Association of PET4 Response With Outcomes of BV-CHP vs CHOP in the ECHELON-2 Trial in CD30+ Peripheral T-cell Lymphoma.

In the phase 3 ECHELON-2 trial, brentuximab vedotin, cyclophosphamide, doxorubicin, and prednisone (BV-CHP) significantly improved progression-free survival (PFS) and overall survival (OS) compared with cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) in patients with CD30+ peripheral T-cell lymphoma (PTCL), benefits that were maintained at 5 years. Interim positron emission tomography (PET) scan can be used to assess prognosis and risk stratify patients. The prognostic value of interim PET was assessed in this post hoc exploratory analysis from ECHELON-2 evaluating interim 18F-FDG PET scans after cycle 4 (PET4) and end-of-treatment-based response and correlated with PFS per investigator and OS.

Impact of CD34 cell dose on outcomes of haploidentical peripheral blood stem cell transplantation in acute leukemia.

Purpose: Allogeneic hematopoietic stem cell transplantation remains a curative option for acute leukemia. While an adequate CD34 cell dose is essential for engraftment, the optimal upper threshold in haploidentical peripheral blood stem cell transplantation (haplo-PBSCT) remains unclear.

Methods: We retrospectively analyzed 81 patients with acute leukemia who underwent haplo-PBSCT with reduced-intensity conditioning between 2010 and 2020.

Efficacy and safety of momelotinib in Janus kinase inhibitor-experienced Asian patients with myelofibrosis and anemia.

Introduction: This post hoc analysis investigated the efficacy and safety of momelotinib in the Asian subpopulation of MOMENTUM (NCT04173494).

Methods: Patients were randomized 2:1 to momelotinib 200 mg once daily (QD) plus danazol placebo (momelotinib group) or danazol 600 mg QD plus momelotinib placebo (danazol group) for 24 weeks (W), after which they could receive open-label momelotinib or danazol.

Primary Endpoint: W24 total symptom score (TSS) response rate (≥ 50% reduction from baseline).

Romiplostim with ciclosporin A in patients with aplastic anaemia naïve to immunosuppressive therapy: A phase 2/3 study.

Romiplostim has been shown to restore multi-lineage haematopoiesis and is effective in patients with aplastic anaemia (AA) refractory to immunosuppressive therapy (IST). This open-label, phase 2/3 study (NCT04095936) recruited adult AA patients in Japan and Korea who had not received prior IST and evaluated the efficacy and safety of romiplostim plus ciclosporin A (CsA). Romiplostim was initiated at 10 μg/kg once weekly through Week 4 and adjusted between 0 and 20 μg/kg from Week 5 onwards.

Serial next-generation sequencing for detecting germline predisposition in acute myeloid leukemia.

Not available.

Efficacy and Safety of Ropeginterferon Alfa-2b in Low-Risk Polycythemia Vera.

Purpose: Conventional management of low-risk polycythemia vera (PV), consisting of phlebotomy and aspirin, often fails to adequately control symptoms and hematologic parameters. This study evaluated the efficacy and safety of ropeginterferon alfa-2b (Ropeg) in low-risk PV patients requiring cytoreductive therapy.

Materials And Methods: In this sub-analysis of an open-label, multicenter trial, 42 patients received Ropeg for 48 weeks.

In vivo tracking of transfused platelets by next-generation sequencing of mitochondrial DNA.

Tracking transfused platelets is important to evaluate platelet transfusion efficiency. Traditionally, corrected count increments were used; however, quantitative polymerase chain reaction-based methods have recently been developed. As both these methods have some limitations, we developed a new method based on next-generation sequencing (NGS) of platelet mitochondrial DNA (mtDNA).

Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric and Young Adult Patients with Chronic Myeloid Leukemia in Tyrosine Kinase Inhibitor Era: A Study of the Korean Blood and Marrow Transplantation Registry.

Purpose: Chronic myeloid leukemia (CML) in children, adolescents, and young adults is rare and differs from older adults. This study evaluated the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) in young Korean CML patients during the tyrosine kinase inhibitor (TKI) era.

Materials And Methods: A retrospective analysis of 35 CML patients aged <40 years who underwent allogeneic HSCT from 2009 to 2019 was conducted using Korean Blood and Marrow Transplantation Registry data.

NKG2D-mediated cytotoxicity of CD4 cytotoxic T cells in multiple myeloma.

Emerging evidence indicates that CD4+ T cells contribute to antitumor immunity beyond their traditional roles as helpers or regulators. However, the specific subset of CD4+ T cells mediating beneficial outcomes in patients with multiple myeloma remains unclear. Here, we performed single-cell RNA sequencing and T-cell receptor sequencing on CD4+ T cells sorted from the bone marrow of patients across the stages of myeloma progression.

Hematologic and molecular responses to ropeginterferon alfa-2b therapy of polycythemia vera: 48-week results from a prospective study.

To prevent thrombosis in patients with polycythemia vera (PV), achieving a complete hematologic response (CHR) is highly recommended in practice. In addition, a reduced JAK2 V617F mutation burden is expected to have a disease-modifying effect, and its molecular response (MR) is currently of significant interest. This study aimed to assess the association between CHR and MR in patients with PV following treatment with ropeginterferon alfa-2b.

Prognostic factors and treatment outcomes of allogeneic stem cell transplantation in lymphoid malignancy.

Allogeneic stem cell transplantation (allo-SCT) is a salvage treatment option for patients with relapsed or refractory lymphoid malignancies. However, the clinical variables impacting outcomes in these patients remain unclear. We analyzed 58 patients who underwent allo-SCT for lymphoid malignancies, including B-cell lymphoma (BCL, n = 20), Hodgkin's disease (n = 3), multiple myeloma (n = 9), natural killer/T-cell lymphoma (NK/TCL, n = 4), and TCL (n = 22).

A phase 1/2 study of gilteritinib in combination with chemotherapy in newly diagnosed patients with AML in Asia.

Objective: This interim analysis of a phase 1/2, open-label, single-arm study assessed the safety, efficacy, and pharmacokinetics of gilteritinib plus chemotherapy in adults with newly diagnosed FLT3 mutation-positive acute myeloid leukemia.

Methods: In sequential phase 1 and 2 studies, induction and consolidation therapy with gilteritinib 120 mg/day plus chemotherapy (induction: idarubicin/cytarabine once daily; consolidation: cytarabine twice daily) was followed by maintenance gilteritinib 120 mg/day monotherapy. Endpoints included maximum tolerated dose (MTD), recommended expansion dose (RED), and dose-limiting toxicity (phase 1), and complete remission (CR) rate following induction therapy (primary endpoint), overall survival (OS), safety, and pharmacokinetics (phase 2).

Poor Mobilization-Associated Factors in Autologous Hematopoietic Stem Cell Harvest.

Peripheral blood stem cell transplantation (PBSCT) is an important therapeutic measure for both hematologic and non-hematologic diseases. For PBSCT to be successful, sufficient CD34 cells need to be mobilized and harvested. Although risk factors associated with poor mobilization in patients with hematologic diseases have been reported, studies of patients with non-hematologic diseases and those receiving plerixafor are rare.

Comprehensive insights into AML relapse: genetic mutations, clonal evolution, and clinical outcomes.

Introduction: Acute myeloid leukemia (AML) is a complex hematologic malignancy characterized by uncontrolled proliferation of myeloid precursor cells within bone marrow. Despite advances in understanding of its molecular underpinnings, AML remains a therapeutic challenge due to its high relapse rate and clonal evolution.

Methods: In this retrospective study, we analyzed data from 24 AML patients diagnosed at a single institution between January 2017 and August 2023.

Validation of the 2022 European LeukemiaNet risk stratification for acute myeloid leukemia.

This study aimed to validate the 2022 European LeukemiaNet (ELN) risk stratification for acute myeloid leukemia (AML). A total of 624 newly diagnosed AML patients from 1998 to 2014 were included in the analysis. Genetic profiling was conducted using targeted deep sequencing of 45 genes based on recurrent driver mutations.

Clinical outcomes of etoposide and cytarabine as consolidation in elderly patients with primary CNS lymphoma.

Background: A consolidation strategy has not been established for transplant-ineligible elderly patients with primary central nervous system lymphoma (PCNSL). In this study, we aimed to retrospectively evaluate the clinical outcomes of etoposide and cytarabine (EA) as consolidation chemotherapy for transplant-ineligible patients with PCNSL following high-dose methotrexate (MTX)-based induction chemotherapy.

Materials And Methods: Between 2015 and 2021, newly diagnosed transplant-ineligible patients with PCNSL with diffuse large B-cell lymphoma were consecutively enrolled.

Unraveling trajectories from aplastic anemia to hematologic malignancies: genetic and molecular insights.

Background: Aplastic anemia (AA), characterized by hematopoietic stem cell deficiency, can evolve into different hematologic malignancies. Our understanding of the genetic basis and mechanisms of this progression remains limited.

Methods: We retrospectively studied 9 acquired AA patients who later developed hematologic malignancies.

Clearing soluble MIC reverses the impaired function of natural killer cells from patients with multiple myeloma.

Background: Major histocompatibility complex (MHC) class I chain-related protein (MIC) is a stress-induced ligand released from multiple myeloma (MM) cells during progression, and soluble MIC impairs natural killer group 2D (NKG2D) activating receptor-mediated recognition and function of natural killer (NK) cells. However, whether clearing soluble MIC with a monoclonal antibody (mAb) can restore NK cell activity of MM patients remains undetermined.

Methods: We analyzed The Cancer Genome Atlas (TCGA) Multiple Myeloma Research Foundation (MMRF) CoMMpass data set to examine the prognostic significance of expression in MM.

Utility of Plasma Microbial Cell-Free DNA Whole-Genome Sequencing for Diagnosis of Invasive Aspergillosis in Patients With Hematologic Malignancy or COVID-19.

Background: We evaluated the clinical accuracy and utility of whole-genome sequencing (WGS) of plasma microbial cell-free DNA (cfDNA) as a novel noninvasive method in diagnosing invasive aspergillosis (IA) in patients with hematologic malignancy (HM) or coronavirus disease 2019 (COVID-19).

Methods: Adults with HM or COVID-19 and suspected IA were recruited. IA cases were retrospectively diagnosed according to EORTC/MSG definitions and ECMM/ISHAM criteria for HM and COVID-19 patients, respectively.

In vitro erythrocyte production using human-induced pluripotent stem cells: determining the best hematopoietic stem cell sources.

Background: Blood transfusion is an essential part of medicine. However, many countries have been facing a national blood crisis. To address this ongoing blood shortage issue, there have been efforts to generate red blood cells (RBCs) in vitro, especially from human-induced pluripotent stem cells (hiPSCs).

Development of a Next-generation Sequencing-based Gene Panel Test to Detect Measurable Residual Disease in Acute Myeloid Leukemia.

Background: AML is a heterogeneous disease, and despite intensive therapy, recurrence is still high in AML patients who achieve the criterion for cytomorphologic remission (residual tumor burden [measurable residual disease, MRD]<5%). This study aimed to develop a targeted next-generation sequencing (NGS) panel to detect MRD in AML patients and validate its performance.

Methods: We designed an error-corrected, targeted MRD-NGS panel without using physical molecular barcodes, including 24 genes.