Blood Reviews最新文献

英文中文

Navigating acute myeloid leukemia towards better outcomes: Treatment pathways and challenges for patients ineligible for intensive chemotherapy 引导急性髓系白血病走向更好的结局：不适合强化化疗的患者的治疗途径和挑战。

IF 5.7 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-08-01 DOI: 10.1016/j.blre.2025.101288

Raffaele Palmieri , Anna Candoni , Francesco Di Raimondo , Giuseppe Rossi , Massimo Breccia , Fabrizio Pane , Paola Volpicelli , Benedetta Neri , Paola Finsinger , Morena Caira , Felicetto Ferrara

Acute myeloid leukemia (AML) is an aggressive hematologic malignancy that affects primarily older individuals. Patients ineligible to receive intensive standard chemotherapy followed by consolidation with/without hematopoietic stem cell transplant have a suboptimal prognosis. In recent years, significant advances have been made in the AML field leading to the development of new anti-leukemic approaches, including lower-intensity therapies specifically developed for patients who are ineligible for intensive chemotherapy. As the available options for this hard-to-manage and historically undertreated patient category are increasing, selecting the best treatment for each patient is crucial and ever more challenging. Accordingly, accurate patient evaluation is required to guide this decision-making process. There is currently no consensus on how to evaluate patients' fitness status, and the available tools that were originally developed for this purpose might not be adequate in the setting of the new treatment options. In this review we describe current management of AML patients unfit for intensive chemotherapy, aiming to highlight current challenges and suggest possible strategies for an accurate therapeutic selection. For this purpose, we will first provide an overview of epidemiology and classification of AML, and then move to current anti-leukemic treatments for unfit patients and the tools used for evaluating patient eligibility for a specific treatment. Finally, we will suggest possible measures to improve the management of AML patients in the era of novel lower-intensity regimens.

急性髓性白血病（AML）是一种侵袭性血液系统恶性肿瘤，主要影响老年人。不适合接受强化标准化疗并合并/不合并造血干细胞移植的患者预后不佳。近年来，AML领域取得了重大进展，导致了新的抗白血病方法的发展，包括专门为不适合进行强化化疗的患者开发的低强度治疗。随着这类难以管理且历史上治疗不足的患者的可用选择越来越多，为每位患者选择最佳治疗方法至关重要，而且更具挑战性。因此，需要准确的患者评估来指导这一决策过程。目前对于如何评估患者的健康状况还没有达成共识，而最初为此目的开发的可用工具可能不适用于新的治疗方案。在这篇综述中，我们描述了目前不适合强化化疗的AML患者的管理，旨在强调当前的挑战，并提出准确治疗选择的可能策略。为此，我们将首先概述AML的流行病学和分类，然后转向当前针对不适合患者的抗白血病治疗以及用于评估患者是否适合特定治疗的工具。最后，我们将提出在新型低强度方案时代改善AML患者管理的可能措施。

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引用次数: 0

Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies 镰状细胞病新药物治疗临床结果的证据和差距：一项系统的文献综述，突出临床试验和现实世界研究的见解。

IF 5.7 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-08-01 DOI: 10.1016/j.blre.2025.101298

Mohamed Yassin , Caterina Minniti , Nirmish Shah , Salam Alkindi , Fateen Ata , Mohammed Qari , Abdullah Al Zayed , Jaffer Altooq , Mona Al Rasheed , Maria Domenica Capellini

This systematic review aims to summarise the clinical outcomes of l-glutamine, crizanlizumab, and voxelotor in the treatment of sickle cell disease (SCD) based on clinical trials and real-world data and to identify any gaps in the observations. The review identified 97 studies reporting data until 31 May 2024.

A pivotal phase III study of l-glutamine showed that patients treated with l-glutamine had a 25 % reduction in pain crises and 33 % fewer hospital days compared to placebo. l-glutamine was generally well tolerated with minimal side effects. Real-world studies of l-glutamine emphasize patient adherence and obstacles to medication accessibility and approval as key concerns. In the SUSTAIN study, a 5-mg/kg dose of crizanlizumab reduced the occurrence of vaso-occlusive crises (VOCs) and hospitalizations by 45 % and 41 %, respectively. Real-world studies of crizanlizumab showed a reduction in complicated VOC events. The high discontinuation rate and results of the STAND trial led to a significant decrease in the use of crizanlizumab. The HOPE trial demonstrated a 51 % improvement in hemoglobin response and a reduction in hemolytic markers in patients treated with voxelotor. While some real-world studies have reported a decrease in VOCs and hospitalizations, the results are inconsistent and not conclusive. Further studies are needed to assess the impact of these novel therapies on end-organ-specific complications of SCD.

本系统综述旨在总结基于临床试验和真实世界数据的l-谷氨酰胺、克里赞单抗和voxelotor治疗镰状细胞病（SCD）的临床结果，并确定观察结果中的任何空白。该综述确定了截至2024年5月31日报告数据的97项研究。一项关键的l-谷氨酰胺III期研究表明，与安慰剂相比，接受l-谷氨酰胺治疗的患者疼痛危机减少25%，住院天数减少33%。l-谷氨酰胺通常耐受性良好，副作用最小。l-谷氨酰胺的实际研究强调患者的依从性和药物可及性和批准的障碍是关键问题。在SUSTAIN研究中，5mg /kg剂量的crizanlizumab分别将血管闭塞危像（VOCs）和住院率降低了45%和41%。实际研究表明，crizanlizumab可减少复杂的VOC事件。STAND试验的高停药率和结果导致crizanlizumab的使用显著减少。HOPE试验表明，在接受voxelotor治疗的患者中，血红蛋白反应改善了51%，溶血标志物降低。虽然一些现实世界的研究报告了挥发性有机化合物和住院治疗的减少，但结果并不一致，也不是结论性的。需要进一步的研究来评估这些新疗法对SCD终末器官特异性并发症的影响。

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引用次数: 0

Suppression to removal, an emerging therapeutic approach for AL amyloidosis: A comprehensive review with early human data and pharmacokinetics of CAEL-101 antibody 抑制去除，AL淀粉样变性的一种新兴治疗方法：早期人类数据和CAEL-101抗体药代动力学的综合综述

IF 5.7 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-08-01 DOI: 10.1016/j.blre.2025.101299

Anam Ashfaque , Yara Shatnawi , Shahzad Raza , Diana Basali , Jack Khouri , Louis Williams , Sandra Mazzoni , Christy Samaras , Hamza Hassan , Utkarsh Acharya , Chakra Chaulagain , Faiz Anwer

Light chain (AL amyloidosis) is a rare disorder characterized by the deposition of misfolded light chains in various organs, causing progressive organ damage. Current therapeutic agents do not remove amyloid aggregates already present in the organs, which are the major determinants of morbidity and mortality. Therefore, drugs targeting amyloid fibrils are currently being investigated. This article provides a comprehensive review of a novel, fibril-directed antibody, CAEL-101 (Anselamimab), which removes fibrillary aggregates from the organs, restoring organ function. Overall, CAEL-101 has demonstrated a favorable toxicity profile and improved organ responses, with faster treatment response time than current therapies in phase I/II trials, and is expected to have promising outcomes in the ongoing phase III studies. Combining anti-plasma cell dyscrasia (suppression) and fibril-directed agents (removal) is a novel therapeutic approach, providing optimism for organ function recovery and enhanced quality of life and survival, especially in patients with severe diseases.

轻链（AL）淀粉样变性是一种罕见的疾病，其特征是在各种器官中沉积错误折叠的轻链，引起进行性器官损害。目前的治疗药物不能去除已经存在于器官中的淀粉样蛋白聚集体，这是发病率和死亡率的主要决定因素。因此，目前正在研究针对淀粉样蛋白原纤维的药物。这篇文章提供了一种新的，原纤维定向抗体CAEL-101 （Anselamimab）的全面回顾，它从器官中去除原纤维聚集体，恢复器官功能。总体而言，在I/II期试验中，CAEL-101已显示出良好的毒性特征和改善的器官反应，治疗反应时间比目前的治疗更快，并且预计在正在进行的III期研究中有很好的结果。结合抗浆细胞增生（抑制）和原纤维定向药物（去除）是一种新的治疗方法，为器官功能恢复和提高生活质量和生存提供了乐观的希望，特别是对于严重疾病的患者。

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引用次数: 0

Navigating the dynamic landscape of lower-risk MDS: Advances and emerging insights 导航低风险MDS的动态景观：进展和新兴见解。

IF 5.7 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-08-01 DOI: 10.1016/j.blre.2025.101301

Alain Mina , Yazan Madanat , Yasmin Abaza , Amer M. Zeidan

Myelodysplastic syndromes/neoplasms (MDS) are a group of clonal myeloid malignancies characterized by ineffective hematopoiesis, cytopenias, and an increased risk of transformation to acute myeloid leukemia (AML). In lower-risk (LR) MDS, as defined by the revised and molecular international prognostic scoring systems (IPSS-R and IPSS-M), anemia is often the predominant clinical manifestation. Treatment strategies have traditionally focused on supportive care, including transfusion support and erythropoiesis stimulating agents (ESAs). While allogeneic hematopoietic stem cell transplantation remains the only potentially curative option for select patients, LR-MDS remain otherwise incurable with current therapies. With the exception of lenalidomide which was approved in 2005 in USA, therapeutic advancements in LR-MDS have stalled for almost 15 years. Progress has been limited by the disease's inherent complexity, indolent nature, and significant heterogeneity, as well as challenges in clinical trial design and execution. Recent advances in gene sequencing and molecular analyses have significantly increased our understanding of disease biology. These insights, coupled with collaborative efforts across the academic community, have led to meaningful shifts in classification, prognostication, and response assessment paradigms in LR-MDS. This evolution has led to a number of approvals, including luspatercept approved in 2020, and imetelstat, which was approved in 2024 in USA. As the therapeutic landscape of LR-MDS continues to evolve, there is growing optimism that these recent milestones will pave the way for further advancements and improved patient outcomes. Next set of studies should focus on the optimal sequencing and combinations of existing agents, as well as moving forward novel effective agents.

骨髓增生异常综合征/肿瘤（MDS）是一组克隆性髓系恶性肿瘤，其特征是造血功能低下、细胞减少和转化为急性髓系白血病（AML）的风险增加。根据修订后的分子国际预后评分系统（IPSS-R和IPSS-M）的定义，在低风险（LR） MDS中，贫血通常是主要的临床表现。治疗策略传统上侧重于支持性护理，包括输血支持和促红细胞生成剂（ESAs）。虽然同种异体造血干细胞移植仍然是特定患者的唯一潜在治疗选择，但目前的治疗方法仍然无法治愈LR-MDS。除了来那度胺（lenalidomide）于2005年在美国获得批准外，LR-MDS的治疗进展已经停滞了近15年。由于该病固有的复杂性、惰性和显著的异质性，以及临床试验设计和执行方面的挑战，进展受到限制。基因测序和分子分析的最新进展大大增加了我们对疾病生物学的了解。这些见解，加上整个学术界的合作努力，导致了LR-MDS分类、预测和反应评估范式的有意义的转变。这一演变导致了许多批准，包括2020年批准的luspatercept和2024年在美国批准的imetelstat。随着LR-MDS的治疗前景不断发展，人们越来越乐观地认为，这些最近的里程碑将为进一步的进展和改善患者的预后铺平道路。下一步的研究应侧重于现有药物的最佳排序和组合，以及开发新的有效药物。

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引用次数: 0

Practical guidance on the prevention and management of infection in multiple myeloma patients: A case-based approach 预防和管理多发性骨髓瘤患者感染的实用指南：基于病例的方法。

IF 6.9 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-04-11 DOI: 10.1016/j.blre.2025.101287

A. Kannan , K. Jeffrey , S. Misbah , K. Ramasamy

The risk of infection in multiple myeloma patients is significant, due to immune dysfunction secondary to myeloma, immunosenescence and age-related comorbidities, given the elderly myeloma patient demographic. Newer treatments, despite providing unprecedented improvements in disease-control, have further elevated infection risk. This risk is so substantial that we are approaching a period where a subset of older myeloma patients may be more likely to die secondary to infectious complications imposed by redirected T-cell therapy rather than from myeloma. As a result, it is essential to provide myeloma patients with the appropriate prophylaxis and monitoring against infection. In this review, we discuss disease-related, patient-related and treatment-related reasons for the increased infection risk in myeloma patients, and how to both prevent and manage this risk through creating a dynamic, infection prevention plan that is personalised to the individual patient.

考虑到老年骨髓瘤患者的人口统计，多发性骨髓瘤患者感染的风险是显著的，因为骨髓瘤继发的免疫功能障碍、免疫衰老和年龄相关的合并症。新的治疗方法尽管在疾病控制方面提供了前所未有的改善，但却进一步提高了感染风险。这种风险是如此巨大，以至于我们正在接近这样一个时期，即一部分老年骨髓瘤患者可能更有可能死于重定向t细胞治疗引起的感染性并发症，而不是死于骨髓瘤。因此，为骨髓瘤患者提供适当的预防和监测感染是至关重要的。在这篇综述中，我们讨论了骨髓瘤患者感染风险增加的疾病相关、患者相关和治疗相关的原因，以及如何通过制定针对个体患者的动态感染预防计划来预防和管理这种风险。

引用次数: 0

Haploidentical transplantation: An optimal platform for graft manipulation and cellular therapies 单倍体移植：移植操作和细胞治疗的最佳平台。

IF 6.9 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-03-21 DOI: 10.1016/j.blre.2025.101286

C.C. Astigarraga , Klauberg MPMS , L. Iovino , F. Milano

Allogeneic hematopoietic stem cell transplantation (allo-HCT) remains a curative therapeutic option for patients with high-risk hematologic malignancies. When a fully matched donor is unavailable, haploidentical hematopoietic stem cell transplantation (haplo-HCT) provides a viable alternative. Over time, haplo-HCT procedures have significantly evolved, improving outcomes in treatment related mortality (TRM), especially in graft-versus-host disease (GvHD). However, challenges such as delayed immune reconstitution and disease relapse persist. Advances in in vivo graft manipulation techniques, such as post-transplant cyclophosphamide (PTCy) and ex vivo approaches, including TCRα/β and CD19 depletion, have shown promise in reducing the risk of severe GvHD without increasing the relapse rates. Innovative strategies, such as haploidentical donor lymphocyte infusions, “suicide-switch” mechanisms, ORCA-Q product infusions, and CAR based therapies offer potential to further optimize outcomes. This review examines the graft manipulation modalities in the haplo-HCT setting, highlighting their role in advancing cellular therapies and providing new hope in the fight against life-threatening diseases.

同种异体造血干细胞移植（allogene hematopoietic stem cell transplantation, alloo - hct）仍然是治疗高危恶性血液病的有效选择。当没有完全匹配的供体时，单倍体造血干细胞移植（haploo - hct）提供了一个可行的选择。随着时间的推移，单倍hct技术已经显著发展，改善了治疗相关死亡率（TRM）的结果，特别是在移植物抗宿主病（GvHD）中。然而，免疫重建延迟和疾病复发等挑战仍然存在。体内移植操作技术的进展，如移植后环磷酰胺（PTCy）和体外方法，包括TCRα/β和CD19去除，已经显示出在不增加复发率的情况下降低严重GvHD风险的希望。创新策略，如单倍体供体淋巴细胞输注、“自杀开关”机制、ORCA-Q产品输注和基于CAR的疗法，提供了进一步优化结果的潜力。本文综述了单倍hct环境下的移植物操作方式，强调了它们在推进细胞治疗方面的作用，并为对抗危及生命的疾病提供了新的希望。

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引用次数: 0

Neutrophils in BCR::ABL1 negative MPN: Contributors or bystanders of fibrosis? BCR中性粒细胞：ABL1阴性MPN：纤维化的贡献者还是旁观者？

IF 6.9 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-03-20 DOI: 10.1016/j.blre.2025.101285

Gaël Vermeersch , Mieke Gouwy , Paul Proost , Sofie Struyf , Timothy Devos

BCR::ABL1 negative myeloproliferative neoplasms (MPNs) are a heterogenous group of disorders characterized by clonal proliferation of hematopoietic stem and progenitor cells (HSPCs) within the bone marrow. Although the identification of somatic key driver mutations significantly increased both understanding and diagnostic accuracy of MPNs, many questions about the exact pathophysiology remain unanswered. Increased neutrophil count at diagnosis is a well-recognized predictor of worse disease evolution and survival, nonetheless the exact role of neutrophilic granulocytes within MPN pathophysiology is almost unexplored. As the majority of these cells are residing within the bone marrow, they represent a non-negligible entity within the bone marrow niche and its homeostasis. This review describes how neutrophils might contribute to the development of the inflammatory bone marrow niche, and hereby also fibrosis, associated with MPNs. The versatile functions and effects in different contexts emphasize the necessity for future research oriented to bone marrow in addition to peripheral blood.

BCR: ABL1阴性骨髓增生性肿瘤（mpn）是一种异质性疾病，其特征是骨髓内造血干细胞和祖细胞（HSPCs）的克隆性增殖。尽管体细胞关键驱动突变的识别显著提高了对mpn的理解和诊断准确性，但关于确切病理生理的许多问题仍未得到解答。诊断时中性粒细胞计数增加是疾病恶化和生存的一个公认的预测因素，尽管如此，中性粒细胞在MPN病理生理中的确切作用几乎未被探索。由于这些细胞大多数存在于骨髓中，它们代表了骨髓生态位及其稳态中不可忽视的实体。这篇综述描述了中性粒细胞如何促进炎症性骨髓生态位的发展，从而促进与mpn相关的纤维化。在不同情况下的多种功能和作用强调了除外周血外进一步研究骨髓的必要性。

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引用次数: 0

Low flow: Selecting a limited flow cytometry panel where resources are constrained 低流量：在资源有限的情况下选择有限的流式细胞仪面板。

IF 6.9 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-03-17 DOI: 10.1016/j.blre.2025.101284

Ailie Ross , Donna Rudd , Joel Wight

Diagnosis and treatment of patients with haematological malignancies (HM) is hampered by access to pathology services in resource-limited settings (RLS). Internationally accepted guidelines and diagnostic criteria for HM require access to sophisticated analysis including comprehensive flow cytometry (FCM) for minimum essential diagnosis and treatment, which is technically challenging in RLS. This review will define these shortcomings and examine the use of limited FCM panels in RLS.

While a consensus guideline exists for a limited chronic lymphocytic leukaemia (CLL) FCM panel, this has yet to be validated in a large cohort. Currently, there are no consensus-based and resource-stratified diagnostic protocols defining limited FCM panels for the diagnosis of acute leukaemia where resources are limited.

There is an unmet need for such guidelines, supported by evidence, for the diagnosis of the most common HM. This systematic review defines consensus-based limited FCM panels from the literature that may be used in the interim.

血液系统恶性肿瘤（HM）患者的诊断和治疗受到资源有限的环境（RLS）获得病理服务的阻碍。国际公认的HM指南和诊断标准要求使用复杂的分析，包括全面的流式细胞术（FCM），以进行最低限度的基本诊断和治疗，这在RLS中具有技术挑战性。这篇综述将定义这些缺点，并检查有限的FCM面板在RLS中的使用。虽然存在有限慢性淋巴细胞白血病（CLL） FCM小组的共识指南，但这尚未在大型队列中得到验证。目前，在资源有限的情况下，没有基于共识和资源分层的诊断方案来定义有限的FCM小组，以诊断急性白血病。对于诊断最常见的HM，这种有证据支持的指南的需求尚未得到满足。本系统综述从文献中定义了可能在此期间使用的基于共识的有限FCM小组。

引用次数: 0

Bridging the evidence-to-practice gap: Advancing neonatal blood transfusion. A narrative review of recent guidelines 弥合从证据到实践的差距：推进新生儿输血。对近期指导方针的叙述性回顾。

IF 6.9 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-03-08 DOI: 10.1016/j.blre.2025.101282

Rozeta Sokou , Eleni A. Gounari , Andreas G. Tsantes , Daniele Piovani , Stefanos Bonovas , Argirios E. Tsantes , Nicoletta Iacovidou

Neonates represent a distinct population within the context of transfusion medicine. Blood transfusions in neonates are vital interventions for multiple conditions, despite their inherent risks and potential complications. Differences in physiology and other transfusion risk factors unique to this group require careful adaptation of transfusion guidelines. This article seeks to offer a thorough overview of the current evidence-based practices for RBC administration in neonates. It covers the collection, processing and storage of RBCs and discusses the research underpinning the most recent transfusion guidelines. Furthermore, it emphasizes the challenges in establishing precise cut-off values for these conditions in both preterm and critically ill neonates and discusses indications for transfusion, thresholds, current guidelines, and potential complications. Finally, it highlights gaps in critical areas of transfusion related research and proposes future targets for research.

新生儿在输血医学的背景下代表一个独特的人群。新生儿输血是多种情况下的重要干预措施，尽管其固有风险和潜在并发症。这一群体的生理差异和其他输血危险因素需要仔细调整输血指南。这篇文章旨在提供一个全面的概述目前的证据为基础的做法，RBC管理在新生儿。它涵盖了红细胞的收集、处理和储存，并讨论了支持最新输血指南的研究。此外，它强调了在早产儿和危重新生儿中为这些条件建立精确的临界值所面临的挑战，并讨论了输血的指征、阈值、现行指南和潜在并发症。最后，它强调了输血相关研究关键领域的差距，并提出了未来的研究目标。

引用次数: 0

Innovations in red blood cell preservation 红细胞保存方面的创新。

IF 6.9 2区医学 Q1 HEMATOLOGY

Blood Reviews

Pub Date : 2025-03-07 DOI: 10.1016/j.blre.2025.101283

Nishaka William , Jason P. Acker

The global infrastructure supporting nearly 100 million transfusions annually relies on the ability to store red cell concentrates (RCCs) for up to 42 days at hypothermic temperatures or indefinitely at low sub-zero temperatures. While these methods are generally effective, there is both an opportunity and, in specific settings, a need to refine storage techniques that have remained largely unchanged since the 1980s. Recent research has identified ways to address limitations that were not fully understood when these methods were first implemented in blood banks, with much of it focusing on modifying conventional storage strategies, while some studies explore alternative approaches. In this review, we explore the current state of RBC preservation and the future prospects for advancing both short- and long-term storage strategies.

每年支持近1亿次输血的全球基础设施依赖于在低温或零下低温下无限期储存红细胞浓缩物（rcc）长达42天的能力。虽然这些方法一般来说是有效的，但是有机会而且在特定情况下需要改进自1980年代以来基本保持不变的储存技术。最近的研究已经确定了解决这些方法首次在血库中实施时未完全理解的局限性的方法，其中大部分研究侧重于修改传统的存储策略，而一些研究则探索替代方法。在这篇综述中，我们探讨了RBC保存的现状以及推进短期和长期存储策略的未来前景。

引用次数: 0

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