Haematologica最新文献

{"title":"Severe toxicity and poor efficacy of reinduction chemotherapy are associated with overall poor outcomes in relapsed B-cell acute lymphoblastic leukemia: a report from the Children's Oncology Group AALL1331 trial.","authors":"Laura E Hogan,Teena Bhatla,Xinxin Xu,Lia Gore,Elizabeth A Raetz,Deepa Bhojwani,David T Teachey,Stephen P Hunger,Mignon L Loh,Patrick A Brown,Lingyun Ji","doi":"10.3324/haematol.2025.287386","DOIUrl":"https://doi.org/10.3324/haematol.2025.287386","url":null,"abstract":"Children's Oncology Group AALL1331 utilized an intensive chemotherapy induction (Block 1) based on UK ALLR3 induction for children, adolescents, and young adults with acute lymphoblastic leukemia in first relapse, followed by risk-stratified therapy. High/intermediate risk patients were subsequently randomized to receive 2 blocks of chemotherapy or 2 blocks of blinatumomab followed by hematopoietic stem cell transplant. Low risk patients were randomized to chemotherapy or chemotherapy cycles intercalated with three blinatumomab blocks. Patients who had an early treatment failure were eligible to receive blinatumomab for up to 2 salvage cycles. We reviewed Block 1 responses, risk stratification, randomization rates, adverse events (AE), and event-free survival and overall survival for all enrolled patients. AALL1331 enrolled 661 patients: 24 died during Block 1 and 42 experienced early treatment failure. Overall, 531/661 (80.3%) attained complete remission with 586 risk-assigned and only 471 were randomized. Of 532 patients with marrow involvement, 290 (54.5%) were minimal residual disease positive (≥0.01%) after Block 1. Grade 3/4/5 AE occurred in Block 1 in 44.9, 24.1, and 3.6% patients respectively, with febrile neutropenia, infections, and sepsis most frequent. Notably, 190 enrolled patients (28.7%) did not proceed with post-induction therapy, including 115 (17.4%) risk stratified but not randomized. These patients had dismal survival. More effective and less toxic reinduction strategies are needed for B-ALL in first relapse. Trial Registration Number: NCT02101853.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"52 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488155","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":"Revised renal stratification and progression models for predicting long-term renal outcomes in immunoglobulin light chain amyloidosis.","authors":"Muhammad Umaid Rauf,Steven Law,Marisa Santostefano,Philip N Hawkins,Aviva Petrie,Francesco Cappelli,Federico Perfetto,Yousuf Razvi,Aldostefano Porcari,Sriram Ravichandran,Adam Ioannou,Joshua Bomsztyk,Alessia Argirò,Costanza Gaudio,Elisabetta Antonioli,Alessandro Barilaro,Marco Delsante,Vittorio Di Maso,Maria G Chiappini,Olabisi Ogunbiyi,Oliver C Cohen,Ana Martinez-Naharro,Carol Whelan,Helen J Lachmann,Ashutosh D Wechalekar,Federico Alberici,Marianna Fontana,Marco Allinovi,Julian D Gillmore","doi":"10.3324/haematol.2025.287703","DOIUrl":"https://doi.org/10.3324/haematol.2025.287703","url":null,"abstract":"Renal prognosis in light-chain amyloidosis (AL) is determined by categorizing patients into three renal stages at diagnosis and assessing Renal Response or Renal Progression following chemotherapy after 6 months. We evaluated, in a test (N=1935) cohort of patients with renal AL amyloidosis who were followed for a median of 95 months, a modified 4-stage model where Renal Stage 2 was sub-categorized according to preserved (2A) or reduced (2B) estimated Glomerular Filtration Rate (eGFR). A hybrid model for evaluation of Renal Progression was also introduced, using an eGFR cut-off of 30ml/min/1.73 m2. These models were compared with existing models; namely those of Palladini and Kastritis, and results were validated in a multicenter cohort (N=438). The risk of progression to renal replacement therapy (RRT) increased progressively across all Renal Stages of the revised staging model (Hazard ratio [HR]: 3.25, 5.13, 10.66 for Stages 2A, 2B and 3 respectively vs Stage 1, each p.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"70 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488165","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":"Rethinking the feasibility and safety of venetoclax-obinutuzumab in chronic lymphocytic leukemia: non-traditional factors may play a role in clinical practice.","authors":"Anna Maria Frustaci,Andrea Galitzia,Mariano Lucignano,Lorena Appio,Jacopo Olivieri,Alessandro Sanna,Claudia Baraté,Fabrizio Pane,Luana Schiattone,Beatrice Casadei,Isacco Ferrarini,Amalia Figuera,Paolo Sportoletti,Giacomo Loseto,Caterina Stelitano,Andrea Visentin,Melania Celli,Francesca Romana Mauro,Massimiliano Palombi,Marta Coscia,Vanessa Innao,Riccardo Moia,Marina Motta,Filomena Russo,Monica Tani,Annalisa Arcari,Elia Boccellato,Chiara Borella,Enrico Capochiani,Angela Ferrari,Massimo Gentile,Roberta Giachetti,Annamaria Giordano,Martina Bullo,Enrico Lista,Luigi Malandruccolo,Maurizio Musso,Marzia Varettoni,Federico Vozella,Francesca Cibien,Michele Merli,Laura Nocilli,Maria Cristina Pasquini,Azzurra Anna Romeo,Valentina Rossi,Gloria Turri,Anna Vanazzi,Marina Cavaliere,Alessandro Gozzetti,Lara Crucitti,Moira Lucesole,Marina Deodato,Annamaria Tomasso,Arianna Zappaterra,Roberta Murru,Caterina Patti,Luca Laurenti,Alessandra Tedeschi","doi":"10.3324/haematol.2025.287799","DOIUrl":"https://doi.org/10.3324/haematol.2025.287799","url":null,"abstract":"The concept of fitness for novel agents in chronic lymphocytic leukemia (CLL) remains debated. Comorbidities and treatment-related logistics are increasingly recognized as key factors in treatment feasibility. Venetoclax-obinutuzumab (VO) has demonstrated efficacy in both fit and unfit patients in clinical trials, yet real-world data remain limited. This retrospective, multicenter study analyzed disease- and patient-related factors affecting VO management and outcomes in 271 patients. Fitness was assessed using comorbidity indices (CLL-CI, CIRS, CCI), ECOG-PS, and caregiver need. Adverse events (AEs) and treatment modifications were evaluated across four treatment phases. Median age was 66 years (19% ≥75); 83% had comorbidities, 34% required polypharmacy, and 10% needed caregiver support. Overall, 96% completed debulking, 89% the full regimen, while 11% discontinued due to toxicity (Tox-DTD). Grade ≥3 AEs occurred in 55%, tumor lysis syndrome in 6%, severe infusion-related reactions in 5%. Overall, 3.3% died during treatment. Unfit patients did not show a significantly higher risk of treatment modifications due to AEs. Dose adjustments were more frequent during debulking. None of the validated fitness scores predicted treatment feasibility or Tox-DTD. Global feasibility was impacted by age (p .002), prior malignancies (p .003), prolonged steroid pre-treatment (p.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"75 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488209","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":"Fecal microbiota transplantation for decolonization from multidrug-resistant bacteria in pediatric allogeneic hematopoietic stem cell transplantation recipients: a retrospective real-word data study.","authors":"Riccardo Masetti,Giovanni Barbara,Edoardo Muratore,Giovanni Marasco,Cesare Cremon,Antonella Marangoni,Patrizia Brigidi,Lorenza Putignani,Giulia Angelino,Francesco Quagliarella,Federica Galaverna,Davide Leardini,Tiziana Lazzarotto,Maria Gabelli,Edoardo Savarino,Marco Zecca,Maura Faraci,Arcangelo Prete,Alessandra Biffi,Franco Locatelli,Pietro Merli","doi":"10.3324/haematol.2025.288067","DOIUrl":"https://doi.org/10.3324/haematol.2025.288067","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"67 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144488111","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":"Radiopsy: a prospective observational study on quantitative multiparametric whole-body magnetic resonance imaging to discriminate between smoldering and multiple myeloma.","authors":"Giacomo Feliciani,Emiliano Loi,Elena Amadori,Eleonora Antognoni,Andrea Prochowski Iamurri,Danila Diano,Daniele Bruno,Arrigo Cattabriga,Paola Caroli,Matteo Marchesini,Davide Bezzi,Delia Cangini,Federica Matteucci,Domenico Barone,Davide Nappi,Gerardo Musuraca,Michela Ceccolini,Sonia Ronconi,Irene Azzali,Giovanni Martinelli,Lorenzo Stefano Maffioli,Nicola Normanno,Anna Sarnelli,Alice Rossi,Claudio Cerchione","doi":"10.3324/haematol.2025.287563","DOIUrl":"https://doi.org/10.3324/haematol.2025.287563","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"5 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144319777","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":"Randomized trial of anti-thymocyte globulin plus low-dose post-transplant cyclophosphamide to prevent graft-versus-host disease in haploidentical transplantation.","authors":"Zheng-Li Xu,Ting-Ting Han,Xiao-Lu Zhu,Jing Liu,Meng Lv,Yu-Qian Sun,Xiao-Dong Mo,Yi-Fei Cheng,Lan-Ping Xu,Xiao-Hui Zhang,Xiao-Jun Huang,Yu Wang","doi":"10.3324/haematol.2025.287504","DOIUrl":"https://doi.org/10.3324/haematol.2025.287504","url":null,"abstract":"The combination of anti-thymocyte globulin (ATG) and posttransplant cyclophosphamide (PTCy) appears to be a potentially effective graft-versus-host disease (GVHD) prevention strategy for haploidentical transplantation. However, the majority of the evidence originated from retrospective studies without uniform protocols. Our previous findings indicated that 10 mg/kg ATG plus low-dose PTCy could decrease GVHD among high-risk populations transplanted from maternal or collateral relatives. We designed an open-label, phase III, randomized controlled trial to compare patients receiving granulocyte colony-stimulating factor (G-CSF)/ATG-based haploidentical transplantation with or without low-dose PTCy (14.5 mg/kg on days 3 and 4) in nonmaternal, noncollateral haploidentical transplants from fathers, children or siblings. A total of 66 patients were randomly assigned to ATG-PTCy (n=44) or ATG (n=22) when the first interim analysis was performed. The interim analysis revealed that the 100-day cumulative incidences (CI) of grade II-IV (18.2% [95% CI 6.6-29.7] vs. 18.2% [1.7-34.7]; P = 0.996) and III-IV acute GVHD (2.3% [95% CI 0-6.7] vs. 0; P = 0.480) were comparable between the ATG-PTCy and ATG cohorts, as was chronic GvHD at 1 year. The estimated 1-year disease free survival (DFS) rates were also similar between ATG-PTCy and ATG cohorts (95.5% [95% CI 89.5-100] vs. 95.2% [86.6-100]; P = 0.979). These results suggested that ATG/PTCy (low-dose) had no advantage over 10 mg/kg ATG-based prophylaxis in patients with haploidentical transplantation other than that of maternal donors or collateral relatives. Future work needs to focus on identifying which populations might benefit from the combined strategy in the context of G-CSF/ATG-based protocols.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"13 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144319804","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 STAT3-VDAC1 axis modulates mitochondrial function and plays a critical role in the survival of acute myeloid leukemia cells.","authors":"Kellen B Gil, Jamie Borg, Rosana Moreira Pereira, Anagha Inguva-Sheth, Geovana Araujo, Jeremy Rahkola, William Showers, Abby Grier, Angelo D'Alessandro, Clayton Smith, Christine McMahon, Daniel A Pollyea, Austin E Gillen, Maria L Amaya","doi":"10.3324/haematol.2025.287352","DOIUrl":"https://doi.org/10.3324/haematol.2025.287352","url":null,"abstract":"<p><p>Signal transducer and activator of transcription 3 (STAT3) is a well-described transcription factor that mediates oxidative phosphorylation and glutamine uptake in bulk acute myeloid leukemia (AML) cells and leukemic stem cells (LSCs). STAT3 has also been shown to translocate to the mitochondria in AML cells, and phosphorylation at the serine 727 (pSTAT3 S727) residue has been shown to be especially important for STAT3's mitochondrial functions. We demonstrate that inhibition of STAT3 results in impaired mitochondrial function and decreased leukemia cell viability. We discovered a novel interaction of STAT3 with voltage-dependent anion channel 1 (VDAC1) in the mitochondria which provides a mechanism through which STAT3 modulates mitochondrial function and cell survival. Through VDAC1, STAT3 regulates calcium and oxidative phosphorylation in the mitochondria. STAT3 and VDAC1 inhibition also result in significantly reduced engraftment potential of LSCs, including primary samples resistant to venetoclax. These results implicate STAT3 as a therapeutic target in AML.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144325486","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":"Treatment approaches and clinical outcomes in primary colorectal MALT lymphoma: a single institution retrospective study of 66 patients.","authors":"Suheil Albert Atallah-Yunes,Matthew J Rees,Thomas M Habermann,Yucai Wang,Javier Munoz,Madiha Iqbal,Jose C Villasboas,Ellen D McPhail,Thomas E Witzig,Stephen M Ansell,Grzegorz S Nowakowski","doi":"10.3324/haematol.2025.288145","DOIUrl":"https://doi.org/10.3324/haematol.2025.288145","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"38 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144320293","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":"TET2 mutation does not impact the prognosis of adult acute myeloid leukemia patients receiving a hematopoietic stem cell transplantation in first remission: similar outcome following matched sibling and unrelated versus haploidentical donor transplants in a multi-center retrospective analysis from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.","authors":"Lin Li,Yishan Ye,Jacques-Emmanuel Galimard,Myriam Labopin,Depei Wu,Jia Chen,Nicolaus Kröger,Jakob Passweg,Urpu Salmenniemi,Maija Itäla-Remes,Xavier Poiré,Matthias Eder,Johan Maertens,David Burns,Henrik Sengeloev,Gitte Olesen,Didier Blaise,Jürgen Finke,Alain Gadisseur,Ali Bazarbachi,Eolia Brissot,Arnon Nagler,Yi Luo,Jimin Shi,Mohamad Mohty,He Huang,Fabio Ciceri,Norbert Claude Gorin","doi":"10.3324/haematol.2025.287326","DOIUrl":"https://doi.org/10.3324/haematol.2025.287326","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"100 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144319806","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":"Olverembatinib in chronic myeloid leukemia: is less actually better?","authors":"Maria Agustina Perusini,Dennis Dong Hwan Kim","doi":"10.3324/haematol.2025.288065","DOIUrl":"https://doi.org/10.3324/haematol.2025.288065","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"191 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144319776","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}