British Journal of Haematology最新文献_第3页

Measurable residual disease-driven treatment in first-line chronic lymphocytic leukaemia. 一线慢性淋巴细胞白血病的可测量残留疾病治疗。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-13 DOI: 10.1111/bjh.19902

M S Davids, K H Lin, A I Mohamed, T Munir, T A Eyre

引用次数: 0

Predicting therapy-related myeloid neoplasms after CAR-T with the Clonal Haematopoiesis Risk Score (CHRS). 用克隆性造血风险评分（CHRS）预测 CAR-T 治疗后与治疗相关的髓系肿瘤。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-13 DOI: 10.1111/bjh.19905

Eugenio Galli, Monica Rossi, Ilaria Pansini, Marcello Viscovo, Tanja Malara, Maria Colangelo, Eleonora Alma, Caterina Giovanna Valentini, Luciana Teofili, Stefan Hohaus, Simona Sica, Federica Sorà, Patrizia Chiusolo

{"title":"Predicting therapy-related myeloid neoplasms after CAR-T with the Clonal Haematopoiesis Risk Score (CHRS).","authors":"Eugenio Galli, Monica Rossi, Ilaria Pansini, Marcello Viscovo, Tanja Malara, Maria Colangelo, Eleonora Alma, Caterina Giovanna Valentini, Luciana Teofili, Stefan Hohaus, Simona Sica, Federica Sorà, Patrizia Chiusolo","doi":"10.1111/bjh.19905","DOIUrl":"https://doi.org/10.1111/bjh.19905","url":null,"abstract":"The clonal haematopoiesis risk score (CHRS) was proposed to predict the rate of progression from clonal haemopoiesis of indeterminate potential (CHIP)/clonal cytopenia with unknown significance (CCUS) to myeloid neoplasms in the general population. CHRS encompasses the type and VAF of the mutation, the presence of a single DNMT3A mutation, cytopenia, age, red cell distribution width (RDW) and mean corpuscular volume (MCV). We studied clonal haematopoiesis in a cohort of 55 consecutive patients treated with CD19-directed CAR-T cells: CHIP and CCUS were present in 7% and 33% of patients before CAR-T. Three therapy-related myeloid neoplasms (t-MN) were observed after treatment with CAR-T (2 MDS and 1 AML). Only patients with an intermediate-high baseline CHRS developed a t-MN. Patients with an intermediate-high CHRS had more than a twofold increased risk of developing a t-MN within the first 9 months after CAR-T (odds ratio 2.89, 95% C.I. 1.98-4.19, p < 0.001). Overall, CHRS was able to predict the occurrence of t-MN after CAR-T with good specificity.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613345","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

Methodology and clinical utility of longitudinal UBA1 tracking in VEXAS syndrome. 纵向 UBA1 追踪 VEXAS 综合征的方法和临床实用性。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-13 DOI: 10.1111/bjh.19897

Carmelo Gurnari, Elisa Galossi, Eleonora Lumia, Alfonso Piciocchi, Mariadomenica Divona, Elisa Casciani, Francesca Romano, Elisa Diral, Alessandro Tomelleri, Federico Caroni, Antonio Vitale, Gregorio Maria Bergonzi, Annalisa Condorelli, Giorgia Battipaglia, Erika Morsia, Elena Crisà, Paola Triggianese, Arianna Savi, Chiara Cardamone, Matteo Dragani, Giulia Rivoli, Federica Pilo, Davide Firinu, Sara Plebani, Francesco D'Agostino, Alessandro D'Ambrosio, Katja Sockel, Cristina Papayannidis, Silvia Salmoiraghi, Fabrizio Pane, Monica Bocchia, Luca Cantarini, Marco Frigeni, Corrado Campochiaro, Lorenzo Dagna, Raffaella Greco, Fabio Ciceri, Orietta Spinelli, Christian Thiede, Maria Teresa Voso

{"title":"Methodology and clinical utility of longitudinal UBA1 tracking in VEXAS syndrome.","authors":"Carmelo Gurnari, Elisa Galossi, Eleonora Lumia, Alfonso Piciocchi, Mariadomenica Divona, Elisa Casciani, Francesca Romano, Elisa Diral, Alessandro Tomelleri, Federico Caroni, Antonio Vitale, Gregorio Maria Bergonzi, Annalisa Condorelli, Giorgia Battipaglia, Erika Morsia, Elena Crisà, Paola Triggianese, Arianna Savi, Chiara Cardamone, Matteo Dragani, Giulia Rivoli, Federica Pilo, Davide Firinu, Sara Plebani, Francesco D'Agostino, Alessandro D'Ambrosio, Katja Sockel, Cristina Papayannidis, Silvia Salmoiraghi, Fabrizio Pane, Monica Bocchia, Luca Cantarini, Marco Frigeni, Corrado Campochiaro, Lorenzo Dagna, Raffaella Greco, Fabio Ciceri, Orietta Spinelli, Christian Thiede, Maria Teresa Voso","doi":"10.1111/bjh.19897","DOIUrl":"https://doi.org/10.1111/bjh.19897","url":null,"abstract":"Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) is a haemato-inflammatory syndrome genetically defined by somatic mutations in the X-linked UBA1 gene, typically Val/Thr/Leu substitutions at the Met41 hotspot. Clinical manifestations are heterogeneous and refractory to most haemato-rheumatological treatments. To date, no guidelines exist for the management of VEXAS, and scarce is the evidence on methodology and clinical significance of longitudinal UBA1 clonal burden evaluation upon therapy. Here, we validated a method to quantify UBA1 clonal burden and explored its applicability in patients with VEXAS. Given the different treatment interactions, droplet digital polymerase chain reaction (ddPCR) may allow for informed therapeutic decisions and implementation of personalized strategies.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613293","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

A case of acquired factor XI deficiency and severe bleeding tendency associated with Streptococcus pyogenes cellulitis. 一例与化脓性链球菌蜂窝组织炎相关的获得性 XI 因子缺乏症和严重出血倾向病例。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-13 DOI: 10.1111/bjh.19908

Nancy El Beayni, Riitta Lassila, Anna-Elina Lehtinen, Mirka Sivula, Timea Szanto

引用次数: 0

Pembrolizumab as salvage treatment for T-cell/histiocyte-rich and Epstein-Barr virus-positive large B-cell lymphoma. 将 Pembrolizumab 作为富含 T 细胞/组织细胞和 Epstein-Barr 病毒阳性大 B 细胞淋巴瘤的挽救治疗。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-13 DOI: 10.1111/bjh.19883

A Schena, F M Quaglia, A Parisi, I Ferrarini, A Moioli, E Tagliavini, A Bernardelli, C Visco

引用次数: 0

Red cell specifications for blood group matching in patients with haemoglobinopathies: An updated systematic review and clinical practice guideline from the International Collaboration for Transfusion Medicine Guidelines. 血红蛋白病患者血型配型的红细胞规格：来自国际输血医学指南合作组织的最新系统综述和临床实践指南。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-13 DOI: 10.1111/bjh.19837

Julia Wolf, Isabelle Blais-Normandin, Aarti Bathla, Homa Keshavarz, Stella T Chou, Arwa Z Al-Riyami, Cassandra D Josephson, Edwin Massey, Heather A Hume, Jacob Pendergrast, Gregory Denomme, Rada M Grubovic Rastvorceva, Sara Trompeter, Simon J Stanworth

{"title":"Red cell specifications for blood group matching in patients with haemoglobinopathies: An updated systematic review and clinical practice guideline from the International Collaboration for Transfusion Medicine Guidelines.","authors":"Julia Wolf, Isabelle Blais-Normandin, Aarti Bathla, Homa Keshavarz, Stella T Chou, Arwa Z Al-Riyami, Cassandra D Josephson, Edwin Massey, Heather A Hume, Jacob Pendergrast, Gregory Denomme, Rada M Grubovic Rastvorceva, Sara Trompeter, Simon J Stanworth","doi":"10.1111/bjh.19837","DOIUrl":"https://doi.org/10.1111/bjh.19837","url":null,"abstract":"Red blood cell (RBC) antigen matching beyond ABO and RhD is commonly recommended for patients with sickle cell disease (SCD) and thalassaemia. We present an updated systematic literature review to inform evidence-based guidelines on RBC matching. The Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) tool was used to develop recommendations. Six new observational studies (4 prospective, 2 retrospective) were identified. The six studies reported on 583 patients in total, including cross-over designs, with sample sizes from 10 to 343. Studies were heterogeneous, utilising varying degrees of RBC matching and different definitions for 'extended' matching. All reported on alloimmunisation. One study reported on molecular matching. The reported prevalence of alloimmunisation using limited matching was 0%-50% and with extended matching was 0%-24%. Eighty-two patients were alloimmunised before study entry. The risk of bias across studies was moderate to critical. The guideline panel recommends that ABO, RhDCcEe, and K-compatible RBCs are selected for individuals with SCD and thalassaemia, even in the absence of alloantibodies, and that RBCs which are antigen-negative to already existing clinically significant antibodies are chosen. There is a need for comparative research to define the benefit, impact, cost-effectiveness, and feasibility of extended RBC matching strategies to prevent alloimmunisation.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613346","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

Minimal residual disease detection for acute lymphoblastic leukaemia in peripheral blood-Are we there yet? 外周血中急性淋巴细胞白血病最小残留病检测--我们做到了吗？

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-11 DOI: 10.1111/bjh.19888

Jan Trka, Eva Fronkova

引用次数: 0

Long-term tracking of haematopoietic clonal dynamics and mutations in non-human primate undergoing transplantation of lentivirally barcoded haematopoietic stem and progenitor cells. 长期跟踪接受慢病毒条形码造血干细胞和祖细胞移植的非人灵长类动物的造血克隆动态和突变。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-10 DOI: 10.1111/bjh.19889

Rohan V Hosuru, Jack Yang, Yifan Zhou, Ashley Gin, Taha B Hayal, So Gun Hong, Cynthia E Dunbar, Chuanfeng Wu

{"title":"Long-term tracking of haematopoietic clonal dynamics and mutations in non-human primate undergoing transplantation of lentivirally barcoded haematopoietic stem and progenitor cells.","authors":"Rohan V Hosuru, Jack Yang, Yifan Zhou, Ashley Gin, Taha B Hayal, So Gun Hong, Cynthia E Dunbar, Chuanfeng Wu","doi":"10.1111/bjh.19889","DOIUrl":"https://doi.org/10.1111/bjh.19889","url":null,"abstract":"Haematopoietic stem and progenitor cell (HSPC) autologous gene therapies are promising treatment for a variety of blood disorders. Investigation of the long-term HSPC clonal dynamics and other measures of safety and durability following lentiviral-mediated gene therapies in predictive models are crucial for assessing risks and benefits in order to inform decisions regarding wider utilization. We established an autologous lentivirally barcoded HSPC transplantation model in rhesus macaque (RM), a model offering insights into haematopoiesis and gene therapies with direct relevance to human. Healthy young adult RMs underwent total body irradiation, followed by transplantation of autologous HSPCs transduced with a lentiviral vector containing a diverse genetic barcode library, uniquely labelling individual HSPCs and their progeny. With up to 131 months of follow-up, we now report quantitative clonal dynamics, characterizing the number, diversity, stability and lineage bias of hundreds of thousands of HSPC clones tracked in five RMs. We documented long-term stable and multi-lineage output from a highly polyclonal pool of HSPCs. Clonal succession after stable haematopoietic reconstitution was minimal. There was no evidence for accelerated acquisition of acquired somatic mutations following autologous lentivirally transduced HSPC transplantation. Our results provide relevant insights into long-term HSPC behaviours in vivo following transplantation and gene therapies.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-11-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613290","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

Hydroxy-wybutosine tRNA modifications as indicators of disease progression and therapeutic targets in leukaemia. 羟基-胞嘧啶 tRNA 修饰作为白血病病情发展的指标和治疗目标。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-10 DOI: 10.1111/bjh.19873

Xu Chen, Rui-Ze Gong, Liu-Ying Mo, Ya-Ting Cheng, Yu Ma, Yi-Tao Qi, Tong-Meng Yan, Zhi-Hong Jiang

{"title":"Hydroxy-wybutosine tRNA modifications as indicators of disease progression and therapeutic targets in leukaemia.","authors":"Xu Chen, Rui-Ze Gong, Liu-Ying Mo, Ya-Ting Cheng, Yu Ma, Yi-Tao Qi, Tong-Meng Yan, Zhi-Hong Jiang","doi":"10.1111/bjh.19873","DOIUrl":"https://doi.org/10.1111/bjh.19873","url":null,"abstract":"Therapeutic approaches for acute myeloid leukaemia (AML) and myelodysplastic syndromes (MDS) differ due to distinct diagnostic criteria and treatment strengths. However, reliable biomarkers to differentiate AML from MDS are needed. This study investigated transfer RNA (tRNA) modifications, particularly hydroxy-wybutosine (OHyW), in the transition from MDS to AML. We found a significant decrease in OHyW and its biosynthetic enzyme leucine carboxyl methyltransferase 2 (LCMT2, alias symbol is TYW4) levels in AML compared to MDS. Mass spectrometric analysis revealed distinct tRNA modification patterns, with AML showing decreased OHyW and increased precursor levels, indicating a disrupted biosynthetic pathway. Lower LCMT2 expression correlated with reduced drug sensitivity and limited differentiation potential in AML cell lines. The results highlight the pivotal role of tRNA modifications in the progression from MDS to AML and suggest that targeting LCMT2 may enhance therapeutic outcomes in AML. By understanding these molecular mechanisms, we can develop new diagnostic markers and therapeutic strategies, potentially transforming the clinical management of AML and improving patient outcomes.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-11-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142613319","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

Targeting RNA modifications in leukaemia: Epitranscriptomic drugs are the new kids on the block. 针对白血病中的 RNA 修饰：外显子转录组药物是新宠。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-11-10 DOI: 10.1111/bjh.19894

Manel Esteller

引用次数: 0