Blood Cancer Journal最新文献

{"title":"Venetoclax and azacitidine in untreated patients with therapy-related acute myeloid leukemia, antecedent myelodysplastic syndromes or chronic myelomonocytic leukemia","authors":"Vinod Pullarkat, Keith W. Pratz, Hartmut Döhner, Christian Recher, Michael J. Thirman, Courtney D. DiNardo, Pierre Fenaux, Andre C. Schuh, Andrew H. Wei, Arnaud Pigneux, Jun-Ho Jang, Gunnar Juliusson, Yasushi Miyazaki, Dominik Selleslag, Martha L. Arellano, Chenglong Liu, Jean A. Ridgeway, Jalaja Potluri, Jovita Schuler, Marina Konopleva","doi":"10.1038/s41408-025-01263-3","DOIUrl":"https://doi.org/10.1038/s41408-025-01263-3","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"95 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143733940","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":"Deep immune cell profiling in blood and bone marrow of early stage monoclonal gammopathy: an iStopMM and ECRIN-M3 collaborative study","authors":"Oihane Pérez-Escurza, Juan Flores-Montero, Jón Þórir Óskarsson, Luzalba Sanoja-Flores, Julio Pozo, Quentin Lécrevisse, Silvia Martín, Elín Ruth Reed, Guðlaug Katrín Hákonardóttir, Stephen Harding, Sigrún Þorsteinsdóttir, Sæmundur Rögnvaldsson, Thorvardur Jon Love, Brian Durie, Sigurdur Yngvi Kristinsson, Alberto Orfao","doi":"10.1038/s41408-025-01255-3","DOIUrl":"https://doi.org/10.1038/s41408-025-01255-3","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"59 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143713051","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":"Double hit & double expressor lymphomas: a multicenter analysis of survival outcomes with CD19-directed CAR T-cell therapy","authors":"Reem Karmali, Geoffrey Shouse, Pallawi Torka, Tamara K. Moyo, Jason Romancik, Stefan K. Barta, Rahul Bhansali, Jonathon B. Cohen, Nirav N. Shah, Joanna Zurko, Vaishalee P. Kenkre, Brian Hess, Deborah M. Stephens, Lindsey Fitzgerald, Thomas Ollila, Bukky Tabiti, Ishan Roy, Shuo Ma, Jane Winter, Barbara Pro, Jonathan Moreira, Alexey V. Danilov, Kevin David, Leo I. Gordon, Narendranath Epperla","doi":"10.1038/s41408-025-01250-8","DOIUrl":"https://doi.org/10.1038/s41408-025-01250-8","url":null,"abstract":"<p>Double-hit (DHL) and double expressor (DEL) DLBCL have poor prognosis with standard therapy but CART may overcome this poor prognostic impact. In this multicenter retrospective study, we sought to confirm this observation by evaluating survival outcomes among patients with relapsed/refractory DHL and DEL treated with CART and evaluate outcomes of relapse post-CART. A total of 408 adult patients with relapsed/refractory DLBCL from 13 academic centers were included based on the availability of DHL and DEL. All 408 patients were included in the DHL (<i>n</i> = 80) vs non-DHL (<i>n</i> = 328) analysis, while 333 patients were included in the analysis of DHL (<i>n</i> = 80) vs DEL (<i>n</i> = 74) vs non (<i>n</i> = 179). On MVA, there were no differences for PFS for DHL vs non-DHL (HR 0.8, 95%CI 0.5–1.3, <i>p</i> = 0.35) or DHL vs DEL vs other (three-way <i>p</i> value, <i>p</i> = 0.5). Response rates and toxicities were similar among groups. Patients with DEL had the highest relapse rates post-CART, while DHL had the worst overall survival after CART relapse. In sum, our data support the notion that CART cell therapy can overcome the poor prognostic impact of DHL and DEL DLBCL in the relapsed/refractory setting. Additionally, patients with DHL that relapse after CART have a very poor prognosis.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"35 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143713048","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":"Correction: The clinical journey of belantamab mafodotin in relapsed or refractory multiple myeloma: lessons in drug development.","authors":"Pralay Mukhopadhyay, Hesham A Abdullah, Joanna B Opalinska, Prani Paka, Eric Richards, Katja Weisel, Suzanne Trudel, Maria-Victoria Mateos, Meletios Athanasios Dimopoulos, Sagar Lonial","doi":"10.1038/s41408-025-01243-7","DOIUrl":"10.1038/s41408-025-01243-7","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"44"},"PeriodicalIF":12.9,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11947437/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143717832","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":"Outcomes in patients with classic Hodgkin lymphoma refractory or intolerant to brentuximab vedotin and anti-PD-1 therapy: a real world analysis from 15 U.S. academic centers","authors":"Timothy J. Voorhees, Eric M. McLaughlin, Pallawi Torka, Jorge Florindez, Na Hyun Kim, Tamara K. Moyo, Heather Reves, Nuttavut Sumransub, Saarang Deshpande, Ashley Rose, Cassandra Duarte, Muhammad Salman Faisal, Showkat Hamid, Suki Subbiah, Sabarish Ayyappan, Lauren Shea, Matt Cortese, Krish Patel, Ajay Major, Hayder Saeed, Jakub Svoboda, Sanjal Desai, Praveen Ramakrishnan Geethakumari, Mehdi Hamadani, Natalie Grover, Narendranath Epperla","doi":"10.1038/s41408-025-01257-1","DOIUrl":"https://doi.org/10.1038/s41408-025-01257-1","url":null,"abstract":"<p>Anti-PD-1 based therapies and brentuximab vedotin (BV) have significantly improved survival in patients with classic Hodgkin lymphoma (cHL) and have been incorporated into earlier lines of therapy. However, there is insufficient data regarding the clinical outcomes in patients who develop refractory disease or who become intolerant of BV and anti-PD-1 therapies (double refractory/intolerant; DR/INT). Here, we evaluated outcomes in patients with DR/INT cHL from 15 US academic medical centers. A total of 173 patients were identified as DR/INT. The median overall survival from the time of cHL diagnosis (OS-1) was 14.8 years (95% CI: 10.9–20.9 years) and the 10-year OS-1 estimate was 62% (95% CI: 52–70%). After accounting for differences in age, patients who underwent autologous stem cell transplant prior to developing DR/INT had significantly longer OS-1 (HR 0.53, 95% CI: 0.29–0.96, <i>p</i> = 0.04). Median OS from time of DR/INT (OS-2) was 7.4 years (95% CI: 4.3-NR) and the 5-year OS-2 estimate was 57% (95% CI: 48-66%). Both anti-PD-1 and BV based therapy rechallenge were effective with median PFS of 237 days (95% CI: 155-357 days) and 183 days (95% CI: 108–273 days), respectively. Finally, advanced therapy options such as CD30 directed chimeric antigen receptor T-cell therapy and allogeneic stem cell transplant after DR/INT were associated with improved OS-2 (<i>p</i> &lt; 0.001). To our knowledge, this represents the largest cohort of patients with DR/INT cHL. OS-2 will serve as a benchmark for future studies aiming to improve survival in DR/INT cHL.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"4 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143713049","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":"Allo-HCT refined ELN 2022 risk classification: validation of the Adverse-Plus risk group in AML patients undergoing allogeneic hematopoietic cell transplantation within the Spanish Group for Hematopoietic Cell Transplantation (GETH-TC)","authors":"Carlos Jiménez-Vicente, Jordi Esteve, Mónica Baile-González, Estefanía Pérez-López, Carmen Martin Calvo, Clara Aparicio, Itziar Oiartzabal Ormategi, Albert Esquirol, Felipe Peña-Muñoz, Sara Fernández-Luis, Inmaculada Heras Fernando, Ana Pilar González-Rodríguez, Alberto López-García, Jose Luis López-Lorenzo, Tamara Torrado, Adolfo Jesús Sáez Marín, Cynthia Acosta Fleytas, Lucía García, Sara Villar, Silvia Filaferro, Pascual Balsalobre, María Jesús Pascual Cascón, María Queralt Salas","doi":"10.1038/s41408-025-01223-x","DOIUrl":"https://doi.org/10.1038/s41408-025-01223-x","url":null,"abstract":"<p>This multicenter retrospective study by GETH-TC validates the prognostic value of the Allo-HCT Refined ELN 2022 risk classification in allografted AML patients. The new classification refines the ELN 2022 risk classification, dividing adverse-risk patients into two subgroups: Adv-Plus (AdvP), including those with complex karyotype, <i>MECOM (EVI1)</i> rearrangement, or <i>TP53</i> mutations/del(17p), and an additional adverse group (Adv*). The study included 651 AML patients treated with at least one line of anthracycline-based induction therapy and in complete remission. According to the Allo-HCT Refined ELN 2022 risk classification, 19.4% (<i>n</i> = 126) patients were classified into the Favorable (Fav) risk, 38.1% (<i>n</i> = 248) into the Intermediate (Int) risk, 27.2% (<i>n</i> = 177) in the Adv* and 15.4% (<i>n</i> = 100) in the AdvP. Outcomes were significantly poorer for patients allocated in the AdvP risk group (5-year OS rate: 32.3%, 5-year LFS rate: 24.3%, both <i>p</i> &lt; 0.001 with the rest of subgroups) and a higher CIR (5-year CIR: 64.3%, <i>p</i> &lt; 0.001). Patients in the Adv* risk group had similar outcomes than patients in the Int risk group (5-year OS rate: 70.2% vs. 66.7%, <i>p</i> = 0.69, 5-year LFS rate: 63.8% vs. 55.9%, <i>p</i> = 0.33). Multivariate analysis confirmed the dismal outcomes for AdvP patients for OS: Hazard Ratio (HR) = 3.05, and LFS: HR = 2.66, both <i>p</i> &lt; 0.001. Our findings validate the Allo-HCT Refined ELN 2022 classification as a robust prognostic tool, particularly highlighting the poor outcomes for the AdvP subgroup.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"16 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143672282","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":"Refining precision prognostics in multiple myeloma: loss of miR-221/222 cluster in CD138+ plasma cells results in short-term progression and worse treatment outcome","authors":"Konstantinos Soureas, Panagiotis Malandrakis, Maria-Alexandra Papadimitriou, Christos Minopoulos, Ioannis Ntanasis-Stathopoulos, Christine-Ivy Liacos, Maria Gavriatopoulou, Efstathios Kastritis, Meletios-Athanasios Dimopoulos, Andreas Scorilas, Margaritis Avgeris, Evangelos Terpos","doi":"10.1038/s41408-025-01248-2","DOIUrl":"https://doi.org/10.1038/s41408-025-01248-2","url":null,"abstract":"<p>The persistence of high relapse rates and therapy resistance continues to challenge the effective management of multiple myeloma (MM). The identification of novel MM-specific molecular markers could ameliorate risk-stratification tools and accurately identify high-risk patients towards personalized prognosis and therapy. miRNA-seq analysis of CD138+ plasma cells (n = 24) unveiled miR-221-3p and miR-222-3p (miR-221/222 cluster) as the most downregulated miRNAs in R-ISS III compared to R-ISS I/II patients. Subsequently, miR-221/222 levels were quantified by RT-qPCR in CD138+ plasma cells of our screening cohort (n = 141), assessing patients’ mortality and disease progression as clinical endpoints. Internal validation was performed by bootstrap analysis, while clinical benefit was estimated by decision curve analysis. Kryukov et al. (n = 149) and Aass et al. (n = 86) served as institutional-independent validation cohorts. Loss of miR-221/222 cluster was strongly associated with patients’ short-term progression and poor overall survival, which was confirmed by Kryukov et al. and Aass et al. validation cohorts. Intriguingly, miR-221/222-fitted multivariate models offered superior risk-stratification within R-ISS staging and risk-based cytogenetics. Moreover, miR-221/222 loss could effectively discriminate optimal 1st-line treatment responders with inferior treatment outcome. Our study identified the loss of miR-221/222 cluster as a powerful independent predictor of patients’ post-treatment progression, ameliorating prognosis and supporting precision medicine in MM.</p><figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"42 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143631388","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":"Characteristics and predictors of central nervous system relapse in newly diagnosed acute promyelocytic leukemia in the era of arsenic: a 13-year monocenter cohort study.","authors":"Bingqian Jiang, Hongyan Tong, Haitao Meng, Wanzhuo Xie, Wenjuan Yu, Jian Huang, Huafeng Wang, Liangshun You, Liping Mao, Min Yang, Jiejing Qian, Yanling Ren, Chunmei Yang, Liya Ma, Jie Jin, Yinjun Lou","doi":"10.1038/s41408-025-01247-3","DOIUrl":"10.1038/s41408-025-01247-3","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"39"},"PeriodicalIF":12.9,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11910538/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143633440","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":"Outcomes and prognostic factors in patients with Burkitt lymphoma/leukemia in adolescents and adults: an experience from hematology cancer consortium","authors":"Akhil Rajendra, Manju Sengar, Anu Korula, Prasanth Ganesan, Hasmukh Jain, Divya K, Prasanna Samuel, Jayachandran Perumal Kalaiyarasi, Gaurav Prakash, M. Joseph John, Rasmi Palassery, Chandran K. Nair, Tanuja Shet, Sushil Selvarajan, Lingaraj Nayak, Parathan Karunakaran, N. A. Fouzia, Om Prakash, Bhausaheb Bagal, Nikita Mehra, Saranya Kumaran, Sridhar Epari, Jayshree Thorat, Venkatraman Radhakrishnan, Aby Abraham","doi":"10.1038/s41408-025-01240-w","DOIUrl":"https://doi.org/10.1038/s41408-025-01240-w","url":null,"abstract":"<p>Treatment of Burkitt Lymphoma/Leukemia (BL/L) in adults has evolved from the use of pediatric inspired regimens (CODOX-M/IVAC, hyper-CVAD, GMALL) to the use of lower intensity EPOCH regimens. The addition of rituximab has led to improvements in overall survival. Survival with these regimens in the real world was shown to be inferior as compared to those found in the prospective trials. In low- and middle-income country (LMIC) settings, unique problems like delays in seeking care, treatment-related toxicities, and treatment abandonment may hamper outcomes. We performed this retrospective multicenter analysis amongst eight centers in India, to study the disease characteristics, treatment patterns, outcomes, and prognostic factors for BL/L. Between 2012–2019, 265 patients were treated at these centers. Common regimens were methotrexate-based (N – 108(40.7%)) and EPOCH-based (N – 103(38.8%)). After a median follow-up of 42 months, 3-year event-free and overall survival were 58% (95% CI: 55–61%) and 66% (95%CI: 63–69%) respectively. In a propensity matched analysis comparing methotrexate-based protocol and EPOCH-based protocol, the EFS and OS were similar with both the protocols. EPOCH based protocol yielded inferior outcomes in patients with bone marrow, and central nervous system involvement. Factors like rituximab incorporation, baseline ECOG PS 0–2, lower serum LDH, early stage(I/II), achievement of complete response (CR) and low/intermediate BL-IPI risk scores were associated with better survival. However, on multivariable analysis, major factor impacting outcome was achievement of CR.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"5 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143627551","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":"Superior preclinical efficacy of co-treatment with BRG1/BRM and FLT3 inhibitor against AML cells with FLT3 mutations","authors":"Warren Fiskus, Christopher P. Mill, Jessica Piel, Mike Collins, Murphy Hentemann, Branko Cuglievan, Christine E. Birdwell, Kaberi Das, Hanxi Hou, John A. Davis, Antrix Jain, Anna Malovannaya, Tapan M. Kadia, Naval Daver, Koji Sasaki, Koichi Takahashi, Danielle Hammond, Patrick K. Reville, Lauren B. Flores, Sanam Loghavi, Xiaoping Su, Courtney D. DiNardo, Kapil N. Bhalla","doi":"10.1038/s41408-025-01251-7","DOIUrl":"https://doi.org/10.1038/s41408-025-01251-7","url":null,"abstract":"<p>Although treatment with standard frontline therapies, including a FLT3 inhibitor (FLT3i) reduces AML burden and achieves clinical remissions, most patients with AML with FLT3 mutation relapse due to therapy-resistant stem/progenitor cells. The core ATPases, BRG1 (SMARCA4) and BRM (SMARCA2) of the canonical (c) BAF (BRG1/BRM-associated factor) complex is a dependency in AML cells, including those harboring FLT3 mutations. We have previously reported that treatment with FHD-286, a BRG1/BRM ATPases inhibitor, induces differentiation and loss of viability of AML stem/progenitor cells. Findings of present studies demonstrate that treatment with FHD-286 induces lethality in AML cells, regardless of sensitivity or resistance to FLT3i. This efficacy is associated with the induction of gene-expression perturbations responsible for growth inhibition, differentiation, as well as a reduced AML-initiating potential of the AML cells. Additionally, co-treatment with FHD-286 and FLT3i exerts superior pre-clinical efficacy against AML cells and patient-derived (PD) xenograft (PDX) models of AML with FLT3 mutations.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143627552","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}