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The Science Behind Clinical Practice: What is a CAR-T Cell? 临床实践背后的科学:什么是CAR-T细胞?
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-29 DOI: 10.1016/j.tmrv.2025.150933
Aswath P Chandrasekar
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引用次数: 0
New Horizons in Transfusion Medicine 输血医学的新视野。
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1016/j.tmrv.2025.150934
Sunny Dzik , Jeannie Callum , Zoe McQuilten
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引用次数: 0
Efficacy of Granulocyte Transfusions in Treating Neutropenic Infections: A Systematic Review and Meta-Analysis of Intervention Studies 粒细胞输注治疗中性粒细胞减少感染的疗效:干预研究的系统回顾和荟萃分析。
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1016/j.tmrv.2025.150929
Alejandra López-Arredondo , Saúl Karr de León , Anna-Maria Lampousi , Marion E.G. Brunck
The therapeutic value of granulocyte transfusions (GTX) remains debated. We conducted a systematic review and meta-analysis of intervention studies evaluating GTX efficacy in treating neutropenic infections. MEDLINE, EMBASE, and Cochrane Central were searched from inception to March 2025 to identify interventional studies evaluating the efficacy of GTX for neutropenic infections. Studies were qualitatively summarized. Summary risk ratios (RR) with 95% confidence intervals (CIs) were estimated for randomized controlled trials (RCTs), and non-randomized controlled trials (NRCTs) using random-effects models. Certainty of evidence was evaluated using GRADE. There were 110 studies meeting inclusion criteria: 16 RCTs, 14 NRCTs, and 80 uncontrolled trials. The most frequent underlying disease was leukemia, and the most frequently reported pathogen was Candida. In RCTs, GTX showed no significant all-cause mortality reduction over standard-of-care in pediatric/adult patients or neonates, both associations with low certainty of evidence. In contrast, prospective NRCTs including pediatric/adult patients showed that GTX led to lower all-cause mortality (RR 0.40; 95% CI: 0.23-0.68, I2: 64%), particularly among recipients of high-dose GTX (≥1 × 1010cells/transfusion), with very low-certainty evidence. Results support a dose-response relationship and highlight heterogeneity in patients, treatment settings, and infections. This work recommends carefully designed future RCTs, including strict patient stratification.
粒细胞输注(GTX)的治疗价值仍有争议。我们对评估GTX治疗中性粒细胞减少感染疗效的干预研究进行了系统回顾和荟萃分析。检索MEDLINE、EMBASE和Cochrane Central从成立到2025年3月,以确定评估GTX治疗中性粒细胞减少感染疗效的介入性研究。对研究进行定性总结。采用随机效应模型估计随机对照试验(rct)和非随机对照试验(NRCTs)的总风险比(RR)和95%置信区间(ci)。使用GRADE评价证据的确定性。有110项研究符合纳入标准:16项随机对照试验,14项非随机对照试验和80项非对照试验。最常见的基础疾病是白血病,最常见的病原体是念珠菌。在随机对照试验中,GTX显示儿科/成人患者或新生儿的全因死亡率与标准护理相比没有显著降低,这两种关联的证据确定性都较低。相比之下,包括儿童/成人患者在内的前瞻性nrct显示,GTX导致较低的全因死亡率(RR 0.40; 95% CI: 0.23-0.68, I2: 64%),特别是在高剂量GTX(≥1 × 1010个细胞/输血)的接受者中,证据的确定性非常低。结果支持剂量-反应关系,并强调患者、治疗环境和感染的异质性。这项工作建议仔细设计未来的随机对照试验,包括严格的患者分层。
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引用次数: 0
A Practical Review of Adaptive Platform Trials 适应性平台试验的实践回顾
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1016/j.tmrv.2025.150935
Natalia Alejandra Angeloni , Neill KJ Adhikari , François Lamontagne
Traditional randomized controlled trials (RCTs) can provide rigorous evidence but are often slow and resource-intensive, requiring separate trials for each intervention. Adaptive platform trials (APTs) have been promoted as a solution, offering a framework that tests multiple therapies under a single protocol, with arms added or dropped as evidence accumulates. However, their advantages come with trade-offs that warrant scrutiny. In this review, we critically appraise 3 landmark APTs. The I-SPY2 trial accelerated Phase II oncology research by utilizing Bayesian adaptive randomization and surrogate endpoints; however, much of its efficiency stemmed from relying on intermediate outcomes, which may not reliably predict survival. RECOVERY demonstrated the power of scale on a pragmatic UK-wide platform, but its success reflected health system infrastructure, political leadership, and the unique circumstances of the COVID-19 pandemic as much as its design. REMAP-CAP, a perpetual platform trial for pneumonia, rapidly switched to pandemic mode in 2020 and tested COVID-19 therapies using Bayesian models and response-adaptive randomization (RAR); however, the RAR amplified random noise in some domains, exposing patients to interventions later shown to be ineffective. A recent systematic review confirmed wide heterogeneity in APTs and suboptimal reporting. APTs are not inherently better than classical RCTs. Gains in speed may depend on less rigorous endpoints, complex adaptive methods, or streamlined oversight, each of which introduces new risks of error. As APTs spread to new fields such as transfusion medicine, clinicians and researchers must learn to recognize both the potential benefits and the pitfalls of this design.
传统的随机对照试验(RCTs)可以提供严格的证据,但通常速度缓慢且资源密集,每次干预都需要单独的试验。适应性平台试验(APTs)作为一种解决方案得到了推广,它提供了一个框架,可以在单一方案下测试多种疗法,并随着证据的积累而增加或减少武器。然而,它们的优势也伴随着值得仔细审视的权衡。在这篇综述中,我们对3种具有里程碑意义的apt进行了批判性评价。I-SPY2试验利用贝叶斯自适应随机化和替代终点加速了II期肿瘤学研究;然而,它的大部分效率源于依赖于中间结果,这可能无法可靠地预测生存。在一个务实的全英国平台上,“复苏”展示了规模的力量,但它的成功不仅反映了它的设计,也反映了卫生系统基础设施、政治领导和COVID-19大流行的独特环境。REMAP-CAP是一项针对肺炎的永久性平台试验,于2020年迅速切换到大流行模式,并使用贝叶斯模型和反应适应性随机化(RAR)测试了COVID-19疗法;然而,RAR放大了某些领域的随机噪声,使患者暴露于后来被证明无效的干预措施中。最近的一项系统综述证实了APTs的广泛异质性和次优报告。apt并不天生就比经典随机对照试验好。速度的提高可能取决于不太严格的端点、复杂的自适应方法或流线型的监督,每一种都会引入新的错误风险。随着apt扩散到输血医学等新领域,临床医生和研究人员必须学会认识到这种设计的潜在好处和缺陷。
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引用次数: 0
Gene Therapies for Hemoglobinopathies: Efficacy, Cell Collection & Transfusion Support 血红蛋白病的基因治疗:疗效、细胞收集和输血支持。
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1016/j.tmrv.2025.150930
Zaina Inam , HyoJeong Han , Jennifer Webb , Meghan Delaney
Sickle cell disease (SCD) and transfusion dependent β-thalassemia (TDT) are complex disorders, often resulting in lifelong morbidity and reduced life expectancy. Allogeneic hematopoietic stem cell transplant (HSCT) is curative, with matched-related donor (MRD) transplant having the highest success. MRD availability is limited for both disorders, and HCT carries the risk of transplant-related complications, such as graft-versus-host disease (GHVD) and graft failure. Gene therapy (GT) offers an alternative curative option by modifying autologous hematopoietic stem and progenitor cells (HSPCs), making the treatment available to all, while eliminating the risk of GVHD. The U.S. Food and Drug Administration (FDA) has approved GTs for both SCD and TDT: lovotibeglogene autotemcel (Lyfgenia) and exagamglogene autotemcel (Casgevy) in 2023 for SCD and betibeglogene autotemcel (Zynteglo) in 2022 and exagamglogene autotemcel (Casgevy) in 2024 for TDT. This article appraises the studies the FDA approvals were based upon, with comments on transfusion and stem collection regimens. The latter aspects highlighting variability in practice and the need for additional studies to optimize pretransfusion regimens and the collection process for successful GT.
镰状细胞病(SCD)和输血依赖性β-地中海贫血(TDT)是复杂的疾病,往往导致终身发病率和预期寿命缩短。同种异体造血干细胞移植(HSCT)具有治愈性,其中匹配相关供体(MRD)移植成功率最高。这两种疾病的MRD可用性都是有限的,并且HCT具有移植相关并发症的风险,例如移植物抗宿主病(GHVD)和移植物衰竭。基因疗法(GT)通过修饰自体造血干细胞和祖细胞(HSPCs)提供了一种替代治疗选择,使所有人都能获得治疗,同时消除了GVHD的风险。美国食品和药物管理局(FDA)已于2023年批准了用于SCD和TDT的gt: lovotibeglogene autotemcel (Lyfgenia)和glogene autotemcel (Casgevy)于2023年批准用于SCD和glogene autotemcel (Zynteglo), glogene autotemcel (Casgevy)于2024年批准用于TDT。本文评价了FDA批准所基于的研究,并对输血和干细胞收集方案进行了评论。后一个方面强调了实践中的可变性,需要进一步的研究来优化输血前方案和收集过程,以实现成功的GT。
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引用次数: 0
TMR Paper for Issue on “Technologies in Transfusion Medicine” Dried Plasma – Where Are We and Where Next? 关于“输血医学技术”的TMR论文-我们在哪里,下一步在哪里?
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1016/j.tmrv.2025.150931
Matt Ellington , Ed Barnard , Laura Green , Tom Woolley , Rebecca Cardigan
Traumatic hemorrhage is a major cause of morbidity and mortality, in both military and civilian settings. Early hemostatic resuscitation including red cell and plasma administration is a mainstay of treatment. Prehospital blood transfusion benefits trauma patients, but delivery is logistically challenging. Dried plasma, stored in ambient conditions and reconstituted rapidly, without specialist equipment, offers a pragmatic solution to the logistical barriers to prehospital transfusion. This review outlines mechanisms of action, and approaches to evaluating efficacy of plasma, summarizes advances in drying technologies and their sequelae on plasma quality, and critically appraises 4 published studies.
创伤性出血是军事和民用环境中发病和死亡的主要原因。早期止血复苏包括红细胞和血浆给药是主要的治疗方法。院前输血有利于创伤患者,但在运送过程中存在后勤方面的挑战。干燥的血浆,在环境条件下储存并快速重建,没有专业设备,为院前输血的后勤障碍提供了一个实用的解决方案。本文概述了血浆的作用机制和疗效评价方法,总结了干燥技术的进展及其对血浆质量的影响,并对已发表的4项研究进行了批判性评价。
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引用次数: 0
Monitoring Mitochondrial Oxygen Tension: mitoPO2 as Physiologic Transfusion Trigger? 监测线粒体氧张力:mitoPO2作为生理性输血触发因素?
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1016/j.tmrv.2025.150932
Lucia W.J.M. Streng, Floor A. Harms, Egbert G. Mik
Hemoglobin-based red blood cell transfusion (RBC) triggers are inadequate for personalized transfusion decisions because they are population-based and therefore unable to identify individual patients that will benefit from RBC transfusion. Therefore, physiological transfusion triggers are sought after to provide tools for a more individualize approach. Since mitochondria are the ultimate destination of oxygen it seems reasonable to suggest that measuring oxygen at the mitochondrial level might provide insight in the need for RBC transfusion. Mitochondrial oxygen tension (mitoPO2) is a novel clinical parameter that can be measured by an optical technology. This narrative review provides a brief introduction on mitoPO2 monitoring and uses 5 recent studies to explore the potential of mitoPO2 as tool for assessing need for transfusion and/or monitoring the effect of transfusion. A mathematical model shows an ideal behavior of mitoPO2 on critical hematocrit and from 4 recent clinical studies we learn that mitoPO2 is an independent parameter that can be used in transfusion-related studies. Further investigation into the potential role of mitoPO2 in transfusion medicine is needed.
基于血红蛋白的红细胞输血(RBC)触发因素不适合个性化的输血决策,因为它们是基于人群的,因此无法识别个体患者将受益于RBC输血。因此,生理输血触发被寻求为更个性化的方法提供工具。由于线粒体是氧气的最终目的地,因此似乎有理由认为,在线粒体水平上测量氧气可能有助于了解红细胞输血的需求。线粒体氧张力(mitoPO2)是一种新的临床参数,可以通过光学技术测量。本文简要介绍了mitoPO2监测,并利用最近的5项研究来探讨mitoPO2作为评估输血需求和/或监测输血效果的工具的潜力。数学模型显示mitoPO2对临界红细胞压积的理想行为,从最近的4项临床研究中,我们了解到mitoPO2是一个独立的参数,可用于输血相关研究。需要进一步研究mitoPO2在输血医学中的潜在作用。
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引用次数: 0
Toward Personalized Transfusion Strategies: The Emerging Role of Wearable Biosensors in Chronic Anemia Management 个性化输血策略:可穿戴生物传感器在慢性贫血管理中的新作用
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-10-01 DOI: 10.1016/j.tmrv.2025.150927
R.P.B. Tonino , M.R. Schipperus , J.J. Zwaginga
Chronic transfusion-dependent anemia presents ongoing challenges in optimizing symptom control and functional outcomes, particularly in an older, often comorbid patient group. Conventional transfusion strategies based on fixed hemoglobin thresholds may inadequately address the individual variability in oxygen delivery needs and symptom burden. Wearable biosensor technologies enable continuous monitoring of physiological parameters such as heart rate, respiratory rate, and physical activity in real-world settings. These tools offer the potential to detect early deterioration and support more responsive, patient-centered transfusion decisions and improve hemovigilance. This review evaluates current evidence on the feasibility, acceptability, and clinical relevance of biosensor use in transfusion medicine. Findings from recent pilot studies demonstrate high data quality, favorable tolerability, and preliminary indications of physiological response following transfusion. However, the clinical utility of biosensor-guided transfusion strategies remains unproven, with key challenges including data interpretation, workflow integration, and validation of clinically meaningful endpoints. As the field moves toward personalized supportive care, biosensors may offer a novel means to optimize transfusion timing, preserve functional capacity, and enhance quality of life.
慢性输血依赖性贫血在优化症状控制和功能结果方面提出了持续的挑战,特别是在老年,通常合并症患者组中。基于固定血红蛋白阈值的传统输血策略可能不足以解决氧气输送需求和症状负担的个体差异。可穿戴生物传感器技术可以在现实环境中持续监测心率、呼吸频率和身体活动等生理参数。这些工具提供了发现早期恶化的潜力,支持更及时、以患者为中心的输血决策,并提高血液警惕性。这篇综述评估了生物传感器在输血医学中应用的可行性、可接受性和临床相关性的现有证据。最近的初步研究结果表明,高数据质量,良好的耐受性和输血后生理反应的初步适应症。然而,生物传感器引导输血策略的临床应用仍未得到证实,主要挑战包括数据解释、工作流程整合和临床有意义终点的验证。随着该领域向个性化支持护理的发展,生物传感器可能提供一种优化输血时机、保持功能能力和提高生活质量的新方法。
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引用次数: 0
ABO matching for platelet transfusions for prevention or treatment of bleeding: A systematic review with meta-analysis. 用于预防或治疗出血的血小板输注ABO匹配:一项荟萃分析的系统综述。
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-09-11 DOI: 10.1016/j.tmrv.2025.150928
Lorna Cain, Asha Aggarwal, Louise J Geneen, Carolyn Dorée, Lise J Estcourt, Rebecca Cardigan, Michael Desborough

Transfusion of ABO identical platelets is recommended in national guidelines, though transfusion of ABO non-identical platelets has been widely adopted to ensure availability and reduce wastage. When ABO non-identical platelets are necessitated, there is a lack of consensus on prioritisation of major or minor compatibility. We conducted a systematic review and meta-analysis (PROSPERO CRD42023450792) of randomised and non-randomised studies to assess whether there is a difference when comparing ABO-identical and non-identical (major, minor, bi-directional mismatch) platelet transfusions. From 4177 potential references, 18 studies met our criteria: 3 randomised controlled trials (RCTs), 8 prospective and 7 retrospective observational studies. Evidence was very low certainty as to whether there was a difference from transfusion of ABO identical or non-identical platelets, where data were available, for clinically significant (WHO grade 2+ and 3+) bleeding, mortality, acute transfusion reactions, platelet refractoriness. Platelet increments were the most frequently reported outcomes. Overall, there was a paucity of evidence for clinical outcome data including bleeding risk for ABO identical compared to non-identical transfusion. We make recommendations for designing and reporting future platelet ABO matching studies based on our observations in this review. Future studies should consider the effect of repeated exposure to ABO identical or non-identical transfusions and known confounders.

国家指南建议输血ABO相同的血小板,但输血ABO不相同的血小板已被广泛采用,以确保可获得性并减少浪费。当需要ABO不相同的血小板时,对主要或次要相容性的优先次序缺乏共识。我们对随机和非随机研究进行了系统回顾和荟萃分析(PROSPERO CRD42023450792),以评估abo血型相同和非abo血型相同(主要、次要、双向错配)血小板输注是否存在差异。从4177个潜在参考文献中,18个研究符合我们的标准:3个随机对照试验(rct), 8个前瞻性和7个回顾性观察性研究。在有数据的情况下,输血ABO相同或不相同的血小板在临床上显著(WHO分级2+和3+)出血、死亡率、急性输血反应、血小板难耐性方面是否存在差异,证据的确定性非常低。血小板增加是最常报道的结果。总的来说,缺乏临床结果数据的证据,包括ABO相同输血与非ABO相同输血的出血风险。基于我们在这篇综述中的观察,我们提出了设计和报告未来血小板ABO匹配研究的建议。未来的研究应考虑反复接触ABO相同或不相同输血和已知混杂因素的影响。
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引用次数: 0
Artificial Intelligence and Machine Learning in Transfusion Practice: An Analytical Assessment 输血实践中的人工智能和机器学习:分析性评估。
IF 2.5 2区 医学 Q2 HEMATOLOGY Pub Date : 2025-08-24 DOI: 10.1016/j.tmrv.2025.150926
Na Li , Ruchika Goel , Sheharyar Raza , Kiarash Riazi , Jie Pan , Huong Quynh Nguyen , Andrew W. Shih , Adam D’Souza , Rounak Dubey , Aaron A.R. Tobian , Donald M. Arnold
Transfusion medicine is vital to healthcare and affects clinical outcomes, patient safety, and system resilience while addressing challenges such as blood shortages, donor variability, and rising costs. The integration of artificial intelligence (AI) and machine learning (ML) presents new opportunities to improve clinical decision-making and operational effectiveness in this field. This structured narrative review identified and evaluated studies applying AI and ML in transfusion medicine. A search of PubMed and Scopus for articles published between January 2018 and April 2025 yielded 565 publications. Studies were included if they applied AI or ML techniques, focused on transfusion management or decision support, and were evaluated using electronic health records or expert review. Four exemplar studies were selected, each representing a distinct AI paradigm: supervised, unsupervised, reinforcement, and generative learning. These studies were critically appraised for methodological rigor, clinical relevance, and potential for implementation in practice. The reviewed studies reflected a clear shift from traditional analytic methods toward more advanced computational approaches to improve prediction accuracy, optimize resource allocation, and support clinical decision-making. Three overarching themes emerged: the need to balance model complexity with interpretability and clinical feasibility; the impact of data quality and preprocessing on model performance and fairness; and the barriers to broader applicability and cross-institutional deployment. As technological barriers continue to decline, future challenges will increasingly center on privacy regulations, infrastructure constraints, and aligning model complexity with practical utility. Thoughtful integration of these considerations through scalable, clinical-grade, and transparent solutions will be critical in realizing the full potential of AI and ML in transfusion medicine.
输血医学对医疗保健至关重要,影响临床结果、患者安全和系统弹性,同时应对血液短缺、献血者多样性和成本上升等挑战。人工智能(AI)和机器学习(ML)的融合为提高该领域的临床决策和操作效率提供了新的机会。这篇结构化的叙述性综述确定并评估了在输血医学中应用人工智能和ML的研究。在PubMed和Scopus上搜索2018年1月至2025年4月间发表的文章,得到565篇。如果研究应用了人工智能或机器学习技术,专注于输血管理或决策支持,并使用电子健康记录或专家审查进行评估,则纳入研究。选择了四个范例研究,每个研究都代表了一个不同的人工智能范式:监督学习、无监督学习、强化学习和生成学习。这些研究在方法的严谨性、临床相关性和在实践中实施的潜力方面得到了严格的评价。回顾的研究反映了从传统分析方法向更先进的计算方法的明显转变,以提高预测准确性,优化资源分配,并支持临床决策。出现了三个总体主题:需要平衡模型的复杂性与可解释性和临床可行性;数据质量和预处理对模型性能和公平性的影响;更广泛的适用性和跨机构部署的障碍。随着技术壁垒的不断下降,未来的挑战将越来越多地集中在隐私法规、基础设施约束以及将模型复杂性与实际效用相结合上。通过可扩展、临床级和透明的解决方案将这些考虑周到地整合在一起,对于实现人工智能和机器学习在输血医学中的全部潜力至关重要。
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引用次数: 0
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Transfusion Medicine Reviews
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