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Volume 3, Issue 15

 

Cover Image

Cover Figure: Features of light chain proximal tubulopathy. Immunohistochemistry stained strongly positive for κ light chain in proximal tubules; λ, not shown, was negative (original magnification ×400; DAB + Harris’s hematoxylin stain).
See the article Jain et al.

WASHINGTON, D.C., August 13, 2019 – Welcome to the “Advance Notice,”  newsletter which provides highlights from issues of Blood Advances, the open-access journal of the American Society of Hematology (ASH), that  are hand-picked by Blood Advances Editor-in-Chief Robert Negrin, MD.

A number of recurring mutations in patients with acute myeloid leukemia that carry important prognostic implications have been identified. Chan and colleagues explored the extent to which these mutations impact the recovery of hematopoiesis following induction chemotherapy. They found that certain mutations result in clinically relevant delays in blood count recovery.

 

 

Chimeric antigen receptor (CAR) T cells directed against CD19 and CD22 have resulted in dramatic responses in a subset of patients with B-cell malignancies. These cells are constructed with either retroviral or lentiviral vectors where possible integration near key control genes has been a concern. In an Exceptional Case Report, Shah and colleagues describe a patient whose CAR T cells expanded after disease relapse that demonstrated clonal expansion with integration of the CAR into the CBL gene, a known regulator of T-cell function. These findings suggest that specific integration may impact function of the resulting engineered cells.

 

Tyrosine kinase inhibitors have revolutionized the treatment of chronic myelogenous leukemia. Ponatinib has the widest inhibitory spectrum and can be used in patients with the T315I mutation. However, ponatinib has resulted in a high risk of thrombotic events. This topic is explored in the manuscript by Schmaier and colleagues who have evaluated the mechanisms of thrombosis and strategies to mitigate this complication of treatment.

 

 

It is well recognized that monoclonal gammopathy can result in significant renal dysfunction. This review by Ramasamy and colleagues will be informative and useful in describing the pathophysiology and management of patients with monoclonal gammopathy of renal significance.

 

Activation and damage to the endothelium have been thought to be involved in a number of complications following allogeneic hematopoietic stem cell transplantation. Although difficult to measure, strategies to limit and repair endothelial damage have been explored through a number of pharmacological and other approaches. This challenging topic is reviewed by a group of experts in this area led by Socié.

 

Apixaban and rivaroxaban have both been approved for the treatment of acute venous thromboembolism (VTE). In a Systematic Review, Kouides and colleaguescompare these 2 agents with respect to the efficacy of preventing recurrent VTE and the safety with respect to bleeding.

 

 

Plant homeodomain finger 6 (Phf6) has been implicated in a number of different diseases, including leukemia Tien and colleagues explore the role of Phf6 in hematopoiesis through the generation of knockout mice, identifying many different ways in which the loss of this 365-amino-acid protein impacts hematopoietic stem cell biology.

 

Evaluating the pain that children with sickle cell disease (SCD) suffer from and how that impacts behavior can be difficult. To overcome this challenge, the Patient Reported Outcomes Measurement Information System (PROMIS) has been developed. Singh and Panepinto evaluated clinical interpretations in a large cohort of children with SCD to provide guidance for PROMIS users in the clinic.

 

Splenic injury and dysfunction are common complications in patients with sickle cell anemia. Hoss and colleagues systematically studied the determinants of spleen injury in infants with sickle cell anemia and now provide an in-depth analysis of this often-devastating complication of this disease.

 


Featured Visual Abstract



A phase 2 study of brentuximab vedotin in patients with CD30-positive advanced systemic mastocytosis

Jason Gotlib, John H. Baird, Tracy I. George, Cheryl Langford, Isabel Reyes, Justin Abuel, Cecelia Perkins, Kurt Schroeder, Prithviraj Bose and Srdan Verstovsek

 

 

 

Blood Advances is the open-access 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.