Cord blood (CB) transplantation has fallen into disfavor because of its association with low cell dose and high non-relapse mortality (NRM). However, a phase I to II trial using UM171-expanded CB transplantation in patients with high-risk hematologic malignancies who lacked a suitable donor demonstrated promising results, including prompt neutrophil engraftment, the ability to use smaller, better human leucocyte antigen (HLA)- matched CB units, and a low NRM rate of 5%. This retrospective matched paired analysis was conducted to test the hypothesis that UM171-expanded CB transplantation provides outcomes equivalent or superior to those of conventional hematopoietic stem cell donor sources.
View Article and Find Full Text PDFThe graft-versus-leukaemia effect (GVL) is closely associated with graft-versus-host disease (GVHD) after human leucocyte antigen (HLA)-matched allogeneic stem-cell transplantation (SCT) in acute lymphoblastic leukaemia (ALL). However, there are no data on this association following haploidentical SCT (haploSCT) with post-transplant cyclophosphamide (PTCy). We assessed the impact of acute and chronic GVHD on haploSCT outcomes in 516 adult ALL patients.
View Article and Find Full Text PDFThe role of allogeneic hematopoietic cell transplantation (allo-HCT) in older patients with Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL) is not well established. In this retrospective analysis we evaluated outcomes of 566 patients with median age of 60 (range 55-76) years treated in first complete remission with allo-HCT from either a matched sibling (n = 138), unrelated (n = 343) or haploidentical (n = 85) donor between the years 2016 and 2022. The probability of overall survival (OS) and leukemia-free survival (LFS) at 2 years was 71% and 59.
View Article and Find Full Text PDFSelection 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 PDFBone 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.
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View Article and Find Full Text PDFWhether one or two agents added to post-transplant cyclophosphamide (PTCy) are needed in HLA-matched allogeneic hematopoietic stem cell transplantation (allo-HSCT) with peripheral blood stem cells (PBSC) is debated. We retrospectively compared PTCy in association with a calcineurin inhibitor (PTCy+CNI) or with a CNI plus mycophenolate mofetil (PTCy+CNI+MMF) in adult patients transplanted for acute myeloid leukemia in first complete remission and receiving PBSC in the period from 2010 to 2020. Propensity score matching was performed using exact matching for donor type (related or unrelated) and the nearest neighbor for other variables (i.
View Article and Find Full Text PDFBackground: The role of autologous hematopoietic stem cell transplantation (AHSCT) in adults with Philadelphia chromosome-negative acute lymphoblastic leukemia (Ph-ALL) remains controversial. The aim of this retrospective study was to analyze results of AHSCT and to identify prognostic factors.
Methods: Overall, 700 patients transplanted in first complete remission between the years 1999-2020 were included.
Core-binding factor acute myeloid leukemia (CBF-AML) generally has a favorable prognosis, with allogeneic hematopoietic stem cell transplantation (allo-SCT) recommended for relapsed/ refractory (R/R) cases achieving second complete remission (CR). However, clinical outcomes remain suboptimal for patients who relapse or fail to achieve CR following induction chemotherapy. Allo-SCT in non-CR is a potential strategy for such patients, though supporting evidence in CBF-AML is limited.
View Article and Find Full Text PDFAllogeneic hematopoietic cell transplantation (allo-HCT) remains a curative treatment for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in their first complete remission (CR1). Recent results using the combination of blinatumomab and second- or third-generation tyrosine kinase inhibitors have challenged the necessity of allo-HCT in CR1. Here we assessed real-world changes over time in transplant characteristics and outcomes in adult patients with Ph+ ALL in CR1, using a large dataset from the European Society for Blood and Marrow Transplantation registry.
View Article and Find Full Text PDFPatients (pts) with myelodysplasia-related AML (MR-AML) are now genetically recategorized, with three different groups in the International Consensus Classification: AML with mutated TP53 (TP53-AML), with myelodysplasia-related gene mutations (MR-GM AML), and with myelodysplasia-related cytogenetic abnormalities (MR-CG AML). Moreover, TP53-AML is determined by the presence of an additional complex karyotype (TP53-mut CK and non-CK AML, respectively). Nonetheless, the relevance of this classification to transplantation outcomes is largely unknown.
View Article and Find Full Text PDFWe retrospectively analyzed the impact of conditioning intensity on transplant outcomes according to their cytogenetic/molecular risk in a cohort of 1823 patients with acute myeloid leukemia (AML) and intermediate- or adverse-risk cytogenetics in first complete remission (CR1). These patients received their first hematopoietic stem cell transplantation (HSCT) using post-transplant cyclophosphamide (PTCy). The intermediate-risk cytogenetic group included 1386 (76%) patients, and 608 (34%) had mutated FLT3-ITD.
View Article and Find Full Text PDFBone Marrow Transplant
April 2025
Post-transplant cyclophosphamide (PTCY) is increasingly used as effective graft-versus-host disease (GvHD) prophylaxis in allogeneic hematopoietic-cell transplantation (allo-HCT). However, PTCY is associated with toxicities. Whether patients with specific comorbidities are more vulnerable to cyclophosphamide-induced toxicity is unclear.
View Article and Find Full Text PDFAcute myeloid leukemia (AML) with translocation t(8;16)(p11;p13) represents a rare entity that has been categorized as a disease-defining recurring cytogenetic abnormality with adverse risk in the 2022 European LeukemiaNet classification. This rating was mainly based on a retrospective analysis comprising patients from several large clinical trials, which, however, included only 21 patients treated with allogeneic stem cell transplantation (alloSCT). Therefore, the European Society for Blood and Marrow Transplantation performed a registry study on a larger cohort to evaluate the role of alloSCT in t(8;16) AML.
View Article and Find Full Text PDFInformation on late complications in patients with acute leukemia who have undergone allogeneic hematopoietic cell transplantation (HCT) is limited. We performed a left-truncated analysis of long-term survival in patients with acute leukemia who were alive and disease-free 2 years after HCT. We included 2701 patients with acute lymphoblastic leukemia (ALL) and 9027 patients with acute myeloid leukemia (AML) who underwent HCT between 2005 and 2012.
View Article and Find Full Text PDFBone Marrow Transplant
January 2025
The best donor option for acute myeloid leukemia (AML) patients lacking an HLA-matched donor has remained intensively debated. We herein report the results of a large retrospective registry study comparing hematopoietic cell transplantation (HCT) outcomes between double-unit umbilical cord blood transplantation (dCBT, n = 209) versus 9/10 HLA-matched unrelated donor (UD) with posttransplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis (UD 9/10, n = 270) in patients with AML in first complete remission (CR1). Inclusion criteria consisted of adult patient, AML in CR1 at transplantation, either peripheral blood stem cells (PBSC) from UD 9/10 with PTCy as GVHD prophylaxis or dCBT without PTCy, transplantation between 2013 and 2021, and no in vivo T-cell depletion.
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