Targeting C3 in autoimmune hemolytic anemias; venetoclax testing to predict AML response; an improved understanding of the structure of FXIII complex
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.Featured Articles:Safety and efficacy of pegcetacoplan treatment for cold agglutinin disease and warm antibody autoimmune hemolytic anemiaEx vivo venetoclax sensitivity predicts clinical response in acute myeloid leukemia in the prospective VenEx trialCryo-EM structure of the human native plasma coagulation factor XIII complex
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Review Series on Globin Disorders
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.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
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. Featured Articles:Brentuximab vedotin, nivolumab, doxorubicin, and dacarbazine for advanced-stage classical Hodgkin lymphomaIFN-I promotes T-cell–independent immunity and RBC autoantibodies via modulation of B-1 cell subsets in murine SCDGenetic 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
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.Featured Articles:Genetic iron overload aggravates, and pharmacological iron restriction improves, MDS pathophysiology in a preclinical studyLong-term follow-up of the STOPAGO studyAcute 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
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.Featured Articles:Dexamethasone dose intensity does not impact outcomes in newly diagnosed multiple myeloma: a secondary SWOG analysisAn erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSASafety and efficacy of standard-of-care ciltacabtagene autoleucel for relapsed/refractory multiple myeloma
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