Blood
Articles in this Issue
Title: Clinical trial availability and receipt of guideline concordant care among adolescents and young adults (AYAs) with acute lymphoblastic leukemia
Abstract BACKGROUND: Adolescents and young adults (AYA: 15-39y) with acute lymphoblastic leukemia (ALL) face poor survival with minimal hist...
Phase 1/2 study of MP0533, a tetra-specific T cell engager (CD33 x CD123 x CD70 x CD3), in patients with relapsed/refractory AML or MDS/AML: Initial results from optimized treatment regimen including densified MP0533 dosing and adapted premedication
Abstract BACKGROUND: MP0533 is a tetra-specific CD3-engaging DARPin designed for avidity-driven T cell-mediated killing of acute myeloid leuk...
Outcomes in children and adolescents with down syndrome and B-lymphoblastic leukemia: A report from Children's oncology group study AALL1731
Abstract Background: Individuals with DS have a 20-fold increased risk of B-ALL, and experience increased rates of both relapse and treatmen...
Effect of FLT3 maintenance in older FLT3-ITD+ adults undergoing allogeneic HCT. a post hoc analysis of BMT CTN 1506
Abstract INTRODUCTION Allogeneic hematopoietic cell transplantation (alloHCT) is indicated in patients (pts) with FLT3-ITD mutated AML. The...
Caplacizumab as a single agent without plasma exchange in relapsed and refractory thrombotic thrombocytopenic purpura: A systematic review.
Abstract Introduction: Caplacizumab is an anti-von Willebrand factor nanobody that has transformed the treatment landscape of thrombotic thro...
Real-world outcomes of primary gastric diffuse large B cell lymphoma in Latin America: A multicenter cohort from the grupo de estudio latinoamericano de linfoproliferativos (GELL)
Abstract INTRODUCTION: Primary gastric diffuse large B-cell lymphoma (PG-DLBCL) is a rare and heterogeneous entity within extranodal non-Hod...
Current practices, organizational structures, technologies and challenges in developing atmps: A survey by dare-NL, nxtgen hightech, T2EVOLVE and ctiwp of EBMT - a gocart-coalition initiative
Abstract Introduction The path from academic development of Advanced Therapy Medicinal Products (ATMPs) to marketing authorization remains c...
Nfkb signaling in idecabtagene vicleucel infusion product drives response in relapsed/refractory multiple myeloma
Abstract Background: B-cell maturation antigen (BCMA) directed chimeric antigen receptor T-cell (CAR-T) therapy has revo...
Venetoclax-based regimens are non-inferior to chemotherapy in core binding factor AML, including AML1-ETO
Abstract Background Core binding factor acute myeloid leukemia (CBF-AML) is a favorable-risk molecular subtype defined by the presence of ei...
Longitudinal quality improvement curriculum for hematology/oncology fellows in an academic training program
Abstract Background: Quality improvement (QI) is a critical component of medicine and an Accreditation Council for Gradu...
The global PNH patient registry: real-world data on PNH symptom burden and quality of life
Abstract Introduction: Current knowledge of the lived experience of individuals with Paroxysmal Nocturnal Hemoglobinuria (PNH) is informed b...
Clinical features and outcomes of adult T-cell leukemia/lymphoma across world regions: An international retrospective cohort study
Abstract Background: Adult T-cell leukemia/lymphoma (ATL) has a dismal prognosis and disproportionately affects regions endemic in Human T-ly...
Updated Results from a Phase 1b study of olverembatinib (HQP1351) combined with lisaftoclax (APG-2575) in pediatric patients with relapsed/refractory Philadelphia Chromosome–Positive acute lymphoblastic leukemia
Abstract Background Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL) in pediatric patients remains a high-risk subtyp...
Next generation platelet storage bags for better and safer transfusions
Abstract Platelet transfusion is a lifesaving medical procedure in contexts ranging from hemorrhagic bleeding to chemotherapy. However, plat...
The efficacy and safety of orelabrutinib-based regimens in the treatment of marginal zone lymphoma: A retrospective real-world study
Abstract Background: Bruton's tyrosine kinase (BTK), a pivotal regulator of B-cell receptor (BCR) signaling, plays a critical role in the su...
Marrow-ly we learn along: A needs assessment of the educational needs of pediatric and young adult patients with bone marrow failure
Abstract Diagnoses of bone marrow failure (BMF), whether acquired or inherited, are rare. The incidence of idiopathic aplastic anemia, the m...
Iptacopan monotherapy demonstrated improved clinical outcomes in a real-world cohort with paroxysmal nocturnal hemoglobinuria: Evidence from a managed access program
Abstract Background: Iptacopan, the first-in-class oral complement factor B inhibitor that selectively inhibits the alternative pathway of th...
Telomere attrition is common in patients with germline RUNX1 pathogenic variants
Abstract Introduction Familial platelet disorder with associated myeloid malignancy (FPDMM) is a rare, autosomal dominant hematologic malign...
Blood microbial DNA as a non-invasive diagnostic surrogate for pneumonia pathogens in immunocompromised patients: A real-world outcomes analysis
Abstract While blood testing is a non-invasive tool for pneumonia diagnosis, its utility depends on specific clinical conditions. Given the...
Characterizing differences in CAR-T cell therapy outcomes based on sex in patients with large B-cell lymphoma
Abstract Background: Chimeric antigen receptor T-cell (CAR-T) therapy has demonstrated improved survival in patients with relapsed, refracto...
The novel allosteric NEK7 inhibitor ofirnoflast (HT-6184) suppresses IL-8 and other pro-inflammatory cytokines in patients with low-risk myelodysplastic syndrome and symptomatic anemia
Abstract Background Chronic inflammation is a key driver of disease progression in myelodysplastic syndromes (MDS), contributing to ineffect...
Psychometric evaluation of the self-management skills checklist across Brazil and the United States: Toward global measurement of transition readiness in sickle cell disease
Abstract Background: The transition from pediatric to adult care is a critical period of vulnerability for adolescents and young adults (AYA...
Outcomes of stem cell transplantation in pediatric and adolescent/young adult patients with trisomy 21: A single-center experience
Abstract Background: For patients with Trisomy 21/Down Syndrome (DS), advances in chemotherapy regimens have increased the 5-year overall su...
A parsimonious, clinically-interpretable logistic model for anemia in PLWHA: Feature selection with MCP penalization and validation in a retrospective cohort
Abstract Background:Anemia remains a common comorbidity in persons living with HIV/AIDS (PLWHA). Among people infected with HIV, anemia is a...
Incidence and characteristics of myeloid neoplasms in patients with multiple myeloma treated with novel myeloma-directed therapies
Abstract BackgroundTherapy-related myeloid neoplasms (t-MNs), including therapy-related myelodysplastic syndromes (t-MDS) and acute myeloid...