Blood Cancer Journal最新文献

{"title":"Association between diabetic control and progression of monoclonal gammopathy of undetermined significance to multiple myeloma","authors":"Lawrence Liu, Byron Sigel, Mei Wang, Nikhil Grandhi, Martin W. Schoen, Kristen M. Sanfilippo, Kenneth R. Carson, Murali Janakiram, Tzeyu L. Michaud, Graham A. Colditz, Theodore S. Thomas, Su-Hsin Chang","doi":"10.1038/s41408-026-01592-x","DOIUrl":"https://doi.org/10.1038/s41408-026-01592-x","url":null,"abstract":"Diabetes Mellitus (DM) is a risk factor for multiple myeloma (MM), but the relationship between diabetic control and risk of progression to MM in patients with monoclonal gammopathy of undetermined significant (MGUS) is unclear. We conducted a retrospective cohort study of US Veterans diagnosed with MGUS from 10/1/1999-12/31/2023 with prior DM. MGUS and MM diagnoses were confirmed using published natural language processing-assisted models. The exposure was time-varying glycated hemoglobin (HbA1c) > 7% during the follow-up (time of MGUS to MM, death, or being censored on 4/15/2025, whichever occurred first). The outcome was time from MGUS to MM. The association was estimated by multivariable-adjusted hazard ratio (aHR) using a Fine-Gray subdistribution hazard model with death as a competing event, adjusted by inverse probability of treatment weighting (IPTW). Among 11,061 patients with DM, HbA1c > 7% was associated with increased progression risk (aHR 1.15; 95% confidence interval [CI] 1.01–1.32). This association was stronger in the subgroups of Type-2 DM (T2DM) with insulin use (n = 7070; aHR 1.46; 95% CI 1.19–1.78) and non-use (n = 3724; aHR 1.42; 95% CI 1.14–1.77) throughout follow-up. For patients with T2DM and MGUS, poorly controlled diabetes may be associated with progression risk to MM. Future studies are needed to confirm this relationship.","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"91 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148693479","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":"Venetoclax, azacitidine, and G-CSF targeting molecular residual disease in CBFB::MYH11 AML resistant to high-dose cytarabine.","authors":"Ting Shi, Ruicong Xue, Jing Jia, Menghan Liu, Yalan Zhou, Chuanying Geng, Yuan Jian, Ying Tian, Yanru Zhang, Wenjing Li, Lefu Huang, Huixing Zhou, Aijun Liu, Wen Gao, Wenming Chen, Yun Leng, Yin Wu, Hong-Hu Zhu","doi":"10.1038/s41408-026-01588-7","DOIUrl":"10.1038/s41408-026-01588-7","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"16 1","pages":""},"PeriodicalIF":13.8,"publicationDate":"2026-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13424342/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148629415","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":"Luvometinib in recurrent/refractory pediatric Langerhans cell histiocytosis: a single-arm, multi-center, phase 2 trial","authors":"Rui Zhang, Rong Liu, Jie Yu, Jianpei Fang, Ju Gao, Aiguo Liu, Sixi Liu, Wei Liu, Xuequn Luo, Yongmin Tang, Jian Ge, Xingli Wang, Zhuli Wu, Xin Huang, Zhen Wei, Yang Zheng","doi":"10.1038/s41408-026-01585-w","DOIUrl":"https://doi.org/10.1038/s41408-026-01585-w","url":null,"abstract":"Although mitogen-activated protein kinase pathway inhibitors have been approved for adult histiocytic neoplasms, none has been approved for pediatric Langerhans cell histiocytosis (LCH). This single-arm, open-label, phase 2 trial (NCT05997602) evaluated the efficacy and safety of luvometinib, a MEK1/2 inhibitor, in recurrent/refractory pediatric LCH, regardless of genotypes. Primary endpoint was independent review committee-assessed objective response rate (ORR) per positron emission tomography response criteria (PRC). Between September 21, 2023, and February 19, 2025, 46 patients were enrolled. The median follow-up was 15.1 months (range, 6.0–22.7). Among 42 patients in efficacy analysis set, the ORR and disease control rate assessed by IRC per PRC were 90.5% (95% CI, 77.4–97.3) and 95.2% (95% CI, 83.8–99.4), respectively. Based on Histiocyte Society Evaluations and Treatment guidelines, investigator-assessed ORR was 97.8% (95% CI, 88.5–99.9) and 12-month progression-free survival rate was 97.8% (95% CI, 85.6–99.7). Variant allele frequency of MAPK pathway mutations in cell-free DNA became undetectable in 78.9% (15/19) of patients. Grade ≥ 3 treatment-related adverse events (TRAEs) were reported in 4 (8.7%) patients. No TRAE led to treatment discontinuation. In the first prospective genotype-agnostic phase 2 study in pediatric LCH, luvometinib demonstrated profound response in the recurrent/refractory setting across diverse genotypes with an acceptable safety profile. Trial registration: This study was registered with ClinicalTrials.gov on August 8, 2023 (Identifier: NCT05997602).","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"40 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148612665","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":"Toward more accurate adverse event attribution in multiple myeloma clinical trials.","authors":"Manisha Bhutani, Peter M Voorhees","doi":"10.1038/s41408-026-01590-z","DOIUrl":"10.1038/s41408-026-01590-z","url":null,"abstract":"<p><p>Adverse event (AE) attribution and reporting in multiple myeloma (MM) clinical trials rely on frameworks developed for cytotoxic chemotherapy that are poorly suited to the modern therapeutic landscape. This review identifies systematic limitations at every stage of AE reporting and proposes a comprehensive reform framework tailored to MM. Errors begin at AE identification and accumulate through each subsequent stage - terminology classification, severity grading, and causality attribution. The five-tier causality attribution system produces inconsistent and often uninformative assessments, and current grading frameworks cannot adequately characterize novel toxicities or capture the cumulative burden of AEs that wax and wane with disease control and treatment response. These challenges are amplified in MM, where older patients with disease-related symptom burden, combination regimens with overlapping toxicities, continuous treatment until progression, and racial differences in baseline physiology create attribution complexities that existing systems cannot address. We propose transitioning to a simplified two-tier attribution system, mandating comprehensive baseline assessments with longitudinal reassessment to capture dynamic changes with treatment response, integrating patient-reported outcomes with MM-specific symptom items, adopting objective criteria for determining clinical significance of laboratory abnormalities, implementing longitudinal toxicity analysis methods, enforcing standardized reporting requirements, and developing AI-based decision support tools. These reforms aim to improve consistency, accuracy, and clinical relevance of safety data in MM trials, ultimately supporting better patient care and more informed regulatory decisions.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"16 1","pages":""},"PeriodicalIF":13.8,"publicationDate":"2026-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13588798/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149001220","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":"Fixed-dose inotuzumab ozogamicin (INO) combined with Vincristine and Prednisone (VP) regimen as induction therapy for newly diagnosed B-cell acute lymphocytic leukemia (B-ALL): a multi-center, open-label, single-arm, prospective clinical study.","authors":"Lei Ju, Sifeng Gao, JiLei Zhang, Yuyu Liu, Qiang Song, Lin Dong, Yan Guo, Xuemei Qin, Jie Zou, Chenglu Yuan, Yan Shi, Dexiao Kong, Kaifeng Wang, Qiang Li, Donglin Yang, Qingliang Teng, Erlie Jiang, Jun Peng, Guanchen Bai","doi":"10.1038/s41408-026-01589-6","DOIUrl":"10.1038/s41408-026-01589-6","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"16 1","pages":""},"PeriodicalIF":13.8,"publicationDate":"2026-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13415782/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148598927","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":"Impact of extramedullary plasmacytoma on outcomes in patients with relapsed refractory multiple myeloma treated with talquetamab: U.S. Myeloma Immunotherapy Consortium","authors":"Aimaz Afrough, Oren Pasvolsky, Andrew J. Portuguese, Saurabh Zanwar, Mahmoud R. Gaballa, Aishwarya Sannareddy, Danai Dima, Utkarsh Goel, Hossam M. Ali, Kelley Julian, Andre De Menezes Silva Corraes, Murali Janakiram, Scott Goldsmith, James A. Davis, Kimberly Green, Noa Biran, Lindsay Fogel, Eli Zolotov, Megan M. Herr, Hamza Hassan, Leyla Shune, Jeries Kort, Christina Ye, Susan Bal, Luciano J. Costa, Laura Joiner, Samer AL Hadidi, Adeel M. Khan, Shahzad Raza, Faiz Anwer, Jack Khouri, Rahul Banerjee, Raffaela Cassano Cassano, Surbhi Sidana, Masooma Shifa Rana, Lekha Mikkilineni, Hitomi Hosoya, Shebli Atrash, Christopher Ferreri, Cindy Varga, Douglas W. Sborov, Shonali Midha, Omar Nadeem, Ariel Grajales-Cruz, Rachid Baz, Brandon Blue, Ciara Freeman, Frederick L. Locke, Krina K. Patel, Yi Lin, Shaji K. Kumar","doi":"10.1038/s41408-026-01565-0","DOIUrl":"https://doi.org/10.1038/s41408-026-01565-0","url":null,"abstract":"Talquetamab, a GPRC5D-targeting bispecific antibody, shows promising activity in relapsed-refractory multiple myeloma. However, real-world outcomes in patients with extramedullary disease (EMD) remain poorly described. We studied 360 patients treated with talquetamab at 15 U.S. centers by Dec 2024. Soft tissue plasmacytomas (STP) were classified as EMD (not contiguous with bone) or paraskeletal disease (PSD; contiguous with bone). If both were present, patients were assigned to the EMD category. Of those, 97 (27%) had EMD, 22 (6%) had PSD, and 241 (67%) had No-STP. Median follow-up was 12.8 months. Overall response rates were 68% in EMD, 63% in PSD, and 65% in No-STP (p = 0.8). Median progression-free survival was 4.3, 4.5, and 7.8 months, respectively (p = 0.009), and median overall survival was 10.3 months, 13.0 months, and not reached (p = 0.070). These findings demonstrate that EMD and PSD are associated with preserved response rates but shorter PFS and OS with talquetamab. While systemic markers of tumor burden and inflammation—particularly lactate dehydrogenase (LDH) and ferritin—emerged as independent predictors of inferior PFS, elevated LDH also retained independent prognostic significance for OS. While EMD was associated with shorter PFS and OS on univariate analysis, it did not retain independent prognostic significance on multivariable analysis, suggesting that inferior outcomes are driven by aggressive disease biology rather than by lesion anatomy alone. These findings are hypothesis-generating and require prospective validation. Talquetamab retains clinical utility in this high-risk population with EMD, though outcomes are suboptimal with a need for further refinement of treatment approaches.","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"14 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148612669","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":"Pre-transplant molecular international prognostic scoring system score reduction is not associated with improved outcomes in myelodysplastic syndromes.","authors":"Masataka Sakashita, Junya Kanda, Keiichiro Hattori, Nobuhiro Hiramoto, Nobuhiro Kanemura, Senji Kasahara, Mitsumasa Watanabe, Satoshi Yoshihara, Michiko Ichii, Nobuo Sezaki, Masaaki Tsuji, Takeshi Maeda, Ko Sasaki, Toshiyuki Kitano, Yasuko Miyahara, Tadakazu Kondo, Mamiko Sakata-Yanagimoto, Akifumi Takaori-Kondo, Seishi Ogawa, Yasuhito Nannya","doi":"10.1038/s41408-026-01586-9","DOIUrl":"10.1038/s41408-026-01586-9","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"16 1","pages":""},"PeriodicalIF":13.8,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13408187/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148598978","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":"Olverembatinib plus immunotherapy versus olverembatinib plus allogeneic hematopoietic cell transplantation in adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia.","authors":"Chenying Li, Hongyan Tong, Huafeng Wang, Yanling Ren, Jiejing Qian, Xinping Zhou, Gaixiang Xu, Liangshun You, Liping Mao, Haitao Meng, Wenjuan Yu, Xingnong Ye, Min Yang, Jian Huang, De Zhou, Wanzhuo Xie, Jie Jin, Yinjun Lou","doi":"10.1038/s41408-026-01591-y","DOIUrl":"10.1038/s41408-026-01591-y","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"16 1","pages":""},"PeriodicalIF":13.8,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13408405/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148598966","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":"Selinexor for myelofibrosis or a drug in search of a disease?","authors":"Naseema Gangat, Ayalew Tefferi","doi":"10.1038/s41408-026-01587-8","DOIUrl":"10.1038/s41408-026-01587-8","url":null,"abstract":"<p><p>Selinexor for the Treatment of Myelofibrosis. SVR35- spleen volume reduction ≥ 35%, TSS- total symptom score.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"16 1","pages":""},"PeriodicalIF":13.8,"publicationDate":"2026-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13401593/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148590412","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":"Linvoseltamab outcomes by frailty status in patients with relapsed/refractory multiple myeloma: a subgroup analysis of the LINKER-MM1 study","authors":"Hira Mian, Naresh Bumma, Joshua Richter, Mina Awad, Timothy J. Inocencio, C. Akunna Otele, Megan Seraphin, Tito Roccia, Glenn S. Kroog, Tanya M. Wildes","doi":"10.1038/s41408-026-01572-1","DOIUrl":"https://doi.org/10.1038/s41408-026-01572-1","url":null,"abstract":"There is a paucity of data in frail patients with multiple myeloma (MM) receiving bispecific antibodies (bsAbs). In LINKER-MM1 (NCT03761108), linvoseltamab (human BCMA×CD3 bsAb) demonstrated high response rates and a generally manageable safety profile in relapsed/refractory (RR)MM. This LINKER-MM1 post hoc analysis evaluated outcomes of linvoseltamab 200 mg in patients categorized for frailty using the International Myeloma Working Group Frailty Proxy (IMWG-FP), Simplified Frailty Score (SFS), and patient-reported frailty phenotype (PRFP). As of July 23, 2024, 117 patients received linvoseltamab (median follow-up: 21.3 months). The percentage of frail patients varied by measure: IMWG-FP, 27.4%; SFS, 26.5%; and PRFP, 20.5% (8/4 patients were excluded due to missing IMWG-FP/PRFP scores, respectively). Objective response rates in non-frail and frail groups, respectively, were: IMWG-FP, 72.7% and 65.6%; SFS, 69.8% and 74.2%; PRFP, 73.0% and 62.5%. Incidence of Grade 3/4 treatment-emergent adverse events was consistent across non-frail and frail groups (IMWG-FP: 72.7% and 75.0%; SFS%: 73.3% and 74.2; PRFP: 74.2% and 70.8, respectively). Clinical outcomes were consistent in non-frail and frail groups, indicating a favorable risk–benefit profile for linvoseltamab in the frail patients with RRMM from LINKER-MM1. Continued evaluation of linvoseltamab in frail patients with RRMM in clinical trials and real-world settings is warranted.","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"13 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148587267","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}