Publications by authors named "Leo F Verdonck"

Given the selection of elderly patients with AML in first complete remission (CR1) the advantage of consolidation with allogeneic hematopoietic cell transplantation (HCT) over chemotherapy is still unclear. Newly diagnosed AML patients in CR1 aged 60-75 years were registered and a donor search initiated. After one consolidation cycle, patients with a matched donor were randomized to HCT with fludarabine/lowdose total body irradiation and cyclosporine/mycophenolate mofetil immunosuppression or conventional non-HCT.

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  • - The study investigates the survival outcomes of patients receiving umbilical cord blood transplants (UCBT) across different racial and ethnic groups, focusing on Black, Latinx, White, and Asian patients, as previous research indicated disparities in survival rates.
  • - A retrospective analysis of data from the Center for International Blood and Marrow Transplant Research (CIBMTR) included 983 single and 1529 double UCBT recipients, measuring outcomes like overall survival (OS), disease-free survival, and transplant-related mortality over two years.
  • - Results showed that while overall survival rates varied by race/ethnicity, with Latinx patients having significantly lower OS compared to Blacks, no significant differences were observed in child patients,
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  • - The study analyzed how different types of donors affect outcomes of hematopoietic cell transplantation (HCT) in patients with myelofibrosis, finding that the use of haploidentical donors rose significantly from 3% in 2013 to 19% in 2019.
  • - Among 1,032 patients with chronic-phase myelofibrosis, matched sibling donor HCTs showed better overall survival in the first three months compared to haploidentical and matched unrelated donor HCTs, with notably lower rates of graft failure.
  • - While matched sibling donors had superior early outcomes, there were no significant differences in long-term survival or disease-free survival among the different donor types, suggesting hap
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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy with a poor prognosis and considered incurable with conventional chemotherapy. Small observational studies reported allogeneic hematopoietic cell transplantation (allo-HCT) offers durable remissions in patients with BPDCN. We report an analysis of patients with BPDCN who received an allo-HCT, using data reported to the Center for International Blood and Marrow Transplant Research (CIBMTR).

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  • Researchers created a prognostic model to predict outcomes for patients with myelofibrosis undergoing allogeneic hematopoietic cell transplantation by analyzing data from 623 patients in the U.S. (CIBMTR cohort) from 2000 to 2016.
  • They identified key factors that influence mortality, assigning weighted scores based on age, donor matching, hemoglobin levels, and donor compatibility, which resulted in differing survival rates among low, intermediate, and high score groupings.
  • The model was validated in a European cohort (EBMT), proving effective for predicting overall survival and transplant-related mortality, aiding clinicians in discussing transplantation prospects with myelofibrosis patients.
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Allogeneic hematopoietic cell transplantation (allo-HCT) remains the only curative treatment for myelofibrosis. However, the optimal conditioning regimen either with reduced-intensity conditioning (RIC) or myeloablative conditioning (MAC) is not well known. Using the Center for International Blood and Marrow Transplant Research database, we identified adults aged ≥18 years with myelofibrosis undergoing allo-HCT between 2008-2019 and analyzed the outcomes separately in the RIC and MAC cohorts based on the conditioning regimens used.

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Post-transplantation cyclophosphamide (PTCy) has been shown to effectively control graft-versus-host disease (GvHD) in haploidentical (Haplo) transplantations. In this retrospective registry study, we compared GvHD organ distribution, severity, and outcomes in patients with GvHD occurring after Haplo transplantation with PTCy GvHD prophylaxis (Haplo/PTCy) versus HLA-matched unrelated donor transplantation with conventional prophylaxis (MUD/conventional). We evaluated 2 cohorts: patients with grade 2 to 4 acute GvHD (aGvHD) including 264 and 1163 recipients of Haplo and MUD transplants; and patients with any chronic GvHD (cGvHD) including 206 and 1018 recipients of Haplo and MUD transplants, respectively.

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  • * A study analyzed 313 adult CMML patients who underwent HCT from 2001 to 2017, finding that higher prognostic scoring (CPSS and CPSS-Mol) correlated with poorer overall survival rates.
  • * Common mutations like ASXL1, TET2, and TP53 were linked to adverse outcomes, with TP53 mutations specifically showing an increase in relapse rates, though their impact should be viewed cautiously due to their infrequency in CMML cases.
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  • T cell prolymphocytic leukemia (T-PLL) is a rare and aggressive cancer with limited treatment options and poor survival rates, prompting this study to evaluate allogeneic hematopoietic cell transplantation (alloHCT) outcomes in affected patients.* -
  • The analysis utilized data from 266 T-PLL patients who underwent alloHCT from 2008 to 2018, revealing a 4-year overall survival rate of 30% and highlighting significant factors affecting survival, including the conditioning regimen and patient age.* -
  • Findings indicated that myeloablative conditioning and poor performance status lead to worse survival and increased treatment-related mortality, while stable disease or progression correlated with a higher relapse risk, suggesting
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  • Matched sibling donors (MSDs) are generally preferred for allogeneic hematopoietic cell transplantation in myelodysplastic syndrome, but it's uncertain if older MSDs yield better outcomes than younger unrelated donors (MUDs).
  • The study aimed to determine if using younger MUDs leads to better disease-free survival and lower relapse rates compared to older MSDs.
  • Results showed that disease-free survival rates were significantly lower for older MSDs compared to younger MUDs, although overall survival did not show a significant difference between the two groups.
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Cytogenetic and molecular abnormalities are known to influence post-transplant outcomes in acute myeloid leukemia (AML) but data assessing the prognostic value of combined genetic models in the HCT setting are limited. We developed an adapted European LeukemiaNet (aELN) risk classification based on available genetic data reported to the Center for International Blood and Marrow Transplant Research, to predict post-transplant outcomes in 2289 adult AML patients transplanted in first remission, between 2013 and 2017. Patients were stratified according to aELN into three groups: favorable (Fav, N = 181), intermediate (IM, N = 1185), and adverse (Adv, N = 923).

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Little is known about whether risk classification at diagnosis predicts post-hematopoietic cell transplantation (HCT) outcomes in patients with acute myeloid leukemia (AML). We evaluated 8709 patients with AML from the CIBMTR database, and after selection and manual curation of the cytogenetics data, 3779 patients in first complete remission were included in the final analysis: 2384 with intermediate-risk, 969 with adverse-risk, and 426 with KMT2A-rearranged disease. An adjusted multivariable analysis detected an increased risk of relapse for patients with KMT2A-rearranged or adverse-risk AML as compared to those with intermediate-risk disease (hazards ratio [HR], 1.

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  • - The study analyzed the outcomes of haploidentical hematopoietic cell transplantation (HCT) using posttransplant cyclophosphamide (PTCy) for adults with acute lymphoblastic leukemia (ALL) in remission, comparing it with other donor types like HLA-matched sibling, unrelated, and umbilical cord blood.
  • - Results showed that overall survival (OS), leukemia-free survival (LFS), and relapse rates were generally similar across donor types; however, haploidentical HCT had lower rates of chronic graft-versus-host disease (cGVHD) than matched donors.
  • - The findings suggest that haploidentical HCT with PTCy is a preferable alternative for adults with
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Patients who develop therapy-related myeloid neoplasm, either myelodysplastic syndrome (t-MDS) or acute myelogenous leukemia (t-AML), have a poor prognosis. An earlier Center for International Blood and Marrow Transplant Research (CIBMTR) analysis of 868 allogeneic hematopoietic cell transplantations (allo-HCTs) performed between 1990 and 2004 showed a 5-year overall survival (OS) and disease-free survival (DFS) of 22% and 21%, respectively. Modern supportive care, graft-versus-host disease prophylaxis, and reduced-intensity conditioning (RIC) regimens have led to improved outcomes.

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Reduced-intensity conditioning (RIC) regimens developed to extend the use of allogeneic hematopoietic stem cell transplantation (HSCT) to older patients have resulted in encouraging outcomes. We aimed to compare the 2 most commonly used RIC regimens, i.v.

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Acute myeloid leukemia (AML) patients often undergo allogeneic hematopoietic cell transplantation (alloHCT) in first complete remission (CR). We examined the effect of depth of clinical response, including incomplete count recovery (CRi) and/or measurable residual disease (MRD), in patients from the Center for International Blood and Marrow Transplantation Research (CIBMTR) registry. We identified 2492 adult patients (1799 CR and 693 CRi) who underwent alloHCT between January 1, 2007 and December 31, 2015.

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  • Myeloablative conditioning (MAC) generally leads to lower relapse rates than reduced-intensity conditioning (RIC) for patients with acute myelogenous leukemia (AML) and myelodysplastic syndromes (MDS) after allogeneic hematopoietic cell transplantation (HCT).
  • In patients classified with low/intermediate-risk Disease Risk Index (DRI), RIC resulted in lower nonrelapse mortality but higher relapse rates, resulting in worse disease-free survival compared to MAC.
  • For high/very high-risk DRI patients, both RIC and MAC showed similar disease-free survival outcomes, indicating that MAC is preferred for low/intermediate-risk patients, while new, less toxic MAC options are needed for high-risk patients
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Comparative outcomes of allogeneic hematopoietic cell transplantation (HCT) for BCR-ABL1- myeloproliferative neoplasms (MPNs) in blast phase (MPN-BP) vs de novo acute myeloid leukemia (AML), and AML with prior myelodysplastic syndromes (MDSs; post-MDS AML), are unknown. Using the Center for International Blood and Marrow Transplant Research (CIBMTR) database, we compared HCT outcomes in 177 MPN-BP patients with 4749 patients with de novo AML, and 1104 patients with post-MDS AML, using multivariate regression analysis in 2 separate comparisons. In a multivariate Cox model, no difference in overall survival (OS) or relapse was observed in patients with MPN-BP vs de novo AML with active leukemia at HCT.

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There is a lack of large comparative study on the outcomes of reduced intensity conditioning (RIC) in acute myeloid leukemia (AML) transplantation using fludarabine/busulfan (FB) and fludarabine/melphalan (FM) regimens. Adult AML patients from Center for International Blood and Marrow Transplant Research who received first RIC allo-transplant between 2001 and 2015 were studied. Patients were excluded if they received cord blood or identical twin transplant, total body irradiation in conditioning, or graft-versus-host disease (GVHD) prophylaxis with in vitro T-cell depletion.

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Allogeneic hematopoietic cell transplantation (HCT) is the only curative therapy for myelofibrosis (MF). In this large multicenter retrospective study, overall survival (OS) in MF patients treated with allogeneic HCT (551 patients) and without HCT (non-HCT) (1377 patients) was analyzed with Cox proportional hazards model. Survival analysis stratified by the Dynamic International Prognostic Scoring System (DIPSS) revealed that the first year of treatment arm assignment, due to upfront risk of transplant-related mortality (TRM), HCT was associated with inferior OS compared with non-HCT (non-HCT vs HCT: DIPSS intermediate 1 [Int-1]: hazard ratio [HR] = 0.

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Purpose: There is no consensus on the best choice of an alternative donor (umbilical cord blood [UCB], haploidentical, one-antigen mismatched [7/8]-bone marrow [BM], or 7/8-peripheral blood [PB]) for hematopoietic cell transplantation (HCT) for patients lacking an HLA-matched related or unrelated donor.

Methods: We report composite end points of graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) and chronic GVHD (cGVHD)-free relapse-free survival (CRFS) in 2,198 patients who underwent UCB (n = 838), haploidentical (n = 159), 7/8-BM (n = 241), or 7/8-PB (n = 960) HCT. All groups were divided by myeloablative conditioning (MAC) intensity or reduced intensity conditioning (RIC), except haploidentical group in which most received RIC.

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Cytogenetic risk stratification at diagnosis has long been one of the most useful tools to assess prognosis in acute lymphoblastic leukemia (ALL). To examine the prognostic impact of cytogenetic abnormalities on outcomes after allogeneic hematopoietic cell transplantation, we studied 1731 adults with Philadelphia-negative ALL in complete remission who underwent myeloablative or reduced intensity/non-myeloablative conditioning transplant from unrelated or matched sibling donors reported to the Center for International Blood and Marrow Transplant Research. A total of 632 patients had abnormal conventional metaphase cytogenetics.

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