首页 > 最新文献

Annals of Hematology最新文献

英文 中文
Vitamin D deficiency in sickle cell disease: a neglected comorbidity in Tunisia 镰状细胞病的维生素D缺乏:突尼斯被忽视的合并症
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-12 DOI: 10.1007/s00277-026-06750-6
Mariem Othmani, Yessine Amri, Yosr Jouini, Faida Ouali, Siwar Chelbi, Sondess Hadj Fredj, Rym Dabboubi, Taieb Messaoud

Sickle cell disease (SCD) is a hereditary hemoglobinopathy that imposes a significant burden on global health. Individuals with SCD often experience multiple nutritional deficiencies, among which vitamin D deficiency has emerged as a critical yet under recognized factor contributing to disease complications. This study aimed to assess the vitamin D status in patients with SCD compared to a matched healthy control group, and to explore its correlation with clinical and biological parameters, including the frequency of vaso-occlusive crises (VOC). A cross-sectional study was conducted between January and June 2024 at Bechir Hamza Children’s Hospital, Tunis. Sixty-four SCD patients and 63 healthy controls were enrolled. Vitamin D [25(OH)D] levels, along with hematologic and biochemical markers, were measured. Statistical analyses included t-tests, chi-square test, ANOVA, and Spearman correlation. Vitamin D levels were significantly lower in the SCD group (15.05 ± 6.6 ng/mL) compared to controls (22.51 ± 12.54 ng/mL, p = 0.026). Among SCD patients, 78.2% (50) had Vitamin D deficiency (< 20ng/mL), 20.3% (13) had Vitamin D insuffisancy (20–30 ng/mL) and only one patient (1.5%) had normal vitamin D status (> 30 ng/mL). Vitamin D levels were inversely correlated with age, total bilirubin, parathyroid hormone (PTH), and frequency of VOC, and positively correlated with hemoglobin concentration and socioeconomic status. SCD patients with vitamin D deficiency experienced significantly more VOC episodes per year. Vitamin D deficiency is highly prevalent among SCD patients and is associated with increased disease severity. Routine screening and appropriate supplementation should be considered as part of comprehensive SCD management strategies.

镰状细胞病(SCD)是一种遗传性血红蛋白病,对全球健康造成重大负担。SCD患者经常经历多种营养缺乏,其中维生素D缺乏已成为导致疾病并发症的关键因素,但尚未得到充分认识。本研究旨在评估SCD患者与匹配健康对照组的维生素D状态,并探讨其与临床和生物学参数的相关性,包括血管闭塞危象(VOC)的频率。一项横断面研究于2024年1月至6月在突尼斯Bechir Hamza儿童医院进行。64名SCD患者和63名健康对照者入组。测量维生素D [25(OH)D]水平,以及血液学和生化指标。统计分析包括t检验、卡方检验、方差分析和Spearman相关。SCD组的维生素D水平(15.05±6.6 ng/mL)明显低于对照组(22.51±12.54 ng/mL, p = 0.026)。在SCD患者中,78.2%(50例)存在维生素D缺乏症(20ng/mL), 20.3%(13例)存在维生素D不足(20 ~ 30ng /mL),仅有1例(1.5%)维生素D正常(30ng /mL)。维生素D水平与年龄、总胆红素、甲状旁腺激素(PTH)和VOC频率呈负相关,与血红蛋白浓度和社会经济地位呈正相关。维生素D缺乏的SCD患者每年出现的VOC发作明显更多。维生素D缺乏症在SCD患者中非常普遍,并与疾病严重程度增加有关。常规筛查和适当补充应被视为SCD综合管理策略的一部分。
{"title":"Vitamin D deficiency in sickle cell disease: a neglected comorbidity in Tunisia","authors":"Mariem Othmani,&nbsp;Yessine Amri,&nbsp;Yosr Jouini,&nbsp;Faida Ouali,&nbsp;Siwar Chelbi,&nbsp;Sondess Hadj Fredj,&nbsp;Rym Dabboubi,&nbsp;Taieb Messaoud","doi":"10.1007/s00277-026-06750-6","DOIUrl":"10.1007/s00277-026-06750-6","url":null,"abstract":"<div><p>Sickle cell disease (SCD) is a hereditary hemoglobinopathy that imposes a significant burden on global health. Individuals with SCD often experience multiple nutritional deficiencies, among which vitamin D deficiency has emerged as a critical yet under recognized factor contributing to disease complications. This study aimed to assess the vitamin D status in patients with SCD compared to a matched healthy control group, and to explore its correlation with clinical and biological parameters, including the frequency of vaso-occlusive crises (VOC). A cross-sectional study was conducted between January and June 2024 at Bechir Hamza Children’s Hospital, Tunis. Sixty-four SCD patients and 63 healthy controls were enrolled. Vitamin D [25(OH)D] levels, along with hematologic and biochemical markers, were measured. Statistical analyses included t-tests, chi-square test, ANOVA, and Spearman correlation. Vitamin D levels were significantly lower in the SCD group (15.05 ± 6.6 ng/mL) compared to controls (22.51 ± 12.54 ng/mL, <i>p</i> = 0.026). Among SCD patients, 78.2% (50) had Vitamin D deficiency (&lt; 20ng/mL), 20.3% (13) had Vitamin D insuffisancy (20–30 ng/mL) and only one patient (1.5%) had normal vitamin D status (&gt; 30 ng/mL). Vitamin D levels were inversely correlated with age, total bilirubin, parathyroid hormone (PTH), and frequency of VOC, and positively correlated with hemoglobin concentration and socioeconomic status. SCD patients with vitamin D deficiency experienced significantly more VOC episodes per year. Vitamin D deficiency is highly prevalent among SCD patients and is associated with increased disease severity. Routine screening and appropriate supplementation should be considered as part of comprehensive SCD management strategies.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-026-06750-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145948079","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Hypomethylating agents alone or in combination with venetoclax in very elderly acute myeloid leukemia patients: less treatment, better care? 低甲基化药物单独或联合venetoclax治疗高龄急性髓系白血病患者:治疗少,护理好?
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-11 DOI: 10.1007/s00277-026-06737-3
Francesco Tarantini, Corinne Contento, Ernesto Vigna, Vera Carluccio, Giuseppina Greco, Crescenza Pasciolla, Lucia Ciuffreda, Giovanni Rossi, Marina Aurora Urbano, Alessandro D’Ambrosio, Lara Aprile, Vito Pier Gagliardi, Mario Delia, Immacolata Attolico, Paola Carluccio, Vincenzo Federico, Antonella Bruzzese, Nicola Di Renzo, Massimo Gentile, Giuseppe Tarantini, Anna Mele, Attilio Guarini, Lorella Maria Antonia Melillo, Angelo Michele Carella, Domenico Pastore, Ferdinando Frigeri, Alessandro Maggi, Cosimo Cumbo, Giorgina Specchia, Pellegrino Musto, Francesco Albano

Hypomethylating agents (HMA) alone or in combination with venetoclax (VEN) are a mainstay for disease control in elderly acute myeloid leukemia (AML). We evaluated the non-inferiority of HMA monotherapy compared to HMA/VEN combination in 227 AML patients aged ≥ 75 years receiving HMA or HMA/VEN combination. No difference in overall survival (OS) was observed between the two groups, with HMA monotherapy demonstrating statistical non-inferiority. HMA-treated patients with favorable performance status had longer OS. The HMA/VEN group experienced higher mortality and worse QoL. HMA monotherapy offers comparable survival outcomes to HMA/VEN with reduced toxicity in elderly AML patients.

低甲基化药物(HMA)单独或联合venetoclax (VEN)是老年急性髓性白血病(AML)疾病控制的主要手段。我们评估了227例年龄≥75岁接受HMA或HMA/VEN联合治疗的AML患者的HMA单药治疗与HMA/VEN联合治疗的非劣效性。两组总生存期(OS)无差异,HMA单药治疗显示统计学上的非劣效性。hma治疗的患者表现状态良好,OS较长。HMA/VEN组死亡率较高,生活质量较差。HMA单药治疗在老年AML患者中提供与HMA/VEN相当的生存结果,且毒性降低。
{"title":"Hypomethylating agents alone or in combination with venetoclax in very elderly acute myeloid leukemia patients: less treatment, better care?","authors":"Francesco Tarantini,&nbsp;Corinne Contento,&nbsp;Ernesto Vigna,&nbsp;Vera Carluccio,&nbsp;Giuseppina Greco,&nbsp;Crescenza Pasciolla,&nbsp;Lucia Ciuffreda,&nbsp;Giovanni Rossi,&nbsp;Marina Aurora Urbano,&nbsp;Alessandro D’Ambrosio,&nbsp;Lara Aprile,&nbsp;Vito Pier Gagliardi,&nbsp;Mario Delia,&nbsp;Immacolata Attolico,&nbsp;Paola Carluccio,&nbsp;Vincenzo Federico,&nbsp;Antonella Bruzzese,&nbsp;Nicola Di Renzo,&nbsp;Massimo Gentile,&nbsp;Giuseppe Tarantini,&nbsp;Anna Mele,&nbsp;Attilio Guarini,&nbsp;Lorella Maria Antonia Melillo,&nbsp;Angelo Michele Carella,&nbsp;Domenico Pastore,&nbsp;Ferdinando Frigeri,&nbsp;Alessandro Maggi,&nbsp;Cosimo Cumbo,&nbsp;Giorgina Specchia,&nbsp;Pellegrino Musto,&nbsp;Francesco Albano","doi":"10.1007/s00277-026-06737-3","DOIUrl":"10.1007/s00277-026-06737-3","url":null,"abstract":"<div><p>Hypomethylating agents (HMA) alone or in combination with venetoclax (VEN) are a mainstay for disease control in elderly acute myeloid leukemia (AML). We evaluated the non-inferiority of HMA monotherapy compared to HMA/VEN combination in 227 AML patients aged ≥ 75 years receiving HMA or HMA/VEN combination. No difference in overall survival (OS) was observed between the two groups, with HMA monotherapy demonstrating statistical non-inferiority. HMA-treated patients with favorable performance status had longer OS. The HMA/VEN group experienced higher mortality and worse QoL. HMA monotherapy offers comparable survival outcomes to HMA/VEN with reduced toxicity in elderly AML patients.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-026-06737-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145948080","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The effects of adding decitabine to conditioning regimen for patients with acute myeloid leukemia or myelodysplastic syndrome undergoing allogeneic hematopoietic stem cell transplantation: a systematic review and meta-analysis 在异基因造血干细胞移植的急性髓系白血病或骨髓增生异常综合征患者的调理方案中加入地西他滨的效果:系统回顾和荟萃分析。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-23 DOI: 10.1007/s00277-025-06342-w
Chengxin Luo, Jianmin Zhang, Xiangtao Huang, Guixian Wu, Yarui Huang, Yaqun Ding, Zhen Huang, Qiuyue Song, Jieping Chen, Xi Li, Shuangnian Xu

Decitabine shows favorable efficacy in induction and maintenance therapy for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), but its role in pre-transplant conditioning still has not been established. We performed a systematic review and meta-analysis of comparative studies investigating the effects of adding decitabine to conditioning regimens in AML/MDS patients undergoing allogeneic hematopoietic stem cell transplantation. Meta-analysis was conducted with Review Manager 5.4. For time-to-event outcomes, pooled hazard ratios (HRs) were calculated using generic inverse-variance method. For dichotomous data, pooled risk ratios (RRs) were calculated using Mantel-Haenszel method. Fixed-effects model was adopted if there is no significant heterogeneity. Literature search and study selection identify 9 eligible studies, including 2 randomized controlled trials and 7 retrospective comparative studies. Meta-analysis show that addition of decitabine to conditioning regimen is associated with significantly better overall survival (HR 0.61; 95% CI: 0.49–0.76; P < 0.00001), reduced risk of relapse (HR 0.58; 95% CI: 0.46–0.73; P < 0.00001), and better disease-free survival (HR 0.67; 95% CI: 0.55–0.81; P < 0.0001) without increasing non-relapse mortality (HR 0.82; 95% CI: 0.56–1.18; P = 0.28) and grade II-IV acute graft-versus-host disease (RR 0.75; 95% CI: 0.54–1.04; P = 0.08).

地西他滨在急性髓性白血病(AML)和骨髓增生异常综合征(MDS)患者的诱导和维持治疗中显示出良好的疗效,但其在移植前调节中的作用尚未确定。我们对进行同种异体造血干细胞移植的AML/MDS患者的调节方案中添加地西他滨的效果进行了系统回顾和荟萃分析。采用Review Manager 5.4进行meta分析。对于时间-事件结局,采用通用反方差法计算合并风险比(hr)。对于二分类数据,采用Mantel-Haenszel方法计算合并风险比(pooled risk ratio, rr)。如果不存在显著异质性,则采用固定效应模型。文献检索和研究选择共获得9项符合条件的研究,包括2项随机对照试验和7项回顾性比较研究。荟萃分析显示,在调理方案中加入地西他滨可显著提高总生存率(HR 0.61; 95% CI: 0.49-0.76; P
{"title":"The effects of adding decitabine to conditioning regimen for patients with acute myeloid leukemia or myelodysplastic syndrome undergoing allogeneic hematopoietic stem cell transplantation: a systematic review and meta-analysis","authors":"Chengxin Luo,&nbsp;Jianmin Zhang,&nbsp;Xiangtao Huang,&nbsp;Guixian Wu,&nbsp;Yarui Huang,&nbsp;Yaqun Ding,&nbsp;Zhen Huang,&nbsp;Qiuyue Song,&nbsp;Jieping Chen,&nbsp;Xi Li,&nbsp;Shuangnian Xu","doi":"10.1007/s00277-025-06342-w","DOIUrl":"10.1007/s00277-025-06342-w","url":null,"abstract":"<div><p>Decitabine shows favorable efficacy in induction and maintenance therapy for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), but its role in pre-transplant conditioning still has not been established. We performed a systematic review and meta-analysis of comparative studies investigating the effects of adding decitabine to conditioning regimens in AML/MDS patients undergoing allogeneic hematopoietic stem cell transplantation. Meta-analysis was conducted with Review Manager 5.4. For time-to-event outcomes, pooled hazard ratios (HRs) were calculated using generic inverse-variance method. For dichotomous data, pooled risk ratios (RRs) were calculated using Mantel-Haenszel method. Fixed-effects model was adopted if there is no significant heterogeneity. Literature search and study selection identify 9 eligible studies, including 2 randomized controlled trials and 7 retrospective comparative studies. Meta-analysis show that addition of decitabine to conditioning regimen is associated with significantly better overall survival (HR 0.61; 95% CI: 0.49–0.76; <i>P</i> &lt; 0.00001), reduced risk of relapse (HR 0.58; 95% CI: 0.46–0.73; <i>P</i> &lt; 0.00001), and better disease-free survival (HR 0.67; 95% CI: 0.55–0.81; <i>P</i> &lt; 0.0001) without increasing non-relapse mortality (HR 0.82; 95% CI: 0.56–1.18; <i>P</i> = 0.28) and grade II-IV acute graft-versus-host disease (RR 0.75; 95% CI: 0.54–1.04; <i>P</i> = 0.08).</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6175 - 6186"},"PeriodicalIF":2.4,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06342-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145809010","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Intensified pegylated-asparaginase in consolidation therapy improved outcome of allogeneic hematopoietic stem cell transplantation for adult T-cell acute lymphoblastic leukemia 强化聚乙二醇天冬酰胺酶巩固治疗可改善同种异体造血干细胞移植治疗成人t细胞急性淋巴细胞白血病的预后。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-23 DOI: 10.1007/s00277-025-06576-8
Junjie Chen, Jia Li, Zhixiang Wang, Zicong Huang, Jieping Lin, Jiawang Ou, Xiuli Xu, Bingqing Tang, Chenhao Ding, Zihong Cai, Ren Lin, Xuan Li, Qifa Liu, Hongsheng Zhou

Research on the optimal consolidation therapy for adult T-cell acute lymphoblastic leukemia (T-ALL) remains limited, and how to integrate pediatric-inspired chemotherapy with allogeneic hematopoietic stem cell transplantation (SCT) is less addressed. Based on retrospective analysis indicating that 4 doses of pegylated-asparaginase (PEG-Asp) prior to SCT were optimal for survival, we designed a PEG-Asp-intensified pediatric-inspired regimen, PDT-ALL-2016, for adult T-ALL (NCT03564704). In this protocol, SCT administered following 4 or 5 doses of PEG-Asp. The impact of PEG-Asp on transplantation outcomes was evaluated in two cohorts: the prospective PDT-ALL-2016 cohort (2016.1-2021.12, N = 63) and the retrospective adult regimen cohort (2008.1-2015.12, N = 61). With intensified PEG-Asp consolidation, the prospective cohort exhibited superior survival compared to the retrospective cohort. Furthermore, within the prospective cohort, patients who received non-truncated PEG-Asp demonstrated better survival than those who received a truncated dose. Then, the entire cohort was stratified into high-dose (4–5 doses, N = 45), medium-dose (2–3 doses; N = 33) and low-dose (0–1 doses; N = 46) groups, based on the number of PEG-Asp doses administrated pre-SCT. The high-dose group showed superior survival compared to the other two groups in both the entire cohort and in subgroup analysis. Our findings indicate that high-dose PEG-Asp is associated with improved outcome in adult T-ALL undergoing SCT.

关于成人t细胞急性淋巴细胞白血病(T-ALL)最佳巩固治疗的研究仍然有限,如何将儿科启发的化疗与同种异体造血干细胞移植(SCT)结合起来的研究较少。基于回顾性分析表明,SCT前4剂量PEG-Asp(聚乙二醇天冬酰胺酶)对生存期最优,我们设计了一种针对成人T-ALL (NCT03564704)的PEG-Asp强化儿科方案PDT-ALL-2016。在该方案中,SCT在4或5剂PEG-Asp之后施用。PEG-Asp对移植结果的影响分为两个队列:前瞻性PDT-ALL-2016队列(2016.1-2021.12,N = 63)和回顾性成人方案队列(2008.1-2015.12,N = 61)。随着PEG-Asp巩固的加强,与回顾性队列相比,前瞻性队列显示出更高的生存率。此外,在前瞻性队列中,接受未截断的PEG-Asp的患者比接受截断剂量的患者表现出更好的生存率。然后,根据sct前给药PEG-Asp的剂量,将整个队列分为高剂量组(4-5剂量,N = 45)、中剂量组(2-3剂量,N = 33)和低剂量组(0-1剂量,N = 46)。在整个队列和亚组分析中,与其他两组相比,高剂量组显示出更高的生存率。我们的研究结果表明,高剂量PEG-Asp与成人T-ALL接受SCT的预后改善有关。
{"title":"Intensified pegylated-asparaginase in consolidation therapy improved outcome of allogeneic hematopoietic stem cell transplantation for adult T-cell acute lymphoblastic leukemia","authors":"Junjie Chen,&nbsp;Jia Li,&nbsp;Zhixiang Wang,&nbsp;Zicong Huang,&nbsp;Jieping Lin,&nbsp;Jiawang Ou,&nbsp;Xiuli Xu,&nbsp;Bingqing Tang,&nbsp;Chenhao Ding,&nbsp;Zihong Cai,&nbsp;Ren Lin,&nbsp;Xuan Li,&nbsp;Qifa Liu,&nbsp;Hongsheng Zhou","doi":"10.1007/s00277-025-06576-8","DOIUrl":"10.1007/s00277-025-06576-8","url":null,"abstract":"<div><p>Research on the optimal consolidation therapy for adult T-cell acute lymphoblastic leukemia (T-ALL) remains limited, and how to integrate pediatric-inspired chemotherapy with allogeneic hematopoietic stem cell transplantation (SCT) is less addressed. Based on retrospective analysis indicating that 4 doses of pegylated-asparaginase (PEG-Asp) prior to SCT were optimal for survival, we designed a PEG-Asp-intensified pediatric-inspired regimen, PDT-ALL-2016, for adult T-ALL (NCT03564704). In this protocol, SCT administered following 4 or 5 doses of PEG-Asp. The impact of PEG-Asp on transplantation outcomes was evaluated in two cohorts: the prospective PDT-ALL-2016 cohort (2016.1-2021.12, <i>N</i> = 63) and the retrospective adult regimen cohort (2008.1-2015.12, <i>N</i> = 61). With intensified PEG-Asp consolidation, the prospective cohort exhibited superior survival compared to the retrospective cohort. Furthermore, within the prospective cohort, patients who received non-truncated PEG-Asp demonstrated better survival than those who received a truncated dose. Then, the entire cohort was stratified into high-dose (4–5 doses, <i>N</i> = 45), medium-dose (2–3 doses; <i>N</i> = 33) and low-dose (0–1 doses; <i>N</i> = 46) groups, based on the number of PEG-Asp doses administrated pre-SCT. The high-dose group showed superior survival compared to the other two groups in both the entire cohort and in subgroup analysis. Our findings indicate that high-dose PEG-Asp is associated with improved outcome in adult T-ALL undergoing SCT.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6339 - 6349"},"PeriodicalIF":2.4,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06576-8.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145809084","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Quantitative assessment of innate myeloid cells in myelofibrosis: insights into myeloid and plasmacytoid dendritic cell depletion and disease progression 骨髓纤维化中先天髓样细胞的定量评估:髓样和浆细胞样树突状细胞耗竭和疾病进展的见解
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-18 DOI: 10.1007/s00277-025-06463-2
Nathália Aparecida Cavalcante Silva, Fábio Pinotti Guirao, Míriam Cristina Rodrigues Barbosa, Diego Alonso Olavarría Bernal, Laura Coutinho Vassalli, Alex Freire Sandes

Myelofibrosis (MF) is a myeloproliferative neoplasm characterized by bone marrow fibrosis, abnormal hematopoiesis, and extramedullary hematopoiesis. Abnormalities of the tumor microenvironment and immune system may be involved in the MF physiopathology and progression, particularly affecting innate myeloid cells (IMCs) such as myeloid dendritic cells (mDCs), plasmacytoid dendritic cells (pDCs) and monocytes. This study aims to quantitatively assess these cell populations in MF patients and their association with clinical outcomes. A cohort of 21 MF patients from the Universidade Federal de São Paulo was analyzed. Peripheral blood samples were evaluated using flow cytometry to quantify pDCs and monocytic subpopulations, including classical, intermediate, and non-classical monocytes. Comparative analyses were conducted with 12 healthy controls. MF patients exhibited a significant reduction in both the relative and absolute numbers of circulating mDCs and pDCs compared to healthy controls. The pDC reduction was more pronounced in patients with grade 3 fibrosis, anemia and splenomegaly. While total monocyte percentages were slightly lower in MF patient, the distribution of monocytic subpopulations showed no significant differences between groups. Patients with JAK2 mutations demonstrated higher concentrations of intermediate monocytes. The findings reveal a remarkable depletion of pDCs in MF patients, particularly those with advanced clinical manifestations such as anemia and splenomegaly. This study highlights the potential role of dendritic cells as biomarkers for disease severity and progression in MF. Further research is needed to elucidate the functional implications of these cells and their interactions within the MF microenvironment, potentially guiding new diagnostic and therapeutic strategies.

骨髓纤维化(MF)是一种骨髓增生性肿瘤,以骨髓纤维化、造血功能异常和髓外造血功能为特征。肿瘤微环境和免疫系统的异常可能参与MF的生理病理和进展,特别是影响先天性髓样细胞(IMCs),如髓样树突状细胞(mDCs)、浆细胞样树突状细胞(pDCs)和单核细胞。本研究旨在定量评估MF患者的这些细胞群及其与临床结果的关系。对来自圣保罗联邦大学的21例MF患者进行队列分析。使用流式细胞术评估外周血样本,定量pDCs和单核细胞亚群,包括经典、中间和非经典单核细胞。与12名健康对照进行比较分析。与健康对照相比,MF患者的循环mDCs和pDCs的相对和绝对数量均显著减少。在3级纤维化、贫血和脾肿大患者中,pDC降低更为明显。虽然MF患者单核细胞总百分比略低,但各组间单核细胞亚群分布无显著差异。JAK2突变患者表现出较高的中间单核细胞浓度。研究结果显示,在MF患者中,特别是那些有贫血和脾肿大等晚期临床表现的患者,pDCs明显减少。这项研究强调了树突状细胞作为MF疾病严重程度和进展的生物标志物的潜在作用。需要进一步的研究来阐明这些细胞的功能含义及其在MF微环境中的相互作用,从而潜在地指导新的诊断和治疗策略。
{"title":"Quantitative assessment of innate myeloid cells in myelofibrosis: insights into myeloid and plasmacytoid dendritic cell depletion and disease progression","authors":"Nathália Aparecida Cavalcante Silva,&nbsp;Fábio Pinotti Guirao,&nbsp;Míriam Cristina Rodrigues Barbosa,&nbsp;Diego Alonso Olavarría Bernal,&nbsp;Laura Coutinho Vassalli,&nbsp;Alex Freire Sandes","doi":"10.1007/s00277-025-06463-2","DOIUrl":"10.1007/s00277-025-06463-2","url":null,"abstract":"<div>\u0000 \u0000 <p>Myelofibrosis (MF) is a myeloproliferative neoplasm characterized by bone marrow fibrosis, abnormal hematopoiesis, and extramedullary hematopoiesis. Abnormalities of the tumor microenvironment and immune system may be involved in the MF physiopathology and progression, particularly affecting innate myeloid cells (IMCs) such as myeloid dendritic cells (mDCs), plasmacytoid dendritic cells (pDCs) and monocytes. This study aims to quantitatively assess these cell populations in MF patients and their association with clinical outcomes. A cohort of 21 MF patients from the Universidade Federal de São Paulo was analyzed. Peripheral blood samples were evaluated using flow cytometry to quantify pDCs and monocytic subpopulations, including classical, intermediate, and non-classical monocytes. Comparative analyses were conducted with 12 healthy controls. MF patients exhibited a significant reduction in both the relative and absolute numbers of circulating mDCs and pDCs compared to healthy controls. The pDC reduction was more pronounced in patients with grade 3 fibrosis, anemia and splenomegaly. While total monocyte percentages were slightly lower in MF patient, the distribution of monocytic subpopulations showed no significant differences between groups. Patients with JAK2 mutations demonstrated higher concentrations of intermediate monocytes. The findings reveal a remarkable depletion of pDCs in MF patients, particularly those with advanced clinical manifestations such as anemia and splenomegaly. This study highlights the potential role of dendritic cells as biomarkers for disease severity and progression in MF. Further research is needed to elucidate the functional implications of these cells and their interactions within the MF microenvironment, potentially guiding new diagnostic and therapeutic strategies.</p>\u0000 </div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6187 - 6196"},"PeriodicalIF":2.4,"publicationDate":"2025-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06463-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145773326","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Safety and efficacy of venetoclax-azacitidine combined with low-dose Idarubicin and cytarabine regimens for the treatment of newly diagnosed acute myeloid leukemia patients compared to the standard chemotherapy: a propensity score-matched real-world single-center experience 与标准化疗相比,venetoclaxa -azacitidine联合低剂量伊达柔比星和阿糖胞苷治疗新诊断急性髓系白血病患者的安全性和有效性:倾向评分匹配的真实世界单中心经验。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-18 DOI: 10.1007/s00277-025-06653-y
Xiao-Tong Chen, Qi Li, Yan-Qiu Zhao, Sheng-Jin Fan

Despite advances in treatment, long -term survival in patients with acute myeloid leukemia (AML) remains unsatisfactory. Standard induction chemotherapy achieves complete remission (CR) rates of 60%-80% in newly diagnosed AML patients; however, relapse and chemoresistance persist as major challenges. Combined multi-target therapeutic regimens have the potential to enhance response rates and improve survival outcomes. In this propensity score matching (PSM), single-center retrospective study, we evaluated the efficacy and safety of a novel regimen-venetoclax-azacitidine combined with low-dose idarubicin and cytarabine (VAIA)།versus standard idarubicin-cytarabine (IA) therapy in newly diagnosed AML patients. Twenty-six patients treated with VAIA were compared with 52 patients treated with IA, with well-matched clinical and molecular baseline characteristics. The VAIA cohort demonstrated significantly higher CR and measurable residual disease (MRD)-negative CR rates compared to the IA cohort (84.3% vs. 61.5%, P = 0.037; and 80.8% vs. 53.8%, P = 0.026, respectively). Moreover, VAIA was associated with improved overall survival (1-year OS: 76.9% vs. 57.7%, P = 0.031) and leukemia-free survival (1-year LFS: 65.4% vs. 46.2%, P = 0.042). Subgroup analysis revealed that adverse-risk patients particularly benefited from VAIA (1-year OS: 69.2% vs. 32%, P = 0.045; 1-year LFS: 61.5% vs. 20%, P = 0.046). In both treatment groups, MRD-negative patients exhibited significantly longer survival than MRD-positive patients. The VAIA regimen also showed superior therapeutic effects in patients particularly in patients with adverse or complex cytogenetics and adverse risk, with an acceptable and manageable toxicity profile. These findings support further prospective evaluation of the VAIA regimen in AML.

尽管治疗取得了进展,急性髓性白血病(AML)患者的长期生存率仍然不令人满意。标准诱导化疗在新诊断的AML患者中达到完全缓解(CR)率为60%-80%;然而,复发和化疗耐药仍然是主要的挑战。联合多靶点治疗方案有可能提高反应率和改善生存结果。在这项倾向评分匹配(PSM)的单中心回顾性研究中,我们评估了一种新方案-维托克拉-阿扎胞苷联合低剂量阿达比星和阿糖胞苷(VAIA):与标准阿达比星-阿糖胞苷(IA)治疗新诊断的AML患者的有效性和安全性。26例接受VAIA治疗的患者与52例接受IA治疗的患者进行了比较,临床和分子基线特征匹配良好。与IA队列相比,VAIA队列的CR和可测量的残留病(MRD)阴性CR率显著更高(分别为84.3%对61.5%,P = 0.037; 80.8%对53.8%,P = 0.026)。此外,VAIA与总生存率(1年OS: 76.9% vs. 57.7%, P = 0.031)和无白血病生存率(1年LFS: 65.4% vs. 46.2%, P = 0.042)相关。亚组分析显示不良风险患者尤其受益于VAIA(1年OS: 69.2% vs. 32%, P = 0.045; 1年LFS: 61.5% vs. 20%, P = 0.046)。在两个治疗组中,mrd阴性患者的生存时间明显长于mrd阳性患者。VAIA方案在患者中也显示出优越的治疗效果,特别是在具有不良或复杂细胞遗传学和不良风险的患者中,具有可接受和可控的毒性特征。这些发现支持进一步对VAIA方案在AML中的前瞻性评估。
{"title":"Safety and efficacy of venetoclax-azacitidine combined with low-dose Idarubicin and cytarabine regimens for the treatment of newly diagnosed acute myeloid leukemia patients compared to the standard chemotherapy: a propensity score-matched real-world single-center experience","authors":"Xiao-Tong Chen,&nbsp;Qi Li,&nbsp;Yan-Qiu Zhao,&nbsp;Sheng-Jin Fan","doi":"10.1007/s00277-025-06653-y","DOIUrl":"10.1007/s00277-025-06653-y","url":null,"abstract":"<div>\u0000 \u0000 <p>Despite advances in treatment, long -term survival in patients with acute myeloid leukemia (AML) remains unsatisfactory. Standard induction chemotherapy achieves complete remission (CR) rates of 60%-80% in newly diagnosed AML patients; however, relapse and chemoresistance persist as major challenges. Combined multi-target therapeutic regimens have the potential to enhance response rates and improve survival outcomes. In this propensity score matching (PSM), single-center retrospective study, we evaluated the efficacy and safety of a novel regimen-venetoclax-azacitidine combined with low-dose idarubicin and cytarabine (VAIA)།versus standard idarubicin-cytarabine (IA) therapy in newly diagnosed AML patients. Twenty-six patients treated with VAIA were compared with 52 patients treated with IA, with well-matched clinical and molecular baseline characteristics. The VAIA cohort demonstrated significantly higher CR and measurable residual disease (MRD)-negative CR rates compared to the IA cohort (84.3% vs. 61.5%, <i>P</i> = 0.037; and 80.8% vs. 53.8%, <i>P</i> = 0.026, respectively). Moreover, VAIA was associated with improved overall survival (1-year OS: 76.9% vs. 57.7%, <i>P</i> = 0.031) and leukemia-free survival (1-year LFS: 65.4% vs. 46.2%, <i>P</i> = 0.042). Subgroup analysis revealed that adverse-risk patients particularly benefited from VAIA (1-year OS: 69.2% vs. 32%, <i>P</i> = 0.045; 1-year LFS: 61.5% vs. 20%, <i>P</i> = 0.046). In both treatment groups, MRD-negative patients exhibited significantly longer survival than MRD-positive patients. The VAIA regimen also showed superior therapeutic effects in patients particularly in patients with adverse or complex cytogenetics and adverse risk, with an acceptable and manageable toxicity profile. These findings support further prospective evaluation of the VAIA regimen in AML.</p>\u0000 </div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6197 - 6204"},"PeriodicalIF":2.4,"publicationDate":"2025-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06653-y.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145773276","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Targeting relapsed/refractory and MRD + Ph + ALL: olverembatinib-venetoclax bridging enhances allo-HSCT outcome​ 针对复发/难治性和MRD + Ph + ALL: olverembatinib-venetoclax桥接提高了同种异体造血干细胞移植的结果。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-13 DOI: 10.1007/s00277-025-06708-0
Xiaoyu Zhang, Yanhong Zhao, Weihua Zhai, Qiaoling Ma, Yigeng Cao, Jialin Wei, Chen Liang, Xin Chen, Wenbin Cao, Donglin Yang, Aiming Pang, Yi He, Sizhou Feng, Mingzhe Han, Rongli Zhang, Erlie Jiang

Olverembatinib is a novel third-generation TKI available in mainland China. However, there are only a few published clinical reports. In this study, we aimed to investigate the efficacy and safety of an olverembatinib-venetoclax regimen before bridging to HSCT in adult Ph/BCR-ABL1 + ALL patients with refractory/relapsed disease (n = 2) or persistent minimal residual disease (MRD) (n = 15). Seventeen Ph + ALL patients who were admitted to our center between February 2022 and January 2023 were enrolled. Four patients harbored a T315I mutation. In all, 100% achieved hematologic complete remission, and 70.6% achieved complete molecular remission. With a median follow-up of 856-day post-HSCT, the 2-year overall survival and relapsed free survival rates was 88.2 ± 7.8% and 79.4 ± 10.9%, respectively. The findings of this study suggest that in Ph + ALL patients with disease recurrence and persistent MRD positivity, olverembatinib-venetoclax regimen showed a high molecular response rate and was well-tolerated in MRD clearance bridged to allo-HSCT.

Olverembatinib是在中国大陆上市的新型第三代TKI。然而,只有少数发表的临床报告。在这项研究中,我们的目的是研究成人Ph/BCR-ABL1 + ALL患者难治性/复发性疾病(n = 2)或持续性微小残留疾病(MRD) (n = 15)桥接HSCT前的olverembatinib-venetoclax方案的有效性和安全性。17例Ph + ALL患者于2022年2月至2023年1月期间入住本中心。四名患者携带T315I突变。总的来说,100%的血液完全缓解,70.6%的分子完全缓解。hsct后中位随访856天,2年总生存率和无复发生存率分别为88.2±7.8%和79.4±10.9%。本研究结果表明,在Ph + ALL复发且MRD持续阳性的患者中,olverembatinib-venetoclax方案显示出较高的分子反应率,并且在异体造血干细胞移植的MRD清除中耐受性良好。
{"title":"Targeting relapsed/refractory and MRD + Ph + ALL: olverembatinib-venetoclax bridging enhances allo-HSCT outcome​","authors":"Xiaoyu Zhang,&nbsp;Yanhong Zhao,&nbsp;Weihua Zhai,&nbsp;Qiaoling Ma,&nbsp;Yigeng Cao,&nbsp;Jialin Wei,&nbsp;Chen Liang,&nbsp;Xin Chen,&nbsp;Wenbin Cao,&nbsp;Donglin Yang,&nbsp;Aiming Pang,&nbsp;Yi He,&nbsp;Sizhou Feng,&nbsp;Mingzhe Han,&nbsp;Rongli Zhang,&nbsp;Erlie Jiang","doi":"10.1007/s00277-025-06708-0","DOIUrl":"10.1007/s00277-025-06708-0","url":null,"abstract":"<div><p>Olverembatinib is a novel third-generation TKI available in mainland China. However, there are only a few published clinical reports. In this study, we aimed to investigate the efficacy and safety of an olverembatinib-venetoclax regimen before bridging to HSCT in adult <i>Ph/BCR-ABL1</i> + ALL patients with refractory/relapsed disease (<i>n</i> = 2) or persistent minimal residual disease (MRD) (<i>n</i> = 15). Seventeen Ph + ALL patients who were admitted to our center between February 2022 and January 2023 were enrolled. Four patients harbored a <i>T315I</i> mutation. In all, 100% achieved hematologic complete remission, and 70.6% achieved complete molecular remission. With a median follow-up of 856-day post-HSCT, the 2-year overall survival and relapsed free survival rates was 88.2 ± 7.8% and 79.4 ± 10.9%, respectively. The findings of this study suggest that in <i>Ph +</i> ALL patients with disease recurrence and persistent MRD positivity, olverembatinib-venetoclax regimen showed a high molecular response rate and was well-tolerated in MRD clearance bridged to allo-HSCT.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6463 - 6466"},"PeriodicalIF":2.4,"publicationDate":"2025-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06708-0.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145740703","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Importance of the 3′UTR region in globin synthesis: identification of two novel HBA1 mutations causing α-Thalassemia 3'UTR区域在珠蛋白合成中的重要性:鉴定两种导致α-地中海贫血的新型HBA1突变。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-13 DOI: 10.1007/s00277-025-06624-3
Sara Ferrer Benito, Mariola Abío, Eduardo J. Bardón-Cancho, Jorge M. Nieto, Belén Ortega, F. A. González, Ana Villegas, Celina Benavente, Paloma Ropero

Alpha-thalassemia is primarily caused by large deletions in the HBA1 and HBA2 genes, but a minority of cases result from non-deletional mutations, including variants in untranslated regions (UTRs) that affect mRNA regulation. The 3′UTR is crucial for post-transcriptional control, influencing mRNA stability, localization, and translation via binding elements for RNA-binding proteins and microRNAs. We describe two patients with persistent microcytosis and normal iron levels. Standard hematological analyses were complemented by high-performance liquid chromatography (HPLC) and capillary electrophoresis to rule out common hemoglobinopathies. Molecular analysis was performed using multiplex PCR and direct sequencing of the HBA1 3′UTR. Patient 1, a 3-year-old Nigerian boy, carried a novel insertion mutation (HBA1:c.*119_120insT) five nucleotides downstream of the transcription termination signal. Patient 2, a 31-year-old Moroccan woman, exhibited a missense variant (HBA1:c.*150C > A) located 40 nucleotides downstream of the TTS. Both variants are located within regulatory regions of the HBA1 3′UTR. In silico analyses suggest disruption of RNA secondary structure, impaired interactions with HuR and AUF1 proteins, altered microRNA binding (e.g., miR-16-5p), and potential interference with polyadenylation signals, resulting in mRNA instability and reduced alpha-globin synthesis. These two novel mutations in the 3′UTR of HBA1 expand the spectrum of non-deletional alpha-thalassemia variants and highlight the importance of regulatory regions in globin gene expression. Their inclusion in routine molecular screening may enhance diagnostic accuracy in cases of unresolved microcytic anemia.

α -地中海贫血主要是由HBA1和HBA2基因的大缺失引起的,但少数病例是由非缺失突变引起的,包括影响mRNA调节的非翻译区(utr)的变异。3'UTR对转录后控制至关重要,通过rna结合蛋白和microrna的结合元件影响mRNA的稳定性、定位和翻译。我们描述了两例持续小细胞增多和正常铁水平的患者。标准血液学分析辅以高效液相色谱(HPLC)和毛细管电泳,以排除常见的血红蛋白病。采用多重PCR和hba13 ' utr直接测序进行分子分析。患者1是一名3岁的尼日利亚男孩,在转录终止信号下游5个核苷酸处携带一种新的插入突变(HBA1:c.*119_120insT)。患者2,一名31岁的摩洛哥妇女,表现出位于TTS下游40个核苷酸的错义变异(HBA1:c.*150C > a)。这两种变异都位于hba13 ' utr的调控区域内。硅分析表明,RNA二级结构被破坏,与HuR和AUF1蛋白的相互作用受损,microRNA结合改变(例如miR-16-5p),以及对聚腺苷化信号的潜在干扰,导致mRNA不稳定和α -珠蛋白合成减少。HBA1 3'UTR的这两个新突变扩大了非缺失型α -地中海贫血变异的范围,并突出了珠蛋白基因表达调控区域的重要性。将其纳入常规分子筛查可提高未解决的小细胞性贫血病例的诊断准确性。
{"title":"Importance of the 3′UTR region in globin synthesis: identification of two novel HBA1 mutations causing α-Thalassemia","authors":"Sara Ferrer Benito,&nbsp;Mariola Abío,&nbsp;Eduardo J. Bardón-Cancho,&nbsp;Jorge M. Nieto,&nbsp;Belén Ortega,&nbsp;F. A. González,&nbsp;Ana Villegas,&nbsp;Celina Benavente,&nbsp;Paloma Ropero","doi":"10.1007/s00277-025-06624-3","DOIUrl":"10.1007/s00277-025-06624-3","url":null,"abstract":"<div><p>Alpha-thalassemia is primarily caused by large deletions in the HBA1 and HBA2 genes, but a minority of cases result from non-deletional mutations, including variants in untranslated regions (UTRs) that affect mRNA regulation. The 3′UTR is crucial for post-transcriptional control, influencing mRNA stability, localization, and translation via binding elements for RNA-binding proteins and microRNAs. We describe two patients with persistent microcytosis and normal iron levels. Standard hematological analyses were complemented by high-performance liquid chromatography (HPLC) and capillary electrophoresis to rule out common hemoglobinopathies. Molecular analysis was performed using multiplex PCR and direct sequencing of the HBA1 3′UTR. Patient 1, a 3-year-old Nigerian boy, carried a novel insertion mutation (HBA1:c.*119_120insT) five nucleotides downstream of the transcription termination signal. Patient 2, a 31-year-old Moroccan woman, exhibited a missense variant (HBA1:c.*150C &gt; A) located 40 nucleotides downstream of the TTS. Both variants are located within regulatory regions of the HBA1 3′UTR. In silico analyses suggest disruption of RNA secondary structure, impaired interactions with HuR and AUF1 proteins, altered microRNA binding (e.g., miR-16-5p), and potential interference with polyadenylation signals, resulting in mRNA instability and reduced alpha-globin synthesis. These two novel mutations in the 3′UTR of HBA1 expand the spectrum of non-deletional alpha-thalassemia variants and highlight the importance of regulatory regions in globin gene expression. Their inclusion in routine molecular screening may enhance diagnostic accuracy in cases of unresolved microcytic anemia.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6169 - 6174"},"PeriodicalIF":2.4,"publicationDate":"2025-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06624-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145740727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Long term outcomes of older patients with classical hodgkin lymphoma: an analysis of the Texas cancer registry 经典霍奇金淋巴瘤老年患者的长期预后:德克萨斯州癌症登记的分析。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-12 DOI: 10.1007/s00277-025-06539-z
Ethan A. Burns, Hala Hassanain, Sunil Mathur, Darshil Choksi, Yuqi Zhang, Cesar Gentille Sanchez, Chih-hang Anthony Tang, Hanh Mai, Carrie Yuen, Shilpan Shah, Siddhartha Ganguly, Chih-chi Andrew Hu, Sai Ravi Pingali

Treatment advances for classical Hodgkin Lymphoma (cHL) have improved outcomes, but studies are lacking in older patients. This analysis aims to investigate survival trends in older individuals with cHL using the Texas Cancer Registry (TCR). Adults ≥ 65 years with cHL were separated into three eras: 1995–2002, 2003–2010, and 2011–2017. Pearson chi-squared test and cox-proportional hazard models along with hazard ratios (HR) and 95% confidence intervals (95% CI) compared demographic factors, and survival trends were assessed by Kaplan-Meier methodology. There were 386 (37.4%), 336 (32.5%), and 311 (30.1%) individuals diagnosed in 1995–2002, 2003–2010, and 2011–2017, respectively. Median DSS per era was 97 months (95% CI 50.7, 143.3), 158 months (95% CI 132.5, 183.5), and not reached, (p=0.383), and median OS was 32 months (95% CI 23.4, 40.6), 37 months (95% CI 23.5, 50.5), and 40 months (95% CI 26.8, 53.1), respectively (p=0.693). Median OS was worse for blacks and Hispanics vs non-Hispanic whites (p=0.007), increasing age (p<0.001), and higher poverty index (p=0.006). While DSS and OS have not improved over the past two decades for older individuals, there are disparity dependent survival differences by race, age, and poverty index. Strategies to reduce these disparities are needed.

经典霍奇金淋巴瘤(cHL)的治疗进展改善了预后,但缺乏老年患者的研究。本分析旨在利用德克萨斯州癌症登记处(TCR)调查老年cHL患者的生存趋势。≥65岁cHL成人分为1995-2002年、2003-2010年和2011-2017年三个时期。Pearson卡方检验和cox比例风险模型以及风险比(HR)和95%置信区间(95% CI)比较了人口统计学因素,并通过Kaplan-Meier方法评估了生存趋势。1995-2002年、2003-2010年和2011-2017年确诊病例分别为386例(37.4%)、336例(32.5%)和311例(30.1%)。每个时代的中位DSS为97个月(95% CI 50.7, 143.3), 158个月(95% CI 132.5, 183.5),未达到(p=0.383),中位OS分别为32个月(95% CI 23.4, 40.6), 37个月(95% CI 23.5, 50.5)和40个月(95% CI 26.8, 53.1) (p=0.693)。黑人和西班牙裔白人的中位OS比非西班牙裔白人更差(p=0.007),随着年龄的增加(p
{"title":"Long term outcomes of older patients with classical hodgkin lymphoma: an analysis of the Texas cancer registry","authors":"Ethan A. Burns,&nbsp;Hala Hassanain,&nbsp;Sunil Mathur,&nbsp;Darshil Choksi,&nbsp;Yuqi Zhang,&nbsp;Cesar Gentille Sanchez,&nbsp;Chih-hang Anthony Tang,&nbsp;Hanh Mai,&nbsp;Carrie Yuen,&nbsp;Shilpan Shah,&nbsp;Siddhartha Ganguly,&nbsp;Chih-chi Andrew Hu,&nbsp;Sai Ravi Pingali","doi":"10.1007/s00277-025-06539-z","DOIUrl":"10.1007/s00277-025-06539-z","url":null,"abstract":"<div><p>Treatment advances for classical Hodgkin Lymphoma (cHL) have improved outcomes, but studies are lacking in older patients. This analysis aims to investigate survival trends in older individuals with cHL using the Texas Cancer Registry (TCR). Adults ≥ 65 years with cHL were separated into three eras: 1995–2002, 2003–2010, and 2011–2017. Pearson chi-squared test and cox-proportional hazard models along with hazard ratios (HR) and 95% confidence intervals (95% CI) compared demographic factors, and survival trends were assessed by Kaplan-Meier methodology. There were 386 (37.4%), 336 (32.5%), and 311 (30.1%) individuals diagnosed in 1995–2002, 2003–2010, and 2011–2017, respectively. Median DSS per era was 97 months (95% CI 50.7, 143.3), 158 months (95% CI 132.5, 183.5), and not reached, (p=0.383), and median OS was 32 months (95% CI 23.4, 40.6), 37 months (95% CI 23.5, 50.5), and 40 months (95% CI 26.8, 53.1), respectively (p=0.693). Median OS was worse for blacks and Hispanics vs non-Hispanic whites (p=0.007), increasing age (p&lt;0.001), and higher poverty index (p=0.006). While DSS and OS have not improved over the past two decades for older individuals, there are disparity dependent survival differences by race, age, and poverty index. Strategies to reduce these disparities are needed.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6305 - 6314"},"PeriodicalIF":2.4,"publicationDate":"2025-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06539-z.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145740715","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Predicting the toxicity-efficacy ratio of venetoclax in real-world patients 预测维托克拉克斯在现实世界患者中的毒效比。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-11 DOI: 10.1007/s00277-025-06531-7
Loïc Osanno, Lucy Brocque, Laurent Bourguignon, Carla Delpech, Laure Farnault, Julien Colle, Pauline Roche, Jacques Chiaroni, Caroline Izard, Régis Costello, Mathilde Dacos, Caroline Solas, Chems Djezzar, Joseph Ciccolini, Thomas Cluzeau, Geoffroy Venton, Raphaëlle Fanciullino

This study investigates the pharmacokinetic variability and exposure-response relationships of Venetoclax (VEN) in adult patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. The study was conducted in a real-world clinical setting and included 48 patients who were treated with VEN in combination with azacytidine. We found significant inter-individual variability of 68% and intra-individual variability of 39% in plasma VEN concentrations. In addition, higher VEN concentrations were associated with better hematological responses. Median overall survival for the entire cohort was 17.8 months, with 1- and 2-year survival rates of 51% and 36.4%, respectively. A comparison of the 14-day VEN and 28-day VEN protocols showed that patients benefited from longer treatment durations, which resulted in more courses being administered. Plasma concentrations in the 14-day VEN protocol were higher than in the 28-day VEN protocol (2330 + 1675 ng/mL vs. 1503 + 966 ng/mL, respectively) without an increase in toxicities. The optimal protocol would be 400 mg/14d if we consider survival as a function of the 1818ng/mL cutoff. These results highlight the importance of considering therapeutic drug monitoring (TDM) as a strategy to optimize treatment outcomes by balancing efficacy and safety.

本研究探讨了Venetoclax (VEN)在不适合接受强化化疗的急性髓性白血病(AML)成年患者中的药代动力学变异性和暴露-反应关系。该研究是在现实世界的临床环境中进行的,包括48名接受VEN联合阿扎胞苷治疗的患者。我们发现血浆VEN浓度的个体间变异性为68%,个体内变异性为39%。此外,较高的VEN浓度与更好的血液学反应相关。整个队列的中位总生存期为17.8个月,1年和2年生存率分别为51%和36.4%。比较14天和28天的VEN方案表明,患者受益于较长的治疗时间,这导致了更多的疗程。14天VEN方案的血浆浓度高于28天VEN方案(分别为2330 + 1675 ng/mL和1503 + 966 ng/mL),但毒性没有增加。如果我们考虑生存是1818ng/mL临界值的函数,那么最佳方案是400mg /14d。这些结果强调了将治疗性药物监测(TDM)作为一种平衡疗效和安全性来优化治疗结果的策略的重要性。
{"title":"Predicting the toxicity-efficacy ratio of venetoclax in real-world patients","authors":"Loïc Osanno,&nbsp;Lucy Brocque,&nbsp;Laurent Bourguignon,&nbsp;Carla Delpech,&nbsp;Laure Farnault,&nbsp;Julien Colle,&nbsp;Pauline Roche,&nbsp;Jacques Chiaroni,&nbsp;Caroline Izard,&nbsp;Régis Costello,&nbsp;Mathilde Dacos,&nbsp;Caroline Solas,&nbsp;Chems Djezzar,&nbsp;Joseph Ciccolini,&nbsp;Thomas Cluzeau,&nbsp;Geoffroy Venton,&nbsp;Raphaëlle Fanciullino","doi":"10.1007/s00277-025-06531-7","DOIUrl":"10.1007/s00277-025-06531-7","url":null,"abstract":"<div><p>This study investigates the pharmacokinetic variability and exposure-response relationships of Venetoclax (VEN) in adult patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. The study was conducted in a real-world clinical setting and included 48 patients who were treated with VEN in combination with azacytidine. We found significant inter-individual variability of 68% and intra-individual variability of 39% in plasma VEN concentrations. In addition, higher VEN concentrations were associated with better hematological responses. Median overall survival for the entire cohort was 17.8 months, with 1- and 2-year survival rates of 51% and 36.4%, respectively. A comparison of the 14-day VEN and 28-day VEN protocols showed that patients benefited from longer treatment durations, which resulted in more courses being administered. Plasma concentrations in the 14-day VEN protocol were higher than in the 28-day VEN protocol (2330 + 1675 ng/mL vs. 1503 + 966 ng/mL, respectively) without an increase in toxicities. The optimal protocol would be 400 mg/14d if we consider survival as a function of the 1818ng/mL cutoff. These results highlight the importance of considering therapeutic drug monitoring (TDM) as a strategy to optimize treatment outcomes by balancing efficacy and safety.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"104 12","pages":"6327 - 6337"},"PeriodicalIF":2.4,"publicationDate":"2025-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-025-06531-7.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145720584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
期刊
Annals of Hematology
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
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
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术
文献互助 智能选刊 最新文献 互助须知 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 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1