Hematology最新文献

筛选
英文 中文
Characterization of a novel α-thalassemia deletion (Dongxing deletion; --DX) by CNV-seq in a Chinese female with Hb H disease: a case report. 用CNV-seq检测中国女性Hb H病患者α-地中海贫血缺失(东兴缺失;—DX)的特征:1例报告
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-06-19 DOI: 10.1080/16078454.2026.2692179
Youqiong Li, Ting Qin, Liang Liang, Ying Li, Lihong Zheng
{"title":"Characterization of a novel α-thalassemia deletion (Dongxing deletion; --<sup>DX</sup>) by CNV-seq in a Chinese female with Hb H disease: a case report.","authors":"Youqiong Li, Ting Qin, Liang Liang, Ying Li, Lihong Zheng","doi":"10.1080/16078454.2026.2692179","DOIUrl":"10.1080/16078454.2026.2692179","url":null,"abstract":"<p><strong>Objective: </strong>This study was initiated to resolve a discrepancy between conventional genetic findings and clinical presentation in a proband with <i>α</i>-thalassemia. The initial genotype (-α<sup>4.2</sup>/-α<sup>4.2</sup>) was inconsistent with the Hb H disease phenotype, suggesting the presence of an undetected pathogenic variant.</p><p><strong>Methods: </strong>To characterize the underlying genetic defect, we employed a multi-technique approach. The proband's DNA was analyzed using multiplex ligation-dependent probe amplification (MLPA), third-generation sequencing (TGS), and copy number variation sequencing (CNV-seq). Prenatal diagnosis was performed because the husband carried the --<sup>SEA</sup>/αα genotype.</p><p><strong>Results: </strong>Investigations revealed a novel, large deletion on the <i>α</i>-globin gene cluster. Unlike MLPA and TGS, which indicated a deletion but could not precisely define its breakpoints due to its telomeric location, CNV-seq successfully mapped the deletion to chr16:97840-230167. This represents a 132,327 bp (132 kb) deletion, which we designated the 'Dongxing' (--<sup>DX</sup>) deletion. The proband's corrected genotype was confirmed as --<sup>DX</sup>/-α<sup>4.2</sup>. Prenatal diagnosis revealed the fetus had Hb H disease (--<sup>SEA</sup>/-α<sup>4.2</sup>).</p><p><strong>Conclusions: </strong>We identified and characterized a novel <i>α</i>-thalassemia deletion (Dongxing deletion) using CNV-seq. This case highlights the limitations of conventional genotyping and underscores the importance of comprehensive genetic analysis in cases of genotype-phenotype discrepancy.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2692179"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148283585","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}
引用次数: 0
Correction. 修正。
IF 1.6 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-04-01 DOI: 10.1080/16078454.2026.2654947
{"title":"Correction.","authors":"","doi":"10.1080/16078454.2026.2654947","DOIUrl":"10.1080/16078454.2026.2654947","url":null,"abstract":"","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2654947"},"PeriodicalIF":1.6,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147591799","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}
引用次数: 0
Temporal trends and global comparison of the burden of thalassemia in China: an analysis of the Global Burden Of Disease Study 2021. 中国地中海贫血负担的时间趋势和全球比较:对2021年全球疾病负担研究的分析
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-07-10 DOI: 10.1080/16078454.2026.2692175
Changqun He, Meiting Long, Yiming Yang, Zhenglian Yao, Linqiang Shen, Fufei Dai, Rong Gao, Xiaoyan Yang
{"title":"Temporal trends and global comparison of the burden of thalassemia in China: an analysis of the Global Burden Of Disease Study 2021.","authors":"Changqun He, Meiting Long, Yiming Yang, Zhenglian Yao, Linqiang Shen, Fufei Dai, Rong Gao, Xiaoyan Yang","doi":"10.1080/16078454.2026.2692175","DOIUrl":"https://doi.org/10.1080/16078454.2026.2692175","url":null,"abstract":"<p><strong>Objective: </strong>This study aims to analyze the temporal trends, age‒sex patterns, and disease burden of thalassemia in China from 1990 to 2021 using the Global Burden of Disease (GBD) 2021 data, so as to optimize resource allocation and health strategy planning.</p><p><strong>Methods: </strong>Data on incidence, prevalence, death, and disability-adjusted life years (DALYs) for thalassemia in China and worldwide are extracted from the GBD 2021 database. Trends are analyzed using Joinpoint regression, and the autoregressive integrated moving average (ARIMA) model is applied to forecast the burden over the next 15 years.</p><p><strong>Results: </strong>Compared to the global average, China demonstrates a more pronounced decline in age-standardized mortality rate (ASMR) and age-standardized DALYs rate (ASDR). In contrast, the age-standardized incidence rate (ASIR) and age-standardized prevalence rate (ASPR) remained stable and significantly higher than the global level. The disease burden was higher in males and concentrated in children under five, with a visible shift toward older age groups. Projections indicate a potential rise in ASIR, stable ASPR with minor fluctuations, and continued decline in ASMR and ASDR over the next 15 years.</p><p><strong>Conclusion: </strong>China has made significant progress in reducing the disease burden associated with thalassemia. However, the disease burden remains higher than the global average, with persistent disparities by sex. These findings underscore the need for sustained and targeted public health strategies.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2692175"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148420964","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}
引用次数: 0
Clinical characteristics, treatment options, and prognosis of subcutaneous panniculitis-like T-cell lymphoma from the SEER database. 来自SEER数据库的皮下泛膜炎样t细胞淋巴瘤的临床特征、治疗选择和预后。
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-03-24 DOI: 10.1080/16078454.2026.2648344
Lijuan Zhang, Zehui Chen, Longhua Lu, Bing Xiang
{"title":"Clinical characteristics, treatment options, and prognosis of subcutaneous panniculitis-like T-cell lymphoma from the SEER database.","authors":"Lijuan Zhang, Zehui Chen, Longhua Lu, Bing Xiang","doi":"10.1080/16078454.2026.2648344","DOIUrl":"10.1080/16078454.2026.2648344","url":null,"abstract":"<p><strong>Introduction: </strong>Subcutaneous panniculitis-like T-cell lymphoma (SPTCL) is a rare type of cutaneous lymphoma lacking standardized treatments. Consequently, patient outcomes vary significantly.</p><p><strong>Methods: </strong>This study explored the clinical characteristics and prognostic factors of 205 SPTCL patients from 2000 to 2021 in the Surveillance, Epidemiology, and End Results (SEER) database.</p><p><strong>Results: </strong>Overall survival (OS) at 1, 3, and 5 years were 78.3%, 75.7%, and 66.4%, respectively. Patients diagnosed after 2008 (possibly excluding the γ-δ subtype) (HR = 0.197, 95% CI = 0.106-0.364, <i>p</i> = 0.000) and Asian or Pacific Islanders (HR = 0.210, 95% CI = 0.049-0.902, <i>p</i> = 0.036) were independent predictors of favorable survival, whereas age 50-60 years (HR = 3.213, 95% CI = 1.357-7.607, <i>p</i> = 0.008) and age > 60 years (HR = 5.039, 95% CI = 2.327-10.911, <i>p</i> = 0.000) were independently associated with poor survival. Patients who received radiotherapy alone exhibited a significantly lower hazard risk compared to those receiving no chemotherapy or radiation (HR = 0.216, 95% CI = 0.048-0.983, <i>p</i> = 0.048). No statistically significant differences in prognosis were observed between patients who received no chemotherapy or radiation and those who received either chemotherapy alone (HR = 1.276, 95% CI = 0.644-2.529, <i>p</i> = 0.485) or radiochemotherapy (HR = 1.283, 95% CI = 0.463-3.558, <i>p</i> = 0.632). These associations persisted after IPTW adjustment, with age, race, year of diagnosis, and treatment remaining independent predictors of OS in SPTCL.</p><p><strong>Conclusions: </strong>The Ann Arbor staging was not suitable for SPTCL. Radiotherapy represents an appropriate therapeutic option for patients with single or localized skin lesions. No statistically significant differences in prognosis were observed between patients who received no chemotherapy or radiation and those who received either chemotherapy alone or radiochemotherapy. This finding suggests that immunomodulatory agents may be preferable to cytotoxic therapy as initial treatment for SPTCL, an inflammatory lymphoma.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2648344"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147511704","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}
引用次数: 0
A survival prognostic model for high-risk myelodysplastic syndrome patients treated with decitabine. 地西他滨治疗高危骨髓增生异常综合征患者的生存预后模型。
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-04-13 DOI: 10.1080/16078454.2026.2657628
Yumei Liu, Haiyue Niu, Yansong Wei, Liyan Yang, Huijuan Jiang, Lijuan Li, Limin Xing, Zonghong Shao, Rong Fu, Huaquan Wang
{"title":"A survival prognostic model for high-risk myelodysplastic syndrome patients treated with decitabine.","authors":"Yumei Liu, Haiyue Niu, Yansong Wei, Liyan Yang, Huijuan Jiang, Lijuan Li, Limin Xing, Zonghong Shao, Rong Fu, Huaquan Wang","doi":"10.1080/16078454.2026.2657628","DOIUrl":"10.1080/16078454.2026.2657628","url":null,"abstract":"<p><strong>Background: </strong>Decitabine is widely used in China for high-risk myelodysplastic syndromes (MDS). However, the prognostic factors associated with overall survival (OS) remain incompletely defined.</p><p><strong>Methods: </strong>We retrospectively analyzed 130 high-risk MDS patients treated with decitabine. Clinical characteristics and laboratory parameters were collected. Treatment response was assessed, and survival outcomes were evaluated using Cox regression models. A prognostic nomogram was developed to improve risk stratification beyond the conventional IPSS/IPSS-R systems.</p><p><strong>Results: </strong>The median age was 60.5 years, with 57.7% male patients. Median OS was 15.5 months. Univariate analysis showed that age ≥50 years, bone marrow blasts ≥10%, and iron overload (serum ferritin ≥500 µg/L) were significantly associated with inferior OS. Multivariate Cox regression confirmed age ≥50 (HR = 2.31, 95% CI: 1.21-4.40, <i>p </i>= 0.011) and elevated bone marrow blasts (HR = 1.92, 95% CI: 1.16-3.17, <i>p </i>= 0.012) as independent adverse prognostic factors. Poor karyotype also conferred poor outcomes. Next-generation sequencing data were available for 92 patients. Given the low mutation rate, the mutational analysis was conducted exploratorily; results suggested that TP53 mutations were significantly associated with adverse prognosis. A prognostic nomogram integrating age, ECOG score, bone marrow blasts, cytogenetics, hemoglobin, platelet count, absolute neutrophil count, ring sideroblasts, and ferritin demonstrated improved risk stratification, though validation was limited to internal cohorts.</p><p><strong>Conclusion: </strong>Decitabine provides short-term hematologic benefits in high-risk MDS, but overall prognosis remains poor. Age, bone marrow blasts, iron overload, and high-risk cytogenetics are key prognostic factors. Incorporating clinical, cytogenetic, and mutational variables into new prognostic models may enhance individualized treatment decision-making for high-risk MDS patients.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2657628"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147672675","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}
引用次数: 0
VGLL4 regulates the Wnt/β-catenin signaling pathway and acts as an oncogenic factor in acute myeloid leukemia. VGLL4调节Wnt/β-catenin信号通路,在急性髓系白血病中作为致瘤因子。
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-04-15 DOI: 10.1080/16078454.2026.2644710
Chen Zhou, Yujin Yin, Juan Gu, He Shen, Yuchu Yuan, Mingyan Wang
{"title":"VGLL4 regulates the Wnt/β-catenin signaling pathway and acts as an oncogenic factor in acute myeloid leukemia.","authors":"Chen Zhou, Yujin Yin, Juan Gu, He Shen, Yuchu Yuan, Mingyan Wang","doi":"10.1080/16078454.2026.2644710","DOIUrl":"10.1080/16078454.2026.2644710","url":null,"abstract":"<p><strong>Objective: </strong>To investigate the expression, function, and underlying mechanism of VGLL4 in AML.</p><p><strong>Methods: </strong>Bioinformatic analysis of TCGA data by GEPIA website was performed. VGLL4 protein expression was analyzed by Western blot. We then performed functional assays (CCK-8, colony formation, and Transwell) to evaluate the effects of VGLL4 knockdown and overexpression on the proliferation, migration, and Matrigel matrix penetration ability of AML cell lines. Apoptosis was assessed by flow cytometry, and the expression of key Wnt/β-catenin pathway proteins was examined by Western blot.</p><p><strong>Results: </strong>Compared with controls, VGLL4 was significantly upregulated in AML. Functionally, VGLL4 knockdown potently suppressed AML cell proliferation, migration, and Matrigel matrix penetration ability, promoted apoptosis, and downregulated the expression of key pathway proteins, including β-catenin and GSK3β.</p><p><strong>Conclusion: </strong>VGLL4 acts as an oncogenic factor in AML, potentially through activating the Wnt/β-catenin pathway. Thus, we propose VGLL4 as a promising molecular target for AML therapy.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2644710"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147689895","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}
引用次数: 0
The prognostic value of AQP1 in hyperbilirubinemia related to ABO hemolytic disease of the newborn. AQP1在新生儿ABO溶血性疾病相关高胆红素血症中的预后价值
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-05-09 DOI: 10.1080/16078454.2026.2665904
Zhenzhen Liu, Wei Shi, Jian Zhu, Lihong Liu
{"title":"The prognostic value of AQP1 in hyperbilirubinemia related to ABO hemolytic disease of the newborn.","authors":"Zhenzhen Liu, Wei Shi, Jian Zhu, Lihong Liu","doi":"10.1080/16078454.2026.2665904","DOIUrl":"10.1080/16078454.2026.2665904","url":null,"abstract":"<p><strong>Background: </strong>ABO hemolytic disease of the newborn (ABO-HDN) is a major cause of hyperbilirubinemia in neonates, often inducing significant elevation of serum bilirubin levels shortly after birth.</p><p><strong>Purpose: </strong>This study aimed to assess the prognostic value of Aquaporin 1 (AQP1) for hyperbilirubinemia in ABO-HDN as reflected by the duration of phototherapy, a key clinical indicator of disease severity and treatment response.</p><p><strong>Methods: </strong>A retrospective cohort study was conducted, enrolling 168 neonates with ABO-HDN-related hyperbilirubinemia. Serum AQP1 expression was detected by RT-qPCR, and neonates were divided into a high-AQP1 expression group (<i>n</i> = 84) and a low-AQP1 expression group (<i>n</i> = 84) according to the median. All neonates were followed up for 7 days after the initiation of phototherapy, and a cure was defined as no total bilirubin (TBIL) rebound within 48 h after reaching the normal TBIL level. The cure rate was defined as the proportion of participants who achieved a cure within the 7‑day follow‑up period.</p><p><strong>Results: </strong>The AQP1 expression in the ABO-HDN group was significantly higher than that in the control group. Additionally, the high-AQP1 group had a longer cure time and a lower cure rate within 7 days. Spearman analysis showed that AQP1 was positively correlated with TBIL (R = 0.481, <i>P</i> < 0.001). Cox regression analysis confirmed that high AQP1 expression was associated with a reduced cure rate (HR = 0.623, 95%CI: 0.449-0.861, <i>P</i> = 0.004).</p><p><strong>Conclusion: </strong>High-AQP1 expression was closely associated with prolonged treatment duration in ABO-HDN-related hyperbilirubinemia. AQP1 can serve as a potential molecular marker for prognostic assessment and individualized treatment guidance in such neonates.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2665904"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147864023","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}
引用次数: 0
Outcomes of patients with myelofibrosis treated with ruxolitinib and anemia-supporting medications. 鲁索利替尼和贫血支持药物治疗骨髓纤维化患者的结局。
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-09-04 DOI: 10.1080/16078454.2026.2725339
Pankit Vachhani, Jennifer Repp, J E Hamer-Maansson, Evan Braunstein, Valkal Bhatt, Haifa Kathrin Al-Ali
{"title":"Outcomes of patients with myelofibrosis treated with ruxolitinib and anemia-supporting medications.","authors":"Pankit Vachhani, Jennifer Repp, J E Hamer-Maansson, Evan Braunstein, Valkal Bhatt, Haifa Kathrin Al-Ali","doi":"10.1080/16078454.2026.2725339","DOIUrl":"10.1080/16078454.2026.2725339","url":null,"abstract":"<p><strong>Objective: </strong>This post hoc analysis of the phase 3b JUMP trial evaluated addition of anemia-supporting medications to ruxolitinib in patients with myelofibrosis and anemia.</p><p><strong>Methods: </strong>101 patients with baseline hemoglobin <12.0 g/dL initiated an erythropoiesis-stimulating agent (ESA) or danazol <3 months post-enrollment and maintained ESA/danazol ≥3 months; 97% initiated ESAs. Patients enrolled in JUMP who had hemoglobin <12.0 g/dL but did not initiate ESAs/danazol within 3 months were evaluated as an unmatched comparator. Total JUMP population data were also analyzed for spleen length (≥50% reduction from baseline) and symptom response (≥6.5-point improvement in FACT-Lym score).</p><p><strong>Results: </strong>Baseline characteristics were similar to comparator JUMP patients (no ESAs/danazol within 3 months, <i>n</i> = 1242). Mean total daily ruxolitinib dose remained >25 mg. Week 24 spleen length response was achieved by 37% of patients; 26% achieved symptom response, similar to the comparator population (28% and 26%, respectively) and comparable to the total JUMP population (<i>N</i> = 2233). Outcomes were similar between the hemoglobin <12.0 g/dL analysis population and patients with baseline hemoglobin <10.0 g/dL (<i>n</i> = 52). In all groups, hemoglobin levels increased after Week 4 following an expected initial decrease.</p><p><strong>Discussion: </strong>This analysis suggests that patients treated with ruxolitinib and anemia-supporting care continued to receive optimal ruxolitinib dosing; spleen-length and symptom response rates in these patients were comparable to the overall JUMP population, the majority of whom did not have anemia.</p><p><strong>Conclusion: </strong>Results support use of anemia-supporting medications with ruxolitinib and may allow maintenance of ruxolitinib dose intensity in patients with myelofibrosis and anemia.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2725339"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148891520","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}
引用次数: 0
Teclistamab-associated adverse events: a disproportionality analysis of the FDA adverse event reporting system. teclistamab相关不良事件:FDA不良事件报告系统的歧化分析。
IF 2 4区 医学
Hematology Pub Date : 2026-12-31 Epub Date: 2026-09-15 DOI: 10.1080/16078454.2026.2730041
Xiaoyu He, Gang Chen, Zhengyu Zhan, Zhenjiang Li
{"title":"Teclistamab-associated adverse events: a disproportionality analysis of the FDA adverse event reporting system.","authors":"Xiaoyu He, Gang Chen, Zhengyu Zhan, Zhenjiang Li","doi":"10.1080/16078454.2026.2730041","DOIUrl":"https://doi.org/10.1080/16078454.2026.2730041","url":null,"abstract":"<p><strong>Introduction: </strong>Teclistamab, a B-cell maturation antigen (BCMA) × CD3 bispecific antibody, is effective in relapsed/refractory multiple myeloma, but its real-world post-marketing safety profile remains incompletely characterized.</p><p><strong>Objectives: </strong>To evaluate adverse event (AE) signals, temporal patterns, and clinical priorities associated with teclistamab using the FDA Adverse Event Reporting System (FAERS).</p><p><strong>Methods: </strong>We retrospectively analyzed FAERS reports from 2022 to 2025 using four disproportionality methods: reporting odds ratio, proportional reporting ratio, Bayesian confidence propagation neural network, and multi-item gamma Poisson shrinker. Time-to-onset, Weibull, Kaplan-Meier, and semi-quantitative clinical prioritization analyses were also performed.</p><p><strong>Results: </strong>Among 2,631 reports, 85.2% involved serious outcomes and 21.7% fatal outcomes. The most frequently reported system organ class was infections and infestations. Seventy significant preferred-term signals were identified, including 52 previously documented and 18 novel signals. Cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, infection, pneumonia, pyrexia, and neutropenia were most frequently reported. Sepsis was the sole high-priority signal, with fatal outcomes in 58.57% of sepsis-related reports. Immune-related and neurological events occurred earlier, whereas infectious complications tended to occur later. Safety profiles were generally consistent across sex and age groups, although septic shock was less frequently reported in females.</p><p><strong>Discussion: </strong>The safety profile broadly aligned with clinical trial data, but novel signals and sepsis-related fatality highlighted clinically important risks.</p><p><strong>Conclusion: </strong>These findings support targeted risk mitigation and continued monitoring, particularly for severe infections and immune-related toxicities.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2730041"},"PeriodicalIF":2.0,"publicationDate":"2026-12-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148968203","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}
引用次数: 0
Leukemia-derived exosomes induce immunosuppression of dendritic cell function via TGFB2-MRPL58 axis. 白血病来源的外泌体通过TGFB2-MRPL58轴诱导树突状细胞功能的免疫抑制。
IF 2 4区 医学
Hematology Pub Date : 2026-12-01 Epub Date: 2026-01-20 DOI: 10.1080/16078454.2026.2616554
Jinglong Lv, Yang Tao, Hui Zhong, Xiaohong Xie, Hongxia Chen, Zhudan Hou, Wenfang Luo, Yangjun Liu, Rui Huang
{"title":"Leukemia-derived exosomes induce immunosuppression of dendritic cell function via TGFB2-MRPL58 axis.","authors":"Jinglong Lv, Yang Tao, Hui Zhong, Xiaohong Xie, Hongxia Chen, Zhudan Hou, Wenfang Luo, Yangjun Liu, Rui Huang","doi":"10.1080/16078454.2026.2616554","DOIUrl":"10.1080/16078454.2026.2616554","url":null,"abstract":"<p><strong>Objectives: </strong>To elucidate the mechanisms by which leukemia-derived exosomes induce immunosuppression in dendritic cells (DCs) and identify potential therapeutic targets.</p><p><strong>Methods: </strong>The optimal exosome dosage (20 µg/ml) for DC treatment was determined via gradient concentration experiments. Transcriptomics and qPCR validation explored molecular pathways. DC phenotype was assessed for maturation markers (CD83/CD86), antigen presentation (CD1a), and immune receptors (TLR2). Cytokine levels (IL-6, IL-17, TNF-α, IL-4) were quantified. Multi-omics analysis identified key signaling pathways. Clinical validation utilized samples from AML patients.</p><p><strong>Results: </strong>Exosome treatment induced broad DC immunosuppression, characterized by significant downregulation of pro-inflammatory cytokines (IL-6, IL-17, TNF-α) and upregulation of anti-inflammatory IL-4. Phenotypic analysis revealed selective inhibition of CD1a and TLR2, while maturation markers (CD83/CD86) remained unaltered. Multi-omics identified TNF-β signaling as the primary immunosuppressive pathway. qPCR confirmed elevated TGF-β2 and mitochondrial ribosomal protein MRPL58, alongside reduced TLR2. Clinical studies in AML patients validated upregulation of TGF-β2, MRPL58, and exosomal markers. Exosomes impaired DC antigen presentation, immune response, and metabolic activity.</p><p><strong>Discussion: </strong>MRPL58 is a novel mediator of exosome-driven immunosuppression, implicating metabolic reprograming. Selective CD1a/TLR2 inhibition suggests exosomes evade immune detection while permitting DC maturation, a strategy favoring leukemia immune escape. Targeting exosome-DC interactions (e.g. blocking exosomal signals or MRPL58) may restore anti-tumor immunity.</p><p><strong>Conclusion: </strong>Leukemia exosomes suppress DC function primarily through MRPL58-associated metabolic modulation and TNF-β signaling. MRPL58 represents a promising therapeutic target. Disrupting exosome-mediated immunosuppression could enhance DC-based vaccines and combination immunotherapies for leukemia.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2616554"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146010198","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}
引用次数: 0
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信
小红书