British Journal of Haematology最新文献

RNA-sequencing: A reliable tool to unveil transcriptional landscape of paediatric B-other acute lymphoblastic leukaemia.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-30 DOI: 10.1111/bjh.20056

Clara Vicente-Garcés, Guerau Fernández, Elena Esperanza-Cebollada, Mercè Richarte-Franqués, Alba Crespo-Carrasco, Sara Montesdeoca, Ignacio Isola, Edurne Sarrate, Esther Cuatrecasas, Susana Rives, José Luis Dapena, Mireia Camós, Nerea Vega-García

{"title":"RNA-sequencing: A reliable tool to unveil transcriptional landscape of paediatric B-other acute lymphoblastic leukaemia.","authors":"Clara Vicente-Garcés, Guerau Fernández, Elena Esperanza-Cebollada, Mercè Richarte-Franqués, Alba Crespo-Carrasco, Sara Montesdeoca, Ignacio Isola, Edurne Sarrate, Esther Cuatrecasas, Susana Rives, José Luis Dapena, Mireia Camós, Nerea Vega-García","doi":"10.1111/bjh.20056","DOIUrl":"https://doi.org/10.1111/bjh.20056","url":null,"abstract":"B-cell precursor acute lymphoblastic leukaemia (BCP-ALL) comprises multiple subtypes characterized by different genetic alterations. With the use of current standard-of-care tests used in clinical practice, 20%-30% of the cases may not be classified into the main genetic subtypes and additional approaches are needed. These patients are grouped in the heterogeneous category B-other ALL. Transcriptome sequencing (RNA-seq) has allowed the identification of novel fusion genes and gene expression profiles that define new molecular subtypes. We present RNA-seq results integrated, in a real-world scenario, with clinical routine diagnostic data to identify new biomarkers and reclassify a cohort of 60 B-other ALL patients in the newly described genetic subtypes. Overall, 49 rearrangements were identified, including 32 different fusion genes in 41 B-other patients (68%). Moreover, we reported six novel rearrangements (IGK::PAX5, PAX5::IL1RAPL1, ETV6::KRT78, IGH::HIC1, IGH::MIR100HG and NKAIN4::PNPLA7). The integration of RNA-seq results with standard-of-care data allowed us to classify 72% of the patients (43/60) in 11 different subtypes, being DUX4 rearranged and PAX5alt the most represented subtypes. In summary, RNA-seq is a reliable tool for the identification of new emerging genetic subtypes contributing to a better genetic risk stratification of BCP-ALL paediatric patients on the path towards a more personalized medicine.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143750423","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

Outcomes of patients with multiple myeloma undergoing autologous transplant with suboptimal pretransplant response.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-30 DOI: 10.1111/bjh.20062

Oren Pasvolsky, Muhammad Bilal Abid, Denái R Milton, Mark R Tanner, Qaiser Bashir, Samer Srour, Neeraj Saini, Asiya Jatoi, Hina N Khan, Paul Lin, Jeremy Ramdial, Yago Nieto, Guilin Tang, Umer Siddiqui, Yosra Aljawai, Partow Kebriaei, Hans C Lee, Krina K Patel, Mahmoud R Gaballa, Sheeba K Thomas, Robert Z Orlowski, Richard E Champlin, Elizabeth J Shpall, Muzaffar H Qazilbash

{"title":"Outcomes of patients with multiple myeloma undergoing autologous transplant with suboptimal pretransplant response.","authors":"Oren Pasvolsky, Muhammad Bilal Abid, Denái R Milton, Mark R Tanner, Qaiser Bashir, Samer Srour, Neeraj Saini, Asiya Jatoi, Hina N Khan, Paul Lin, Jeremy Ramdial, Yago Nieto, Guilin Tang, Umer Siddiqui, Yosra Aljawai, Partow Kebriaei, Hans C Lee, Krina K Patel, Mahmoud R Gaballa, Sheeba K Thomas, Robert Z Orlowski, Richard E Champlin, Elizabeth J Shpall, Muzaffar H Qazilbash","doi":"10.1111/bjh.20062","DOIUrl":"https://doi.org/10.1111/bjh.20062","url":null,"abstract":"There are scarce data in the literature focusing on newly diagnosed multiple myeloma (NDMM) patients who undergo autologous haematopoietic cell transplantation (autoHCT) after achieving suboptimal response to induction. To address this, we performed a retrospective, single-centre analysis of patients with NDMM who underwent upfront autoHCT between 2005 and 2021 with a pretransplant response of less than very good partial response (<VGPR). Primary outcomes were progression-free survival (PFS) and overall survival (OS). 1109 patients were included in our analysis. Median PFS and OS for the entire cohort were 38.6 (95% confidence interval [CI], 35.9-41.9) months and 103.8 (95% CI, 96.4-113.2) months, respectively. Patients with high-risk cytogenetic abnormalities (HRCA) had a median PFS and OS of 24.8 months and 69.9 months respectively. In multivariable analysis, the use of post-transplant maintenance (hazard ratio [HR] 0.75, p = 0.001 and HR 0.75, p = 0.008) and achieving complete response (CR) at best post-transplant response (HR 0.60, p < 0.001 and HR 0.51, p < 0.001) were associated with superior PFS and OS respectively. In conclusion, NDMM patients who received upfront autoHCT with a pretransplant response of <VGPR had a median PFS of >3 years and median OS of >8 years. Post-transplant maintenance further improved survival outcomes.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143750420","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

The wider perspective: Barriers and recommendations for transfusion support for patients with sickle cell disease in low- and middle-income countries.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-27 DOI: 10.1111/bjh.20055

Jeremy W Jacobs, Luiz Amorim, France Pirenne, Claude Tayou, Ijele Adimora, Lydia H Pecker, Aaron A R Tobian, Jeannie Callum, Julie Makani, Mark T Gladwin, Meghan Delaney, Darrell J Triulzi, Isaac Odame, Evan M Bloch

{"title":"The wider perspective: Barriers and recommendations for transfusion support for patients with sickle cell disease in low- and middle-income countries.","authors":"Jeremy W Jacobs, Luiz Amorim, France Pirenne, Claude Tayou, Ijele Adimora, Lydia H Pecker, Aaron A R Tobian, Jeannie Callum, Julie Makani, Mark T Gladwin, Meghan Delaney, Darrell J Triulzi, Isaac Odame, Evan M Bloch","doi":"10.1111/bjh.20055","DOIUrl":"https://doi.org/10.1111/bjh.20055","url":null,"abstract":"Globally, sickle cell disease (SCD) is the most common inherited haemoglobinopathy. The highest burden of SCD is encountered in low- and middle-income countries (LMICs), most of which lack the resources to contend with the disease. There is a marked divide between care for individuals with SCD in high-income countries (HICs) versus LMICs, whereby the few disease-modifying therapies and curative regimens are only accessible to those in HICs. As such, blood transfusion remains central to the emergent treatment and prevention of complications of SCD. However, there are a myriad of related challenges in LMICs, which have impeded efforts to treat patients with SCD effectively. In addition to blood safety and availability, examples that impact SCD specifically include capabilities to detect and/or manage red blood cell alloimmunization, capacity for automated red cell exchange, limited immunohematology, suboptimal quality oversight with a lack of safeguards to prevent transfusion of incompatible blood and limited or absent post-transfusion surveillance to detect and/or manage transfusion-associated adverse events. Consequently, clinical practices that are otherwise regarded as standard of care in HICs remain the exception in LMICs, highlighting disparities in care. A multifaceted approach that prioritizes transfusion support in LMICs is needed to improve care for patients with SCD.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143727200","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

CD19 chimeric antigen receptor-T cell therapy in murine immune thrombocytopenia.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-26 DOI: 10.1111/bjh.20061

Fengjiao Han, Zhengqi Jiang, Qiuyu Guo, Yucan Li, Chaoyang Li, Xiaohong Liang, Lin Han, Reid C Gallant, Ming Hou, Jun Peng, Miao Xu

{"title":"CD19 chimeric antigen receptor-T cell therapy in murine immune thrombocytopenia.","authors":"Fengjiao Han, Zhengqi Jiang, Qiuyu Guo, Yucan Li, Chaoyang Li, Xiaohong Liang, Lin Han, Reid C Gallant, Ming Hou, Jun Peng, Miao Xu","doi":"10.1111/bjh.20061","DOIUrl":"https://doi.org/10.1111/bjh.20061","url":null,"abstract":"Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by antiplatelet autoantibodies, with many patients refractory or relapsing on conventional treatments. GPIbα, an important autoantigen in ITP, is notably linked to refractoriness, highlighting the need for novel treatments. We assessed CD19 chimeric antigen receptor (CAR)-T cell therapy's potential in a modified murine model targeting GPIbα. CD19 CAR-T cell infusion accelerated platelet count recovery compared to the control group, effectively depleted CD19+ B cells and CD138+ plasma cells, and markedly reduced anti-GPIbα autoantibodies in vivo. In vitro CD19 CAR-T cells reduced both plasma cells and B cells in the spleens of mice and ITP patients. CD19 CAR-T cell therapy significantly altered T-cell subsets, increasing regulatory T cells, T helper 1 and T helper 17 populations, suggesting a role in modulating the immune response for sustained ITP remission. Monitoring of body/spleen weights and temperature showed no significant cytokine release syndrome, indicating a favourable safety profile. These promising results support the potential of CD19 CAR-T cell therapy as a novel treatment option for refractory ITP, particularly in GPIbα-positive autoantibody patients. Further clinical studies are warranted to assess the safety and efficacy of this approach in human patients.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143727199","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

Frequency and prognostic value of unconventional genetic subtypes in paediatric and young adult B-cell precursor acute lymphoblastic leukaemia in Taiwan.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-25 DOI: 10.1111/bjh.20057

Ying-Jung Huang, Hsi-Che Liu, Ming-Chung Kuo, Ting-Chi Yeh, Tung-Liang Lin, Shih-Hsiang Chen, Tang-Her Jaing, Shih-Chung Wang, Te-Kau Chang, Hsiu-Ju Yen, Jiunn-Ming Sheen, Ming-Chung Wang, Tung-Huei Lin, Ting-Yu Huang, Hsiao-Wen Kao, Che-Wei Ou, Yu-Shin Hung, Chih-Cheng Hsiao, Lee-Yung Shih

{"title":"Frequency and prognostic value of unconventional genetic subtypes in paediatric and young adult B-cell precursor acute lymphoblastic leukaemia in Taiwan.","authors":"Ying-Jung Huang, Hsi-Che Liu, Ming-Chung Kuo, Ting-Chi Yeh, Tung-Liang Lin, Shih-Hsiang Chen, Tang-Her Jaing, Shih-Chung Wang, Te-Kau Chang, Hsiu-Ju Yen, Jiunn-Ming Sheen, Ming-Chung Wang, Tung-Huei Lin, Ting-Yu Huang, Hsiao-Wen Kao, Che-Wei Ou, Yu-Shin Hung, Chih-Cheng Hsiao, Lee-Yung Shih","doi":"10.1111/bjh.20057","DOIUrl":"https://doi.org/10.1111/bjh.20057","url":null,"abstract":"Unconventional genetic subtypes of B-cell precursor acute lymphoblastic leukaemia (B-ALL) were analysed to compare their frequency and their impact on outcomes between children and young adults in Taiwan. Unconventional subtypes were found in 23.0% of 456 paediatric B-ALL and 24.5% of 139 young adult B-ALL. The most frequently unconventional subtype both in children and young adults was BCR::ABL1-like, which could be subdivided into different kinase-altering aberrations in 67.3% of children and 78.6% of young adults. CRLF2-R was more frequent in children, while IL7R mutations were more common in young adults. In children, favourable outcomes were observed in patients with DUX4-R and PAX5alt, whereas those with BCR::ABL1-like and MEF2D-R had inferior outcomes. BCR::ABL1-like and MEF2D-R were also the independent predictors of inferior event-free survival in children. Conversely, most unconventional subtypes in young adults were associated with adverse outcomes except for DUX4-R. We found a lower incidence of BCR::ABL1-like and a better prognosis for paediatric PAX5alt in Taiwan compared to the West. Additionally, genetic differences were identified between paediatric and young adult BCR::ABL1-like subtypes. The extremely poor prognosis for unclassified young adults highlights the potential use of further subdivision of unfavourable genetic subtypes in refining risk classification and treatment optimization.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143707923","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

The real-world application of T-cell receptor constant beta-1 chain antibody assay in cutaneous T-cell lymphoma.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-24 DOI: 10.1111/bjh.20060

Olivia Pierog, Taylor Craig, Jacky Jennings, Michael J Borowitz, Ananya Munjal, Mariah Owusu-Agyei, David Weiner, J David Peske, Suman Paul, Sima Rozati

引用次数: 0

Are we ignoring sex differences in haematological malignancies? A call for improved reporting.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-23 DOI: 10.1111/bjh.20044

Ora Paltiel, Sumita Ratnasingam, Hui-Peng Lee

{"title":"Are we ignoring sex differences in haematological malignancies? A call for improved reporting.","authors":"Ora Paltiel, Sumita Ratnasingam, Hui-Peng Lee","doi":"10.1111/bjh.20044","DOIUrl":"https://doi.org/10.1111/bjh.20044","url":null,"abstract":"There are clear sex-based differences in the incidence, risk factors and mortality of most haematologic malignancies (HM). Despite known differences in physiology, haematopoiesis, molecular profiles, drug pharmacokinetics, treatment-related toxicities and treatment experience, males and females receive standardized and identical treatment for most HMs. Previous published work has demonstrated disparities in female representation in cancer clinical trials and highlighted a paucity of information on differential treatment outcomes and toxicities by sex. We analysed references of 182 clinical trials which form the basis of recent treatment guidelines from the National Comprehensive Cancer Network and found a minority (17/9.3%) did not report the sex distribution of trial participants. However, a majority (165/90.6%) did not report sex-disaggregated outcomes. Of those that did, 36.5% showed outcome differences by sex. Academic leadership by women in the assessed trials as well as in guidelines committees was disproportionately lower than their representation in the profession. We call on all clinical trials leaders, consortia and guideline builders to include sex-disaggregated data in their analyses, reporting these in a transparent manner (as per regulations mandating such reporting), and for investigators to assess whether aetiological factors differ by sex. These actions will enhance personalized prevention, therapy and follow-up.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143690630","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

Monoclonal gammopathy.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-23 DOI: 10.1111/bjh.20059

Gurmukh Singh

引用次数: 0

Correction to 'Genetic testing to identify hereditary predispositions to haematological malignancy is critical prior to allogenic haematopoietic cell transplant'.

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-03-23 DOI: 10.1111/bjh.20054

引用次数: 0

Enterocolitis associated with glofitamab-First report and clinicopathological findings in three cases.

IF 5.1 2区医学