Haematologica最新文献

{"title":"Plasma exchange for hyperammonemia-induced reduced consciousness after PEG-asparaginase in an adult patient with acute lymphoblastic leukemia.","authors":"Felix Klingler, Anika Forstreuter, Luisa Stegat, Sofie Kämereit, Felix Ullrich, Florian Udonta, Christian Krebs, Dominik Wichmann, Carsten Bokemeyer, Nicola Goekbuget, Walter Fiedler, Franziska Modemann","doi":"10.3324/haematol.2024.286972","DOIUrl":"10.3324/haematol.2024.286972","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1660-1664"},"PeriodicalIF":8.2,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143614726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"New insights into the generation and function of megakaryocytes in health and disease.","authors":"Changhong Du, Jun Chen, Junping Wang","doi":"10.3324/haematol.2024.287236","DOIUrl":"10.3324/haematol.2024.287236","url":null,"abstract":"<p><p>Megakaryocytes are canonically viewed as specialized hematopoietic cells that merely produce platelets and that are generated through a series of hematopoietic stem and progenitor cells in the bone marrow. Despite their essential physiological functions, the generation and function of megakaryocytes remain incompletely understood. Recent studies, mostly in mice, have begun to redefine the cellular hierarchy of megakaryopoiesis, and shed new light on the alternative routes and mechanisms of megakaryopoiesis. Moreover, the conception of megakaryocytes as a homogeneous cell population with the sole purpose of platelet production is being challenged. Indeed, megakaryocytes are being shown to be a heterogeneous population of cells with distinct routes of differentiation and versatile functions. In particular, megakaryocytes show abilities reminiscent of immune cells, and are increasingly considered as the root dictating the hemostatic and immune functions of platelets in various physiopathological conditions. Furthermore, although megakaryocytes are well known as a component of the bone marrow niche, maintaining hematopoietic stem cells during homeostasis, the newly identified properties of megakaryocytes further indicate that they may be key supporters of stressed or diseased hematopoietic stem cells during myeloablative injury, aging and hematopoietic malignancy. Therefore, the generation and function of megakaryocytes are more diverse than we previously thought. Here, we review the recent literature that expands our views of megakaryocyte differentiation and heterogeneity, as well as the functions of megakaryocytes, with special focuses on their immune functions and supporting roles for stressed or diseased hematopoietic stem cells.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1500-1512"},"PeriodicalIF":8.2,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143718804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patients with high-risk acute promyelocytic leukemia need maintenance therapy for 1 year - the CONS.","authors":"Harry J Iland","doi":"10.3324/haematol.2025.287418","DOIUrl":"10.3324/haematol.2025.287418","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1459-1465"},"PeriodicalIF":8.2,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143772244","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Should we move past erythropoietin-stimulating agent monotherapy in lower-risk myelodysplastic syndromes?","authors":"Lee Mozessohn,Rena Buckstein","doi":"10.3324/haematol.2025.288157","DOIUrl":"https://doi.org/10.3324/haematol.2025.288157","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"9 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488074","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chimera's curse: myeloid clonal evolution after CD19-directed CAR T-cell therapy.","authors":"Gregory W Roloff","doi":"10.3324/haematol.2025.287976","DOIUrl":"https://doi.org/10.3324/haematol.2025.287976","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"48 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488110","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"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.","authors":"Roberta S Azevedo,Wei-Ying Jen,Danielle Hammond,Fadi G Haddad,Alexis Geppner,Ghayas C Issa,Koji Sasaki,Jayastu Senapati,Elias Jabbour,Farhad Ravandi,Hagop Kantarjian,Tapan M Kadia","doi":"10.3324/haematol.2025.287932","DOIUrl":"https://doi.org/10.3324/haematol.2025.287932","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"242 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Asciminib for Philadelphia chromosome positive leukemias.","authors":"Timothy P Hughes,Deborah L White,David T Yeung","doi":"10.3324/haematol.2024.286798","DOIUrl":"https://doi.org/10.3324/haematol.2024.286798","url":null,"abstract":"Twenty-five years after the introduction of imatinib, we have entered a new era of therapy for Chronic Myeloid Leukemia (CML). Despite the development of second and third generation (2G and 3G) tyrosine kinase inhibitors (TKIs), their impact have been incremental in improving outcomes for CML patients. While frontline use of 2G TKIs have improved molecular response rates and reduced progression to blast phase, there has been no improvement in overall survival compared to imatinib, likely due to the higher toxicity and consequent higher non CML-related mortality. Imatinib remains the most prescribed therapy for CML worldwide, despite it being the least potent TKI and most prone to resistance and progression. Asciminib, the first STAMP (Specifically Targeting the ABL Myristoyl Pocket) inhibitor, binds to the myristoyl pocket of BCR::ABL1. Its specificity minimises off-target toxicity which enables asciminib to finally break this frustrating link between potency and toxicity. After a decade of clinical trials, both in patients with resistance and intolerance to two or more TKIs, and more recently in the frontline setting, asciminib is fulfilling its early promise of a more rapid and reliable pathway to long-term disease control with minimal toxicity. There are however some unexpected challenges when using asciminib that require further investigation. In this Spotlight we review the key studies and outline the potential impact and current limitations of this first STAMP inhibitor in the CML setting and in other leukemias where ABL1 or ABL2 is the key target.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"21 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488148","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"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.","authors":"Guillaume Moulis,Frederick Chen,Giuseppe Carli,Waleed Ghanima,Karolin Trautmann-Grill,Thomas Stauch,Alexandra Schifferli,Haroon Miah,Manuela Rueter,Lisanna Ghiotto,Riccardo Tomasello,Annabell Georgi,Vickie McDonald,Francesco Zaja,Heidi Hassel Pettersen,Thomas Kühne,Maria Luisa Lozano,Tomás José González-López,Drew Provan,Marc Michel,Nichola Cooper,Francesco Rodeghiero","doi":"10.3324/haematol.2025.287408","DOIUrl":"https://doi.org/10.3324/haematol.2025.287408","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"243 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488167","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early detection and management of extracranial arteriopathy reduces the incidence of silent cerebral infarcts in sickle cell anemia: a long-term prospective cohort study.","authors":"Francoise Bernaudin,Cecile Arnaud,Annie Kamdem,Jenny Youn,Manuela Vasile,Isabelle Hau,Fouad Madhi,Aline Malterre,Celine Delestrain,Ralph Epaud,Camille Jung,Suzanne Verlhac","doi":"10.3324/haematol.2025.287720","DOIUrl":"https://doi.org/10.3324/haematol.2025.287720","url":null,"abstract":"Previous reports about the Creteil newborn-cohort (1988/Apr-2007) showed that the risk of silent cerebral infarcts (SCI) remained high (37.1%) by age 14 in children with sickle cell anemia (SCA) and intracranial time-averaged mean maximum velocity (TAMMV)≥200cm/s despite chronictransfusion. Systematic assessment of extracranial internal carotid artery (eICA) since June-2011 revealed that SCI-risk is associated with chronic or acute anemia and eICA-stenosis. Based on these results, SCA-children with eICA-TAMMV≥200cm/s or eICA-stenosis were placed on chronictransfusion and considered for allogeneic stem-cell transplantation (alloSCT). SCA-children with 160-199cm/s eICA-TAMMV were maintained on hydroxyurea. We hypothesized that detection/management of eICA-arteriopathy and wider use of hydroxyurea could reduce SCI-incidence. Comparison between the new cohort (May-2007/Dec-2014) eICA-assessed before age 4 with wider but not systematic use of hydroxyurea and the earlier cohort (1988/Apr-2007) never eICAassessed until the 2008 update, revealed a significant reduction of SCI-risk (Log-Rank, P=.009) associated with eICA-assessment but not with wider use of hydroxyurea. eICA-TAMMVs≥160cm/s, even with no eICA-stenosis, were risk-factors for SCI, suggesting that all SCA-children with eICATAMMV≥ 160cm/s should be placed on chronic-transfusion. Hydroxyurea initiation at an early age was associated with lower intracranial-arteriopathy incidence, but not with lower eICA-arteriopathy and SCI-incidence. In the overall cohort (1988-2014), including 332 SCA-children, all assessed/managed for eICA-arteriopathy after 2011, the cumulative-SCI-incidence by age 14 was 25.0% (95%CI:19.0%-31.0%). SCI-risk was associated with being older at first-neck-MRA and having high MCV on hydroxyurea. While the impact of hydroxyurea on SCI-incidence remains unclear, making controlled trials necessary, eICA-arteriopathy management by intensive therapy is effective at improving SCIprevention.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"17 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An aggressive hematolymphoid neoplasm with homozygous SMARCB1 loss shows response to EZH2 inhibition.","authors":"Iman Sarami,Amy S Duffield,Suchitra Sundaram,Douglas I Lin,Christian Salib,Shafinaz Hussein,Siraj El Jamal,Nasrin Ghesani,Busra Cangut,Bruce E Petersen","doi":"10.3324/haematol.2024.285776","DOIUrl":"https://doi.org/10.3324/haematol.2024.285776","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"46 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488150","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}