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 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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Stay up to date on the latest research and reviews for both clinical and basic hematology with Blood podcast, published weekly in conjunction with each issue of Blood. Additionally, Blood review series podcasts feature in depth discussion with authors of recently published review articles. Subscribe and never miss a new episode.