Hematology最新文献

{"title":"Sintilimab for treating progressive multifocal leukoencephalopathy caused by human polyomavirus 2 virus infection following allogeneic hematopoietic cell transplantation: a case report.","authors":"Xuelian Jin, Xushu Zhong, Qinyu Liu, Xinchuan Chen","doi":"10.1080/16078454.2025.2458932","DOIUrl":"10.1080/16078454.2025.2458932","url":null,"abstract":"<p><strong>Background: </strong>Progressive multifocal leukoencephalopathy (PML) is characterized by demyelination in the central nervous system. It is caused by infection with human polyomavirus 2 and has a poor prognosis. Therapeutic strategies involve restoring immune function and/or discontinuing immunosuppressive treatment. Immune checkpoint inhibitors such as those targeting programmed death receptor-1 (PD-1) can alleviate PML by restoring T cell function. There are no case reports on the use of the PD-1 inhibitor, Sintilimab, for treating PML. Here, we report a case of successful treatment of PML with sintilimab following allogeneic hematopoietic stem cell transplantation.</p><p><strong>Case presentation: </strong>A 35-year-old woman with high-risk acute myeloid leukemia underwent allogeneic hematopoietic stem cell transplantation after induced remission and developed PML 12 months after transplantation. She received five courses of 100 mg every 4 weeks with monitoring by magnetic resonance imaging (MRI) and viral load in the cerebrospinal fluid, showing clinical improvement, resolution of neurological symptoms, and reduced viral load. MRI showed initial exacerbation of lesions but significant improvement after five courses of treatment. No graft-versus-host disease occurred, but manageable immune reconstitution inflammatory syndrome was observed.</p><p><strong>Conclusion: </strong>Sintilimab, a PD-1 inhibitor, might be used to treat PML in patients with hematologic malignancies undergoing allo-HSCT, which needs further investigation.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2458932"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079693","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":"Outcomes of peripherally inserted central catheter vs conventional central venous catheters in hematological cancer patients: a systematic review and meta-analysis.","authors":"Weilei Ge, Chen Zheng","doi":"10.1080/16078454.2025.2450572","DOIUrl":"10.1080/16078454.2025.2450572","url":null,"abstract":"<p><strong>Objective: </strong>This review aimed to examine if there is any difference in the risk of thrombosis and central line-associated bloodstream infection (CLABSI) with the use of peripherally inserted central catheter (PICC) and conventional central venous catheters (CVC) in hematological cancer patients.</p><p><strong>Methods: </strong>We searched the online databases of PubMed, CENTRAL, Scopus, Web of Science, and Embase for all types of studies comparing the risk of thrombosis and CLABSI between PICC and CVC. The search ended on 23rd September 2024.</p><p><strong>Results: </strong>Eight studies were included. One was a randomized trial while others were observational studies. Meta-analysis showed no statistically significant difference in the risk of thrombosis between PICC and CVC (OR: 1.69 95% CI: 0.75, 3.82 I²= 78%). However, these results were not stable on sensitivity analysis. The exclusion of two studies indicated a higher risk of thrombosis with PICC. Pooled analysis showed that the risk of CLABSI was significantly lower with PICC as compared to CVC (OR: 0.52 95% CI: 0.40, 0.66 I²= 0%). Results of subgroup analysis based on study design and diagnosis showed conflicting results.</p><p><strong>Conclusions: </strong>There is conflicting evidence on the risk of thrombosis between PICC and CVC when used for hematological cancer patients. There could be a tendency of higher risk of thrombosis with PICC which needs to be confirmed by further studies. However, the use of PICC may reduce the risk of CLABSI in such patients. The quality of evidence is low owing to the predominance of observational studies with high inter-study heterogeneity.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2450572"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142983225","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":"Forecasting the incidence of acute lymphoid leukaemia in males and females in the Saudi population from 2020 to 2029: application of ARIMA models and public health implications.","authors":"Saeed M Kabrah, Budhi Handoko, Yasir Aljohani, Abdulrahman Mujalli, Mohammad Alobaidy, Arwa F Flemban, Wesam F Farrash, Abdulaziz H Alharbi, M S J Alzahrani","doi":"10.1080/16078454.2025.2538318","DOIUrl":"https://doi.org/10.1080/16078454.2025.2538318","url":null,"abstract":"<p><strong>Background: </strong>Acute lymphoid leukaemia (ALL) is a significant cause of morbidity and mortality globally, with increasing incidence rates observed in Saudi Arabia. Despite advances in treatment, there is a lack of localized, sex-specific forecasts to guide public health interventions.</p><p><strong>Objective: </strong>This study aims to forecast the future incidence of acute lymphoid leukaemia in males and females using ARIMA models.</p><p><strong>Methods: </strong>Saudi national cancer registries data from 1990 to 2019 were used. The dataset was divided into training (80%) and testing (20%) sets. ARIMA models were developed for male and female incidence, with model parameters determined using ACF and PACF plots. Stationarity was assessed using the Augmented Dickey-Fuller test, and model accuracy was validated using MAE, MSE, and MAPE. Forecasts included point estimates and 95% confidence intervals.</p><p><strong>Results: </strong>For males, the ARIMA (3, 3, 3) model forecasted an increase in ALL incidences from 957 cases in 2020 to 2181 cases in 2029. For females, the ARIMA (4, 3, 0) model projected an increase from 1019 cases in 2020 to 2159 cases in 2029. The models demonstrated high accuracy, with MAE of 1.19895 and 2.749188, MSE of 62.33 and 31.83, and MAPE of 0.6805807 and 1.443453 for males and females, respectively.</p><p><strong>Conclusion: </strong>Forecasts indicate a substantial rise in ALL incidence among both sexes, highlighting the urgent need for improved surveillance, early detection, and healthcare capacity planning. Incorporating ARIMA modelling into routine monitoring could support proactive resource allocation. Further studies should integrate additional predictive variables to enhance model precision.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2538318"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144707318","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":"A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A.","authors":"Johannes Oldenburg, Charles Hay, Flora Peyvandi, Anna-Elina Lehtinen, Ingrid Pabinger, Eveline Nüesch, Håkan Malmström, Eva Bednar, Stefan Lethagen","doi":"10.1080/16078454.2025.2513186","DOIUrl":"10.1080/16078454.2025.2513186","url":null,"abstract":"<p><strong>Background: </strong>Despite recent advances, factor replacement therapy remains a cornerstone in hemophilia A treatment. Efmoroctocog alfa, a recombinant FVIII Fc fusion protein (rFVIIIFc), has an extended half-life allowing higher FVIII levels and less frequent dosing than standard half-life (SHL) products, without increasing factor consumption.</p><p><strong>Methods: </strong>A-SURE was a 24-month prospective, non-interventional study assessing real-world effectiveness of rFVIIIFc prophylaxis. This post-hoc, intra-patient analysis included patients with hemophilia A (PwHA) who switched from SHL FVIII to rFVIIIFc prophylaxis. Effectiveness endpoints included annualised bleeding rate (ABR), annualised joint bleeding rate (AjBR), weekly injection frequency and weekly factor consumption.</p><p><strong>Results: </strong>Of 131 PwHA eligible for analysis, mean ABR and AjBR decreased from 3.7 and 2.4 to 1.8 and 1.1, respectively, after switching (mean [95% confidence interval (CI)] change of -1.9 [-3.0, -0.8] and -1.2 [-2.0, -0.5]). Mean weekly injection frequency decreased from 3.1 to 2.3 (mean [95% CI] change of -0.8 [-1.0, -0.7]); weekly factor consumption reduced from 89.7 to 84.1 international units (IU)/kg, respectively (mean [95% CI] change of -5.7 [-10.7, -0.6]). These trends were consistent across age groups.</p><p><strong>Conclusion: </strong>This intra-patient comparison demonstrates switching from SHL FVIII to rFVIIIFc prophylaxis reduces frequency of bleeds, injection frequency, and factor consumption, complementing previously reported data from A-SURE.<b>Trial registration:</b>ClinicalTrials.gov identifier: NCT02976753.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2513186"},"PeriodicalIF":1.6,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144567383","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":"Gilteritinib maintenance after allogeneic hematopoietic stem cell transplantation for relapsed/refractory acute myeloid leukemia with FLT3-internal tandem duplication mutation.","authors":"Chen Liang, Donglin Yang, Aiming Pang, Yi He, Rongli Zhang, Weihua Zhai, Sizhou Feng, Mingzhe Han, Suning Chen, Liping Dou, Yu Wang, Xiaojin Wu, Erlie Jiang","doi":"10.1080/16078454.2025.2509353","DOIUrl":"https://doi.org/10.1080/16078454.2025.2509353","url":null,"abstract":"<p><p><b>Background:</b> Patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with FLT3-Internal Tandem Duplication (ITD) mutation have a poor prognosis and high risk of relapse, even after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Prevention of disease relapse remains a challenge.<b>Objective:</b> To investigate the efficacy and tolerability of gilteritinib maintenance therapy in R/R AML patients with FLT3-ITD mutation.<b>Methods:</b> We retrospectively analyzed 96 AML patients who received allo-HSCT between 2017 and 2022. Patients were divided into two groups based on whether they received gilteritinib maintenance therapy or not. To minimize selection bias, we implemented propensity score matching and selected 80 propensity score-matched patients for comparison, 40 in each group. The therapeutic process and clinical outcomes were retrospectively analyzed.<b>Results:</b> All patient baseline and transplant characteristics were similar between the gilteritinib and the control group. Gilteritinib therapy conferred significant survival advantages (<i>P</i> = 0.013 for OS and <i>P</i> = 0.026 for RFS). Relapse remained the main reason of treatment failure with 3-year incidence of 25.0% (95%CI 12.8-39.2%) and 55.0% (95%CI 38.1-69.0%) for the gilteritinib group and the control group(<i>P</i> = 0.010). In multivariate Cox regression analysis, gilteritinib maintenance was the only factor associated with improved OS (HR = 0.395, <i>P</i> = 0.040) and RFS (HR = 0.447, <i>P</i> = 0.030).<b>Conclusions:</b> Our results indicated that gilteritinib maintenance therapy reduced the risk of relapse for FLT3-ITD mutated R/R AML. Compared with patients without maintenance therapy, gilteritinib treatment showed a similar incidence of NRM and GVHD, leading to significant survival advantages in this high-risk cohort of patients.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2509353"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144316807","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":"Comprehensive Hematological and molecular Characterization of hemoglobin Hekinan [α27(B8)Glu→Asp(α1), <i>HBA1</i>:c.84G > T] in a Large Thai cohort.","authors":"Amornchai Suksusut, Jidapa Jaitheang, Manussavee Prapphal, Pranee Sutcharitchan, Ponlapat Rojnuckarin, Noppacharn Uaprasert","doi":"10.1080/16078454.2025.2456679","DOIUrl":"10.1080/16078454.2025.2456679","url":null,"abstract":"<p><strong>Background: </strong>Hemoglobin (Hb) Hekinan is a prevalent α-globin variant frequently missed in thalassemia screening centers using high-performance liquid chromatography (HPLC) or capillary electrophoresis. This study aims to investigate the hematological and molecular characteristics of Hb Hekinan in a large cohort.</p><p><strong>Methods: </strong>Hb variants were identified using isoelectric focusing (IEF) and HPLC. Hb Hekinan was confirmed by direct DNA sequencing. Additional genetic determinants, including α-thalassemia, β-thalassemia and other variants, were detected using multiplex GAP-PCR, ARMS-PCR or direct DNA sequencing as appropriate.</p><p><strong>Results: </strong>Among 61,997 Hb typing samples, 149 cases of Hb Hekinan were identified in Thai individuals and classified into 8 genotypic groups. These included 104 Hb Hekinan heterozygotes, 10 Hb Hekinan coexisting with α⁺-thalassemia, 3 Hb Hekinan with non-deletional α-variants, 6 Hb Hekinan with α⁰-thalassemia, 21 double heterozygote for Hb Hekinan and HbE, 3 Hb Hekinan with β-thalassemia trait, 1 triple heterozygotes (Hb Hekinan/α⁰-thalassemia/Hb E) and 1 quadruple heterozygote for Hb Hekinan/α⁺-thalassemia/Hb E/Hb Hope. Hb Hekinan was well-separated from Hb A using IEF but was frequently missed with HPLC. On HPLC, Hb Hekinan could only be identified when coexisting with α⁰-thalassemia. All cases presented with either normal Hb levels or mild anemia.</p><p><strong>Conclusions: </strong>Hb Hekinan is a prevalent α-globin variant that is often undetected by HPLC but reliably identified using IEF. These findings highlight the importance of incorporating IEF for accurate diagnosis of Hb Hekinan. Most cases are clinically benign, even when interacting with other thalassemia syndromes or Hb variants.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2456679"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143058992","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":"Eculizumab treatment for Chinese patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH): efficacy and safety - a single-center study.","authors":"Leyu Wang, Ziwei Liu, Chen Yang, Miao Chen, Bing Han","doi":"10.1080/16078454.2025.2450575","DOIUrl":"10.1080/16078454.2025.2450575","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate the short-term efficacy and safety of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China.</p><p><strong>Method: </strong>Data were retrospectively collected from patients with PNH who received at least 3 months of full-dose eculizumab. Changes in clinical and laboratory indicators after 1, 3, and 6 months of eculizumab therapy and at the end of follow-up were documented. The incidence rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and adverse events were recorded.</p><p><strong>Result: </strong>A total of 48 patients, including 27 males, with a median age of 46 (12-78) years were included. Twenty-four (50%) patients had classic PNH and 24 (50%) had bone marrow failure (BMF)/PNH. Eighteen (37.5%) patients required blood transfusion. The median duration of follow-up was 6 (3-15) months. During the follow-up period, Lactate Dehydrogenase (LDH) levels were lower than those at baseline (<0.05) at all observation points. The patients showed a significant reduction in creatinine levels from baseline (<i>P</i> = 0.022 and <i>P</i> = 0.039, respectively) at 1 and 3 months. At the end of the follow-up, fifteen (83.3%) became transfusion-independent. No new thrombotic events were observed. The FACIT-Fatigue score significantly improved (<i>P</i> < 0.05). No significant differences were observed in the changes in hemoglobin or LDH levels between patients with classic PNH and those with BMF/PNH. BTH was observed in 17.4% of patients and EVH in 10.4%. Mild adverse events occurred in 22.9% of patients. No deaths or clonal evolution was observed.</p><p><strong>Conclusion: </strong>Eculizumab can effectively control the hemolytic symptoms of PNH with good tolerance for Chinese patients.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2450575"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143065405","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":"Clinical trial participation, clinical care, and patient outcomes by practice setting: a real-world database analysis of patients with Chronic Lymphocytic Leukemia or Mantle Cell Lymphoma.","authors":"Debora S Bruno, Manoj Khanal, Xiaohong Li, Maricer P Escalon, Katherine Winfree, Lisa M Hess","doi":"10.1080/16078454.2025.2457809","DOIUrl":"10.1080/16078454.2025.2457809","url":null,"abstract":"<p><strong>Objective: </strong>This study was designed to compare treatment patterns, clinical trial participation, and clinical outcomes among patients with small lymphocytic lymphoma/chronic lymphocytic leukemia (CLL) or Mantle cell lymphoma (MCL) by site of care.</p><p><strong>Methods: </strong>A nationwide electronic health record (EHR)-derived de-identified database was utilized for this study. Eligible patients were diagnosed with either CLL or MCL from 2013-2022 who received systemic therapy for their disease. Overall survival (OS) was analyzed using Kaplan Meier method, censoring patients without events at last observation in the database. Cox proportional hazards regression model was used to adjust for baseline covariates.</p><p><strong>Results: </strong>A total of 6,372 patients with CLL and 3,411 with MCL met eligibility criteria for this analysis; 13.9% and 22.2%, respectively, were treated in academic settings. Academic settings were associated with higher patient volume and were more likely to treat MCL with CAR-T, enroll patients with CLL or MCL to clinical trials, and care for patients who were younger, White, and for CLL with higher rates of del(17p) mutations (all <i>p</i> < 0.01). Survival was significantly longer among patients treated in academic vs community settings (median OS not reached vs 80.5 months for CLL; 95.6 vs 68.7 months for MCL from start of first-line therapy).</p><p><strong>Discussion: </strong>Patients who received care in academic settings differed from those treated in the community; care in academic settings was associated with significantly longer OS and higher trial participation. Further research is warranted to better understand the factors that may contribute to the observed outcomes.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2457809"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255630","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":"Prognostic significance of neutrophil extracellular trap-related genes in childhood acute lymphoblastic leukemia: insights from multi-omics and in vitro experiment.","authors":"Cheng Chen, Yu Ma, Yadai Gao, Huiqing Ge, Xiaochun Zhang","doi":"10.1080/16078454.2025.2452701","DOIUrl":"10.1080/16078454.2025.2452701","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to develop a prognostic model based on extracellular trap-related genes (NETRGs) for patients with cALL.</p><p><strong>Methods: </strong>Data from the TARGET-ALL-P2 and TARGET-ALL-P3 cohorts in the Genomic Data Commons database, the transcriptome dataset GSE26713, the single-cell transcriptome dataset GSE130116 from the Gene Expression Omnibus database and 306 NETRGs identified were analysed. Differentially expressed genes (DEGs) were identified from GSE26713 and differentially expressed NETRGs (DE-NETRGs) were obtained by overlapping DEGs with NETRGs. Functional analyses were conducted. Key feature genes were identified through univariate and least absolute shrinkage and selection operator (LASSO) regression. Prognostic genes were determined via multivariate Cox regression analysis, followed by the construction and validation of a risk model and nomogram. Additional analyses included immune profiling, drug sensitivity, functional differences, cell-type-specific expression, enrichment analysis and RT-qPCR.</p><p><strong>Results: </strong>A total of 1,270 DEGs were identified in GSE26713, of which 74 overlapped with NETRGs. Seven prognostic genes were identified using univariate, LASSO and multivariate Cox regression analyses. Survival analysis revealed lower survival rates in the high-risk group. Independent prognostic analysis identified risk scores and primary diagnosis as independent predictors of prognosis. Immune cell profiling showed significant differences in cell populations such as aDCs, eosinophils and Th2 cells between risk groups. Six cell subtypes were annotated, with prognostic genes predominantly expressed in myeloid cells. RT-qPCR revealed that PTAFR, FCGR2A, RETN and CAT were significantly downregulated, while TLR2 and S100A12 were upregulated in cALL.</p><p><strong>Conclusion: </strong>TLR2, PTAFR, FCGR2A, RETN, S100A12 and CAT may serve as potential therapeutic targets.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2452701"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143004633","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":"Targeting NEDD8 in pediatric acute myeloid leukemia: an integrated bioinformatics and experimental approach.","authors":"Jian Sun, Cui Liu, Guangli Yang, Qian Li, Yang An, Yin Zhu, Pingping Zhang, Yaning Guan, Chang Peng, Zuochen Du, Pei Huang, Yan Chen","doi":"10.1080/16078454.2025.2478650","DOIUrl":"10.1080/16078454.2025.2478650","url":null,"abstract":"<p><p>SUMMARYThis study systematically explored the role of NEDD8 in pediatric acute myeloid leukemia (AML) through patient sample analysis, database mining, and in vitro experiments. Our results demonstrated that NEDD8 was significantly overexpressed in newly diagnosed pediatric AML patients and was associated with poor survival outcomes. Functional enrichment analysis of the TARGET database further revealed a strong correlation between NEDD8 and cancer-related pathways. In vitro experiments showed that NEDD8 knockdown significantly inhibited the proliferation of AML cells (THP-1 and MV4-11) and induced cell cycle arrest. Collectively, these findings highlight the critical role of NEDD8 in pediatric AML pathogenesis and suggest its potential as both a prognostic biomarker and a therapeutic target.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"30 1","pages":"2478650"},"PeriodicalIF":2.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143657094","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}