Blood Cancer Journal最新文献

{"title":"Genomic and immunogenomic profiling of extramedullary acute myeloid leukemia reveals actionable clonal branching and frequent immune editing","authors":"Clément Collignon, Tucker Hansen, Colin Hercus, Marianna B. Ruzinova, Gabrielle Roth Guepin, Caroline Bonmati, Marie Thérèse Rubio, Pierre Feugier, Mélanie Gaudfrin, Hervé Sartelet, Marion Divoux, Marc Muller, Sharon Heath, Geoffrey L. Uy, David H. Spencer, David Y. Chen, Simona Pagliuca, Francesca Ferraro","doi":"10.1038/s41408-025-01345-2","DOIUrl":"https://doi.org/10.1038/s41408-025-01345-2","url":null,"abstract":"<figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"187 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144840028","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":"GPRC5D Bispecific antibody talquetamab-induced dizziness-ataxia syndrome, clinical presentation and management- a case series","authors":"Murali Janakiram, Lawrence Liu, Scott Goldsmith, Azra Borogovac, Sarah Lee, Nitya Nathwani, Michael Rosenzweig, Firoozeh Sahebi, Myo Htut","doi":"10.1038/s41408-025-01342-5","DOIUrl":"https://doi.org/10.1038/s41408-025-01342-5","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"19 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144819214","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":"Subsets of follicular lymphoma 3B have divergent outcomes: results from the prospective multicenter MER and LEO cohorts","authors":"Patrizia Mondello, Brianna Negaard, Andrew L. Feldman, Brian K. Link, Carla Casulo, Dai Chihara, David Russler-Germain, Jason Romancik, Caitlin Gribbin, Sara Haddadi, Eric Mou, Ivana N. Micallef, Patrick B. Johnston, Joseph Novak, Yucai Wang, Rebecca L. King, Anne J. Novak, Thomas M. Habermann, Peter Martin, Brad Kahl, Grzegorz S. Nowakowski, Loretta J. Nastoupil, James R. Cerhan, Christopher R. Flowers, Izidore S. Lossos, Richard W. Burack, Matthew J. Maurer, Stephen M. Ansell","doi":"10.1038/s41408-025-01347-0","DOIUrl":"https://doi.org/10.1038/s41408-025-01347-0","url":null,"abstract":"<p>Follicular lymphoma (FL) 3B is considered an aggressive lymphoma, however recent studies have challenged this paradigm. Additional controversy involves the clinical implication of pure FL3B (FL3Bp) vs FL3B with concurrent diffuse large B cell lymphoma (DLBCL) (FL3Bc). To address these questions, we performed a pooled study of the MER and LEO cohorts comparing 464 newly diagnosed, R-CHOP-treated patients with FL1-2 (<i>n</i> = 216), FL3A (<i>n</i> = 170), FL3B (<i>n</i> = 78) and 739 DLBCL. Among FL3B patients, 19 (24%) had FL3Bc and 59 (76%) FL3Bp. Baseline characteristics and outcomes were similar between the two FL3B subtypes. Compared to FL1-3A, FL3B showed similar clinical features, except for a lower tumor burden. After R-CHOP, FL1-2 patients had an inferior event-free survival (EFS) than those with FL3B, whereas there was no difference with FL3A. Survival was similar across the FL grades. Although FL1-2 patients failed to achieve EFS24 more frequently than FL3B and FL3A, FL3B patients who failed EFS24 had three-fold higher risk of subsequent mortality than other FLs. At 5-year follow-up FL3B patients had twice the risk of relapse with an aggressive subtype than those with FL1-2 and FL3A. Compared to DLBCL, FL3B patients had more favorable clinical features, but similar outcomes to GCB subtype. Our data suggest that most FL3B have a good outcome, while a subset has an aggressive behavior.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"113 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144797044","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":"Value of serum-free light chain measurements in response and progression assessment in multiple myeloma with monoclonal protein measurable by electrophoresis","authors":"Elham Askari, Angela Dispenzieri, Francis K. Buadi, Suzanne R. Hayman, Morie A. Gertz, Prashant Kapoor, Wilson Gonsalves, Taxiarchis Kourelis, David Dingli, Rahma Warsame, Nelson Leung, Yi Lin, Eli Muchtar, Joselle Cook, Moritz Binder, Nadine Abdallah, Lisa Hwa, Miriam Hobbs, Amie Fonder, David Murray, Robert. A. Kyle, S. Vincent Rajkumar, Shaji K. Kumar","doi":"10.1038/s41408-025-01340-7","DOIUrl":"https://doi.org/10.1038/s41408-025-01340-7","url":null,"abstract":"<p>Uniform assessment of response to treatment is crucial to managing multiple myeloma (MM) and developing new therapies. Measurement of monoclonal protein forms the cornerstone of disease assessment in MM. According to International Myeloma Working Group (IMWG) guidelines, serum-free light chain (sFLC) is included in MM response assessment in patients with no measurable disease by electrophoresis and to define stringent complete response. We retrospectively analyzed the independent value of serial FLC on response and progression assessments in 839 patients with measurable disease by sFLC as well as serum/urine electrophoresis. A significant association was observed between sFLC and electrophoretic responses during initial therapy and at best response (<i>p</i> &lt; 0.001). This study revealed comparable percentage changes in serial dFLC and urine M-protein, with parallel trends (<i>p</i> &lt; 0.001) and strong correlations (<i>r</i> 0.55–0.79, <i>p</i> &lt; 0.001). The response was detected earlier by sFLC (1.1 months, 95% CI 1.06–1.17), and sFLC ≥ PR after two cycles of induction demonstrated a strong predictive value for subsequent electrophoresis responses (OR 9.33, <i>p</i> &lt; 0.001). Following induction, no difference in PFS was observed between very good partial response (VGPR) as determined by sFLC, sPEP, and uPEP (<i>p</i> = 0.538). The median second-PFS for patients with only sFLC-progression disease (PD) was similar to those with urine M-protein PD with or without sFLC-PD (HR 1.28, 95% CI 0.77–2.13, <i>p</i> 0.334). However, the median overall survival from the first relapse was significantly better for patients with only sFLC-PD (HR 1.87, 95% CI 1.07–3.27, <i>p</i> 0.03). Among patients with PD, 12% had sFLC as the only detectable tumor marker at the time of second-line therapy. This study supports the incorporation of serial sFLC measurements for monitoring response and progression in MM, even in patients with electrophoretic measurable disease, and further advocates replacing 24-h urine with serial sFLC in response assessment.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"11 1","pages":"133"},"PeriodicalIF":12.8,"publicationDate":"2025-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144796865","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":"Neutrophil-to-Lymphocyte ratio as surrogate for JAK2V617F suppression and event-free survival in polycythemia vera.","authors":"Tiziano Barbui,Arianna Ghirardi,Victoria Empson,Francesca Fenili,Giuseppe Gaetano Loscocco,Annalisa Condorelli,Alessandra Iurlo,Daniele Cattaneo,Elena Rossi,Valerio De Stefano,Paola Guglielmelli,Christoph Klade,Heinz Gisslinger,Alessandro Rambaldi,Joseph M Scandura,Alessandro Maria Vannucchi","doi":"10.1038/s41408-025-01317-6","DOIUrl":"https://doi.org/10.1038/s41408-025-01317-6","url":null,"abstract":"Chronic systemic inflammation is a key driver of polycythemia vera (PV) progression, but the immunomodulatory effects of current treatments remain poorly defined. The neutrophil-to-lymphocyte ratio (NLR) is an accessible biomarker of systemic inflammation proven in other contexts, but its role in monitoring PV disease activity has not been established. Using data from three of the largest PV clinical trials, we evaluated the effects of PV therapies on NLR and its relationship with molecular response and clinical outcomes. In 404 hematocrit-controlled patients from the ECLAP study, hydroxyurea (HU) failed to significantly lower NLR (p = 0.11) due to the parallel declines in ANC and ALC. Neither leukocyte counts nor NLR were significantly reduced by phlebotomy in ECLAP patients treated without cytoreductive therapy. In contrast, the Low-PV study showed that while phlebotomy tended to increase NLR, low-dose ropeginterferon alfa-2b (Ropeg) significantly reduced NLR (-18.2% and -36.3% in patients with low and high baseline NLR, respectively) by suppressing ANC rather than lymphocytes. NLR reduction correlated with the primary Low-PV endpoint (p = 0.021) and reduction of JAK2 variant allele frequency (VAF) [1]. The PROUD-PV/CONTINUATION-PV study confirmed the superior effect of Ropeg over HU, with a significantly greater NLR reduction at 60 months (-56.5% versus -33.6%, respectively, p = 0.019) in patients with high baseline NLR. Moreover, NLR reduction was associated with decreased JAK2V617F VAF (p < 0.0001) and improved event-free survival (p = 0.010). These findings identify NLR as a dynamic biomarker of treatment response and prognosis in PV and support its incorporation into routine monitoring.","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"27 1","pages":"132"},"PeriodicalIF":12.8,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144791932","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":"Co-occurrence of clonally related follicular lymphoma and histiocytic sarcoma","authors":"Sarah Haebe, Debra K. Czerwinski, Anuja Sathe, Susan M. Grimes, Tianqi Chen, Carlos J. Suarez, Brock Martin, Hanlee P. Ji, Ronald Levy, Tanaya Shree","doi":"10.1038/s41408-025-01335-4","DOIUrl":"https://doi.org/10.1038/s41408-025-01335-4","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"58 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144778334","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":"Comprehensive assessment of adverse event profiles associated with bispecific antibodies in multiple myeloma","authors":"Mobina Golmohammadi, Shahzad Raza, Maram Albayyadhi, Hossein Sholehrasa, Jack Khouri, Louis Williams, Doris K. Hansen, Azam Moradi, Xuan Xu, Moath Albliwi, Ali Hajj Ali, Danai Dima, Faiz Anwer, Barry Paul, Majid Jaberi-Douraki","doi":"10.1038/s41408-025-01334-5","DOIUrl":"https://doi.org/10.1038/s41408-025-01334-5","url":null,"abstract":"<p>Bispecific antibodies (BsAbs) have shown promise in the management of relapsed/refractory multiple myeloma (MM). Despite its efficacy, this class of drugs is associated with significant toxicities. In this study, we conducted a pooled analysis of the available clinical trials on BsAbs for the treatment of MM, including full publications and abstracts until April 2025. BsAbs were classified into two groups: B-cell maturation antigen (BCMA), and GPRC5D/FcRH5 BsAbs. Welch’s t-test was performed to compare the safety profiles of each agent. For clustering, we used principal component analysis (PCA). Our study analyzed 22 trials involving 2374 patients with MM from early 2023 to April 2025. Among these, 1276 patients received BCMA BsAbs, 841 treated with GPRC5D/FcRH5 BsAbs, 157 received teclistamab + talquetamab, and 65 patients received a talquetamab + daratumumab, and 35 patients received talquetamab + pomalidomide. The median follow-up for all groups was 11.83 months. Among all-grade hematologic adverse events (AEs), neutropenia occurred in 40.4%, anemia in 39.2%, thrombocytopenia in 21.4%, lymphopenia in 19.2%, infections in 45.8%, and cytokine release syndrome (CRS) in 65%. For grade 3/4 AEs, infections occurred in 20.3%, CRS in 1.5%, neutropenia in 35.2%, anemia in 24.5%%, thrombocytopenia in 13.5%, and lymphopenia in 17.7%. CRS and the need for tocilizumab were significantly less frequent with BCMA BsAbs vs GPRC5D/FcRH5 BsAbs, (P &lt; 0.002). Skillings Mack (Generalized Friedman’s) findings emphasized substantial distinctions between BCMA and GPRC5D/FcRH5×CD3 in both overall and severe grade 3/4 AEs (p ≤ 0.0002). PCA revealed agents with all grades and grade 3/4 showed similar clustering patterns except for three agents. Overall, our findings demonstrated the excellent efficacy on the use of BsAbs in MM; however, these agents have been linked to a unique AE profile. GPRC5D/FcRH5 are associated with less grade 3/4 hematologic toxicity whereas BCMA BsAbs were associated with lower grade 3/4 CRS rates, compared to GPRC5D/FcRH5. These insights are crucial for guiding treatment decisions and developing strategies to improve patient outcomes.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"11 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144763179","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":"Brentuximab vedotin plus chemotherapy for the treatment of front-line systemic anaplastic large cell lymphoma: subgroup analysis of the ECHELON-2 study at 5 years’ follow-up","authors":"Eva Domingo-Domènech, Barbara Pro, Tim Illidge, Steven Horwitz, Lorenz Trumper, Swami Iyer, Ranjana Advani, Nancy L. Bartlett, Jacob Haaber Christensen, Won-Seog Kim, Tatyana Feldman, Ilseung Choi, Giuseppe Gritti, David Belada, Andrei Shustov, Arpad Illes, Pier Luigi Zinzani, Andreas Hüttmann, Marek Trneny, Steven Le Gouill, Deepa Jagadeesh, Jonathan W. Friedberg, Meredith Little, Cassie Dong, Michelle Fanale, Keenan Fenton, Kerry J. Savage","doi":"10.1038/s41408-025-01329-2","DOIUrl":"https://doi.org/10.1038/s41408-025-01329-2","url":null,"abstract":"<p>Trial registration: ClinicalTrials.gov number: NCT01777152</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"14 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144756367","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 clinico-genomic prognostic model for primary myelodysplastic neoplasm in Asia.","authors":"Harinder Gill,Rita Yim,Paul Lee,Xavier Cheng-Hong Tsai,Vivian W K Li,Garret M K Leung,Melissa Ooi,Tsz-Shing Hui,Radha Raghupathy,Lynn Chin,Lester Au,Qi Zhang,Tony K Y Wu,Carmen Y Y Lee,Wee-Joo Chng,Hwei-Fang Tien,Hsin-An Hou,Yok-Lam Kwong","doi":"10.1038/s41408-025-01339-0","DOIUrl":"https://doi.org/10.1038/s41408-025-01339-0","url":null,"abstract":"A personalized prognostic model that takes into account the unique molecular features of primary myelodysplastic neoplasm (MDS) in Asia patients is lacking. Diagnostic clinicopathologic features, cytogenetic changes, and gene mutations of ethnic Asian patients with primary MDS were analyzed. Variables were evaluated for associations with overall survival (OS), leukemia-free survival (LFS), and time to progression to secondary AML (TTP-sAML). Prognostic scores were built as a weighted sum of prognostic variables for each patient. The cohort comprised 1225 patients, with at least one gene mutation identified in 1177 patients (96%). Genomic factors associated with inferior outcomes included monosomy 7, del(5q), and GNAS and TP53 mutations for OS; trisomy 19, del(5q), monosomy 7, and GNAS, PTPN11 and TP53 mutations for LFS; and i(17q), del(5q), and NPM1, NRAS, GNAS, IDH2, SF3B1 and RUNX1 mutations for TTP-sAML. The Asian Prognostic Scoring System (APSS) was determined, stratifying patients into six prognostic risk categories. The APSS, compared with the International Prognostic Scoring System molecular (IPSS-M), showed superior concordance indices (C-indices) for OS (0.73 versus 0.57), LFS (0.72 versus 0.59), and TTP-sAML (0.75 versus 0.65) for this Asian cohort. In conclusion, the APSS enhanced prognostication of primary MDS in Asia.","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"45 1","pages":"128"},"PeriodicalIF":12.8,"publicationDate":"2025-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144756077","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":"Activity of CAR-T cells and bispecific antibodies in multiple myeloma with extramedullary involvement","authors":"Maximilian J. Steinhardt, Christoph Schaefers, Lisa B. Leypoldt, Igor-Wolfgang Blau, Marie Harzer, Xiang Zhou, Christine Riedhammer, Abdulaziz Kamili, Ricardo Kosch, Laura S. Topp, Isabel Molwitz, Nils-Ole Gross-Fengels, Yasmin Fede Melzer, Jule Artzenroth, Maximilian Al-Bazaz, Winfried Alsdorf, Max S. Topp, Johannes Duell, Julia Mersi, Johannes Waldschmidt, Carsten Bokemeyer, Hermann Einsele, K. Martin Kortüm, Katja Weisel, Leo Rasche","doi":"10.1038/s41408-025-01330-9","DOIUrl":"https://doi.org/10.1038/s41408-025-01330-9","url":null,"abstract":"<p>Extramedullary multiple myeloma (EMD) is associated with low response rates, short progression-free survival, and poor prognosis. CAR T cells and bispecific antibodies (bsABs) have shown efficacy in relapsed myeloma, but it remains uncertain whether one T cell redirection strategy should be preferred. We retrospectively analyzed 80 patients with EMD not adjacent to the bone treated with ide-cel, cilta-cel, teclistamab, or talquetamab at three academic centers in Germany. All patients were heavily pretreated, and a high-risk cytogenetic profile was prevalent in &gt;41% of patients. All cohorts had a median of 5 to 7 prior lines of therapy. The vast majority of patients receiving cilta-cel, ide-cel, or teclistamab were BCMA-naive ( &gt;88%). Response rates after CAR T cell infusion were significantly higher (100% with cilta-cel, 82% with ide-cel) than with bsABs (29% for talquetamab, 36% for teclistamab). Complete resolution of EMD was more frequent after CAR T cell therapies (50% and 41%) than after bsABs (16% and 14%). With a median follow-up of 12.2 months, median (m)PFS was not reached in patients that had received cilta-cel; mPFS was 7.3 months after ide-cel and significantly longer for both CAR T products compared to talquetamab or teclistamab (mPFS 4.0 and 2.6 months). Effective debulking therapy prolonged remissions after CAR T cell infusion compared to no debulking or no response to debulking. Visceral and soft tissue manifestations responded significantly less frequently than EMD in other locations. With significantly higher response rates, deeper remissions, and longer mPFS, our retrospective data suggest CAR T cells may provide a meaningful benefit in EMD.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"25 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144737174","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}