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Burixafor, a CXCR4 inhibitor with a differentiated kinetics profile: results of a phase 2 study for rapid cell mobilization in multiple myeloma and lymphoma patients undergoing transplant. Burixafor,一种具有分化动力学特征的CXCR4抑制剂:一项用于移植的多发性骨髓瘤和淋巴瘤患者快速细胞动员的2期研究结果。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-04 DOI: 10.1007/s00277-026-06858-9
Devki D Sukhtankar, Pina M Cardarelli, Bita Jalilizeinali, Gayatri Setia, GaYeon Kim, Li-Wen Chang, Cheng-Yuan Tsai, Niña G Caculitan, Michael W Schuster
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引用次数: 0
Efficacy and safety of epcoritamab in relapsed or refractory large B-cell lymphoma: 3-year update from the EPCORE NHL-1 trial. EPCORE NHL-1试验3年来的最新进展:epcoritamab治疗复发或难治性大b细胞淋巴瘤的疗效和安全性
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-04 DOI: 10.1007/s00277-026-06798-4
Yasmin H Karimi, Chan Y Cheah, Michael Roost Clausen, David Cunningham, Umar Farooq, Tatyana Feldman, Herve Ghesquieres, Wojciech Jurczak, Kim M Linton, Tycel Phillips, Julie M Vose, Won Seog Kim, Pegah Jafarinasabian, Barbara D'Angelo Månsson, David Soong, Andrew J Steele, Zhu Li, Christian W Eskelund, Martin Hutchings, Catherine Thieblemont

Epcoritamab, a CD3xCD20 bispecific antibody, resulted in deep, durable responses with a manageable safety profile in patients with relapsed/refractory large B-cell lymphoma (LBCL) in EPCORE® NHL-1 (NCT03625037). We report results from a 3-year follow-up. Adults with relapsed/refractory LBCL received epcoritamab until progressive disease or unacceptable toxicity. The primary endpoint was overall response rate (ORR). Median age was 64.0 years, 39% of patients received prior CAR T-cell treatment, and 75% were refractory to ≥ 2 consecutive lines of treatment. As of May 3, 2024 (median follow-up 37.1 months [range, 0.3-45.5]), ORR was 59% and complete response (CR) rate 41% by investigator assessment. Median duration of response was 20.8 months (95% confidence interval [CI], 13.0-32.0). Median duration of CR was 36.1 months (20.2-not reached [NR]); the longest ongoing CR was > 43 months. Median progression-free survival was 4.2 months (95% CI, 2.8-5.5) in all patients and 37.3 months (26.0-NR) in patients with CR. Median overall survival was 18.5 months (95% CI, 11.7-27.7) in all patients and NR in patients with CR. Of 119 patients evaluable for minimal residual disease (MRD) assessments, 54 (45%) were MRD-negative at any time during the study. Most common adverse events were cytokine release syndrome (51%), fatigue (25%), and pyrexia (25%), with no new safety signals. Grade 1, 2, and 3 infections occurred in 23%, 34%, and 24% of patients, respectively. The durability of responses and prolonged survival in complete responders suggest long-term disease-free survival with epcoritamab in these patients with relapsed/refractory LBCL.

Epcoritamab是一种CD3xCD20双特异性抗体,在EPCORE®NHL-1 (NCT03625037)治疗复发/难治性大b细胞淋巴瘤(LBCL)患者中产生了深度、持久的反应,具有可管理的安全性。我们报告了3年随访的结果。复发/难治性LBCL的成人患者在疾病进展或不可接受的毒性之前接受依可他单抗治疗。主要终点是总缓解率(ORR)。中位年龄为64.0岁,39%的患者既往接受过CAR - t细胞治疗,75%的患者对≥2个连续治疗线难以治愈。截至2024年5月3日(中位随访37.1个月[范围,0.3-45.5]),研究者评估ORR为59%,完全缓解(CR)率为41%。中位反应持续时间为20.8个月(95%可信区间[CI], 13.0-32.0)。中位CR持续时间为36.1个月(20.2个月未达到[NR]);持续时间最长的CR为1043个月。所有患者的中位无进展生存期为4.2个月(95% CI, 2.8-5.5), CR患者的中位无进展生存期为37.3个月(26.0-NR),所有患者的中位总生存期为18.5个月(95% CI, 11.7-27.7), CR患者的中位无进展生存期为NR,在119例可评估最小残留疾病(MRD)的患者中,54例(45%)在研究期间的任何时候均为MRD阴性。最常见的不良事件是细胞因子释放综合征(51%)、疲劳(25%)和发热(25%),没有新的安全信号。1、2和3级感染分别发生在23%、34%和24%的患者中。完全缓解者反应的持久性和延长的生存期表明,在这些复发/难治性LBCL患者中,使用依可单抗可以长期无病生存。
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引用次数: 0
Efficacy and safety of belumosudil for refractory chronic graft-versus-host disease in routine practice. 白莫硫地尔治疗难治性慢性移植物抗宿主病的临床疗效和安全性。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-04 DOI: 10.1007/s00277-026-06760-4
Garret M K Leung, Joycelyn P Y Sim, Thomas S Y Chan, Carol Y M Cheung, Eric Tse, Albert K W Lie, Harinder Gill, Yok-Lam Kwong
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引用次数: 0
Plasma exchange combined with anti-B-cell therapy enables comparable outcomes between DSA-positive and DSA-negative aplastic anemia after haplo-HSCT: a propensity-score-matched cohort study. 血浆置换联合抗b细胞治疗使得单倍造血干细胞移植后dsa阳性和dsa阴性再生贫血的结果具有可比性:一项倾向评分匹配的队列研究。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-04 DOI: 10.1007/s00277-026-06815-6
Zhengwei Tan, Jinyu Hu, Yuechao Zhao, Huijin Hu, Qinghong Yu, Yu Zhang, Liqiang Wu, Tonglin Hu, Dijiong Wu, Baodong Ye, Wenbin Liu

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative treatment for aplastic anemia (AA). For patients lacking a matched sibling donor (MSD) or matched unrelated donor (MUD), haploidentical HSCT (haplo-HSCT) has become an alternative with comparable efficacy. However, donor-specific anti-HLA antibodies (DSAs) remain the principal barrier. In this study, we compared transplant outcomes in DSA-positive recipients after desensitization with plasma exchange (PE) combined with anti-B-cell therapy. Between January 2019 and December 2023, we enrolled 30 DSA-positive AA patients who underwent haplo-HSCT at our center. All received a desensitization regimen combining PE with anti-B-cell agents. By propensity-score matching (1:2 ratio, caliper 0.02), we additionally enrolled 60 DSA-negative controls matched for key clinical variables to compare therapeutic efficacy and long-term outcomes between the two groups. This study enrolled 90 AA patients who underwent haplo-HSCT: 30 were DSA-positive and 60 were DSA-negative. Baseline characteristics were comparable between the two groups. After desensitization with PE combined with anti-B-cell agents, the DSA-positive group achieved outcomes equivalent to those of the DSA-negative group. Specifically, EBV reactivation (46.7% vs. 68.3%; P = 0.002) was significantly better in the DSA-positive group. Engraftment, aGVHD, Effacacy, OS, and GRFS rates were similar between two groups. However, the risk of cGVHD remains higher in the DSA-positive group (40% vs. 12%; P = 0.009). For DSA-positive AA patients who are candidates for haplo-HSCT and for whom no DSA-negative donor can be identified, desensitization with PE combined with anti-B-cell agents represents an established strategy to improve transplant outcomes; nevertheless, close surveillance for cGVHD is warranted after transplantation.

同种异体造血干细胞移植(allo-HSCT)仍然是治疗再生障碍性贫血的唯一方法。对于缺乏匹配兄弟姐妹供体(MSD)或匹配非亲属供体(MUD)的患者,单倍同型HSCT(单倍-HSCT)已成为具有相当疗效的替代方法。然而,供体特异性抗hla抗体(dsa)仍然是主要的障碍。在这项研究中,我们比较了dsa阳性受体在血浆置换(PE)联合抗b细胞治疗脱敏后的移植结果。在2019年1月至2023年12月期间,我们招募了30名dsa阳性AA患者,他们在我们的中心接受了单倍造血干细胞移植。所有患者均接受PE联合抗b细胞药物的脱敏治疗。通过倾向评分匹配(1:2比,卡尺0.02),我们额外招募了60名dsa阴性对照,匹配关键临床变量,比较两组的治疗效果和长期结局。本研究招募了90例接受单倍造血干细胞移植的AA患者:30例dsa阳性,60例dsa阴性。两组患者的基线特征具有可比性。经PE联合抗b细胞药物脱敏后,dsa阳性组的结果与dsa阴性组相当。具体而言,dsa阳性组EBV再激活(46.7% vs. 68.3%; P = 0.002)明显更好。两组间植入率、aGVHD、Effacacy、OS和GRFS相似。然而,dsa阳性组cGVHD的风险仍然较高(40% vs. 12%; P = 0.009)。对于dsa阳性的AA患者,作为单倍hsct的候选者,并且无法确定dsa阴性供体,PE脱敏联合抗b细胞药物代表了改善移植结果的既定策略;然而,移植后密切监测cGVHD是必要的。
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引用次数: 0
Neurofibromatosis type I -related severe aplastic anemia: an unusual association with a complicated bone marrow transplantation course. I型神经纤维瘤病相关的严重再生障碍性贫血:与复杂骨髓移植病程的不寻常关联。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-04 DOI: 10.1007/s00277-026-06752-4
Farah Malaeb, Nahla Mobarak, Dayel Alshahrani, Fahad Almanjomi
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引用次数: 0
Dynamic changes in mortality risk factors for acute leukemia patients in the ICU and Post-ICU periods. 急性白血病患者ICU期及ICU后死亡危险因素的动态变化
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-04 DOI: 10.1007/s00277-026-06744-4
Linli Lu, Qi Liang, YuHan Yan, Yishu Tang, Erhua Wang, Ying Li, Jian Zhang, Han Xiao, Qian Cheng, Xin Li

Acute leukemia (AL) is an aggressive hematologic malignancy that often causes serious complications and requires intensive care unit (ICU) treatment. Identifying risk factors for mortality in AL patients during and post-ICU admission can improve prognosis. This retrospective study enrolled AL patients first admitted to the ICU of the Third Xiangya Hospital, Central South University, from November 2008 to October 2023. It analyzed risk factors associated with ICU mortality, 1-month mortality and 6-month mortality, and conducted a subgroup analysis of the prognosis of ICU survivors. A total of 126 patients were included in this study, with a median age of 42 years. Acute respiratory failure (46%) and sepsis (23%) were the main reasons for ICU admission. Overall, the mortality rates were 57.9% in the ICU, 67.5% at 1 month, and 69% at 6 months. Notably, among the ICU survivors, 26.4% (14/53) died within 6 months, with 85.7% (12/14) occurring within the first month. The time from hospital admission to ICU transfer >10 days and invasive mechanical ventilation were independent risk factors for ICU mortality, 1-month mortality and 6-month mortality. Relapsed or refractory leukemia (OR = 20.715) and low albumin levels (OR = 34.428) were independent risk factors for death within one month among ICU survivors. The risk factors for mortality in AL patients admitted to the ICU exhibit dynamic changes across different stages. For ICU survivors, optimizing nutritional support and initiating anti-tumor treatment as early as possible after discharge are crucial for improving prognosis.

急性白血病(AL)是一种侵袭性血液系统恶性肿瘤,经常引起严重的并发症,需要重症监护病房(ICU)治疗。确定AL患者在icu住院期间和住院后死亡的危险因素可以改善预后。本回顾性研究纳入2008年11月至2023年10月首次入住中南大学湘雅第三医院ICU的AL患者。分析ICU病死率、1个月病死率、6个月病死率相关危险因素,并对ICU存活患者预后进行亚组分析。本研究共纳入126例患者,中位年龄42岁。急性呼吸衰竭(46%)和脓毒症(23%)是住院的主要原因。总体而言,ICU的死亡率为57.9%,1个月死亡率为67.5%,6个月死亡率为69%。值得注意的是,在ICU幸存者中,26.4%(14/53)在6个月内死亡,85.7%(12/14)发生在第一个月内。入院至转ICU时间bbb10 d和有创机械通气是ICU死亡率、1个月死亡率和6个月死亡率的独立危险因素。复发或难治性白血病(or = 20.715)和低白蛋白水平(or = 34.428)是ICU存活患者1个月内死亡的独立危险因素。ICU住院AL患者死亡率的危险因素在不同阶段呈现动态变化。对于ICU存活患者,优化营养支持,出院后尽早开始抗肿瘤治疗对改善预后至关重要。
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引用次数: 0
Addition of rituximab to CEAC conditioning for autologous hematopoietic stem cell transplantation provides no survival benefit in diffuse large B-Cell lymphoma: A propensity score-matched cohort study. 弥漫性大b细胞淋巴瘤患者弥漫性大b细胞淋巴瘤患者弥漫性大b细胞淋巴瘤患者:一项倾向评分匹配的队列研究
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-03 DOI: 10.1007/s00277-026-06834-3
Chaoshuai Fan, Jiabang Yang, Yanni Peng, Juan Hu, Mingyue Cao, Ruobing Li, Dongmei Jiang, Tao Wang, Wen Luo, Li Chen, Yanrong Luo, Guihua Lu
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引用次数: 0
Impact of marital status on survival in patients with follicular lymphoma. 婚姻状况对滤泡性淋巴瘤患者生存的影响。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-02-02 DOI: 10.1007/s00277-026-06731-9
Songyuan He, Hao Song, Weiyi Zuo, Shu Liu, Xiaojie Fang, Xudong Li

Although marital status has been associated with survival in several cancers, evidence regarding its prognostic relevance in follicular lymphoma (FL), an indolent lymphoma characterized by prolonged survival, remains limited. In this population-based study using the SEER database, we identified 23,434 adult patients diagnosed with FL between 2013 and 2022. Propensity score matching was performed to balance baseline characteristics, and survival outcomes were assessed using Kaplan-Meier analysis and Cox proportional hazards models. In the overall cohort, unmarried patients were more likely to be female, older, and present with B symptoms (all P < 0.001), and demonstrated significantly lower 5-year overall survival (OS: 72.8% vs. 81.0%, P < 0.001) and cause-specific survival (CSS: 84.9% vs. 90.6%, P < 0.001) compared with married patients. After 1:1 propensity score matching on age, sex, race/ethnicity, Ann Arbor stage, B symptoms, treatment status, prior cancer history, and diagnosis year (matched cohort, n = 16,384), this survival disparity persisted (5-year OS: 72.5% vs. 81.3%; CSS: 84.7% vs. 90.7%; both P < 0.001). Multivariable Cox regression confirmed that unmarried status was independently associated with increased risks of OS (HR, 1.62; 95% CI, 1.53-1.71, P < 0.001) and CSS (HR, 1.77; 95% CI, 1.63-1.92, P < 0.001). These findings suggest that unmarried status is an independent adverse prognostic factor in patients with FL and highlight additional attention may be warranted for unmarried individuals during long-term disease management.

尽管婚姻状况与几种癌症的生存有关,但其与滤泡性淋巴瘤(FL)预后相关性的证据仍然有限,滤泡性淋巴瘤是一种以生存时间延长为特征的惰性淋巴瘤。在这项使用SEER数据库的基于人群的研究中,我们确定了2013年至2022年间诊断为FL的23,434名成年患者。进行倾向评分匹配以平衡基线特征,并使用Kaplan-Meier分析和Cox比例风险模型评估生存结果。在整个队列中,未婚患者更可能是女性,年龄较大,并出现B型症状(均为P
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引用次数: 0
Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia 儿童再生障碍性贫血环孢素治疗的机器学习死亡率预测模型
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-31 DOI: 10.1007/s00277-026-06842-3
Xianhao Wen, Li Xiao, Danni Li, Meiling Liao, Yuye Liu, Qihui Liu, Xianmin Guan, Ying Dou, Ziyu Hua

The outcomes of children with aplastic anemia receiving cyclosporine monotherapy vary significantly in terms of mortality risk; therefore, a prognostic model for predicting mortality risk was constructed to optimize risk-stratified treatment strategies. This retrospective cohort study included children with acquired AA receiving cyclosporine-based immunosuppression, stratified by disease severity (vSAA/SAA/NSAA) and randomly split into training (70%) and validation (30%) cohorts. Ten machine learning models were developed; hyperparameters were optimized via grid search with 10-fold cross-validation exclusively within the training cohort to prevent data leakage. Model performance was evaluated using area under the ROC curve (AUC), accuracy, recall, specificity, precision, F1 score, and Brier score. Decision curve analysis (DCA) quantified clinical net benefit. The calibration curve was used to evaluate the reliability of the predicted probabilities. The SHapley Additive exPlanations (SHAP) framework was used to interpret feature contributions and ensure model transparency. Least absolute shrinkage and selection operator (LASSO) regression on the training cohort identified 5 predictors: reticulocyte count (RC), platelet count (PLT), disease subtype (vSAA/SAA/NSAA), total bilirubin (TB), and bone marrow myeloid proportion. The CatBoost model achieved the highest performance: AUC 0.834 (95% CI: 0.774–0.895) in training and 0.826 (95% CI: 0.743–0.910) in validation, with acceptable calibration (Brier score: 0.206 in training cohort, 0.207 in validation cohort). SHAP analysis confirmed RC as the top contributor, with lower RC values associated with higher predicted mortality risk. The CatBoost model demonstrates robust performance and transparency for predicting mortality risk in children with AA after cyclosporine treatment. Adherence to TRIPOD + AI guidelines ensures methodological rigor, supporting its potential as a clinical decision tool to stratify patients into distinct mortality risk groups and optimize individualized treatment strategies.

再生障碍性贫血儿童接受环孢素单药治疗的结果在死亡风险方面存在显著差异;因此,我们构建了预测死亡风险的预后模型,以优化风险分层治疗策略。本回顾性队列研究纳入接受环孢素免疫抑制的获得性AA患儿,按疾病严重程度(vSAA/SAA/NSAA)分层,随机分为训练(70%)和验证(30%)队列。开发了10个机器学习模型;通过网格搜索优化超参数,并在训练队列中进行10倍交叉验证,以防止数据泄漏。采用ROC曲线下面积(AUC)、准确率、召回率、特异性、精确度、F1评分和Brier评分来评价模型的性能。决策曲线分析(DCA)量化临床净收益。用标定曲线评价预测概率的可靠性。SHapley加性解释(SHAP)框架用于解释特征贡献并确保模型透明度。训练队列的最小绝对收缩和选择算子(LASSO)回归确定了5个预测因子:网织红细胞计数(RC)、血小板计数(PLT)、疾病亚型(vSAA/SAA/NSAA)、总胆红素(TB)和骨髓比例。CatBoost模型获得了最高的性能:训练中的AUC为0.834 (95% CI: 0.774-0.895),验证中的AUC为0.826 (95% CI: 0.743-0.910),校准可接受(训练队列中的Brier评分为0.206,验证队列中的Brier评分为0.207)。SHAP分析证实RC是最大贡献者,较低的RC值与较高的预测死亡风险相关。CatBoost模型在预测AA患儿环孢素治疗后的死亡风险方面表现出稳健的性能和透明度。遵守TRIPOD + AI指南可确保方法的严密性,支持其作为临床决策工具的潜力,将患者划分为不同的死亡风险组并优化个性化治疗策略。
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引用次数: 0
Next-generation Bruton’s tyrosine kinase inhibitors for chronic lymphocytic leukemia-associated membranoproliferative glomerulonephritis: a case report 新一代布鲁顿酪氨酸激酶抑制剂治疗慢性淋巴细胞白血病相关膜增生性肾小球肾炎1例报告
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2026-01-30 DOI: 10.1007/s00277-026-06730-w
Li Chen, Yuting Huang, Jing Xu, Sinian Huang, Qianying Zhang, Fanli Hua, Xiaoxia Pan, Jian-Qing Mi

Renal involvement in chronic lymphocytic leukemia (CLL) is uncommon but can lead to significant morbidity. Membranoproliferative glomerulonephritis (MPGN) is among the most frequently reported glomerular lesions associated with CLL and may presents with nephrotic syndrome. Early recognition of the association between renal lesions and CLL is crucial for guiding treatment and improving both renal and hematologic outcomes. We report two biopsy-proven cases of CLL-associated MPGN successfully treated with next-generation Bruton’s tyrosine kinase inhibitors (BTKis). Both patients presented with nephrotic-range proteinuria. In Case 1, the patient exhibited monoclonal IgG-κ gammopathy and isolated low serum C3, suggestive of complement-mediated injury without direct immunoglobulin deposition. He achieved sustained hematologic and renal remission with orelabrutinib following early discontinuation of rituximab–chlorambucil due to infection. In Case 2, renal biopsy showed interstitial infiltration by CLL cells and immune complex deposition, supporting a leukemic infiltration and immune-complex mediated mechanism. Zanubrutinib led to clinical improvement, and rituximab was later added to further reduce proteinuria. These cases underscore the critical role of kidney biopsy in clarifying diagnosis and underlying mechanisms. In this case series, the treatment regimen centered on next-generation BTKis enabled patients to achieve concurrent favorable renal and hematologic remission with good tolerability.

慢性淋巴细胞白血病(CLL)肾脏受累并不常见,但可导致显著的发病率。膜增生性肾小球肾炎(MPGN)是最常报道的与CLL相关的肾小球病变之一,可能表现为肾病综合征。早期识别肾脏病变与CLL之间的关系对于指导治疗和改善肾脏和血液学预后至关重要。我们报告了两例活检证实的cll相关MPGN病例,成功地用下一代布鲁顿酪氨酸激酶抑制剂(BTKis)治疗。两例患者均表现为肾范围蛋白尿。在病例1中,患者表现出单克隆IgG-κ γ病和孤立的低血清C3,提示补体介导的损伤,没有直接的免疫球蛋白沉积。他在早期因感染停用利妥昔单抗-氯霉素后,使用奥瑞鲁替尼实现了持续的血液学和肾脏缓解。病例2,肾活检显示间质有CLL细胞浸润和免疫复合物沉积,支持白血病浸润和免疫复合物介导的机制。扎努鲁替尼导致临床改善,随后加入利妥昔单抗进一步减少蛋白尿。这些病例强调了肾活检在明确诊断和潜在机制中的关键作用。在这个病例系列中,以下一代BTKis为中心的治疗方案使患者同时获得良好的肾脏和血液缓解,并具有良好的耐受性。
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引用次数: 0
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Annals of Hematology
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