Clinical Lymphoma, Myeloma & Leukemia最新文献_第5页

Cytogenetic Features and Their Implications in Clinical Practice: A Real-World Analysis of a Large Cohort of Multiple Myeloma Patients 细胞遗传学特征及其在临床实践中的意义：对大量多发性骨髓瘤患者的现实世界分析。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.09.001

Sonia Morè , Massimo Offidani , Laura Corvatta , Silvia Aloisi , Tommaso Za , Francesca Fazio , Martina Gherardini , Velia Bongarzoni , Barbara Anaclerico , Luca Franceschini , Silvia Ferraro , Luca Cupelli , Carmine Liberatore , Francesca Fioritoni , Laura De Padua , Angela Rago , Silvia Gentili , Roberto Latagliata , Mariagrazia Garzia , Giancarlo Discepoli , Maria Teresa Petrucci

We retrospectively analysed cytogenetics by fluorescence in situ hybridization (FISH) in 1.026 patients with multiple myeloma treated in real-world with the aim to establish its role in daily clinical practice. Thirty-seven percent of patients had no FISH data available. Based on median PFS of each cytogenetic abnormality found, we identified 3 group of patients with a significantly different PFS and determined which patients can best benefit from anti-CD38 regimens and double transplant. These findings support cytogenetic testing in all patients at diagnosis.

Background

Cytogenetics by fluorescence in situ hybridization (FISH) plays an increasing prognostic role in multiple myeloma (MM).

Methods

we analysed cytogenetics and its implications in 1.026 patients treated in real-world from 2019 to 2023. Low-risk (LR) patients had normal cytogenetic or del(13q); intermediate-risk (IR) had t(11;14), hyperdiploidy, gain(1q) or del(1p); high-risk (HR) group had del(17p)/TP53, amp1q21, t(4;14), t(14:16) or t(14;20). Co-existence of 2 high risk abnormalities was named double hit.

Results

FISH data were not evaluable in 383 patients (37%). Out of 643 evaluable patients, chromosome 1 alterations were observed in 119 (18.5%), high risk chromosome 14 translocations in 65 (10%), del(17p)/TP53 in 37 (6%) and double-hit in 7 patients (1%). Cytogenetic was normal or hyperdiploid in 252 (39%) and 59 (13%) patients, respectively. Median PFS of LR, IR and HR group were 57.5, 43.2 and 30.5 months, respectively (P < .001). Although anti-CD38 regimens resulted in significantly longer PFS in both TE and NTE pts, in the former significant benefit was documented in the LR and IR group but not in HR group while in the latter only in the LR group. Tandem transplantation did not improve PFS both overall and in the 3 risk groups. Multivariate Cox regression analysis selected ECOG-PS ≥2, R-ISS II-III and our cytogenetic score HR as factors affecting PFS.

Conclusions

in real world, more than one third of MM patients do not have baseline FISH data. Nevertheless, cytogenetics and ECOG PS can be used for prognostic staging and for tailoring therapy.

我们回顾性分析了1.026例在现实世界中接受治疗的多发性骨髓瘤患者的荧光原位杂交（FISH）细胞遗传学，目的是确定其在日常临床实践中的作用。37%的患者没有可用的FISH数据。根据所发现的每个细胞遗传学异常的中位PFS，我们确定了3组PFS显著不同的患者，并确定哪组患者最能从抗cd38方案和双重移植中获益。这些发现支持在诊断时对所有患者进行细胞遗传学检测。背景：荧光原位杂交细胞遗传学（FISH）在多发性骨髓瘤（MM）的预后中起着越来越重要的作用。方法：我们分析了2019年至2023年在现实世界接受治疗的1.026例患者的细胞遗传学及其意义。低危（LR）患者细胞遗传学正常或del(13q)；中危（IR）患者有t（11;14）、高二倍体、增益（1q）或缺失（1p）；高危（HR）组有del(17p)/TP53、amp1q21、t（4;14）、t（14; 16）、t（14;20）。两种高危异常同时存在称为双重打击。结果：383例（37%）患者的FISH数据无法评估。在643例可评估的患者中，1号染色体改变119例（18.5%），14号染色体易位65例（10%），del(17p)/TP53 37例（6%），双重命中7例（1%）。252例（39%）和59例（13%）患者细胞遗传学为正常或超二倍体。LR组、IR组和HR组的中位PFS分别为57.5、43.2和30.5个月（P < 0.001）。尽管抗cd38方案显著延长了TE和NTE患者的PFS，但前者在LR组和IR组中有显著的获益，而在HR组中没有，而后者仅在LR组中有显著获益。串联移植在总体和3个危险组中均未改善PFS。多因素Cox回归分析选择ECOG-PS≥2、R-ISS II-III和我们的细胞遗传学评分HR作为影响PFS的因素。结论：在现实世界中，超过三分之一的MM患者没有基线FISH数据。然而，细胞遗传学和ECOG PS可用于预后分期和定制治疗。

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引用次数: 0

Tyrosine Kinase Inhibitors During Frontline Therapy in Adults With Acute Lymphoblastic Leukemia With ABL-class Rearrangements 酪氨酸激酶抑制剂在成人急性淋巴细胞白血病abl级重排的一线治疗中的应用。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.09.010

Julie Braish MD , Hagop Kantarjian MD , Nicholas J. Short MD , Guilin Tang MD , Naval Daver MD , Tapan Kadia MD , Jayastu Senapati MD , Wei-Ying Jen MD , Koichi Takahashi MD, PhD , Rebecca Garris BA, MSW , Guillermo Garcia-Manero MD , Farhad Ravandi MD , Elias Jabbour MD , Nitin Jain MD

引用次数: 0

Transdifferentiation From Multiple Myeloma to Myeloid Sarcoma in the Setting of CAR T-Cell Therapy CAR - t细胞治疗下多发性骨髓瘤向髓系肉瘤的转分化。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.09.013

Diana Cirstea , Kathleen Gallagher , Maxx King , Paola Dal Cin , Mark J Sloan , Marcela V Maus , Noopur Raje , Matthew Frigault , Valentina Nardi

•
The introduction of anti-BCMA CAR T-cell therapies has improved the prognosis for patients with multiple myeloma. However, long-term remission remains elusive for many patients due to various resistance mechanisms. Here we discuss a unique case of a 56-year-old woman whose multiple myeloma transdifferentiated into myeloid sarcoma shortly after receiving BCMA-targeted CAR T-cell therapy (ciltacabtagene autoleucel).
•
Our report presents the first documented case of multiple myeloma transdifferentiating into myeloid sarcoma following BCMA-targeted CAR T-cell therapy, specifically with ciltacabtagene autoleucel, making a novel observation in the field of oncology.
•
Myeloid sarcoma cells retaining the same genetic abnormalities as the original myeloma cells, including the IgH/FGFR3 translocation, suggest a clonal relationship, highlighting potential lineage plasticity in myeloma and its role in therapy resistance.
•
Clinicians should be vigilant for signs of lineage switch in patients undergoing CAR T-cell therapy, particularly in those with high-risk genetic features.
•
In cases of suspected transdifferentiation, a multidisciplinary approach involving hematology, oncology, and pathology is essential for diagnosis and management.
•
This case underscores the need for further research into the mechanisms of lineage plasticity in multiple myeloma and the potential role of CAR T-cell therapy in driving such changes.

•抗bcma CAR - t细胞疗法的引入改善了多发性骨髓瘤患者的预后。然而，由于各种耐药机制，许多患者的长期缓解仍然难以捉摸。在这里，我们讨论一个56岁的女性，她的多发性骨髓瘤在接受bcma靶向CAR -t细胞治疗后不久就转分化为髓系肉瘤。•我们的报告提出了第一个记录的多发性骨髓瘤在bcma靶向CAR -t细胞治疗后转分化为髓系肉瘤的病例，特别是西他卡他烯自体甲醇，在肿瘤学领域进行了新的观察。•髓系肉瘤细胞保留与原始骨髓瘤细胞相同的遗传异常，包括IgH/FGFR3易位，表明存在克隆关系，突出了骨髓瘤中潜在的谱系可塑性及其在治疗耐药性中的作用。•临床医生应警惕接受CAR - t细胞治疗的患者谱系转换的迹象，特别是那些具有高风险遗传特征的患者。•在疑似转分化的病例中，包括血液学、肿瘤学和病理学在内的多学科方法对诊断和治疗至关重要。•该病例强调需要进一步研究多发性骨髓瘤谱系可塑性的机制以及CAR - t细胞治疗在推动这种变化中的潜在作用。

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引用次数: 0

Real-World Comparison of Treatment Outcomes for Multiple Myeloma in Elderly Transplant Ineligible Patients Receiving First-Line VRd, Rd and VCd: Results From the Australian and New Zealand Myeloma and Related Diseases Registry 接受一线VRd、Rd和VCd的老年移植不合格患者多发性骨髓瘤治疗结果的现实世界比较：来自澳大利亚和新西兰骨髓瘤及相关疾病登记处的结果

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.08.004

Sarah Zhao , Cameron Wellard , Elizabeth M Moore , Andrew Spencer , Hang Quach , P. Joy Ho , Emma-Jane McDonald , Peter Mollee , Simon J Harrison , Bradley Augustson , Erica M Wood , Zoe K McQuilten , Rajeev Rajagopal

Background

Real-world data on treatment outcomes for elderly transplant-ineligible patients with newly diagnosed multiple myeloma are limited. The difference in treatment subsidization in Australia compared with New Zealand enables comparison of bortezomib-cyclophosphamide-dexamethasone (VCd), lenalidomide-bortezomib-dexamethasone (VRd) with Rd maintenance, and continuous Rd.

Methods

Using data from the ANZ Myeloma and Related Diseases Registry, we evaluated 1092 patients over 70 years of age between February 2013 and February 2024. Those who received Rd, VRd or VCd induction, and did not undergo an autologous stem cell transplant were included.

Results

Overall response rates were 85.6%, 73.7%, and 91.5% for VCd, Rd, and VRd, respectively (P < 0.001). At a median follow-up of 37 months, VRd showed the longest median progression-free survival (PFS) of 27.5 months, compared to 23.7 months for Rd and 20.5 months for VCd (P = 0.01). After adjustment, PFS for VRd and Rd remained superior compared to VCd. Rd patients had the longest median time to next treatment (35.1 months), compared to 28.7 months for VRd and 20.1 months for VCd. Overall survival (OS) was superior with VRd (P = 0.039), with 3-year survival rates of 80% for VRd, 67% for Rd, and 67% for VCd. However, multivariate analysis did not show a significant difference in OS.

Conclusion

Our study confirms VRd demonstrates superior PFS compared to Rd and VCd, but was underpowered to detect a significant difference in OS. More prospective real-world studies are needed to establish the optimal choice of induction therapy balancing efficacy and toxicity for this cohort in resource limited settings.

背景：新诊断的老年不适合移植的多发性骨髓瘤患者的治疗结果的真实数据是有限的。与新西兰相比，澳大利亚在治疗补贴方面的差异使得硼替佐米-环磷酰胺-地塞米松（VCd）、来那度胺-硼替佐米-地塞米松（VRd）与Rd维持和持续Rd进行比较。方法：使用来自ANZ骨髓瘤和相关疾病登记处的数据，我们评估了2013年2月至2024年2月期间1092名70岁以上的患者。接受Rd、VRd或VCd诱导且未接受自体干细胞移植的患者也包括在内。结果：VCd、Rd和VRd的总有效率分别为85.6%、73.7%和91.5% （P < 0.001）。在中位随访37个月时，VRd显示最长的中位无进展生存期（PFS）为27.5个月，而Rd为23.7个月，VCd为20.5个月（P = 0.01）。调整后，VRd和Rd的PFS仍优于VCd。Rd患者到下一次治疗的中位时间最长（35.1个月），而VRd患者为28.7个月，VCd患者为20.1个月。VRd组的总生存率（OS）优于VRd组（P = 0.039）， VRd组的3年生存率为80%，Rd组为67%，VCd组为67%。然而，多变量分析未显示OS有显著差异。结论：我们的研究证实VRd与Rd和VCd相比具有更好的PFS，但不足以检测OS的显着差异。在资源有限的情况下，需要更多的前瞻性现实世界研究来确定诱导治疗平衡疗效和毒性的最佳选择。

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引用次数: 0

Intravascular Complications of Central Venous Catheterization in Chronic Hematological Diseases: Low Risk with Peripherally Inserted Catheters in a Single-Center Retrospective Large Study 慢性血液病中心静脉置管的血管内并发症：在一项单中心回顾性大型研究中，外周置管的低风险

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.09.011

Claudia Giordano, Marco Picardi, Annamaria Vincenzi, Alessia Scarpa, Fabrizio Pane

Introduction

To evaluate the impact of long-term peripherally inserted central catheters (PICC) on symptomatic venous thrombosis (s-VT), we reviewed clinical charts of patients suffering from chronic hematological diseases with in-situ PICC placement for at least 90 days after catheterization at the Hematology Unit of the Federico II University Medical School of Naples (Italy). The period of observation was between January 2014 and December 2023.

Methods

A total of 1150 PICCs were inserted into 1150 patients at the hematologic diagnosis. Underlying chronic diseases were the following: non-Hodgkin lymphoma (n = 760, 66.1%), Hodgkin lymphoma (n = 200, 17.4%), multiple myeloma (n = 100, 8.7%), chronic lymphocytic leukemia (n = 50, 4.3%), severe aplastic anemia (n = 15, 1.3%), sickle cell disease (n = 10, 0.9%), thalassemia (n = 10, 0.9%), and hemophilia (n = 5, 0.4%). PICCs were successfully inserted in all cases. The median duration of in-situ PICC placement was 300 days (range, 120-400 days).

Results

A s-VT occurred in 30 cases (2.6%), with a rate of 0.13 (95% CI, 0.04-0.30) per 1000 implantation days. The median period time between PICC insertion and thrombotic episode was 30 days (range, 4-95 days). No serious complication was associated with these events. Hodgkin lymphoma (compared to the other diseases) resulted in a numerically higher incidence of PICC-related s-VT.

Conclusion

PICCs represent a useful and safe frontline central vascular approach for patients with chronic hematologic diseases, with a thrombotic risk profile comparable to that reported with centrally inserted totally implantable venous access devices (TIVADs).

简介：为了评估长期外周插入中心导管（PICC）对症状性静脉血栓形成（s-VT）的影响，我们回顾了那不勒斯（意大利）费德里克二世大学医学院血液科原位放置PICC的慢性血液病患者在置管后至少90天的临床图表。观察期为2014年1月至2023年12月。方法：对1150例血液学诊断患者共置入1150个picc。潜在的慢性疾病如下：非霍奇金淋巴瘤（n = 760, 66.1%）、霍奇金淋巴瘤（n = 200, 17.4%）、多发性骨髓瘤（n = 100, 8.7%）、慢性淋巴细胞白血病（n = 50, 4.3%）、严重再生障碍性贫血（n = 15, 1.3%）、镰状细胞病（n = 10, 0.9%）、地中海贫血（n = 10, 0.9%）和血友病（n = 5, 0.4%）。所有病例均成功插入picc。PICC原位放置的中位持续时间为300天（范围为120-400天）。结果：30例（2.6%）发生s-VT，发生率为0.13 (95% CI, 0.04 ~ 0.30) / 1000天。PICC置入至血栓发作的中位时间为30天（范围4-95天）。这些事件均无严重并发症。霍奇金淋巴瘤（与其他疾病相比）导致picc相关s-VT的发生率更高。结论：PICCs为慢性血液病患者提供了一种有效且安全的一线中心血管途径，其血栓形成风险与中心插入式全植入式静脉通路装置（TIVADs）相当。

{"title":"Intravascular Complications of Central Venous Catheterization in Chronic Hematological Diseases: Low Risk with Peripherally Inserted Catheters in a Single-Center Retrospective Large Study","authors":"Claudia Giordano, Marco Picardi, Annamaria Vincenzi, Alessia Scarpa, Fabrizio Pane","doi":"10.1016/j.clml.2025.09.011","DOIUrl":"10.1016/j.clml.2025.09.011","url":null,"abstract":"<div><h3>Introduction</h3><div>To evaluate the impact of long-term peripherally inserted central catheters (PICC) on symptomatic venous thrombosis (s-VT), we reviewed clinical charts of patients suffering from chronic hematological diseases with in-situ PICC placement for at least 90 days after catheterization at the Hematology Unit of the Federico II University Medical School of Naples (Italy). The period of observation was between January 2014 and December 2023.</div></div><div><h3>Methods</h3><div>A total of 1150 PICCs were inserted into 1150 patients at the hematologic diagnosis. Underlying chronic diseases were the following: non-Hodgkin lymphoma (n = 760, 66.1%), Hodgkin lymphoma (n = 200, 17.4%), multiple myeloma (n = 100, 8.7%), chronic lymphocytic leukemia (n = 50, 4.3%), severe aplastic anemia (n = 15, 1.3%), sickle cell disease (n = 10, 0.9%), thalassemia (n = 10, 0.9%), and hemophilia (n = 5, 0.4%). PICCs were successfully inserted in all cases. The median duration of in-situ PICC placement was 300 days (range, 120-400 days).</div></div><div><h3>Results</h3><div>A s-VT occurred in 30 cases (2.6%), with a rate of 0.13 (95% CI, 0.04-0.30) per 1000 implantation days. The median period time between PICC insertion and thrombotic episode was 30 days (range, 4-95 days). No serious complication was associated with these events. Hodgkin lymphoma (compared to the other diseases) resulted in a numerically higher incidence of PICC-related s-VT.</div></div><div><h3>Conclusion</h3><div>PICCs represent a useful and safe frontline central vascular approach for patients with chronic hematologic diseases, with a thrombotic risk profile comparable to that reported with centrally inserted totally implantable venous access devices (TIVADs).</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages e114-e119"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145318216","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

SOHO State of the Art Updates and Next Questions | Current and Emerging Novel Treatments for Marginal Zone Lymphoma SOHO最新进展和下一个问题|边缘区淋巴瘤当前和新兴的新治疗方法。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.05.021

Izidore S. Lossos

Marginal zone lymphoma (MZL) is a heterogeneous disease representing 7% to 10% of non-Hodgkin lymphomas (NHL). There are no clear guidelines defining the need for treatment initiation and no standardized therapeutic approach for patients with MZL. Previously, many treatments were extrapolated from clinical trials that mainly enrolled patients with follicular lymphoma and did not have statistical power to show treatment efficacy specifically in MZL patients. Currently, the field is moving toward conducting specific trials in MZL patients with specific inclusion criteria for each MZL subtype, assessing response distinctively in FDG avid and nonavid MZL tumors and using MZL-relevant primary endpoints. Herein I briefly describe these changes, summarize previous therapeutic studies in MZL and present preliminary publicly available data on novel emerging treatments, including bispecific antibodies, antibody drug conjugates and novel targeted therapies.

边缘带淋巴瘤（MZL）是一种异质性疾病，占非霍奇金淋巴瘤（NHL）的7%至10%。目前还没有明确的指南来定义MZL患者开始治疗的必要性，也没有标准化的治疗方法。以前，许多治疗方法都是从临床试验中推断出来的，这些临床试验主要招募滤泡性淋巴瘤患者，并且没有统计学上的力量来显示治疗效果，特别是在MZL患者中。目前，该领域正朝着在MZL患者中开展特定试验的方向发展，针对每种MZL亚型制定特定的纳入标准，评估FDG依赖型和非依赖型MZL肿瘤的独特反应，并使用MZL相关的主要终点。在此，我简要地描述了这些变化，总结了MZL先前的治疗研究，并介绍了新的新兴治疗方法的初步公开数据，包括双特异性抗体，抗体药物偶联物和新的靶向治疗。

引用次数: 0

Curability Potential of Low-Grade Follicular Lymphoma 低级别滤泡性淋巴瘤的治愈潜力。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.09.009

F. Cabanillas , J.G. Conde , T. Gutierrez , A. Torres , J. Abreu , F.B. Hagemeister

Background

Classic follicular lymphoma is generally considered incurable. Standard management includes a “watch and wait” strategy for patients with low tumor burden, while those with high tumor burden typically receive chemotherapy.

Objectives

We aimed to evaluate the potential for curability in a cohort of patients managed with systemic therapy, without the use of a watch-and-wait approach.

Methods

This study is a retrospective, real-world analysis of patients treated in routine clinical practice. We analyzed 183 previously untreated patients with low-grade follicular lymphoma (LGFL) treated at Auxilio Mutuo Cancer Center in Puerto Rico between June 2002 and July 2023. All patients began therapy shortly after diagnosis, without observation. We stratified treatment based on the presence or absence of “clinically discordant indolent histology” (CDIH), defined by aggressive clinical features such as B symptoms, high lactic dehydrogenase (LDH), SUV >14 on PET, Ki-67 > 30%, or involvement of atypical sites including lung, pleura, soft-tissue, CNS and bones. Patients with CDIH typically received R-CHOP x6 followed by FND (fludarabine, Novantrone [mitoxantrone] and dexamethasone). Non-CDIH patients mostly received FND-R. All patients received 2 years of rituximab maintenance. The primary endpoint was failure-free survival (FFS).

Results

Among 183 patients, the median follow-up for censored cases was 8.5 years (range: 6 to 261 months). At 10 years, the FFS was 80%; at 15 years, 75%. Of 61 patients followed for more than 10 years, 57 (93%) remain relapse-free, suggesting a potential for cure in those who remain failure-free beyond a decade.

Conclusion

Our findings support the hypothesis that early initiation of chemotherapy may potentially result in cure for patients with classic follicular NHL. These results warrant confirmation in future prospective studies.

背景：典型滤泡性淋巴瘤通常被认为是无法治愈的。标准管理包括对肿瘤负担低的患者采取“观察和等待”策略，而肿瘤负担高的患者通常接受化疗。目的：我们旨在评估在不使用观察和等待方法的情况下，接受全身治疗的患者队列的治愈潜力。方法：本研究对在常规临床实践中接受治疗的患者进行回顾性分析。我们分析了2002年6月至2023年7月期间在波多黎各Auxilio Mutuo癌症中心治疗的183例未经治疗的低级别滤泡性淋巴瘤（LGFL）患者。所有患者在诊断后不久开始治疗，未进行观察。我们根据是否存在“临床不一致的惰性组织学”（CDIH）对治疗进行分层，CDIH的定义是侵略性的临床特征，如B症状、高乳酸脱氢酶（LDH）、PET上的SUV >4、Ki-67 > 30%，或累及非典型部位，包括肺、胸膜、软组织、中枢神经系统和骨骼。CDIH患者通常接受R-CHOP x6，随后接受FND（氟达拉滨、诺vantrone[米托蒽醌]和地塞米松）。非cdih患者大多接受FND-R治疗。所有患者均接受2年美罗华维持治疗。主要终点为无故障生存期（FFS）。结果：在183例患者中，审查病例的中位随访时间为8.5年（范围：6至261个月）。十年后，FFS占80%；15年，75%。在61例随访超过10年的患者中，57例（93%）仍然没有复发，这表明那些在10年以上没有复发的患者有治愈的潜力。结论：我们的研究结果支持了早期化疗可能导致经典滤泡性NHL患者治愈的假设。这些结果值得在未来的前瞻性研究中得到证实。

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引用次数: 0

SOHO State of the Art Updates and Next Questions | The Optimal Management of Waldenström Macroglobulinemia SOHO国家的艺术更新和下一个问题| Waldenström巨球蛋白血症的最佳管理。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.06.017

Alberto Guijosa , Alicia de las Heras , Shayna Sarosiek , Jorge J. Castillo

Waldenström macroglobulinemia (WM) is a rare IgM-secreting lymphoplasmacytic lymphoma with recurrent somatic mutations in MYD88 and CXCR4 observed in the malignant cells of >90% and 30% to 40% of the patients. Given its rarity, WM poses specific diagnostic and management challenges. The diagnosis of WM is clinicopathological and no pathognomonic findings exist. The combination of a monoclonal IgM paraproteinemia, lymphoplasmacytic lymphoma in the bone marrow or other organs, and the MYD88 L265P mutation makes a diagnosis of WM with a high specificity. Approximately, a third of the patients will be asymptomatic at diagnosis and the best approach is to observe, as these patients have similar survival rates than age, sex and year of diagnosis-matched individuals of the general population. Eighty percent of patients diagnosed with asymptomatic WM will need treatment within 10 years. Treatment is indicated in symptomatic patients in whom the symptoms affect the patients’ activities and are likely to be caused by the disease process. Multiple standard treatment options are safe and effective in symptomatic patients, including rituximab in combination with alkylating agents or proteasome inhibitors, covalent BTK inhibitors, and BCL2 antagonists. Noncovalent BTK inhibitors have emerged as a novel treatment option. Second-generation BCL2 antagonists, BTK degraders, antibody-drug conjugates and bispecific T-cell engagers are being evaluated in clinical trials. Multinational collaborative consortia to accelerate clinical trial design and execution in WM have emerged in Europe and the United States.

Waldenström巨球蛋白血症（macroglobulinemia， WM）是一种罕见的igm分泌性淋巴浆细胞性淋巴瘤，MYD88和CXCR4的体细胞突变在bbb90 %和30% ~ 40%的患者的恶性细胞中反复出现。鉴于其罕见性，WM提出了具体的诊断和管理挑战。WM的诊断是临床病理的，没有病理表现。结合单克隆IgM副蛋白血症、骨髓或其他器官淋巴浆细胞性淋巴瘤和MYD88 L265P突变，诊断WM具有高特异性。大约三分之一的患者在诊断时无症状，最好的方法是观察，因为这些患者的生存率与一般人群中年龄、性别和年份的诊断相匹配的个体相似。80%被诊断为无症状WM的患者将在10年内需要治疗。治疗适用于有症状的患者，这些患者的症状影响患者的活动，并且很可能是由疾病过程引起的。对于有症状的患者，多种标准治疗方案是安全有效的，包括利妥昔单抗联合烷基化剂或蛋白酶体抑制剂、共价BTK抑制剂和BCL2拮抗剂。非共价BTK抑制剂已成为一种新的治疗选择。第二代BCL2拮抗剂、BTK降解剂、抗体-药物偶联物和双特异性t细胞接合物正在临床试验中进行评估。加速WM临床试验设计和执行的跨国合作联盟已经在欧洲和美国出现。

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引用次数: 0

When ICAN(S) Becomes ICAN’T: Clinician and Staff Perspectives on In-Hospital Neurotoxicity Grading 当ICAN(S)变成ICAN’t：临床医生和工作人员对院内神经毒性分级的看法。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.08.021

Grace M. Ferri , Allison Frank , Daniel Li , Pria Anand , Maya Abdallah , Vanessa Avalone , J. Mark Sloan , Vaishali Sanchorawala , Adam Lerner , Raphael E. Szalat , Fabio Petrocca , Camille V. Edwards , Britney N. Bell

Purpose

Guidelines from the American Society for Transplantation and Cellular Therapy (ASTCT) propose use of the Immune Effector Cell-Associated Encephalopathy (ICE) score as a means by which to grade Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS). However, ICE scoring may not appropriately capture ICANS among patients with limited English proficiency or diverse educational or cultural backgrounds. With the development of protocols for early ICANS treatment and the advent of CAR-T repurposing for solid tumors, creation of an accessible neurotoxicity grading framework (and an accurate clinical correlate) for all patients is paramount.

Methods

Using a quantitative and qualitative descriptive study design, we surveyed staff members at a United States safety-net hospital experienced in grading the ICE score. We then performed an iterative thematic analysis of data embedded within free-text responses and used a modified version of the theoretical framework of acceptability (TFA) to guide evaluation of the anticipated intervention.

Results

Of the 36 survey respondents, most (27/36, 75%) agreed that lack of language concordance could lead to inaccurate ICE scores. While translation services were thought to be used appropriately (33/36, 92%), logistical barriers including availability of interpreter services (in-person, phone, tablet) were thought to impact quality of care for non-native English-speaking patients. Additional barriers to accurate ICE scoring included patient literacy, numeracy (eg, cultural differences in measuring time), education level, or disability status (eg, hearing or vision loss, memory or cognitive impairment).

Conclusion

This needs assessment demonstrated stakeholder perspectives on the standard ICE score; associated challenges among patients with limited English proficiency and illiteracy; and the utility of an alternative language-concordant and culturally humble grading system for neurotoxicity among non-native English speakers.

目的：美国移植和细胞治疗学会（ASTCT）的指南建议使用免疫效应细胞相关脑病（ICE）评分作为对免疫效应细胞相关神经毒性综合征（ICANS）进行分级的一种手段。然而，ICE评分可能不能恰当地反映英语水平有限或教育或文化背景不同的患者的ICANS。随着早期ICANS治疗方案的发展和CAR-T重新用于实体瘤的出现，为所有患者创建一个可访问的神经毒性分级框架（以及准确的临床相关性）是至关重要的。方法：采用定量和定性描述性研究设计，我们调查了美国一家安全网医院的工作人员，他们对ICE评分有经验。然后，我们对自由文本回复中嵌入的数据进行了迭代主题分析，并使用可接受性理论框架（TFA）的修改版本来指导预期干预的评估。结果：在36个调查对象中，大多数（27/ 36,75%）认为缺乏语言一致性可能导致ICE评分不准确。虽然翻译服务被认为得到了适当的使用（33/ 36,92%），但后勤障碍，包括口译服务（面对面、电话、平板电脑）的可用性，被认为会影响非英语母语患者的护理质量。影响准确ICE评分的其他障碍包括患者读写能力、计算能力（如测量时间的文化差异）、教育水平或残疾状况（如听力或视力丧失、记忆或认知障碍）。结论：该需求评估展示了利益相关者对标准ICE评分的看法；英语水平有限和文盲患者的相关挑战；以及在非英语母语者中使用另一种语言一致和文化谦逊的神经毒性评分系统的效用。

{"title":"When ICAN(S) Becomes ICAN’T: Clinician and Staff Perspectives on In-Hospital Neurotoxicity Grading","authors":"Grace M. Ferri , Allison Frank , Daniel Li , Pria Anand , Maya Abdallah , Vanessa Avalone , J. Mark Sloan , Vaishali Sanchorawala , Adam Lerner , Raphael E. Szalat , Fabio Petrocca , Camille V. Edwards , Britney N. Bell","doi":"10.1016/j.clml.2025.08.021","DOIUrl":"10.1016/j.clml.2025.08.021","url":null,"abstract":"<div><h3>Purpose</h3><div>Guidelines from the American Society for Transplantation and Cellular Therapy (ASTCT) propose use of the Immune Effector Cell-Associated Encephalopathy (ICE) score as a means by which to grade Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS). However, ICE scoring may not appropriately capture ICANS among patients with limited English proficiency or diverse educational or cultural backgrounds. With the development of protocols for early ICANS treatment and the advent of CAR-T repurposing for solid tumors, creation of an accessible neurotoxicity grading framework (and an accurate clinical correlate) for all patients is paramount.</div></div><div><h3>Methods</h3><div>Using a quantitative and qualitative descriptive study design, we surveyed staff members at a United States safety-net hospital experienced in grading the ICE score. We then performed an iterative thematic analysis of data embedded within free-text responses and used a modified version of the theoretical framework of acceptability (TFA) to guide evaluation of the anticipated intervention.</div></div><div><h3>Results</h3><div>Of the 36 survey respondents, most (27/36, 75%) agreed that lack of language concordance could lead to inaccurate ICE scores. While translation services were thought to be used appropriately (33/36, 92%), logistical barriers including availability of interpreter services (in-person, phone, tablet) were thought to impact quality of care for non-native English-speaking patients. Additional barriers to accurate ICE scoring included patient literacy, numeracy (eg, cultural differences in measuring time), education level, or disability status (eg, hearing or vision loss, memory or cognitive impairment).</div></div><div><h3>Conclusion</h3><div>This needs assessment demonstrated stakeholder perspectives on the standard ICE score; associated challenges among patients with limited English proficiency and illiteracy; and the utility of an alternative language-concordant and culturally humble grading system for neurotoxicity among non-native English speakers.</div></div>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":"26 1","pages":"Pages e77-e82"},"PeriodicalIF":2.7,"publicationDate":"2026-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145181802","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Impact of Measurable Residual Disease Status on Outcomes After HLA-Matched Donor Allogeneic Hematopoietic Cell Transplantation in Acute Myeloid Leukemia 可测量的残留疾病状态对hla匹配供体异体造血细胞移植后急性髓系白血病预后的影响。

IF 2.7 4区医学 Q2 HEMATOLOGY

Clinical Lymphoma, Myeloma & Leukemia

Pub Date : 2026-01-01 DOI: 10.1016/j.clml.2025.08.011

Moazzam Shahzad , Muhammad Kashif Amin , Sohaib Irfan , Abhinav Vyas , Rania Ahsan , Sibgha Gull Chaudhary , Iqra Anwar , Matthew McGuirk , Raheel Iftikhar , Haitham Abdelhakim , Anurag K. Singh , Mehdi Hamadani , Joseph P. Mcguirk , Muhammad Umair Mushtaq

Background

Allogeneic hematopoietic stem cell transplantation (allo-HCT) is a key treatment for acute myeloid leukemia (AML). Measurable residual disease (MRD) predicts post-transplant outcomes. This study evaluates the impact of pretransplant MRD status on outcomes in AML patients undergoing allo-HCT.

Methods

We retrospectively analyzed AML patients who underwent allo-HCT from matched related or unrelated donors (2013–2018) using the CIBMTR P-5646 dataset. Patients were stratified by pretransplant MRD status. Outcomes included overall survival (OS), disease-free survival (DFS), relapse, nonrelapse mortality (NRM), acute graft-versus-host disease (aGVHD), GVHD-free relapse-free survival (GRFS), and engraftment. Hazard ratios (HR) with 95% confidence intervals (CI) were calculated using multivariate Cox regression, adjusted for significant univariate variables (P < .05).

Results

Of 2404 AML patients (354 MRD-positive, 2050 MRD-negative), MRD-positive patients had a lower Karnofsky performance status (≥90%: 46.1% vs. 55.1%, P = .004) and were more likely to undergo myeloablative conditioning (66.6% vs. 52.7%, P < .001). MRD positivity predicted worse OS (HR 1.91, 95% CI 1.62-2.23, P < .001), DFS (HR 2.05, 95% CI 1.77–2.36, P < .001), relapse (HR 2.25, 95% CI 1.91-2.64, P < .001), aGVHD grade II to IV (HR 1.24, 95% CI 1.03-1.50, P = .024), GRFS (HR 1.59, 95% CI 1.41-1.81, P < .001), and slower platelet engraftment (HR 0.71, 95% CI 0.63-0.81, P < .001). NRM (P = .387) and neutrophil engraftment (P = .159) were similar.

Conclusion

Pretransplant MRD status predicts post-allo-HCT outcomes, with MRD positivity associated with reduced overall and disease-free survival and increased relapse risk. Personalized MRD-directed strategies are needed to optimize outcomes in AML patients undergoing allogeneic transplantation.

背景：同种异体造血干细胞移植是急性髓系白血病（AML）的关键治疗手段。可测量的残留疾病（MRD）预测移植后的预后。本研究评估移植前MRD状态对接受同种异体hct治疗的AML患者预后的影响。方法：我们使用CIBMTR P-5646数据集回顾性分析了2013-2018年从匹配的相关或非相关供体接受同种异体hct治疗的AML患者。根据移植前MRD状态对患者进行分层。结果包括总生存期（OS）、无病生存期（DFS）、复发、非复发死亡率（NRM）、急性移植物抗宿主病（aGVHD）、无移植物抗宿主病无复发生存期（GRFS）和移植物。采用多变量Cox回归计算风险比（HR）和95%置信区间（CI），并对显著单变量进行校正（P < 0.05）。结果：2404例AML患者（354例mrd阳性，2050例mrd阴性）中，mrd阳性患者的Karnofsky表现状态较低（≥90%:46.1%对55.1%,P = 0.004），更有可能接受清髓调节（66.6%对52.7%,P < 0.001）。MRD阳性预测较差的OS （HR 1.91, 95% CI 1.62-2.23, P < 0.001）、DFS （HR 2.05, 95% CI 1.77-2.36, P < 0.001）、复发（HR 2.25, 95% CI 1.91-2.64, P < 0.001）、aGVHD II至IV级（HR 1.24, 95% CI 1.03-1.50, P = 0.024）、GRFS （HR 1.59, 95% CI 1.41-1.81, P < 0.001）和血小板植入较慢（HR 0.71, 95% CI 0.63-0.81, P < 0.001）。NRM （P = .387）和中性粒细胞移植（P = .159）相似。结论：移植前MRD状态预测同种异体移植后的预后，MRD阳性与总生存率和无病生存率降低以及复发风险增加相关。需要个性化的mrd导向策略来优化接受同种异体移植的AML患者的预后。

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引用次数: 0