductions in these cells in those with SIADs, particularly in those with VEXAS syndrome.
  • The findings suggest potential causes for the decreased immune cell counts, including movement to areas of inflammation, increased cell death, or issues with cell formation in the bone marrow.
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Objectives: Myelodysplastic syndromes (MDS) and chronic myelomonocytic leukaemia (CMML) are associated with systemic inflammatory and autoimmune diseases (SIADs) in 10-30% of cases. The aims of this study were (i) to evaluate the prevalence of venous thromboembolism VTE in patients presenting with both MDS/CMML and SIADs, (ii) to describe risk factors associated with thrombosis, and (iii) to analyse the impact of VTE on overall survival and transformation to acute myeloid leukaemia in comparison to patients with MDS/CMML-associated SIADs without VTE.

Methods: This retrospective multicentre case-control study was conducted among patients with MDS/CMML and dysimmune disorders and featured in the French retrospective database of the French Network of Dysimmune Disorders Associated with Hemopathies (MINHEMON), diagnosed with MDS/CMML and dysimmune disorders.

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In order to improve the outcome observed with azacitidine (AZA) in higher-risk Myelodysplastic syndrome (MDS), its combination with other drugs in MDS must be evaluated. So far, no combination has not been shown to be more effective than AZA alone. AZA-PLUS was a phase II trial that, in a "pick a winner" approach, randomly assigned patients with higher-risk MDS, CMML and low blast count AML to: AZA; AZA plus lenalidomide; AZA plus Valproic Acid or AZA plus Idarubicin.

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Background: The general practitioner (GP) is central to managing patients with cancer, whose numbers are increasing worldwide. The GP's involvement requires better coordination between involved partners, in particular oncologists and GPs.

Objectives: To conduct a feasibility study of remote participation of GPs in multi-disciplinary consultation meetings (MCMs).

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Data about the presentation and the management of primary immune thrombocytopenia (ITP) in very elderly patients (VEPs; aged ≥80 years) are lacking. The aim of the present study was to describe ITP in this subgroup. The data source was the prospective CARMEN-France registry.

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Objective: Patients with inflammatory myopathies (IM) are known to have an increased risk of developing malignancies. Autoimmune and inflammatory diseases occur in up to 25% of patients with myelodysplastic syndrome (MDS). This study aimed to describe the rare association between IM and MDS.

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Article Synopsis
  • Thrombocytopenia in MDS and CMML:
  • Despite being moderate in prevalence for low-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukaemia (CMML), thrombocytopenia poses significant risks for severe bleeding, and current treatment options remain limited.
  • Efficacy of Eltrombopag (ELT):
  • In a retrospective study involving 61 patients (50 with MDS and 11 with CMML), ELT demonstrated a platelet response in 77% of patients, with a median duration of response lasting about 8 months, and none of the patients who stopped treatment relapsed within a follow-up period.
  • Safety and Comp
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Introduction: The identification of specific molecular signatures and the development of new targeted drugs have changed the paradigm of onco-nephrology, now allowing a multiscale approach of kidney involvement related to hematologic malignancies relying on combined hematologic and molecular assessments. In this study, we aimed to refine the spectrum of kidney disorders associated with chronic myelomonocytic leukemia (CMML) or BCR-ABL-negative myeloproliferative neoplasms (MPNs), 2 very rare conditions scarcely described.

Methods: Case series.

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Myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML) are associated with systemic inflammatory or autoimmune diseases in 10-20 % of cases. Among them, immune thrombocytopenia (ITP) has been reported but large studies assessing this association are missing. Whether such patients have a particular phenotype and require particular management is unclear.

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Purpose: -mutated () 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, and a median overall survival (OS) of approximately 6 months. Eprenetapopt (APR-246), a novel first-in-class drug, leads to p53 protein reconformation and reactivates its proapoptotic and cell-cycle arrest functions.

Patients And Methods: This phase II study assessed the safety and efficacy of eprenetapopt in combination with AZA in untreated high or very high International Prognostic Scoring System-R MDS and AML patients.

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Objective: Hematological treatment decisions in older adults with hematological malignancies are complex. Our objective is to study the impact of a comprehensive geriatric assessment on hematological treatment decision in older patients and the factors associated with change in treatment plan.

Methods: We conducted a cross-sectional analysis of patients aged 65 years and above with hematological malignancies, hospitalized between 2008 and 2019 at the University Cancer Institute of Toulouse.

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Background: Patients with anemia and lower-risk myelodysplastic syndromes in whom erythropoiesis-stimulating agent therapy is not effective generally become dependent on red-cell transfusions. Luspatercept, a recombinant fusion protein that binds transforming growth factor β superfamily ligands to reduce SMAD2 and SMAD3 signaling, showed promising results in a phase 2 study.

Methods: In a double-blind, placebo-controlled, phase 3 trial, we randomly assigned patients with very-low-risk, low-risk, or intermediate-risk myelodysplastic syndromes (defined according to the Revised International Prognostic Scoring System) with ring sideroblasts who had been receiving regular red-cell transfusions to receive either luspatercept (at a dose of 1.

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In response to the complexity of medical care in oncology, 2 years ago, we designed a new teaching method (SPOC, Small Private Online Course) to improve cancer treatment and its management by emphasizing the community-hospital interface. The educational objective of this study was to evaluate after 1 year if the interest for this teaching remained constant over the long term to meet both educational and financial requirements. We designed a questionnaire including 18 questions grouped in 3 main parts describing the profile of the participants, his/her own experience, and the current utilization of the SPOC.

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Progression-free survival (PFS) of patients with lower-risk myelodysplastic syndromes (MDS) treated with red blood cell transfusions is usually reduced, but it is unclear whether transfusion dose density is an independent prognostic factor. The European MDS Registry collects prospective data at 6-monthly intervals from newly diagnosed lower-risk myelodysplastic syndromes patients in 16 European countries and Israel. Data on the transfusion dose density - the cumulative dose received at the end of each interval divided by the time since the beginning of the interval in which the first transfusion was received - were analyzed using proportional hazards regression with time-varying co-variates, with death and progression to higher-risk MDS/acute myeloid leukemia as events.

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Article Synopsis
  • High-risk myelodysplastic syndrome and acute myeloid leukemia patients often experience poor survival rates after treatment with azacitidine, prompting the investigation of the novel drug guadecitabine.
  • In a phase II study involving 56 patients with a median age of 75, guadecitabine showed an 14.3% response rate, with some patients achieving prolonged survival, particularly those with fewer genetic mutations.
  • Overall survival for the group was 7.1 months, with responders living significantly longer, and factors like initial azacitidine failure type and blood demethylation rates influencing survival outcomes.
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Commentary on « Ipilimumab induced vasculitis » by Padda A. et al., J Immunother Cancer.

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Objectives: To assess the reduction of transfusions rate in transfusion-dependent patients with low-risk myelodysplastic syndrome (MDS) with iron overload treated with deferasirox.

Methods: Prospective observational study. Primary endpoint was reduction in transfusion requirements (RTR) at 3 months, (assessed on 8-week period).

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Treatment with azacitidine (AZA) has been suggested to be of benefit for higher-risk myelodysplastic syndrome (HR-MDS) patients with chromosome 7 abnormalities (Abn 7). This retrospective study of 235 HR-MDS patients with Abn 7 treated with AZA (n = 115) versus best supportive care (BSC; n = 120), assessed AZA treatment as a time-varying variable in multivariable analysis. A Cox Regression model with time-interaction terms of overall survival (OS) at different time points confirmed that, while chromosome 7 cytogenetic categories (complex karyotype [CK] versus non-CK) and International Prognostic Scoring System risk (high versus intermediate-2) retained poor prognosis over time, AZA treatment had a favourable impact on OS during the first 3 years of treatment compared to BSC (Hazard ratio [HR] 0·5 P < 0·001 at 1 year, 0·7 P = 0·019 at 2 years; 0·73 P = 0·029 at 3 years).

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Background: Oncology involves complex care and multidisciplinary management of patients; however, misinformation and ineffective communication remain problematic.

Objective: The educational objective of our study was to develop a new teaching method to improve cancer treatment and management by emphasizing the link between hospitals (inpatients) and their surrounding communities (outpatients).

Methods: A team of 22 professionals from public and private institutions developed a small private online course (SPOC).

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