British Journal of Haematology最新文献_第10页

Update on B-cell maturation antigen-directed therapies in AL amyloidosis b细胞成熟抗原导向治疗AL淀粉样变性的最新进展。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-02 DOI: 10.1111/bjh.19960

Krzysztof Jamroziak, Klaudia Zielonka, Jahanzaib Khwaja, Ashutosh D. Wechalekar

{"title":"Update on B-cell maturation antigen-directed therapies in AL amyloidosis","authors":"Krzysztof Jamroziak, Klaudia Zielonka, Jahanzaib Khwaja, Ashutosh D. Wechalekar","doi":"10.1111/bjh.19960","DOIUrl":"10.1111/bjh.19960","url":null,"abstract":"<div>\u0000 \u0000 <p>Systemic light chain (AL) amyloidosis is a rare clonal plasma cell disorder characterized by the production of amyloidogenic immunoglobulin light chains, which causes the formation and deposition of amyloid fibrils, leading to multi-organ dysfunction. Current treatment is directed at the underlying plasma cell clone to achieve a profound reduction in the monoclonal free light chain production. The standard-of-care first-line therapy is a combination of daratumumab, cyclophosphamide, bortezomib and dexamethasone (D-VCd regimen), resulting in high rates of haematological and organ responses. However, AL amyloidosis remains incurable, and all patients inevitably relapse. Hence, novel treatment options are needed for patients with an inadequate response or relapsed/refractory disease. B-cell maturation antigen (BCMA) is a tumour necrosis factor (TNF receptor superfamily receptor overexpressed on plasma cells in multiple myeloma (MM) and AL amyloidosis. Recently, several novel anti-BCMA immunotherapies have been approved for the treatment of relapsed/refractory MM, including antibody–drug conjugate belantamab mafodotin, bispecific antibodies teclistamab and elranatamab and chimeric antigen receptor T-cell therapies idecabtagene vicleucel and ciltacabtagene autoleucel. Despite lower expression than in MM, BCMA is also a promising target in AL amyloidosis. This review aims to provide up-to-date information on the efficacy and toxicity of anti-BCMA therapy in AL amyloidosis.</p>\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"817-831"},"PeriodicalIF":5.1,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19960","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142918728","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

EGLN1 mutations in Cis can induce congenital erythrocytosis with thromboses by increasing protein instability EGLN1突变可通过增加蛋白不稳定性诱导先天性红细胞增多伴血栓形成。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-02 DOI: 10.1111/bjh.19932

Serge Carillo, Marine Delamare, Laurent Henry, Nada Maaziz, Hana Safraou, Betty Gardie, Thierry Lavabre-Bertrand

引用次数: 0

Systemic lupus erythematosus-associated autoantibodies in sickle cell disease: Spontaneous emergence in a patient and in transgenic sickle mice. 镰状细胞病中的系统性红斑狼疮相关自身抗体：在患者和转基因镰状细胞小鼠中自发出现。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-02 DOI: 10.1111/bjh.19972

Urshita Sinha, Suman Setty, Céleste Pilon, Julia J Brown, Maria Armila Ruiz, Guohui Ren, Joyce Rauch, Santosh L Saraf, Jerrold S Levine

引用次数: 0

Plasma inflammatory and angiogenic protein profiling of patients with sickle cell disease 镰状细胞病患者血浆炎症和血管生成蛋白谱分析

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-01 DOI: 10.1111/bjh.19970

L. A. de Ligt, A. E. Gaartman, K. Konté, S. Thakoerdin, K. Fijnvandraat, T. W. Kuijpers, R. van Bruggen, B. J. Biemond, E. Nur

{"title":"Plasma inflammatory and angiogenic protein profiling of patients with sickle cell disease","authors":"L. A. de Ligt, A. E. Gaartman, K. Konté, S. Thakoerdin, K. Fijnvandraat, T. W. Kuijpers, R. van Bruggen, B. J. Biemond, E. Nur","doi":"10.1111/bjh.19970","DOIUrl":"10.1111/bjh.19970","url":null,"abstract":"<p>In this study, we aimed to explore the inflammatory and angiogenic pathways in sickle cell disease (SCD). We used proximity extension assay technology (Olink) to measure 92 plasma proteins involved in inflammation and angiogenesis. Plasma samples were collected from 57 SCD patients (sickle cell anaemia/HbS-β<sup>0</sup> thalassaemia-thalassaemia) in steady-state and 13 healthy ethnicity-matched healthy controls (HCs). From 15 patients, paired samples were collected during both steady-state and vaso-occlusive episodes (VOEs) and from 23 SCD patients longitudinal samples were collected before and after treatment with either voxelotor (<i>n</i> = 10), hydroxyurea (<i>n</i> = 8) or allogeneic haematopoietic stem-cell transplantation (<i>n</i> = 5). Fifty plasma proteins were differentially expressed in steady-state SCD patients as compared to HC. These included proteins involved in angiogenesis (i.e. ANGPT1, ANGPT2 and VEGFA), the IL-18 signalling pathway (i.e. IL-6, IL-10, IL-18), T-cell activation (i.e. LAG3, PDCD1) and natural killer (NK)-cell activation (CD244, NCR1, GZMB). While proteins involved in angiogenesis and the IL-18 signalling pathway were further upregulated during VOE, levels of several proteins involved in the IL-18 pathway, T-cell and NK-cell activation and angiogenesis, restored towards levels detected in HCs after curative or disease-modifying treatment. These findings might contribute to a better understanding of SCD pathophysiology and identifying potential new targets for therapeutic interventions.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"954-964"},"PeriodicalIF":5.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19970","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142913164","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

Long-term remission of TP53-mutated therapy-related acute myeloid leukaemia following allogeneic stem cell transplantation in a heart transplant recipient: A case report and literature review 心脏移植受者异体干细胞移植后tp53突变治疗相关急性髓性白血病的长期缓解：1例报告和文献综述

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-30 DOI: 10.1111/bjh.19982

Annie Xiao, Rachel Davidians, Monzr M. Al Malki

引用次数: 0

HOPX as a tumour-suppressive protein in T-cell acute lymphoblastic leukaemia HOPX在t细胞急性淋巴细胞白血病中的肿瘤抑制作用。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-30 DOI: 10.1111/bjh.19965

Chien-Chin Lin, Chia-Lang Hsu, Chi-Yuan Yao, Yu-Hung Wang, Chang-Tsu Yuan, Yuan-Yeh Kuo, Jhih-Yi Lee, Pin-Tsen Shih, Chein-Jun Kao, Po-Han Chuang, Yueh-Chwen Hsu, Hsin-An Hou, Wen-Chien Chou, Hwei-Fang Tien

{"title":"HOPX as a tumour-suppressive protein in T-cell acute lymphoblastic leukaemia","authors":"Chien-Chin Lin, Chia-Lang Hsu, Chi-Yuan Yao, Yu-Hung Wang, Chang-Tsu Yuan, Yuan-Yeh Kuo, Jhih-Yi Lee, Pin-Tsen Shih, Chein-Jun Kao, Po-Han Chuang, Yueh-Chwen Hsu, Hsin-An Hou, Wen-Chien Chou, Hwei-Fang Tien","doi":"10.1111/bjh.19965","DOIUrl":"10.1111/bjh.19965","url":null,"abstract":"<div>\u0000 \u0000 <p>The homeodomain protein homeobox (<i>HOPX</i>), a multifaceted regulator of cellular functions and developmental processes, is predominantly expressed in stem cells across diverse tissues; it has also emerged as a tumour suppressor in various solid cancers. However, its role in haematological malignancies still remains undefined. This study aimed to elucidate its significance in T-cell acute lymphoblastic leukaemia (T-ALL). We firstly uncovered a novel link between reduced <i>HOPX</i> expression, its promoter hypermethylation and increased tumour burden in patients with T-ALL, suggesting its tumour-suppressive role. Next, we induced T-ALL by transducing intracellular NOTCH1 (ICN1) into mice with either conditional knock-in at the Rosa26 locus or knockout of <i>Hopx</i>. We found that T-ALL development was markedly accelerated and impeded in backgrounds with low and high Hopx expression respectively. Further analysis revealed Hopx's roles in modulating the Wnt-β-catenin pathway, a pivotal regulator of the downstream Myc signalling involved in T-ALL transformation and progression.</p>\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 2","pages":"505-516"},"PeriodicalIF":5.1,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19965","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142906465","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

Successful autologous haematopoietic stem cell transplantation in a refractory anti-myelin-associated glycoprotein antibody neuropathy

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-29 DOI: 10.1111/bjh.19969

Fanny Urbain, Céline Labeyrie, Cristina Castilla-Llorente, Nicolas Noel, Dominique Farge, Andoni Echaniz-Laguna

引用次数: 0

Monoclonal gammopathy of undetermined significance and the risk of thrombotic events: Results from iStopMM, a prospective population-based screening study 未确定意义的单克隆γ病和血栓事件的风险：来自iStopMM的结果，这是一项前瞻性的基于人群的筛查研究。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-25 DOI: 10.1111/bjh.19957

Sæmundur Rögnvaldsson, Alessandro Gasparini, Sigrun Thorsteinsdottir, Ingigerdur Sverrisdottir, Elias Eythorsson, Thorir Einarsson Long, Robert Palmason, Brynjar Vidarsson, Pall Torfi Onundarson, Bjarni A. Agnarsson, Margret Sigurdardottir, Isleifur Olafsson, Ingunn Thorsteinsdottir, Jon Thorir Oskarsson, Asbjorn Jonsson, Runolfur Palsson, Olafur Skuli Indriðason, Andri Olafsson, Malin Hultcrantz, Brian G. M. Durie, Stephen Harding, Ola Landgren, Thorvardur Jon Love, Sigurdur Yngvi Kristinsson

{"title":"Monoclonal gammopathy of undetermined significance and the risk of thrombotic events: Results from iStopMM, a prospective population-based screening study","authors":"Sæmundur Rögnvaldsson, Alessandro Gasparini, Sigrun Thorsteinsdottir, Ingigerdur Sverrisdottir, Elias Eythorsson, Thorir Einarsson Long, Robert Palmason, Brynjar Vidarsson, Pall Torfi Onundarson, Bjarni A. Agnarsson, Margret Sigurdardottir, Isleifur Olafsson, Ingunn Thorsteinsdottir, Jon Thorir Oskarsson, Asbjorn Jonsson, Runolfur Palsson, Olafur Skuli Indriðason, Andri Olafsson, Malin Hultcrantz, Brian G. M. Durie, Stephen Harding, Ola Landgren, Thorvardur Jon Love, Sigurdur Yngvi Kristinsson","doi":"10.1111/bjh.19957","DOIUrl":"10.1111/bjh.19957","url":null,"abstract":"<div>\u0000 \u0000 <p>Monoclonal gammopathy of undetermined significance (MGUS) is the asymptomatic precursor of multiple myeloma and related diseases but has also been associated with thrombosis. Prior studies have not been based on screened cohorts leading to bias. We assessed the risk of thrombosis in a cohort of 75 422 individuals over 40 years old who were screened for MGUS in Iceland. We also evaluated the association of M protein concentration with thrombotic risk. A total of 3668 participants had MGUS. After a median follow-up of ~3.7 years, 124 venous and 252 arterial thrombotic events (10.3 and 21.0 per 1000 person years respectively) were observed in the MGUS group, compared to 1509 and 3471 in the non-MGUS group (6.0 and 13.8 per 1000 person years respectively). After adjusting for multiple confounders, MGUS was associated with an increased risk of venous thrombosis (hazard ratio [HR] = 1.43; 95% confidence interval [CI]: 1.19–1.73) but not arterial thrombosis (HR = 0.96; 95% CI: 0.87–1.13). M protein concentration was not associated with venous (<i>p</i> = 0.72) or arterial (<i>p</i> = 0.95) thrombosis. The findings show, in a screened cohort, that MGUS is associated with venous, but not arterial, thrombosis. Furthermore, they suggest that there is a subset of individuals with MGUS with subclinical monoclonal gammopathy of thrombotic significance.</p>\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"899-906"},"PeriodicalIF":5.1,"publicationDate":"2024-12-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142890704","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

CPX-351 +/− gemtuzumab ozogamicin as induction therapy for adult patients with newly diagnosed, favourable-intermediate risk, FLT3-ITD negative, AML: A pilot study CPX-351 +/- gemtuzumab ozogamicin作为新诊断、有利-中等风险、FLT3-ITD阴性的AML成人患者的诱导治疗：一项试点研究

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-25 DOI: 10.1111/bjh.19967

Chezi Ganzel, Avraham Frisch, Ofir Wolach, Yakir Moshe, Baher Krayem, Neta Dor, Etti Broide, Emmanuel Benayoun, Jacob M. Rowe, Yishai Ofran

引用次数: 0

Identification of a GFI1B variant associated with abnormal platelet function and normal platelet count 与血小板功能异常和血小板计数正常相关的GFI1B变异的鉴定

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-23 DOI: 10.1111/bjh.19964

Nikesh Kawankar, Chandrakala Shanmukhaiah, Bipin Kulkarni

引用次数: 0