Haematologica最新文献

{"title":"CSF3R mutations and variants in myeloid neoplasms: associated phenotypes, co-mutations, and survival trends.","authors":"Abiola Bolarinwa,Natasha Szuber,Rong He,Animesh Pardanani,Naseema Gangat,Ayalew Tefferi","doi":"10.3324/haematol.2025.288026","DOIUrl":"https://doi.org/10.3324/haematol.2025.288026","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"51 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594045","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":"Improving chronic myeloid leukemia management and quality of life: patient and physician survey on unmet needs from the CML SUN survey.","authors":"Fabian Lang,Zack Pemberton-Whiteley,Joannie Clements,Cristina Ruiz,Delphine Rea,Lisa Machado,Naoto Takahashi,Sung-Ho Moon,Andrew Grigg,Cornelia Borowczak,Peter Schuld,Pauline Frank,Cristina Constantinescu,Carla Boquimpani,Jorge E Cortes","doi":"10.3324/haematol.2025.287772","DOIUrl":"https://doi.org/10.3324/haematol.2025.287772","url":null,"abstract":"For patients with chronic myeloid leukemia in chronic phase (CML-CP), disease management, treatment experiences, and decisions around switching therapies due to resistance or intolerance can have significant impacts on their lives. Experiences and perspectives regarding the roles of patients and treating physicians in shared decision-making are poorly understood. The CML Survey on Unmet Needs (CML SUN), the largest CML survey to date, was initiated to gather insights from patients with CML-CP and physicians on disease management, including treatment goals, decision-making, satisfaction, tolerability and the impact of CML on daily life. The survey was deployed in 11 countries with 361 patient and 198 physician participants and comprised separate questionnaires for each group. Results indicated that nearly three-quarters of physicians saw themselves as the ultimate initial treatment decision-makers; only a quarter of patients reported that these decisions were discussed and decided together with their physician. Nearly half of physicians reported making treatment decisions across all lines of therapy with little to no input from the patient. Disparities between patient and physician opinions were observed regarding treatment goals, especially the balance between efficacy and tolerability. The CML SUN highlights the need for improvements in communication about treatment options and the importance of shared treatment decisionmaking to unify treatment goals.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"1 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594088","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":"The small GTPase ARF6 regulates sphingolipid homeostasis and supports proliferation in acute myeloid leukemia.","authors":"Helong Gary Zhao,Nataly Cruz-Rodriguez,Kent C Johnson,Anthony D Pomicter,Briana Bates,Benjamin Bateman,Torsten Haferlach,Tongjun Gu,Jonathan Ahmann,Dongqing Yan,Greg S Lee,Weiquan Zhu,Jenna Bishop,Shannon J Odelberg,Michael W Deininger","doi":"10.3324/haematol.2024.286228","DOIUrl":"https://doi.org/10.3324/haematol.2024.286228","url":null,"abstract":"Metabolic dependencies are emerging as promising therapy targets in cancer, including acute myeloid leukemia (AML). Several metabolic vulnerabilities have been identified in AML cells, including a requirement for balanced sphingolipid metabolism to maintain survival and proliferation. Here we describe a novel function of the RAS superfamily small GTPase ARF6 in maintaining sphingolipid homeostasis in AML. Genetic depletion of ARF6 inhibited the proliferation of AML cell lines and reduced colony formation of primary AML CD34+ cells. Mechanistically, ARF6 promotes conversion of ceramide to sphingomyelin by enhancing sphingomyelin synthase (SGMS1/2) expression, thereby preventing accumulation of cytotoxic ceramide levels. Accordingly, higher expression of ARF6 and its effectors SGMS1/2 in AML patient cells correlates with shorter survival in two independent AML cohorts, with ARF6 exhibiting an adverse prognostic effect independent of European Leukemia Net genetic risk. Small molecule inhibitors of ARF6 suppressed colony formation by primary AML CD34+ cells, but not cord blood CD34+ cells and showed activity in xenograft models. The dependency of AML cells on ARF6 to regulate sphingolipid homeostasis may present a therapeutic opportunity.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"16 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594092","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":"Cytopenias and infections following ciltacabtagene autoleucel in heavily-pretreated relapsed or refractory multiple myeloma.","authors":"Danai Dima,Jennifer M Logue,Syed Hamza Bin Waqar,Lauren C Peres,Christelle M Colin-Leitzinger,Gabriel De Avila,Eric C Smith,Lawrence Skelson,Kristy L Matte,Brandon Blue,Vanna N Hovanky,Mahmoud Gaballa,Oren Pasvolsky,Laura B Oswald,Gliceida Galarza M Fortuna,Charlotte B Wagner,Shaun DeJarnette,Christen Dillard,Fabiana Perna,Lekha Mikkilineni,Hitomi Hosoya,Ciara L Freeman,Kenneth H Shain,Rachid C Baz,Ariel Grajales-Cruz,Omar Castaneda Puglianini,Melissa Alsina,Frederick L Locke,Leyla O Shune,Douglas W Sborov,Krina K Patel,Surbhi Sidana,Doris K Hansen","doi":"10.3324/haematol.2025.287783","DOIUrl":"https://doi.org/10.3324/haematol.2025.287783","url":null,"abstract":"Ciltacabtagene autoleucel (cilta-cel) was FDA-approved in February 2022 for the treatment of relapsed/refractory multiple myeloma after 4 lines of therapy. On CARTITUDE-1 trial, grade ≥3 cytopenias and infections were common. Herein, we sought to characterize cytopenias and infections after cilta-cel infusion in the standard-of-care setting. This multicenter retrospective study included 105 patients who received cilta-cel; 91 reached day-90 and 49 reached day-180 follow-up. Grade ≥3 cytopenia was present among 52% of patients on day-30, and 24% of patients on day-90. Based on the newer immune effector cell-associated hematotoxicity (ICAHT) grading for neutropenia severity, 11 patients (10%) experienced grade ≥3 early ICAHT in the first 30 days, while only 3 (3.3%) experienced grade ≥3 late ICAHT after day-30. On univariate analysis, any grade thrombocytopenia at apheresis was associated with grade ≥3 cytopenia at both days 30 and 90. Granulocyte colony-stimulating factor was administered to 65%, transfusion support to 38%, thrombopoietin agonists to 10%, intravenous immunoglobulin to 52%, and CD34+ stem cell boosts to 9.5% of patients. Infections occurred in 49% of patients and were severe in 32%. Earlier infections in the first 30 days were equally bacterial (42%) and viral (42%). Later infections between days 31-100 and after day 100 were mostly viral (59% and 60%), with only 32% and 12% being grade ≥3 at each time period. On univariate analysis, worse ECOG performance status at lymphodepletion, higher maximum grade of CRS, delayed neurotoxicity, steroid and anakinra use, and lower IgA levels at day 90 were associated with severe infections.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"697 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594093","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":"Flow cytometric analysis of cerebrospinal fluid identifies patients with occult central nervous system involvement in relapsed pediatric acute lymphoblastic leukemia.","authors":"Maria Thastrup,Hanne Vibeke Marquart,Mette Levinsen,Aina Ulvmoen,Birgitte Klug Albertsen,Cornelis Jan Pronk,Marianne Olsen,Peder Wehner,Mervi Taskinen,Bodil Als-Nielsen,Kjeld Schmiegelow","doi":"10.3324/haematol.2025.287392","DOIUrl":"https://doi.org/10.3324/haematol.2025.287392","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"9 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594090","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":"Ovarian tissue autotransplantation in acute leukemia: balancing the risk of relapse and the hope of parenthood.","authors":"Florian Chevillon,Elsa Labrune,Emmanuelle Clappier,Hélène Lapillonne,Paola Ballerini,Virginie Barraud Lange,Anne Fortin,Raphael Itzykson,Gérard Socié,Agnès Buzyn,Guy Leverger,Jean-Hugues Dalle,Nathalie Dhédin,Nicolas Boissel,Régis Peffault De Latour,Bruno Salle,Christophe Roux,Catherine Poirot","doi":"10.3324/haematol.2025.287942","DOIUrl":"https://doi.org/10.3324/haematol.2025.287942","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"14 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594034","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":"Integrating killer cell immunoglobulin-like receptor high-resolution genotyping for predicting transplant outcomes in allogeneic hematopoietic stem cell transplantation.","authors":"Antonia Schäfer,Stéphane Buhler,Ticiana D J Farias,Katherine M Kichula,Helen Baldomero,Zuleika Calderin Sollet,Sylvie Ferrari-Lacraz,Baptiste Micheli,Stavroula Masouridi-Levrat,Vanessa Mesquita,Oliver Kürsteiner,Gayathri Nair,Jörg Halter,Tayfun Güngör,Dominik Schneidawind,Yves Chalandon,Jakob R Passweg,Paul J Norman,Jean Villard","doi":"10.3324/haematol.2024.287061","DOIUrl":"https://doi.org/10.3324/haematol.2024.287061","url":null,"abstract":"The success of hematopoietic stem cell transplantation (HSCT) partly relies on the beneficial graft-versus-leukemia effect, mediated by alloreactive NK cells through their killer cell immunoglobulin-like receptors (KIR). Conflicting results have been reported regarding the impact of the KIR immunogenetic system on HSCT outcomes with a scarcity of data interrogating the effect of KIR allelic polymorphism. With the aim to fill this gap, donor KIR genes derived from a national cohort of 1247 HLA-matched transplanted donor/recipient pairs were determined at a high-resolution and tested in Cox proportional hazards models. Donor/recipient (D/R) pairs bearing a KIR2DS4*00101 - HLA-C1/C2/A11 interaction showed a significant detrimental impact on progression-free survival (PFS), overall survival (OS), transplant-related mortality (TRM) and chronic graft-versus-host disease (cGvHD) in multivariable analysis. Strong KIR2DL2/L3 - HLAC1 and especially KIR2DL3*00501 and *015 interactions showed a significant increase in the incidence of cGvHD compared to missing ligand D/R pairs. Highly inhibiting KIR3DL1 - HLA-B and HLA-A (Bw4) interactions were associated with a reduced relapse incidence as compared to weak and non-inhibiting interactions. Our study indicates that high-resolution KIR genotyping informs post-transplant outcomes with a seemingly higher protection of educated NK cells.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"39 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594044","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":"Real-world validation study of the LSC17 score for risk prediction in patients with newly diagnosed acute myeloid leukemia.","authors":"Tracy Murphy,Boyang Zhang,Tong Zhang,Ian King,Jose-Mario Capo-Chichi,Vikas Gupta,Dawn Maze,Caroline J McNamara,Mark D Minden,Aaron D Schimmer,Andre C Schuh,Hassan Sibai,Karen W L Yee,Andrea Arruda,Zhibin Lu,Dina Khalaf,Chantal Rockwell,Brian Leber,Mitchell Sabloff,Anne Tierens,Tracy L Stockley,Steven M Chan,Stanley W K Ng,Jean C Y Wang","doi":"10.3324/haematol.2025.287777","DOIUrl":"https://doi.org/10.3324/haematol.2025.287777","url":null,"abstract":"Acute myeloid leukemia (AML) patients exhibit diverse molecular and cytogenetic changes with heterogeneous outcomes. The functionally-derived LSC17 gene expression score has demonstrated strong prognostic significance in retrospective analyses of adult and pediatric AML cohorts, where above-median scores are associated with worse outcomes compared to below-median scores in intensively-treated patients. Here we used a laboratory-developed clinically-validated NanoStringbased LSC17 assay to test the prognostic value of the LSC17 score in a prospective multicentre study of 276 newly-diagnosed AML patients. Each patient's score was classified as high or low by comparison to a previously-established reference score. In the entire cohort, a high LSC17 score was associated with poor risk features, including advanced age and unfavorable genetic mutations. In the subset of 190 patients treated intensively, a high LSC17 score was associated with lower remission rates (63% vs 94% after induction, P.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"4 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144594089","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":"Unveiling platelet aging with progressive β-galactose exposure as a signature of platelet senescence in humans and mice.","authors":"Catherine Angénieux,Thomas Nipoti,Gabriel Rojas-Jiménez,Mélanie Daniel,Frédéric Adam,Annabelle Dupont,Blandine Maître,Alexandre Kauskot","doi":"10.3324/haematol.2025.287812","DOIUrl":"https://doi.org/10.3324/haematol.2025.287812","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"76 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144547769","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":"BTKi +humanized anti-CD20 for advanced follicular lymphoma: ready for an ALTERNATIVE?","authors":"Irene Dogliotti,Massimo Federico","doi":"10.3324/haematol.2025.288196","DOIUrl":"https://doi.org/10.3324/haematol.2025.288196","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"20 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144547768","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}