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Real-world evaluation of belumosudil for chronic ocular graft-versus-host-disease: Efficacy and outcomes. 白莫硫地尔治疗慢性眼部移植物抗宿主病的实际评价:疗效和结果。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-23 DOI: 10.1038/s41409-026-02846-9
Leonie Menghesha, Tim Richardson, Jana Knuever, Udo Holtick, Christof Scheid, Michael E Stern, Philipp Steven
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引用次数: 0
Is sex chromosome loss a predictive marker for outcome after sex-mismatched allogeneic hematopoietic stem cell transplantation? 性染色体缺失是性别不匹配异基因造血干细胞移植后预后的预测指标吗?
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-20 DOI: 10.1038/s41409-026-02828-x
Lu Gao, Na Li, Lin Feng, Xiaohui Zhang, Lanping Xu, Yu Wang, Yingjun Chang, Xiaojun Huang, Yueyun Lai

Sex chromosome loss (SCL) occasionally occurs after sex-mismatched allogeneic hematopoietic stem cell transplantation (allo-HSCT), but its clinical significance remains unclear. This retrospective study analyzed 78 patients who developed SCL after sex-mismatched HSCT, comprising 27 cases with initial-onset SCL (detected within first month post-transplant) and 51 cases with late-onset SCL (median onset time: 6.1 months; range: 2-73.1 months). Based on the results of chimerism analysis, initial-onset SCL was found to predominantly reflect the physiological aging hematopoietic cells from older donors, rather than relapse-related clonal abnormalities. In contrast, late-onset SCL was significantly associated with worse overall survival (OS) (HR 8.190,95% CI 2.842-23.600), inferior event-free survival (EFS) (HR 4.691,95% CI 1.839-11.966), and higher relapse risk (HR 6.751,95% CI 1.912-23.841). Among late-onset SCL patients, the optimal initial SCL clone size threshold for predicting relapse was 6% (sensitivity of 88.6%, specificity of 75.0%). The multivariate analysis confirmed ≥6% initial SCL clone size as an independent risk factor for relapse (HR 3.546, 95%CI 1.540-8.161), EFS(HR 3.418, 95% CI 1.572-7.433) and OS (HR 11.665, 95% CI 3.926-34.663). In conclusion, late-onset SCL may be associated with poor clinical outcomes, with the ≥6% clonal threshold serving as a critical marker for identifying high-risk patients prone to treatment failure.

性染色体丢失(SCL)偶尔发生在性别不匹配的同种异体造血干细胞移植(alloo - hsct)后,但其临床意义尚不清楚。本回顾性研究分析了78例性别不匹配HSCT后发生SCL的患者,包括27例初发性SCL(移植后1个月内发现)和51例迟发性SCL(中位发病时间:6.1个月;范围:2-73.1个月)。根据嵌合分析的结果,发现初始性SCL主要反映来自老年供者的生理性老化造血细胞,而不是复发相关的克隆异常。相比之下,迟发性SCL与较差的总生存期(OS) (HR 8.190,95% CI 2.842-23.600)、较差的无事件生存期(EFS) (HR 4.691,95% CI 1.839-11.966)和较高的复发风险(HR 6.751,95% CI 1.912-23.841)显著相关。在迟发性SCL患者中,预测复发的最佳初始SCL克隆大小阈值为6%(敏感性为88.6%,特异性为75.0%)。多因素分析证实,初始SCL克隆大小≥6%是复发的独立危险因素(HR 3.546, 95%CI 1.540-8.161)、EFS(HR 3.418, 95%CI 1.572-7.433)和OS (HR 11.665, 95%CI 3.926-34.663)。综上所述,迟发性SCL可能与较差的临床结果相关,≥6%克隆阈值可作为识别易发生治疗失败的高危患者的关键标志。
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引用次数: 0
Impact of acute GvHD severity on survival and disease relapse in pediatric patients undergoing allogeneic stem cell transplantation for acute lymphoblastic leukemia: an analysis based on the EBMT-PDWP registry. 急性GvHD严重程度对接受同种异体干细胞移植治疗急性淋巴细胞白血病的儿童患者生存和疾病复发的影响:基于EBMT-PDWP登记的分析
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-19 DOI: 10.1038/s41409-026-02797-1
Z Szmit, G Lucchini, A Bertaina, J E Galimard, J H Dalle, F Locatelli, M Taskinen, O Mirci-Danicar, P Sedlacek, A Balduzzi, I Ghemlas, J Styczynski, V Kitra-Roussou, H Hashem, F Fagioli, Y Bertrand, M Aljurf, M Ifversen, A Dalissier, P Bader, K Kalwak, S Corbacioglu

This retrospective registry-based study investigated the role of acute GvHD in the largest reported pediatric population with acute lymphoblastic leukemia (ALL) undergoing allogeneic stem cell transplantation (HSCT). The study included 3328 children. Median age was 9.6 years (IQR 6-13.9 and range 0.4-18). Forty-nine percent were transplanted from an MSD, 37% from MUD, 14% from MMUD; 68% received a TBI-based conditioning. CSA alone or in combination with methotrexate/MMF was administered as GVHD prophylaxis in 94%. The 100-day cumulative incidence of grade II-IV acute GVHD (aGVHD) was 35% (95% CI: 34-37), while that of grade III-IV was 11% (95% CI: 10-12). Two-year cumulative incidence of chronic GVHD (cGVHD) was 17% (95% CI: 15-18)/extensive cGVHD 8% (95% CI: 7-9). With a median follow-up of 3.1 years, OS, leukemia-free survival (LFS), and GVHD-free/relapse-free survival were 74% (95% CI 72-76), 65% (95% CI 64-67) and 51% (95% CI 49-53), respectively. Using multivariable Cox models in 100-day landmark analysis, any degree of aGVHD was associated with reduced risk of relapse; grade III-IV significantly increased NRM and reduced OS; grade II aGVHD offers the best chances of achieving prolonged LFS (HR = 0.78 (0.64-0.94), p = 0.008). Overall, these data confirm that grade II aGvHD prevents the occurrence of leukaemia relapse without causing unacceptable mortality.

这项基于登记的回顾性研究调查了急性GvHD在接受同种异体干细胞移植(HSCT)的急性淋巴细胞白血病(ALL)儿童中最大的报道人群中的作用。该研究包括3328名儿童。中位年龄为9.6岁(IQR 6-13.9,范围0.4-18)。49%移植自MSD, 37%移植自MUD, 14%移植自MMUD;68%的人接受了基于tbi的调理。单独使用CSA或联合使用甲氨蝶呤/MMF预防GVHD的比例为94%。II-IV级急性GVHD (aGVHD)的100天累积发病率为35% (95% CI: 34-37),而III-IV级的累积发病率为11% (95% CI: 10-12)。慢性GVHD (cGVHD)的两年累积发病率为17% (95% CI: 15-18)/广泛性cGVHD为8% (95% CI: 7-9)。中位随访时间为3.1年,OS、无白血病生存率(LFS)和无gvhd /无复发生存率分别为74% (95% CI 72-76)、65% (95% CI 64-67)和51% (95% CI 49-53)。在100天里程碑分析中使用多变量Cox模型,任何程度的aGVHD都与复发风险降低相关;III-IV级显著增加NRM,降低OS;II级aGVHD获得延长LFS的机会最大(HR = 0.78 (0.64-0.94), p = 0.008)。总的来说,这些数据证实,II级aGvHD可以防止白血病复发,而不会造成不可接受的死亡率。
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引用次数: 0
Can pre-transplant sickle cell disease characteristics predict early post-transplant complications? 移植前镰状细胞病的特征能否预测移植后早期并发症?
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-19 DOI: 10.1038/s41409-026-02847-8
Alaa Kintab, Mohamed Bayoumy, Enass H Raffa, Amal AlSeraihy, Ibraheem Abosoudah, Amany Orabe, Hassan Altrabolsi, Shady Kamel, Shaker Abdulla, Rania Jaha, Wasil Jastaniah
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引用次数: 0
Donor age ≥50 years combined with donor-CI ≥3 identifies a high-risk cohort for adverse outcomes after allogeneic hematopoietic stem cell transplantation in B-cell acute lymphoblastic leukemia. 供者年龄≥50岁且供者ci≥3确定了b细胞急性淋巴细胞白血病异体造血干细胞移植后不良后果的高风险队列。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-18 DOI: 10.1038/s41409-026-02844-x
Ning Wu, Jun Kong, Meng Lv, Xiao-Dong Mo, Yu-Qian Sun, Yi-Fei Cheng, Lan-Ping Xu, Xiao-Hui Zhang, Xiao-Jun Huang, Yu Wang
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引用次数: 0
Hematopoietic stem cell transplantation in pediatric congenital erythropoietic porphyria: a French retrospective multicenter registry study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC). 造血干细胞移植治疗儿童先天性红细胞生成性卟啉症:代表法语国家骨髓移植和细胞治疗协会(SFGM-TC)进行的一项法国回顾性多中心注册研究。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-18 DOI: 10.1038/s41409-026-02824-1
Charlotte Nazon, Eléna Muninger, Nicole Raus, Cécile Renard, Anne Sirvent, Arthur Sterin, Despina Moshous, Catherine Paillard
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引用次数: 0
Differential clinical impact of HLA-DPA1~DPB1 linkage mismatches in 14/14-matched unrelated donor HSCT: a multicenter retrospective study from CMDP revealing age- and disease-specific risk patterns. HLA-DPA1~DPB1连锁错配在14/14匹配的非亲属供体HSCT中的差异临床影响:一项来自CMDP的多中心回顾性研究揭示了年龄和疾病特异性风险模式。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-18 DOI: 10.1038/s41409-026-02835-y
Tengteng Zhang, Xiaojing Bao, Ying Han, Xiaoni Yuan, Yang Li, Xue Jiang, Tianjie Yang, Dan Du, Jun He
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引用次数: 0
Pacritinib in transplant-eligible myelofibrosis: final analysis of the phase II HOVON-134 trial. 帕西替尼治疗符合移植条件的骨髓纤维化:HOVON-134 II期试验的最终分析
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-18 DOI: 10.1038/s41409-026-02841-0
R Van Dijck, B van der Holt, A E C Broers, M D Hazenberg, T Devos, M W M van der Poel, D Breems, L Deleu, B Schuthof, C M C van Hooije, J J Cornelissen, N P M Schaap, P A W Te Boekhorst
{"title":"Pacritinib in transplant-eligible myelofibrosis: final analysis of the phase II HOVON-134 trial.","authors":"R Van Dijck, B van der Holt, A E C Broers, M D Hazenberg, T Devos, M W M van der Poel, D Breems, L Deleu, B Schuthof, C M C van Hooije, J J Cornelissen, N P M Schaap, P A W Te Boekhorst","doi":"10.1038/s41409-026-02841-0","DOIUrl":"https://doi.org/10.1038/s41409-026-02841-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147479750","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advanced deep learning enables prediction of allogeneic stem cell mobilization success. 先进的深度学习能够预测异体干细胞动员的成功。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-17 DOI: 10.1038/s41409-026-02811-6
Asif Adil, Jingyu Xiang, Nicola Piccirillo, Hillary G Harris, Simona Sica, John F DiPersio, Stephanie N Hurwitz
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引用次数: 0
Imaging biomarkers to predict the development of sinusoidal obstruction syndrome of the liver in pediatric hematopoietic cell transplant recipients. 预测儿童造血细胞移植受者肝窦阻塞综合征发展的影像学生物标志物。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2026-03-17 DOI: 10.1038/s41409-026-02815-2
Elizabeth M Holland, Shruthi Suryaprakash, Yu Bi, Akshay Patwardhan, Dinesh Keerthi, Cara E Morin, Sherwin Chan, Subodh Selukar, Akshay Sharma
{"title":"Imaging biomarkers to predict the development of sinusoidal obstruction syndrome of the liver in pediatric hematopoietic cell transplant recipients.","authors":"Elizabeth M Holland, Shruthi Suryaprakash, Yu Bi, Akshay Patwardhan, Dinesh Keerthi, Cara E Morin, Sherwin Chan, Subodh Selukar, Akshay Sharma","doi":"10.1038/s41409-026-02815-2","DOIUrl":"https://doi.org/10.1038/s41409-026-02815-2","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473054","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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Bone Marrow Transplantation
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