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Volume 131, Issue 9

 

 

March 1, 2018 – 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.

Cover Figure: Platelet glycoprotein VI contributes to local antibacterial responses to pneumonia in mice.  See the article by Mayer et al.

Nbeal2 interacts with Dock7, Sec16a, and Vac14
These investigations illuminate the molecular mechanisms of platelet α-granule biogenesis and the molecular defects of gray platelet syndrome through the identification of the Nbeal2 interactome.


Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B
Miesbach et al show that adeno-associated virus 5 vector liver-directed gene therapy in severe and moderate hemophilia B is clinically effective and safe and leads to stable factor IX expression.


A model for predicting effect of treatment on progression-free survival using MRD as a surrogate end point in CLL
Dimier and colleagues quantitate for the first time the relationship between minimal residual disease (MRD) negativity and progression-free survival in patients receiving chemoimmunotherapy as a first-line treatment for chronic lymphocytic leukemia (CLL), establishing its validity as a surrogate marker for treatment outcome.


Red cell exchange transfusions lower cerebral blood flow and oxygen extraction fraction in pediatric sickle cell anemia
The authors report that white matter of the brain is chronically hypoxic in sickle cell disease and that transfusions acutely lower the volume of brain tissue at risk for stroke.


Syndecan-1 promotes Wnt/β-catenin signaling in multiple myeloma by presenting Wnts and R-spondins
The authors investigate mechanisms underlying a novel strategy to block multiple myeloma cell proliferation by targeting the heparin sulfate proteoglycan syndecan-1. They show that the Wnt/β-catenin pathway is a critical factor in this process.


Preclinical efficacy of daratumumab in T-cell acute lymphoblastic leukemia
This paper reports that CD38 is expressed in pediatric T-cell acute lymphoblastic leukemia (T-ALL) at the time of diagnosis and at the time of relapse. Targeting CD38 with the monoclonal antibody daratumumab shows substantial therapeutic efficacy in multiple patient-derived xenograft models of T-ALL.

 

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Blood (www.bloodjournal.org), 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) (www.hematology.org), 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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