British Journal of Haematology最新文献

{"title":"Impact of red blood cell exchange on echocardiographic parameters among adults with sickle cell disease.","authors":"Jules Mercier-Ross, Renata Bahous, Marie-Claude Beaulieu, Arman Sarshoghi, Denis Soulières, Stéphanie Forté, Brian J Potter","doi":"10.1111/bjh.70120","DOIUrl":"https://doi.org/10.1111/bjh.70120","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144936995","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}

{"title":"Comparable peri-transplant mortality and incidence of febrile neutropenia after high-dose melphalan and autologous stem cell transplantation in patients with plasma cell dyscrasias treated as outpatient versus inpatient.","authors":"Ana Maria Avila Rodriguez, Xiao Hu, Cindy Varga, Raymond Comenzo, Shayna Sarosiek, J Mark Sloan, Vaishali Sanchorawala, Gheorghe Doros, Karen Quillen","doi":"10.1111/bjh.70104","DOIUrl":"https://doi.org/10.1111/bjh.70104","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937000","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}

{"title":"NEAT1 lncRNA overexpression results in short-term progression and poor treatment outcome in childhood B-ALL.","authors":"Marieta Xagorari, Antonios Marmarinos, Dimitrios Doganis, Maria Nikita, Evgenia Magkou, Angeliki-Eleni Sfetsiori, Margarita Baka, Lydia Kossiva, Sofia Pasparaki, Alexandra Soldatou, Maria Tsolia, Andreas Scorilas, Dimitrios Gourgiotis, Margaritis Avgeris","doi":"10.1111/bjh.70127","DOIUrl":"https://doi.org/10.1111/bjh.70127","url":null,"abstract":"<p><p>Childhood acute lymphoblastic leukaemia (chALL) remains the most prevalent malignancy in children and adolescents. Improving risk stratification and providing personalized prognosis and treatment remain major clinical challenges. Herein, we analysed the clinical utility of NEAT1 lncRNA for the prognosis and prediction of treatment outcome of childhood B-cell precursor ALL (chB-ALL). NEAT1_1 isoform was quantified in bone marrow samples of chB-ALL patients at diagnosis (n = 160) and at the end of induction (n = 108) of ALL-BFM protocol, and in age-matched healthy children (n = 68). Relapse and death served as clinical end-points for survival analysis. Bootstrap analysis was performed for internal validation and decision curve analysis assessed the clinical net benefit for chB-ALL prognosis. Our analysis showed that chB-ALL patients with NEAT1 overexpression at diagnosis are at significantly higher risk for progression (HR = 2.957, 95% CI: 1.122-7.790, p = 0.011) and worse survival (HR = 5.832, 95% CI: 1.259-27.01, p = 0.012), independently of clinicopathological and treatment data. Moreover, NEAT1-fitted multivariate models resulted in improved risk stratification compared to the conventional disease markers of white blood cells, bone marrow response and minimal residual disease, while decision curve analysis highlighted the superior clinical net benefit for chB-ALL prognosis. In conclusion, NEAT1 overexpression constitutes a powerful, independent predictor of poor treatment outcomes and disease progression of chB-ALL, providing refined stratification of patient's risk.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937062","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}

{"title":"Two-year follow-up of relmacabtagene autoleucel in relapsed or refractory follicular lymphoma in RELIANCE study.","authors":"Yuqin Song, Dehui Zou, Haiyan Yang, Jianqiu Wu, Ye Guo, Wenyu Li, Hui Liu, Zhen Xia, Yongping Zhang, Zisong Zhou, Jun Zhu","doi":"10.1111/bjh.20122","DOIUrl":"https://doi.org/10.1111/bjh.20122","url":null,"abstract":"<p><p>Relmacabtagene autoleucel (relma-cel) demonstrated significant clinical outcomes for ≥3 lines of treatment for relapsed or refractory (R/R) follicular lymphoma (FL) in the RELIANCE study. The primary end-point was the complete response rate (CRR) at 3 months. Key secondary end-points included the duration of response (DOR), progression-free survival (PFS), overall survival (OS) and frequency/severity of adverse events (AEs). Relma-cel-treated patients (N = 28) had a median age of 54 years (range: 36-71 years), and more than 60% of the patients received ≥3 lines of prior systemic therapies. The median follow-up was 24.4 months. As previously reported, in the primary analyses set (n = 27), the 3-month CRR was 85.2% and the 3-month overall response rate was 100.0%. The median DOR, PFS and OS were not reached. As updated in this article, the estimated 2-year PFS and OS rates were 80.3% and 100% respectively. During the 90-day treatment-emergent period (N = 28), no patients had cytokine release syndrome of grade ≥3, and neurological toxicity of grade ≥3 occurred in only one patient treated with 100 × 10⁶ chimeric antigen receptor T cells. Neutropenia (39.3%) was the most common grade ≥3 treatment-emergent adverse event. There were no AEs leading to death. Relma-cel demonstrated durable remissions and manageable safety without new safety signals during the 2-year follow-up in patients with R/R FL (ClinicalTrials.gov, NCT04089215).</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937108","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}

{"title":"The efficacy of targeted and immune-based therapies in adults with TP53-mutated acute lymphoblastic leukaemia.","authors":"Hoda Pourhassan, Jose Tinajero, Huiyan Ma, Ryan Jackson, Raju Pillai, Dat Ngo, Vaibhav Agrawal, Paul Koller, Brian Ball, Amanda Blackmon, Amandeep Salhotra, Monzr Al Malki, Ahmed Aribi, Salman Otoukesh, Andrew Artz, Pamela Becker, Ryotaro Nakamura, Anthony Stein, Stephen Forman, Guido Marcucci, Vinod Pullarkat, Ibrahim Aldoss","doi":"10.1111/bjh.20260","DOIUrl":"https://doi.org/10.1111/bjh.20260","url":null,"abstract":"<p><p>We retrospectively evaluated the treatment outcomes of 47 adult patients with TP53-mutated acute lymphoblastic leukaemia (ALL) treated with either blinatumomab, inotuzumab or/and CD19 CAR T-cell therapy. The complete remission with or without count recovery (CR/CRi) (negative minimal residual disease (MRD-) rate) following treatment with blinatumomab (n = 46), inotuzumab (n = 26) and CD19 CAR T cells (n = 6) was 58.7% (96.3%), 61.5% (60%) and 66.7% (75%) respectively. The median OS was 13.6 months (95% CI: 9.6-17.2) for all patients, and it was not significantly different based on the individual novel salvage therapy (p = 0.40). The 12-month leukaemia-free survival (LFS) for responders to blinatumomab, InO and CAR T cells was 20%, 11% and 0% (p = 0.743) respectively. Patients who had undergone allogeneic haematopoietic stem cell transplantation (HSCT) post-response had improved 12-month LFS compared to those who did not (35% vs. 9%, p = 0.014). Among relapsed patients following blinatumomab, 11 (61%) presented with CD19-negative disease. Hence, targeted and immune-based therapies are effective in inducing high MRD-negative remission rates in adults with B-cell ALL harbouring TP53 mutations. Nonetheless, the durability of remission is short in the absence of allogeneic HCT consolidation and relapse frequently manifested as CD19-negative disease following blinatumomab.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937028","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}

{"title":"Anti-thrombotic therapy in patients with cancer at the end of life and associated clinical outcomes: A cohort study using population-linked routinely collected data.","authors":"Sarah J Aldridge, Ashley Akbari, Adrian Edwards, Kate J Lifford, Denise Abbel, Suzanne Cannegieter, Jamilla Goedegebuur, Eva K Kempers, Mette Søgaard, Chantal Visser, Geert-Jan Geersing, Marieke J H Kruip, Anne Gulbech Ording, Carline van den Dries, Eric C T Geijteman, Frederikus A. Klok, Isabelle Mahé, Simon P Mooijaart, Sebastian Szmit, Simon Noble","doi":"10.1111/bjh.70032","DOIUrl":"10.1111/bjh.70032","url":null,"abstract":"<p><p>Anti-thrombotic therapy (ATT) in cancer patients approaching the end of life presents significant clinical challenges, balancing thrombotic and bleeding risks. This study analysed ATT prescribing patterns and associated outcomes in patients diagnosed with poor prognosis cancer, defined as cancer diagnoses associated with a 1-year life expectancy, using the Welsh national Secure Anonymised Information Linkage Databank. Retrospective cohort study of adults in Wales diagnosed with poor prognosis cancer between 2013 and 2021, following up patients from cancer diagnosis until death, end of follow-up or study end (31 December 2021). Outcomes included ATT discontinuation, bleeding and thromboembolic events in secondary care. We identified a cohort of 25 783 adults with a median survival of 145 days. Of these, 32% were receiving ATT at diagnosis, with 77% continuing until death. One-year cumulative incidence of ATT discontinuation was 19% (95% CI: 18%-20%). The 1-year cumulative incidence of bleeding was 3.2% (95% CI: 3.0%-3.4%) and of thromboembolic events was 5.3% (95% CI: 5.0%-5.6%). ATT was prevalent at cancer diagnosis and discontinuation before death was uncommon. The management of ATT is complex in patients with advanced cancer and there is a need for clearer guidance on appropriate discontinuation strategies as well as when to continue these medicines.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937008","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}

{"title":"Delayed onset vacuoles in a confirmed case of VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome","authors":"Stephen Deering,&nbsp;Luke Y. C. Chen,&nbsp;Richard Xiang,&nbsp;Alexandra Legge","doi":"10.1111/bjh.20171","DOIUrl":"10.1111/bjh.20171","url":null,"abstract":"<p>A 64-year-old male, previously healthy, presented in July 2021 with an unprovoked pulmonary embolism and subsequently developed recurrent fevers, myalgias, arthralgias, neck space and orbital inflammation, lymphadenopathy, cutaneous small-vessel vasculitis and panniculitis. Investigations demonstrated a normocytic anaemia (mean corpuscular volume [MCV]: 87–93 fL) and elevated CRP (45–303 mg/L), but otherwise were non-diagnostic.</p><p>Positron emission tomography (left panel: PET image of the thorax, abdomen and pelvis) showed fluorodeoxyglucose (FDG) avidity in the bone marrow, lymph nodes, spleen, anterior globes, thyroid and peripheral vasculature. Bone marrow biopsy in December 2021 (middle panel: Initial bone marrow aspirate, 10× objective, May–Grünwald–Giemsa stain) revealed a normocellular marrow with occasional hypolobulated megakaryocytes (circled) and maturing trilineage haematopoiesis; no vacuolisation was noted. Lymph node, tonsillar and thyroid biopsies were also performed and were non-contributory.</p><p>The patient responded to high-dose prednisone, but would flare if tapered below 20 mg daily. Methotrexate provided minimal benefit. After referrals to Rheumatology and Haematology, genetic testing was performed and confirmed a p.Met41Val <i>UBA1</i> gene mutation, consistent with a diagnosis of VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome.<span>¹</span> In August 2023, a repeat bone marrow biopsy (right panel: Repeat bone marrow aspirate, 20× objective, May–Grünwald–Giemsa stain) was performed after the patient developed a new progressive macrocytic anaemia (MCV: 95–105 fL). This now revealed trilineage dysplasia with vacuolated erythroid and granulocyte precursors (circled). Treatment was initiated with azacitidine and ruxolitinib, leading to a sustained steroid-free remission. The initial bone marrow biopsy was re-reviewed with again no vacuolisation found.</p><p>Although rare cases of VEXAS with atypical splice site mutations have presented without vacuoles, this is the first reported case of a classic p.Met41Val mutation with initially absent vacuoles that developed over the patient's disease course.<span>²</span> A recent study estimated the prevalence of <i>UBA1</i> pathological variants at 1 in 13 591 for unrelated individuals and 1 in 4269 for men over age 50.<span>³</span> Given VEXAS may be more prevalent than previously thought, this case emphasises the importance of considering molecular testing for <i>UBA1</i> mutations in autoinflammatory conditions to avoid underdiagnosis in early disease or atypical clinical phenotypes.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"207 3","pages":"701-702"},"PeriodicalIF":3.8,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12436209/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937035","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}

{"title":"Twin pregnancy in patients with sickle cell disease: A French cohort study.","authors":"Edouard Leyne, Louise Strube, François Lionnet, Alexandra Benachi, Cyril Touboul, Emile Darai, Fernanda Volt, Eliane Gluckman, Aline Santin, Anne-Gael Cordier","doi":"10.1111/bjh.70121","DOIUrl":"https://doi.org/10.1111/bjh.70121","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937017","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}

{"title":"Measurable residual disease-guided therapy in acute myeloid leukaemia: Practical insights.","authors":"Boaz Nachmias, Arnon Haran, Meira Yisraeli Salman, Eytan M Stein","doi":"10.1111/bjh.70038","DOIUrl":"https://doi.org/10.1111/bjh.70038","url":null,"abstract":"<p><p>Measurable residual disease (MRD) is a prognostic marker in patients with acute myeloid leukaemia (AML). This review examines the role of MRD assessment in guiding treatment strategies. We evaluate key questions, such as the prognostic power of MRD in specific genetic subgroups of AML, the optimal pre-emptive approach for MRD persistence or recurrence, the effect of MRD on allogeneic stem cell transplantation decisions and MRD-guided maintenance considerations. The absence of randomized trials comparing MRD-guided treatment strategies precludes definitive statements. However, by critically evaluating recent studies and emerging trends, we highlight the evolving role of MRD as a decision-making tool in AML, identify key gaps in evidence and suggest directions for future research to optimize personalized treatment approaches.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144936987","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}

{"title":"Reduced incidence of relapse after checkpoint inhibitors relative to brentuximab vedotin as salvage therapy before allogeneic stem cell transplantation for refractory/relapsed Hodgkin lymphoma: A retrospective analysis.","authors":"Jacopo Mariotti, Chiara Pinton, Laura Giordano, Daniela Taurino, Barbara Sarina, Chiara De Philippis, Daniele Mannina, Cristina Tentori, Armando Santoro, Stefania Bramanti","doi":"10.1111/bjh.70091","DOIUrl":"https://doi.org/10.1111/bjh.70091","url":null,"abstract":"<p><p>Eighty-two patients were eligible for allogeneic transplantation (Allo-SCT) at our Institution between April 2014 and August 2022. Of these, 72 actually received Allo-SCT, n = 44 after checkpoint inhibitor (CPI) containing salvage therapy (CPI cohort) and n = 28 with brentuximab-vedotin (BV) containing therapy without CPI (BV cohort). With a median follow-up of 63 months, the CPI cohort had improved cumulative incidence of relapse (5% vs. 37%, p = 0.002) and progression-free survival (PFS) (79% and 56%, p = 0.049) relative to the BV group. By multivariable analysis, pretransplant CPI resulted in the only independent predictor of relapse (hazard ratio [HR]: 0.124, 95% confidence interval 0.270-0.570, p = 0.007) and a strong predictor for PFS (HR 0.43, 95% CI 0.18-1.02, p = 0.057). Our findings support the use of CPI over BV as the first-line salvage therapy for R/R HL patients relapsing after autologous stem cell transplantation when consolidation with Allo-SCT is planned.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937048","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}