Blood NeoplasiaPub Date : 2025-05-01DOI: 10.1016/j.bneo.2025.100088
{"title":"Moura PL, Nannya Y, Aliouat A, et al. Competition of dual SF3B1mt clones in MDS-RS is associated with distinct RNA mis-splicing in hematopoietic stem cells. Blood Neoplasia. 2024;1(2):100011.","authors":"","doi":"10.1016/j.bneo.2025.100088","DOIUrl":"10.1016/j.bneo.2025.100088","url":null,"abstract":"","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100088"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143923022","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}
Blood NeoplasiaPub Date : 2025-05-01DOI: 10.1016/j.bneo.2025.100089
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
{"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}
Blood NeoplasiaPub Date : 2025-05-01DOI: 10.1016/j.bneo.2025.100094
Brian Cuzzo , Maegan Ford , Saagar Jain , Hua-Jay Cherng , Evelyn Orlando , Patrick Gould , Amy Song , Benjamin May , Yuxuan Chen , Govind Bhagat , Andrew H. Lipsky , Barbara Pro , Jennifer E. Amengual
{"title":"Retrospective evaluation of R-EPOCH in the frontline treatment of adult patients with PTLD after solid organ transplant","authors":"Brian Cuzzo , Maegan Ford , Saagar Jain , Hua-Jay Cherng , Evelyn Orlando , Patrick Gould , Amy Song , Benjamin May , Yuxuan Chen , Govind Bhagat , Andrew H. Lipsky , Barbara Pro , Jennifer E. Amengual","doi":"10.1016/j.bneo.2025.100094","DOIUrl":"10.1016/j.bneo.2025.100094","url":null,"abstract":"<div><h3>Abstract</h3><div>Posttransplant lymphoproliferative disorders (PTLD) are rare complications of solid organ transplantation, which carry significant morbidity and mortality. Phase 2 trials that use sequential rituximab (R) followed by R, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) have become an acceptable approach for B-cell PTLD, although it carries a high risk of treatment-related mortality (up to 11%). Many aspects of B-cell PTLD biology and patient characteristics parallel AIDS-related lymphomas in which dose-modified R, etoposide, prednisone, vincristine, cyclophosphamide, and doxorubicin (DM-R-EPOCH) has been demonstrated to be highly efficacious and safe. In this single-institution retrospective study of (N = 101) adult transplant recipients with B-cell PTLD, 65 received DM-R-EPOCH, 8 received R-CHOP, and 17 received R monotherapy. Median progression-free and overall survival was 4.4 years and 6.4 years, respectively, for DM-R-EPOCH and 1 year and 1.1 years, respectively, for R-CHOP. Rates of neutropenia and infection were 70% and 77%, respectively, for DM-R-EPOCH, and 88% each for R-CHOP. Treatment-related mortality for patients treated with DM-R-EPOCH and R-CHOP was 4.7% and 25%, respectively. The median number of cycles of DM-R-EPOCH was 6, and between 73% and 89% of patients received a relative dose intensity of ≥80% for cyclophosphamide, etoposide, and doxorubicin. The relative dose intensity of vincristine was <80% in 56% of patients because of frequent omission for gastrointestinal involvement of PTLD. Collectively, these data suggest that DM-R-EPOCH does not lead to excessive toxicity in patients with B-cell PTLD and support the need for further prospective clinical studies.</div></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100094"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143923019","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}
Blood NeoplasiaPub Date : 2025-05-01DOI: 10.1016/j.bneo.2025.100084
{"title":"Uemura Y, Yamamoto M, Ishimura M, et al. JAK1/2 inhibitor ruxolitinib for the treatment of systemic chronic active Epstein-Barr virus disease: a phase 2 study. Blood Neoplasia. 2025;2(1):100053.","authors":"","doi":"10.1016/j.bneo.2025.100084","DOIUrl":"10.1016/j.bneo.2025.100084","url":null,"abstract":"","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100084"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143923021","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}
Blood NeoplasiaPub Date : 2025-05-01DOI: 10.1016/j.bneo.2025.100090
Hannah Kunstek , Janneke Kieviet , Caroline Lindemans , Coco de Koning ∗ , Stefan Nierkens ∗
{"title":"Thymic peptides in immune reconstitution and clinical outcome after allogeneic hematopoietic cell transplantation","authors":"Hannah Kunstek , Janneke Kieviet , Caroline Lindemans , Coco de Koning ∗ , 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}
Blood NeoplasiaPub Date : 2025-05-01DOI: 10.1016/j.bneo.2025.100093
Tamara Westover , Michael P. Walsh , Sherif Abdelhamed , Emily Xiong , Jing Ma , Guangchun Song , Melvin E. Thomas III , Masayuki Umeda , Jamie L. Maciaszek , Jasmine C. Wong , Astrid Wintering , Lu Wang , Peter D. Emanuel , Mignon L. Loh , Sarah K. Tasian , Elliot Stieglitz , Jason R. Schwartz , Kevin M. Shannon , Jeffery M. Klco
{"title":"Genomic landscape and clonal architecture in pediatric myeloid neoplasms with chromosome 7 deletions","authors":"Tamara Westover , Michael P. Walsh , Sherif Abdelhamed , Emily Xiong , Jing Ma , Guangchun Song , Melvin E. Thomas III , Masayuki Umeda , Jamie L. Maciaszek , Jasmine C. Wong , Astrid Wintering , Lu Wang , Peter D. Emanuel , Mignon L. Loh , Sarah K. Tasian , Elliot Stieglitz , Jason R. Schwartz , Kevin M. Shannon , Jeffery M. Klco","doi":"10.1016/j.bneo.2025.100093","DOIUrl":"10.1016/j.bneo.2025.100093","url":null,"abstract":"","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":"2 2","pages":"Article 100093"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143923020","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 , Naoki Osada , Hirokazu Kubota , Ko Kikuzato , Hiroo Koyama , Takeshi Sonoda , Akiko Idei , Minoru Yoshida , Masaki Kikuchi , Takashi Umehara , Chiduru Watanabe , Teruki Honma , Hiroshi Yasui , Sho Ikeda , Naoto Takahashi , Hideki Nakasone , Jiro Kikuchi , 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)<sup>+</sup> MM compared with t(4;14)<sup>-</sup> 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}
Blood NeoplasiaPub Date : 2025-03-10DOI: 10.1016/j.bneo.2025.100092
Binoy Yohannan , Matthew Rees , Morie A. Gertz , Angela Dispenzieri , Prashant Kapoor , Francis K. Buadi , David Dingli , Nelson Leung , Martha Q. Lacy , Suzanne R. Hayman , Wilson Gonsalves , Taxiarchis Kourelis , Joselle Cook , Moritz Binder , Mustaqeem Siddiqui , Yi Lin , Lisa Hwa , Michelle G. Rogers , Miriam Hobbs , Amie Fonder , Eli Muchtar
{"title":"Improved survival with daratumumab-CyBorD compared with CyBorD as frontline therapy for AL amyloidosis","authors":"Binoy Yohannan , Matthew Rees , Morie A. Gertz , Angela Dispenzieri , Prashant Kapoor , Francis K. Buadi , David Dingli , Nelson Leung , Martha Q. Lacy , Suzanne R. Hayman , Wilson Gonsalves , Taxiarchis Kourelis , Joselle Cook , Moritz Binder , Mustaqeem Siddiqui , Yi Lin , Lisa Hwa , Michelle G. Rogers , Miriam Hobbs , Amie Fonder , 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> < .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> < .001). Fewer patients treated with Dara-CyBorD required second-line therapy at the 12-month landmark (14.9% vs 42.9%; <em>P</em> < .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}
Blood NeoplasiaPub Date : 2025-03-03DOI: 10.1016/j.bneo.2025.100082
Katie Erdos , Aya Alshareef , Richard T. Silver , Joseph M. Scandura , Ghaith Abu-Zeinah
{"title":"Outcomes for ruxolitinib only versus combination with interferon in treating patients with myelofibrosis","authors":"Katie Erdos , Aya Alshareef , Richard T. Silver , Joseph M. Scandura , 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}