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Prognostic performance of machine learning in predicting haematological cancer outcomes: systematic review and meta-analysis 机器学习在预测血液学癌症结果中的预后表现:系统回顾和荟萃分析。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/j.blre.2025.101325
Adugnaw Zeleke Alem , Itismita Mohanty , Nalini Pati , Theophile Niyonsenga
Diagnosis and treatment of haematologic malignancies present significant challenges, underscoring the need for highly individualized therapeutic approaches. Incorporating machine learning (ML) algorithms into predictions of haematological cancer outcomes has been increasingly investigated in recent years. However, it has not been investigated whether ML-based approach is superior to standard regression methods. Therefore, this review aims to assess their performance as compared to standard regression-based prediction methods. Studies from Web of Science, Medline, SCOPUS, CINHAL were reviewed, and associated risk of bias (ROB) assessed using PROBAST (Prediction Model Risk of Bias Assessment Tool). Standard and three-level random-effects meta-analysis were performed to obtain the ML pooled discriminative ability. Of 4204 retrieved studies, 48 were included in the systematic review. Pooled area under the curve (AUC) values of 24 top-performing ML and all 71 ML models from 19 studies were 0.80 (95% CI: 0.76, 0.84) and 0.779 (95% CI: 0.731, 0.827) in the testing set, respectively. The discrimination ability was similar between top-performing ML algorithms (AUC = 0.78, 95% CI: 0.70–0.86) and standard regression (AUC = 0.72, 95% CI: 0.66–0.78) in testing set. The three-level meta-analysis that incorporated all ML algorithms revealed similar results. However, externally validated top-performing ML algorithms outperformed standard models with a pooled AUC of 0.87 (95% CI: 0.76–0.98) compared with 0.72 (95% CI: 0.66–0.79). Although ML models' performance was acceptable, studies generally exhibited high ROB, emphasizing the need for future ML models to adhere to PROBAST guidelines.
血液恶性肿瘤的诊断和治疗面临重大挑战,强调需要高度个性化的治疗方法。近年来,将机器学习(ML)算法纳入血液学癌症结果预测的研究越来越多。然而,基于机器学习的方法是否优于标准回归方法尚未得到研究。因此,本综述旨在评估它们与基于标准回归的预测方法相比的性能。我们回顾了Web of Science、Medline、SCOPUS、CINHAL的研究,并使用PROBAST(预测模型偏倚风险评估工具)评估相关的偏倚风险(ROB)。进行标准和三水平随机效应荟萃分析以获得ML池判别能力。在4204项被检索的研究中,48项被纳入系统评价。来自19项研究的24个表现最好的ML模型和所有71个ML模型的合并曲线下面积(AUC)值在测试集中分别为0.80 (95% CI: 0.76, 0.84)和0.779 (95% CI: 0.731, 0.827)。测试集中表现最好的ML算法(AUC = 0.78, 95% CI: 0.70 ~ 0.86)与标准回归(AUC = 0.72, 95% CI: 0.66 ~ 0.78)的判别能力相似。纳入所有ML算法的三层元分析显示了类似的结果。然而,外部验证的最佳ML算法优于标准模型,合并AUC为0.87 (95% CI: 0.76-0.98),而标准模型的AUC为0.72 (95% CI: 0.66-0.79)。虽然ML模型的表现是可以接受的,但研究通常显示出较高的ROB,强调未来ML模型需要遵守PROBAST指南。
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引用次数: 0
Metabolomics in sickle cell disease: Current knowledge and gaps – A scoping review 镰状细胞病的代谢组学:目前的知识和差距-范围综述。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/j.blre.2025.101338
Sigrid van der Veen , Judith J.M. Jans , Eduard J. van Beers , Bart J. Biemond , Pablo Bartolucci , Maria Paola Boaro , Anna Collado Gimbert , Raffaella Colombatti , Mirco D'Agnolo , Karin J. Fijnvandraat , Amira Idrizovic , Petros Kountouris , Mar Mañú Pereira , Elisabetta Mezzalira , Minke A.E. Rab , Anita W. Rijneveld , Tiziana Sanavia , Nanda M. Verhoeven-Duif , Marjon H. Cnossen
Sickle cell disease (SCD) has large phenotypic variability. Systematic metabolomic profiling may provide insights into phenotypes and treatment responses. We conducted a scoping review on associations between blood metabolites, SCD-related complications, and therapies in studies analyzing ≥10 metabolites in red blood cells, whole blood, and plasma. Lipidomics-focused studies were excluded. Fifteen studies were included, focusing on metabolic profiling, clinical outcomes, or therapies (hydroxyurea, transfusion, and mitapivat). Metabolic profiling differentiated SCD from healthy controls and patients with HbSS and HbSC genotypes. Associations with hemolysis, vaso-occlusive events, nephropathy, TRV, and mortality were identified. Overall, metabolites were involved in arginine, tryptophan, glutamate metabolism, glycolysis, pentose phosphate pathway, and the Lands cycle. Some metabolites showed opposite correlations across complications or sample types. Despite growing interest, gaps remain in study designs, metabolite selection, genotype representation, and underexplored complications and therapies. Standardized, large-scale metabolomics studies are needed to advance personalized treatment in SCD.
镰状细胞病(SCD)具有很大的表型变异性。系统代谢组学分析可以提供对表型和治疗反应的见解。我们在分析红细胞、全血和血浆中≥10种代谢物的研究中,对血液代谢物、scd相关并发症和治疗之间的关系进行了范围综述。以脂质组学为重点的研究被排除在外。纳入了15项研究,重点是代谢谱、临床结果或治疗(羟脲、输血和米他伐特)。代谢分析将SCD与健康对照、HbSS和HbSC基因型患者区分开来。确定了溶血、血管闭塞事件、肾病、TRV和死亡率的相关性。总的来说,代谢产物涉及精氨酸、色氨酸、谷氨酸代谢、糖酵解、戊糖磷酸途径和Lands循环。一些代谢物在并发症或样本类型中显示相反的相关性。尽管人们的兴趣越来越大,但在研究设计、代谢物选择、基因型代表以及未充分探索的并发症和治疗方面仍然存在差距。需要标准化、大规模的代谢组学研究来推进SCD的个性化治疗。
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引用次数: 0
Patient-reported outcomes - the missing link to advancing light chain (AL) amyloidosis clinical research 患者报告的结果-推进轻链(AL)淀粉样变性临床研究的缺失环节。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/j.blre.2025.101303
Tobias Dittrich , Lina Weinert , Anita D'Souza
Systemic light chain (AL) amyloidosis often results in multi-organ dysfunction and significant morbidity. Clinical assessments may not capture the full impact of disease and treatment on patients. Patient-reported outcomes (PROs) can help fill this gap. Although evidence suggests that the use of PROs provides additional predictive value beyond established cardiac staging systems, their integration into standard AL amyloidosis management remains limited. Our review examines the prognostic and therapeutic value of PROs and their current use as endpoints in clinical trials. We also discuss practical considerations, including instrument selection and administration, data interpretation, and reporting. Finally, we present a roadmap for integrating PROs into routine AL amyloidosis management, focusing on the selection of appropriate ePRO platforms and implementation strategies. We advocate a framework for data sharing and a coordinated research agenda. By addressing evidence gaps and prioritizing the patient perspective, PROs have the potential to advance AL amyloidosis care and research.
系统性轻链(AL)淀粉样变常导致多器官功能障碍和显著发病率。临床评估可能无法充分反映疾病和治疗对患者的影响。患者报告的结果(PROs)可以帮助填补这一空白。尽管有证据表明,使用pro提供了比已建立的心脏分期系统更多的预测价值,但它们与标准AL淀粉样变管理的整合仍然有限。我们的综述考察了PROs的预后和治疗价值,以及它们目前在临床试验中作为终点的使用情况。我们还讨论了实际考虑,包括仪器的选择和管理,数据解释和报告。最后,我们提出了将pro纳入常规AL淀粉样变性治疗的路线图,重点是选择合适的ePRO平台和实施策略。我们主张建立数据共享框架和协调研究议程。通过解决证据差距和优先考虑患者的观点,PROs有可能推进AL淀粉样变性的治疗和研究。
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引用次数: 0
Old tests and new paradigms: How to interpret iron studies and related biomarkers for the diagnosis of iron deficiency in adults 旧的测试和新的范式:如何解释铁研究和相关的生物标志物诊断成人缺铁。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/j.blre.2025.101337
Magdalena Kriel , Jessica Opie , Jody Rusch , David Richardson , Vernon Louw
Clinicians need a good understanding of available tools to diagnose iron deficiency (ID). Interpretation of commonly used laboratory tests can be challenging due to the dynamic nature of iron homeostasis and concurrent inflammation, which influence results. The misinterpretation of iron studies, inconsistencies in ID diagnostic guidelines, and low awareness of non-anaemic ID may lead to missed diagnoses and opportunities for treatment. Serum ferritin (SF) is the most specific routine test for diagnosing ID, but should be interpreted in the correct context. Low SF levels provide a clearcut diagnosis, whilst normal and raised SF levels in the setting of inflammation require a nuanced diagnostic approach. Limited evidence to support diagnostic SF cut-offs and uncertainty surrounding SF reference intervals represent ongoing challenges. This paper aims to provide clarity on the utility and limitations of various laboratory tests for ID and to demonstrate their interpretation with practical examples.
临床医生需要很好地了解可用的工具来诊断缺铁(ID)。由于铁稳态和并发炎症的动态性影响结果,解释常用的实验室测试可能具有挑战性。对铁研究的误解,ID诊断指南的不一致,以及对非贫血性ID的低认识可能导致错过诊断和治疗机会。血清铁蛋白(SF)是诊断ID最具体的常规检查,但应在正确的背景下解释。低SF水平提供了明确的诊断,而正常和升高的SF水平在炎症设置需要细致的诊断方法。支持诊断性SF截止点的证据有限,SF参考区间的不确定性是目前面临的挑战。本文的目的是提供澄清的效用和限制的各种实验室测试的ID和演示他们的解释与实际的例子。
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引用次数: 0
Bispecific antibodies in multiple myeloma: maximizing potential through rational combination therapies 多发性骨髓瘤的双特异性抗体:通过合理的联合治疗最大化潜力。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/j.blre.2025.101342
Xiang Zhou , Johannes M. Waldschmidt , Hermann Einsele
Bispecific antibodies have emerged as a transformative immunotherapeutic strategy in multiple myeloma (MM). Early constructs such as BiTEs paved the way for full-length IgG-based antibodies with improved pharmacokinetics and safety profiles. As of 2025, teclistamab, elranatamab, linvoseltamab (all BCMA×CD3) and talquetamab (GPRC5D × CD3) have received regulatory approval for relapsed/refractory MM. Investigational agents targeting FcRH5 (cevostamab) and next-generation constructs with altered binding configurations targeting BCMA are under clinical evaluation. Despite high response rates in late-line therapy, resistance via antigen loss, T-cell dysfunction and soluble BCMA presents a major challenge. Combinations with IMiDs, checkpoint inhibitors and dual-targeting approaches are being tested to enhance durability. Frontline studies currently investigate if bispecific antibodies can further deepen responses and induce MRD negativity when used as part of induction therapy. As clinical data matures, bispecific antibodies are likely to redefine MM treatment, by offering an off-the-shelf, scalable alternative to CAR-T therapy with curative potential in selected patient populations.
双特异性抗体已成为多发性骨髓瘤(MM)的一种变革性免疫治疗策略。早期构建如BiTEs为全长igg抗体的开发铺平了道路,并改善了其药代动力学和安全性。截至2025年,teclistamab、elranatamab、linvoseltamab(均为BCMA×CD3)和talquetamab (GPRC5D ×CD3)已获得监管机构批准,用于治疗复发/难治性MM。针对FcRH5 (cevostamab)的研究药物和靶向BCMA的改变结合构型的下一代构建物正在临床评估中。尽管晚期治疗的应答率很高,但抗原丢失、t细胞功能障碍和可溶性BCMA引起的耐药是一个主要挑战。目前正在测试与IMiDs、检查点抑制剂和双靶向方法的联合使用,以提高耐久性。目前,一线研究正在调查双特异性抗体作为诱导治疗的一部分是否能进一步加深反应并诱导MRD阴性。随着临床数据的成熟,双特异性抗体可能会重新定义MM治疗,提供一种现成的、可扩展的CAR-T疗法替代方案,在选定的患者群体中具有治疗潜力。
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引用次数: 0
Tackling myeloma bone disease: From pathophysiology to cutting-edge therapies 治疗骨髓瘤骨病:从病理生理学到尖端疗法。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/j.blre.2025.101305
Sophie Roux , Françoise Debiais , Marie-Hélène Vieillard
Bone involvement in multiple myeloma (MM) is marked by osteolysis, driven by excessive bone resorption and a profound suppression of bone formation. Interactions between MM cells and the bone microenvironment—mediated by integrins,chemokines, and bone marrow stromal cells—play a critical role in the development of myeloma bone disease (MBD). Key players include osteoclasts, osteoblasts, and osteocytes. Osteoclast activation is mainly driven by RANK/RANKL and other pro-osteoclastogenic factors that disrupt bone remodeling, while impaired bone formation involves Wnt signaling inhibition and Runx2/Cbfa1 suppression. Emerging therapeutic strategies are focused on addressing both the tumor burden and the bone remodeling imbalance, with advances in molecular targeting and microRNA-based approaches. Similarly, novel anti-myeloma therapies show promise for MBD, though their full impact is not yet defined. This review highlights recent findings in MM-associated bone disease and discusses current and prospective therapies aimed at improving patient outcomes.
多发性骨髓瘤(MM)的骨受累以骨溶解为特征,由过度的骨吸收和骨形成的严重抑制所驱动。MM细胞与骨微环境之间的相互作用由整合素、趋化因子和骨髓基质细胞介导,在骨髓瘤骨病(MBD)的发展中起着关键作用。主要参与者包括破骨细胞、成骨细胞和骨细胞。破骨细胞激活主要由RANK/RANKL等破坏骨重塑的促破骨因子驱动,而骨形成受损涉及Wnt信号抑制和Runx2/Cbfa1抑制。随着分子靶向和基于微rna的方法的进展,新兴的治疗策略集中在解决肿瘤负担和骨重塑失衡上。类似地,新的抗骨髓瘤疗法显示出治疗MBD的希望,尽管它们的全部影响尚未确定。这篇综述强调了mm相关骨病的最新发现,并讨论了旨在改善患者预后的当前和未来治疗方法。
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引用次数: 0
Caplacizumab for pediatric immune thrombotic thrombocytopenic Purpura: A scoping review of current evidence 卡普拉珠单抗治疗儿童免疫性血栓性血小板减少性紫癜:当前证据的范围审查。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-11-01 DOI: 10.1016/j.blre.2025.101326
Abdulrahman F. Al-Mashdali , Samah M. Salih , Priyadarshini Asmita Vatsyayan , Deena Mudawi , Sarah A. Elkourashy , Honar Cherif , Shehab F. Mohamed
While caplacizumab, a von Willebrand factor inhibitor, has dramatically improved iTTP management in adults, its role in pediatric iTTP remains understudied. This scoping review synthesizes current evidence on the safety and efficacy of caplacizumab in children with iTTP.
We conducted a systematic literature search (PubMed, Scopus) up to March 2025, identifying 15 studies (36 patients) meeting inclusion criteria. The cohort (median age 14 years, range 1–17) exhibited female predominance (72 %) and diverse ethnic backgrounds. Neurological symptoms (53 %) and bleeding manifestations (petechiae, purpura, or ecchymoses in 74 %) were common presentations. Caplacizumab was administered at 5 mg (17 %), 10 mg (75 %), or 11 mg (8 %), alongside plasma exchange (22 % received >10 sessions) and immunosuppression (97 % corticosteroids, 92 % rituximab).
Key outcomes included rapid platelet recovery (median 3.5 days post-caplacizumab initiation) and relatively short hospitalization (median 14 days). Adverse events were uncommon (11 % minor bleeding), with no major or life-threatening hemorrhagic complications reported. Mortality was 0 %, with all patients surviving to follow-up. During a median follow-up period of 6–12 months, 94 % of patients remained relapse-free. Notably, 22 % of patients were under 12 years of age, underscoring the off-label use of caplacizumab in younger children.
These findings suggest caplacizumab is effective and safe in pediatric iTTP, mirroring outcomes observed in adults while demonstrating a favorable bleeding profile. However, variability in dosing and limited data on long-term effects highlight the need for pediatric-specific guidelines and prospective studies. This review supports caplacizumab as a promising adjunct in childhood iTTP but calls for further research to optimize its use in this vulnerable population.
虽然卡普拉珠单抗(一种血管性血血病因子抑制剂)已显著改善成人iTTP的治疗,但其在儿童iTTP中的作用仍未得到充分研究。本综述综合了目前关于卡普拉珠单抗治疗iTTP患儿安全性和有效性的证据。我们进行了截至2025年3月的系统文献检索(PubMed, Scopus),确定了15项研究(36例患者)符合纳入标准。该队列(中位年龄14岁,范围1-17岁)表现出女性优势(72%)和多样化的种族背景。神经系统症状(53%)和出血表现(积点、紫癜或瘀斑74%)是常见的症状。Caplacizumab给予5mg (17%), 10mg(75%)或11mg(8%),同时血浆置换(22%接受bbb10疗程)和免疫抑制(97%皮质类固醇,92%美罗华)。主要结局包括血小板快速恢复(卡普拉珠单抗启动后中位3.5天)和相对较短的住院时间(中位14天)。不良事件不常见(11%轻微出血),无重大或危及生命的出血并发症报道。死亡率为0%,所有患者均存活至随访。在6-12个月的中位随访期间,94%的患者保持无复发。值得注意的是,22%的患者年龄在12岁以下,这强调了卡普拉珠单抗在年幼儿童中的标签外使用。这些研究结果表明,caplacizumab在儿童iTTP中是有效和安全的,与在成人中观察到的结果一致,同时显示出良好的出血情况。然而,剂量的可变性和有限的长期影响数据突出了儿科特定指南和前瞻性研究的必要性。本综述支持卡普拉珠单抗作为儿童iTTP的一种有前景的辅助药物,但需要进一步研究以优化其在这一易感人群中的应用。
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引用次数: 0
The microbiome-lymphoma Axis: A systematic review and Meta-analysis of gut Dysbiosis pattern in diffuse large B-cell lymphoma. 微生物组-淋巴瘤轴:弥漫性大b细胞淋巴瘤肠道生态失调模式的系统回顾和荟萃分析。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-10-30 DOI: 10.1016/j.blre.2025.101341
Sarah A Elkourashy, Rasha Abu-El-Ruz, Medhat Z Askar, Ahmad Hamdan, Susu M Zughaier

Background: Gut microbiota has emerged as a critical mediator of immune homeostasis and cancer biology. Increasing evidence suggests that gut dysbiosis may play a significant role in the pathogenesis of diffuse large B-cell lymphoma (DLBCL), the most common and aggressive subtype of non-Hodgkin lymphoma (NHL). However, a comprehensive synthesis of the microbial alterations associated with DLBCL remains poorly defined.

Methods: We systematically reviewed and meta-analyzed thirteen studies (n = 4087 cases) to assess gut microbiota alterations in DLBCL. Both observational and Mendelian randomization designs were included. Pooled odds ratios (OR) were calculated for mendelian randomization studies using random-effects models, and microbial shifts were analyzed at the species level to contextualize biological relevance. Observational studies were used for qualitative assessment.

Results: Meta analysis was done for the mendelian randomization studies (n = 3737 cases). The overall pooled OR was 0.96 (95 % CI: 0.93-1.00), suggested a slight shift towards depletion, with considerable heterogeneity (I2 = 78.7 %). Species-level analysis revealed significant enrichment of pro-inflammatory or potentially oncogenic taxa in DLBCL, including Bilophila (OR = 1.78), Desulfovibrionaceae (OR = 1.58), and Coprobacter (OR = 1.37). Conversely, beneficial commensals with anti-inflammatory and metabolic regulatory roles such as Eubacterium coprostanoligenes group (OR = 0.19), Alistipes (OR = 0.57), Ruminococcaceae UCG011 (OR = 0.75) were significantly depleted.

Conclusions: This first comprehensive synthesis demonstrates a reproducible species-level microbial signature in DLBCL, characterized by depletion of protective commensals and enrichment of pro-tumorigenic taxa. Mechanistically, these shifts may promote lymphomagenesis via inflammatory, metabolic, and immune-modulatory pathways. Our findings highlight the gut microbiota as a latent biomarker source and therapeutic target, supporting microbiota-modulating strategies in precision lymphoma care.

背景:肠道微生物群已成为免疫稳态和癌症生物学的重要媒介。越来越多的证据表明,肠道生态失调可能在弥漫性大b细胞淋巴瘤(DLBCL)的发病机制中发挥重要作用,DLBCL是非霍奇金淋巴瘤(NHL)中最常见和最具侵袭性的亚型。然而,与DLBCL相关的微生物改变的综合合成仍然没有明确的定义。方法:我们系统地回顾和荟萃分析了13项研究(n = 4087例),以评估DLBCL患者肠道微生物群的改变。包括观察和孟德尔随机化设计。使用随机效应模型计算孟德尔随机化研究的合并优势比(OR),并在物种水平上分析微生物的变化,以确定生物学相关性。观察性研究用于定性评估。结果:对孟德尔随机化研究(n = 3737例)进行Meta分析。总体合并OR为0.96 (95% CI: 0.93-1.00),表明有轻微的向枯竭转移,具有相当大的异质性(I2 = 78.7%)。物种水平分析显示,DLBCL中促炎或潜在致癌分类群显著富集,包括Bilophila (or = 1.78)、Desulfovibrionaceae (or = 1.58)和Coprobacter (or = 1.37)。相反,具有抗炎和代谢调节作用的有益共生菌如coprostanoligenes菌群(OR = 0.19)、Alistipes菌群(OR = 0.57)、Ruminococcaceae菌群UCG011菌群(OR = 0.75)显著减少。结论:这是第一次全面的合成,证明了DLBCL中可复制的物种水平微生物特征,其特征是保护性共栖物的消耗和促肿瘤分类群的富集。从机制上讲,这些转变可能通过炎症、代谢和免疫调节途径促进淋巴瘤的发生。我们的研究结果强调肠道微生物群作为潜在的生物标志物来源和治疗靶点,支持精确治疗淋巴瘤的微生物群调节策略。
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引用次数: 0
Transplant-acquired allergy in HCT-recipients: Reference for clinical management hct受者移植获得性过敏:临床管理参考。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-08-01 DOI: 10.1016/j.blre.2025.101289
Akihiro Ohmoto , Yoshiyuki Yamada , Shigeo Fuji
Transplant-acquired allergy (TAA) is well known in pediatric patients undergoing liver transplantation. Regarding allogeneic hematopoietic cell transplantation (allo-HCT), the clinical characteristics of TAA have not been fully elucidated. Clinical manifestations of TAA include eczematous dermatitis, allergic rhinitis, and asthma. It is known that allergic diseases are transferable from allergic donors to non-allergic recipients via allo-HCT. The potential mechanism is the transfer of allergen-specific memory B cells resulting in Th2-skewed allergy-specific immune responses. Retrospective studies have suggested that cord blood transplantation (CBT) and the immunosuppressant tacrolimus have a significant impact on the development of TAA. The reported prevalence varies according to study design, diagnostic methods, and study population, and the suspected allergens vary widely among individuals. Prospective cohort studies and further mechanistic investigations are warranted to provide robust evidence on the prevalence and risk factors associated with TAA.
移植获得性过敏(TAA)在儿童肝移植患者中是众所周知的。关于同种异体造血细胞移植(allogeneic hematopoietic cell transplantation, allo-HCT), TAA的临床特征尚未完全阐明。TAA的临床表现包括湿疹性皮炎、变应性鼻炎和哮喘。众所周知,变应性疾病可通过同种异体hct从过敏供体转移到非过敏受体。潜在的机制是过敏原特异性记忆B细胞的转移导致th2偏斜的过敏原特异性免疫反应。回顾性研究表明,脐带血移植(CBT)和免疫抑制剂他克莫司对TAA的发展有显著影响。报告的患病率因研究设计、诊断方法和研究人群而异,疑似过敏原在个体之间差异很大。前瞻性队列研究和进一步的机制调查有必要为TAA的患病率和危险因素提供强有力的证据。
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引用次数: 0
The new era of primary immune thrombocytopenia management in adults: A narrative review of current and emerging treatments 成人原发性免疫性血小板减少症管理的新时代:对当前和新兴治疗方法的叙述回顾。
IF 5.7 2区 医学 Q1 HEMATOLOGY Pub Date : 2025-08-01 DOI: 10.1016/j.blre.2025.101300
Tomás José González-López , Drew Provan
The purpose of this review is to highlight the treatments currently available and those under- going evaluation in clinical trials for the treatment of ITP in order to achieve optimal use of the various existing ITP treatments. Specifically, we point out the indications for use of the various therapies available: corticosteroids, intravenous immunoglobulins (IVIG), thrombo- poietic agents (TPO-RAs), Syk inhibitors: Fostamatinib, antiCD20 monoclonal antibodies i.e. rituximab and the use of splenectomy in ITP. A review of the use of new drugs in ITP is also included in our manuscript: Neonatal Fc receptor (FcRn) antagonists; Bruton tyrosine kinase (BTK) inhibition; B-cell activating factor (BAFF) pathway inhibition; plasma cell depletion (an- tiCD38 monoclonal antibodies); new Syk inhibitors and complement inhibition. We believe that a reader with little knowledge of ITP can gain a clear understanding of the current treatment of ITP and its more or less immediate treatment prospects.
本综述的目的是强调目前可用的治疗方法和正在临床试验中评估的治疗ITP的方法,以实现各种现有ITP治疗方法的最佳使用。具体来说,我们指出了使用各种可用疗法的适应症:皮质类固醇,静脉注射免疫球蛋白(IVIG),血栓生成剂(TPO-RAs), Syk抑制剂:Fostamatinib, antid20单克隆抗体,即利妥昔单抗,以及在ITP中使用脾切除术。我们的手稿中还包括对ITP中新药使用的回顾:新生儿Fc受体(FcRn)拮抗剂;布鲁顿酪氨酸激酶抑制;b细胞活化因子(BAFF)通路抑制;血浆细胞耗竭(an- tiCD38单克隆抗体);新的Syk抑制剂和补体抑制。我们相信,一个对ITP知之甚少的读者可以清楚地了解ITP的目前治疗方法及其或多或少的直接治疗前景。
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引用次数: 0
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Blood Reviews
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