Alle afleveringen
403 afleveringen, van nieuw naar oud
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Hypercalcemia in monoclonal gammopathy of undetermined significance, neutrophil gelatinase-associated lipocalin in hemostasis, and CD19-targeted NK- or T-cell therapy combined with anti-CD19 monoclonal antibodies
27 februari 2025 · 19 min
In this week's episode we’ll learn more about the significance of hypercalcemia in monoclonal gammopathy of undetermined significance, the role of neutrophil gelatinase-associated lipocalin in hemostasis, and the feasibility of combining CD19-targeted NK- or T-cell therapy with anti-CD19 monoclonal antibodies. Lees meer
Featured Articles:
Approaching Hypercalcemia in Gammopathy of Undetermined Significance: Insights from the iStopMM study
Deficiency of neutrophil gelatinase-associated lipocalin elicits Hemophilia-like bleeding and clotting disorder
Anti-CD19 antibody cotreatment enhances serial killing activity of anti-CD19 CAR-T/-NK cells and reduces trogocytosis
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Review Series on a Quarter Century of TKIs in CML
27 februari 2025 · 33 min
In this Review Series episode Reflections on a Quarter Century of TKIs in CML introduced by Associate Editor Dr. Jason Gottlieb, we’ll hear from contributing authors Drs. Brian Drucker, Francois Guillot, Tim Hughes and Michael Deininger, as they discuss how CML treatment has been impacted since the introduction of tyrosine kinase inhibitors 25 years ago. Lees meer
Click here to view the complete Review Series featured in Volume 145 Issue 9 of Blood.
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A “belt and suspenders” approach to PNH; oral pathogens exacerbate chronic GVHD; microbial metabolome changes may adversely impact CAR-T outcomes
21 februari 2025 · 19 min
In this week's episode, we'll hear about a “belt and suspenders” approach to paroxysmal nocturnal hemoglobinuria, or PNH. We’ll review long-term efficacy data for danicopan—an oral complement factor D inhibitor recently approved as an add-on to C5 inhibitor therapy.
After that: researchers show how oral pathogens may exacerbate chronic graft-versus-host disease. And they assess targeted interventions to mitigate these effects. Finally, we move from the oral to the gut microbiome. New findings suggest that intestinal dysbiosis, induced by antibiotics, may adversely affect outcomes among patients who are receiving CAR-T cell therapy. Lees meerFeatured Articles:
Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH
Oral inflammation and microbiome dysbiosis exacerbate chronic graft-versus-host disease
Antibiotic-induced loss of gut microbiome metabolic output correlates with clinical responses to CAR T-cell therapy
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Mosunetuzumab in relapsed/refractory follicular lymphoma, CD70 CAR T-cells and an anti-CD33/anti-CD3 bispecific antibody in acute myeloid leukemia, and ferroptosis in stored red blood cells
13 februari 2025 · 23 min
In this week's episode we’ll find out about longer term results from a pivotal trial of mosunetuzumab in relapsed/refractory follicular lymphoma, potential use of CD70 CAR T-cells that secrete an anti-CD33/anti-CD3 bispecific antibody as a therapy for acute myeloid leukemia, and how ferroptosis regulates hemolysis in stored red blood cells. Lees meer
Featured Articles:
Long-term 3-year follow-up of mosunetuzumab in relapsed or refractory follicular lymphoma after ≥2 prior therapies
CD70 CAR T cells secreting an anti-CD33/anti-CD3 dual-targeting antibody overcome antigen heterogeneity in AML
Ferroptosis regulates hemolysis in stored murine and human red blood cells
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Review Series on Basic/Translational Science of Factor VIII, Factor IX, and von Willebrand Factor
13 februari 2025 · 32 min
In this Review Series episode on Basic/Translational Science of Factor VIII, Factor IX, and von Willebrand Factor introduced by Dr. Thomas Ortel, we’ll hear from contributing authors Drs. Ben Samuelson Jones, Mac Monroe and Peter Lenting as they discuss how the functional roles of these 3 proteins are interconnected. Lees meer
Click here to view the complete Review Series featured in Volume 144 Issue 21 of Blood.
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Dual TKI targeting in Ph+ ALL; role of liver endothelial ferroportin in iron sensing and homeostasis; severe anemia in pregnant patients with beta-thalassemia minor
6 februari 2025 · 19 min
In this week's episode we'll learn about asciminib plus dasatinib in Philadelphia chromosome-positive acute leukemia. In a phase 1 study, combining two tyrosine kinase inhibitors with distinct mechanisms of action had encouraging activity in patients with de novo disease. After that: new research demonstrates that the iron exporter ferroportin contributes to the iron-sensing properties of liver endothelial cells. But is it the primary mediator of systemic iron homeostasis? Finally: anemia in pregnant patients with beta-thalassemia minor. In the third trimester, about one-third of patients have hemoglobin levels of 9 grams per deciliter or lower. This report may help to guide the appropriate diagnosis of anemia while limiting unnecessary testing and interventions. Lees meer
Featured Articles:
Asciminib plus dasatinib and prednisone for Philadelphia chromosome–positive acute leukemia
The hepcidin-ferroportin axis modulates liver endothelial cell BMP expression to influence iron homeostasis in mice
β-Thalassemia minor is associated with high rates of worsening anemia in pregnancy
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First-line zanubrutinib, obinutuzumab, and venetoclax in mantle cell lymphoma; hyperdiploidy in multiple myeloma; and pegcrisantaspase plus venetoclax in relapsed/refractory acute myeloid leukemia
30 januari 2025 · 21 min
In this week's episode we’ll learn more about a phase 2 trial of first-line zanubrutinib, obinutuzumab, and venetoclax in TP53-mutated mantle cell lymphoma; early development of hyperdiploidy in multiple myeloma; and a phase 1 trial of the asparaginase pegcrisantaspase plus venetoclax in relapsed/refractory acute myeloid leukemia. Lees meer
Featured Articles:
Zanubrutinib, obinutuzumab, and venetoclax for first-line treatment of mantle cell lymphoma with a TP53 mutation
Development of hyperdiploidy starts at an early age and takes a decade to complete
A phase 1 study of the amino acid modulator pegcrisantaspase and venetoclax for relapsed or refractory acute myeloid leukemia
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Targeting C3 in autoimmune hemolytic anemias; venetoclax testing to predict AML response; an improved understanding of the structure of FXIII complex
23 januari 2025 · 19 min
In this week's episode we'll learn about C3-targeted therapy in autoimmune hemolytic anemias. Based on results of an open-label, phase 2 study, pegcetacoplan was well-tolerated, with encouraging efficacy, particularly in patients with cold agglutinin disease. After that: Venetoclax sensitivity testing to predict AML response. In a significant step toward precision medicine, researchers have validated an assay capable of predicting response to the venetoclax-azacitidine regimen. Finally, an improved basic understanding of human coagulation factor XIII. Investigators present the cryo-electron microscopy structure of Factor XIII complex, providing new insights into etiology and the deleterious effects of certain specific mutations. Lees meer
Featured Articles:
Safety and efficacy of pegcetacoplan treatment for cold agglutinin disease and warm antibody autoimmune hemolytic anemia
Ex vivo venetoclax sensitivity predicts clinical response in acute myeloid leukemia in the prospective VenEx trial
Cryo-EM structure of the human native plasma coagulation factor XIII complex
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Review Series on Globin Disorders
23 januari 2025 · 23 min
In this Review Series episode on Globin Disorders introduced by Drs. Thomas Coates and Irene Roberts, we’ll hear from Dr. Douglas Higgs and Dr. Mitchell Weiss as they address advances in our understanding of globin gene biology. Lees meer
Click here to view the complete Review Series featured in Volume 144 Issue 8 of Blood.
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Brentuximab vedotin, nivolumab, and chemotherapy in classical Hodgkin lymphoma; type 1 interferon signaling in sickle cell disease; and type 1 human leukocyte antigen gene mutations in cutaneous T-cell lymphoma
16 januari 2025 · 22 min
In this week's episode we’ll learn more about an experimental regimen for classical Hodgkin lymphoma that combines brentuximab vedotin, nivolumab, and chemotherapy; a possible role for type 1 interferon signaling in developing autoantibodies to red blood cells in sickle cell disease; and how genomic alterations affecting class I human leukocyte antigen molecules may affect patients with cutaneous T-cell lymphoma. Lees meer
Featured Articles:
Brentuximab vedotin, nivolumab, doxorubicin, and dacarbazine for advanced-stage classical Hodgkin lymphoma
IFN-I promotes T-cell–independent immunity and RBC autoantibodies via modulation of B-1 cell subsets in murine SCD
Genetic alteration of class I HLA in cutaneous T-cell lymphoma
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Iron overload aggravates MDS pathophysiology; long-term outcomes after TPO-RA treatment in ITP; new insights into APL treatment outcomes by age and risk
9 januari 2025 · 17 min
In this week's episode we'll learn about the role of iron in myelodysplastic syndromes, or MDS. After that: long-term treatment outcomes in immune thrombocytopenia from the STOPAGO study. Finally, new insights into APL treatment outcomes and prognostic factors from the large-scale Harmony APL project which used ATRA-Arsenic combination therapy. Lees meer
Featured Articles:
Genetic iron overload aggravates, and pharmacological iron restriction improves, MDS pathophysiology in a preclinical study
Long-term follow-up of the STOPAGO study
Acute promyelocytic leukemia: long-term outcomes from the HARMONY project
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Dexamethasone dosing in newly diagnosed multiple myeloma, a new experimental model of X-linked sideroblastic anemia, and ciltacabtagene autoleucel in real-world myeloma treatment
2 januari 2025 · 20 min
In this week's episode, we’ll learn about the prevalence and impact of dexamethasone dose reductions during triple or quadruple therapy for newly diagnosed multiple myeloma, a conditional knockout mouse model for testing gene therapy in X-linked sideroblastic anemia, and real-world efficacy and safety of ciltacabtagene autoleucel in patients with relapsed/refractory multiple myeloma. Lees meer
Featured Articles:
Dexamethasone dose intensity does not impact outcomes in newly diagnosed multiple myeloma: a secondary SWOG analysis
An erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSA
Safety and efficacy of standard-of-care ciltacabtagene autoleucel for relapsed/refractory multiple myeloma
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Comparing BTK inhibitors in relapsed/refractory CLL; unraveling the genetic background of the AnWj-negative blood type; rapid adaptation of CLL cells to venetoclax
26 december 2024 · 19 min
In this week's episode, we'll be comparing BTK inhibitors in relapsed/refractory CLL. Then, we'll hear how researchers in the UK unraveled the genetic background of the AnWj blood group. Finally we'll learn about the role of BCL-2 and BAFF in CLL cell survival following venetoclax therapy. Lees meer
Featured Articles:
Deletions in the MAL gene result in loss of Mal protein, defining the rare inherited AnWj-negative blood group phenotype
Sustained benefit of zanubrutinib vs ibrutinib in patients with R/R CLL/SLL: final comparative analysis of ALPINE
Venetoclax dose escalation rapidly activates a BAFF/BCL-2 survival axis in chronic lymphocytic leukemia
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Primary resistance to BCMA-targeted bispecifics in multiple myeloma, emapalumab therapy in hemophagocytic lymphohistiocytosis, and chemotherapy-induced pyroptosis as a mechanism of thrombus formation in patients with cancer
19 december 2024 · 20 min
In this week's episode, we’ll learn more about mechanisms of primary resistance to BCMA-targeted bispecific T-cell engagers in relapsed/refractory multiple myeloma, the effects of emapalumab therapy on outcomes in patients with pediatric hemophagocytic lymphohistiocytosis who receive stem cell transplants, and a cell death process that may help account for increased thromboembolic risk in patients receiving cancer chemotherapy. Lees meer
Featured Articles:
Impact of soluble BCMA and non–T-cell factors on refractoriness to BCMA-targeting T-cell engagers in multiple myeloma
Emapalumab therapy for hemophagocytic lymphohistiocytosis before reduced-intensity transplantation improves chimerism
GSDME-mediated pyroptosis contributes to chemotherapy-induced platelet hyperactivity and thrombotic potential
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Unravelling follicular lymphoma subtypes; stroke rate trends in sickle cell disease; procoagulant platelet activation promotes venous thrombosis
12 december 2024 · 19 min
In this week's episode, unravelling follicular lymphoma subtypes. Researchers dissect the biological diversity of follicular lymphoma and introduce a new prognostic mode, that could change the way this B-cell neoplasm is subtyped and treated. Then, concerning stroke rate trends in sickle cell disease. A new report shows increasing rates of cerebrovascular events among people with SCD in California. Finally, procoagulant platelet activation promotes venous thrombosis. Investigators report finding procoagulant platelets in the circulation and in thrombi of patients and mice with DVT or PE. Lees meer
Featured Articles:
Follicular lymphoma comprises germinal center–like and memory-like molecular subtypes with prognostic significance
Rates of strokes in Californians with sickle cell disease in the post-STOP era
Procoagulant platelet activation promotes venous thrombosis
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Clonal hematopoiesis in patients with telomere biology disorders, genomic alterations in extracutaneous juvenile xanthogranulomas, and the BCMA-CD3 bispecific antibody teclistamab in relapsed/refractory multiple myeloma
5 december 2024 · 20 min
In this week's episode we’ll learn more about how clonal hematopoiesis affects prognosis in patients with telomere biology disorders, consider recently uncovered molecular subtypes of extracutaneous juvenile xanthogranulomas, and discuss a clinical trial of the BCMA-CD3 bispecific antibody teclistamab in patients with relapsed/refractory multiple myeloma who have received previous BCMA-targeted therapy. Lees meer
Featured Articles:
Clonal landscape and clinical outcomes of telomere biology disorders: somatic rescue and cancer mutations
Recurrent CLTC::SYK fusions and CSF1R mutations in juvenile xanthogranuloma of soft tissue
Efficacy and safety of teclistamab in patients with relapsed/refractory multiple myeloma after BCMA-targeting therapies
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Targeting mutant calreticulin in MPNs; focusing on bleeding risk in patients with cancer; revisiting diagnostic guidelines for familial HLH
28 november 2024 · 19 min
In this week's podcast, we'll learn about targeting mutant calreticulin in MPNs, or myeloproliferative neoplasms. Then, it’s time to address bleeding and clotting in cancer. Results of the prospective, observational CAT-BLED study provide much needed data on the high risk of clinically relevant bleeding, and its association with all-cause mortality, among cancer patients receiving systemic therapy. Finally, we'll revisit diagnostic guidelines for familial HLH, or hemophagocytic lymphohistiocytosis. The authors present new and improved diagnostic criteria with revised clinical criteria, and guidelines on cellular and genetic diagnostic assays. Lees meer
Featured Articles:
Selective targeting of mutated calreticulin by the monoclonal antibody INCA033989 inhibits oncogenic function of MPN
Bleeding events in patients with cancer: incidence, risk factors, and impact on prognosis in a prospective cohort study
Diagnostic guidelines for familial hemophagocytic lymphohistiocytosis revisited
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Review Series on T-Cell Lymphoma
21 november 2024 · 33 min
In this Review Series episode on T-Cell Lymphomas introduced by Dr. Philippe Armand we’ll hear from Drs. Laurence de Leval, Javeed Iqbal, and Enrica Marchi about their work concerning new treatment practices and insights to various t-cell lymphomas. Lees meer
Click here to view the complete Review Series featured in Volume 144 Issue 18 of Blood.
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Stratifying risk in acute myeloid leukemia, daratumumab plus chemotherapy in relapsed-refractory pediatric leukemias, and a method for creating genetically engineered platelets
21 november 2024 · 18 min
In this week's episode we’ll learn more about a new risk classification scheme for use in patients with acute myeloid leukemia who are ineligible for intensive therapy, efficacy and safety of daratumumab plus chemotherapy in pediatric patients with acute lymphoblastic leukemia or lymphoblastic lymphoma, and a blood bank-compatible method for creating genetically engineered platelets with a wide range of potential uses. Lees meer
Featured Articles:
Genetic Risk Stratification and Outcomes Among Treatment-Naive Patients with AML Treated With Venetoclax and Azacitidine
Daratumumab in Pediatric Relapsed/Refractory Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma: DELPHINUS Study
Genetic Engineering of Transfusable Platelets with mRNA-Lipid Nanoparticles is Compatible with Blood Banking Practices
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Exploring the ecosystem of extramedullary myeloma; role of ruxolitinib for pediatric acute GvHD; “YAP”-ing about YAP1 and thrombopoiesis in ITP
14 november 2024 · 19 min
In this week's episode we'll learn about new explorations in extramedullary myeloma. Then, we'll hear about the role of ruxolitinib in children with acute GvHD. Finally: yapping about YAP1 as a new treatment target in immune thrombocytopenia. Lees meer
Featured Articles:
Spatial transcriptomics reveals profound subclonal heterogeneity and T-cell dysfunction in extramedullary myeloma
Ruxolitinib for pediatric patients with treatment-naïve and steroid-refractory acute graft-versus-host disease: the REACH4 study
YAP1 regulates thrombopoiesis by binding to MYH9 in immune thrombocytopenia
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First-line asciminib for chronic myeloid leukemia, a new risk score for clonal cytopenia, and genotyped D-positive blood transfusion in sickle cell anemia
7 november 2024 · 21 min
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. Lees meer
Featured Articles
Asciminib Monotherapy as Frontline Treatment of Chronic Phase Chronic Myeloid Leukaemia - Results from the ASCEND Study
Risk 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
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Update on fixed-dose venetoclax-obinutuzumab in previously untreated CLL; dual epi-immunotherapy in cHL; predicting treatment failure in TKI-treated CML
31 oktober 2024 · 19 min
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. Lees meer
Featured Articles:
Venetoclax-obinutuzumab for previously untreated chronic lymphocytic leukemia: 6-year results of the randomized phase 3 CLL14 study
Epigenetic agents plus anti-PD-1 reprogram the tumor microenvironment and restore antitumor efficacy in Hodgkin lymphoma
A predictive model for therapy failure in patients with chronic myeloid leukemia receiving tyrosine kinase inhibitor therapy
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Bone marrow failure related to ERG insufficiency, factor 11 in vascular hyperpermeability, and new criteria for evaluating anemia in myelofibrosis
24 oktober 2024 · 21 min
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. Lees meer
Featured Articles:
Germ line ERG haploinsufficiency defines a new syndrome with cytopenia and hematological malignancy predisposition
Coagulation factor XI regulates endothelial cell permeability and barrier function in vitro and in vivo
Proposals for revised International Working Group–European LeukemiaNet criteria for anemia response in myelofibrosis
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Platelet glycoprotein receptor VI in abdominal aortic aneurysms, TP53 mutations in 5q-deleted myelodysplastic syndrome, and immunostimulatory cytokine plus CAR T-cell therapy in acute lymphoblastic leukemia
17 oktober 2024 · 19 min
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. Lees meer
Featured Articles:
Soluble glycoprotein VI predicts abdominal aortic aneurysm growth rate and is a novel therapeutic target
Influence of TP53 gene mutations and their allelic status in myelodysplastic syndromes with isolated 5q deletion
A phase 1 clinical trial of NKTR-255 with CD19-22 CAR T-cell therapy for refractory B-cell acute lymphoblastic leukemia
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Using NGS to refine risk stratification in T-ALL; a novel gene therapy approach in severe α-thalassemia; molecular taxonomy of MDS
10 oktober 2024 · 22 min
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. Lees meer
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
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A novel tripartite fusion drives treatment resistance in atypical APL; managing iTTP without TPE; hope for motherhood after allogeneic HCT
3 oktober 2024 · 18 min
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. Lees meer
Featured Articles:
Critical role of tripartite fusion and LBD truncation in certain RARA- and all RARG-related atypical APL
Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange
Hope for motherhood: pregnancy after allogeneic hematopoietic cell transplantation (a national multicenter study)
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Pirtobrutinib plus venetoclax with or without rituximab in relapsed/refractory CLL; murine models of erythroid 5ALA synthesis disorders; targeting PKCa in diabetes and hemochromatosis
26 september 2024 · 23 min
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. Lees meer
Featured Articles:
Fixed-duration pirtobrutinib plus venetoclax with or without rituximab in relapsed/refractory CLL: Phase 1b BRUIN trial
Murine models of erythroid 5ALA synthesis disorders and their conditional synthetic lethal dependency on pyridoxine
Targeting PKCα alleviates iron overload in diabetes and hemochromatosis through the inhibition of ferroportin
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Transplant outcomes in pediatric Fanconi anemia; hepatic Foxo1 regulates iron homeostasis; clinical networks improve APL outcomes in Latin America
19 september 2024 · 19 min
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. Lees meer
Featured Articles:
Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia
Foxo1 is an iron-responsive transcriptional factor regulating systemic iron homeostasis
Clinical networking results in continuous improvement of the outcome of patients with acute promyelocytic
leukemia -
CD8+ T-cell differentiation and treatment response in AML; ATM germline pathogenic variants affect cancer outcomes in ataxia-telangiectasia; efficacy of a selective menin-KMT2A inhibitor in KMT2A- and NPM1-altered leukemias
12 september 2024 · 23 min
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. Lees meer
Featured Articles:
CD8+ T-cell differentiation and dysfunction inform treatment response in acute myeloid leukemia
ATM 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 -
TCR-T cells for post-HCT leukemia recurrence; platelets are the predominant source of procoagulant membranes in hemostasis; BR + R maintenance for MCL
5 september 2024 · 18 min
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. Lees meer
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
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Outcomes of younger patients with mantle cell lymphoma; M-protein assessment during maintenance therapy in multiple myeloma; in vivo CAR T-cell generation using lentiviral vectors
29 augustus 2024 · 1 min
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. Lees meer
Featured Articles:
Outcomes of younger patients with mantle cell lymphoma experiencing late relapse (>24 months): the LATE-POD study
Mass 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
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Review Series on Factor XI
29 augustus 2024 · 45 min
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. Lees meer
Review Series Articles:
Introduction to a review series on factor XI
Biology of factor XI
Targeting factor XI and factor XIa to prevent thrombosis
Factor XI deficiency: phenotypic age-related considerations and clinical approach towards bleeding risk assessment
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Protective role of neonatal CMV infection in B-ALL; GVHD targets organoid-forming bile duct stem cells; seven-year outcomes for venetoclax-ibrutinib therapy in MCL
22 augustus 2024 · 17 min
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. Lees meer
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 manner
Seven-year outcomes of venetoclax-ibrutinib therapy in mantle cell lymphoma: durable responses and
treatment-free remissions -
Iron, HFE haemochromatosis, and infections; bispecific antibodies improve CAR T-cell response; determinants of outcome in NPM1-mutated AML
15 augustus 2024 · 24 min
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. Lees meer
Featured Articles:
Iron, hemochromatosis genotypes, and risk of infections: a cohort study of 142 188 general population individuals
Molecular, clinical, and therapeutic determinants of outcome in NPM1-mutated AML
CD20-bispecific antibodies improve response to CD19-CAR T cells in lymphoma in vitro and CLL in vivo models
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Immune fitness and response to taclistamab in R/R multiple myeloma; BCR::ABL1 M244V mutation causes resistance to asciminib; HSCT and myocardial fibrosis in young patients with SCD
8 augustus 2024 · 17 min
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. Lees meer
Featured Articles:
Correlation of immune fitness with response to teclistamab in relapsed/refractory multiple myeloma in MajesTEC-1
BCR::ABL1 kinase N-lobe mutants confer moderate to high degrees of resistance to asciminib
Impact of hematopoietic cell transplantation on myocardial fibrosis in young patients with sickle cell disease
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Decitabine in older patients with AML; EPCR and sickle chronic kidney disease; lymphocyte depletion versus post-transplant cyclophosphamide for mismatched donor transplant for inborn errors of immunity
1 augustus 2024 · 23 min
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. Lees meer
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
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Impact of MRD-negative on PFS in myeloma; IPSS-R down-staging before transplant in MDS; hereditary angioedema and venous thromboembolism
25 juli 2024 · 22 min
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. Lees meer
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
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Acalabrutinib, venetoclax and obinutuzumab in relapsed/refractory CLL; alternative donor transplantation for severe aplastic anemia; clonal relapse dynamics in AML following allogeneic HCT
18 juli 2024 · 24 min
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. Lees meer
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
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Clinical impact of CRh in AML; thrombosis features in a real-life PNH cohort; and restriction of SOX11 expression to EBV-negative Burkitt lymphomas
11 juli 2024 · 20 min
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. Lees meer
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
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Mutational and transcriptional landscape of pediatric BCP lymphoblastic lymphoma; the role of platelet-derived TGF-1 in immune thrombocytopenia; phase 3 trial of mavorixafor in WHIM syndrome
4 juli 2024 · 23 min
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. Lees meer
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
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Review Series on Oncogenic Signaling and Immune Evasion in Hematologic Malignancies
27 juni 2024 · 16 min
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. Lees meer
Featured Series:
Introduction to a review series on oncogenic signaling and immune evasion in hematologic malignancies
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GM-CSF coordinates shelter in the cytokine storm; Interleukin-1 as a therapeutic target in TTP; CAR T-cell outcomes across race and ethnicity in LBCL
27 juni 2024 · 20 min
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. Lees meer
Featured Articles:
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
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Reassessing the CLL prognostic index; ADAMTS13 conformation and risk of relapse in immune-mediated TTP; haploidentical bone marrow transplant in sickle cell disease
20 juni 2024 · 24 min
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. Lees meer
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
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A biomarker for disease severity in Wiskott-Aldrich Syndrome; loss of Dnmt3a confers resistance to IFNα; unrelated donor selection in HSCT with PTCy
13 juni 2024 · 19 min
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. Lees meer
Featured Articles:
Wiskott-Aldrich Syndrome: A retrospective study on 577 patients defines the genotype as a predictive biomarker for disease severity and survival
Loss 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
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Nilotinib with or without cytarabine for Philadelphia-positive AML; thrombosis risk in Factor V Leiden and Prothrombin G20210A; ctDNA improves prognostic prediction in relapsed/refractory multiple myeloma
6 juni 2024 · 23 min
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. Lees meer
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
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Hemophilia prophylaxis with fitusiran vs BPA/CFC; germline NBN variants in pediatric B-ALL; Histone demethylation and cell-intrinsic immune response in AML
30 mei 2024 · 19 min
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. Lees meer
Featured Articles:
Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial
Germline 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
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Effects of voxelotor on cerebral blood flow in pediatric sickle cell disease; thrombosis in Vexas syndrome; response to tyrosine kinase inhibitors in ABL-class acute lymphoblastic leukemia
23 mei 2024 · 22 min
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. Lees meer
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
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Stamping out high-risk myeloma with D-KRd and tandem transplant; plasmin-cleaved VWF in microthrombosis; SCD plasma sensitizes iPSC-derived sensory neurons
16 mei 2024 · 20 min
In this week's podcast we'll discuss another step forward in stamping out high-risk myeloma. Then we'll hear about the plasmin-cleaved von Willebrand factor, or VWF, in microthrombosis. Finally we'll hear about new insights into the origins of sickle cell pain. Lees meer
Featured Articles:
Daratumumab, carfilzomib, lenalidomide, and dexamethasone with tandem transplant for high-risk newly
diagnosed myelomaPlasmin-cleaved von Willebrand factor as a biomarker for microvascular thrombosis
Sickle cell disease iPSC-derived sensory neurons exhibit increased excitability and sensitization to patient
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Allogeneic transplant in first remission for post-induction molecular MRD; circulating HSPCs actively participate in human hematopoiesis; let-7 miRNAs repress HIC2 to regulate BCL11A transcription and hemoglobin switching
9 mei 2024 · 22 min
In this week's episode we’ll discuss the utility of MRD in identifying patients with NPM1 AML who benefit from allogeneic transplant in first remission, learn more about the contribution of circulating hematopoietic stem/progenitor cell subsets to human hematopoietic hemostasis, and discuss the role of Let-7 miRNAs in regulation of BCL11A transcription and hemoglobin switching. Lees meer
Featured Articles:
Postinduction molecular MRD identifies patients with NPM1 AML who benefit from allogeneic transplant in first remission
let-7 miRNAs repress HIC2 to regulate BCL11A transcription and hemoglobin switching
Circulating Hematopoietic Stem/Progenitor Cell subsets contribute to human hematopoietic homeostasis
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ADAMTS13 recovery in caplacizumab-treated TTP; inhibition of venetoclax-resistant BCL2 variants with sonrotoclax; genomic insights from Castleman disease in twins
2 mei 2024 · 19 min
In this week's epside we'll learn about ADAMTS recovery after caplacizumab therapy in patients with immune thrombotic thrombocytopenic purpura. Then, we'll hear about preclinical characterization of sonrotoclax, a potential second-generation BCL2 inhibitor. Finally we'll hear about new insights into the etiology of Castleman disease where researchers describe the unusual occurrence of idiopathic multicentric Castleman disease in identical twins. Lees meer
Featured Articles:
ADAMTS13 recovery in acute thrombotic thrombocytopenic purpura after caplacizumab therapy
Sonrotoclax overcomes BCL2 G101V mutation–induced venetoclax resistance in preclinical models of
hematologic malignancySingle-cell landscape of idiopathic multicentric Castleman disease in identical twins