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Successful treatment with Tislelizumab and Zanubrutinib in a patient with relapsed/refractory, CD19/CD20 negative Richter’s transformed CLL Tislelizumab和Zanubrutinib成功治疗复发/难治性CD19/CD20阴性Richter’s转化型CLL患者。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-15 DOI: 10.1038/s41409-025-02737-5
Rina Latscha, Dominik Heim, Benjamin Kasenda, Thomas Menter, Jakob Passweg, Andreas Holbro
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引用次数: 0
Transplant conditioning intensity (TCI) score predicts allo-HCT outcomes in patients with myelofibrosis: a study of the Chronic Malignancies Working Party of EBMT 移植适应强度(TCI)评分预测骨髓纤维化患者的同种异体hct结果:EBMT慢性恶性肿瘤工作组的一项研究。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-15 DOI: 10.1038/s41409-025-02732-w
Patryk Sobieralski, Tomasz Czerw, Luuk Gras, Linda Koster, Nicolaus Kröger, Thomas Schroeder, Lone Friis, Elisabetta Metafuni, Jakob Passweg, Marie Robin, Matthias Stelljes, Annoek E. C. Broers, Patrice Chevallier, Robert Zeiser, Marie Therese Rubio, Mareike Verbeek, Ipek Yonal-Hindilerden, Domenico Pastore, Jan Zaucha, Kavita Raj, Joanna Drozd-Sokołowska, Giorgia Battipaglia, Nicola Polverelli, Juan Carlos Hernández-Boluda, Donal P. McLornan
Outcomes in myelofibrosis (MF) patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) appear unaffected by the intensity of the preparative regimen, defined traditionally as myeloablative (MAC) or reduced intensity conditioning (RIC). The Transplant Conditioning Intensity (TCI) index is an objective tool offering a precise measure of conditioning intensity. We explored the potential association between TCI score and overall survival (OS), progression-free survival (PFS), cumulative incidence of relapse (CIR) and non-relapse mortality (NRM) in 2454 MF patients undergoing allo-HCT between 2012 and 2021, selected from the EBMT registry. Patients receiving TCI-intermediate/high regimens had similar OS (HR 1.12, 95% CI 0.97–1.30) and PFS (HR 1.00, 95% CI 0.88–1.14) compared to TCI-low regimens. However, TCI-intermediate/high regimens were associated with lower risk of relapse (HR 0.74, 95% CI 0.61–0.91, p = 0.008) and higher risk of NRM (HR 1.24, 95% CI 1.04–1.48, p = 0.02). Our findings suggest that the TCI score provides a more clinically relevant stratification of conditioning intensity than the conventional MAC/RIC classification. While higher intensity TCI regimens are associated with lower RI, this benefit is offset by increased NRM, resulting in no survival advantage. However, the TCI index may enable a more personalized approach to conditioning regimen selection by balancing relapse risk with patient frailty.
骨髓纤维化(MF)患者接受同种异体造血细胞移植(allogenic hematopoietic cell transplantation, alloo - hct)的结果似乎不受预备方案强度的影响,传统上定义为骨髓清除(MAC)或降低强度调节(RIC)。移植调节强度(TCI)指数是提供调节强度精确测量的客观工具。我们从EBMT注册表中选择了2454例2012年至2021年间接受异位肝移植的MF患者,探讨了TCI评分与总生存期(OS)、无进展生存期(PFS)、累积复发发生率(CIR)和非复发死亡率(NRM)之间的潜在关联。与低tci方案相比,接受tci中高方案的患者具有相似的OS (HR 1.12, 95% CI 0.97-1.30)和PFS (HR 1.00, 95% CI 0.88-1.14)。然而,tci -中/高方案与较低的复发风险(HR 0.74, 95% CI 0.61-0.91, p = 0.008)和较高的NRM风险(HR 1.24, 95% CI 1.04-1.48, p = 0.02)相关。我们的研究结果表明,与传统的MAC/RIC分类相比,TCI评分提供了更具有临床相关性的调节强度分层。虽然高强度TCI方案与较低的RI相关,但这种益处被增加的NRM抵消,导致没有生存优势。然而,TCI指数可以通过平衡复发风险和患者虚弱来实现更个性化的治疗方案选择。
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引用次数: 0
Multiple myeloma incidence, transplant utilization, and mortality- impact of social vulnerability 多发性骨髓瘤发病率、移植利用和死亡率——社会脆弱性的影响。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-13 DOI: 10.1038/s41409-025-02733-9
James Fan Wu, Noel Estrada-Merly, Yuhong Zhou, Bethany Canales, Tina W. F. Yen, Anita D’Souza
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引用次数: 0
Preserved measles immunity after allogeneic hematopoietic stem cell transplantation in a cohort of mainly αβ T-cell-depleted graft recipients 异体造血干细胞移植后在主要αβ t细胞缺失的移植受体中保留麻疹免疫
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-06 DOI: 10.1038/s41409-025-02731-x
M. J. M. Janssen, M. A. de Witte, L.G.M. Daenen, F. Verduyn Lunell, D. van Baarle, J. Kuball, A.H.W. Bruns
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引用次数: 0
Upfront memory T cell add-back with haploidentical TCRαβ-depleted graft in adults with haematological malignancies: a nationwide, multicentre, single-arm, prospective study 一项全国性、多中心、单臂、前瞻性研究:成人血液学恶性肿瘤患者的前期记忆T细胞加回与单倍体tcr αβ-耗尽移植
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-06 DOI: 10.1038/s41409-025-02728-6
Liang-Piu Koh, Yeh Ching Linn, Yang Liang Boo, Victor Ling, Zi Yi Lim, Michelle Poon, Jeffrey Quek, Hein Than, Colin Phipps Diong, Balamurugan Vellayappan, Aloysius Ho, Francesca Lim, William Hwang, Lip Kun Tan, Jean Rachel M. Catapia, Joanne Lee, Ian Wu, Kheng Wei Yeoh, Bryan Ho, Wen Shen Looi, Lawrence Ng, Tertius Tansloan Tuy, Yvonne Loh, Teck Guan Soh, Gina Gan, Kee Khiang Heng, Yin Jie Koh, Wing Leung
In haploidentical hematopoietic cell transplantation (Haplo-HCT), in vivo or ex vivo T-cell depletion (TCD) can prevent graft-versus-host disease (GVHD) but increase risk of infection and relapse. We hypothesized that TCRαβ-depleted allograft together with upfront infusion with CD45RA-depleted memory T cells (αβTCD + TMDLI) may result in favourable GVHD-free and relapse-free survival (GRFS). Between January 2017 and July 2023, 145 adult patients with various haematological malignancies received αβTCD + TMDLI. All except 2 patients had robust engraftment at a median of 12 days for neutrophil and 11 days for platelet. The cumulative incidence (CI) of CMV, EBV, HHV6 or ADV infection was only 43% (n = 63) at day+120. CI of grade II–IV and III–IV acute GVHD at 180 days was 31% and 8% respectively. Chronic GVHD was seen in only 5 patients with a 2-year CI of 4%. CI of non-relapse mortality and relapse at 2 years were 17% and 22% respectively. At a median follow up of 28 months, 3-year overall (OS), event-free (EFS), and GRFS were 67%, 62%, and 59%, respectively. This was significantly improved over a propensity score-matched contemporary cohort (n = 53) who received PTCy as GVHD prophylaxis. This first multi-center study demonstrated the potential benefits of the αβTCD + TMDLI approach for Haplo-HCT.
在单倍体造血细胞移植(haploo - hct)中,体内或体外t细胞消耗(TCD)可以预防移植物抗宿主病(GVHD),但增加感染和复发的风险。我们假设tcr αβ缺失同种异体移植物与cd45ra缺失记忆T细胞(αβTCD + TMDLI)的预先输注可能导致良好的无gvhd和无复发生存(GRFS)。2017年1月至2023年7月,145例不同血液病恶性肿瘤患者接受αβTCD + TMDLI治疗。除2例患者外,其余患者在中性粒细胞和血小板的中位植入时间分别为12天和11天。在第120天,CMV、EBV、HHV6或ADV感染的累积发生率(CI)仅为43% (n = 63)。II-IV级和III-IV级急性GVHD 180 d时CI分别为31%和8%。慢性GVHD仅出现在5例患者中,2年CI为4%。非复发死亡率和2年复发CI分别为17%和22%。在中位随访28个月时,3年总体(OS)、无事件(EFS)和GRFS分别为67%、62%和59%。在倾向评分匹配的当代队列(n = 53)中,接受PTCy作为GVHD预防的患者,这一点得到了显著改善。这项首次多中心研究证明了αβTCD + TMDLI方法治疗haploi - hct的潜在益处。
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引用次数: 0
Comparison of single-unit umbilical cord blood transplantation and haploidentical transplantation using posttransplant cyclophosphamide during first complete remission of acute myeloid leukemia 急性髓系白血病首次完全缓解时单单位脐带血移植与单倍体移植后环磷酰胺的比较。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-05 DOI: 10.1038/s41409-025-02729-5
Masamitsu Yanada, Satoshi Yamasaki, Shohei Mizuno, Junichi Sugita, Takahiro Fujino, Yukiko Misaki, Masatsugu Tanaka, Naoyuki Uchida, Makoto Onizuka, Noriko Doki, Shuichi Ota, Masashi Sawa, Toshiro Kawakita, Yuta Hasegawa, Hirohisa Nakamae, Kazuya Ishiwata, Nobuhiro Hiramoto, Fumihiko Ishimaru, Junya Kanda, Marie Ohbiki, Yoshiko Atsuta, Hideki Nakasone, Takaaki Konuma
Choosing an optimal alternative donor is an important clinical concern in allogeneic hematopoietic cell transplantation (HCT). In Japan, single-unit umbilical cord blood transplantation (UCBT) has been widely used in the last two decades, whereas HCT from HLA-haploidentical related donors (haplo-HCT) has been increasingly used following the advent of posttransplant cyclophosphamide (PTCY) for graft-versus-host disease (GVHD) prophylaxis. This registry-based study aimed to compare outcomes between single-unit UCBT (n = 848) and PTCY-based haplo-HCT (n = 241) performed during first complete remission in patients with acute myeloid leukemia. UCBT was associated with a lower likelihood of engraftment (P < 0.001), a higher risk of grade 2–4 and grade 3–4 acute GVHD (P = 0.003 each), and a lower risk of extensive chronic GVHD (P = 0.048). The UCBT and haplo-HCT groups did not significantly differ in 3-year probabilities of overall survival (68% versus 69%, P = 0.686), GVHD/relapse-free survival (55% versus 54%, P = 0.866), relapse (14% versus 16%, P = 0.463), and non-relapse mortality (21% versus 19%, P = 0.403), respectively, which were confirmed with multivariate analysis. These results indicate that both procedures should be considered viable options for patients lacking a matched donor.
在同种异体造血细胞移植(HCT)中,选择最佳的替代供体是一个重要的临床问题。在日本,单单位脐带血移植(UCBT)在过去二十年中被广泛使用,而随着移植后环磷酰胺(PTCY)用于预防移植物抗宿主病(GVHD)的出现,来自hla -单倍体相关供体的HCT (haploi -HCT)的使用越来越多。这项基于注册表的研究旨在比较急性髓系白血病患者首次完全缓解期间进行的单单位UCBT (n = 848)和基于ptcy的单倍- hct (n = 241)的结果。UCBT与较低的移植可能性相关(P
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引用次数: 0
Real-world experience of belumosudil and belumosudil/ruxolitinib combination in steroid-refractory chronic graft-versus-host disease 白莫硫地尔和白莫硫地尔/鲁索利替尼联合治疗类固醇难治性慢性移植物抗宿主病的实际经验。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-04 DOI: 10.1038/s41409-025-02721-z
Gagan Raju, Moneeza Walji, David Nemirovsky, Sean Devlin, Ilan Goldstein, Amandeep Singh, Pamela Susman, Natasia Rodriguez, Miguel-Angel Perales, Doris M. Ponce
Belumosudil is approved after failure of ≥2 lines of therapy in chronic graft-versus-host disease cGVHD. However, real-world data is limited. We conducted a retrospective analysis of 67 patients with steroid-refractory or dependent (SR/SD) cGVHD. At baseline, most patients had advanced multi-organ cGVHD. The 6- and 12-month overall response rate (ORR) was 61%. However, a subset of patients achieved deeper responses with ongoing therapy at 12 months. The 6-month failure-free survival (FFS) was 75% (95%CI: 65–86) whereas the 12-month FFS was 66% (95%CI: 55–78). A low incidence of drug-related grade ≥3 toxicities was observed. A cohort of patients with immune function analysis showed gradual improvement in immune subsets at 1-year post-treatment. The combined bel-rux cohort (n = 14) showed a 6- and 12-month ORR of 64% and 57%, respectively. Overall, belumosudil was associated with high treatment response and survival outcomes. Notably, deeper responses were observed with ongoing therapy, and it was overall well tolerated. In a cohort of patients, immune cell populations had preserved to improved values throughout treatment. Patients who received bel-rux demonstrated efficacy and safety as well. Overall, our real-world study indicates similar findings to the clinical trial and supports the use of belumosudil in cGVHD.
白莫硫地尔在治疗慢性移植物抗宿主病(cGVHD)失败≥2线后获批。然而,真实世界的数据是有限的。我们对67例类固醇难治性或依赖性(SR/SD) cGVHD患者进行回顾性分析。在基线时,大多数患者患有晚期多器官cGVHD。6个月和12个月的总有效率(ORR)为61%。然而,一部分患者在持续治疗12个月后获得了更深层次的反应。6个月无故障生存率(FFS)为75% (95%CI: 65-86),而12个月无故障生存率为66% (95%CI: 55-78)。观察到低发生率的药物相关≥3级毒性。免疫功能分析的患者队列显示,在治疗后1年,免疫亚群逐渐改善。联合bel-rux队列(n = 14)显示6个月和12个月的ORR分别为64%和57%。总体而言,白莫硫地尔与高治疗反应和生存结果相关。值得注意的是,持续治疗观察到更深的反应,总体耐受性良好。在一组患者中,免疫细胞群在整个治疗过程中一直保持到改善值。接受bel-rux治疗的患者也表现出了疗效和安全性。总的来说,我们的真实世界研究显示了与临床试验相似的结果,并支持在cGVHD中使用白莫硫地尔。
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引用次数: 0
The 51st Annual Meeting of the European Society for Blood and Marrow Transplantation: Van Bekkum Awards. 第51届欧洲血液和骨髓移植学会年会:Van Bekkum奖。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1038/s41409-025-02647-6
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引用次数: 0
The 51st Annual Meeting of the European Society for Blood and Marrow Transplantation: Organising committee. 第51届欧洲血液和骨髓移植学会年会:组委会。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1038/s41409-025-02645-8
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引用次数: 0
The 51st Annual Meeting of the European Society for Blood and Marrow Transplantation: Physicians - Poster Session (P001-P909). 第51届欧洲血液和骨髓移植学会年会:医师-海报会议(P001-P909)。
IF 5.2 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1038/s41409-025-02651-w
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引用次数: 0
期刊
Bone Marrow Transplantation
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