British Journal of Haematology最新文献_第9页

Distinct subtypes of post-transplant lymphoproliferative disorders: CHIP-like mutations in early lesions and substantial mutational differences between EBV-positive and EBV-negative diffuse large B-cell lymphomas 移植后淋巴增生性疾病的不同亚型：早期病变中的chip样突变以及ebv阳性和ebv阴性弥漫性大b细胞淋巴瘤之间的实质性突变差异。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-07 DOI: 10.1111/bjh.19952

Vanesa-Sindi Ivanova, Thomas Menter, Ningxuan Cui, Peter Leary, Carl Zinner, Jörg P. Halter, Frank Stenner, Stefan Dirnhofer, Anne Müller, Alexandar Tzankov

{"title":"Distinct subtypes of post-transplant lymphoproliferative disorders: CHIP-like mutations in early lesions and substantial mutational differences between EBV-positive and EBV-negative diffuse large B-cell lymphomas","authors":"Vanesa-Sindi Ivanova, Thomas Menter, Ningxuan Cui, Peter Leary, Carl Zinner, Jörg P. Halter, Frank Stenner, Stefan Dirnhofer, Anne Müller, Alexandar Tzankov","doi":"10.1111/bjh.19952","DOIUrl":"10.1111/bjh.19952","url":null,"abstract":"<div>\u0000 \u0000 Post-transplant lymphoproliferative disorders (PTLD) and lymphomas in immunocompromised individuals represent significant clinical challenges, with a limited understanding of their pathogenesis. We investigated a PTLD cohort (n = 50) consisting of ‘early lesions’ (infectious mononucleosis-like PTLD, plasmacytic and follicular hyperplasias), polymorphic PTLD and post-transplant diffuse large B-cell lymphomas (PT-DLBCL). The study also included 15 DLBCL with autoimmune/immunocompromised backgrounds (IS-DLBCL) and 14 DLBCL, not otherwise specified (DLBCL, NOS), as control. To investigate microarchitectural and genetic changes, immunohistochemistry, multiplex immunofluorescence (mIF), fluorescence in situ hybridisation and high-throughput sequencing were performed. Scarcity of viral infections other than Epstein–Barr virus (EBV) was observed. mIF revealed lower Treg infiltration in PT-DLBCL and high CD8+/PD1+ T cells in IS-DLBCL. MYC rearrangements were most common in PT-DLBCL, followed by IS-DLBCL and DLBCL, NOS, all EBV-negative. TP53 mutations were frequent in EBV-negative PT-DLBCL and DLBCL, NOS but absent in ‘early lesions’. NOTCH1 mutations were predominant in PT-DLBCL (N1 DLBCL-subgroup). Gene expression profiling showed a significant overlap between ‘early lesions’ and polymorphic PTLD. The presence of clonal haematopoiesis of indeterminate potential (CHIP)-like mutations and the absence of immune-escape gene mutations in ‘early lesions’ suggest these disorders may represent clonal expansions driven by exogenic immunosuppression and/or EBV infection ‘substituting’ for mutations of the latter group of genes.\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 2","pages":"484-504"},"PeriodicalIF":5.1,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142941825","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

MicroRNA-199a-5p may be a diagnostic biomarker of primary ITP. MicroRNA-199a-5p可能是原发性ITP的诊断性生物标志物。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-07 DOI: 10.1111/bjh.19987

Lamya Garabet, Anbjørg Rangberg, Anna Maria Eriksson, Christine Monceyron Jonassen, Raul Teruel-Montoya, Maria Luisa Lozano, Constantino Martinez, Heidi Hassel Pettersen, Åse-Berit Mathisen, Eirik Tjønnfjord, Hoa Tran, Ellen Brodin, Galina Tsykunova, Johanna Gebhart, James Bussel, Waleed Ghanima

{"title":"MicroRNA-199a-5p may be a diagnostic biomarker of primary ITP.","authors":"Lamya Garabet, Anbjørg Rangberg, Anna Maria Eriksson, Christine Monceyron Jonassen, Raul Teruel-Montoya, Maria Luisa Lozano, Constantino Martinez, Heidi Hassel Pettersen, Åse-Berit Mathisen, Eirik Tjønnfjord, Hoa Tran, Ellen Brodin, Galina Tsykunova, Johanna Gebhart, James Bussel, Waleed Ghanima","doi":"10.1111/bjh.19987","DOIUrl":"https://doi.org/10.1111/bjh.19987","url":null,"abstract":"There is no diagnostic test for primary immune thrombocytopenia (ITP). Certain microRNAs have shown to have diagnostic potential in ITP. We validated 12 microRNAs identified from two previous studies to find a diagnostic biomarker. The study included two ITP cohorts (n = 61) and healthy controls (n = 28). The first ITP cohort involved 24 patients from the Prolong study, patients with newly diagnosed/persistent ITP (<1 year) treated with corticosteroids ± IVIG but relapsed/failed to respond. The second cohort comprised 37 patients from ITP biobank, Østfold Hospital, Norway, patients had different disease stages and therapies. Twelve microRNAs were measured: miR-199a-5p, miR-33a-5p, miR-195-5p, miR-130a-3p, miR-144-3p, miR-146a-5p, miR-222-3p, miR-374b-5p, miR-486-5p, miR-1341-5p, miR-766-3p and miR-409-3p. miR-199a-5p, miR-33a-5p, miR-374b-5p, miR-146a-5p and miR-409-3p were expressed differentially in the entire ITP cohort compared to controls; of those only miR-199a-5p showed good discriminative ability between ITP and controls with area under the curve (AUC) of 0.718 (95% CI: 0.599-0.836). In the Prolong cohort (ITP < 1 year), miR-199a-5p and miR-374b-5p showed very good discriminative ability between ITP and controls with AUC of 0.824 (0.708-0.940) and 0.806 (0.688-0.924) respectively. This study confirmed that miR-199a-5p has good discriminative ability between primary ITP and healthy controls, thus may be a diagnostic biomarker of ITP.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142941827","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Diagnosis and management of monoclonal gammopathy of renal significance: A British Society for Haematology good practice paper 肾意义单克隆伽玛病的诊断和管理：英国血液学学会良好实践论文。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-07 DOI: 10.1111/bjh.19956

Jennifer Pinney, Candice Roufosse, Andreas Kousios, Aristeidis Chaidos, Julian D. Gillmore, Francesco Rainone, Satarupa Choudhuri, Karthik Ramasamy, Sarah Blakey, John Ashcroft, Y. L. Tracey Chan, Paul Cockwell, Guy Pratt, the BSH Committee

引用次数: 0

Venetoclax therapy in chronic lymphocytic leukaemia patients relapsed after allogeneic haematopoietic stem cell transplantation 异基因造血干细胞移植后复发的慢性淋巴细胞白血病患者的Venetoclax治疗。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-07 DOI: 10.1111/bjh.19976

Francesca Perutelli, Elia Boccellato, Maria Chiara Montalbano, Gioacchino Catania, Marina Deodato, Anna Maria Frustaci, Idanna Innocenti, Riccardo Moia, Francesca Maria Quaglia, Giulia Quaresmini, Paolo Rivela, Gianluca Gaidano, Mauro Krampera, Luca Laurenti, Alessandro Rambaldi, Benedetto Bruno, Candida Vitale, Marta Coscia

{"title":"Venetoclax therapy in chronic lymphocytic leukaemia patients relapsed after allogeneic haematopoietic stem cell transplantation","authors":"Francesca Perutelli, Elia Boccellato, Maria Chiara Montalbano, Gioacchino Catania, Marina Deodato, Anna Maria Frustaci, Idanna Innocenti, Riccardo Moia, Francesca Maria Quaglia, Giulia Quaresmini, Paolo Rivela, Gianluca Gaidano, Mauro Krampera, Luca Laurenti, Alessandro Rambaldi, Benedetto Bruno, Candida Vitale, Marta Coscia","doi":"10.1111/bjh.19976","DOIUrl":"10.1111/bjh.19976","url":null,"abstract":"Allogeneic hematopoietic stem cell transplantation (alloHSCT) remains an option for young and fit chronic lymphocytic leukaemia (CLL) patients with high-risk disease features. However, allotransplanted patients are generally excluded from clinical trials, making data regarding the use of venetoclax after alloHSCT extremely rare. We report data from 7 CLL patients who received venetoclax after alloHSCT among 53 Italian centers. These patients underwent alloHSCT between 2006 and 2021 after failing chemoimmunotherapy (7/7), ibrutinib (5/7) and/or idelalisib (1/7). Of note, 3/7 patients had already received venetoclax-based therapy before alloHSCT. Post-allo HSCT venetoclax treatment resulted safe, with adverse events not different from what reported in clinical trials. Importantly, no meaningful impact on graft versus host disease (GvHD) course was observed: 4/7 patients with pre-existing chronic GvHD had no exacerbation after venetoclax start, and only one patient developed GvHD during venetoclax therapy, that was managed as per standard clinical practice. Concerning efficacy, 5/7 patients presented a clinical response to venetoclax, with two patients achieving an undetectable minimal residual disease. To our knowledge, this is the largest reported series of CLL patients treated with venetoclax after alloHSCT. In these heavily pretreated and high-risk patients, previous alloHSCT did not compromise the feasibility of venetoclax therapy, that lacked unexpected toxicities and did not exacerbate GvHD.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"924-929"},"PeriodicalIF":5.1,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19976","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142941830","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Increased relative eosinophil counts portend neck oedema after chimeric antigen receptor-T therapy 嗜酸性粒细胞相对计数增加预示着嵌合抗原受体-T疗法后颈部水肿。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-06 DOI: 10.1111/bjh.19992

Naokazu Nakamura, Tomoyasu Jo, Yasuyuki Arai, Toshio Kitawaki, Momoko Nishikori, Chisaki Mizumoto, Junya Kanda, Kouhei Yamashita, Miki Nagao, Akifumi Takaori-Kondo

引用次数: 0

Management strategies for patients with chronic lymphocytic leukaemia harbouring complex karyotype

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-06 DOI: 10.1111/bjh.19986

Andrea Serafin, Valeria Ruocco, Alessandro Cellini, Francesco Angotzi, Laura Bonaldi, Livio Trentin, Andrea Visentin

引用次数: 0

An approach to Hemequity: Identifying the barriers and facilitators of iron deficiency reduction strategies in low- to middle-income countries

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-06 DOI: 10.1111/bjh.19984

Shiliang Ge, Saif Ali, Victoria Haldane, Carine Bekdache, Grace H. Tang, Michelle Sholzberg

{"title":"An approach to Hemequity: Identifying the barriers and facilitators of iron deficiency reduction strategies in low- to middle-income countries","authors":"Shiliang Ge, Saif Ali, Victoria Haldane, Carine Bekdache, Grace H. Tang, Michelle Sholzberg","doi":"10.1111/bjh.19984","DOIUrl":"10.1111/bjh.19984","url":null,"abstract":"Approximately 1.92 billion people worldwide are anaemic, and iron deficiency is the most common cause. Iron deficiency anaemia (IDA) disproportionately affects women of reproductive age and remains under-addressed in low- to middle-income countries (LMICs). The primary objective of our scoping review is to evaluate the barriers and facilitators to IDA management in LMICs by using an intersectionality-enhanced implementation science lens adapted from the consolidated framework for implementation research and the theoretical domains framework. A total of 53 studies were identified. Contextual barriers included the deprioritization of IDA risk, unequal gender norms and stigma from the HIV/AIDS epidemic. Regional poverty, conflict and natural disasters led to supply chain barriers. Individual-level facilitators included partner support and antenatal care access while barriers included forgetfulness and having medical comorbidities. Successful interventions also utilized education initiatives to empower women in community decision-making. Moreover, community mobilization and the degree of community ownership determined the sustainability of IDA reduction strategies. IDA is not only a medical problem, but one that is rooted in the sociocultural and political context. Future approaches must recognize the resilience of LMIC communities and acknowledge the importance of knowledge translation rooted in community ownership and empowerment.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 2","pages":"428-442"},"PeriodicalIF":5.1,"publicationDate":"2025-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19984","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143416886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

mRNA-laden lipid nanoparticle-enabled humanized CD19 CAR-T-cell engineering for the eradication of leukaemic cells

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-06 DOI: 10.1111/bjh.19988

Zhaozhao Chen, Anqi Ren, Yingying Li, Jinhui Shu, Jianghua Wu, Hekuan Huang, Jingming Wang, Yu Hu, Heng Mei

{"title":"mRNA-laden lipid nanoparticle-enabled humanized CD19 CAR-T-cell engineering for the eradication of leukaemic cells","authors":"Zhaozhao Chen, Anqi Ren, Yingying Li, Jinhui Shu, Jianghua Wu, Hekuan Huang, Jingming Wang, Yu Hu, Heng Mei","doi":"10.1111/bjh.19988","DOIUrl":"10.1111/bjh.19988","url":null,"abstract":"Chimeric antigen receptor T-cell (CAR-T) therapy has shown transformative potential in treating malignant tumours, with increasing global approval of CAR-T products. However, high-production costs and risks associated with viral vector-based CAR-T cells—such as insertional mutagenesis and secondary tumour formation—remain challenges. Our study introduces an innovative CAR-T engineering approach using mRNA delivered via lipid nanoparticles (LNPs), aiming to reduce costs and enhance safety while maintaining strong anti-tumour efficacy. We developed an LNP-based transfection protocol for efficient delivery of mRNA encoding full-human CAR constructs, achieving high CAR expression and significant cytotoxicity against leukaemic cells in vitro. Co-culture with Raji cells showed increased cytokine secretion and tumour cell killing by mRNA-LNP CAR-T cells. Therapeutic efficacy was further demonstrated in an NOD-scid-IL2Rγnull (NSG) mouse model with Raji engraftment, where treated mice exhibited marked tumour regression and extended survival. These findings underscore the potential of mRNA-LNPs as a non-viral, effective CAR-T engineering platform, offering a promising alternative to traditional methods that could improve CAR-T safety, efficacy and accessibility in clinical cancer immunotherapy.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 2","pages":"628-643"},"PeriodicalIF":5.1,"publicationDate":"2025-01-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19988","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143416902","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Biochemical screening of glucose-6-phosphate dehydrogenase deficiency in borderline cases: Complementary inputs of standardization enzymes and comparison with genetic status

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-06 DOI: 10.1111/bjh.19990

Alexandre Raynor, Basile Jacquel, Stanislas François, Soraya Fellahi, Nadir Mouri, Claire Berquet, Pablo Bartolucci, Frédéric Galactéros, Marc Conti, Sylvain Loric, Jean-Philippe Bastard, Benoît Funalot, Michel Bahuau, Stéphane Moutereau

引用次数: 0

Long-term efficacy and safety of avatrombopag in Chinese children with primary immune thrombocytopenia: A real-world observational study 阿伐波帕治疗原发性免疫性血小板减少症儿童的长期疗效和安全性：一项真实世界的观察性研究。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-05 DOI: 10.1111/bjh.19973

Nan Wang, Zhifa Wang, Jingjing Liu, Yu Hu, Shuyue Dong, Hui Chen, Jinxi Meng, Jingyao Ma, Zhenping Chen, Xiaoling Cheng, Runhui Wu

{"title":"Long-term efficacy and safety of avatrombopag in Chinese children with primary immune thrombocytopenia: A real-world observational study","authors":"Nan Wang, Zhifa Wang, Jingjing Liu, Yu Hu, Shuyue Dong, Hui Chen, Jinxi Meng, Jingyao Ma, Zhenping Chen, Xiaoling Cheng, Runhui Wu","doi":"10.1111/bjh.19973","DOIUrl":"10.1111/bjh.19973","url":null,"abstract":"<div>\u0000 \u0000 Avatrombopag is a newly approved thrombopoietin receptor agonist for second-line treatment of chronic immune thrombocytopenia (ITP) in adults. Our previous study showed its efficacy and safety in a small sample of paediatric ITP patients. However, large samples and long-term data are still lacking. Children diagnosed with ITP and treated with avatrombopag for at least 4 weeks were enrolled. In 94 ITP patients with a median age of 7.43 (interquartile range (IQR), 4.82, 10.80) years, the median effective dose was 10 (IQR, 10, 20) mg for children under 6 years old and 20 (IQR, 20, 40) mg for children under 18 years old. The overall response was achieved in 72.3% (68/94) and 73.4% (58/79) of patients within 4 weeks and 12 weeks. The sustained response at 24 weeks and 48 weeks were 62.3% (33/53) and 51.6% (16/31) respectively. The occurrence of bleeding events, rescue therapy and concomitant ITP medication decreased during the follow-up period. For safety, thrombocytosis (platelet count ≥400 × 109/L) was the most frequent adverse event (AE) observed in 44 children 97 times. Long-term treatment with avatrombopag in ITP children showed a rapid and sustained platelet response and good bleeding control without significant or new AEs.\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"935-943"},"PeriodicalIF":5.1,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142930097","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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