Publications by authors named "Fatiha Chermat"
Article Synopsis
- Membranopathies include blood disorders caused by genetic changes in red blood cell membrane proteins, with hereditary spherocytosis and stomatocytosis being key examples, while CDA II shows similar symptoms.
- Mitapivat, a new drug that activates pyruvate kinase, has shown promise in raising hemoglobin levels and reducing blood cell breakdown in various diseases and is being tested in a trial with around 25 adults who have membranopathies or CDA II.
- The SATISFY trial aims to determine the safety and effectiveness of mitapivat over an 8-week period, with a focus on measuring its impact on hemoglobin, hemolysis, and patient-reported outcomes.
Introduction: Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) are clonal myeloid malignancies, characterized by bone marrow failure leading to cytopenias (and possible myeloproliferation for CMML) and a high propensity to evolve to Acute Myeloid Leukemia (AML).
Objective And Methods: The aim of our retrospective study was to evaluate the clinical and hematological features; the prevalence of MDS subtypes, R-IPSS, and the outcome of 106 Armenian MDS/CMML patients diagnosed over the 2008-2020 period in a single Armenian Hematology center and compare them to French MDS patients included in the GFM registry.
Results: Median age in the Armenian cohort was 64 years (range 19-84) and 55% were males.
Article Synopsis
- CPX-351, a drug combining cytarabine and daunorubicin, shows better results than traditional treatments for secondary acute myeloid leukemia and is being tested for its safety and efficacy in related blood disorders.
- A phase 2 trial included patients with higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia, focusing on those in their first-line treatment, while a second cohort was halted due to low patient enrollment.
- The trial's results indicated a strong response rate, with 87% of participants showing improvement after treatment with CPX-351, backed by data collected from 31 patients during the study period.
Purpose: Hydroxyurea (HY) is a reference treatment of advanced myeloproliferative neoplasms. We conducted a randomized phase III trial comparing decitabine (DAC) and HY in advanced myeloproliferative chronic myelomonocytic leukemias (CMML).
Patients And Methods: Newly diagnosed myeloproliferative CMML patients with advanced disease were randomly assigned 1:1 to intravenous DAC (20 mg/m/d days 1-5) or HY (1-4 g/d) in 28-day cycles.
Article Synopsis
- - The EUROPE phase 2 trial examined how well biomarkers predict the effectiveness of romiplostim (ROM) treatment for patients with lower-risk myelodysplastic neoplasms (LR-MDS) and low platelet counts, involving 77 patients from the EMSCO network.
- - Out of the participants, 42% showed significant improvements in platelet counts, lasting a median of 340 days, while other types of responses (neutrophil and erythroid) were much less common.
- - The study found that while certain mutations (like SRSF2) and baseline hemoglobin levels were linked to positive responses, ROM treatment did not affect the progression of genetic mutations in patients, confirming its safety and efficacy.
Article Synopsis
- MDS-RS patients experience chronic anemia and often become dependent on red blood cell transfusions, with a low risk of developing acute myeloid leukemia.
- In a 6-month study involving 100 transfusion-dependent patients across 12 French centers, researchers examined transfusion patterns, hospitalization frequency, care costs, and related health issues.
- Results showed that 79% of patients had a high transfusion burden, leading to increased hospital visits and significant treatment costs (16,188€ per patient), highlighting the need to evaluate more effective treatment options.
Patient-reported outcomes (PROs) are relevant and valuable end points in the care of patients with myelodysplastic syndromes (MDS). However, a consensus-based selection of PROs for MDS, derived by both patients and hematologists, is lacking. We aimed to develop a core set of PROs for patients with MDS as part of the prospective European LeukemiaNet MDS (EUMDS) Registry.
View Article and Find Full Text PDFThe aim of this study was to characterize a large series of 154 patients with acute promyelocytic leukemia (median age, 53 years; range, 18-90 years) and evaluate real-life outcome after up-front treatment with arsenic trioxide and all-trans retinoic acid. All patients were included in the prospective NAPOLEON registry (NCT02192619) between 2013 and 2019. The acute promyelocytic leukemia was de novo in 91% (n=140) and therapy-related in 9% (n=14); 13% (n=20) of the patients were older than 70 years.
View Article and Find Full Text PDFPurpose: -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.
Myelodysplastic syndrome (MDS) defines a group of heterogeneous hematologic malignancies that often progresses to acute myeloid leukemia (AML). The leading treatment for high-risk MDS patients is azacitidine (Aza, Vidaza), but a significant proportion of patients are refractory and all patients eventually relapse after an undefined time period. Therefore, new therapies for MDS are urgently needed.
View Article and Find Full Text PDFArticle 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.
Article Synopsis
- The study investigated the effectiveness of azacitidine alone versus azacitidine combined with epoetin-β in patients with lower-risk myelodysplastic syndromes who were resistant to previous treatments.
- Out of 98 patients, the likelihood of achieving transfusion independence after six cycles was similar between the two treatment groups (16.3% for azacitidine alone and 14.3% for the combination).
- Key findings highlighted that mutations in the SF3B1 gene were linked to a better response, while certain genetic abnormalities indicated worse overall survival rates, suggesting limited benefit from combining treatments.
Survival after azacitidine (AZA) failure in higher-risk myelodysplastic syndromes (MDS) is poor and new treatment options are needed. Erlotinib, an oral inhibitor of the epidermal-growth-factor-receptor (EGFR), has shown in preclinical models some efficacy in higher risk MDS and acute myeloid leukemia (AML). In this phase I/II trial, 30 patients received 100mg/day (n=5) or 150mg/day (n=25) of Erlotinib orally after primary or secondary resistance to AZA treatment.
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