Leukemia research最新文献

{"title":"Double IGHV rearrangements in a Latin American cohort of chronic lymphocytic leukemia patients","authors":"Carmen Stanganelli, Juana Cabrera, Andrea Bender, Evangelina Agriello, Davi Coe Torres, Gerson Moura Ferreira, Maria Paula Mota dos Santos, Eliana Saul Furquim Werneck Abdelhay, Irma Slavutsky","doi":"10.1016/j.leukres.2025.108151","DOIUrl":"10.1016/j.leukres.2025.108151","url":null,"abstract":"","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"160 ","pages":"Article 108151"},"PeriodicalIF":2.2,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145827879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"IGF2BP3 promotes acute myeloid leukemia cell progression by regulating Semaphorin 4D stability in an m6A-dependent manner","authors":"Juan Li ,&nbsp;Shulan Shi ,&nbsp;Kefu Zhu ,&nbsp;Linyang Zhe ,&nbsp;Tianle Lu ,&nbsp;Quanzhen Deng ,&nbsp;Qingfang Li ,&nbsp;Qiuling Hou ,&nbsp;Tilong Huang ,&nbsp;Qiangming Sun ,&nbsp;Ming Yu ,&nbsp;Hongchao Jiang","doi":"10.1016/j.leukres.2025.108119","DOIUrl":"10.1016/j.leukres.2025.108119","url":null,"abstract":"<div><h3>Background</h3><div>Our previous findings indicate Semaphorin 4D (Sema4D) as a potential pediatric leukemia biomarker, promoting leukemogenesis via PI3K/AKT and ERK pathways, but its upstream regulatory mechanisms remain unclear. This study was conducted to explore the potential regulatory relationship between Insulin growth factor 2 mRNA binding protein 3 (IGF2BP3) and Sema4D in acute myeloid leukemia (AML).</div></div><div><h3>Methods</h3><div>The expression levels of Sema4D and IGF2BP3 were analyzed in PBMCs from 41 newly diagnosed patients with pediatric acute myeloid leukemia (AML) and 35 healthy pediatric donors who presented no history of leukemia using western blotting and qRT-PCR. IGF2BP3 overexpression and knockdown models were established in Kasumi-1 and HL-60 cells via lentiviral infection. Cell proliferation, apoptosis, and cell cycle distribution were assessed using CCK-8 and flow cytometry. The stability of Sema4D mRNA and m6A methylation levels were evaluated via mRNA stability assay and qPCR.</div></div><div><h3>Results</h3><div>This study discovered that Sema4D and IGF2BP3 were overexpressed in the peripheral blood mononuclear cells (PBMCs) of AML patients, and their expression levels were positively correlated. IGF2BP3 overexpression enhanced cell proliferation and cell cycle progression, and inhibited apoptosis in AML cells, while knockdown had the opposite effect. Mechanistic exploration revealed that IGF2BP3 enhances the stability of Sema4D mRNA through m6A-dependent mechanisms.</div></div><div><h3>Conclusion</h3><div>In this study, we demonstrate that the IGF2BP3/Sema4D axis is a crucial regulator in AML development, driving cell proliferation and survival through post-transcriptional regulation of Sema4D by IGF2BP3 in an m6A-dependent manner. Our findings highlight the potential of targeting this axis as a therapeutic strategy for AML treatment.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108119"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145445437","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High JAK2V617F allele burden in low-risk PV: an additional recommendation to start “early” cytoreduction? Results from a multicentric study","authors":"Luca Tosoni,&nbsp;Sara Di Giusto,&nbsp;Gianluca Morelli,&nbsp;Melissa Bergnach,&nbsp;Chiara Callegari,&nbsp;Rossella Stella,&nbsp;Francesco Zaja,&nbsp;Antonia Schwab,&nbsp;Albert Wölfler,&nbsp;Eleonora Toffoletti,&nbsp;Daniela Damiani,&nbsp;Renato Fanin,&nbsp;Mario Tiribelli","doi":"10.1016/j.leukres.2025.108123","DOIUrl":"10.1016/j.leukres.2025.108123","url":null,"abstract":"","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108123"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145409507","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The role of interim bone marrow assessments in acute myeloid leukemia – A systematic review and meta-analysis","authors":"Yuanli Lei ,&nbsp;Zhiting Tang ,&nbsp;Jessica A. Reese ,&nbsp;Omer Jamy ,&nbsp;Mohamad Khawandanah ,&nbsp;Manu Pandey ,&nbsp;Muhammad Faisal ,&nbsp;Adam Asch ,&nbsp;Zimu Gong","doi":"10.1016/j.leukres.2025.108121","DOIUrl":"10.1016/j.leukres.2025.108121","url":null,"abstract":"<div><h3>Background</h3><div>The current standard of care for acute myeloid leukemia (AML) patients undergoing intensive induction chemotherapy includes an interim bone marrow biopsy around day 14 (D14BM), and reinduction for patients with more than 5 % residual blasts on D14BM (Positive D14BM). However, this approach has become increasingly controversial. This systematic review and meta-analysis assess the sensitivity, specificity, and predictive value of D14BM in patients treated with one cycle of induction chemotherapy and evaluate the efficacy of reinduction versus observation in patients with Positive D14BM.</div></div><div><h3>Methods</h3><div>A systematic literature search was conducted using PubMed, Embase, Web of Science, and Cochrane databases. A bivariate model was used for pooled sensitivity and specificity. The positive predictive value was estimated based on pooled mean sensitivity, specificity, and historical refractory leukemia prevalence. Risk ratios (RR) for complete remission (CR) were meta-analyzed to compare reinduction versus observation in Positive D14BM patients.</div></div><div><h3>Result</h3><div>Among 1044 identified articles, 12 met inclusion criteria. Ten studies (1683 patients) evaluated the predictive value of D14BM, with a sensitivity of 49.7 % (proportion of refractory cases correctly identified by Positive D14BM) and specificity of 86.2 %. The estimated positive predictive value was 38.9 %, assuming a 15 % prevalence of true refractory leukemia. Eleven studies (832 patients) evaluated efficacy of reinduction, with a pooled RR of 1.00 (61 % vs. 60 %, 95 % CI: 0.76–1.31) for CR.</div></div><div><h3>Conclusion</h3><div>Positive D14BM has limited predictive power for refractory leukemia. Reinduction based on Positive D14BM does not improve CR rates compared to observation alone only and may expose patients to undue toxicity.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108121"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145318432","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world treatment patterns and clinical outcomes of patients with treatment-naïve and relapsed/refractory diffuse large B-cell lymphoma in the United States","authors":"Jennifer K. Lue ,&nbsp;Priyanka J. Bobbili ,&nbsp;Mahek Garg ,&nbsp;Christopher W. Yee ,&nbsp;Katherine E. Ryland ,&nbsp;Sonia Gupta ,&nbsp;Daisy Liu ,&nbsp;Monika Raut ,&nbsp;Mei Sheng Duh","doi":"10.1016/j.leukres.2025.108117","DOIUrl":"10.1016/j.leukres.2025.108117","url":null,"abstract":"<div><h3>Background</h3><div>With the evolving treatment landscape and growing therapeutic options, it is essential to understand the treatments currently used in real-world clinical practice and the associated outcomes among patients with DLBCL to identify potential unmet treatment needs.</div></div><div><h3>Patients and Methods</h3><div>This retrospective longitudinal cohort study examined treatment patterns and outcomes for diffuse large B-cell lymphoma (DLBCL) patients using the COTA electronic health records database from 2016 to 2023. The study population included 3569 patients, treated in community oncology settings, who received first-line (1L) therapy.</div></div><div><h3>Results</h3><div>Nineteen percent of patients relapsed after 1L chemoimmunotherapy (primarily R-CHOP). The most common second-line treatments included rituximab-based regimens and autologous stem cell transplant (ASCT). Antibody-drug conjugates (ADCs) and CAR T-cell therapy were administered in third-line or later. Median progression-free survival (PFS) and overall survival (OS) decreased with each line. Relapsed/refractory real-world DLBCL patients experienced poor outcomes (24-month OS of 41.7 %). Patients receiving ASCT or CAR T-cell therapy had better OS than those who did not receive cellular therapies, with 24-month OS of 78 %, 54 %, and 31 %, respectively.</div></div><div><h3>Conclusion</h3><div>This study highlights the need for improved treatment options and increased access to novel therapies, emphasizing gaps in community oncology settings.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108117"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145517199","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rusfertide rapidly decreases hematocrit in patients with suboptimally controlled polycythemia vera","authors":"Lee Ping Chew ,&nbsp;Yelena Z. Ginzburg ,&nbsp;Kamini Kirubamoorthy ,&nbsp;Sung-Eun Lee ,&nbsp;Jae Hoon Lee ,&nbsp;Nishit B. Modi ,&nbsp;Sarita Khanna ,&nbsp;Phillip Dinh ,&nbsp;Frank Valone ,&nbsp;Arturo Molina ,&nbsp;Suneel Gupta","doi":"10.1016/j.leukres.2025.108132","DOIUrl":"10.1016/j.leukres.2025.108132","url":null,"abstract":"<div><div>Polycythemia vera (PV) is characterized by overproduction of erythrocytes, leukocytes, and platelets. Rusfertide is a hepcidin mimetic polypeptide that binds to ferroportin and decreases iron delivery to bone marrow, reducing the production of red blood cells. The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (&gt;48 %) baseline hematocrit. Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached &lt; 45 %; once achieved, patients received weekly rusfertide dosing. Of the 20 patients enrolled, 15 (75 %) completed 24 weeks of treatment, 13 (65 %) completed 52 weeks, and 7 discontinued rusfertide therapy. Mean baseline hematocrit was 51.6 %. By Week 8, 17 (85 %) patients achieved hematocrit &lt; 45 %, and 19 (95 %) achieved ≥ 5 % absolute reduction from baseline. Median time to first hematocrit &lt; 45 % was 4.9 weeks. Mean hematocrit was &lt; 45 % at Week 4 and subsequently remained &lt; 45 % throughout the study. No patients received phlebotomy during rusfertide treatment. Rusfertide led to a significant reduction in erythrocyte count (5.98 ×10¹²/L to 4.79 ×10¹²/L; nominal p-value 0.0036) and an improvement in iron deficiency markers (eg, ferritin and mean corpuscular volume) relative to baseline. Seventeen patients (85 %) experienced treatment-emergent adverse events, most of which were Grade 1/2. In this open-label phase 2 study, rusfertide resulted in a rapid and sustained improvement in hematocrit and was generally well tolerated. Rapid and sustained control of hematocrit reduces the need for therapeutic phlebotomy and may help reduce thrombotic and cardiovascular events over the long term in patients with PV.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108132"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145418537","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Differential impact of graft-versus-host disease on post-transplant outcomes of chronic myelomonocytic leukemia according to transplant procedures","authors":"Hidehiro Itonaga ,&nbsp;Yasushi Miyazaki ,&nbsp;Machiko Fujioka ,&nbsp;Shuhei Kurosawa ,&nbsp;Yasunobu Nagata ,&nbsp;Yutaka Shimazu ,&nbsp;Tomoaki Ueda ,&nbsp;Naoyuki Uchida ,&nbsp;Noriko Doki ,&nbsp;Tetsuya Nishida ,&nbsp;Noboru Asada ,&nbsp;Masatsugu Tanaka ,&nbsp;Satoru Takada ,&nbsp;Masatoshi Sakurai ,&nbsp;Mineo Kurokawa ,&nbsp;Makoto Yoshimitsu ,&nbsp;Yoshinobu Kanda ,&nbsp;Tatsuo Ichinohe ,&nbsp;Yoshiko Atsuta ,&nbsp;Takayoshi Tachibana","doi":"10.1016/j.leukres.2025.108134","DOIUrl":"10.1016/j.leukres.2025.108134","url":null,"abstract":"<div><div>Allogeneic hematopoietic stem cell transplantation (allo-HSCT) provides curative outcomes for chronic myelomonocytic leukemia (CMML). We conducted a retrospective study to clarify the protective impact of acute and chronic graft-versus-host disease (aGVHD and cGVHD) on relapse by transplant procedures in 314 CMML patients. Interaction effects of conditioning intensity were observed with aGVHD (P_interaction=0.181) and cGVHD (P_interaction=0.256) for the cumulative incidence of relapse (CIR), whereas interaction effects of donor type were not detected with GVHD. In patients with the myeloablative conditioning regimens, the multivariate analysis showed that the development of aGVHD was not associated with overall survival (OS). The development of limited cGVHD correlated with better OS (HR [95 % CI], 0.34 [0.14–0.81]; P = 0.015); and that of extensive cGVHD correlated with better OS (HR, 0.44 [0.21–0.91]; P = 0.026) and lower CIR (HR, 0.28 [0.08–0.94]; P = 0.040). In patients with the reduced-intensity conditioning regimens, the development of grade I-II aGVHD correlated with better OS (HR, 0.39 [0.20–0.76]; P = 0.005) and lower CIR (HR, 0.30 [0.13–0.70]; P = 0.006). The development of extensive cGVHD correlated with better OS (HR, 0.44 [0.20–0.96]; P = 0.039). The present results suggest that the type and severity of GVHD mediating graft-versus-leukemia effects against relapse were influenced by conditioning intensity in CMML patients.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108134"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145505508","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exosomal miR-140–3p produced by bone marrow stromal cells affects acute myeloid leukemia cell growth and apoptosis by targeting SUZ12","authors":"Jiajia Li ,&nbsp;Feifan Li ,&nbsp;Mengmeng Zhang ,&nbsp;Yanping Wu ,&nbsp;Meng Wang ,&nbsp;Pingping Zhang","doi":"10.1016/j.leukres.2025.108122","DOIUrl":"10.1016/j.leukres.2025.108122","url":null,"abstract":"<div><div>There is growing evidence that exosomes produced by bone marrow stromal cells (BMSCs) are associated with the progression of several cancers, including acute myeloid leukemia (AML), but intrinsic molecular mechanisms remain elusive. Here, we applied previous microarray analysis (GSE133276 and GSE209871) to identify differentially expressed exosomal miRNAs in BMSCs of AML patients, and candidate miR-140–3p, miR-142–5p and miR-142–3p were selected. This study aimed to investigate whether BMSCs affects AML progression are mediated by the candidates, and to explore regulatory mechanisms. The levels of the candidates were examined in bone marrow and BMSCs from AML patients, and exosomes from BMSCs of normal subjects and AML cells by qRT-PCR. Cell proliferation and apoptosis of AML cells co-cultured with BMSCs were detected through CCK-8, colony formation, flow cytometry, and Caspase-3 activity assays with manipulation of the candidate miRNAs expression. RNA pull-down and luciferase reporter assays to identify the downstream target mRNAs of the miRNAs. We confirmed that miR-140–3p, miR-142–5p and miR-142–3p levels were downregulated in bone marrow and BMSCs of AML patients, and were significantly enriched in exosomes of BMSCs but not AML cells. BMSCs co-cultured with AML cells could transfer these miRNAs into AML cells, and suppressed the proliferative potential and promoted the apoptotic behavior of AML cells. Furthermore, miR-140–3p agomir in BMSCs exacerbated the effects of the co-culture system on the AML cell proliferation and apoptosis, which were attenuated by miR-140–3p antagomir. In contrast, co-culture data showed that miR-142–5p and miR-142–3p had no significant effect on cell proliferation and apoptosis. Moreover, SUZ12 polycomb repressive complex 2 subunit (SUZ12) was directly targeted by miR-140–3p, overexpression or inhibition of SUZ12 in AML cells partially counteracted miR-140–3p agomir or antagomir-mediated cellular effects, respectively. Our study suggested that the BMSCs-derived exosomal miR-140–3p, rather than miR-142–5p and miR-142–3p, has a regulatory effect on the growth and apoptosis of AML cells by targeting SUZ12.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108122"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145364939","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multicenter retrospective analysis of the short-term efficacy and safety of the VAH regimen in the treatment of acute myeloid leukemia","authors":"Ruihua Mi ,&nbsp;Lin Chen ,&nbsp;Lin Wang ,&nbsp;Haiping Yang ,&nbsp;Hongmian Zhao ,&nbsp;Sun Wu ,&nbsp;Yixuan Ma ,&nbsp;Jia Liu ,&nbsp;Xudong Wei","doi":"10.1016/j.leukres.2025.108114","DOIUrl":"10.1016/j.leukres.2025.108114","url":null,"abstract":"<div><h3>Objective</h3><div>To retrospectively investigate the short-term efficacy and safety of the triple-drug combination regimen of venetoclax + azacitidine + homoharringtonine (VAH regimen) in patients with acute myeloid leukemia (AML).</div></div><div><h3>Methods</h3><div>Retrospective analysis was conducted on a total of 45 patients with newly diagnosed or refractory/relapsed AML, who were admitted to the Affiliated Cancer Hospital of Zhengzhou University, the First Affiliated Hospital, and College of Clinical Medicine of Henan University of Science and Technology, the Huaihe Hospital of Henan University, and The First Affiliated Hospital of Xinxiang Medical University from July 2022 to October 2024 and treated with the VAH regimen. The overall response rate and safety of this regimen were analyzed, and the relevant literature was reviewed.</div></div><div><h3>Results</h3><div>In the 23 newly treated patients, the overall response rate (ORR) (CR+CRi) was 65.2 % on day 15 of the VAH regimen and 82.6 % on day 29 after the end of cycle 1. The median duration of grade 3–4 neutropenia was 12.6 days. In the 22 refractory/relapsed patients, the ORR (CR+CRi) was 68.2 % on day 15 of the VAH regimen and 86.4 % on day 29 after the end of cycle 1, and the median duration of neutropenia was 14.4 days. The most common adverse reactions were myelosuppression and infection, both of which were within controllable limits. There was no death due to adverse reactions during treatment.</div></div><div><h3>Conclusion</h3><div>The VAH regimen yields a high remission rate in newly diagnosed and refractory/relapsed AML patients. This retrospective study provides a novel treatment strategy for AML patients.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"159 ","pages":"Article 108114"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145390688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pathogenic mechanisms and targeted therapies for anemia in myelodysplastic syndromes","authors":"Doudou Chang ,&nbsp;Linhua Yang ,&nbsp;Ruijuan Zhang","doi":"10.1016/j.leukres.2025.108099","DOIUrl":"10.1016/j.leukres.2025.108099","url":null,"abstract":"<div><div>Myelodysplastic Syndromes (MDS) are malignant neoplasms of the myeloid lineage originating from hematopoietic stem cells (HSCs), characterized by ineffective hematopoiesis, blood cell dysplasia, and a high risk of transformation to acute leukemia. Anemia affects up to 90 % of MDS patients, significantly increasing symptom burden, impacting quality of life, accelerating disease progression, and is associated with increased morbidity and mortality. The limited therapeutic landscape for addressing anemia in MDS presents a significant challenge in clinical management. This review explores the mechanisms of anemia in MDS（including low-risk MDS and high-risk MDS）, covering disturbances in hematopoiesis, telomere dynamics, inflammation, immune dysregulation, alterations in iron metabolism, and the role of the bone marrow microenvironment. We examine how these factors contribute to the complex pathophysiology of MDS-related anemia and discuss their implications for treatment strategies. Furthermore, we highlight recent advances in understanding the molecular basis of MDS, which have paved the way for novel therapeutic approaches. These include targeted therapies addressing specific genetic mutations, immunomodulatory agents, and innovative approaches to stimulate effective erythropoiesis. We also review emerging treatments such as Luspatercept, along with other novel agents targeting specific pathways, and ongoing clinical trials exploring combination therapies and personalized treatment strategies.</div></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"158 ","pages":"Article 108099"},"PeriodicalIF":2.2,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145098913","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}