Blood Neoplasia最新文献

{"title":"Response and outcomes of patients with IDH1/2- mutated accelerated/blast-phase myeloproliferative neoplasms","authors":"Leah A. Goldberg , James J. Yoon , Hannah Johnston , Marta B. Davidson , Alexa Siddon , Rory M. Shallis , Evan C. Chen , Madelyn Burkart , Timothy S. Oh , Sunil G. Iyer , Ellen Madarang , Chandrasekar Muthiah , Joshua Kassner , Raajit K. Rampal , Guru Subramanian Guru Murthy , Terrence Bradley , Yasmin Abaza , Jacqueline S. Garcia , Vikas Gupta , Kristen M. Pettit , Anand A. Patel","doi":"10.1016/j.bneo.2025.100089","DOIUrl":"10.1016/j.bneo.2025.100089","url":null,"abstract":"","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100089"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143886562","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Thymic peptides in immune reconstitution and clinical outcome after allogeneic hematopoietic cell transplantation","authors":"Hannah Kunstek ,&nbsp;Janneke Kieviet ,&nbsp;Caroline Lindemans ,&nbsp;Coco de Koning ∗ ,&nbsp;Stefan Nierkens ∗","doi":"10.1016/j.bneo.2025.100090","DOIUrl":"10.1016/j.bneo.2025.100090","url":null,"abstract":"<div><h3>Abstract</h3><div>Patients with hematologic malignancies or nonmalignant diseases may undergo allogeneic hematopoietic cell transplantation (HCT), which represents a potential curative treatment. However, they are still at risk of life-threatening complications, such as relapse, acute graft-versus-host disease, and opportunistic infections. These complications are more likely if T-cell reconstitution is delayed during the initial 3 to 4 months after HCT. Therefore, it is of clinical importance to advance early peripheral T-cell expansion. The thymus is the cradle of T-cell production, but it is extremely sensitive to conditioning drugs used in HCT. As a result, egress of T cells from the thymus is abrogated during the first 3 to 6 months after HCT. Instead, early T-cell reconstitution depends on peripheral expansion of engrafted donor T cells. However, besides its established function to produce T cells, the thymus also produces thymic peptides with hormone-like activity. These molecules play an essential part in the development and nature of immune responses and may have a role in modulating T-cell expansion and function after HCT. In this review, we investigate the role of thymic peptides in shaping the dynamics of immune reconstitution early after HCT. Furthermore, we summarize current reports on clinical application of thymic peptides post-HCT and discuss their potential use in improving patient outcomes.</div></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100090"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143886378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Discovery of a novel class NSD2 inhibitor for multiple myeloma with t(4;14)+","authors":"Sae Matsuoka ,&nbsp;Naoki Osada ,&nbsp;Hirokazu Kubota ,&nbsp;Ko Kikuzato ,&nbsp;Hiroo Koyama ,&nbsp;Takeshi Sonoda ,&nbsp;Akiko Idei ,&nbsp;Minoru Yoshida ,&nbsp;Masaki Kikuchi ,&nbsp;Takashi Umehara ,&nbsp;Chiduru Watanabe ,&nbsp;Teruki Honma ,&nbsp;Hiroshi Yasui ,&nbsp;Sho Ikeda ,&nbsp;Naoto Takahashi ,&nbsp;Hideki Nakasone ,&nbsp;Jiro Kikuchi ,&nbsp;Yusuke Furukawa","doi":"10.1016/j.bneo.2025.100091","DOIUrl":"10.1016/j.bneo.2025.100091","url":null,"abstract":"<div><h3>Abstract</h3><div>The prognosis for multiple myeloma (MM) has continued to improve with the development of a series of novel molecular targeted drugs over time. However, the prognosis remains poor for cases with high-risk chromosomal abnormalities. Of such abnormalities, t(4;14) is the second most common, occurring in 15% of patients with MM. MM cells carrying t(4;14) strongly express histone methyltransferase with a SET domain, called NSD2, making them resistant to drugs against MM. Therefore, NSD2 is a promising therapeutic target for MM carrying t(4;14). Subsequently, we performed high-throughput screening and identified RK-0080552 (RK-552) as a novel class NSD2 inhibitor. RK-552 was significantly cytotoxic against t(4;14)⁺ MM compared with t(4;14)^- MM cells in vitro and in vivo via transcriptional suppression of the <em>IRF4</em> gene, coincided with a decrease in histone H3 lysine 36 dimethylation. Moreover, RK-552 acted additively with pomalidomide in vitro and prolonged the survival of recipient mice without side effects. These results suggest that RK-552 may be a clinically relevant NSD2 inhibitor with specific cytotoxicity to MM cells carrying t(4;14). Our study also provides a molecular basis and rationale for the inclusion in current treatment strategies. Therefore, the clinical use of RK-552 may significantly improve the treatment outcome of MM with t(4;14).</div></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100091"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143906976","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Improved survival with daratumumab-CyBorD compared with CyBorD as frontline therapy for AL amyloidosis","authors":"Binoy Yohannan ,&nbsp;Matthew Rees ,&nbsp;Morie A. Gertz ,&nbsp;Angela Dispenzieri ,&nbsp;Prashant Kapoor ,&nbsp;Francis K. Buadi ,&nbsp;David Dingli ,&nbsp;Nelson Leung ,&nbsp;Martha Q. Lacy ,&nbsp;Suzanne R. Hayman ,&nbsp;Wilson Gonsalves ,&nbsp;Taxiarchis Kourelis ,&nbsp;Joselle Cook ,&nbsp;Moritz Binder ,&nbsp;Mustaqeem Siddiqui ,&nbsp;Yi Lin ,&nbsp;Lisa Hwa ,&nbsp;Michelle G. Rogers ,&nbsp;Miriam Hobbs ,&nbsp;Amie Fonder ,&nbsp;Eli Muchtar","doi":"10.1016/j.bneo.2025.100092","DOIUrl":"10.1016/j.bneo.2025.100092","url":null,"abstract":"<div><h3>Abstract</h3><div>In the ANDROMEDA phase 3 trial, the addition of daratumumab to cyclophosphamide, bortezomib, and dexamethasone (Dara-CyBorD) as frontline therapy significantly improved hematological and organ responses, and event-free survival (EFS) compared with CyBorD. To validate its results, we performed a retrospective study of 361 consecutive patients with newly diagnosed light chain (AL) amyloidosis treated between 2018 and 2022. Patients who received Dara-CyBorD (n = 147) were compared with those treated with CyBorD (n = 214) in key outcome endpoints. The 2-month hematological very good partial response or better rate was higher with Dara-CyBorD than with CyBorD (60.8% vs 31.1%; <em>P</em> &lt; .001). In addition, 2- and 6-month hematological complete response was also higher with Dara-CyBorD (15.3% vs 3.0% and 39.5% vs 17.8%, respectively; both <em>P</em> &lt; .001). Fewer patients treated with Dara-CyBorD required second-line therapy at the 12-month landmark (14.9% vs 42.9%; <em>P</em> &lt; .001). The 6- and 12-month cardiac responses were higher and deeper in the Dara-CyBorD group than in the CyBorD group. Dara-CyBorD was associated with a lower 6-month mortality rate (8.8% vs 16.3%; <em>P</em> = .04) and superior EFS and overall survival (OS). An OS difference between the treatment groups was statistically significant among patients with stage II cardiac disease, and borderline significant for stage IIIA but not for cardiac stage IIIB. In conclusion, the addition of daratumumab to frontline CyBorD significantly improved hematological and organ response rates, reduced early deaths, and prolonged EFS and OS compared with CyBorD.</div></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100092"},"PeriodicalIF":0.0,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143860617","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes for ruxolitinib only versus combination with interferon in treating patients with myelofibrosis","authors":"Katie Erdos ,&nbsp;Aya Alshareef ,&nbsp;Richard T. Silver ,&nbsp;Joseph M. Scandura ,&nbsp;Ghaith Abu-Zeinah","doi":"10.1016/j.bneo.2025.100082","DOIUrl":"10.1016/j.bneo.2025.100082","url":null,"abstract":"","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100082"},"PeriodicalIF":0.0,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143791842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prospective clinical trials of venetoclax and hypomethylating agents for relapsed BPDCN","authors":"Naveen Pemmaraju ,&nbsp;Luan Hai Phan ,&nbsp;Geoffrey Fell ,&nbsp;Marlise R. Luskin ,&nbsp;Mahesh Swaminathan ,&nbsp;Sherry Pierce ,&nbsp;Courtney DiNardo ,&nbsp;Abhishek Maiti ,&nbsp;Marina Konopleva ,&nbsp;Andrew A. Lane","doi":"10.1016/j.bneo.2025.100086","DOIUrl":"10.1016/j.bneo.2025.100086","url":null,"abstract":"","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100086"},"PeriodicalIF":0.0,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143838021","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multiplex gene editing models of del(7q) reveal combined CUX1 and EZH2 loss drives clonal expansion and drug resistance","authors":"Matthew R. M. Jotte ,&nbsp;Angela Stoddart ,&nbsp;Tanner C. Martinez ,&nbsp;Raven Moten ,&nbsp;Yuqing Xue ,&nbsp;Molly K. Imgruet ,&nbsp;Hunter Blaylock ,&nbsp;Henna S. Nam ,&nbsp;Bonnie Hu ,&nbsp;Jermaine Austin ,&nbsp;Ningfei An ,&nbsp;Saira Khan ,&nbsp;Sandeep K. Gurbuxani ,&nbsp;Megan E. McNerney","doi":"10.1016/j.bneo.2025.100083","DOIUrl":"10.1016/j.bneo.2025.100083","url":null,"abstract":"<div><h3>Abstract</h3><div>Loss of all or part of chromosome 7 [−7/del(7q)] is recurrent in myeloid neoplasms and associated with a poor response to chemotherapy. Chromosome 7–encoded genes driving drug resistance and the consequences of combinatorial 7q tumor suppressor gene loss have remained unclear, the latter question largely because of the challenges of modeling aneuploidy. Here, we use in silico data mining to uncover 7q genes involved in chemotherapy resistance. We establish murine models of del(7q) clonal hematopoiesis and drug resistance with multiplex CRISPR-Cas9 (CRISPR–associated protein 9)–mediated inactivation of 4 genes, <em>Cux1</em>, <em>Ezh2</em>, <em>Kmt2c</em>, and <em>Kmt2e</em>. Postgenotoxic exposure, combined deficiency of <em>Cux1</em> and <em>Ezh2</em> preferentially promotes clonal myeloid expansion in vivo, with compounding defects in DNA damage recognition and repair. Human acute myeloid leukemia cell lines similarly illustrate central roles for <em>CUX1</em> and <em>EZH2</em> loss in survival and DNA damage resolution after chemotherapy exposure. Transcriptome analysis reveals combined <em>Cux1</em> and <em>Ezh2</em> loss recapitulates gene signatures of -7 patients and defective DNA damage response pathways, to a greater extent than single gene loss. This work reveals a genetic interaction between <em>CUX1</em> and <em>EZH2</em>, and sheds light on how −7/del(7q) contributes to leukemogenesis and drug resistance characteristic of these adverse-risk neoplasms. These data support the concept of 7q as a contiguous gene syndrome region, in which combined loss of multiple gene drives pathogenesis. Furthermore, our CRISPR-based approach may serve as a framework for interrogating other recurrent aneuploid events in cancer.</div></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100083"},"PeriodicalIF":0.0,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143860681","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A novel 3-way translocation involving ETV6::IL3 drives AML with eosinophilia","authors":"Ariel Siegel ,&nbsp;Joseph Tripodi ,&nbsp;Dianna Hardatt ,&nbsp;Brianna Kelly ,&nbsp;Sebastian El Ghaity-Beckley ,&nbsp;Michelle Becker ,&nbsp;Brenton Francisco ,&nbsp;Kayleen Bailey ,&nbsp;Bruce Petersen ,&nbsp;Douglas Tremblay ,&nbsp;Jonathan Feld ,&nbsp;Hannah Levavi ,&nbsp;Marina Kremyanskaya ,&nbsp;Alla Keyzner ,&nbsp;Bridget K. Marcellino ,&nbsp;Christian Salib ,&nbsp;Amy S. Duffield ,&nbsp;John Mascarenhas ,&nbsp;Vesna Najfeld ,&nbsp;Alan H. Shih","doi":"10.1016/j.bneo.2025.100079","DOIUrl":"10.1016/j.bneo.2025.100079","url":null,"abstract":"","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100079"},"PeriodicalIF":0.0,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143704103","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world treatment patterns and clinical outcomes in patients with follicular lymphoma: a SEER-Medicare analysis","authors":"Dai Chihara ,&nbsp;Shibing Yang ,&nbsp;Savreet Bains Chawla ,&nbsp;Guihua Zhang ,&nbsp;Anthony Wang ,&nbsp;Junhua Yu ,&nbsp;Donald Arnette ,&nbsp;Fernando Rivas Navarro ,&nbsp;Julie Blaedel ,&nbsp;Alex Mutebi","doi":"10.1016/j.bneo.2025.100080","DOIUrl":"10.1016/j.bneo.2025.100080","url":null,"abstract":"<div><h3>Abstract</h3><div>Treatment sequencing and survival outcomes in follicular lymphoma (FL) are heterogeneous. This study describes real-world treatment patterns and outcomes among older patients with FL in the United States. Patients aged ≥65 years diagnosed with FL were identified from Surveillance, Epidemiology, and End Results–Medicare (2000-2017) and followed up through 2019. A total of 13 423 patients with FL (median age, 76 years at diagnosis) were included. With a median follow-up of 57.1 months, 38% of patients had no record of initiating any systemic treatment during the observation window; 62%, 23%, 9%, and 4% received ≥1, ≥2, ≥3, and ≥4 lines of therapy (LOTs), respectively. Survival rates increased significantly (<em>P</em> &lt; .0001) over time, as evidenced by a 21% and 36% reduction in mortality risk among patients diagnosed in 2006-2011 and 2012-2017, respectively, compared with those diagnosed in 2000-2005. Chemoimmunotherapy was the most common treatment across LOTs. In ≥1, ≥2, ≥3, and ≥4 LOTs, median event-free survival was 33.1, 19.3, 15.5, and 13.0 months, respectively, and median overall survival (OS) was 79.6, 47.5, 32.8, and 26.1 months, respectively. Older age, advanced FL stage, and high comorbidity index at diagnosis were associated with shorter OS. Patients who progressed within 24 months following first-line therapy and those who received third-line therapy within 36 months also had shorter OS from diagnosis, as did patients with double-refractory disease. Despite recent improvement in treatment outcomes, there remains an unmet medical need for older, high-risk patients with FL.</div></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100080"},"PeriodicalIF":0.0,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143838020","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adult leukemia in Florida 2010-2019: diverse, aging population as an indicator for the United States","authors":"Ming S. Lee ∗ ,&nbsp;Rebecca E. Kaiser ∗ ,&nbsp;Christopher D. Armstrong ,&nbsp;David B. Dukenik ,&nbsp;Nancy S. Elliott ,&nbsp;Raymond R. Balise ,&nbsp;Justin M. Watts ,&nbsp;Mikkael A. Sekeres ,&nbsp;Erin N. Kobetz ,&nbsp;Justin Taylor","doi":"10.1016/j.bneo.2025.100078","DOIUrl":"10.1016/j.bneo.2025.100078","url":null,"abstract":"<div><h3>Abstract</h3><div>According to recent data released by the National Cancer Institute, Florida has the highest incidence of adult leukemia in the United States. There is limited population-based research on aging and sociodemographic disparities associated with leukemia in Florida, which can have a national impact on the assessment of leukemia burden. Using geocoded cancer data from the Florida Cancer Data System and population data from the US Census, this study evaluated socioeconomic and regional disparities associated with leukemia and found that leukemia disparities by race/ethnicity and rurality exist in Florida. The non-Hispanic White population had the highest incidence rates for most subtypes of leukemia, whereas the non-Hispanic Black population had the highest odds of dying from leukemia. Rural counties and urban neighborhoods with lower socioeconomic status were associated with higher mortality odds for leukemia. Leukemia-treating physician numbers were mismatched in regions in which patients with leukemia exhibit higher incidence and mortality odds. These results suggest that leukemia incidence rate in Florida is likely to remain among the highest in the United States due to population aging; however, physician shortages may exacerbate disparities and limit care in rural areas. Florida demographically looks like what the entire US population may be in the future and is therefore an indicator of the coming needs in the United States for increased leukemia diagnosis, treatment, and survivorship care. Larger national and international studies can build on this study by applying our methodology on a larger scale and can also be applied to other hematologic malignancies and other cancer types.</div></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100078"},"PeriodicalIF":0.0,"publicationDate":"2025-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143714450","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}