Publications by authors named "Cecile Pautas"

Despite recent refinements in the diagnostic and prognostic assessment of CEBPA mutations in AML, several questions remain open, i.e. implications of different types of basic region leucin zipper (bZIP) mutations, the role of co-mutations and the allelic state.

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  • A study assessed the safety and efficacy of gilteritinib in 167 patients with relapsed/refractory FLT3-mutated acute myeloid leukemia (AML), comparing it to data from the ADMIRAL trial.
  • The key differences in patient characteristics included higher rates of poor performance status and FLT3-TKD mutations, along with more complex treatment histories among participants.
  • The results showed similar rates of complete remission and overall survival between treatment cohorts and highlighted the potential for outpatient treatment benefits for heavily pretreated patients.
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  • Several fusion genes linked to leukemia, like BCR::ABL1 and PML::RARA, have targeted therapies that improve outcomes, but relapse still poses a challenge.
  • Current myeloid next-generation sequencing (NGS) methods often miss detecting these fusion genes, complicating analysis of disease progression and treatment strategies.
  • A new sequencing approach (aCAP-Seq) and bioinformatics tool (HmnFusion) demonstrate high accuracy in detecting these fusions, allowing better understanding of clonal dynamics in leukemia patients and supporting insights into their resistance to treatments.
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  • HSCT recipients have a high risk of severe COVID-19 and show altered immune responses to vaccinations; this study focused on how well they responded to the BNT162b2 mRNA vaccine.
  • A significant number of HSCT recipients were given a third vaccine dose if their antibody levels were low a month after the second dose; six months later, the antibody levels declined but 72% still had protective levels.
  • Factors like immunosuppressive treatment and low lymphocyte counts were linked to reduced antibody levels, and a small number of participants experienced COVID-19 infections, with one resulting in death.
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  • The study examines the real-world use of antifungal prophylaxis (AFP) in patients with acute myeloblastic leukaemia (AML) after they receive induction chemotherapy, highlighting variations across different treatment centers.
  • Out of 677 patients, four AFP strategies were identified, with the group receiving no prophylaxis having the highest incidence of invasive fungal infections (IFI) and a significantly earlier onset compared to those on posaconazole.
  • AFP was found to delay the onset of IFIs and reduce their overall occurrence; however, misidentification of IFIs by investigators affects treatment management, impacting overall patient outcomes and rate of complete remission in AML.
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  • The study looks at how certain unusual features in blood cells, called dysplastic features, affect treatment outcomes in patients with acute myeloid leukemia (AML).
  • Researchers used a machine learning method to analyze data from 190 AML patients and found that most had a complete response to treatment.
  • They discovered that specific cell features like hypolobulated megakaryocytes seemed to predict lower chances of treatment success, which could help doctors decide on better treatment options.
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  • Researchers studied 471 patients aged 60 or older with a type of leukemia called acute myeloid leukemia (AML) to create a tool that predicts how long they might survive after treatment.
  • They found that certain gene mutations in these patients affected their chances of survival, especially for those with poor genetic conditions.
  • The new decision tool can help doctors figure out the best treatment plans based on the patients' genetic info and can be used in future clinical trials.
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In patients with isocitrate dehydrogenase (IDH)-mutated acute myeloid leukemia (AML) treated by intensive chemotherapy (IC), prognostic significance of co-occurring genetic alterations and allogeneic hematopoietic stem cell transplantation (HSCT) are of particular interest with the advent of IDH1/2 mutant inhibitors. We retrospectively analyzed 319 patients with newly diagnosed AML (127 with IDH1, 135 with IDH2R140, and 57 with IDH2R172 mutations) treated with IC in 3 Acute Leukemia French Association prospective trials. In each IDH subgroup, we analyzed the prognostic impact of clinical and genetic covariates, and the role of HSCT.

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The phase 3 ALFA-0701 trial demonstrated improved outcomes with fractionated-dose gemtuzumab ozogamicin (GO) combined with standard chemotherapy vs. standard chemotherapy alone in adults with de novo acute myeloid leukemia (AML). We examined post-transplant outcomes and occurrence of hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) in patients who received hematopoietic stem cell transplantation (HSCT) as follow-up therapy in ALFA-0701.

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A multistage model instructed by a large dataset (knowledge bank [KB] algorithm) has recently been developed to improve outcome predictions and tailor therapeutic decisions, including hematopoietic stem cell transplantation (HSCT) in acute myeloid leukemia (AML). We assessed the performance of the KB in guiding HSCT decisions in first complete remission (CR1) in 656 AML patients younger than 60 years from the ALFA-0702 trial (NCT00932412). KB predictions of overall survival (OS) were superior to those of European LeukemiaNet (ELN) 2017 risk stratification (C-index, 68.

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In this study, we aimed to refine prognostication of older with acute myeloid leukemia (AML) after intensive chemotherapy. Five hundred and nine patients aged 60 years or older (median age, 68 years) were prospectively enrolled in the intensive Acute Leukemia French Association (ALFA)-1200 trial between 2012 and 2016, and 471 patient samples were submitted to multigene analysis. Mutations in any of 8 genes frequently altered in myelodysplastic syndromes (MDS), including ASXL1, SRSF2, STAG2, BCOR, U2AF1, EZH2, SF3B1, and ZRSR2, defined a secondary AML (sAML)-like disease, as reported.

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Measles can be a life-threatening infection in immunocompromised patients, especially after allogeneic hematopoietic cell transplantation (HCT) because of the corresponding loss of immunity. However, measles vaccines are live-attenuated, which is why measles vaccinations are recommended only in seronegative HCT recipients and in specific conditions. However, little data exist on the rates of seroprotection to measles with the current conditioning regimens and in long-term follow-up.

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Acute myeloid leukemia (AML) is a highly heterogeneous disease both in terms of genetic background and response to chemotherapy. Although molecular aberrations are routinely used to stratify AML patients into prognostic subgroups when receiving standard chemotherapy, the predictive value of the genetic background and co-occurring mutations remains to be assessed when using newly approved antileukemic drugs. In the present study, we retrospectively addressed the question of the predictive value of molecular events on the benefit of the addition of gemtuzumab ozogamicin (GO) to standard front-line chemotherapy.

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Background: International guidelines recommend vaccinating allogeneic hematopoietic cell transplant (HCT) recipients at 3 months after transplant, giving 3 doses of pneumococcal conjugate vaccine (PCV) followed by either a dose of 23-valent pneumococcal polysaccharide vaccine (PSV23) or a fourth PCV dose in the case of graft-versus-host disease (GvHD). However, the long-term immunity after this regimen is unknown, and there is no recommendation from 24 months after transplant regarding boosts. Our objective was to assess the antipneumococcal antibody titers and seroprotection rates of allogeneic HCT recipients years after different schedules of vaccination.

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Among 419 consecutive allogeneic hematopoietic cell transplant recipients, we observed 17 (4.0%) cases of toxoplasmosis at a median time of day 45 (range, 6 to 322) after transplant. Seven of these 17 cases occurred before day 30 after transplant.

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Pneumocystis jirovecii pneumonia (PCP) is a life-threatening disease in allogeneic hematopoietic cell transplantation (HCT) recipients. Trimethoprim-sulfamethoxazole (TMP-SMX) is the preferred prophylaxis but has significant toxicity. We assessed 139 consecutive HCT patients for PCP prophylaxis in our center.

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The randomized, phase III ALFA-0701 trial showed that a reduced and fractionated dose of gemtuzumab ozogamicin added to standard front-line chemotherapy significantly improves event-free survival (EFS) in adults with acute myeloid leukemia (AML). Here we report an independent review of EFS, final overall survival (OS), and additional safety results from ALFA-0701. Patients (n=271) aged 50-70 years with AML were randomized to receive conventional front-line induction chemotherapy (3+7daunorubicin+cytarabine) with/without gemtuzumab ozogamicin 3 mg/m on days 1, 4, and 7 during induction.

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Febrile neutropenia (FN) is the main reason for antibiotic prescription in hematology wards where, on the other hand, antibiotic stewardship (AS) is poorly explored. The objectives of the present study were to evaluate (1) the impact of an AS intervention on antibiotic consumption and (2) the applicability and acceptance rate of the intervention and its clinical impact. A persuasive AS intervention based on European Conference on Infection in Leukaemia (ECIL) guidelines for FN was implemented in a high-risk hematology ward in a tertiary referral public university hospital.

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Background: Cytomegalovirus (CMV) infection and disease (CMV episodes) are global concerns after allogeneic hematopoietic stem cell transplantation (HSCT). They affect survival, both by direct and indirect effects. Due to safety issues of current anti-CMV antivirals, long-term CMV prophylaxis is poorly tolerated and the most common strategy to decrease the incidence of CMV disease is preemptive.

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Disease relapse remains the first cause of mortality of hematological malignancies after allogeneic hematopoietic stem cell transplantation (allo-HCT). The risk of recurrence is elevated in patients with high-risk cytogenetic or molecular abnormalities, as well as when allo-HCT is performed in patients with refractory disease or with persistent molecular or radiological (PET-CT scan) residual disease. Within the frame of the 7th annual workshops of the francophone society for bone marrow transplantation and cellular therapy, the working group reviewed the literature in order to elaborate unified guidelines for the prevention and treatment of relapse after allo-HCT.

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