Publications by authors named "Charbonnier A"

Introduction: Hematopoietic stem cell transplantation (HCST) is a widely used therapy in the management of hematological malignancies, leading to cytopenias that require transient transfusions. Platelet recovery (PR) following HSCT is assessed by monitoring platelet count (PC). Immature platelet fraction (IPF) is a research parameter offered by Sysmex® on XN series analyzers, enabling rapid diagnostic orientation in the event of thrombocytopenia.

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  • Elderly AML patients (60-75 years) with poor-risk cytogenetics typically have poor outcomes with intensive chemotherapy, but the effectiveness of Venetoclax (VEN) combined with other treatments is being studied.
  • A study at Institut Paoli Calmettes involved comparing 26 patients treated with VEN to a historical cohort of 90 patients treated with intensive chemotherapy, focusing on treatment response and overall survival rates.
  • The findings suggested that VEN showed promising results, with a 69% composite response rate and a median overall survival of 7.9 months, making it a potential alternative to intensive chemotherapy for high-risk elderly patients.
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In the context of T-cell replete haploidentical stem cell transplantation (Haplo-SCT) using post-transplantation cyclophosphamide (PT-Cy), it is still unknown whether peripheral blood (PB) or bone marrow (BM) is the best graft source. While PB is associated with a higher incidence of graft-versus-host disease (GVHD), it may induce a stronger graft-versus-leukemia effect compared to BM, notably in acute myeloid leukemia (AML). From the EBMT registry database, we compared T-cell replete PB (n = 595) versus BM (n = 209) grafts in a large cohort of 804 patients over the age of 60 years who underwent Haplo-SCT with PT-Cy for an AML in first or second complete remission.

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Mutations in spliceosome genes (SRSF2, SF3B1, U2AF1, ZRSR2) correlate with inferior outcomes in patients treated with intensive chemotherapy for Acute Myeloid Leukemia. However, their prognostic impact in patients treated with less intensive protocols is not well known. This study aimed to evaluate the impact of Spliceosome mutations in patients treated with Venetoclax and Azacitidine for newly diagnosed AML.

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Fragrance encapsulates are widely used in consumer care applications such as fabric softeners or other liquid laundry products; they provide multiple benefits, from fragrance protection in the commercial product to a controlled release and improved sensorial experience for the consumers. Polymeric fragrance encapsulates are in the scope of the EU regulation restricting the use of intentionally added microplastic particles, and industry is actively working on innovation programs to find biodegradable alternatives. However, particular attention needs to be paid to claims that a fragrance encapsulation system is biodegradable, because biodegradation test results can vary considerably depending on how a test material is prepared, which can even lead to false-positive biodegradation test results, as shown in our study.

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In patients awaiting an allogeneic haematopoietic stem cell transplantation, platelet transfusion is a risk factor for anti-HLA class I immunization because the resulting donor-specific antibodies complicate the allograft process. The objective of the present study was to determine the feasibility of a novel eplet-based strategy for identifying HLA class I mismatches between potential donors and the recipient when pre-allograft platelet transfusions were required. We included 114 recipient/haploidentical relative pairs.

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The BYOND study evaluated the efficacy and safety of bosutinib 500 mg once daily in patients with chronic myeloid leukemia (CML) resistant/intolerant to prior tyrosine kinase inhibitors (TKIs). These post-hoc analyses assessed the efficacy and safety of bosutinib by resistance or intolerance to prior TKIs (imatinib-resistant vs dasatinib/nilotinib-resistant vs TKI-intolerant), and cross-intolerance between bosutinib and prior TKIs (imatinib, dasatinib, nilotinib), in patients with Philadelphia chromosome-positive chronic phase CML. Data are reported after ≥3 years' follow-up.

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The use of peripheral blood (PB) or bone marrow (BM) stem cells graft in haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis remains controversial. Moreover, the value of adding anti-thymoglobulin (ATG) to PTCy is unknown. A total of 1344 adult patients received an unmanipulated haploidentical transplant at 37 centers from 2012 to 2019 for hematologic malignancy.

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  • Chronic myeloid leukemia (CML) is treated with special pills called tyrosine kinase inhibitors, and one of them is called bosutinib.
  • Bosutinib is used to help adult patients with CML, either as a first treatment or if other treatments haven't worked.
  • Doctors found that bosutinib can cause side effects like stomach problems and liver issues, so a group of experts made guidelines to help manage these side effects and keep patients safe while they are being treated.
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Objectives: To compare the efficacy of venetoclax-azacitidine (VEN-AZA) with AZA in the real-life for patients with first relapsed or refractory acute myeloid leukaemia (R/R AML).

Methods: We retrospectively analysed R/R AML patients treated with VEN-AZA at the Institut Paoli Calmettes between September 2020 and February 2022. We compared them to a historical cohort of patients treated with AZA between 2010 and 2021.

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Background: Failure of gastrointestinal acute graft--host disease (GI-aGvHD) to respond to steroid therapy is associated with limited further therapeutic options. We aimed to assess the safety and efficacy of the first-in-human use of the pooled allogeneic faecal microbiota, MaaT013, for the treatment of steroid-refractory GI-aGvHD.

Methods: This prospective, international, single-arm, phase 2a study reports clinical outcomes from a 24-patient cohort with grade III-IV, steroid refractory GI-aGvHD treated with the pooled allogeneic faecal microbiota MaaT013.

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  • Accelerated phase chronic myeloid leukemia (AP-CML) has a poorer prognosis than chronic phase (CP-CML), prompting researchers to test second-generation tyrosine kinase inhibitors (TKI2) as an initial treatment due to their reduced toxicity.
  • In a study of 69 newly diagnosed patients with AP-CML, various health parameters were measured, revealing worse conditions in those with hematologic AP compared to cytogenetically defined AP.
  • Regardless of the type of TKI2 treatment used, the patients achieved similar rates of clinical response and overall survival, suggesting that TKI2 can effectively manage AP-CML and mitigate the challenges associated with its advanced disease phase.
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Background: Advanced-stage cutaneous T-cell lymphomas (CTCLs) are rare, usually refractory, and fatal diseases. Case series have suggested that allogeneic haematopoietic stem cell transplantation (HSCT) might improve the prognosis of advanced-stage CTCLs. The objective of this study was to investigate the effect of allogeneic HSCT compared with non-HSCT therapy on the outcome of individuals with advanced-stage CTCLs.

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Background: Retrospective studies in hematological unit have suggested that single red blood cell (1-RBC) unit transfusion policy may reduce the number of RBC used without negative clinical impact.

Method: Acute leukemia patients requiring intensive chemotherapy or patients receiving autologous or allogeneic transplantation were randomly assigned to receive either single RBC (1-RBC arm) or double RBC (2-RBC arm) per transfusion with a hemoglobin trigger of 8 g/dL. The primary composite endpoint was the percentage of patients experiencing serious complications, such as a non-hematological adverse event grade ≥ 3 or intensive care admission or death.

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Relapse after allogeneic hematopoietic cell transplantation (allo-HCT) remains a major concern because it is associated with poor survival. A second allo-HCT is a valid option in this situation. During the 13th annual harmonization workshops of the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to update the second allo-HCT recommendations elaborated during the previous workshop (2016).

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  • Late relapse (LR) after a special blood stem cell treatment for leukemia happens in about 4.5% of patients and is studied to find out how well they do after this happens.
  • A study looked at 7,582 patients who had the treatment, and 319 of them had late relapses, mostly after about 38 months.
  • The chances of living longer after a late relapse were about 19.9 months, with many patients getting better after a second treatment.
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Allogeneic hematopoietic stem cell transplantation remains the best curative option for higher-risk myelodysplastic syndrome. The presence of monosomal karyotype and/or complex karyotype abnormalities predicts inferior survival after allo-SCT in MDS patients. Haploidentical allo-SCT has been increasingly used in acute leukemia (AL) and has similar results as using HLA-matched donors, but data on higher-risk MDS is sparse.

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Introduction: Allogeneic hematopoietic stem-cell transplantation (allo-HSCT) remains the best curative option for high-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Unfortunately, it is still associated with a significant risk of relapse due to mechanisms of escape from the control of alloreactive T cells. Repetitive adjuvant donor lymphocyte infusion (DLI), termed prophylactic DLI (proDLI), as an effective strategy in preventing relapse is still debated.

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  • Ruxolitinib is an effective treatment for steroid-refractory graft-versus-host disease (SR-GVHD), with overall response rates exceeding 70%, but it increases the risk of cytomegalovirus (CMV) reactivation.
  • A study of 57 patients showed that starting ruxolitinib significantly raised the chances of first CMV (HR=1.747) and Epstein-Barr virus (EBV) reactivation (HR=2.657).
  • Despite the risk of viral reactivation, ruxolitinib's benefits in treating SR-GVHD are substantial, highlighting the need for careful monitoring of viral loads rather than restricting its use.
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  • * A total of 79 patients participated, and the primary goal was to assess the cumulative molecular response rates over 12 months, with results showing significant rates of deep molecular response at 5 years.
  • * While grade 3 neutropenia was common, it didn't lead to severe infections, and most patients continued the Peg-IFN treatment for a substantial time, resulting in notable molecular response rates after 12 and 24 months.
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The optimal conditioning regimen prior haploidentical stem cell transplantation (Haplo-SCT) with post transplantation cyclophosphamide (PT-Cy) for acute myeloid leukemia (AML) remains unknown. A non-myeloablative conditioning (NMAC) regimen (cyclophosphamide + fludarabine + TBI 2 Gy [CyFluTBI]) is a safe approach, but relapse incidence remains high in this setting. Alternatively, a reduced intensity conditioning (RIC) regimen combining thiotepa and reduced-dose busulfan with fludarabine (TBF) may decrease AML relapse.

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  • Discontinuing tyrosine kinase inhibitors in chronic phase chronic myeloid leukemia (CML) is feasible, but more research is needed on factors that predict recurrence after stopping treatment and the long-term effects of treatment-free remission (TFR).
  • In a study updating data from the STIM2 trial, 199 patients were analyzed, showing a significant percentage (43.4%) achieved TFR at 5 years, but many experienced a loss of major molecular response over time.
  • The timing of molecular recurrence varied, with most recurrences occurring within the first 6 months after stopping treatment, and certain factors such as treatment duration and molecular response levels were linked to recurrence within the first 24 months but not later.
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Venetoclax (VEN) belongs the BH3-mimetic class that selectively targets BCL-2, activating apoptosis. The combination of VEN and azacitidine (AZA) has changed the paradigm of treatment of newly diagnosed (ND) acute myeloid leukemia (AML) patients ineligible for intensive chemotherapy. There is scarce evidence for the use of VEN-AZA for relapsed or refractory (R/R) AML.

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