In this bonus episode of Blood Podcast, Coordinating Editor, Dr. Jason Gotlib is joined by Drs. Sioban Keel, Margaret Ragni, and Andoni Echaniz-Laguna to discuss the Review Series on RNA Therapeutics in Hematology. The series highlights areas in which RNA therapeutics are most advanced: acute hepatic porphyria, transthyretin amyloidosis, and hemophilia.

Review Series on RNA Therapeutics in Hematology

The Blood Podcast summarizes content recently published in Blood, the most cited peer-reviewed publication in the field of hematology.

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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.  Featured Articles: ADAMTS13 recovery in acute thrombotic thrombocytopenic purpura after caplacizumab therapySonrotoclax overcomes BCL2 G101V mutation–induced venetoclax resistance in preclinical models of
hematologic malignancySingle-cell landscape of idiopathic multicentric Castleman disease in identical twins

ADAMTS13 recovery in caplacizumab-treated TTP; inhibition of venetoclax-resistant BCL2 variants with sonrotoclax; genomic insights from Castleman disease in twins

In this week's episode, we’ll discuss the findings from a study assessing responses after treatment with tisagenlecleucel [LM1] in adults with relapsed/refractory follicular lymphoma, learn more about the association between high microRNA-145 plasma levels and decreased risk of future incident venous thromboembolism, and discuss how epigenetic and immunogenetic signatures can be used in the prediction of outcomes for high-count monoclonal B lymphocytosis. 
Featured Articles: Durable Response After Tisagenlecleucel in Adults With Relapsed/Refractory Follicular Lymphoma: ELARA Trial Update Prediction of outcomes for high-count monoclonal B lymphocytosis using an epigenetic and immunogenetic signature High microRNA-145 plasma levels are associated with decreased risk of future incident venous thromboembolism: The HUNT study 

Durable response after tisa-cel in adults with relapsed/refractory follicular lymphoma; high microRNA-145 plasma levels indicate decreased risk of VTE; epigenetic/immunogenetic signature in the predic

In this week's episode, we'll discuss gut microbiota exploitation by CPX-351 in acute myeloid leukemia. Then we'll learn about optimizing anti-myeloma immunity. New research shows that regulatory T cells suppress myeloma-specific immunity during autologous stem cell mobilization and transplantation. Finally we'll discuss among pediatric patients with ITP or other autoimmune cytopenias, which ones will go on to develop systemic lupus?  Featured Articles: CPX-351 exploits the gut microbiota to promote mucosal barrier function, colonization resistance, and immune homeostasisRegulatory T cells suppress myeloma-specific immunity during autologous stem cell mobilization and transplantationAntinuclear antibody–associated autoimmune cytopenia in childhood is a risk factor for systemic lupus erythematosus

Gut microbiota exploitation by CPX-351 in AML; optimizing anti-myeloma immunity for ASCT; which pediatric ITP patients will develop lupus?

In this week's episode we’ll discuss the mechanism by which Jak2V617F clonal hematopoiesis promotes arterial thrombosis, discuss how Staphylococcus aureus induces drug resistance in cancer T cells in Sézary syndrome, and learn more about the clinical and functional features of RAC2-related immunodeficiency. Featured Articles:Jak2V617F clonal hematopoiesis promotes arterial thrombosis via platelet activation and cross talkStaphylococcus aureus induces drug resistance in cancer T cells in Sézary syndromeClinical and functional spectrum of RAC2-related immunodeficiency



Jak2V617F clonal hematopoiesis promotes arterial thrombosis; Staphylococcus aureus infection in Sézary syndrome; clinical features of RAC2-related immunodeficiency

In this week's episode we'll discuss molecular mimicry in aplastic anemia, novel experiments show that antigens associated with viral infections can mimic epitopes presented on hematopoietic progenitor cells. Then, we'll learn about prophylaxis with subcutaneous emicizumab in infants with hemophilia. Finally we'll see how hydroxyurea is associated with a significant reduction in infections among children with sickle cell anemia in Uganda. Articles featured this week:Virus-reactive T cells expanded in aplastic anemia eliminate hematopoietic progenitor cells by molecular
mimicryEmicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label
trialHydroxyurea reduces infections in children with sickle cell anemia in Uganda

Review Series on RNA Therapeutics in Hematology

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