Table of Contents
CLINICAL TRIALS AND OBSERVATIONS
Phase 1 study of the selective BTK inhibitor zanubrutinib in B-cell malignancies and safety and efficacy evaluation in CLL
Clinical Trials & Observations
This paper offers the first description of zanubrutinib, a new Bruton tyrosine kinase (BTK) inhibitor with an improved safety profile.
IMMUNOBIOLOGY AND IMMUNOTHERAPY
The results of this study show that chimeric antigen receptor (CAR) T-cell therapy with tisagenlecleucel, a CD19-specific CAR T-cell product, is a feasible option for patients with secondary central nervous system (CNS) diffuse large B-cell lymphoma, with limited toxicity and potential efficacy.
The investigators used genomic techniques to interrogate a large series of neutrophilic myeloid neoplasms, including atypical chronic myeloid leukemia, chronic neutrophilic leukemia, and others. They identify unique mutation patterns and a detailed genomic landscape that suggest that the current clinicopathologically defined entities may not be biologically relevant.
THROMBOSIS AND HEMOSTASIS
Genetic determinants of VWF clearance and FVIII binding modify FVIII pharmacokinetics in pediatric hemophilia A patients
Factor VIII (FVIII) pharmacokinetic (PK) properties show high interpatient variability in hemophilia A patients. This study evaluates how von Willebrand factor (VWF) genetic variants modify the PKs of FVIII and provides important new mechanistic insights in this area.
Posttransplant cyclophosphamide vs antithymocyte globulin in HLA-mismatched unrelated donor transplantation
LETTER TO BLOOD
Natural history and cell of origin of TCF3-ZNF384 and PTPN11 mutations in monozygotic twins with concordant BCP-ALL
In a series of elegant studies, the investigators elucidate the developmental path of a TCF3-ZNF384/PTPN11–driven B-cell acute lymphoblastic leukemia (BCP-ALL) with different PTPN11 mutations. Through the analysis of a pair of identical twins, they formally demonstrate for the first time that TCF3-ZNF384 is a primary prenatal event; these results are relevant both for understanding the disease and for potential therapies.
B-cell receptor sequencing network for a monozygotic twin with concordant B-cell precursor acute lymphoblastic leukemia to track the clonal origin of the leukemia. See the article by Bueno et al on page 900.
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