Acta Haematologica最新文献

{"title":"Deep Learning Applications in Lymphoma Imaging.","authors":"Vera Sorin, Israel Cohen, Ruth Lekach, Sasan Partovi, Daniel Raskin","doi":"10.1159/000547427","DOIUrl":"https://doi.org/10.1159/000547427","url":null,"abstract":"<p><p>Lymphomas are a diverse group of disorders characterized by the clonal proliferation of lymphocytes. While definitive diagnosis of lymphoma relies on histopathology, immune-phenotyping and additional molecular analyses, imaging modalities such as PET/CT, CT, and MRI play a central role in the diagnostic process and management, from assessing disease extent, to evaluation of response to therapy and detecting recurrence. Artificial intelligence (AI), particularly deep learning models like convolutional neural networks (CNNs), is transforming lymphoma imaging by enabling automated detection, segmentation, and classification. This review elaborates on recent advancements in deep learning for lymphoma imaging and its integration into clinical practice. Challenges include obtaining high-quality, annotated datasets, addressing biases in training data, and ensuring consistent model performance. Ongoing efforts are focused on enhancing model interpretability, incorporating diverse patient populations to improve generalizability, and ensuring safe and effective integration of AI into clinical workflows, with the goal of improving patient outcomes.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-17"},"PeriodicalIF":1.7,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144635905","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Early-Onset or Worsening Anemia in Patients With Myelofibrosis Treated With Ruxolitinib: A Post Hoc Analysis of the JUMP Study.","authors":"Haifa Kathrin Al-Ali, Paola Guglielmelli, Claire N Harrison, Ruben A Mesa, J E Hamer-Maansson, Evan Braunstein, Vikas Gupta","doi":"10.1159/000546585","DOIUrl":"https://doi.org/10.1159/000546585","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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 RBC transfusions through Week 12.</p><p><strong>Results: </strong>Overall, 2233 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 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.</p><p><strong>Conclusions: </strong>These results support the use of ruxolitinib in patients with myelofibrosis regardless of baseline anemia or development of treatment-related anemia.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":1.7,"publicationDate":"2025-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144625253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Complexities of MRD Assays in ALL: A Guide for the Practicing Clinician.","authors":"Jerry Luo, Lori Muffly","doi":"10.1159/000547423","DOIUrl":"https://doi.org/10.1159/000547423","url":null,"abstract":"<p><p>Assessment of measurable residual disease (MRD) is an important component of acute lymphoblastic leukemia (ALL) management. Quantification of MRD may be performed through a variety of assays, each with differing sensitivity for leukemia detection. In this review article, we focus on complexities that the treating physician may face when considering MRD in ALL. New information on the optimal MRD assay for Philadelphia chromosome positive ALL, specimen source for MRD testing (eg, blood, bone marrow, cerebrospinal fluid), when to test, and updates on MRD in T-cell ALL are included.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":1.7,"publicationDate":"2025-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144625252","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Venetoclax-based regimen in refractory or relapsed pediatric acute lymphoblastic leukemia.","authors":"Lin Zhang, Zhixiao Zhang, Aidong Lu, Yueping Jia, Lin Huang, Leping Zhang, Huimin Zeng","doi":"10.1159/000547080","DOIUrl":"https://doi.org/10.1159/000547080","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Patients and methods: </strong>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 response (CR) or complete response with incomplete recovery of blood count (CRi) rates and objective response rates (ORR) were analyzed.</p><p><strong>Results: </strong>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 1371 days. For the refractory patients, the median OS was unreached. For T-ALL patients, the median OS was 1371 days. For the patients with B-ALL, the median OS was 543 days. All patients had hematologic adverse reactions within an acceptable range.</p><p><strong>Conclusion: </strong>Children with R/R ALL who received the VEN-based regimen achieved a high response 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.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-18"},"PeriodicalIF":1.7,"publicationDate":"2025-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144590221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"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.","authors":"Shreekant Parasuraman, Kimberly Saverno, Kristin Zimmerman Savill, Philomena Colucci","doi":"10.1159/000546910","DOIUrl":"https://doi.org/10.1159/000546910","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":1.7,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144574673","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploration of B and T cell receptor repertoires reveals distinct mechanisms in pure red cell aplasia, autoimmune hemolytic anemia, and aplastic anemia.","authors":"Ruoxi Zhang, Bing Han","doi":"10.1159/000547027","DOIUrl":"https://doi.org/10.1159/000547027","url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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 AA group was more diverse than that of NC group (p < 0.05). Regarding BCR and TCR repertoires, PRCA, AIHA, and AA groups had uniform gene characteristics. The preferential gene of immunoglobulin heavy (IGH) chains in PRCA patients was correlated with memory cell and red blood cell antigen recognition; genes expressed in AIHA group was associated with 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 NC group, 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, 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.</p><p><strong>Conclusion: </strong>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>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-18"},"PeriodicalIF":1.7,"publicationDate":"2025-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144525949","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Genomic Subclassification and Risk Stratification of ALL.","authors":"Anna Østergaard, Ilaria Iacobucci","doi":"10.1159/000547135","DOIUrl":"https://doi.org/10.1159/000547135","url":null,"abstract":"<p><p>Over the last two 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. 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. In this review, we describe the updated genomic classification of ALL highlighting new biological insights and discussing their implications for prognostication and outcome.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-21"},"PeriodicalIF":1.7,"publicationDate":"2025-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144525950","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment of Philadelphia positive acute lymphoblastic leukemia.","authors":"Anna Torrent, Josep Maria Ribera","doi":"10.1159/000547026","DOIUrl":"https://doi.org/10.1159/000547026","url":null,"abstract":"<p><p>Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) has historically been associated with poor prognosis and limited therapeutic options. Over the past two decades, however, the treatment paradigm has markedly shifted. 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 (MRD) monitoring has emerged as a pivotal tool for risk stratification and therapeutic decision-making. Consequently, the role of allogeneic hematopoietic stem cell transplantation (allo-HSCT), considered a cornerstone of curative treatment, is being re-evaluated 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. This review outlines the major advances in the management of Ph+ ALL, emphasizing the move toward more personalized, targeted, and less toxic treatment approaches.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-22"},"PeriodicalIF":1.7,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144482831","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Artificial Intelligence in Hemophilia Management: Revolutionizing Patient Care and Future Directions.","authors":"Sarina Levy-Mendelovich, Benjamin S Glicksberg, Shelly Soffer, Moran Gendler, Orly Efros, Eyal Klang","doi":"10.1159/000546954","DOIUrl":"https://doi.org/10.1159/000546954","url":null,"abstract":"<p><p>Recent advancements in artificial intelligence (AI) hold significant promise for transforming hemophilia care. This review explores AI's impact on critical aspects of hemophilia management, including bleeding risk prediction, biomarker identification, personalized treatment strategies, and patient education. 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 AI's potential to enhance personalized care and improve outcomes for individuals with hemophilia.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":1.7,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144482829","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identification of Needs of Patients with Multiple Myeloma: A Questionnaire-Based Study in Israeli Patients.","authors":"Ilana Levy Yurkovski, Shira Ben-Dov, Varda Shoam, Micha Yuz, Noa Lavi","doi":"10.1159/000547025","DOIUrl":"10.1159/000547025","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-7"},"PeriodicalIF":1.7,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12258863/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144482830","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}