Blood Cancer Journal最新文献

{"title":"Whole body MRI by MY-RADS for imaging response assessment in multiple myeloma","authors":"Christina Messiou, Nuria Porta, Dow-Mu Koh, Angela Riddell, Katherine Downey, James Croft, Leonora Conneely, Georgina Hopkinson, Alina Dragan, Tommy Brown, Simon Stern, Betty Cheung, Charalampia Kyriakou, Pawel Kaczmarek, Kevin Boyd, Charlotte Pawlyn, Jessica Winfield, Martin F. Kaiser","doi":"10.1038/s41408-025-01327-4","DOIUrl":"https://doi.org/10.1038/s41408-025-01327-4","url":null,"abstract":"<p>Minimal residual disease (MRD) testing has underpinned the evaluation and expansion of therapeutic options for patients with multiple myeloma (MM). Imaging is essential for evaluating residual disease status, overcoming sampling errors inherent with other MRD modalities. The accuracy of whole-body MRI (WB-MRI) has led to its incorporation into MM diagnostic imaging guidelines. We report here on the prospective iTIMM trial (image-guided theranostics in MM; NCT02403102), designed to evaluate imaging residual disease using contemporary, functional WB-MRI as per MY-RADS protocol. In iTIMM, 70 MM patients planned to undergo autologous stem cell transplantation ASCT in newly diagnosed MM or at first relapse, underwent WB-MRI before start of induction and at day 100 post-ASCT. Patients with residual disease post-ASCT (RAC2 or higher) had shorter progression-free survival (median 24 months, 95% confidence interval (CI): 19–41 vs. 42 months, 95% CI: 37–not evaluable (NE), log-rank <i>p</i> = 0.013; hazard ratio (HR) 2.09 (95% CI: 1.15–3.78) and overall survival (median 47 months, 95% CI: 30.9–NE vs. NE (95% CI: NE–NE), <i>p</i> = 0.002, HR = 5.45 (95% CI: 1.67–17.87) than those without (RAC1). Imaging response also refined the prognostic association of bone marrow MRD and serological response. Our results support WB-MRI implementation for evaluation of residual disease alongside conventional laboratory-based assessments.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"46 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645588","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":"Age-dependent phenotypic and molecular evolution of pediatric MDS arising from GATA2 deficiency","authors":"Lili Kotmayer, Emilia J. Kozyra, Guolian Kang, Brigitte Strahm, Ayami Yoshimi, Sushree S. Sahoo, Victor B. Pastor, Enrico Attardi, Rebecca Voss, Luca Vinci, Max Kaiser, Michael N. Dworzak, Barbara De Moerloose, Martina Sukova, Jan Starý, Henrik Hasle, Kirsi Jahnukainen, Sophia Polychronopoulou, Krisztián Kállay, Owen P. Smith, Andrea Malone, Shlomit Barzilai Birenboim, Riccardo Masetti, Jochen Buechner, Marek Ussowicz, Paula Kjöllerström, Ivana Bodova, Marko Kavcic, Albert Català, Dominik Turkiewicz, Markus Schmugge, Valerie de Haas, Victoria I. Okhomina, Cristian Sotomayor, Paula Catalán, Claudia Wehr, Ulrich Salzer, Ulrich Germing, Norbert Gattermann, Csaba Bödör, Nathan Gray, Sara Lewis, Akiko Shimamura, Alessandra Giorgetti, Miriam Erlacher, Charlotte M. Niemeyer, Marcin W. Wlodarski","doi":"10.1038/s41408-025-01309-6","DOIUrl":"https://doi.org/10.1038/s41408-025-01309-6","url":null,"abstract":"<p>GATA2 deficiency is an autosomal dominant transcriptopathy disorder with high risk for myelodysplastic syndrome (MDS). To elucidate genotype-phenotype associations and identify new genetic risk factors for MDS, we analyzed 218 individuals with germline heterozygous <i>GATA2</i> variants. We observed striking age-dependent incidence patterns in GATA2-related MDS (GATA2-MDS), with MDS being absent in infants, rare before age 6 years, and steeply increasing in older children. Among 108 distinct <i>GATA2</i> variants (67 novel), null mutations conferred a 1.7-fold increased risk for MDS, had earlier MDS onset compared to other variants (12.2 vs. 14.6 years, <i>p</i> = 0.009) and were associated with lymphedema and deafness. In contrast, intron 4 variants exhibited reduced penetrance and lower risk for MDS development. Analysis of the somatic landscape revealed unique patterns of clonal hematopoiesis. <i>SETBP1</i> mutations occurred exclusively in patients with monosomy 7 and their frequency decreased with age. Conversely, the frequency of <i>STAG2</i> mutations and trisomy 8 increased with age and appeared protective against early development of advanced MDS. Overall, the majority (73.9%) of mutation-positive cases harbored monosomy 7, suggesting it serves as a major driver in malignant progression. Our findings provide evidence for age-appropriate surveillance, and a foundation for genotype-driven risk stratification in GATA2 deficiency.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"12 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144629884","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":"Integrated genomics with refined cell-of-origin subtyping distinguishes subtype-specific mechanisms of treatment resistance and relapse in diffuse large B-cell lymphoma","authors":"Janek S. Walker, Kerstin Wenzl, Joseph P. Novak, Matthew E. Stokes, Melissa A. Hopper, Abigail R. Dropik, Miranda S. Siminski, Allison M. Bock, Vivekananda Sarangi, Maria Ortiz, Nicholas Stong, C. Chris Huang, Matthew J. Maurer, Brian K. Link, Stephen M. Ansell, Thomas M. Habermann, Thomas E. Witzig, Rebecca L. King, Grzegorz Nowakowski, James R. Cerhan, Anita K. Gandhi, Anne J. Novak","doi":"10.1038/s41408-025-01326-5","DOIUrl":"https://doi.org/10.1038/s41408-025-01326-5","url":null,"abstract":"<p>Up to 40% of diffuse large B-cell lymphoma (DLBCL) patients do not experience a durable response to frontline immunochemotherapy, and prospective identification of high-risk cases that may benefit from personalized therapeutic management remains an unmet need. Molecular phenotyping techniques have established a landscape of genomic variants in diagnostic DLBCL; however, these have not yet been applied in large-scale studies of relapsed/refractory DLBCL, resulting in incomplete characterization of mechanisms driving tumor progression and treatment resistance. Here, we performed an integrated multiomic analysis on 228 relapsed/refractory DLBCL samples, including 24 with serial biopsies. Refined cell-of-origin subtyping identified patients harboring GCB and DZsig+ relapsed/refractory tumors in cases with primary refractory disease with remarkably poor outcomes, and comparative analysis of genomic features between relapsed and diagnostic samples identified subtype-specific mechanisms of therapeutic resistance driven by frequent alteration to <i>MYC</i>, <i>BCL2</i>, <i>BCL6</i>, and <i>TP53</i> among additional strong lymphoma driver genes. Tumor evolution dynamics suggest innate mechanisms of chemoresistance are present in many DLBCL tumors at diagnosis, and that relapsed/refractory tumors are primarily comprised of a homogenous clonal expansion with reduced tumor microenvironment activity. Adaptation of personalized therapeutic strategies targeting DLBCL subtype-specific resistance mechanisms should be considered to benefit these high-risk populations.</p><figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"37 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144612872","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":"Fine particulate matter exposure is linked to worse myeloma outcomes in a diverse urban cohort","authors":"Michael Wysota, Kith Pradhan, Sarah Jacobs, Joel Rosiene, Charles Hall, George Downward, Yocheved Halberstam, Nishi Shah, Dean Hosgood, Aditi Shastri","doi":"10.1038/s41408-025-01301-0","DOIUrl":"https://doi.org/10.1038/s41408-025-01301-0","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"31 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144568323","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":"Efficacy and safety of immunosuppressive therapy versus cyclosporine combined with avatrombopag in older adults with severe aplastic anemia: a multicenter prospective study","authors":"Leyu Wang, Lei Ye, Xin Zhao, Miao Chen, Fengkui Zhang, Bing Han","doi":"10.1038/s41408-025-01328-3","DOIUrl":"https://doi.org/10.1038/s41408-025-01328-3","url":null,"abstract":"<p>This trial compared antithymocyte globulin (ATG) + cyclosporine A (CsA) + avatrombopag (AVA) and CsA + AVA in older adults with severe aplastic anemia (SAA). The patients were randomized to receive either ATG + CsA + AVA or CsA + AVA. Of 84 included patients, 42 were treated with ATG + CsA + AVA and 42 with CsA + AVA. With a median follow-up of 13 (0.3–17) months, the objective response rates (ORRs) at 3, 6, and 12 months and the end of follow-up were 53.7%, 65.9%, 80.6%, and 71.4% in the ATG + CsA + AVA group and 61.9%, 73.2%, 77.4%, and 64.3% in the CsA + AVA group, respectively (P &gt; 0.05 at any time point). Three-month ORR was an independent predictor of 6-month complete response rates (P = 0.019). Patients in the ATG + CsA + AVA group showed a higher incidence of adverse events than those in the CsA + AVA group (64.3% vs. 35.7%, P = 0.009). The rates of relapse (P = 0.667), mortality (P = 1.000) and clonal evolution (P = 1.000) were comparable between the groups. The combination of CsA + AVA achieved comparable efficacy with superior safety compared to the combination of ATG + CsA + AVA in older adults newly diagnosed with SAA.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"3 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144565630","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":"Comparable outcomes with 14-, 21-, or standard 28-day venetoclax in the first cycle of azacitidine-venetoclax in untreated acute myeloid leukemia: real-world experience from the Hokkaido Leukemia Net.","authors":"Minoru Kanaya, Masahiro Onozawa, Toshihiro Matsukawa, Naoki Miyashita, Fumiaki Fujii, Shota Yoshida, Jun Nagai, Masayuki Aiba, Daisuke Hidaka, Junichi Hashiguchi, Hajime Senjo, Tetsuyuki Igarashi, Masahiro Chiba, Satoshi Yamamoto, Taku Shimizu, Takashi Ishio, Shota Yokoyama, Ko Ebata, Satoshi Iyama, Tatsuo Oyake, Takeshi Kondo, Takanori Teshima","doi":"10.1038/s41408-025-01324-7","DOIUrl":"10.1038/s41408-025-01324-7","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"118"},"PeriodicalIF":12.9,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12229622/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144558945","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":"Incidental vs. symptomatic diagnosis of follicular lymphoma: implications of earlier detection.","authors":"Suheil Albert Atallah-Yunes, Matthew J Rees, Raphael Mwangi, Robyn L Kuchler, Ahmed Alnughmush, Grzegorz S Nowakowski, Thomas M Habermann, Yucai Wang, Jose C Villasboas Bisneto, Andrew L Feldman, Matthew J Maurer, James R Cerhan, Stephen M Ansell, Thomas E Witzig","doi":"10.1038/s41408-025-01322-9","DOIUrl":"10.1038/s41408-025-01322-9","url":null,"abstract":"<p><p>Follicular lymphoma (FL) is usually diagnosed at an advanced stage when the patient presents with a palpable lymph node or symptoms such as pain and fatigue. However, due to advances in imaging techniques used for many diseases and cancer screening, incidental diagnosis of FL is expected to rise. In this study, we investigated FL disease characteristics and outcomes in patients diagnosed incidentally versus symptomatically, providing insights into what might be detected with multi-cancer early detection tests (MCEDs). We conducted a review of 908 patients with newly diagnosed FL enrolled in the Mayo Clinic component of the Molecular Epidemiology Resource (MER) from 2002 to 2015. We compared disease characteristics and outcomes between the incidental and symptomatic groups. Of the 908 patients, 259 (28.5%) were diagnosed incidentally. The incidental group was more likely to present with early-stage disease (stage I/II: 43.2% vs. 30.6%, p = 0.0003), normal lactate dehydrogenase (LDH) levels (87.2% vs. 80.8%, p = 0.03), and trended towards having lower FLIPI scores (49.8% vs. 42.2%, p = 0.1). However, there were no significant differences in event-free survival (EFS), overall survival (OS) or lymphoma-specific survival (LSS) between the two groups. In conclusion, incidental detection of FL is associated with earlier stages and more favorable disease characteristics. However, this did not translate into improved survival outcomes. Whether even earlier detection of FL using emerging MCEDs translates into improved outcomes remains an open question requiring further investigation.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"116"},"PeriodicalIF":12.9,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12229598/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144558946","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":"Is immune effector cell-associated enterocolitis a CAR T-cell lymphoproliferative disorder?","authors":"Kayra Somay, Hulya Azakli, Joeffrey J Chahine, Thomas M Loughney, Metin Ozdemirli","doi":"10.1038/s41408-025-01319-4","DOIUrl":"10.1038/s41408-025-01319-4","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"117"},"PeriodicalIF":12.9,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12229613/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144558947","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":"Adult AML with NUP98 rearrangements should be stratified into adverse-risk group","authors":"Wusixian Huang, Man Wang, Jundan Xie, Qian Wang, Haiping Dai, Suning Chen, Qinrong Wang","doi":"10.1038/s41408-025-01325-6","DOIUrl":"https://doi.org/10.1038/s41408-025-01325-6","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"27 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144547186","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":"Humoral vaccine responses following Chimeric Antigen Receptor T-cell therapy for hematological malignancies","authors":"Sigrun Einarsdottir, Stephanie Lobaugh, Danny Luan, Marina Gomez-Llobell, Padmapriya Subramanian, Sean Devlin, David Chung, Parastoo B. Dahi, Lorenzo Falchi, Sergio Giralt, Heather Landau, Alexander M. Lesokhin, Richard Lin, Jennifer Lue, Sham Mailankody, M. Lia Palomba, Jae H. Park, Gilles Salles, Michael Scordo, Silvia Escribano-Serrat, Jaime Sanz, Kai Rejeski, Roni Shouval, Saad Usmani, Miguel-Angel Perales, Gunjan Shah, Zainab Shahid","doi":"10.1038/s41408-025-01321-w","DOIUrl":"https://doi.org/10.1038/s41408-025-01321-w","url":null,"abstract":"<p>This single-center, retrospective study analyzed vaccine responses in patients who received post-Chimeric Antigen Receptor (CAR) T-cell therapy vaccination between 2018 and 2024. Vaccinations were administered according to EBMT/CIBMTR recommendations and pathogen-specific IgG responses to 12 vaccine-preventable infections were assessed. Seroprotection was defined by established cut-offs or a significant fold increase in titers. A total of 73 patients that had not received intravenous immunoglobulins within the eight weeks prior to pre- or post titer were included. The median time to vaccination initiation was 13 months (range 6–66) post-CAR T. Pre and post-vaccination titers were available for 49 patients. Pre-vaccination seroprotection was high (&gt; 85%) for tetanus and poliovirus. Among patients not seroprotected prior to vaccination, vaccine response rates were high for tetanus and polio (100%), moderate for diphtheria (75%) and haemophilus influenzae type b (62%), and lower for pertussis (48%), hepatitis A (43%), hepatitis B (44%), and pneumococcal disease (33%). CD19 CAR T recipients had higher pre-vaccination seroprotection rates than BCMA recipients, but vaccine responses did not differ significantly between groups. Pre-vaccination IgA levels were significantly associated with vaccine response, and absolute B-cell counts trended higher among responders (<i>p</i> = 0.054). Our findings highlight the importance of immune reconstitution in vaccine responses post-CAR T.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"41 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144547187","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}