Blood
Articles in this Issue
Identification of novel “driver” genes of multiple myeloma through genome-scale CRISPR activation studies
Abstract Loss-of-function studies (LOF) have historically been the main functional approach to identify and study genes which drive the biol...
The humanistic burden of patients with chronic myeloid leukemia (CML) treated with first line (1L) tyrosine kinase inhibitors (TKIs)
Abstract INTRODUCTION: While TKIs significantly extend survival in CML, their adverse events (AEs) may lead to treatment discontinuation and...
Development and validation of synthetic data generation over a federated learning computing framework to accelerate innovation and boost personalized medicine in hematological diseases
Abstract Background: Access to large, diverse, and high-quality datasets is essential for advancing research in rare hematological diseases...
Tumor-specific risk of acute kidney injury: A multi-cancer comparison across matched real-world cohorts
Abstract Despite its frequency and prognostic significance, acute kidney injury (AKI) remains poorly stratified across cancer types, limitin...
Behavioral health and biophysical complexity as predictors of healthcare utilization in adults with sickle cell disease
Abstract Background: Identifying predictors of high healthcare utilization in adults with Sickle Cell Disease (SCD) remains a clinical and o...
Experience with prolgolimab in the treatment of patients with relapsed and refractory classical Hodgkin lymphoma
Abstract Background: Modern treatment regimens for classical Hodgkin lymphoma (cHL) enable the cure of the majority of patients; however, re...
Unlocking the genetics of sickle cell disease
Abstract Introduction Sickle cell disease (SCD) is associated with wide variability in clinical disease. Factors like β-globin genotype, co-...
The unique role of PSMD2/Rpn1 among ubiquitin receptors in multiple myeloma highlights its potential as a therapeutic target to overcome proteasome inhibitor resistance
Abstract Background and Significance Therapeutic inhibition of the proteasomal catalytic activity of the 20S core particle (CP) by proteasom...
Disrupted DNA repair and oxidative stress responses underlie hematopoietic stem cell failure after spinal cord injury
Abstract Hematopoietic stem cells (HSCs) sustain lifelong blood and immune cell production through tightly regulated transitions between dor...
Transportation barriers and Medicaid's transportation service use in sickle cell disease: A mixed methods study in Wisconsin
Abstract Background: Individuals with sickle cell disease (SCD) often have socioeconomic barriers, limiting access to obtain care to manage...
Phase II Study of post transplant cyclophosphamide-based graft versus host disease prophylaxis after HLA-mismatched unrelated donor transplantation and reduced intensity conditioning: Results from the NMDP-sponsored access expansion Study
Abstract Allogeneic hematopoietic cell transplantation (HCT) using an HLA mismatched unrelated donor (MMUD) with calcineurin inhibitor-based...
First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD: Initial results of PRIME-0101
Abstract P47phox deficient phagocytic oxidase Chronic Granulomatous Disease (p47phox CGD) is an inherited immunodeficiency caused by mutatio...
Rare occurrence of liver failure secondary to sickle cell crisis and It's management
Abstract Intro: Sickle cell disease (SCD) affects more than 8 million people worldwide and liver disease is an important...
Avapritinib achieves deep and durable symptom control with a well-tolerated safety profile in ism: Long-term outcomes from pioneer
Abstract Introduction: Indolent systemic mastocytosis (ISM) is a clonal mast cell disease driven by the KIT D816V mutation in ~95% of cases...
Ruxolitinib increases immunogenicity and primes the tumor microenvironment for immune checkpoint blockade in 9p24.1 amplified lymphomas
Abstract Introduction: Amplification of 9p24.1 (9p+) upregulates PDL-1/2 and JAK2, leading to an immune-privileged tumor microenvironment (TM...
Simultaneous high-throughput identification of myeloid leukemia driver mutation-derived public neoantigens and cognate functional TCRs for TCR-T therapy
Abstract Many myeloid malignancies are driven by acquisition of recurrent somatic mutations which increase cancer cell fitness and growth. T...
Impact of uncontrolled hyperglycemia during induction chemotherapy on response and survival outcomes in patients with adult B-cell acute lymphoblastic leukemia (B-ALL) on HCVAD-based regimens
Abstract Hyperglycemia is a well-recognized complication of induction chemotherapy in patients with B cell acute lymphoblastic leukemia (B-A...
Efficacy and safety of zanubrutinib combined with g-CVP (Obinutuzumab, Cyclophosphamide, Vindesine, Prednisone) for first-line treatment of follicular lymphoma
Abstract IntroductionObinutuzumab combined with immunochemotherapy significantly improves outcomes in follicular lymphoma (FL) with high tum...
Clinical characterization of the rare G6PD pawnee variant: A five-patient case series
Abstract Glucose-6-phosphate dehydrogenase (G6PD) deficiency is a common genetic condition, affecting an estimated 500 million people worldw...
NXT007 prophylaxis in people with hemophilia A with or without FVIII inhibitors: A global Phase I/II multiple-ascending-dose study
Abstract Background: NXT007 is a next-generation bispecific antibody, based on emicizumab, that mimics the cofactor function of activated fa...
Cumulative burden of myelodysplasia-related gene mutations is associated with poor leukemia-free survival in ELN-2022 favorable-risk AML
Abstract Background: The new category acute myeloid leukemia (AML) with myelodysplasia-related (MDS-R) gene is designated as “adverse-risk”...
Altered bone marrow (BM) plasma amino acid (AA) composition reflects microenvironmental remodeling in patients with multiple myeloma (MM) compared to monoclonal gammopathy of unknown significance (MGUS)
Abstract Altered Bone Marrow (BM) Plasma Amino Acid (AA) Composition Reflects Microenvironmental Remodeling in Patients with Multiple Myelom...
Comparison of clinical outcomes in multiple myeloma patients treated with chimeric antigen receptor (CAR)-T cell therapy across clinical trials: Intention-to-treat vs. per-protocol analyses
Abstract Background: CAR T-cell therapy is an effective treatment for relapsed/refractory multiple myeloma (MM). CAR T-cell therapy is regar...
Smartphone app for noninvasive, at-home pediatric hemoglobin monitoring in sickle cell disease.
Abstract Anemia is a widespread health issue impacting more than 2 billion people worldwide. In sickle cell disease (SCD), which impacts mor...
Treatment patterns and outcomes among patients treated with second-generation BTK inhibitors in CLL
Abstract Background Next-generation covalent Bruton tyrosine kinase inhibitors (BTKi), including zanubrutinib (ZANU) and acalabrutinib (ACA)...