Episodit
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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 -
Puuttuva jakso?
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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 -
Introduced by Associate Editor Robert Zeiser, this Review Series focuses on the problem of immune escape by acute myeloid leukemia (AML). The series opens with a review of how AML escapes T-cell–driven elimination and then focuses on how p53 function impinges on AML recognition by immune cells. The series finishes with a summary of new approaches to tackling this major problem.
Featured Series:
Introduction to a review series on oncogenic signaling and immune evasion in hematologic malignancies -
In this week's episode we'll discuss new insights on the role of GM-CSF in establishing immune memory. The authors propose that the coordination of opposing immune memory programs, driven by GM-CSF, may be essential to efficient, yet controlled, innate immune responses. After that: Interleukin-1 inhibition in TTP. Researchers explore the potential of recombinant IL-1 receptor antagonist, anakinra, in a murine model of thrombotic thrombocytopenic purpura—an uncommon but potentially fatal disorder with limited therapeutic options. Finally, we'll learn about CAR T-cell therapy outcomes by race and ethnicity in large B-cell lymphoma. Non-Hispanic Black patients had lower rates of response and progression-free survival in axi-cel clinical trials and real-world data, raising awareness and giving further insights into potential inequitable access to care.
GM-CSF receptor expression determines opposing innate memory phenotypes at different stages of myelopoiesis Mortality, cardiac and cerebral damage reduction by IL-1 inhibition in a murine model of TTP Real-World and Clinical Trial Outcomes in Large B-cell Lymphoma with Axicabtagene Ciloleucel Across Race and Ethnicity
Featured Articles: -
In this week's episode we’ll discuss the prognostic value of the Chronic Lymphocytic Leukemia International Prognostic Index in the era of targeted therapies, learn how peak ADAMTS13 activity can be used to assess ADAMTS13 conformation and risk of relapse in immune-mediated thrombotic thrombocytopenic purpura, and discuss the findings from a phase 2 trial of haploidentical bone marrow transplant in sickle cell disease.
Featured Articles
Reassessing the Chronic Lymphocytic Leukemia International Prognostic Index in the era of targeted therapies Peak ADAMTS13 activity to assess ADAMTS13 conformation and risk of relapse in immune-mediated Thrombotic Thrombocytopenic Purpura An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease -
In this week's episode we'll discuss assessing risk profile in Wiskott-Aldrich Syndrome, or WAS. Then, we'll learn about how the loss of DNMT3A confers resistance to interferon-alpha in hematopoietic stem cells carrying the JAK2-V617F mutation. Finally, we'll explore unrelated donor selection for AML patients undergoing hematopoietic stem cell transplantation with post-transplant cyclophosphamide.
Featured Articles:
Wiskott-Aldrich Syndrome: A retrospective study on 577 patients defines the genotype as a predictive biomarker for disease severity and survivalLoss of Dnmt3a increased self-renewal and resistance to pegylated interferon-alpha in JAK2-V617F-positive myeloproliferative neoplasms Younger unrelated donors may be preferable over HLA match in the PTCy era: A study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation -
In this week's episode we’ll discuss the safety and efficacy of nilotinib with or without cytarabine in Philadelphia-positive acute lymphoblastic leukemia; learn more about thrombosis risk in double heterozygous carriers of Factor V Leiden and prothrombin G20210A and discuss the utility of circulating cell-free tumor DNA in prognostic prediction in patients with relapsed/refractory multiple myeloma.
Featured Articles:
Nilotinib with or without cytarabine for Philadelphia positive acute lymphoblastic leukemia Thrombosis risk in double heterozygous carriers of Factor V Leiden and Prothrombin G20210A in FinnGen and the UK Biobank ctDNA improves prognostic prediction in relapsed/refractory multiple myeloma receiving ixazomib, lenalidomide, and dexamethasone -
In this week's episode we'll learn about fitusiran prophylaxis in patients with hemophilia A or B, with or without inhibitors. Next we'll hear about new findings that heterozygous germline variants in the NBN gene that are linked to increased risk of B-cell acute lymphoblastic leukemia in children. Finally, we'll explore new insights on the histone demethylase PHF8, which has been identified as a master regulator of cell-intrinsic immune responses in acute myeloid leukemia.
Featured Articles:
Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trialGermline genetic NBN variation and predisposition to B-cell acute lymphoblastic leukemia in children Epigenetic control over the cell-intrinsic immune response antagonizes self-renewal in acute myeloid leukemia -
In this week's episode we’ll discuss the effects of voxelotor on cerebral blood flow in pediatric sickle cell disease, learn more about venous and arterial thrombosis in patients with Vexas syndrome, and discuss differential sensitivity to tyrosine kinase inhibitors in ABL-class acute lymphoblastic leukemia.
Featured Articles:
The influence of voxelotor on cerebral blood flow and oxygen extraction in pediatric sickle cell disease Venous and arterial thrombosis in patients with VEXAS syndrome Tyrosine kinase inhibitor response of ABL-class acute lymphoblastic leukemia: The role of kinase type and SH3 domain - Näytä enemmän