Blood Cancer Journal最新文献

{"title":"Impact of extranodal involvement at CAR T-cell therapy on outcomes in patients with relapsed or refractory large B-cell lymphoma-Results from a multicenter cohort study.","authors":"Frederique St-Pierre,Subodh Bhatta,Peter G Doukas,Madeline Jenkin,Kaitlin Annunzio,Alexandra E Rojek,Alyssa Gibson,Yun Kyoung Tiger,Brittany McCall,Khaled Alhamad,Alec Hansen,Juan P Alderuccio,Olutobi Adewale,Keem Patel,Asaad Trabolsi,Izidore S Lossos,Lindsey Fitzgerald,Thomas A Ollila,Matthew J Matasar,Justin Kline,Reem Karmali,Narendranath Epperla","doi":"10.1038/s41408-025-01318-5","DOIUrl":"https://doi.org/10.1038/s41408-025-01318-5","url":null,"abstract":"Extranodal (EN) diffuse large B-cell lymphoma (DLBCL) has been historically associated with inferior survival outcomes compared to nodal DLBCL. However, outcomes of patients with EN DLBCL following chimeric antigen receptor T-cell (CAR-T) therapy are not well established. In this multi-center retrospective cohort study, we evaluated the outcomes of patients with EN DLBCL who underwent CAR-T in the relapsed/refractory (R/R) setting. The primary objective was overall survival (OS), while secondary objectives included progression-free survival (PFS), response rates, and toxicity rates. A total of 218 patients were included in the analysis. The most common sites of EN involvement were skin/soft tissue (25%), bone (22%), and lung (17%). Overall response rate (ORR) and complete response rate (CRR) at first post-treatment evaluation were 62% (n = 127) and 40% (n = 82), respectively. Median follow-up was 3.5 years. Median PFS and OS were 4.0 months (95% CI = 3.1-7.2) and 25.7 months (95% CI = 16.1-51.6), respectively. Cytokine release syndrome (CRS) of any grade occurred in 73% (n = 159) of patients, and 6% (n = 12) had grade ≥ 3 CRS. Immune effector cell-associated neurotoxicity syndrome (ICANS) of any grade occurred in 37% (n = 81) of patients, and 19% (n = 41) developed grade ≥ 3 ICANS. In the multivariable analysis, factors that were independently prognostic of inferior OS were 3 or more lines of therapy prior to CAR-T, bulky disease at the time of CAR-T, hepatobiliary, and pancreas involvement, while refractory disease to the most recent therapy prior to CAR-T was associated with inferior PFS. Future studies should further evaluate outcomes of CAR-T in patients with specific EN sites of involvement that appear to be associated with inferior survival such as the liver and pancreas.","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"25 1","pages":"110"},"PeriodicalIF":12.8,"publicationDate":"2025-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144337469","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":"Guidelines for the testing and reporting of cytogenetic results for risk stratification of multiple myeloma: a report of the Cancer Genomics Consortium Plasma Cell Neoplasm Working Group","authors":"Xinyan Lu, Erica F. Andersen, Rahul Banerjee, Celeste C. Eno, Patrick R. Gonzales, Shaji Kumar, Angela M. Lager, Patricia M. Miron, Trevor Pugh, Fabiola Quintero-Rivera, Virginia C. Thurston, Daynna J. Wolff, Jian Zhao, Rafael Fonseca, Linda B. Baughn","doi":"10.1038/s41408-025-01286-w","DOIUrl":"https://doi.org/10.1038/s41408-025-01286-w","url":null,"abstract":"<p>Fluorescence in situ hybridization (FISH) remains the gold-standard clinical assay to detect genetic abnormalities in multiple myeloma (MM). However, FISH panel design, use of conventional chromosome banding analysis and reporting practices have been reported to vary among laboratories. Therefore, standardization in FISH testing and reporting practices is needed to improve report clarity and avoid misinterpretation. The recommendations in this paper represent a consensus of our Cancer Genomics Consortium Plasma Cell Neoplasm Working Group, comprising a joint panel of cytogenetic laboratory directors and clinical investigators with expertise in the diagnosis, risk stratification, and treatment of multiple myeloma. Prior to developing these consensus recommendations, we performed a full literature review and conducted a survey of 102 oncologists to assess current variations and challenges in MM cytogenetic/FISH testing and reporting. Our guidelines establish best practices for the optimization of FISH panel selection, and recommendations for standardized reporting of cytogenetic results to align with the 2025 International Myeloma Society (IMS)/International Myeloma Working Group (IMWG) Updated Risk Stratification.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"145 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144312199","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 phase 2 study of Daratumumab with Thalidomide and dexamethasone in relapsed and/or Refractory Myeloma (RRMM)","authors":"Chandramouli Nagarajan, Wei-Ying Jen, Melissa Ooi, Sanjay De Mel, Cinnie Soekojo, Sung Soo Yoon, Melinda Tan, Yunxin Chen, Xinhua Li, Yogesh Pokharkar, Shi Ning Tham, Nur Shahidah Binte Hashim, Neha Awasthi, Sarah M. Burkill, Brian Durie, Wee Joo Chng","doi":"10.1038/s41408-025-01296-8","DOIUrl":"https://doi.org/10.1038/s41408-025-01296-8","url":null,"abstract":"<p>Trial Registration: NCT03153036</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"10 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144305172","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 growth response 1 as a key regulator of PD-L1 expression and immune evasion in extranodal NK/T-cell lymphoma","authors":"Ji Yun Lee, Kui-Jin Kim, Woochan Park, Jeongmin Seo, Minsu Kang, Eun Hee Jung, Sang-A Kim, Koung Jin Suh, Ji-Won Kim, Se Hyun Kim, Jeong-Ok Lee, Jin Won Kim, Yu Jung Kim, Keun-Wook Lee, Jee Hyun Kim, Soo-Mee Bang, Tae Min Kim, Jin Ho Paik","doi":"10.1038/s41408-025-01313-w","DOIUrl":"https://doi.org/10.1038/s41408-025-01313-w","url":null,"abstract":"<p>This study investigates the role of Early Growth Response 1 (EGR1) in extranodal natural killer/T-cell lymphoma (ENKTL) and its correlation with PD-L1 expression. Analysis of 62 ENKTL patient samples revealed that high EGR1 expression was linked to PD-L1 positivity, the immune evasion-A subtype, and early-stage disease. Although EGR1 expression was not an independent prognostic factor for overall survival, patients with higher EGR1 levels showed a trend toward better outcomes. In ENKTL cell lines (YT, SNK6), EGR1 positively regulated LMP1 and PD-L1 expression. Knockdown of EGR1 reduced PD-L1 levels, decreased PTEN, increased AKT phosphorylation, and abrogated STAT3 phosphorylation. Conversely, EGR1 overexpression enhanced PD-L1. Treatment with the histone deacetylase inhibitor entinostat upregulated both EGR1 and PD-L1, but this effect was lost in EGR1-depleted cells, indicating EGR1’s necessity for HDAC inhibitor–induced PD-L1 expression. These findings reveal EGR1’s pivotal role in tumor immune modulation and highlight potential combination therapies targeting EGR1, epigenetic regulators, and PD-1/PD-L1 checkpoints.</p><figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"12 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144288408","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":"Sex-associated differences in outcome of patients with acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation.","authors":"Jule Ussmann, Lara Bischof, Donata Backhaus, Dominic Brauer, Jacob Jendro, Georg-Nikolaus Franke, Vladan Vucinic, Marco Herling, Klaus H Metzeler, Maximilian Merz, Uwe Platzbecker, Sebastian Schwind, Madlen Jentzsch","doi":"10.1038/s41408-025-01316-7","DOIUrl":"10.1038/s41408-025-01316-7","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"107"},"PeriodicalIF":12.9,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12163082/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144282255","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":"Daratumumab for PRCA after HCT: study and practical considerations from the EBMT Transplant Complications Working Party","authors":"Nico Gagelmann, Moniek de Witte, Christophe Peczynski, William Boreland, Annoek E. C. Broers, Edgar Jost, Alexander Kulagin, Albert Esquirol, Simona Sica, Jürgen Kuball, Gerardo Errico, Wolfgang Bethge, Johan Maertens, Friedrich Stölzel, Edouard Forcade, Matthew Collin, Matteo Parma, Goda Choi, Nicolaus Kröger, Maria Chiara Di Chio, Maria Chiara Finazzi, Lucia López Corral, Jose Rifón, Alberto Mussetti, Adrian Bloor, Marco Ladetto, Hélène Schoemans, Olaf Penack, Ivan Moiseev, Zinaida Peric","doi":"10.1038/s41408-025-01315-8","DOIUrl":"https://doi.org/10.1038/s41408-025-01315-8","url":null,"abstract":"<p>Pure red cell aplasia (PRCA) is a relevant complication after ABO-mismatched allogeneic hematopoietic cell transplantation (HCT). No standard treatment exists, and practice is heterogenous. In this study, we took advantage of an international collaboration to describe characteristics and outcomes of patients receiving daratumumab for PRCA following first allogeneic HCT. We identified 45 patients meeting these criteria (median patient age, 56 years). The median time from HCT to PRCA was 55 days (IQR, 36–116) and all patients were transfusion-dependent at time of daratumumab start. Daratumumab was first-line treatment in 16 patients (36%), most patients (67%) received daratumumab intravenously, and median time from PRCA diagnosis and daratumumab start was 88 days (IQR, 59–219). Incidence of transfusion independence was 69% (95% confidence interval [CI], 52–80%) at 6 months and 80% (95% CI, 62–90%) at 12 months. Incidences of hemoglobin and reticulocyte recoveries were respectively 56 and 78% at 6 months and 65 and 83% at 12 months. Survival at 12 months was 81%, and of 8 deaths, 7 were GVHD- or infection-related. One death was associated with hemolytic anemia. This is the first international and largest study on the use of daratumumab for PRCA after allogeneic HCT, showing high response rates superior to that reported for other treatments. Seven incidents of severe adverse events (mostly infections) underscore the need for close monitoring, proactive management, and comparative studies to determine the role for daratumumab for PRCA. Last, based on these data and a comprehensive literature review, we provide practical consideration for modern PRCA treatment.</p><figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"51 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144211182","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: Revised free light chain reference intervals enhance risk stratification in monoclonal gammopathy of undetermined significance and reduce overdiagnosis.","authors":"Cecilie Velsoe Maeng, Sæmundur Rögnvaldsson, Thórir Einarsson Long, Christian Brieghel, Emil Hermansen, Carsten Utoft Niemann, Kirsten Grønbæk, Sigurður Yngvi Kristinsson, Sigrún Thorsteinsdóttir","doi":"10.1038/s41408-025-01307-8","DOIUrl":"10.1038/s41408-025-01307-8","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"105"},"PeriodicalIF":12.9,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12117119/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144156718","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":"Observation or treatment for smoldering multiple myeloma? A systematic review and meta-analysis of randomized controlled studies","authors":"Ioannis Ntanasis-Stathopoulos, Charalampos Filippatos, Panagiotis Malandrakis, Efstathios Kastritis, Evangelos Terpos, Meletios-Athanasios Dimopoulos, Maria Gavriatopoulou","doi":"10.1038/s41408-025-01312-x","DOIUrl":"https://doi.org/10.1038/s41408-025-01312-x","url":null,"abstract":"<p>While observation is the current standard of care for smoldering multiple myeloma (sMM), emerging evidence suggests that early therapeutic intervention may delay progression and improve outcomes especially for high-risk patients. A systematic review and meta-analysis of randomized clinical trials was performed according to the PRISMA guidelines in order to evaluate the effect of treatment compared to observation in outcomes of patients with sMM. Five studies (7 articles) involving 844 patients with intermediate or high risk sMM and comparing treatment to observation or placebo were deemed eligible. All studies reported progression-free survival results, with progression defined as time to active myeloma (without high heterogeneity, I² = 42%, <i>p</i> = 0.14). A statistically significant 60% reduced pooled risk for disease progression or death (HR = 0.40, 95%CI:0.29–0.55) was revealed for patients who underwent treatment compared to those who did not. An exploratory sensitivity analysis involving 3 trials with only observation in the control group, revealed a 66% lower risk for disease progression or death (HR = 0.34, 95%CI: 0.21–0.56) for patients in the treatment group compared to the control group. Furthermore, time-to-progression was reported in 3 studies; the pooled effect estimate revealed a statistically significant 58% reduced risk for progression to symptomatic MM (HR = 0.42, 95%CI: 0.29–0.61) for patients who underwent treatment compared to those who did not. Only 2 trials reported mature overall survival outcomes, and the pooled effect estimate showed a 45% lower risk for death (HR = 0.55, 95%CI: 0.37–0.82) for sMM patients who received treatment compared to those on observation. Regarding safety, the odds for serious adverse events for those on treatment was as 3.5 times as high (OR = 3.53, 95%CI: 1.14–10.91) compared to those on observation or placebo. In conclusion, this meta-analysis highlights the significant benefits of early treatment in selected patients with sMM, across key clinical outcomes. However, close monitoring is essential for the management of treatment-related toxicities.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"83 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144145518","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":"Attrition rates and treatment outcomes in multiple myeloma: real-world data over a 40-year period","authors":"Luis Gerardo Rodríguez-Lobato, Anna de Daniel, Arturo Pereira, Carlos Fernández de Larrea, Natalia Tovar, M. Teresa Cibeira, David F. Moreno, Jose Miguel Mateos, Noemí Llobet, Esther Carcelero, Daniel Munárriz, Joan Bladé, Laura Rosiñol","doi":"10.1038/s41408-025-01311-y","DOIUrl":"https://doi.org/10.1038/s41408-025-01311-y","url":null,"abstract":"<p>The treatment landscape of multiple myeloma (MM) has evolved significantly over four decades, driven by novel therapies and optimized supportive care. However, the attrition rate (AR), defined as the proportion of patients who die without advancing to the next line of therapy (LOT) after treatment failure, remains a major challenge. To assess how treatment patterns and outcomes have evolved, we analyzed 1,297 MM patients treated between 1980 and 2020, stratified by diagnosis period and age. ARs declined from 38–55% in the 1980s to 15–20% in 2010–2020, but remained high in older patients, with 46.9% of those over 80 unable to proceed beyond first LOT. While progression-free survival gains were primarily observed in the first LOT (15.8 to 24.1 months, <i>p</i> = 0.001), overall survival (OS) improved across all LOTs and age groups, likely due to more effective salvage therapies and supportive care. Achieving a complete response in first-line therapy was associated with a significant OS benefit (4.5 vs. 1.6 years, <i>p</i> &lt; 0.001), underscoring its importance, as many patients, particularly older ones, are less likely to reach subsequent LOTs. Despite advances in MM treatment, patient loss to attrition remains a challenge, highlighting the need for more effective therapies early in the disease course.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"12 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144136651","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":"Poor prognostic implication of CDKN2 deletion in adult patients with Philadelphia chromosome-positive ALL","authors":"So Yeon Park, Daehun Kwag, Jin Jung, Gi June Min, Sung-Soo Park, Silvia Park, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Chang-Ki Min, Seok-Goo Cho, Yonggoo Kim, Myungshin Kim, Seok Lee, Jae-Ho Yoon","doi":"10.1038/s41408-025-01303-y","DOIUrl":"https://doi.org/10.1038/s41408-025-01303-y","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"45 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144130222","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}