Blood Cancer Journal最新文献

{"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":"https://doi.org/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":"","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":"","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}

{"title":"Real world outcomes with elotuzumab-based therapies for patients with relapsed refractory multiple myeloma: a Mayo Clinic experience","authors":"Ricardo D. Parrondo, Saurav Das, Hanna Sledge, Leif Bergsagel, Rafael Fonseca, Nelson Leung, Prashant Kapoor, Morie Gertz, Francis Buadi, Angela Dispenzieri, Jamie Elliott, Andre Fernandez, Caitlin Flott, Asher A. Chanan-Khan, Vivek Roy, Sikander Ailawadhi","doi":"10.1038/s41408-025-01310-z","DOIUrl":"https://doi.org/10.1038/s41408-025-01310-z","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"20 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144122617","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":"Disease characteristics and monitoring of IDH1/IDH2-mutated acute myeloid leukemia","authors":"Mélinda Dantec, Maxime Dufossé, Augustin Boudry, Elise Fournier, Laure Goursaud, Delphine Lebon, Claire Bories, Isabelle Plantier, Sabine Tricot, Adrien Daniel, Alexandre Willaume, Valérie Coiteux, Céline Berthon, Jean-Pierre Marolleau, Claude Preudhomme, Nicolas Duployez, Laurène Fenwarth","doi":"10.1038/s41408-025-01304-x","DOIUrl":"https://doi.org/10.1038/s41408-025-01304-x","url":null,"abstract":"<figure></figure>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"133 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144130086","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: Targeting of epigenetic co-dependencies enhances anti-AML efficacy of Menin inhibitor in AML with MLL1-r or mutant NPM1.","authors":"Warren Fiskus, Christopher P Mill, Christine Birdwell, John A Davis, Kaberi Das, Steffen Boettcher, Tapan M Kadia, Courtney D DiNardo, Koichi Takahashi, Sanam Loghavi, Michael J Soth, Tim Heffernan, Gerard M McGeehan, Xinjia Ruan, Xiaoping Su, Christopher R Vakoc, Naval Daver, Kapil N Bhalla","doi":"10.1038/s41408-025-01306-9","DOIUrl":"10.1038/s41408-025-01306-9","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"99"},"PeriodicalIF":12.9,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12095577/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144118690","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":"Genomic and transcriptomic determinants of clinical outcomes in patients with AML and DNMT3A mutations","authors":"Sao-Chih Ni, Chi-Yuan Yao, Xavier Cheng-Hong Tsai, Min-Yen Lo, Chien-Yuan Chen, Wan-Hsuan Lee, Chien-Chin Lin, Yuan-Yeh Kuo, Yen-Ling Peng, Mei-Hsuan Tseng, Yu-Sin Wu, Ming-Chih Liu, Liang-In Lin, Ming-Kai Chuang, Bor-Sheng Ko, Ming Yao, Jih-Luh Tang, Feng-Ming Tien, Wen-Chien Chou, Hsin-An Hou, Hwei-Fang Tien","doi":"10.1038/s41408-025-01287-9","DOIUrl":"https://doi.org/10.1038/s41408-025-01287-9","url":null,"abstract":"<p>Acute myeloid leukemia (AML) and <i>DNMT3A</i> mutations (<i>DNMT3A</i>^mut) are considered to carry intermediate risk under the 2022 European LeukemiaNet (ELN-2022) classification in the absence of other co-mutations or cytogenetic abnormalities. However, this group is highly heterogeneous. In this study, the genomic and transcriptomic features influencing outcomes in <i>DNMT3A</i>-mutated AML were examined in a cohort of 884 patients with AML receiving standard chemotherapy. Stratification by <i>NPM1</i> and <i>FLT3</i>-ITD status revealed worse survival among patients with <i>NPM1</i> mutations and wild-type <i>FLT3</i>-ITD (<i>NPM1</i>^mut/<i>FLT3-</i>ITD^wt) than patients in the ELN-2022 favorable risk group. The other three subgroups (<i>NPM1</i>^mut/<i>FLT3-</i>ITD^mut, <i>NPM1</i>^wt/<i>FLT3-</i>ITD^mut, and <i>NPM1</i>^wt/<i>FLT3-</i>ITD^wt) exhibited worse prognoses than patients in the ELN-2022 intermediate risk group. Additionally, the presence of <i>TET2</i>^mut in patients with AML and <i>DNMT3A</i>^mut/<i>NPM1</i>^mut/<i>FLT3</i>-ITD^wt led to reclassification from favorable risk to intermediate risk in the ELN-2022. RNA-sequencing analysis revealed a distinct transcriptomic profile in patients with <i>TET2</i>^mut, highlighting the enrichment of leukemic stem cell signatures and dendritic cell migration, with <i>MMP14</i>, <i>CD200</i>, and <i>CT45A5</i> identified as key differentially expressed genes. In conclusion, co-mutation patterns strongly affected AML outcomes in patients with <i>DNMT3A</i>^mut. Patients with <i>TET2</i>^mut constituted a unique subgroup within the ELN-2022 favorable <i>DNMT3A</i>^mut/<i>NPM1</i>^mut/<i>FLT3</i>-ITD^wt group, characterized by distinct transcriptomic features and an unfavorable prognosis.</p>","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"131 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144097234","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: Novel humanized CD19-CAR-T (Now talicabtagene autoleucel, Tali-cel™) cells in relapsed/ refractory pediatric B-acute lymphoblastic leukemia- an open-label single-arm phase-I/Ib study.","authors":"Gaurav Narula, Swaminathan Keerthivasagam, Hasmukh Jain, Sachin Punatar, Akanksha Chichra, Chetan Dhamne, Prashant Tembhare, Papagudi Ganesan Subramanian, Nikhil Patkar, Minal Poojary, Anant Gokarn, Sumeet Mirgh, Nishant Jindal, Albeena Nisar, Deepali Pandit, Khushali Pandit, Alka Dwivedi, Atharva Karulkar, Ankesh Kumar Jaiswal, Aalia Khan, Shreshtha Shah, Afrin Rafiq, Moumita Basu, Juber Pendhari, Sweety Asija, Ambalika Chowdury, Ankit Banik, Nirmalya Roy Moulik, Shyam Srinivasan, Shilpushp Bhosle, Sumathi Hiregoudar, Shashank Ojha, Lingaraj Nayak, Jayshree Thorat, Bhausaheb Bagal, Manju Sengar, Navin Khattry, Shripad Banavali, Steven Highfill, Nirali N Shah, Rahul Purwar","doi":"10.1038/s41408-025-01308-7","DOIUrl":"10.1038/s41408-025-01308-7","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"15 1","pages":"98"},"PeriodicalIF":12.9,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12092698/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144109683","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":"Two ways to complex karyotype in MDS—the role of del(5q) and TP53","authors":"Sandra Huber, Stephan Hutter, Constance Baer, Manja Meggendorfer, Gregor Hoermann, Wolfgang Kern, Torsten Haferlach, Claudia Haferlach","doi":"10.1038/s41408-025-01305-w","DOIUrl":"https://doi.org/10.1038/s41408-025-01305-w","url":null,"abstract":"","PeriodicalId":8989,"journal":{"name":"Blood Cancer Journal","volume":"73 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144097235","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}