Acta Haematologica[JOURNAL]
Sun
200 papers
Future Directions in Allogeneic Stem Cell Transplantation in Acute Lymphoblastic Leukemia.
Rafaeli N, Kebriaei P
Acta Haematol · 2026 · PMID 40716436 · Publisher ↗
BACKGROUND: Allogeneic stem cell transplantation (ASCT) has been a cornerstone of acute lymphoblastic leukemia (ALL) treatment for decades, with significant improvements in patient survival. These advancements are linked...
BACKGROUND: Allogeneic stem cell transplantation (ASCT) has been a cornerstone of acute lymphoblastic leukemia (ALL) treatment for decades, with significant improvements in patient survival. These advancements are linked to better therapies, refined conditioning regimens, and minimal residual disease monitoring. Despite progress, challenges remain in reducing relapse, treatment-related mortality, and toxicity. SUMMARY: This article explores recent advancements in ASCT for ALL, focusing on conditioning regimens, graft engineering, and post-transplant therapies. Key developments include identifying patients who benefit from reduced-intensity conditioning over myeloablative conditioning or reduction in dose of total body irradiation (TBI), leading to improved outcomes and lower toxicity. Innovative graft manipulation strategies, such as Orca-T and Orca-Q, aim to enhance graft-versus-leukemia effects while minimizing graft-versus-host disease (GVHD). Additionally, post-transplant therapies, including targeted treatments, immunotherapies, and CAR-T cells, are showing promise in preventing relapse. Advances in mismatched donor use and GVHD prophylaxis are broadening donor options and reducing adverse effects, improving ASCT's safety and effectiveness in ALL. KEY MESSAGES: ASCT remains a critical treatment for high-risk ALL, with advancements in conditioning, graft engineering, and post-transplant strategies. Future focus will be on optimizing patient selection, use in post-CAR-T consolidation, and refining graft manipulation, aiming to personalize ASCT and enhance survival and quality of life for ALL patients.
Beyond CAR-T: The Rise of CAR-NK/T Cells for Haematological Cancer.
Noorafrooz M, Gale RP, Dashtkoohi M
Acta Haematol · 2026 · PMID 40706581 · Publisher ↗
BACKGROUND: Chimeric antigen receptor (CAR)-natural killer (NK)/T-cells offer a new approach in immune therapy of haematological cancers. NK/T-cells bridge innate and adaptive immunity with strong anti-cancer effects. Un...
BACKGROUND: Chimeric antigen receptor (CAR)-natural killer (NK)/T-cells offer a new approach in immune therapy of haematological cancers. NK/T-cells bridge innate and adaptive immunity with strong anti-cancer effects. Unlike allogeneic CAR-T-cells, CAR-NK/T-cells do not require TCR genetic deletion to prevent major MHC recognition, have a lower risk of graft-versus-host disease, and can be used universally. SUMMARY: Pre-clinical studies report CAR-NK/T-cells effectively target antigens such as CD19 and B-cell maturation antigen in B-cell lymphomas and plasma cell myeloma. Compared with CAR-T-cells, CAR-NK/T-cells have faster immune responses, more cytotoxicity and better safety. Recent innovations increase efficacy of CAR-NK/T-cell therapies. Early clinical trials report promising safety and efficacy. Although still in the early phases of development, advances in NK/T-cell therapy are overcoming prior challenges. KEY MESSAGES: CAR-NK/T-cells may prove a safer, more flexible form of cell therapy of haematological cancers.
Toward Clinically Actionable Machine Learning and Artificial Intelligence Algorithms in Acute Leukemia: A Systematic Narrative Review.
Sabile JMG, Zhang P, Parwani AV … +3 more , Chobrutsiky B, Gandhi AP, Srisuwananukorn A
Acta Haematol · 2025 · PMID 40706562 · Publisher ↗
INTRODUCTION: Acute myeloid leukemia (AML) is a heterogenous hematologic malignancy that maintains high relapse rates and poor survival despite ongoing treatment advances. There is critically unmet need for consistently...
INTRODUCTION: Acute myeloid leukemia (AML) is a heterogenous hematologic malignancy that maintains high relapse rates and poor survival despite ongoing treatment advances. There is critically unmet need for consistently providing long-term survival with minimal treatment toxicity for AML patients. Advances in artificial intelligence/machine learning (AI/ML) offer new approaches to addressing clinical challenges in AML. METHODS: In this systematic narrative review, 426 publications focusing on the intersection of AML and AI/ML between January 1, 2010, and July 30, 2024, are reviewed. RESULTS: The evolution of AI/ML tools over time is described from a clinically relevant perspective with a distinction between early epochs of AI/ML versus more contemporary algorithms, such as generative adversarial networks and transformer-based algorithms. This review highlights the utilization of contemporary AI/ML algorithms via addressing diagnostic challenges, molecular risk stratification problems, and clinical outcome prediction in the context of AML. CONCLUSION: Overall, AI/ML represents a promising new frontier in approaching clinical problems in AML, though there are still opportunities for utilization, particularly in the setting of allogeneic stem cell transplantation.
Global Challenges in Paediatric Acute Lymphoblastic Leukaemia.
Krishnan S, Saha V
Acta Haematol · 2026 · PMID 40695248 · Full text
<p>Background: Acute lymphoblastic leukaemia (ALL) is the commonest paediatric cancer and represents a fifth of adult leukaemias. Global outcome disparities are linked to variations in socio-demographic indices (SDIs). S...
<p>Background: Acute lymphoblastic leukaemia (ALL) is the commonest paediatric cancer and represents a fifth of adult leukaemias. Global outcome disparities are linked to variations in socio-demographic indices (SDIs). Summary: In high-SDI regions, established collaborative groups report cure rates surpassing 90% in paediatric ALL. The focus is on reducing treatment toxicity using chemotherapy-free strategies, principally T-cell-directed immunotherapies and targeted small molecules, as exemplified in adult Philadelphia-chromosome-positive ALL. High cure rates limit testing of novel approaches outside niche subgroups, while high costs preclude wider real-world adoption of these advances. Mid-SDI regions (50-80% cure) face challenges in fully implementing contemporary risk-adapted therapy to improve outcomes and reduce costs. This necessitates collaborative practice, standardised high-quality risk-stratification diagnostics, and access to quality-assured generic cytotoxics. Low-SDI regions (<50% cure) report rising disease burden and face more fundamental challenges, including timely diagnosis, access to treatment and expertise, and minimising toxicity and abandonment. Solutions require locally adapted protocols, collaborative partnerships, and sustained patient-support programmes. Key Message: Global partnerships across SDI regions are crucial to address shared challenges in ALL, including access to affordable quality therapeutics, continuing refinement of established treatment elements, tailoring biomarkers for diverse populations, and collaborative frameworks to evaluate new treatments, technologies, and treatment paradigms. </p>.
Practical Management of Cardiovascular Adverse Events with BTKi Treatment in Patients with Chronic Lymphocytic Leukemia: A Consensus Report by Hematologists and Cardiologists.
Munir T, Čiburienė E, Graklanov V … +11 more , Gospodinova M, Jakšić O, Lipar L, Marton I, Panovská A, Petríková L, Pileckyte R, Pozsonyi Z, Slanina M, Škerget M, Bulj N
Acta Haematol · 2026 · PMID 40684756 · Full text
<p>Background: Cardiovascular (CV) adverse events (AEs), especially atrial fibrillation (AF) and hypertension, have been reported in patients receiving treatments for chronic lymphocytic leukemia (CLL), including Bruton'...
<p>Background: Cardiovascular (CV) adverse events (AEs), especially atrial fibrillation (AF) and hypertension, have been reported in patients receiving treatments for chronic lymphocytic leukemia (CLL), including Bruton's tyrosine kinase inhibitors (BTKis). Although these AEs are managed effectively in most cases and AE management guidelines exist, practical management approaches are inconsistent across regions and practices. We aimed to address these inconsistencies by developing consensus recommendations. Summary: A European expert panel was assembled comprising eight hematologists and six cardiologists. Literature analysis, expert interviews, and the Delphi method were used to gain consensus on screening, monitoring, and treatment of AF and hypertension statements. Key Messages: Maintaining BTKi treatment is paramount to maximize time to next treatment; for patients at high risk of progression, this can be achieved by appropriately treating hypertension and AF and adjusting the BTKi dose. Patients should be risk-stratified as low, moderate, high, or very-high risk of cancer therapy-related CV toxicity and treated according to their disease status so that CLL treatment can be maintained. Patient education on symptom monitoring, home blood pressure monitoring, and electrocardiograms (baseline, every 3 months) are recommended to detect/monitor AF and hypertension. Close collaboration between hematologists and cardiologists is vital to achieve optimal patient outcomes. </p>.
Deep Learning Applications in Lymphoma Imaging.
Sorin V, Cohen I, Lekach R … +2 more , Partovi S, Raskin D
Acta Haematol · 2025 · PMID 40659002 · Publisher ↗
BACKGROUND: Lymphomas are a diverse group of disorders characterized by clonal proliferation of lymphocytes. While definitive diagnosis relies on histopathology, immunohistochemical, molecular and genomic analyses, imagi...
BACKGROUND: Lymphomas are a diverse group of disorders characterized by clonal proliferation of lymphocytes. While definitive diagnosis relies on histopathology, immunohistochemical, molecular and genomic analyses, imaging modalities including positron emission tomography/computed tomography (PET/CT), computed tomography (CT), and magnetic resonance imaging (MRI) are essential in diagnostic processes and management. Imaging aids in detecting suitable biopsy sites, assessing disease extent, evaluating treatment response, and detecting recurrence. However, accurate diagnosis and staging remain challenging due to tumor heterogeneity, inter-observer variability, and technical imaging issues. SUMMARY: Artificial intelligence (AI), particularly deep learning (DL) models, is transforming lymphoma imaging by enabling automated detection, segmentation, and classification across PET/CT, CT, and MRI modalities. Key applications include automated metabolic response assessment and total metabolic tumor volume quantification in PET/CT, lymph node segmentation and classification in CT, and improved detection of central nervous system involvement in MRI. Despite promising results, significant challenges limit widespread clinical adoption, including variability in imaging protocols affecting model generalizability, reliance on small retrospective datasets, lack of model interpretability, and difficulties integrating AI tools into existing clinical workflows. KEY MESSAGES: (1) DL applications can automate detection, segmentation, and classification in lymphoma imaging, improving diagnostic accuracy and reducing inter-observer variability across PET/CT, CT, and MRI modalities. (2) Challenges in DL adoption include validating model performance across diverse imaging protocols, addressing data biases, and ensuring generalizability to real-world clinical settings. (3) Integrating AI into clinical workflows requires careful validation to ensure safety, consistency, and alignment with existing diagnostic and treatment standards.
Complexities of Measurable Residual Disease Assays in Acute Lymphoblastic Leukemia: A Guide for the Practicing Clinician.
Luo J, Muffly L
Acta Haematol · 2026 · PMID 40652938 · Full text
<p>Background: Assessment of measurable residual disease (MRD) assessment is an internal component of prognostication and management of acute lymphoblastic leukemia (ALL). A range of assays - differing in sensitivity, co...
<p>Background: Assessment of measurable residual disease (MRD) assessment is an internal component of prognostication and management of acute lymphoblastic leukemia (ALL). A range of assays - differing in sensitivity, complexity, and clinical application - are available. As these technologies advance, clinicians face new challenges in selecting and interpreting MRD tests. Summary: MRD testing is essential for risk stratification and treatment guidance in pediatric and adult ALL. Key assay platforms include multiparameter flow cytometry, quantitative PCR, and next-generation sequencing (NGS) for clonal B- or T-cell receptor rearrangements. NGS MRD offers superior detection depth, especially in post-hematopoietic cell transplantation, and post-CAR T-cell therapy. In Philadelphia chromosome-positive ALL, persistent BCR::ABL1 may represent non-leukemic clones, warranting the use of lineage-specific assays. While bone marrow remains the standard MRD source, assessment of MRD in blood and cerebrospinal fluid are gaining support in select contexts. MRD assessment in T-cell ALL remains complex due to antigen heterogeneity and infrequent clonal targets. Key Messages: This review provides a practical overview of MRD testing in ALL, comparing available technologies and highlighting clinical implications of assay selection, sensitivity, and sample type. </p>.
Impact of Early-Onset or Worsening Anemia in Patients with Myelofibrosis Treated with Ruxolitinib: A post hoc Analysis of the JUMP Study.
Al-Ali HK, Guglielmelli P, Harrison CN … +4 more , Mesa RA, Hamer-Maansson JE, Braunstein E, Gupta V
Acta Haematol · 2026 · PMID 40652928 · Full text
UNLABELLED: <p>Introduction: Anemia can influence decisions regarding initiation, dosing, and discontinuation of Janus kinase inhibitor therapy for myelofibrosis. We evaluated the impact of new-onset or worsening anemia...
UNLABELLED: <p>Introduction: Anemia can influence decisions regarding initiation, dosing, and discontinuation of Janus kinase inhibitor therapy for myelofibrosis. We evaluated the impact of new-onset or worsening anemia following ruxolitinib initiation on spleen response, symptom severity, and overall survival in patients with myelofibrosis. METHODS: This post hoc analysis used data from all patients enrolled in the phase 3b JUMP trial. Outcomes were stratified by presence or absence of new-onset or worsening anemia following ruxolitinib initiation, defined as hemoglobin decrease ≥15 g/L from baseline and hemoglobin <100 g/L (female)/<110 g/L (male) at Week 12, new transfusion requirement post-baseline until Week 12 (for baseline non-transfusion-dependent patients), or ≥50% increase from baseline in red blood cell transfusions through Week 12. RESULTS: Overall, 2,233 patients were included; 52.9% developed new-onset or worsening anemia up to Week 12. Ruxolitinib was associated with improvements in spleen length and myelofibrosis symptoms, regardless of the presence or absence of new-onset or worsening anemia or baseline anemia status. No differences in spleen response or overall survival were observed between patients with versus without new-onset or worsening anemia, regardless of baseline anemia status. CONCLUSIONS: These results support the use of ruxolitinib in patients with myelofibrosis, regardless of baseline anemia or development of treatment-related anemia. </p>.
Venetoclax-Based Regimen in Refractory or Relapsed Pediatric Acute Lymphoblastic Leukemia.
Zhang L, Zhang Z, Lu A … +4 more , Jia Y, Huang L, Zhang L, Zeng H
Acta Haematol · 2026 · PMID 40628245 · Publisher ↗
INTRODUCTION: This study investigated the efficacy and survival of pediatric refractory or relapsed (R/R) acute lymphoblastic leukemia (ALL) treated with a venetoclax (VEN)-based regimen. METHODS: Children with R/R ALL t...
INTRODUCTION: This study investigated the efficacy and survival of pediatric refractory or relapsed (R/R) acute lymphoblastic leukemia (ALL) treated with a venetoclax (VEN)-based regimen. METHODS: Children with R/R ALL treated with a VEN-based regimen at Peking University People's Hospital from December 1, 2018, to January 15, 2024, were included in this study. Complete remission (CR) or complete remission with incomplete recovery of blood count (CRi) rates and objective response rates (ORRs) were analyzed. RESULTS: Twenty-two children with R/R ALL were included in this study. The median duration of VEN treatment per cycle was 21 (7-28) days, and the median VEN dose was 100 (50-300) mg/day. Following a cycle of VEN-based therapy, 17 children (77.3%) achieved CR/CRi/morphological leukemia-free state (MLFS), including 8 cases (8/17) with negative MRD. The ORR in the children with B-cell acute lymphoblastic leukemia (B-ALL) (n = 9) and T-cell acute lymphoblastic leukemia (T-ALL) (n = 8) was 75% and 80%, respectively. Patients with early T-cell precursor (ETP) ALL (n = 6) achieved MRD-negative remission, and one KMT2A::USP2-positive child achieved MLFS after receiving a VEN-based regimen. For the relapsed patients, the median overall survival (OS) was 1,371 days. For the refractory patients, the median OS was unreached. For T-ALL patients, the median OS was 1,371 days. For the patients with B-ALL, the median OS was 543 days. All patients had hematologic adverse reactions within an acceptable range. CONCLUSION: Children with R/R ALL who received the VEN-based regimen achieved a high remission rate with an acceptable safety profile. Significantly, the VEN-based regimen was effective in patients with R/R ETP with MRD-negative results while also proving beneficial for KMT2A-rearranged, highlighting VEN-chemotherapy as a treatment option for remission.
Identification and Confirmation of Myeloid/Lymphoid Neoplasms with Fibroblast Growth Factor Receptor 1 Rearrangement and Characterization of Treatment Patterns and Outcomes in a Real-World Setting: A US Retrospective Chart Review.
Parasuraman S, Saverno K, Zimmerman Savill KM … +1 more , Colucci P
Acta Haematol · 2026 · PMID 40618748 · Full text
UNLABELLED: <p>Introduction: Myeloid/lymphoid neoplasms with fibroblast growth factor receptor 1 rearrangement (MLN-FGFR1) are rare, heterogenous, aggressive hematologic malignancies with FGFR1 rearrangements at the 8p11...
UNLABELLED: <p>Introduction: Myeloid/lymphoid neoplasms with fibroblast growth factor receptor 1 rearrangement (MLN-FGFR1) are rare, heterogenous, aggressive hematologic malignancies with FGFR1 rearrangements at the 8p11 locus. Pemigatinib, a potent selective inhibitor of FGFR1-3, is approved for relapsed/refractory MLN-FGFR1. METHODS: This retrospective chart review included US adults with myeloproliferative neoplasm, unclassifiable (MPN-U), myelodysplastic syndrome (MDS)/MPN, post-MPN acute myelogenous leukemia, precursor T- or B-cell acute lymphoblastic leukemia/lymphoma, or mixed-phenotype acute leukemia with bone marrow biopsy and standard cytogenetic and/or molecular results. Probable cases of MLN-FGFR1 were identified and confirmed with cytogenetic or molecular testing results. Patient characteristics, diagnostic testing methods, treatments, and outcomes were abstracted. RESULTS: Of 560 submitted cases, 51 (9.1%) were probable MLN-FGFR1, 33 (5.9%) of which were subsequently confirmed. Among patients with confirmed MLN-FGFR1, 8p11 translocation or FGFR1 rearrangements were detected with standard cytogenetics in 72.7%, break-apart fluorescence in situ hybridization in 66.7%, next-generation sequencing in 21.2%, and real-time polymerase chain reaction in 6.1%. All but 1 patient initiated treatment; 3 patients underwent allogenic stem-cell transplant. CONCLUSION: This study highlights the importance of cytogenetic and molecular evaluations in patients with chronic/blast phase hematologic malignancies to diagnose MLN-FGFR1. This is particularly important following US approval of pemigatinib for this hematologic malignancy. </p>.
Exploration of B- and T-Cell Receptor Repertoires Reveals Distinct Mechanisms in Pure Red Cell Aplasia, Autoimmune Hemolytic Anemia, and Aplastic Anemia.
Zhang R, Han B
Acta Haematol · 2026 · PMID 40587957 · Full text
UNLABELLED: <p>Introduction: Pure red cell aplasia (PRCA), autoimmune hemolytic anemia (AIHA), and aplastic anemia (AA) are immune-mediated diseases that affect mainly erythrocytes or erythroid progenitor cells. This stu...
UNLABELLED: <p>Introduction: Pure red cell aplasia (PRCA), autoimmune hemolytic anemia (AIHA), and aplastic anemia (AA) are immune-mediated diseases that affect mainly erythrocytes or erythroid progenitor cells. This study aimed to investigate changes related to autoimmunity in B-cell receptor (BCR) and T-cell receptor (TCR) repertoires in patients with these diseases. METHODS: Patients with primary PRCA, AIHA, and AA and normal controls (NCs) were recruited. Peripheral blood was collected, and BCR and TCR repertoires were sequenced by next-generation immunosequencing. RESULTS: Ten patients with PRCA, 10 with AIHA, 10 with AA, and 7 NCs were ultimately enrolled. According to the broad repertoire metric analysis, only the TCR repertoire of the AA group was more diverse than that of the NC group (p < 0.05). Regarding BCR and TCR repertoires, the PRCA, AIHA, and AA groups had uniform gene characteristics. The preferential gene of immunoglobulin heavy chains in PRCA patients was correlated with memory cell and red blood cell antigen recognition; genes expressed in the AIHA group were associated with the secretion of autoantibodies, whereas AA patients had more genes related to neutralizing antibodies. For T-cell receptor β (TRB) chains, PRCA patients had skewed use of genes associated with T-cell dysregulation and hyperinflammation, whereas AIHA and AA patients had similar genes as patients with other autoimmune diseases, which correlated with abnormal antigen recognition. PRCA and AA patients had specific TRBV-J gene combinations. For the BCR light chains, PRCA and AIHA patients tended to use more κ chains, whereas AA patients tended to use more λ chains. Regarding the TCRα chains, patients with each of the three diseases expressed more genes related to hypersensitivity reactions (p < 0.05). Compared to the NC group, the PRCA and AIHA groups had greater BCR somatic hypermutation (SHM). The length of CDR3 was similar, but the hydrophobicity differed among the different disease groups. Different motifs were found in BCRs and TCRs of the three diseases. Compared to NCs, the PRCA, AIHA, and AA groups showed a considerable lack of physiological T-cell clusters, and some disease-specific T-cell clusters were found in each disease group. CONCLUSION: PRCA, AIHA, and AA patients had different BCR and TCR repertoire characteristics in terms of genes and gene combinations, hydrophobicity and CDR3 motifs, and T-cell clustering, which might contribute to autoimmune antigen recognition. The abnormalities were mainly T-cell related for PRCA patients, B-cell related for AIHA patients and both for AA patients. </p>.
Genomic Subclassification and Risk Stratification of ALL.
Østergaard A, Iacobucci I
Acta Haematol · 2026 · PMID 40587953 · Publisher ↗
BACKGROUND: Over the last 2 decades, significant improvements have been made in the understanding of the genomic and biological bases of acute lymphoblastic leukemia (ALL), resulting in enhanced genomic classification, m...
BACKGROUND: Over the last 2 decades, significant improvements have been made in the understanding of the genomic and biological bases of acute lymphoblastic leukemia (ALL), resulting in enhanced genomic classification, more precise risk stratification, and improved long-term outcomes. ALL is a hematologic malignancy defined by uncontrolled proliferation of immature B- or T-lymphoid blasts in the bone marrow, blood, and other extramedullary tissues. It affects most commonly children, representing the most common childhood cancer, but it also occurs in adults where outcome tends to be poorer compared to pediatric patients. SUMMARY: A variety of genetic aberrations, including structural and numerical chromosome alterations, translocations generating fusion oncoproteins, cryptic genomic rearrangements, sequence mutations, and genomic copy number changes, define multiple genomic subtypes, influence risk stratification and determine response to therapeutic strategies. KEY MESSAGES: In this review, we describe the updated genomic classification of ALL highlighting new biological insights and discussing their implications for prognostication and outcome.
Identification of Needs of Patients with Multiple Myeloma: A Questionnaire-Based Study in Israeli Patients.
Levy Yurkovski I, Ben-Dov S, Shoam V … +2 more , Yuz M, Lavi N
Acta Haematol · 2026 · PMID 40555219 · Full text
UNLABELLED: <p>Introduction: Multiple myeloma is an incurable chronic malignant disease. The disease itself and its treatment impair quality-of-life (QoL), yet there is no data regarding the biopsychosocial needs of pati...
UNLABELLED: <p>Introduction: Multiple myeloma is an incurable chronic malignant disease. The disease itself and its treatment impair quality-of-life (QoL), yet there is no data regarding the biopsychosocial needs of patients in the era of new treatments. In the current study, we aimed to identify the biopsychosocial needs of patients with multiple myeloma. METHODS: This is a descriptive study on patients with multiple myeloma in Israel in 2024. The information was based on a questionnaire examining physical, psychological, and social needs filled out by myeloma patients. We analyzed the main impairments of QoL and what affected them, the main supporter in dealing with the disease, psychosocial needs reported by the patients and the difficulties in dealing with such difficulties. RESULTS: The main symptom reported by multiple myeloma patients was fatigue. The number of treatment lines worsened QoL (unstandardized coefficient: 0.987, 95% CI: 0.284; 1.691, p = 0.006). The patient's partner mostly helped in dealing with the disease (72.7%). The most desired type of support was assistance in accessing rights (median 5, interquartile range 3-5); however, one-third did not use the support services offered to them. A total of 48% the patients talked to their doctor about the struggle and the accompanying difficulties. CONCLUSION: Myeloma patients report various impairments in the biopsychosocial components of QoL. Although supportive services are offered, adjustments must be made to optimally meet patients' needs. Further studies should test the effectiveness of different interventions on the biopsychosocial components of the QoL of these patients in the era of new drugs. </p>.
Artificial Intelligence in Hemophilia Management: Revolutionizing Patient Care and Future Directions.
Levy-Mendelovich S, Glicksberg BS, Soffer S … +3 more , Gendler M, Efros O, Klang E
Acta Haematol · 2025 · PMID 40555203 · Full text
<p>Background: Recent advancements in artificial intelligence (AI) hold significant promise for transforming hemophilia care. Summary: This review explores the impact of AI on critical aspects of hemophilia management, i...
<p>Background: Recent advancements in artificial intelligence (AI) hold significant promise for transforming hemophilia care. Summary: This review explores the impact of AI on critical aspects of hemophilia management, including bleeding risk prediction, biomarker identification, personalized treatment strategies, and patient education. Key Messages: We discuss the application of machine learning models in predicting bleeding risks among children with hemophilia engaging in physical activities, the use of AI in analyzing factor VIII protein structures to determine disease severity, and the development of AI-powered chatbots and digital platforms for patient education and self-management, particularly in resource-limited settings. Furthermore, we address the challenges inherent in implementing AI technologies in clinical practice, such as data privacy concerns, model interpretability, and the need for robust validation. By highlighting current advancements and future directions, we underscore the potential of AI to enhance personalized care and improve outcomes for individuals with hemophilia. </p>.
Treatment of Philadelphia-Positive Acute Lymphoblastic Leukemia.
Torrent A, Ribera JM
Acta Haematol · 2026 · PMID 40555197 · Publisher ↗
BACKGROUND: Philadelphia chromosome-positive acute lymphoblastic leukemia has historically been associated with poor prognosis and limited therapeutic options. Over the past 2 decades, however, the treatment paradigm has...
BACKGROUND: Philadelphia chromosome-positive acute lymphoblastic leukemia has historically been associated with poor prognosis and limited therapeutic options. Over the past 2 decades, however, the treatment paradigm has markedly shifted. SUMMARY: The introduction of tyrosine kinase inhibitors (TKIs), such as imatinib, dasatinib, and ponatinib, has revolutionized frontline therapy, significantly improving remission rates and long-term survival. These agents, when combined with reduced-intensity chemotherapy or even with corticosteroids, have enabled less toxic regimens, particularly beneficial for older or unfit patients. The implementation of measurable residual disease monitoring has emerged as a pivotal tool for risk stratification and therapeutic decision-making. Consequently, the role of allogeneic hematopoietic stem cell transplantation, considered a cornerstone of curative treatment, is being reevaluated in patients achieving sustained deep molecular responses. More recently, immunotherapeutic strategies - including the bispecific T-cell engager blinatumomab and chimeric antigen receptor (CAR) T-cell therapies - have emerged as effective alternatives to conventional chemotherapy and TKIs. KEY MESSAGES: While TKIs remain the backbone of treatment, the integration of immunotherapeutic strategies - including bispecific antibodies and CAR T-cell therapy - has expanded therapeutic options, not only in the R/R setting but increasingly in frontline regimens. Ongoing research aimed at optimizing the sequencing, combination, and duration of these therapies is essential to further enhance clinical outcomes.
Integrating Artificial Intelligence into Hematology: New Frontiers and Practical Applications.
Soffer S, Klang E
Acta Haematol · 2025 · PMID 40517767 · Publisher ↗
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Machine Learning Application for Bleeding Risk Prediction in Patients with Atrial Fibrillation Treated with Oral Anticoagulation.
Lerman TT, Tiosano S, Beigel R … +8 more , Cohen-Shelly M, Kornowski R, Munitz R, Nace DA, Hassan S, Scandrett K, Forman DE, Fishman B
Acta Haematol · 2025 · PMID 40517766 · Publisher ↗
Atrial fibrillation (AF) is a prevalent cardiac arrhythmia associated with a significantly increased risk of systemic thromboembolism and stroke. Anticoagulation therapy, particularly with direct oral anticoagulants, has...
Atrial fibrillation (AF) is a prevalent cardiac arrhythmia associated with a significantly increased risk of systemic thromboembolism and stroke. Anticoagulation therapy, particularly with direct oral anticoagulants, has become the standard for stroke prevention but comes at the cost of an increased bleeding risk. With the introduction of effective alternatives to anticoagulation, such as percutaneous left atrial appendage occlusion, bleeding risk stratification has become essential to guide therapeutic decision-making. Conventional statistical methods have been used for bleeding risk stratification scores, such as HEMORR2HAGES, HAS-BLED, and ATRIA. However, these methods may inadequately address the multifactorial nature of bleeding risk in diverse patient populations, and their overall performance has been suboptimal. Summary and Key Messages: Recent advancements in machine learning (ML) offer promising opportunities to enhance bleeding risk prediction and optimize anticoagulation therapy. This review explores ML applications in AF patients receiving anticoagulation therapy, focusing on the development and validation of ML-based bleeding risk scores. These models have demonstrated improved predictive performance compared to traditional tools, leveraging complex datasets to identify nuanced patterns and interactions. Furthermore, ML-driven tools in warfarin management, including dose prediction, optimization of time in the therapeutic range, and the identification of drug-drug interactions, show significant potential to enhance patient safety and treatment efficacy.
Ring Chromosomes in Patients with Myeloid Neoplasms Are Associated with a Poor Response to Therapy.
Nathan DI, Hoffman R, Ahire D … +6 more , Mascarenhas J, Marcellino BK, Tremblay D, Silverman LR, Najfeld V, Feld J
Acta Haematol · 2026 · PMID 40505637 · Full text
UNLABELLED: <p>Introduction: Ring chromosomes (RCs) are acquired circular structural abnormalities associated with gain or loss of genetic material, which are thought to be associated with inferior outcomes in patients w...
UNLABELLED: <p>Introduction: Ring chromosomes (RCs) are acquired circular structural abnormalities associated with gain or loss of genetic material, which are thought to be associated with inferior outcomes in patients with myeloid neoplasms (MNs). Responses of patients with MN and RC to the standard therapeutic options have not been previously reported. METHODS: We analyzed the demographics and outcomes of 31 consecutive patients with an MN and RC, comparing overall survival (OS) and progression-free survival (PFS) of patients who received supportive care (n = 9), cytotoxic chemotherapeutic options (n = 3), hypomethylating agents (HMA) alone (n = 6), or HMA in combination with venetoclax (n = 13). RESULTS: Over 60% of all patients with RC had either a TP53 mutation, loss of 17p, or both. Interestingly, 22/31 (71%) of patients had not received prior radiation or chemotherapy. Patients who received supportive care had a shorter OS (p = 0.001), but none of the therapeutic interventions were associated with further improvement in prolonging OS (p = 0.86) or PFS. The presence of a complex karyotype, TP53 mutations/loss of TP53, or a treatment-related MN was not independently associated with an inferior OS in MN patients with RCs. CONCLUSION: These findings indicate that patients with MN and RC have especially poor outcomes and that effective treatment strategies remain an unmet need. </p>.
Treatment of Older Adults with Newly Diagnosed Philadelphia Chromosome-Negative Acute Lymphoblastic Leukemia.
Mendez LM, DeAngelo DJ, Luskin MR
Acta Haematol · 2026 · PMID 40494331 · Publisher ↗
BACKGROUND: Approximately half of newly diagnosed cases of acute lymphoblastic leukemia (ALL) occur in adults, but adults experience significantly higher rates of treatment failure and treatment-related mortality due to...
BACKGROUND: Approximately half of newly diagnosed cases of acute lymphoblastic leukemia (ALL) occur in adults, but adults experience significantly higher rates of treatment failure and treatment-related mortality due to frequent presence of adverse disease biology and limited tolerability of conventional chemotherapy. SUMMARY: Here, we discuss recent data from clinical trials investigating new approaches for initial treatment of Philadelphia chromosome-negative ALL in older adults. These trials investigate the incorporation of novel agents including the anti-CD22 antibody-drug conjugate inotuzumab, the CD19-CD3 bi-specific T-cell engager blinatumomab, and the BCL2 inhibitor venetoclax into treatment regimens, with some studies attenuating or omitting chemotherapy. We also discuss the role of allogeneic stem cell transplantation consolidation for this population and highlight the possibility of frontline CD19-directed chimeric antigen receptor T-cell therapy consolidation approaches for B-ALL. Finally, we discuss improved understanding of the genetic diversity of ALL in older adults including occurrence of ALL with TP53 mutation, ALL with myeloid gene mutations, and therapy-related ALL. KEY MESSAGE: Overall, we highlight progress for older adults with Ph-negative ALL with patients more frequently achieving a measurable residual disease-negative complete remission, but significant work remains to improve the safety of treatment as well as the depth and durability of treatment response.
Ultrasonography-Guided Core-Needle Biopsy for Newly Diagnosed Large B-Cell Lymphomas: Analysis on Diagnostic Efficacy and Safety in an Italian Retrospective Study.
Picardi M, Giordano C, Vincenzi A … +7 more , Pugliese N, Scarpa A, Mascolo M, Vigliar E, Troncone G, Salvatore C, Pane F
Acta Haematol · 2026 · PMID 40418928 · Full text
UNLABELLED: <p>Introduction: Excisional biopsy (EB) is the gold standard for large B-cell lymphomas (LBCLs) diagnosis. Based on recent advances in interventional radiology enabling accurate sampling with core needle biop...
UNLABELLED: <p>Introduction: Excisional biopsy (EB) is the gold standard for large B-cell lymphomas (LBCLs) diagnosis. Based on recent advances in interventional radiology enabling accurate sampling with core needle biopsy (CNB), we evaluated efficacy and safety of CNB under imaging guidance for diagnosing LBCLs. METHODS: At the Hematology and Pathology Units of the Federico II University Medical School of Naples (Italy), we retrospectively collected patients with lymphadenopathies suspected of lymphomas (during 2009-2022) of which the ultrasonography (US)-guided CNB lymph node samples were available. Subsequently, we investigated the accuracy and safety of US-guided CNB for LBCLs diagnosis. RESULTS: Over a 12-year period, 800 (superficial target, n = 560; deep-seated target, n = 240) lymph node biopsies performed with 16-gauge diameter modified Menghini needle under power-Doppler ultrasonographic guidance have been evaluated in 800 patients. According to the reference standard, 220 were suffering from LBCLs (diffuse LBCL NOS [n = 196], and high-grade B-cell lymphoma with MYC and BCL2 rearrangements [n = 24]) subtypes, other malignancy subtypes (n = 510) and non-malignant findings (n = 70). For the series of LBCLs, the overall diagnostic accuracy of the micro-histological sampling was 100% (95% confidence interval: 98%-100%). The complications occurred with very low incidence and severity (grade ≤2). CONCLUSION: US-guided CNB is a less invasive method and can be considered an alternative to EB for LBCL diagnosis. </p>.
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