Volume 130, Issue 21

Cover Figure: Novel pathway by which TGF-β regulates HSC retention in the niche. See the article by Yahata et al.

WASHINGTON, November 23, 2017 – 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.

Ibrutinib for chronic graft-versus-host disease after failure of prior therapy
In this plenary paper, the investigators present results from a prospective phase 1b/2 study on the Bruton tyrosine kinase inhibitor ibrutinib in patients in need of salvage immunosuppressive treatment for chronic graft-versus-host disease (cGVHD). High sustained response rates amongst other findings led for the first time to Food and Drug Administration approval of an agent in refractory cGVHD.

Kinetics and biomarkers of severe cytokine release syndrome after CD19 chimeric antigen receptor–modified T-cell therapy
Clinical trials of CD19 chimeric antigen receptor (CAR) T cells exert a transformative impact on the treatment of multiple relapsed and refractory B-cell malignancies, but the cytokine release syndrome (CRS) has appeared as a serious complication. The authors describe informative cytokine profiles associated with CRS in a large cohort of adults treated with CD19-directed CAR-modified T cells.

Novel insights into the clinical phenotype and pathophysiology underlying low VWF levels
The investigators present an in-depth analysis of the relationship between the variable levels of reduced von Willebrand factor (VWF), bleeding phenotype, laboratory “phenotype,” and genetic background. They also offer clinically important new insights relevant for diagnosis and treatment.

Novel approaches to hemophilia therapy: successes and challenges
This Blood Spotlight review of novel therapeutic approaches for hemophilia highlights newly approved therapies as well as those in early and late clinical trials. It covers extended half-life factors, nonfactor therapies, and important advances in gene therapy. It also predicts their place in future therapy.

Inherited bone marrow failure syndromes: considerations pre- and posttransplant
This review discusses the important aspects of the most common inherited bone marrow failure syndromes and patient risks before and after stem cell transplantation.

Signaling pathways and immune evasion mechanisms in classical Hodgkin lymphoma
The use of antibodies against programmed death protein-1 (PD-1), programmed death-ligand 1 (PD-L1), and PD-L2 is a hot topic in the treatment of malignant lymphomas. This review of classical Hodgkin lymphoma focuses on the role of the PD-1–PD-L1/PD-L2 receptor-ligand pair in Hodgkin lymphoma biology and its importance as a target for treatment.

Thrombocytopenia in pregnancy
This article describes the causes, diagnosis, and management of thrombocytopenia in pregnancy, an important subject for the practicing hematologist.

FCR achieves long-term durable remissions in patients with IGHV-mutated CLL
The authors report a systemic review on the efficacy of the fludarabine-cyclophosphamide-rituximab (FCR) chemotherapy regimen in first-line therapy of chronic lymphocytic leukemia (CLL). Emerging data indicate that patients with IGHV-mutated CLL who receive FCR chemotherapy do well long term. FCR chemotherapy remains a clinically valid option in the era of various new therapeutic agents for CLL.

Deletion of BMP6 worsens the phenotype of HJV-deficient mice and attenuates hepcidin levels reached after LPS challenge
Lack of bone morphogenetic protein BMP6 or the BMP coreceptor hemojuvelin (HJV) leads to a similar phenotype with hepcidin insufficiency and tissue iron accumulation. This work demonstrates that BMP6 also contributes to hepcidin expression in the absence of HJV, showing that BMP6 and HJV can regulate hepcidin independently of each other.

Enhancer profiling identifies critical cancer genes and characterizes cell identity in adult T-cell leukemia
The manuscript describes a novel approach of integrative analysis of superenhancer profiling and gene expression profiling and identifies novel cancer-associated genes in adult T-cell leukemia.

Effective “activated PI3Kδ syndrome”–targeted therapy with the PI3Kδ inhibitor leniolisib
This paper presents the first prospective trial of a PI3Kδ inhibitor in patients with activated PI3Kδ syndrome (APDS) caused by activating mutations in PIK3CD. The authors demonstrate the cellular effects and clinical efficacy of treatment with a newly designed small molecule inhibitor that targets PI3Kδ hyperactivation in APDS.


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.

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