Issue 13, Volume 127

Cover Figure: Podoplanin/CLEC-2 interaction promotes murine megakaryocyte growth and proplatelet formation. See the article by Tamura et al.

WASHINGTON, March 31, 2016 – Welcome to “This Week in Blood,” a weekly snapshot of the hottest studies from each week’s issue of Blood, the official journal of the American Society of Hematology (ASH), hand-picked by Blood Editor-in-Chief Bob Löwenberg, MD, PhD, and Deputy Editor Nancy Berliner, MD.

A first-in-human phase 1 study of ACE910, a novel factor VIII–mimetic bispecific antibody, in healthy subjects
In this week's plenary paper, Uchida and colleagues report the results of a phase 1 study of a novel hemophilia therapy. ACE910 is a bispecific antibody that juxtaposes factors IX and X to activate factor Xa while totally bypassing factor VIII. The antibody has a half-life of 4-5 weeks and is well tolerated in normal volunteers. This agent promises to transform the therapy of patients with hemophilia, especially those with acquired inhibitors.

Janus kinase inhibition lessens inflammation and ameliorates disease in murine models of hemophagocytic lymphohistiocytosis
In this week's Blood, Das and colleagues report that ruxolitinib is an effective treatment in 2 different mouse models of hemophagocytic lymphohistiocytosis (HLH). Using the perforin-null mouse model of familial HLH and a CpG injection model of autoimmune-associated secondary HLH, they demonstrate that Janus kinase inhibition decreases T-cell expansion and inflammatory cytokine production while improving clinical manifestations of HLH. This promising new therapy for the deadly disease is already being examined in human clinical trials.

Serum-resistant CpG-STAT3 decoy for targeting survival and immune checkpoint signaling in acute myeloid leukemia
STAT3 acts as an oncogene in acute myeloid leukemia (AML) by suppressing antitumor immune responses. In this issue of Blood, Zhang and colleagues use a STAT3 "decoy" encoding the STAT3-binding site conjugated to TLR9 ligand to target AML in a mouse model, exploiting the fact that AML cells express TLR9 while normal hematopoietic stem cells do not. They demonstrate remarkable efficacy of this agent in reversing immunosuppressive signaling, leading to AML cell eradication through direct or indirect immune pathways.

What is going on between defibrotide and endothelial cells? Snapshots reveal the hot spots of their romance

Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure
In this issue, 2 articles shed light on the role of defibrotide (DF) in the treatment of severe veno-occlusive disease (VOD), a severe complication of stem cell transplantation that frequently leads to multiorgan failure and death. In one article, Palomo and colleagues investigate the interaction of DF with endothelial cells that might explain its role as an endothelial protective agent. They report that DF binding to endothelial cell membranes is sufficient to induce meaningful anti-inflammatory and antioxidant effects. In the other article, Richardson and colleagues report on a phase 3 trial that confirms that DF improves survival and completes recovery of patients with VOD and multiorgan failure.

A meta-analysis of low-molecular-weight heparin to prevent pregnancy loss in women with inherited thrombophilia
In this week's issue, Skeith and colleagues perform a meta-analysis of the efficacy of low-molecular-weight heparin (LMWH) in preventing pregnancy loss in patients with inherited thrombophilia. They report that in patients with inherited thrombophilia and prior late or recurrent early pregnancy loss, LMWH did not improve live birth rates. They suggest that there is no benefit for testing for thrombophilia or for treatment with LMWH.

This week's complete table of contents

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Blood (, the most cited peer-reviewed publication in the field of hematology, is available weekly in print and online. Blood is the official journal of the American Society of Hematology (ASH) (, the world’s largest professional society concerned with the causes and treatment of blood disorders.

ASH’s mission is to further the understanding, diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems by promoting research, clinical care, education, training, and advocacy in hematology.

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