Volume 131, Issue 12



March 22, 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: Targeting protein S to ameliorate bleeding in hemophilia. See the article by
Prince et al

Minimal/measurable residual disease in AML: a consensus document from the European LeukemiaNet MRD Working Party
In this consensus document, the authors discuss the complex technical issues and scientific challenges of optimizing the use of measurable residual disease (MRD) to prognosticate outcomes in acute myeloid leukemia (AML).

Plasminogen replacement therapy for the treatment of children and adults with congenital plasminogen deficiency
Shapiro et al report successful preliminary results treating patients with plasminogen deficiency with Glu-plasminogen replacement therapy, reporting an increase in trough plasminogen levels and clinical improvement.

SETD1A protects HSCs from activation-induced functional decline in vivo
Using conditional knockout mice, Arndt and colleagues demonstrate that the histone methyltransferase SETD1A is not required for steady-state hematopoiesis but is critical for hematopoietic stem cell DNA repair pathways and for protection from inflammation-induced hematopoietic stress.

Measurable residual disease detection by high-throughput sequencing improves risk stratification for pediatric B-ALL
Wood et al present results of a high throughput sequencing strategy for diagnosing MRD in pediatric B-lymphoblastic leukemia (B-ALL). They demonstrate that this easily standardized approach offers better detection and improved risk stratification.

Targeting anticoagulant protein S to improve hemostasis in hemophilia
Prince and colleagues report that loss of protein S, which is an anticoagulant acting as cofactor for activated protein C and tissue factor pathway inhibitor, ameliorates the bleeding phenotype in hemophilic mice. This suggests that targeting protein S may offer an alternative approach to the treatment of both hemophilia A and B.

In vivo T-depleted reduced-intensity transplantation for GATA2-related immune dysfunction
Tholouli et al report excellent outcomes with reduced-intensity conditioning (RIC) transplantation in 4 patients with GATA2 mutations complicated by life-threatening infections and respiratory complications, suggesting that RIC transplant should be considered even in patients without myelodysplastic syndrome or AML.

α1-Antitrypsin infusion for treatment of steroid-resistant acute graft-versus-host disease
α1-Antitrypsin has been previously demonstrated to downmodulate inflammation and to alter ratios of regulatory and effector T cells. Magenau et al report impressive durable responses to infusion of α1-antitrypsin in patients with steroid-resistant acute graft-vs-host disease.

How I treat type 2B von Willebrand disease
Using 3 illustrative cases, Kruse-Jarres and Johnsen provide a useful approach to the diagnosis and treatment of type 2B von Willebrand disease.


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