Issue Archive

In this issue’s Blood Advances Talk, Tritschler and Wells discuss the role of extended therapy beyond the typical 3 months for patients with unprovoked venous thromboembolism. We hope you enjoy this discussion.

In utero stem cell transplantation holds promise to treat a broad range of congenital disorders. In a Stimulus Report, Magnani and colleagues describe a case of severe combined immunodeficiency successfully treated in utero using bone marrow from a matched sibling donor.

RUNX1 mutations are known to contribute to leukemic transformation. Vellenga and colleagues introduce the RUNX1-S291fs300X mutation into human CD34+ cells and induced pluripotent stem cells to study its role in leukemogenesis. The mutation has important biological effects. 

Hairy cell leukemia (HCL) is a tumor of mature B cells. The role of DNA methylation in this leukemia is explored by Forconi and colleagues to identify a methylation-driven signature of this HCL to better characterize this disease and aid future drug development efforts.

Nicotinamide phosphoribosyltransferase is involved in the generation of NAD and is a potential target in acute myeloid leukemia (AML). Mitchell and colleagues evaluated a novel inhibitor, KPT-9274, in preclinical models that holds promise in future clinical studies of patients with AML. 

The cell of origin of leukemic transformation is an area of ongoing debate. The study from the laboratory of Ema and colleagues explores the impact of self renewal and differentiation potentials in leukemia development and highlights the multiple cells of origin from which transformation can occur.

The asymmetric distribution of specific phospholipids on the cell surface is maintained through phospholipid flippases. Broer and colleagues studied the flippase ATP8A1 in murine and human platelets, revealing a novel mechanism of flippase cleavage that differed between activated and apoptotic platelets. 

Platelets have been identified with diverse functions in coagulation, host tissue repair, and immune activity. Lee and colleagues report another important finding related to platelet function in animal models of lung disease. They find platelets protect against lung injury from pathogen-secreted virulence factors.  

Malglycemia, defined as hyperglycemia or hypoglycemia, is associated with increased complications of adult patients in many clinical settings. Forlenza and colleagues studied the impact of malglycemia in a cohort of pediatric and adolescent hematopoietic stem cell transplant patients, finding the disorder is associated with increased risk of morbidity and mortality in these patients too.