Emicizumab reduces bleeding in hemophilia A

Emicizumab (Hemlibra)
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Final results from the HAVEN 3 study suggest emicizumab prophylaxis can reduce bleeding in hemophilia A patients without factor VIII inhibitors. Compared to patients who did not receive prophylaxis, those who received emicizumab prophylaxis had a 96% to 97% reduction in treated bleeds and a 94% to 95% reduction in all bleeds. An intra-patient comparison showed a 68% reduction in treated bleeds with once-weekly emicizumab, compared to prior factor VIII prophylaxis. [Read Article]

FDA approves drug for thrombocytopenia in CLD

Thrombocytopenia
Image from Erhabor Osaro
The US Food and Drug Administration (FDA) has approved avatrombopag (DOPTELET®), a second-generation thrombopoietin receptor agonist. Avatrombopag is now approved to treat thrombocytopenia in adults with chronic liver disease (CLD) who are scheduled to undergo a procedure. Dova Pharmaceuticals, Inc., plans to launch the drug in June. [Read Article]

Protein may be therapeutic target for AML

AML cells
Image by Lance Liotta
A signaling protein may be a “potent” therapeutic target for acute myeloid leukemia (AML), according to a paper published in the Journal of Experimental Medicine. The protein, interleukin-1 receptor accessory protein (IL1RAP), is often highly expressed on the surface of leukemic stem cells (LSCs) but largely absent from normal hematopoietic stem cells. Despite this, it wasn’t known whether LSCs require IL1RAP to survive and proliferate and whether inhibiting IL1RAP could be a successful way to treat AML. [Read Article]