Haematologica

Reducing complexity and burden of anticoagulant management in patients with cancer-associated venous thromboembolism

Benjamin Brenner — 2025-12-01

Relapsed acute lymphoblastic leukemia: back to the drawing board

Elad Jacoby — 2025-07-03

Leukemia stem cells in acute myeloid leukemia - mimicking the “space and time continuum”?

Adriana Plesa — 2025-07-17

Recurrence of minimal residual disease in acute myeloid leukemia: a window of opportunity for intervention

Aniket Bankar — 2025-07-31

Olverembatinib in chronic myeloid leukemia: is less actually better?

Maria Agustina Perusini — 2025-06-19

Sugar-free transforming growth factor β1 increases the fitness of myelodysplastic neoplasm/acute myeloid leukemia cells

Eric Solary — 2025-06-26

BTKi + humanized anti-CD20 for advanced follicular lymphoma: ready for an ALTERNATIVE?

Irene Dogliotti — 2025-07-03

A breath of relief: nitrous oxide in sickle cell disease

Thiago Trovati Maciel — 2025-07-03

Lessons learned from experience of phosphatidylinositol 3-kinase inhibitors in chronic lymphocytic leukemia and lymphoma

Paola Ghione — 2025-07-24

The development of phosphoinositide 3-kinase (PI3K) pathway inhibitors for the treatment of lymphoma and chronic lymphocytic leukemia (CLL) has been a journey characterized by significant promise and important challenges.1,2 Starting as a very rational targeted approach for B- and T-cell lymphoproliferative disorders, the development of PI3K inhibitors faced regulatory setbacks due to class-specific adverse events and complex interpretation of the survival outcomes.3 The recently published CHRONOS-4 clinical trial,4 a randomized placebo-controlled study comparing copanlisib in combination with bendamustine plus rituximab and placebo with bendamustine plus rituximab demonstrated that adding copanlisib to the standard-of-care bendamustine plus rituximab did not improve survival and increased toxicity. In this article, we examine the scientific rationale, clinical experience, regulatory hurdles, and future developments of PI3K inhibitors. We analyze the complexity of balancing PI3K therapeutic potential and safety.

Rebalancing agents in hemophilia: knowns, unknowns, and uncertainties

Quentin Van Thillo — 2025-07-31

Treatment options for patients with hemophilia and other bleeding disorders have advanced dramatically over the last few years, not only with the availability of safer factor concentrates, but also with the introduction of factor VIII-mimicking agents. Until recently, there were still areas of hemophilia care that required attention and optimization, including the need for repeated venipuncture, often requiring a central venous access device, and the possible development of inhibitors that limit the efficacy of factor replacement, thereby increasing the complexity and burden of therapy. A new class of rebalancing agents aims to address these remaining issues by inhibiting various natural anticoagulants. Fitusiran is a small interfering RNA agent that reduces antithrombin synthesis in hepatocytes, favoring a procoagulant state. Other promising rebalancing agents are concizumab and marstacimab, which selectively bind to the K2 domain of the tissue factor pathway inhibitor, thus restoring thrombin generation. SerpinPC is a subcutaneous biological inhibitor that blocks the anticoagulant activated protein C pathway, while VGA039 is a monoclonal antibody that targets its cofactor protein S. Although the available clinical data are promising, several important challenges remain. These include the thrombotic risk of rebalancing agents, perioperative and bleeding management, availability in low-income countries, efficacy and factor VIII equivalence compared to existing treatments, ideal target populations, and potential application in other hemostatic disorders. The primary aim of this review is to summarize the best available evidence on these novel rebalancing agents, while highlighting the unknowns, and emphasizing the uncertainties that lie ahead.

Recognition, prevention, and management of adverse events associated with asparaginase/ pegaspargase treatment of acute lymphoblastic leukemia in adults: consensus of an expert panel

Emily Curran — 2025-03-20

Asparaginase (ASNase)-based chemotherapy regimens significantly improve survival outcomes in children, adolescents and young adults (AYA), and even adults with acute lymphoblastic leukemia/lymphoma (ALL); however, the incidence and severity of ASNase-associated adverse events (AE) in adults may differ significantly from those reported in children. Strategies to mitigate, monitor for, and manage toxicities that allow adult ALL patients to receive full ASNase courses are needed. A representative 12-member panel of experts who treat AYA and adult ALL patients, incorporate ASNase into their treatment regimens, and conduct related research was assembled to consider opportunities to optimize the use of pediatric-inspired ALL regimens in these adult patients. Following 2 systematic biomedical literature searches from April 2009 through April 2024, a modified Delphi method was used to distill expert opinion into clinical statements that met a standardized definition of consensus. After 2 iterative Delphi method surveys, 23 statements met the standardized definition of consensus, whereas 19 statements did not. Five statements were merged to avoid redundancy. The clinical statements were grouped into 5 distinct categories: 1) hepatotoxicity; 2) hypersensitivity reactions; 3) thromboembolic and coagulopathy complications; 4) pancreatitis and metabolic complications; and 5) dosing. The intent of these statements is to provide health care providers with information that will help them mitigate, monitor for, and manage the most common and/or unique ASNase-induced AE in adult ALL patients, allowing these patients to receive more or all the planned ASNase doses and thereby improve outcomes.

Severe toxicity and poor efficacy of reinduction chemotherapy are associated with overall poor outcomes in relapsed B-cell acute lymphoblastic leukemia: a report from the Children’s Oncology Group AALL1331 trial

Laura E. Hogan — 2025-06-26

Children’s Oncology Group AALL1331 utilized an intensive chemotherapy induction (Block 1) based on UK ALLR3 induction for children, adolescents, and young adults with acute lymphoblastic leukemia in first relapse, followed by risk-stratified therapy. High/intermediate-risk patients were subsequently randomized to receive two blocks of chemotherapy or two blocks of blinatumomab followed by a hematopoietic stem cell transplant. Low-risk patients were randomized to chemotherapy or chemotherapy cycles intercalated with three blinatumomab blocks. Patients who had early treatment failure were eligible to receive blinatumomab for up to two salvage cycles. We reviewed Block 1 responses, risk stratification, randomization rates, adverse events, event-free survival and overall survival for all enrolled patients. AALL1331 enrolled 661 patients: 24 died during Block 1 and 42 experienced early treatment failure. Overall, 531/661 (80.3%) attained complete remission with 586 risk-assigned and only 471 were randomized. Of 532 patients with bone marrow involvement, 290 (54.5%) were positive for minimal residual disease (≥0.01%) after Block 1. Grade 3, 4 or 5 adverse events occurred in Block 1 in 44.9%, 24.1%, and 3.6% of patients, respectively, with febrile neutropenia, infections, and sepsis being most frequent. Notably, 190 enrolled patients (28.7%) did not proceed with post-induction therapy, including 115 (17.4%) risk-stratified but not randomized. These patients had dismal survival. More effective and less toxic reinduction strategies are needed for B-cell acute lymphoblastic leukemia in first relapse. Trial registration number: NCT02101853.

Different features of acute myeloid leukemia stem cell quantification in intensively treated patients

Lok Lam Ngai — 2025-04-30

In acute myeloid leukemia, the burden of CD34+CD38– leukemia stem cells (LSC) has prognostic value at diagnosis and after induction chemotherapy. Since different methods of LSC quantification have been proposed, we determined the prognostic value on overall survival and incidence of relapse of these methods across European LeukemiaNet (ELN) 2017 risk groups, using data from the HOVON-SAKK132 trial. In addition, we evaluated the optimal number of acquired white blood cells for accurate LSC detection and the prognostic value of individual LSC markers. Results show that it is essential to acquire at least 1x106 white blood cells in order to assess LSC-negativity accurately. Among various LSC markers, CD44 overexpression on CD34+CD38– cells was the only marker that was not statistically significant in our panel. Testing the impact of several published variations of LSC analysis on prognostic value for overall survival and cumulative incidence of relapse showed only marginal differences, demonstrating the robust prognostic value of LSC burden. For further clinical implementation, the optimal LSC assessment may differ among ELN risk groups. In conclusion, LSC burden is a robust prognostic factor and insight into the different methods of LSC definition can facilitate the clinical implementation. Trial registration EudraCT Number: 2013-002843-26.

Measurable residual disease recurrence as early warning of relapse in acute myeloid leukemia

Benfa Gong — 2025-06-12

The aim of this study was to investigate the clinical features and outcomes of measurable residual disease recurrence (MRD-R) by multiparameter flow cytometry in acute myeloid leukemia. We retrospectively analyzed clinical characteristics, residual disease status and outcomes of 767 patients with newly diagnosed acute myeloid leukemia who achieved complete remission within two cycles of induction at our center. Overall, 171 (22.3%) patients experienced MRD-R during follow-up. Patients with MRD-R had inferior outcomes compared to those without MRD-R, with 3-year cumulative incidence of morphological relapse (CIR), relapse-free survival (RFS) and overall survival (OS) being 63.6% vs. 30.6% (P<0.001), 13.9% vs. 67.2% (P<0.001) and 39.0% vs. 79.2% (P<0.001), respectively. The outcomes for patients in the groups with minute MRD-R (<0.1%) or overt MRD-R (≥0.1%) were comparable, with 3-year CIR, RFS and OS of 65.2% vs. 68.2% (P=0.76), 28.9% vs. 27.5% (P=0.85), and 44.8% vs. 38.9% (P=0.39), respectively. Early intervention at the time of MRD-R postponed morphological relapse, extending the median interval from MRD-R to morphological relapse to 4.2 months vs. 1.7 months without intervention (P=0.033). Considering these findings together, MRD-R indicated a higher incidence of relapse and poorer outcomes and could serve as an early warning event for relapse in clinical practice. Early intervention could delay relapse, thereby creating a time window for transplantation.

Randomized trial of anti-thymocyte globulin plus lowdose post-transplant cyclophosphamide to prevent graft-versus- host disease in haploidentical transplantation

Zheng-Li Xu — 2025-06-19

The combination of anti-thymocyte globulin (ATG) and posttransplant cyclophosphamide (PTCy) appears to be a potentially effective graft-versus-host disease (GVHD) prevention strategy for haploidentical transplantation. However, the majority of the evidence originated from retrospective studies without uniform protocols. Our previous findings indicated that 10 mg/kg ATG plus low-dose PTCy could decrease GVHD among high-risk populations transplanted from maternal or collateral relatives. We designed an open-label, phase III, randomized controlled trial to compare patients receiving granulocyte colony-stimulating factor (G-CSF)/ATG-based haploidentical transplantation with or without low-dose PTCy (14.5 mg/kg on days 3 and 4) in non-maternal, non-collateral haploidentical transplants from fathers, children or siblings. A total of 66 patients were randomly assigned to ATG-PTCy (N=44) or ATG (N=22) when the first interim analysis was performed. The interim analysis revealed that the 100-day cumulative incidences of grade 2-4 (18.2%, 95% confidence interval [CI]: 6.6-29.7 vs. 18.2%, 95% CI: 1.7-34.7; P=0.996) and 3-4 acute GVHD (2.3%, 95% CI: 0-6.7 vs. 0; P=0.480) were comparable between the ATG-PTCy and ATG cohorts, as was chronic GVHD at 1 year. The estimated 1-year disease-free survival (DFS) rates were also similar between ATG-PTCy and ATG cohorts (95.5%, 95% CI: 89.5–100 vs. 95.2%, 95% CI: 86.6–100; P=0.979). These results suggested that ATG/PTCy (low-dose) had no advantage over 10 mg/kg ATG-based prophylaxis in patients with haploidentical transplantation other than that of maternal donors or collateral relatives. Future work needs to focus on identifying which populations might benefit from the combined strategy in the context of G-CSF/ATG-based protocols (clinicaltrials gov. Identifier: NCT 06108739).

Novel PI3kδ inhibitor roginolisib synergizes with venetoclax in hematologic malignancies

Binu Kandathilparambil Sasi — 2025-06-19

The phosphoinositide 3-kinase (PI3K) pathway remains a potent drug target in hematological malignancies despite the challenges that have affected clinical drug development, particularly unpredictable toxicity, and inherent/acquired drug resistance. Herein, we tested the activity of a novel PI3Kδ selective, non-ATP competitive inhibitor, roginolisib (IOA-244), in hematological malignancies including diffuse large B-cell lymphoma (DLBCL) and chronic lymphocytic leukemia (CLL). To identify rational actionable combination partners that can be tested in hematologic malignancies, an unbiased pharmacological screening of 474 compounds was carried out in two lymphoma cell lines. We identified BCL2 blockade with venetoclax as synergistically active with roginolisib, a finding confirmed in a broad panel of lymphoma cell lines, DLBCL cell lines and primary CLL samples. We further demonstrate that the sensitizing effects of roginolisib to venetoclax correlate with suppression of downstream PI3K/AKT pathways and alterations in the expression of the apoptotic proteins BIM, mediated through FOXO1 transactivation, and MCL1, with ubiquitination and degradation mediated through GSK3α/β activation. These findings support proof of concept for roginolisib development in hematological malignancies as a single agent or in combination with venetoclax. A clinical trial of roginolisib with venetoclax and an anti-CD20 antibody is initiating in CLL.

Optimizing olverembatinib dose in chronic phase chronic myeloid leukemia

Xiaoshuai Zhang — 2025-04-30

Optimizing olverembatinib dose in people with chronic phase chronic myeloid leukemia is important to increase safety without compromising efficacy. We designed a multicenter, retrospective study comparing safety and efficacy of olverembatinib between the recommended dose of 40 mg every other day (QOD; N=216) and a reduced dose of 30 mg QOD (N=66) in subjects failing to have benefited from other tyrosine kinase inhibitors. The cohorts were similar with regard to baseline co-variates and adjusted for by propensity score matching. There were no significant differences in cytogenetic and molecular responses, or in outcomes between the two dose cohorts. However, the proportion of subjects receiving the original olverembatinib dose at the last follow-up was significantly higher in the 30 mg cohort: 64% (95% confidence interval [95% CI]: 53-75%) versus 44% (95% CI: 37-51%; P=0.004). Furthermore, the proportion of subjects receiving a reduced dose or permanently discontinuing because of adverse events was significantly lower in the 30 mg cohort: 21% (95% CI: 9-33%) versus 41% (95% CI: 34-48%; P=0.003). In summary, a starting dose of olverembatinib 30 mg QOD is as effective as a 40 mg starting dose but better tolerated in people with chronic phase chronic myeloid leukemia in whom other tyrosine kinase inhibitors have failed.

Elevated 5-HTR7 deteriorates dysregulated megakaryocytopoiesis in immune thrombocytopenic purpura via upregulating the PKA/Orai1/ERK1/2 pathway

Meng Zhou — 2025-06-19

Dysregulated megakaryocytopoiesis contributes to reduced platelet counts in immune thrombocytopenic purpura (ITP), yet the mechanism remains elusive. Although 5-hydroxytryptamine receptor 7 (5-HTR7) has been implicated in megakaryocyte biology, its pathogenic involvement in ITP is undefined. This study investigated the impact of 5-HTR7 on megakaryocyte maturation in ITP using flow cytometry, immunofluorescence, and single-cell RNA sequencing. Analyses revealed elevated 5-HTR7 expression on megakaryocytes from ITP patients compared to healthy controls. Pharmacological inhibition of 5-HTR7 using SB269970A not only rescued megakaryocyte maturation defects in vitro but also restored circulating platelet levels in a mouse model of active ITP. Single-cell RNA sequencing coupled with western blot validation identified ERK1/2 phosphorylation in SB269970A-treated megakaryocytes. Mechanistically, 5-HTR7 impaired megakaryocyte maturation through the PKA/Orai1/ERK axis by suppressing store-operated calcium entry, as confirmed via confocal microscopy. In conclusion, elevated expression of 5-HTR7 impairs maturation of megakaryocytes, causing lower platelet counts in ITP and offering a potential therapeutic target for ITP management.

Bisecting GlcNAc expression by bone marrow stromal cells modulates TGF-β1-driven macrophage polarization in myeloid leukemias

Jingjing Feng — 2025-05-22

There has been growing evidence highlighting the critical role of tumor-associated macrophages (TAM) in promoting immune evasion and disease progression in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Combined single- cell RNA sequencing, flow cytometry, and immunohistochemistry studies of the innate immune compartment in bone marrow of MDS/AML reveal a shift toward a tumor-supportive M2-polarized macrophage as well as the expression of programmed cell death-ligand 1 (PD-L1) in this cell lineage. We found leukemic stroma cells with a high level of TGFβ1 secretion can determine TAM toward M2-polarized subtype. Further mechanistic investigations revealed that bone marrow (BM) stromal cells with specific glycans, reduced bisecting N-acetylglucosamine (GlcNAc) levels, in MDS/AML promoted M2-polarized subtype through the secretion of TGFβ1, which elevated PD-L1 expression and thereby impaired CD8+ T-cell function. Our study provides insights into the mechanisms of selectively modifying specific glycans in BM stroma cells and how these may contribute to targeting strategies aimed at the tumor microenvironment.

Chemotherapy-free combination of ibrutinib and obinutuzumab for untreated advanced follicular lymphoma: results of a phase II study from the German Lymphoma Alliance

Christian Schmidt — 2025-06-12

Immunochemotherapy induces long-term responses in patients with follicular lymphoma. However, the toxicity of chemotherapy remains a relevant challenge. The Bruton tyrosine kinase inhibitor ibrutinib has shown significant activity in patients with indolent B-cell lymphoma. Combining ibrutinib with obinutuzumab may, therefore, be an attractive chemotherapy-free option. We conducted a prospective, single-arm, multicenter phase II trial to evaluate the chemotherapy-free regimen of obinutuzumab plus ibrutinib in patients with previously untreated advanced-stage follicular lymphoma. Patients received six 21-day cycles of ibrutinib and obinutuzumab for induction and 12 additional 2-month cycles for maintenance. The primary endpoint was 1-year progression-free survival (PFS). The study was powered to detect an improvement of 10% over the 1-year PFS of 85%. Ninety-eight patients were enrolled in the trial. The median follow-up was 5.5 years. After induction, five patients (5%) had a complete response and 82 (85%) had a partial response. The 1-year PFS was 80%, missing the prospected improvement of a 1-year PFS of 85% (P=0.93). The median PFS was 4.5 years; median duration of response and overall survival were not reached. The most common adverse events of grade 3/4 were neutropenia, lung infection, hypertension, fatigue, rash and thrombocytopenia. The trial of a chemotherapy-free regimen of obinutuzumab and ibrutinib in follicular lymphoma patients failed to demonstrate a 10% improvement in the primary efficacy endpoint. However, the combination produced durable and deep responses and had an acceptable safety profile. Trial registration, EudraCT-Number: 2014-005164-15.

Revised renal stratification and progression models for predicting long-term renal outcomes in immunoglobulin light chain amyloidosis

Muhammad Umaid Rauf — 2025-06-26

Renal prognosis in light-chain amyloidosis (AL) is determined by categorizing patients into three renal stages at diagnosis and assessing renal response or renal progression following chemotherapy after 6 months. We evaluated, in a test (N=1,935) cohort of patients with renal AL amyloidosis who were followed for a median of 95 months, a modified 4-stage model where Renal Stage 2 was sub-categorized according to preserved (2A) or reduced (2B) estimated glomerular filtration rate (eGFR). A hybrid model for evaluation of renal progression was also introduced, using an eGFR cut-off of 30 mL/min/1.73 m2. These models were compared with existing models; namely those of Palladini and Kastritis, and results were validated in a multicenter cohort (N=438). The risk of progression to renal replacement therapy (RRT) increased progressively across all Renal Stages of the Revised staging model (hazard ratio [HR] =3.25, HR=5.13, HR=10.66 for stages 2A, 2B and 3 respectively vs. stage 1; each P<0.001). Our revised criteria for renal response (HR=0.26, 95% confidence interval [CI]: 0.18-0.38 at 60 months) and renal progression (HR=8.15, 95% CI: 6.1-10.9) were independently predictive of RRT and outperfomed existing criteria at all follow-up time points. Renal progression was independently associated with mortality (HR=1.5, 95% CI: 1.26-1.86; P<0.001). The enhanced performance of these refined renal staging and response models enables timely and appropriate chemotherapy adjustment in patients with renal AL amyloidosis.

Characterization, outcome and identification of prognostic factors for patients with systemic immunoglobulin lightchain amyloidosis requiring dialysis prior to initial anticlonal therapy

Lilli S. Sester — 2025-06-19

AL amyloidosis is a serious disease characterized by the deposition of immunoglobulin light chains in multiple organs. Renal involvement occurs in up to 70% of patients, but only a minority require dialysis before initiating anti-clonal treatment. Understanding the occurrence of end-stage organ damage is crucial to pave the way for reversing deposition. Currently, there is no detailed analysis available for this rare patient subgroup. We conducted a systematic search across three amyloidosis centers and characterized 68 biopsy-proven AL amyloidosis patients who required dialysis prior to initial anti-clonal therapy. In our cohort, the second most affected organ was the heart. Renal parameters exhibited variability. Residual urine output and proteinuria ranged widely, while anuria had developed in only a few patients. Among the treated patients, 84% received bortezomib as first-line therapy. The median overall survival (OS) was 44.8 months, with a median event-free survival (EFS) of 16.8 months. Our univariate statistical analysis revealed that underlying clonal disease, indicated by plasma cell infiltration, but not the difference between involved (amyloidogenic) and uninvolved free light chain, impacted OS. Importantly, higher levels of Troponins were associated with worse OS, confirmed by multivariate analysis, whereas N-terminal pro-natriuretic peptide type B levels and classical echocardiographic parameters, such as septal thickness and longitudinal strain, did not demonstrate significant prognostic value. This study provides crucial insights into this unique cohort of dialysis-dependent AL amyloidosis patients. The underlying clonal disease and markers of cardiac damage are important prognostic criteria. These findings emphasize the need to refine prognostic scoring systems for dialysis-dependent AL amyloidosis patients to better stratify risk and optimize treatment approaches.

Comprehensive evaluation of disease characteristics and outcomes of patients with extramedullary multiple myeloma in the modern era

Megan Broughton — 2025-06-12

Multiple myeloma (MM) derives from the clonal proliferation of plasma cells, primarily residing in the bone marrow. However, MM cells can disseminate systemically, leading to osseous or soft tissue extramedullary disease (EMM) or plasma cell leukemia (PCL). The presence of EMM or PCL has historically been linked to poor prognosis and aggressive features. In this study, we analyzed 201 patients with EMM treated at our institution between January 1, 2010, and November 30, 2023. Among these patients, 25 had primary PCL, 19 had secondary PCL, 89 were diagnosed with EMM at the time of MM diagnosis, 29 developed EMM after therapy, and 39 had solitary plasmacytoma (SP), with 20 progressing into MM. Patients with EMM at the time of MM diagnosis or SP progressing to MM exhibited a median overall survival (OS) comparable to those with MM alone (7.5 years or not reached). However, the presence of EMM was associated with worse prognosis in specific groups: primary PCL (median OS: 26 months), secondary PCL (median OS: 1.6 months), and secondary EMM (median OS: 16 months). Additional prognostic features included high Revised International Staging System, chromosomal abnormalities (1q+, 17p deletion, and 13q deletion), and elevated lactate dehydrogenase values at presentation. While the site of EMM did not correlate with inferior outcomes, osseous SP increased the risk of progression to overt MM. In conclusion, the presence of EMM confers variable prognosis, emphasizing the need for more effective therapeutic strategies, particularly for patients with PCL or those developing EMM later during treatment.

Extramedullary disease is associated with severe toxicities following B-cell maturation antigen CAR T-cell therapy in multiple myeloma

Andrew J. Portuguese — 2025-06-19

Extramedullary disease (EMD) in multiple myeloma (MM) is associated with poor outcomes following B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T therapy, yet its impact on treatment-related toxicity remains unclear. This study evaluates the impact of active EMD on toxicity, efficacy, and survival in patients with MM treated with idecabtagene vicleucel (ide-cel) or ciltacabtagene autoleucel (cilta-cel). We conducted a retrospective cohort study of all patients with MM who received ide-cel (N=32) or cilta-cel (N=76) as standard-of-care therapy at our institution from August 2021 to October 2024. EMD was defined as the presence of soft tissue masses in extraosseous locations, and outcomes were compared based on EMD status. Among 108 patients, 26 (24%) had EMD. Patients with EMD experienced higher rates of grade (G)1+ (38% vs. 17%; P=0.022) and G3+ immune effector cell-associated neurotoxicity syndrome (19% vs. 1.2%; P=0.003), as well as G1+ (96% vs. 78%; P=0.041) and G3+ early immune effector cell-associated hematoxicity (31% vs. 0; P<0.001). Patients with EMD had more prolonged severe neutropenia (median: 7 vs. 2 days; P<0.001), greater cefepime use (median 10 vs. 6 doses; P=0.039), and higher rates of bacteremia (15% vs. 2.4%; P=0.029). In terms of efficacy, patients with EMD had lower complete response rates (20% vs. 59%; P<0.001), shorter median progression-free survival (7.6 vs. 24.6 months; P<0.001), and shorter median overall survival (20 months vs. not reached; P<0.001; 1-year estimates, 53% vs. 96%) and higher 1-year non-relapse mortality (21% vs. 2.5%; P=0.003). EMD is associated with increased toxicity, delayed hematologic recovery, more infectious complications, and reduced survival in patients with MM receiving CAR T therapy.

B-cell lymphoblastic leukemia/lymphoma with mutated IKZF1 N159Y: clinical and genetic features of an emerging entity

Emily N. Alvey — 2025-02-27

Atypical acute promyelocytic leukemia with tripartite fusion gene PML::RARG::LINE-L2a is resistant to ATRA but sensitive to arsenic-based therapy

Sanyun Wu — 2025-06-12

TET2 mutation does not impact the prognosis of adult acute myeloid leukemia patients receiving a hematopoietic stem cell transplantation in first remission: similar outcome following matched sibling and unrelated versus haploidentical donor transplants in a multi-center retrospective analysis from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Lin Li — 2025-06-19

Use of second-line and beyond maintenance therapies in adult patients with primary immune thrombocytopenia in Europe: a parallel study of six prospective multicenter national registries

Guillaume Moulis — 2025-06-26

Blocking platelet glycoprotein V cleavage reduces bleeding in mouse models of MYH9-related disease

Patricia Öftering — 2025-06-12

Radiopsy: a prospective observational study on quantitative multiparametric whole-body magnetic resonance imaging to discriminate between smoldering and multiple myeloma

Giacomo Feliciani — 2025-06-19

A retrospective study of isatuximab-pomalidomidedexamethasone in relapsed/refractory systemic AL amyloidosis

Sargam Rachit Vohra — 2025-07-03

Oral administration of a liquid containing nitrous oxide abolishes vaso-occlusive pain in mice with sickle cell disease

Ana Oddo — 2025-06-05

Unveiling platelet aging with progressive β-galactose exposure as a signature of platelet senescence in humans and mice

Catherine Angénieux — 2025-07-03

PTPN11 mutations define a rare but highly adverse subset of myelodysplastic syndromes

Alexandre Bazinet — 2025-07-03

Lower-intensity chemo-immunotherapy with cladribine, low-dose cytarabine, venetoclax and blinatumomab produces high response rates in patients with BCR::ABL1-negative B-cell/myeloid mixed phenotype acute leukemia

Roberta S. Azevedo — 2025-06-26

Optical genome mapping reveals complex cytogenetic abnormalities in multiple myeloma

Jorge A. Palacios — 2025-06-19

CSF3R mutations and variants in myeloid neoplasms: associated phenotypes, co-mutations, and survival trends

Abiola Bolarinwa — 2025-07-10

Minimal residual disease status predicts outcomes in patients with follicular lymphoma treated with chemoimmunotherapy on the SWOG S0016 trial

Alexey V. Danilov — 2025-07-03

Fecal microbiota transplantation for decolonization from multidrug-resistant bacteria in pediatric allogeneic hematopoietic stem cell transplantation recipients: a retrospective real-world data study

Riccardo Masetti — 2025-06-26

Treatment approaches and clinical outcomes in primary colorectal MALT lymphoma: a single-institution retrospective study of 66 patients

Suheil Albert Atallah-Yunes — 2025-06-19

Don’t lose sight: rare extramedullary involvement of myeloid/lymphoid neoplasm with ETV6:ABL1 translocation in blast phase

Neta Sternbach — 2025-07-24

Allogeneic hematopoietic cell transplantation rescues congenital red cell aplasia in H syndrome due to SLC29A3 mutations

Troy C. Quigg — 2025-08-28

Hepatitis B virus reactivation following T-cell engager therapy in multiple myeloma despite negative hepatitis B core antibody serology: implications for screening in patients with hematological malignancies

Matthew J. Rees — 2025-07-31

Treatment-relevant misdiagnosis of autoimmune myelofibrosis

Naseema Gangat — 2025-08-28

Lowering the platelet count threshold to 40x10⁹/L for lumbar puncture: can this represent a generalized approach? Comment on: “40 is the new 50: reducing the need for platelet transfusions prior to lumbar puncture in adults with hematologic malignancies”

Ahmet Emre Eşkazan — 2025-04-03

Comment on “Acute pro-B-cell lymphoblastic leukemia evolving from myelodysplastic neoplasm post cytotoxic therapy: a case report”

Arndt Borkhardt — 2025-05-15

Erratum to: “Chemotherapy-free combination of ibrutinib and obinutuzumab for untreated advanced follicular lymphoma: results of a phase II study from the German Lymphoma Alliance”

Christian Schmidt — 2025-12-01

Erratum to: “Consistent clinical factor VIII equivalency is unlikely for non-factor therapies in hemophilic mice”

Thibaud Sefiane — 2025-12-01