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In this week's episode we’ll learn more about the ASCEND study, which investigated first-line asciminib in chronic phase chronic myeloid leukemia; a new risk score for myeloid neoplasm transformation in patients with clonal cytopenia of undetermined significance; and use of RHD genotyped D-positive blood transfusions in patients with sickle cell and unexpected anti-D antibodies.
Featured Articles
Asciminib Monotherapy as Frontline Treatment of Chronic Phase Chronic Myeloid Leukaemia - Results from the ASCEND StudyRisk Prediction for Clonal Cytopenia: Multicenter Real-World Evidence Genotyped RhD+ red cells for D-positive patients with sickle cell disease with conventional RHD and unexpected anti-D -
In this week's episode we'll learn about the updated phase 3 results on fixed-dose venetoclax plus obinutuzumab in previously untreated CLL. Then, we'll hear about dual epi-immunotherapy in classical Hodgkin lymphoma. Finally we'll explore redefining risk of treatment failure in CML.
Featured Articles:
Venetoclax-obinutuzumab for previously untreated chronic lymphocytic leukemia: 6-year results of the randomized phase 3 CLL14 studyEpigenetic agents plus anti-PD-1 reprogram the tumor microenvironment and restore antitumor efficacy in Hodgkin lymphomaA predictive model for therapy failure in patients with chronic myeloid leukemia receiving tyrosine kinase inhibitor therapy -
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In this week's episode we’ll learn more about the role of ERG loss-of-function variants in bone marrow failure and hematological malignancies, a proposed pathway linking factor 11 activation to increased vascular permeability during inflammation, and new clinical criteria for diagnosing anemia, defining transfusion-dependence, and evaluating anemia treatment responses in patients with myelofibrosis.
Featured Articles:
Germ line ERG haploinsufficiency defines a new syndrome with cytopenia and hematological malignancy predispositionCoagulation factor XI regulates endothelial cell permeability and barrier function in vitro and in vivoProposals for revised International Working Group–European LeukemiaNet criteria for anemia response in myelofibrosis -
In this week's episode, we'll learn about the role of a platelet-specific glycoprotein receptor in abdominal aortic aneurysm formation, the influence of TP53 mutations on outcomes for patients with 5q-deleted myelodysplastic syndrome, and a rational combination treatment intended to produce more durable responses in patients with refractory B-cell acute lymphoblastic leukemia who are treated with CAR T-cell therapy.
Featured Articles:
Soluble glycoprotein VI predicts abdominal aortic aneurysm growth rate and is a novel therapeutic targetInfluence of TP53 gene mutations and their allelic status in myelodysplastic syndromes with isolated 5q deletionA phase 1 clinical trial of NKTR-255 with CD19-22 CAR T-cell therapy for refractory B-cell acute lymphoblastic leukemia -
In this week's episode we'll learn about refining risk stratification in T-cell acute lymphoblastic leukemia, or ALL. After that, we'll discuss a novel gene therapy approach in severe alpha-thalassemia. Investigators describe an innovative mouse model and an effective gene therapy approach, renewing prospects for the development of novel strategies to treat this disease. Finally, we'll hear about how genomic profiling has helped identify subgroups associated with distinct clinical phenotypes and outcomes in the molecular taxonomy of myelodysplastic syndromes, or MDS.
Featured Articles
NGS-based stratification refines the risk stratification in T-ALL and identifies a very-high-risk subgroup of
patientsUse of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral
gene therapyMolecular taxonomy of myelodysplastic syndromes and its clinical implications -
In this week's episode we’ll discuss a novel tripartite fusion drives treatment resistance in acute promyelocytic leukemia. In some patients with atypical APL, these novel retinoic acid receptor gene fusions result in truncation of the ligand binding domain of the retinoic acid receptor protein, resulting in non-responsiveness to treatment with all-trans retinoic acid. After that: managing immune thrombotic thrombocytopenia or iTTP without therapeutic plasma exchange, or TPE. Finally, hope for motherhood after allogeneic HCT.
Featured Articles:
Critical role of tripartite fusion and LBD truncation in certain RARA- and all RARG-related atypical APLManagement of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchangeHope for motherhood: pregnancy after allogeneic hematopoietic cell transplantation (a national multicenter study) -
In this week's episode we’ll discuss the safety and efficacy of pirtobrutinib with or without rituximab in relapsed/refractory CLL; learn more about erythroid 5ALA synthesis disorders and their conditional synthetic lethal dependency on pyridoxine; and discuss how targeting PKC alpha alleviates iron overload in diabetes and hemochromatosis through the inhibition of ferroportin.
Featured Articles:
Fixed-duration pirtobrutinib plus venetoclax with or without rituximab in relapsed/refractory CLL: Phase 1b BRUIN trialMurine models of erythroid 5ALA synthesis disorders and their conditional synthetic lethal dependency on pyridoxineTargeting PKCα alleviates iron overload in diabetes and hemochromatosis through the inhibition of ferroportin -
In this week's episode we'll discuss outcomes following hematopoietic stem cell transplantation in pediatric patients with Fanconi anemia. Then, we'll learn about how new research shows that the transcription factor Foxo1, commonly associated with glucose metabolism, regulates hepcidin expression and systemic iron homeostasis. Finally, in Latin America: using clinical networks to improve outcomes in patients with acute promyelocytic leukemia. After clinical networks were established, survival and relapse rates improved substantially, highlighting the effectiveness of this unique intervention strategy in low- and middle-income countries.
Featured Articles:
Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemiaFoxo1 is an iron-responsive transcriptional factor regulating systemic iron homeostasisClinical networking results in continuous improvement of the outcome of patients with acute promyelocytic
leukemia -
In this week's episode we’ll discuss how CD8+ T-cell differentiation and dysfunction inform treatment response in acute myeloid leukemia; learn more about the effect of ATM germline pathogenic variants on the outcomes in children with ataxia-telangiectasia and hematological malignancies; and discuss the preclinical efficacy of a potent, selective menin-KMT2A inhibitor JNJ-75276617 in KMT2A- and NPM1-altered leukemias.
Featured Articles:
CD8+ T-cell differentiation and dysfunction inform treatment response in acute myeloid leukemiaATM germ line pathogenic variants affect outcomes in children with ataxia-telangiectasia and hematological
malignanciesPreclinical efficacy of the potent, selective menin-KMT2A inhibitor JNJ-75276617 (bleximenib) in
KMT2A- and NPM1-altered leukemias -
In this week's episode we'll discuss HA-1-targeted T-cell receptor T-cell therapy for recurrent leukemia after hematopoietic stem cell transplantation. Next, we'll learn about how 4D intravital imaging in mice reveals the key role of platelets as a source of procoagulant membranes in hemostasis. Finally, we'll hear about modifications to a common induction and maintenance treatment for treatment-naïve mantle-cell lymphoma.
Featured Articles
HA-1–targeted T-cell receptor T-cell therapy for recurrent leukemia after hematopoietic stem cell
transplantation4D intravital imaging studies identify platelets as the predominant cellular procoagulant surface in a mouse
hemostasis modelRandomized study of induction with bendamustine-rituximab ± bortezomib and maintenance with
rituximab ± lenalidomide for MCL -
In this week's episode we’ll discuss the outcomes of younger patients with mantle cell lymphoma experiencing late relapse; learn more about mass spectrometry-based assessment of M-protein in peripheral blood during maintenance therapy in multiple myeloma and discuss in vivo CAR T-cell generation in non-human primates using lentiviral vectors.
Featured Articles:
Outcomes of younger patients with mantle cell lymphoma experiencing late relapse (>24 months): the LATE-POD studyMass spectrometry–based assessment of M protein in peripheral blood during maintenance therapy in
multiple myelomaIn vivo CAR T-cell generation in nonhuman primates using lentiviral vectors displaying a multidomain fusion ligand -
Introduced by Associate Editor Dr. Thomas Ortel, this bonus episode discusses the Review Series on Factor XI, published in volume 143 issue 15 of Blood Journal. Dr. Ortel is joined by contributing authors Owen McCarty, PhD, Gili Kenet, MD, and David Gailani, MD.
Review Series Articles:
Introduction to a review series on factor XIBiology of factor XITargeting factor XI and factor XIa to prevent thrombosisFactor XI deficiency: phenotypic age-related considerations and clinical approach towards bleeding risk assessment -
In this week's episode we'll learn how cytomegalovirus infection early in life depletes preleukemic cells in a mouse model of B-cell acute lymphoblastic leukemia. After that we'll discuss new research, where GVHD targets organoid-forming bile duct stem cells in a TGF-beta-dependent manner. Conversely, a TGF-beta inhibitor protects these stem cells against GVHD and mitigates biliary dysfunction. Finally, we'll hear about the seven-year outcomes for venetoclax-ibrutinib in relapsed or refractory mantle cell lymphoma. In addition to long-term survival benefits, researchers report durable treatment-free remissions and effective retreatment in patients with MRD-negative complete responses.
Featured Articles:
Early-life infection depletes preleukemic cells in a mouse model of hyperdiploid B-cell acute lymphoblastic
leukemiaGVHD targets organoid-forming bile duct stem cells in a TGF-β–dependent mannerSeven-year outcomes of venetoclax-ibrutinib therapy in mantle cell lymphoma: durable responses and
treatment-free remissions -
In this week's episode we'll learn about iron, HFE hemochromatosis, and infections. In this large, population-based study, both high and low levels of plasma iron and transferrin saturation were associated with increased risks of infection. Then, we'll discuss how bispecific antibodies improve CAR T-cell response in B-cell malignancies. In-vitro and in-vivo data demonstrate enhanced therapeutic efficacy when a CD20-directed bispecific antibody is given in combination with CD19-directed CAR-T cells. Finally, we'll hear about determinants of outcome in NPM1-mutated AML. In a large series of patients with NPM1-mutated AML, investigators identified several variables beyond FLT3-ITD that adversely impacted outcomes.
Featured Articles:
Iron, hemochromatosis genotypes, and risk of infections: a cohort study of 142 188 general population individualsMolecular, clinical, and therapeutic determinants of outcome in NPM1-mutated AMLCD20-bispecific antibodies improve response to CD19-CAR T cells in lymphoma in vitro and CLL in vivo models -
In this week's episode we’ll discuss how immune fitness impacts response to teclistamab in relapsed/refractory multiple myeloma; learn more about a new mechanism of resistance to asciminib conferred by the BCR::ABL1 M244V mutation, and discuss the impact of hematopoietic cell transplantation on myocardial fibrosis in young patients with sickle cell disease.
Featured Articles:
Correlation of immune fitness with response to teclistamab in relapsed/refractory multiple myeloma in MajesTEC-1BCR::ABL1 kinase N-lobe mutants confer moderate to high degrees of resistance to asciminibImpact of hematopoietic cell transplantation on myocardial fibrosis in young patients with sickle cell disease -
In this week's episode we’ll discuss the safety and efficacy of decitabine in older patients with AML; learn how heme-induced loss of renovascular endothelial protein C receptor promotes chronic kidney disease in sickle mice, and discuss the outcomes of treatment with HLA-mismatched HSCT with TCRab/CD19 lymphocyte depletion or post-HSCT cyclophosphamide for inborn errors of immunity.
Featured Articles:
Decitabine in older patients with AML: Quality of life results of the EORTC-GIMEMA-GMDS-SG randomized phase III trial Heme induced loss of renovascular endothelial protein C receptor promotes chronic kidney disease in sickle mice Outcomes of HLA-mismatched HSCT with TCRab/CD19 depletion or post-HSCT cyclophosphamide for inborn errors or immunity -
In this week's episode we'll discuss minimal residual disease as an intermediate clinical endpoint for multiple myeloma. Then, we'll learn about IPSS-R downstaging before transplant in MDS. Finally we'll hear about the connection between hereditary angioedema and venous thromboembolism.
Featured Articles:
EVIDENCE meta-analysis: evaluating minimal residual disease as an intermediate clinical endpoint for multiple myeloma Does IPSS-R down-staging before transplantation improve the prognosis of patients with Myelodysplastic Neoplasms? Increased risk of venous thromboembolism [or VTE] in young and middle-aged individuals with hereditary angioedema: a family study -
In this week's episode we’ll discuss the safety and efficacy of acalabrutinib, venetoclax and obinutuzumab in relapsed/refractory CLL; learn more about alternative donor transplantation for severe aplastic anemia and discuss clonal relapse dynamics in acute myeloid leukemia following allogeneic hematopoietic cell transplantation.
Featured Articles:
Acalabrutinib, venetoclax and obinutuzumab in relapsed/refractory CLL: Final efficacy and ctDNA analysis of the CLL2-BAAG trial Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT Clonal relapse dynamics in acute myeloid leukemia following allogeneic hematopoietic cell transplantation -
In this week's episode we'll learn about the clinical benefit of complete remission with partial hematological recovery, or CRh, in patients with Acute Myeloid Leukemia (AML) treated with molecularly targeted drugs. Then we'll hear about a large cohort of patients with PNH were studied to detail PNH-related thrombotic events, unravel determinants of thrombosis, and evaluate anti-coagulation strategies. Finally, we'll see how new research shows that SOX11 expression is restricted to EBV-negative Burkitt lymphoma, and is associated with a specific genetic landscape.
Featured Articles:
Complete Remission with Partial Hematological Recovery as a Palliative Endpoint for Treatment of Acute Myeloid Leukemia Paroxysmal nocturnal hemoglobinuria-related thrombosis in the era of novel therapies: a 2043 patient/years analysis SOX11 expression is restricted to EBV-negative Burkitt lymphoma and associates with molecular genetic features -
In this week's episode we’ll discuss the mutational and transcriptional landscape of pediatric BCP lymphoblastic leukemia; learn more about the role of platelet-derived TGF-β1 in immune thrombocytopenia; and discuss the findings from a phase 3 trial of mavorixafor in WHIM syndrome.
Featured Articles:
Mutational and transcriptional landscape of pediatric B-cell precursor lymphoblastic lymphoma Platelet-derived TGF-β1 induces functional reprogramming of myeloid-derived suppressor cells in immune thrombocytopenia Phase 3 randomized trial of mavorixafor, CXCR4 antagonist, in WHIM syndrome - Mehr anzeigen
