Blood Cancer Journal最新文献

{"title":"Switching TKIs during CML therapy is frequent, mostly driven by intolerance, and does not affect survival: a prospective Quebec registry study","authors":"Lambert Busque, Marc-Étienne Beaudet, Michaël Harnois, Hanane Moussa, Natasha Szuber, Luigina Mollica, Robert Delage, Harold Olney, Pierre Laneuville, Ghislain Cournoyer, Inès Chamakhi, Marc Lalancette, Danielle Talbot, Vincent Ethier, Pierre Desjardins, Sarit Assouline","doi":"10.1038/s41408-025-01242-8","DOIUrl":"https://doi.org/10.1038/s41408-025-01242-8","url":null,"abstract":"<figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"32 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143599780","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":"A real-world experience of efficacy and safety of belantamab mafodotin in relapsed refractory multiple myeloma","authors":"Rachel Dileo, Prerna Mewawalla, Kalaivani Babu, Yue Yin, Christopher Strouse, Ethan Chen, Hira Shaikh, James A. Davis, Kimberly M. Green, Omar Alkharabsheh, Aliya Rashid, Bidushi Pokhrel, Nausheen Ahmed, Al-Ola Abdallah, Hamza Hashmi","doi":"10.1038/s41408-025-01226-8","DOIUrl":"https://doi.org/10.1038/s41408-025-01226-8","url":null,"abstract":"<p>While initial trials led to the accelerated approval of belantamab mafodotin, a BCMA-directed antibody-drug conjugate, confirmatory trials failed to establish benefit from this therapy for patients with relapsed refractory multiple myeloma (RRMM), eventually leading to its withdrawal from commercial use. With an imminent approval as an effective combination therapy, as seen in recent randomized trials, we report real-world clinical outcomes with belantamab mafodotin in 81 RRMM patients. With a median of 5 (range 2–15) prior lines of therapy, 92, 45, and 15% of the patients were triple-class refractory, penta-class refractory, and BCMA-refractory. More than half (57%) of the patients had high-risk cytogenetics, 37% had extramedullary disease (EMD), and 67% of the patients would have been considered ineligible for the DREAMM-2 trial. The best overall response (ORR) and complete response rates were 40.0 and 15.0%, respectively. ORRs were lower in patients with EMD, BCMA-refractory, and penta-refractory disease at 23, 17, and 24%, respectively. All-grade ocular toxicity was seen in 69% of patients, with grade 3+ events in 43%. Grade 3+ hematological toxicities included neutropenia (20%), anemia (28%), and thrombocytopenia (31%). With a median follow-up of 11.3 (0.3–44.6) months for the entire population, median PFS and OS were 5 (1–20) months and 12 (3–28) months, respectively. Presence of EMD was the only predictor of both PFS and OS on multivariable analysis. Compared to the pivotal trial and despite several high-risk disease features, belantamab mafodotin demonstrated comparable efficacy and safety in this real-world patient population.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"68 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143589849","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":"Different definitions in intention-to-treat analysis for chimeric antigen receptor T-cell therapy depend on research scope.","authors":"Lisa Argnani, Beatrice Casadei, Pier Luigi Zinzani","doi":"10.1038/s41408-025-01246-4","DOIUrl":"10.1038/s41408-025-01246-4","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"33"},"PeriodicalIF":12.9,"publicationDate":"2025-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11889076/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143584508","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evolution of WHO diagnostic criteria in “Classical Myeloproliferative Neoplasms” compared with the International Consensus Classification","authors":"Jürgen Thiele, Hans Michael Kvasnicka, Umberto Gianelli, Daniel A. Arber, Ayalew Tefferi, Alessandro M. Vannucchi, Tiziano Barbui, Attilio Orazi","doi":"10.1038/s41408-025-01235-7","DOIUrl":"https://doi.org/10.1038/s41408-025-01235-7","url":null,"abstract":"<p>A lively discussion persists regarding the diagnostic criteria for essential thrombocythemia (ET), primary myelofibrosis (PMF) and polycythemia vera (PV), particularly in relation to early/pre-fibrotic myelofibrosis (pre-PMF), a disease entity initially introduced in 2001 by the 3^rd edition of the World Health Organization (WHO) classification. The definition and criteria used to diagnose pre-PMF have been progressively modified over time. The most update definition of pre-PMF can be found in the International Consensus Classification (ICC) published in 2022. An updated largely similar definition is also incorporated in the recently published 5^th edition of WHO classification (2024). Diagnostic criteria for ET have undergone changes up to 2016/17 for the revised 4^th edition of the WHO. In particular the threshold value for platelets were lowered and the important discrimination between “true” and “false” ET (in reality pre-PMF) been widely acknowledged. To avoid misdiagnose in early phase PV, the criteria for gender-adjusted thresholds for hemoglobin/ hematocrit have been lowered and the identification of an appropriate bone marrow (BM) morphology was upgraded as a major diagnostic criterion. Given the prominent role of morphology in MPN-related diagnostic algorithms, the diagnostic adequacy of the BM biopsy (sample procurement and proper laboratory handling) as emphasized in former WHO editions and in the ICC, was not addressed by the WHO 5^th. The essential role of genetic markers is recognized by both classifications. A comparison between the revised 4^th edition WHO classification and the ICC versus the WHO 5^th reveals no significant differences, with the exception of the occurrence of leukoerythroblastosis in pre-PMF considered by the latter as one of the minor diagnostic criteria which seems unwarranted. In contrast to the revised 4^th edition, the majority of the microscopic images used for the WHO 5^th due to their low magnification and poor technique, do not highlight the diagnosis differences among these entities.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"36 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143538369","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":"Dexamethasone for the management of CRS Related to teclistamab in patients with relapsed/refractory multiple myeloma","authors":"James A. Davis, Jordan Snyder, Mikhaila Rice, Donald C. Moore, Christopher Cahoon, Kelley Julian, Charlotte B. Wagner, Katelynn Granger, Kimberly M. Green, Shebli Atrash, Hailey Hill, Jessica McElwee, Grace Elsey, Jack Khouri, Joslyn Rudoni, Zahra Mahmoudjafari, Victoria R. Nachar","doi":"10.1038/s41408-025-01222-y","DOIUrl":"https://doi.org/10.1038/s41408-025-01222-y","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"52 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143538368","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":"Risk factors for CAR T-cell manufacturing failure and patient outcomes in large B-cell lymphoma: a report from the UK National CAR T Panel","authors":"Vaishali Dulobdas, Amy A. Kirkwood, Fabio Serpenti, Brijesh Gautama, Aikaterini Panopoulou, Amrith Mathew, Sumantha Gabriel, Ram Malladi, Jessica Pealing, Denise Bonney, Emma Nicholson, Caroline Besley, Sara Ghorashian, Andrea Kuhnl, Elizabeth Davies, Jackie Chappell, Anne Black, Tobias Menne, Maeve A. O’Reilly, Robin Sanderson, Sridhar Chaganti","doi":"10.1038/s41408-025-01225-9","DOIUrl":"https://doi.org/10.1038/s41408-025-01225-9","url":null,"abstract":"<p>CAR T-cell manufacturing failure (MF) is a situation where the manufacturing process fails to yield a product or results in one which is out-of-specification (OOS). We conducted a multicentre retrospective review of factors contributing to MF and patient outcomes. Of 981 large B-cell lymphoma (LBCL) patients approved for CAR T-cell therapy, 38 (3.87%) had MF. Eleven patients received delayed infusion with a product in-specification (delayed-infused) following 21 remanufacturing attempts. OOS product was infused in 13 (OOS-infused), and 14 were not infused. For comparison, we included 38 LBCL controls without MF; 29 received infusion (controls-infused). Prior bendamustine was the only baseline variable associated with MF risk, largely due to therapy within 6 months; 23.7% MF vs 0% controls (<i>P</i> = 0.0029). Overall survival (OS) and progression-free survival (PFS) were not significantly different for infused patients, with 1-year OS (PFS) of 52.8% (46.2%), 46.8% (24.2%) and 68.4% (41.4%) for OOS-infused, delayed-infused and controls-infused respectively (PFS HR OOS-infused vs controls-infused 1.41, <i>P</i> = 0.40; delayed-infused vs controls-infused 1.64, <i>P</i> = 0.25; and OOS-infused vs delayed-infused 0.86, <i>P</i> = 0.76). CRS, ICANS and cytopenias were not significantly different between cohorts. Outcomes for OOS-infused LBCL patients following MF are encouraging. Remanufacturing led to infusion of a product in-specification in around 50% and may be an option for patients where a suitable OOS product is not available.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"86 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143538395","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":"Myeloid neoplasms with MYC-positive double minutes: towards recognition as a distinct entity","authors":"Isolde Summerer, Wencke Walter, Manja Meggendorfer, Torsten Haferlach, Frank Dicker, Stephan Hutter, Niroshan Nadarajah, Anna Stengel, Constanze Kuehn, Wolfgang Kern, John M. Bennett, Claudia Haferlach, Christian Pohlkamp","doi":"10.1038/s41408-025-01244-6","DOIUrl":"https://doi.org/10.1038/s41408-025-01244-6","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"84 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143532806","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":"Myeloid neoplasms with PHF6 mutations: context-dependent genomic and prognostic characterization in 176 informative cases","authors":"Saubia Fathima, Ali Alsugair, Rong He, Abhishek A. Mangaonkar, Kebede H. Begna, Animesh Pardanani, Cinthya J. Zepeda Mendoza, Kaaren K. Reichard, Naseema Gangat, Ayalew Tefferi","doi":"10.1038/s41408-025-01231-x","DOIUrl":"https://doi.org/10.1038/s41408-025-01231-x","url":null,"abstract":"<p>Recent reports suggest a favorable prognosis for <i>PHF6</i> mutation (<i>PHF6</i>^MUT) in chronic myelomonocytic leukemia (CMML) and unfavorable in acute myeloid leukemia (AML). We accessed 176 consecutive patients with a spectrum of myeloid neoplasms with <i>PHF6</i>^MUT, including AML (<i>N</i> = 67), CMML (<i>N</i> = 49), myelodysplastic syndromes (MDS; <i>N</i> = 36), myeloproliferative neoplasms (MPN; <i>N</i> = 16), and MDS/MPN (<i>N</i> = 8). <i>PHF6</i> mutations were classified as nonsense (43%) or frameshift (30%) with the PHD2 domain being the most frequently (64%) affected region. Median follow-up was 25 months with 110 (63%) deaths and 44 allogenic transplants. Our top-line observations include (a) a distinctly superior overall survival (OS; 81 vs. 18 months; <i>p</i> &lt; 0.01) and blast transformation-free survival (BTFS; “not reached” vs. 44 months; <i>p</i> &lt; 0.01) in patients with CMML vs. those with other myeloid neoplasms, (ii) a higher than expected frequency of isolated loss of Y chromosome, in the setting of CMML (16% vs. expected 6%) and MDS (8% vs expected 2.5%), (iii) a significant association, in MDS, between <i>PHF6</i>^MUT variant allele fraction (VAF) &gt; 20% and inferior OS (HR 3.0, 95% CI 1.1–8.1, multivariate <i>p</i> = 0.02) as well as female gender and inferior BTFS (HR 26.8, 95% CI 1.9–368.3, multivariate <i>p</i> = 0.01), (iv) a relatively favorable median post-transplant survival of 46 months. Multivariable analysis also identified high-risk karyotype (HR 5.1, 95% CI 1.2–20.9, <i>p</i> = 0.02), and hemoglobin &lt;10 g/dL (HR 2.7, 95% CI 1.0–7.2, <i>p</i> = 0.04), as independent predictors of inferior OS in patients with MDS. The current study provides disease-specific information on genotype and prognosis of <i>PHF6</i>-mutated myeloid neoplasms.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"33 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143526039","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":"Impact of baseline genetic profile and treatment on outcome and hematological toxicity in CEBPA-mutated AML","authors":"Francesco Mannelli, Matteo Piccini, Marco Frigeni, Giacomo Gianfaldoni, Sara Bencini, Silvia Salmoiraghi, Barbara Scappini, Benedetta Peruzzi, Roberto Caporale, Gaia Ciolli, Francesca Crupi, Laura Fasano, Elisa Quinti, Andrea Pasquini, Jessica Caroprese, Fiorenza Vanderwert, Giada Rotunno, Fabiana Pancani, Leonardo Signori, Danilo Tarantino, Chiara Maccari, Francesco Annunziato, Orietta Spinelli, Paola Guglielmelli, Alessandro Rambaldi, Alessandro M. Vannucchi","doi":"10.1038/s41408-025-01224-w","DOIUrl":"https://doi.org/10.1038/s41408-025-01224-w","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"84 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143526269","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":"Multiple myeloma as a challenging multidimensional random process: a data-driven web-based application for treatment selection","authors":"Alexander S. Luchinin, Tigran G. Gevorkyan, Anastasia A. Semenova","doi":"10.1038/s41408-025-01238-4","DOIUrl":"https://doi.org/10.1038/s41408-025-01238-4","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"130 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143517917","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}