Blood
Articles in this Issue
BCMA-directed CAR-T cell therapy induces durable drug-free remission in refractory primary immune thrombocytopenia
This first clinical evaluation of BCMA CAR-T cell therapy in four patients with refractory primary ITP demonstrated manageable safety, rapid and sustained platelet recovery, and durable drug-free comp...
How I Treat Thrombotic Microangiopathy in the Setting of Triggers
Complement-mediated thrombotic microangiopathy can be difficult to recognize when it presents in the setting of an apparent clinical trigger, such as pregnancy, severe hypertension, or kidney transpla...
CD70 Overexpression at Diagnosis Predicts Relapse and supports dual CD19-CD70 CAR-T Therapy in Follicular Lymphoma
Although first-line immunotherapy achieves remission in most patients with follicular lymphoma (FL), improved biomarkers and therapeutic strategies are required to identify and manage those who relaps...
IL-6 receptor inhibition promotes expansion of cytolytic CD4+ T cells after cellular immunotherapy.
Graft-versus-host disease (GVHD) remains a major barrier to the success of allogeneic hematopoietic stem cell transplantation (HSCT). In preclinical models, dysregulation of IL-6 in the peri-transplan...
Human precursor labeling in situ reveals a conveyor belt differentiation trajectory and a 6-day neutrophil life span
The kinetics of neutrophil development in human bone marrow remain largely unclear, despite the large implications for the mechanisms underlying host defense and inflammatory responses. Here, in situ...
Anti-αvβ3 antibodies in FNAIT are associated with disturbed angiogenesis and intracranial hemorrhage in men and mice
In Caucasians, maternal allo-immunization against human platelet antigen (HPA)-1a is the most common cause for fetal and neonatal alloimmune thrombocytopenia (FNAIT). Intracranial hemorrhage (ICH) is...
Factor VIII Aurora: A Naturally Occurring Gain of Function FVIII Variant with Enhanced FIXa Affinity
Factor VIII Aurora (FVIII-R571S) is the first described naturally occurring enhanced-potency FVIII variant identified in a patient with recurrent thrombosis and early mortality. The patient's plasma e...
Gut Microbiota Drives Aging-related Erythropoiesis Impairment via Phenylacetic Acid-induced Histone Phenylacetylation
Anemia, the most prevalent hematologic disorder in older adults, imposes a significant burden of cardiovascular events, cognitive decline, and mortality. However, the mechanisms underlying aging-relat...
Rapid Peak Cilta-cel Expansion is Associated with Delayed Neurotoxicity in Multiple Myeloma
The impact of chimeric antigen receptor (CAR)-T cell expansion and persistence on clinical outcomes and treatment-related morbidity in patients with relapsed/refractory multiple myeloma (RRMM) remains...
The CYB5R3 T117S Missense Variant is Associated with Attenuated Riociguat Efficacy in Sickle Cell Disease
Sickle cell disease attenuates nitric oxide signaling, limiting soluble guanylyl cyclase (sGC) stimulation needed for hemo-vascular function. Cytochrome b5 reductase 3 (CYB5R3) regulates sGC activity...
USP22 is a novel vulnerability regulating MEIS1 protein abundance and gene transcription in KMT2Ar acute leukemia
Patients with acute leukemias harboring translocations involving gene lysine methyltransferase 2A (KMT2A) have a poor prognosis due to chemotherapy resistance with rapid relapse following standard tre...
Tagraxofusp, Azacitidine, and Venetoclax in Blastic Plasmacytoid Dendritic Cell Neoplasm
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an orphan, aggressive hematologic malignancy characterized by high CD123 expression. Tagraxofusp (TAG), a CD123-directed toxin, is the only appr...
Single Cell Analysis of the Tumor Microenvironment Landscape Across the Disease Spectrum of Multiple Myeloma
Multiple myeloma progresses from precursor states to active disease, and studying tumor microenvironment (TME) evolution across these stages is key to understanding immune dysregulation and therapeuti...
Concurrent administration of BCMA and GPRC5D chimeric antigen receptor (CAR) T cells in advanced multiple myeloma
BCMA and GPRC5D-directed therapies have shown high efficacy in the treatment of relapsed or refractory myeloma with multiple new immune therapies now approved. Preclinical studies have shown that expr...
The Chaos of Choice in Large B-cell Lymphoma: A Call to Harmonize First-line Trial Design
After five decades of CHOP-based therapy, the treatment landscape for patients with newly diagnosed large B-cell lymphoma (LBCL) is rapidly evolving. Novel agents, including antibody-drug conjugates,...
Precision Transfusion Medicine in the Omics Era
Transfusion medicine has practiced a form of precision medicine for decades through compatibility testing, infectious disease screening, component manufacturing, traceability, and hemovigilance. Yet b...
Decentralized Clinical Trials in Hematology: the Promise and the Peril
Decentralized clinical trials (DCTs) are clinical studies where part or all trial-related activities occur remotely, reducing or eliminating the need for patients to physically visit a research site....
How I Treat Chemotherapy-Induced Thrombocytopenia with Thrombopoietin Receptor Agonists
Chemotherapy-induced thrombocytopenia (CIT) is a common complication of cancer therapy that may result in major bleeding events and reduction of chemotherapy relative dose intensity. While there remai...
Fibrocytes drive JAK2V617F-mutated myelofibrosis: pitavastatin reverses marrow fibrosis and anemia
Bone marrow (BM) fibrosis in primary and post-polycythemia vera/essential thrombocythemia myelofibrosis (MF) has traditionally been considered a reactive process driven by cytokines, such as transform...
First-line ibrutinib plus venetoclax for non-blastoid mantle cell lymphoma in patients ≥65 years or with TP53 mutations
The phase 3 SYMPATICO study included an open-label cohort to evaluate the efficacy and safety of first-line ibrutinib plus venetoclax in patients with non-blastoid mantle cell lymphoma (MCL) ≥65 years...
R spondin 2 regulates regeneration of the hematopoietic stem cell niche
Hematopoietic stem cells (HSCs) depend upon paracrine signals from bone marrow endothelial cells (BM ECs) and perivascular stromal cells for their maintenance and regeneration. Chemotherapy and total...
Durable efficacy and manageable long-term safety of lisocabtagene maraleucel in 3L+ FL: 3-year update from TRANSCEND FL
In the TRANSCEND FL primary analysis, lisocabtagene maraleucel (liso-cel) showed high response rates and favorable safety in patients with relapsed/refractory (R/R) follicular lymphoma (FL). Longer fo...
CD4+CD25+FOXP3+ Regulatory T Cells to Protect Against Graft Versus Host Disease
Allogeneic hematopoietic cell transplantation (HCT) has benefited many patients with hematological malignancies and bone marrow failure states. However, graft vs host disease (GVHD) and immune incompe...
Phosphorylated DEK sustains leukemia stem cells by enabling PBX3-driven transcriptional reprogramming
Leukemia stem cells (LSCs) drive acute myeloid leukemia (AML) initiation, relapse, and chemoresistance, yet the core post-translational events sustaining LSC maintenance remain poorly defined. Here, t...
A single-cell atlas identifies oncogenic transcriptional programs and immune escape mechanisms in CTCL
Primary cutaneous T-cell lymphomas (CTCL) are a heterogeneous group of extranodal non-Hodgkin lymphomas. Outcomes for patients with advanced-stage disease are suboptimal, as few complete and durable r...