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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
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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。
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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。
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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。
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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。
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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
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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。
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引用次数: 0
Can platelet to neutrophil ratio (PNR) serve as a viable alternative tool for monitoring sickle cell disease patients on hydroxyurea in low income countries? 血小板与中性粒细胞比率(PNR)能否作为监测低收入国家镰状细胞病患者羟脲的可行替代工具?
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-10 DOI: 10.1007/s00277-025-06598-2
Efobi Chilota Chibuife, Nri-Ezedi Chisom Adaobi, Franklin Odini, Habib Darbari Deepika, Nana-Bilkisu Aduni, Campbell Andrew

Quantification of foetal haemoglobin (HbF) via high-performance liquid chromatography (HPLC) remains a critical parameter for monitoring therapeutic response to hydroxyurea (HU) in patients with sickle cell disease (SCD). However, its routine application is constrained in low-income settings such as Nigeria due to limited accessibility. The platelet-to-neutrophil ratio (PNR), an emerging inflammatory biomarker, has demonstrated prognostic relevance in several disease conditions. This study investigates the potential utility of PNR as a surrogate monitoring tool in SCD patients receiving HU therapy. This was a retrospective analysis of data derived from the Children’s National Hospital Natural History Study of Sickle Cell Disease, conducted in Washington, D.C., USA. Medical records of patients with SCD were reviewed from 1 September 2013 to 14 September 2023. Associations between PNR, HU use, HbF concentration, and reticulocyte count were evaluated. T-tests was used for continuous variables and chi-square tests for categorical variables. Regression analysis was used to evaluate the predictors of hydroxyurea use. A p-value of < 0.05 was considered statistically significant. Of 437 patient records reviewed, 222 (50.8%) were male, and 312 (71.4%) were receiving HU. The majority (81%) had no history of transfusion. Patients on HU exhibited significantly higher platelet counts, HbF levels, and PNR values (p = 0.0008, p < 0.0001, and p < 0.0001, respectively). In multivariate logistic regression, both HbF (OR: 1.121; 95% CI: 1.051–1.196; p < 0.001) and PNR (OR: 1.014; 95% CI: 1.005–1.024; p = 0.0022) were independently associated with HU use. No significant associations were observed between HU use and age group, haemoglobin phenotype, transfusion history, or BMI (p > 0.05). The PNR demonstrates a significant association with HU use and HbF levels, indicating its potential as a low-cost, accessible biomarker for monitoring HU therapy in SCD. Although this analysis was based on data from a high-resource setting, the findings underscore the relevance of PNR as a feasible monitoring alternative in resource-constrained environments. Further validation in low-income contexts is warranted to establish population-specific reference intervals and inform clinical practice.

通过高效液相色谱(HPLC)定量胎儿血红蛋白(HbF)仍然是监测镰状细胞病(SCD)患者羟基脲(HU)治疗反应的关键参数。然而,由于可及性有限,它在尼日利亚等低收入国家的常规应用受到限制。血小板与中性粒细胞比率(PNR)是一种新兴的炎症生物标志物,已被证明与几种疾病的预后相关。本研究探讨了PNR作为接受HU治疗的SCD患者的替代监测工具的潜在效用。这是一项对美国华盛顿特区国立儿童医院镰状细胞病自然史研究数据的回顾性分析。回顾了2013年9月1日至2023年9月14日SCD患者的医疗记录。评估PNR、HU使用、HbF浓度和网织红细胞计数之间的关系。连续变量采用t检验,分类变量采用卡方检验。采用回归分析评价羟基脲使用的预测因素。p值为0.05)。PNR显示与HU使用和HbF水平显著相关,表明其有潜力作为监测SCD中HU治疗的低成本、可获取的生物标志物。虽然这一分析是基于高资源环境的数据,但研究结果强调了PNR在资源受限环境中作为一种可行的监测替代方案的相关性。在低收入背景下进一步验证是有必要的,以建立特定人群的参考区间,并为临床实践提供信息。
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引用次数: 0
Adapting haematology practices for effective management of blood disorders in pregnancy in a limited resource setting 在资源有限的情况下,调整血液学实践以有效管理妊娠期血液疾病。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2025-12-10 DOI: 10.1007/s00277-025-06566-w
Thushara Balasuriya, Chitranga C. Kariyawasan

Introduction

Haematology plays a crucial role in the management of pregnant females as blood disorders are common in these patients which can significantly impact on maternal and fetal health. This paper explores how haematology can adapt its practice to address the unique challenges posed by physiological/pathological changes during pregnancy.

Methods

A comprehensive review of the physiological changes in blood volume, coagulation, and haematological parameters during pregnancy was conducted. Case series of pregnant women with various blood disorders, including gestational thrombocytopenia, iron deficiency anaemia, venous thromboembolism, sickle cell disease, and thalassemia, were analysed to illustrate management strategies.

Results

The physiological increase in blood volume during pregnancy leads to dilutional effects on haematological parameters, complicating diagnosis and management. Conditions such as gestational thrombocytopenia and anaemia require tailored approaches, while the increased risk of thromboembolic events necessitates proactive risk assessment and prophylactic measures. The management of pre-existing blood disorders demands a multidisciplinary approach, emphasizing collaboration between haematologists and obstetricians.

Discussion

Effective management of blood disorders in pregnancy is essential on understanding physiological changes, individualized care plans, and patient education. Empowering patients with knowledge about their conditions and potential complications is essential for optimizing outcomes.

Conclusion

Adapting haematology practice for pregnancy management is vital for addressing the complexities of blood disorders during the critical pregnancy period. A multidisciplinary approach, ongoing education, and research advancements are essential for improving maternal and fetal health outcomes. By fostering collaboration across specialties, healthcare providers can work towards achieving the best possible outcomes for pregnant woman with haematological conditions.

导读:血液学在孕妇的管理中起着至关重要的作用,因为血液疾病在这些患者中很常见,可以显著影响母体和胎儿的健康。本文探讨了血液学如何适应其实践,以解决怀孕期间生理/病理变化带来的独特挑战。方法:对妊娠期间血容量、凝血和血液学参数的生理变化进行全面回顾。病例系列的孕妇与各种血液疾病,包括妊娠血小板减少症,缺铁性贫血,静脉血栓栓塞症,镰状细胞病,地中海贫血,分析说明管理策略。结果:妊娠期血容量的生理性增加导致血液学参数的稀释,使诊断和治疗复杂化。妊娠期血小板减少和贫血等情况需要量身定制的方法,而血栓栓塞事件风险的增加需要主动风险评估和预防措施。预先存在的血液疾病的管理需要多学科的方法,强调血液科医生和产科医生之间的合作。讨论:妊娠期血液疾病的有效管理对了解生理变化、个性化护理计划和患者教育至关重要。让患者了解自己的病情和潜在的并发症对于优化结果至关重要。结论:适应血液学实践妊娠管理是解决血液疾病的复杂性,在关键的妊娠期至关重要。多学科方法、持续教育和研究进展对改善孕产妇和胎儿健康结果至关重要。通过促进各专业之间的合作,医疗保健提供者可以努力为患有血液病的孕妇实现最佳结果。
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引用次数: 0
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Annals of Hematology
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