Alle afleveringen
403 afleveringen, van nieuw naar oud
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Rivaroxaban after cancer surgery, genetics and survival in severe aplastic anemia, and a 5-year follow up of rituximab-venetoclax in relapsed/refractory CLL
25 augustus 2022 · 20 min
In this week’s episode discuss the efficacy of rivaroxaban as antithrombotic prophylaxis after laparoscopic surgery for colorectal cancer, learn more about the association between genomic variants and survival outcomes after hematopoietic cell transplantation in severe aplastic anemia, and discuss the long-term safety and efficacy of fixed-duration venetoclax plus rituximab in relapsed/refractory CLL. Lees meer
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Stroke in hereditary TTP, and germline DDX41 variants as risk factors and prognostic markers in myeloid neoplasms
18 augustus 2022 · 21 min
In this week’s episode, we’ll learn more about neuropsychiatric manifestations and stroke risk in hereditary TTP, discuss germline DDX41 variants as predisposing factors to myeloid neoplasms, and learn more about the prognostic impact of DDX41 mutations in adults with intensively treated AML. Lees meer
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Cost effectiveness of first-line daratumumab in MM, base-edited CAR T cells for T-ALL, and the role of SETBP1 mutations in chronic neutrophilic leukemia
11 augustus 2022 · 19 min
In this week’s episode new research suggesting that, in patients with transplant-eligible multiple myeloma, adding daratumumab to first-line combination regimens may be a more cost effective strategy than saving it for second-line use. We’ll also review the work of researchers who applied base-editing technology to develop a complex and potent CAR T cell product for potential “off-the-shelf” use in patients with T-cell leukemias and other CD7-positive malignancies. Finally, we’ll review a paper that elucidates the role of SETBP1 mutations in chronic neutrophilic leukemia, pointing the way toward a potential multi-pronged therapeutic approach to this rare myeloproliferative neoplasm. Lees meer
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Immune thrombocytopenia and adenoviral SARS-COV2 vaccines, genomics, and a risk assessment model of anti-CD19 CAR T-cell treatment outcomes in lymphoma
4 augustus 2022 · 21 min
In this week’s episode we’ll discuss the factors influencing the development of immune thrombocytopenia after administration of the ChAdOx1 nCov-19 vaccine, learn about the genomic features underlying anti-CD19 CAR T-cell treatment failure in lymphoma, and introduce a new predictive model for risk assessment before CAR T-cell therapy for large B-cell lymphoma. Lees meer
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Naturally selected CD7 CAR T cell therapy for T-lineage malignancies, CD4+ T cell exhaustion and PD-L1 inhibition in B-ALL, and tPA-induced phosphorylation of occludin in stroke
28 juli 2022 · 18 min
In this week’s episode we review a novel approach to generating autologous CD7-specific CAR T therapy for patients with T-cell malignancies that overcomes a key limitation: target-driven fratricide. We’ll also learn about new research pinpointing a key subset of exhausted CD4+ T cells in B- ALL, which also provides a rationale for combining tyrosine kinase inhibitors and PD-L1 blockers to reverse exhaustion and enhance leukemia clearance. Finally, we’ll discuss studies in a mouse model of acute ischemic stroke, showing that inhibiting phosphorylation of a tight junction protein in endothelial cells reduces risk of intracranial hemorrhage after treatment with recombinant tissue plasminogen activator. Lees meer
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Role of PIEZO1 in leukocyte extravasation, CD8+ T-cell activation in macrophage activation syndrome, and ABO O blood group as a novel risk factor for HIT
21 juli 2022 · 21 min
In this week’s episode we first review new work revealing the critical role of the tension-sensitive cation channel PIEZO1 in the transendothelial migration of leukocytes. We’ll also review new research suggesting that CD8+ T-cells dimly expressing the CD4 antigen are increased in patients with various forms of secondary HLH, a finding that may have diagnostic, prognostic, and therapeutic significance. Finally, we’ll review a large, genome-wide association study identifying the ABO O blood group as a novel risk factor for heparin-induced thrombocytopenia—a finding that could have implications for prediction of this syndrome and for the management of related conditions. Lees meer
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Long-term ibrutinib-rituximab vs FCR in CLL, procoagulant platelet sentinels in prevention of inflammatory bleeding, and new SERPINC1 variants in thrombophilia
14 juli 2022 · 18 min
In this week’s episode we’ll compare the long-term outcomes of ibrutinib-rituximab combination therapy versus FCR chemoimmunotherapy in chronic lymphocytic leukemia, discuss the role of procoagulant platelet sentinels in inflammatory bleeding, and learn more about variants in the SERPINC1 gene encoding antithrombin that cause severe thrombophilia. Lees meer
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Malignancy risk from genetically modified immune effector cells, p53 immunohistochemistry as a new tool in AML, and phenotypic escape from CD19-directed immunotherapies.
7 juli 2022 · 22 min
Today we’ll learn more about the risk of subsequent malignancies in patients treated with genetically modified immune effector cells, discuss how p53 immunohistochemistry can be a global readout for TP53 alterations in AML, and uncover the role of CD19-negative CD22-positive B-cell progenitors in immune escape from CD19-directed therapies. Lees meer
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Overcoming CAR T cell resistance in myeloma, why anti-CCR4 antibody therapy in CTCL sometimes fails, and corticosteroids increase risk of VOE in sickle disease
30 juni 2022 · 19 min
In this week’s episode we review a novel strategy for overcoming resistance to CAR T cell therapy that involves the dual targeting of myeloma cells and cancer-associated fibroblasts. We’ll also explore a recent report demonstrating that loss of CCR4 expression is common after treatment of CTCL with the anti-CCR4 antibody mogamulizumab. Finally, we’ll review real-world data demonstrating an association between corticosteroid exposure and risk of vaso-occlusive episodes in patients with sickle cell disease, providing further evidence that steroids should be avoided in this setting. Lees meer
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Salvage therapy with NICE for Hodgkin lymphoma, BMP2/SMAD pathway activation in leukemic transformation, and the role of zinc in T-cell reconstitution after HSCT
23 juni 2022 · 19 min
In this week’s episode we’ll learn more about salvage therapy with nivolumab plus or minus ICE (or NICE) for Hodgkin lymphoma, discuss the role of BMP2/SMAD pathway activation in leukemic transformation, and learn more about the role of zinc in T-cell reconstitution after transplantation. Lees meer
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MRD and post-transplant outcomes in older AML patients, enhancer hijacking in a novel high-risk B-other ALL subtype, and ruxolitinib in pediatric HLH
16 juni 2022 · 20 min
In this week’s episode we will discuss new data demonstrating that, in older patients with AML, post-transplant relapse risk is driven by clinical and molecular features present at diagnosis, but not remission MRD. We’ll also explore two studies that characterize a novel high-risk B-ALL subtype, defined by two unique genomic alterations that includes a deletion resulting in enhancer hijacking that deregulates expression of the CDX2 homeobox transcription factor. Finally, we’ll review results of a large, single-arm phase 2 trial providing encouraging clinical evidence for the use of ruxolitinib as a front-line treatment for pediatric hemophagocytic lymphohistiocytosis. Lees meer
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Anti-PF4 antibodies after Covid vaccination, CAR T-cell therapy for CNS leukemia, and advances in Diamond-Blackfan anemia
9 juni 2022 · 21 min
In this week's episode, we’ll learn more about the interaction between anti-PF4 antibodies and anticoagulants in vaccine-induced thrombotic thrombocytopenia, discuss the efficacy and safety of CD19-specific CAR T-cell-based therapy in B-cell ALL patients with central nervous system leukemia, and learn more about erythroid cell-extrinsic factors that can inhibit erythropoiesis in adjacent cells. Lees meer
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Fixed-duration ibrutinib + venetoclax for CLL, aryl hydrocarbon receptor in idiopathic pneumonia syndrome, and VWF propeptide and multimerization
2 juni 2022 · 22 min
In this week's episode, we discuss results from the international phase 2 CAPTIVATE study showing that in patients with treatment-naive CLL, fixed-dose ibrutinib plus venetoclax yields deep and durable responses and promising progression-free survival. We’ll also explore data that show that aryl hydrocarbon receptor is a critical inflammation checkpoint in the lung epithelium—a finding that may have therapeutic implications for idiopathic pneumonia syndrome. Lastly, we’ll review new insights into the role of von Willebrand Factor propeptide in facilitating multimer formation, shedding light on a potential novel treatment approach for type 2a von Willebrand disease. Lees meer
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HEATR3 variants as a new cause of Diamond-Blackfan anemia, zanubrutinib in mantle cell lymphoma, and HMGB1 inhibits Epo signaling in anemia of inflammation
26 mei 2022 · 20 min
In this week's episode we discuss the role of HEATR3 variants as a new cause of Diamond-Blackfan anemia, learn more about the long-term efficacy and safety of zanubrutinib in relapsed or refractory mantle cell lymphoma, and uncover an unexpected role for HMGB1 in anemia of inflammation by inhibiting erythropoietin signaling. Lees meer
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HLA-DQ heterodimers and transplantation, the genomic landscape of LGL leukemia, and an updated classification of hemochromatosis
19 mei 2022 · 22 min
In this week’s episode we’ll feature new research demonstrating that certain HLA-DQ heterodimers can help predict clinical outcome following hematopoietic cell transplantation. Next, we’ll review a recent integrated and comprehensive genomic analysis that sheds new light on the molecular characteristics of large granular lymphocyte leukemia and its subtypes. Finally, we’ll review the work of a group that proposes a new and more accessible hemochromatosis classification system based on clinical characteristics and genetic features. Lees meer
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Role of macrophage NOX2 in alveolar homeostasis, hydroxyurea versus peginterferon alfa-2a in high-risk PV and ET, and the emerging PV treatment landscape
12 mei 2022 · 21 min
In this week’s episode we’ll discuss an undescribed role for NOX2 in maintaining lung homeostasis through suppression of alveolar macrophage activation. We’ll also cover results of a phase 3 randomized trial that compares the safety and efficacy of hydroxyurea and peginterferon alfa-2a in patients with high-risk polycythemia vera and essential thrombocythemia (or PV and ET, respectively). Finally, we’ll go more in depth on the emerging treatment landscape for PV, and the limits of current clinical trial endpoints. Lees meer
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Microbiome and CD4 T-cell recovery after allogeneic HCT, and a paradigm shift to CAR T-cells in the second line for large B-cell lymphoma?
5 mei 2022 · 20 min
In this week’s episode we’ll discuss the effects of intestinal microbial diversity on patient outcomes following allogeneic hematopoietic cell transplant and review the results of three different phase 3 trials comparing CAR T-cells to autologous stem cell transplant in patients with refractory or early relapsing large B cell lymphoma, which have the potential to result in a paradigm shift in the standard of care for second-line therapy. Lees meer
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Canakinumab in SCA, antigen-specific donor T-cells to prevent post-transplant relapse in ALL, and nasopharyngeal mucosal immunity defects in transplanted SCID patients
28 april 2022 · 20 min
In this week’s episode, we’ll discuss the efficacy of canakinumab in children and young adults with sickle cell anemia, learn more about the use of donor-derived multiple leukemia antigen specific T-cell therapy to prevent relapse in post-transplant patients with ALL, and discuss the defects in nasopharyngeal mucosal immunity in patients with severe combined immunodeficiency after hematopoietic stem cell transplantation. Lees meer
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Timing (and utility) of CNS prophylaxis in DLBCL, Apollo deficiency in a bone marrow failure syndrome, and GATA1 mutants in congenital anemia
21 april 2022 · 17 min
In this week’s episode we’ll cover a retrospective study on the timing of high-dose methotrexate CNS prophylaxis in diffuse large B-cell lymphoma, results of which suggest end-of-treatment delivery might be preferred to earlier administration. The second research article describes how biallelic Apollo variants lead to an inherited bone marrow failure syndrome that resembles dyskeratosis congenita, albeit with normal telomere length. We’ll conclude with research revealing how a rare form of congenital anemia is caused by novel missense mutations in GATA1. Lees meer
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Obinutuzumab plus lenalidomide in advanced follicular lymphoma, mechanisms of anemia in children with malaria, and granulocyte microvesicles for septic shock
14 april 2022 · 19 min
In this week’s episode we’ll discuss the efficacy of the obinutuzumab-lenalinomide combination in advanced follicular lymphoma, learn more about the underlying mechanisms of anemia in children infected with Plasmodium falciparum, and discuss how granulocyte microvesicles could improve outcomes in septic shock. Lees meer
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Structural racism and AML survival, promoter competition and hemoglobin switching, and CAR T-cell therapy and high-risk cytogenetics in ALL
7 april 2022 · 18 min
In this week’s episode we’ll first cover a research article demonstrating the striking contribution of neighborhood disadvantage to racial and ethnic disparities in survival in patients with acute myeloid leukemia. The second research article provides a model for understanding how disruption of the adult-globin promoter may alleviate promoter competition, thereby reactivating fetal gamma-globin gene expression. We will close with a research article showing that CD19-directed CAR T-cell therapy provides durable remissions in patients with relapsed or refractory ALL across cytogenetic categories, including those patients with high-risk cytogenetics. Lees meer
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Targeting the PBX1-FOXM1 axis in myeloma, SF3B1 mutations in MDS, and pulmonary artery hypertension-associated mortality in β-thalassemia patients
31 maart 2022 · 19 min
In this week's podcast, we’ll discuss the role of the PBX1-FOXM1 axis in multiple myeloma, learn more about the pathobiology of SF3B1 splicing factor mutations in myelodysplastic syndromes with ring sideroblasts, and discuss mortality rates due to pulmonary arterial hypertension in patients with β-thalassemia. Lees meer
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Novel high-risk subtypes in adult B-ALL, Liso-cel CAR-T therapy in CLL, and liquid biopsies in large B-cell lymphomas
24 maart 2022 · 18 min
In this week's podcast, we’ll cover a research article that describes two novel subgroups of adult B-cell ALL with high expression of CDX2 and IDH mutations that are linked to poor prognosis. Next, we'll cover results from TRANSCEND CLL 004, a phase 1 study of the CAR T cell therapy, liso-cel, in patients with relapsed or refractory CLL. We will close with research that provides new insights into how analysis of circulating tumor DNA could inform management of patients with aggressive B-cell lymphomas. Lees meer
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Effective treatments for kaposiform hemangioendothelioma, causes of vascular dysfunction in sickle cell patients, and better strategies to select therapy in older adults with AML
17 maart 2022 · 19 min
In this week's podcast, discuss the efficacy of siroliumus plus prednisolone in kaposiform hemangioendothelioma with the Kasabach-Merritt phenomenon, learn more about the association between SOD2 V16A and vascular dysfunction in patients with sickle cell disease, and discuss the value of geriatric assessment in predicting outcomes in older adults with AML. Lees meer
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COVID-19 vaccination in patients with blood cancers and recalibrating the HLA barrier in haploidentical stem cell transplantation
10 maart 2022 · 19 min
In this week's podcast, we’ll focus on 2 reports on clinical outcomes following COVID-19 vaccination in patients with hematologic malignancies. Despite full or partial vaccination, these patients have an increased risk of poor outcomes compared to matched controls or the general population. In a third report, we discuss new data on HLA-mismatching and outcomes of haploidentical transplants. Lees meer
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Cell therapy options for chemo-sensitive DLBCL, PIGA mutations in a novel subtype of hemochromatosis, and triplet combination therapy in high-risk CLL
3 maart 2022 · 21 min
In this week's podcast, we’ll compare the efficacy of autologous stem cell transplant to CAR T-cell therapy for relapsed diffuse large B-cell lymphoma, learn more about a novel subtype of hemochromatosis caused by constitutional PIGA mutations, and discuss the findings from a phase 2 trial of obinutuzumab, ibrutinib and venetoclax in patients with untreated high-risk CLL. Lees meer
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Germline predisposition mutations in AML, a new way to resolve NETs, and venetoclax and BAX mutations in normal myeloid cells of CLL patients
24 februari 2022 · 19 min
In this week's podcast, we’ll look at work from researchers who found pathogenic or likely pathogenic variants of cancer predisposition genes in nearly 14% of adults with AML. Next, we’ll review research demonstrating that a recently identified series of resolvins reduces neutrophil extracellular trap formation and enhances clearance of these NETs by macrophages, suggesting a new mechanism for the resolution of inflammation and coagulopathies associated with various infections. We’ll conclude with research demonstrating that patients with CLL undergoing long-term venetoclax treatment exhibit a high incidence of cytopenias, clonal hematopoiesis, and myeloid neoplasms, and may acquire BAX mutations in normal myeloid cells. Lees meer
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Ovarian reserve in sickle cell anemia, genomic landscape of adult T-cell leukemia/lymphoma, and a new prognostic index for HLH-associated heme malignancies
17 februari 2022 · 20 min
In this week’s podcast, we’ll discuss diminished ovarian reserve in young women with sickle cell anemia, learn more about genomic alterations in adult T-cell leukemia/lymphoma, and review a novel diagnostic and prognostic index for malignancies associated with hemophagocytic lymphohistiocytosis. Lees meer
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Role of HBO1 in HSC function, robust MRD analysis in multiple myeloma, and cardiac ferroptosis in sickle cell disease
10 februari 2022 · 16 min
In this week’s podcast, we’ll look at research demonstrating that the histone acetyltransferase HBO1 is an essential regulator of hematopoietic stem cell function during adult hematopoiesis. Next, we’ll review a large, combined analysis confirming the prognostic value of MRD assessed with next-generation sequencing in 4 randomized trials evaluating daratumumab-based therapies. We’ll conclude with research demonstrating that ferroptosis, a specific type of regulated cell death, is a key mechanism of cardiomyopathy in sickle cell disease. Lees meer
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The ARID3A transcription factor suppresses leukemia, a COVID-19 booster shot benefits CLL patients, and advances in the genetics of congenital neutropenia
3 februari 2022 · 20 min
In this week’s podcast, we’ll discuss the role of megakaryocytic transcription factor ARID3A in leukemia suppression, learn more about the efficacy of a third Pfizer BNT162b2 mRNA COVID-19 vaccine dose in patients with chronic lymphocytic leukemia, and discuss the association of heterozygous caseinolytic peptidase B variants with congenital neutropenia. Lees meer
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Sustained MRD negativity & outcomes in myeloma, therapeutic targeting of VWF in TTP, and improving CAR T-cells in CLL with PI3 kinase inhibition
28 januari 2022 · 20 min
On this week’s podcast, we’ll review a recent analysis of phase 3 daratumumab studies looking at the prognostic impact of sustained minimal residual disease, or MRD negativity in patients with multiple myeloma. Next, we’ll hear more about a novel thrombolytic agent targeting von Willebrand factor that may represent a promising approach for the treatment of thrombotic thrombocytopenic purpura. We’ll conclude with a study demonstrating that PI3 kinase inhibitor duvelisib can be used to enhance the in vivo efficacy of CAR T cells in CLL. Lees meer
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Does route of CNS prophylaxis for DLBCL matter?, the connection between CHIP and COPD, and ATRA plus low-dose rituximab in ITP
20 januari 2022 · 22 min
In this week’s podcast, we’ll discuss real-world outcomes comparing route of CNS prophylaxis for aggressive non-Hodgkin lymphomas, learn more about the association of clonal hematopoiesis with chronic obstructive pulmonary disease, and discuss the efficacy of all-trans retinoic acid plus low-dose rituximab in corticosteroid-resistant or relapsed immune thrombocytopenia. Lees meer
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A spectrum of ALK-positive histiocytosis, and metabolites as biomarkers for GvHD diagnosis and risk assignment
13 januari 2022 · 15 min
On today’s podcast, we’ll explore the largest case series to date describing ALK-positive histiocytosis. The authors provide new insights on the disease and its treatment with ALK inhibitors, which can provide durable responses. We’ll also review with a study that pinpoints alpha-ketoglutaric acid as consistently elevated in pediatric chronic GvHD, and finds unique metabolomic patterns that appear to distinguish different GvHD subtypes. Lees meer
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Long-term outcomes in severe aplastic anemia, cytoprotective effects of factor VIIa, and the role of IL-1 in hematopoietic stem cell ageing
6 januari 2022 · 21 min
In this week’s podcast, we’ll talk about the long-term outcomes in patients with severe aplastic anemia treated with immunosuppression and eltrombopag, learn more about anti-inflammatory and cytoprotective effects of factor VIIa, and discuss the role of IL-1 in microbiome-induced ageing of hematopoietic stem cells in mice. Lees meer
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CRISPR cure for XMEN disease?, JAK inhibition in T-cell lymphomas, and intracranial hemorrhage in hemophilia
30 december 2021 · 20 min
In this week’s episode, we’ll review a research article that provides the first description of a targeted gene insertion approach to correct the genetic mutation underlying XMEN disease. Next, we’ll cover results of a phase 2 study that provides proof-of-principle that the JAK/STAT pathway is a promising target for the treatment of peripheral T-cell lymphomas. We’ll close with a study providing precise and up-to-date estimates on the incidence of intracranial hemorrhage in patients with hemophilia that could have important implications for preventive strategies. Lees meer
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Antigen presentation by platelet extracellular vesicles, fibril-reactive monoclonal antibody therapy for AL amyloidosis, and targeting gasdermin D in sepsis
23 december 2021 · 19 min
In this week’s episode, we’ll review a research article demonstrating that some platelet-derived extracellular vesicles harbor proteasomes that can process and present antigens via MHC class I molecules. Next, we’ll review results of a phase 1 study of a fibril-reactive monoclonal antibody that was well tolerated and led to rapid, sustained organ responses in patients with AL amyloidosis. We’ll close with a research article suggesting that the pore-forming protein gasdermin D, which plays a crucial role in the release of neutrophil extracellular traps, is a promising drug target in sepsis. Lees meer
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Mesenchymal stromal cells in SCD, CAR T-cell-mediated hematotoxicity in B-cell lymphoma, and dual cytokine blockade in GVHD
16 december 2021 · 18 min
In this week’s episode, we’ll talk about the functional properties of mesenchymal stromal cells in sickle cell disease, learn more about CAR T-cell-mediated hematotoxicity in relapsed/refractory B-cell lymphoma, and discuss dual cytokine blockade in graft-versus-host disease. Lees meer
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PROTACs for JAKs in CRLF2-rearranged ALL, risk stratification in Down syndrome-associated leukemia, and how losing sialic acid activates immune cells in ITP
9 december 2021 · 19 min
In this week’s episode, we’ll learn about a new approach for targeting Janus kinases in CRLF2-rearranged ALL, discuss risk stratification for myeloid leukemia in children with Down syndrome and learn more about how sialic acid alterations on megakaryocyte antigens regulate immune cells and platelet production. Lees meer
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Belumosudil for chronic GVHD, mechanistic insights into VITT, and factors driving molecular response in IFNα-treated MPNs
2 december 2021 · 20 min
First on today’s podcast, we’ll review results of a randomized phase 2 study demonstrating that inhibition of ROCK2 with belumosudil is well tolerated and effective in patients with steroid-refractory graft-versus-host disease. Next, we’ll review the work of researchers who have uncovered new insights into the immunopathogenesis of vaccine-induced immune thrombotic thrombocytopenia. And we’ll close with a report of a prospective longitudinal analysis that elucidates the dynamics of mutated hematopoietic stem and progenitor cells during therapy with interferon-alpha in patients with BCR-ABL1 negative myeloproliferative neoplasms. Lees meer
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Post-vaccination COVID-19 in patients with hematologic malignancies
1 december 2021 · 22 min
All four studies included in this podcast present preliminary data with modest numbers of patients, and therefore, the findings should be interpreted with these limitations. Likewise, the impact of specific strains and waning antibody levels after COVID-19 vaccination will require further follow-up, since the Delta variant was not the dominant strain in circulation at the time of this research. The first two studies assess the immune responses to vaccination in two specific high-risk populations, namely, patients with lymphoma and multiple myeloma. The last two studies, one from a large European consortium and the other based on nationwide data from Israel, report on the incidence, risk factors and short-term outcomes of COVID-19 breakthrough infection in vaccinated patients with hematologic malignancies. Lees meer
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Asciminib versus bosutinib in CML; dopamine signaling and hematopoietic stem cells; complement factor C5 and VTE risk
25 november 2021 · 21 min
In this week’s episode, we’ll learn more about the efficacy of asciminib in patients with chronic myeloid leukemia who are resistant or intolerant to two or more tyrosine kinase inhibitors, discuss the role of dopamine signaling in hematopoietic stem and progenitor cell function, and learn more about elevated plasma concentration of complement factor C5 as a risk factor for venous thromboembolism. Lees meer
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Danicopan add-on therapy in PNH, neural networks to identify bone marrow cells, and RNA editome and hematopoiesis
18 november 2021 · 18 min
In this week’s episode, we’ll review results of a phase 2 study showing the beneficial effects of a first-in-class factor D inhibitor as add-on therapy in PNH patients who remain anemic and are transfusion-dependent despite C5 inhibition. Next, we’ll review the work of researchers who have developed a neural network that they say is highly accurate in differentiating between bone marrow cell morphologies. We’ll close with a report demonstrating that RNA editing of antizyme inhibitor 1, or Azin1, is a novel regulator of hematopoietic cell fate that can influence self-renewal and differentiation. Lees meer
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Machine learning in classification of AML, aging bone marrow in leukemia, and treating bleeding disorders of unknown cause
11 november 2021 · 19 min
In this week’s episode, we’ll learn more about the application of machine learning in molecular subclassification and prognostication of acute myeloid leukemia or AML, discuss the role of aging bone marrow in leukemia progression, and learn more about the challenges in treating bleeding disorders of unknown cause. Lees meer
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Anti-CD36-antibody therapy of neonatal alloimmune thrombocytopenia, costs of hemophilia B gene therapy, and insights into erythroid maturation
4 november 2021 · 18 min
In this week’s episode, we’ll review a research article showing beneficial effects of prenatal immunotherapy in a mouse model of anti-CD36-mediated fetal and neonatal alloimmune thrombocytopenia. Next, we’ll look at results of a simulation analysis suggesting that gene therapy for hemophilia B is more cost-effective than on-demand or prophylactic factor treatment. We’ll conclude with a report which provides important new insights into regulation of terminal erythroid maturation at the transcriptional level that may help improve our understanding of normal and abnormal erythropoiesis. Lees meer
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Genetic risks for CMV after BMT, multiplex gene editing to reactivate HbF, and rewiring plasma cells in light chain amyloidosis and myeloma
28 oktober 2021 · 20 min
In this week’s episode, we’ll discuss an analysis of genetic risks for CMV infection after an allogeneic hematopoietic stem cell transplant, learn more about a new multiplex gene editing approach to reactivate fetal hemoglobin in thalassemia, and discuss transcriptional rewiring of normal plasma cell development in light-chain amyloidosis and myeloma. Lees meer
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Omidubicel vs standard umbilical cord blood transplant, CAR T-cell associated lymphomas, and CD63 and ferritin export
21 oktober 2021 · 20 min
In this week’s episode, we’ll review results of a phase 3 randomized study demonstrating shorter time to neutrophil and platelet recovery with an ex vivo expanded hematopoietic progenitor cell product called omidubicel as compared to standard umbilical cord transplantation. Next, we’ll look at preliminary data on the development of lymphomas originating from piggyBac-modified CD19 CAR T-cells, sounding a note of caution for researchers exploring new gene modification methodologies for CAR T-cell production. We’ll conclude with a report suggesting that the vesicular protein CD63 may orchestrate the transfer of iron-rich ferritin among cells. Lees meer
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Iron homeostasis and anemia of inflammation in TB, and long-term outcomes of gene therapy in adenosine deaminase-deficient SCID patients
14 oktober 2021 · 15 min
In this week’s episode, we will review a study that prospectively followed tuberculosis patients after treatment initiation to evaluate iron handling during the resolution of inflammatory anemia. We will also examine clinical benefit and long-term safety of gamma-retroviral gene therapy in patients with adenosine deaminase deficient severe combined immunodeficiency. Lees meer
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Immune megakaryocytes, PD1 deletions in CTCL, and COVID-19 vaccine and VITT
7 oktober 2021 · 19 min
In this week’s episode, we’ll review a study providing new insights on megakaryocyte diversity and function, including a unique subpopulation that may act as immune cells. Next, we’ll review research that intriguingly reveals a putative role for the PD1 gene in cutaneous T-cell lymphoma. Lastly, we’ll conclude with a report demonstrating a lack of cross-reaction between the antibodies that cause vaccine-induced thrombocytopenia and thrombosis, and the COVID-19 spike protein. Lees meer
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Inhibition of P-selectin as an anticoagulant, prognostic implications of CEBPA mutations in AML, and treating sickle cell disease with voxelitor
30 september 2021 · 15 min
In this week’s episode, we’ll review a study that demonstrates the efficacy of a synthetic inhibitor termed P-G6 that blocks P-selectin signaling and reduces thrombus formation in a pre-clinical model of non-occlusive venous thrombosis, learn more about how biallelic and single bZip CEBPA mutations have an equally favorable prognostic impact in acute myeloid leukemia, and examine the effects of modifying the pathophysiology of sickle cell anemia with therapeutic agents that increase oxygen affinity. Lees meer
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Caution on JAK inhibitors in HLH, CD44 loss sensitizes AML to venetoclax, and new insights on cell cycle control in Richter transformation
23 september 2021 · 15 min
In this week’s episode, we review results of preclinical investigations that sound a note of caution regarding the potential use of JAK inhibitors as treatment for hemophagocytic lymphohistiocytosis (or HLH), research that provides new insights on how CD44 loss of function sensitizes AML cells to the BCL-2 inhibitor venetoclax, and conclude with a report that demonstrates cooperation between B cell receptor signaling and genetic lesions in CDKN2A, CDKN2B and TP53 in Richter transformation. Lees meer