von Willebrand factor levels in the diagnosis of von Willebrand disease: a systematic review and meta-analysis
This comprehensive, systematic review by Kalot and colleagues assesses the clinical practice guidelines on the management and diagnosis of von Willebrand Disease (VWD), developed by a combined effort from the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH), the National Hemophilia Foundation (NHF), the World Federation of Hemophilia (WFH), and the University of Kansas Medical Center (KUMC). This review evaluates published outcomes of different patient groups with suspected and confirmed VWD and looks at the accuracy of commonly used testing strategies.
TCRαβ/CD19 depleted HSCT from an HLA-haploidentical relative to treat children with different nonmalignant disorders
Merli and colleagues report on the results of a prospective study of TCRα/β depleted haploidentical stem cell transplantation in 70 pediatric patients with nonmalignant diseases. The results show excellent outcomes although there was a relatively high (30%) incidence of graft failure. This data will help inform pediatric transplanters when considering haploidentical transplant strategies for their patients with nonmalignant diseases and will help inform future clinical trials using this approach.
KAT2A complexes ATAC and SAGA play unique roles in cell maintenance and identity in hematopoiesis and leukemia
Arede et al demonstrate the roles of KAT2A complexes in acute myeloid leukemia cell maintenance and erythroid differentiation. Specifically, they identify that different KAT2A complexes (ATAC and SAGA) appear to regulate differentiation and ribo/metabolism genes in myeloid leukemias.
Transcriptomic analysis reveals proinflammatory signatures associated with acute myeloid leukemia progression
Stratmann et al report on an RNA-sequencing analysis from samples derived from 70 pediatric and adult patients with acute myeloid leukemia (AML). Their findings reveal novel or previously unappreciated differentially expressed genes associated with tumor progression in AML (eg CR1, GLI2 and IL1R1), many of which indicate the promotion of a pro-inflammatory signature. Furthermore, through a machine learning-based analysis, they were able to depict gene interactive networks and additional relapse-predictive expression patterns in AML. These results emphasize the importance of applying multilevel analysis on patient-matched diagnosis and relapse samples to better understand tumor progression and therapy resistance in AML.
Mutations in CEBPAmu are found in approximately 10% of patients with acute myeloid leukemia and have been found to impact prognosis. In this study by Yamaguchi and colleagues, a large number of patients with different forms of this mutation were examined to assess prognosis and clinical impact.
Liu et al studied how iron levels in cord blood-derived megakaryocytes regulate differentiation in vitro in response to eltrombopag (ELT). ELT showed dose-dependent opposing effects on megakaryocyte differentiation in cultures. Specifically, low concentrations of ELT stimulated megakaryopoiesis while high concentrations suppressed megakaryocyte differentiation and proliferation.