Acute graft-versus-host disease (GVHDa) is one of the leading causes of morbidity and mortality after allogeneic hematopoietic stem cell transplant (HSCT) patients. While the first-line consensus treatment has been based on systemic corticosteroid therapy for many years, ruxolitinib has recently been approved and has become the standard second-line treatment. Nevertheless, the effectiveness of ruxolitinib remains limited to 40 % of cortico-resistant patients, raising the crucial question of selecting a third-line treatment.
View Article and Find Full Text PDFJ Allergy Clin Immunol
August 2025
Reconstitution of the T cell compartment is essential in the treatment of several immune disorders. Similarly, individuals with hematological malignancies undergoing allogeneic hematopoietic stem cell transplantation experience prolonged T cell deficiencies, which increase the risk of infections and relapses. Various strategies for addressing T cell deficiencies are based on adoptive T cell therapies.
View Article and Find Full Text PDFLancet Haematol
September 2025
Background: The combination of a hypomethylating agent with donor lymphocyte infusion as maintenance therapy after haematopoietic stem-cell transplantation (HSCT) in acute myeloid leukaemia and myelodysplastic syndrome might reduce the risk of relapse. We aimed to evaluate the activity and safety of oral decitabine and cedazuridine (ASTX727) as maintenance after allogeneic HSCT in patients with acute myeloid leukaemia or myelodysplastic syndrome at very high risk of relapse post-transplantation.
Methods: We conducted a multicentre, single-arm, phase 2 study (GFM-DACORAL-DLI) at 12 centres in France.
Selection of a suitable donor for allogeneic hematopoietic stem cell transplantation (allo-HCT) has mainly relied on human leukocyte antigen matching, and to date, a matched sibling donor (MSD) remains the first choice. However, patients with acute myeloid leukemia (AML) are older and therefore, have older siblings. Haplo-identical donors (HID) are easily available, and offspring are younger than siblings.
View Article and Find Full Text PDFDonor preference for acute myeloid leukemia (AML) patients transplanted in second complete remission (CR2) remains unclear, and hematopoietic cell transplantation (HCT) with post-transplant cyclophosphamide (PTCy) from a haploidentical donor (HAPLO) merits attention. Data of 3878 adult AML patients receiving a first allo-HCT in CR2 from the European Society of Blood and Marrow Transplantation registry between 2010 and 2022 were analyzed. Univariate analyses and Cox regression models were used.
View Article and Find Full Text PDFThe impact of the number of induction cycles required to achieve first complete remission (CR1) on transplant outcomes in adult acute lymphoblastic leukemia (ALL) patients remains unknown. We conducted a retrospective EBMT registry analysis (2000-2022) of ALL patients who underwent transplantation in CR1 after one (n = 2038), two (n = 296), or three or more (n = 110) induction cycles. Median age was 40 years (range 18-73); 79% had B-ALL.
View Article and Find Full Text PDFNEJM Evid
July 2025
Background: We conducted a randomized controlled trial to compare intermediate doses (IDAC) with high doses of cytarabine (HDAC) as postinduction therapy in patients 18 to 60 years of age with newly diagnosed acute myeloid leukemia (AML). The main objectives were to evaluate noninferiority in overall survival (OS) after IDAC and safety.
Methods: Patients 18 to 60 years of age with newly diagnosed AML, except those with core-binding factor, acute promyelocytic, Philadelphia chromosome-positive, or post-myeloproliferative neoplasm AML, were eligible.
Bone Marrow Transplant
September 2025
In the past decades, treatment for acute myeloid leukemia (AML) has advanced, but allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains vital for improving survival in most patients. This retrospective study, conducted on behalf of the Acute Leukemia Working Party of the EBMT, examines the impact of fludarabine dose in reduced-intensity conditioning regimens on clinical outcomes in patients over 50 years old with AML in first complete remission, without chronic kidney disease. We analyzed 1907 patients who underwent allo-HSCT between 2010 and 2022, stratifying them into four fludarabine dose groups: 110-130 mg/m, 140-150 mg/m, 151-160 mg/m, and 170-190 mg/m.
View Article and Find Full Text PDFCutaneous T-cell lymphomas (CTCLs) are rare, usually refractory, and sometimes fatal diseases. Patients presenting with advanced-stage CTCL usually exhibit poor long-term survival outcomes. Only very few treatments have improved progression-free survival (PFS) in advanced CTCL, and no treatment has increased overall survival (OS).
View Article and Find Full Text PDFAm J Hematol
July 2025
There are very limited data regarding the outcomes of elderly patients with acute lymphoblastic leukemia (ALL) who undergo allogeneic hematopoietic stem cell transplantation (alloHSCT). A total of 316 ALL patients aged ≥ 60 years who underwent alloHSCT between 2010 to 2022 were identified in the SFGM-TC registry. The primary objective was to evaluate progression-free survival (PFS), non-relapse mortality (NRM), relapse incidence (RI), and graft-versus-host disease (GvHD)-free relapse-free survival (GRFS), as well as their risk factors.
View Article and Find Full Text PDFSinusoidal obstruction syndrome (SOS) formerly known as Veno-occlusive disease (VOD) is a potentially fatal complication that occurs mainly after haematopoietic cell transplantation, especially allogeneic transplantation. The liver is the principal organ affected, though other organs, such as the lungs, may also be involved to a lesser extent. The condition is characterised by obstruction of the hepatic venules, leading to sinusoidal congestion, hepatic ischaemia and, in severe cases, fulminant liver failure.
View Article and Find Full Text PDFBone Marrow Transplant
July 2025
The role of ABO blood group system mismatch on allogeneic hematopoietic cell transplantation (allo-HCT) outcomes is controversial since current publications of large datasets are lacking. We retrospectively analyzed 30,487 patients transplanted between 2010 and 2021 using the EBMT registry to assess ABO incompatibility's effect on non-relapse mortality (NRM), overall survival (OS), progression-free survival (PFS), relapse incidence (RI), acute GvHD (aGvHD), chronic GvHD (cGvHD), and neutrophil engraftment. Transplantations were classified as ABO-compatible (56.
View Article and Find Full Text PDFHaematologica
April 2025
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View Article and Find Full Text PDFChronic graft versus host disease is a major cause of morbidity after allogeneic haematopoietic cell transplantation. Belumosudil has recently been approved for the treatment of cGVHD refractory after two lines of treatment. However, few data are available to evaluate its efficacy and safety in real life.
View Article and Find Full Text PDFDonor lymphocyte infusion (DLI) prevents acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) relapses following hematopoietic stem cell transplantation. Given the life-threatening toxicities such as graft versus host disease (GVHD), the identification of variables associated with response without toxicities is warranted. We hypothesized that HLA evolutionary divergence (HED), defined by the diversity between two given alleles of the same HLA gene, may be such a factor.
View Article and Find Full Text PDFIntroduction: The increase in the population of immunocompromised patients due to advances in management of end-stage diseases and transplants poses challenges in treating infections caused by multi-drug resistant (MDR) pathogens. Cefiderocol (FDC), a siderophore cephalosporin, has shown efficacy against carbapenem-resistant Gram-negative bacteria.
Methods: This retrospective multicentre study investigated the real-world use of FDC in 114 immunocompromised adults treated for MDR infections in 12 French hospitals (June 2020-November 2023).
Allogeneic hematopoietic stem cell transplantation (alloHSCT) is a potentially curative treatment for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). However, these transplants are complicated by a high rate of relapse in very high cytogenetic risk or refractory diseases. The benefit of this therapeutic strategy for these serious malignant hemopathies could therefore be reassessed.
View Article and Find Full Text PDFAlthough broadly used, consolidative autologous hematopoietic stem cell transplantation (auto-HCT) for relapsed/refractory (R/R) T-cell/histiocyte-rich large B-cell lymphoma (THRLBCL) has never been specifically investigated. Here, we have analyzed outcomes of auto-HCT for THRLBCL compared with diffuse large cell B-cell lymphoma not otherwise specified (DLBCL). Eligible for this retrospective registry study were adult patients with R/R THRLBCL and DLBCL, respectively, who underwent a first auto-HCT in a salvage-sensitive disease status as assessed by positron emission tomography-computed tomography between 2016 and 2021 and were registered with the European Society for Blood and Marrow Transplantation database.
View Article and Find Full Text PDFLeuk Lymphoma
December 2024
HLA-DP permissive mismatches can be assigned a direction according to their immunopeptidome divergence across core and noncore subsets. Noncore permissive graft-versus-host mismatches show significantly reduced risks of relapse without increased nonrelapse mortality compared with allele-matched pairs.
View Article and Find Full Text PDFFor most patients with childhood myelodysplastic syndrome (cMDS), allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative option. In the case of increased blasts (cMDS-IB), the benefit of pretransplant cytoreductive therapy remains controversial. In this multicenter retrospective study, the outcomes of all French children who underwent allo-HSCT for cMDS reported in the SFGM-TC registry between 2000 and 2020 were analyzed ( = 84).
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