British Journal of Haematology最新文献

{"title":"Real-world outcomes of venetoclax plus hypomethylating agents in unfit acute myeloid leukaemia: Results of the GIMEMA AML2320 trial.","authors":"Raffaele Palmieri, Alfonso Piciocchi, Stefano Soddu, Marco Frigeni, Erika Borlenghi, Nicola Fracchiolla, Maria Paola Martelli, Ernesta Audisio, Alessandro Cignetti, Giacomo Gianfaldoni, Calogero Vetro, Nicola di Rienzo, Daniela Esposito, Ambra Di Veroli, Carmine Selleri, Federica Gigli, Antonio Curti, Antonino Mulè, Catello Califano, Sara Bigliardi, Massimo Breccia, Claudio Fozza, Monica Bocchia, Albana Lico, Federico Simonetti, Elisabetta Pierdomenico, Monia Marchetti, Ilaria Cerroni, Francesco Buccisano, Maria Ilaria Del Principe, Luca Maurillo, Giovangiacinto Paterno, Paola Fazi, Marco Vignetti, Giuseppe Rossi, Alessandro Rambaldi, Adriano Venditti","doi":"10.1111/bjh.70752","DOIUrl":"10.1111/bjh.70752","url":null,"abstract":"<p><p>Venetoclax (VEN) plus hypomethylating agents (HMAs) represents the standard of care for newly diagnosed acute myeloid leukaemia (AML) patients unfit for intensive chemotherapy, but prospective real-world observational data outside randomized clinical trials remain limited. The Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA) AML2320 is a prospective, multicentre, observational study (NCT04589728) including 193 newly diagnosed unfit AML patients receiving VEN plus azacitidine or decitabine between November 2020 and December 2021. Primary end-point was overall survival (OS); secondary end-points included composite complete remission (cCR), disease-free survival and safety. Median age was 74 years with 42% of patients being ≥75 years. After completing cycle 4, cCR was achieved in 73% of the patients, with 54% responding before cycle 2. After a median follow-up of 23 months, median OS was 13.0 months. cCR achievement within cycle 4 correlated with longer OS (19.1 vs. 9.1 months, p = 0.001). Patients receiving 400 mg VEN without azoles had improved OS compared with those on reduced doses with azoles (18.2 vs. 11.4 months, p = 0.015). An anchored matching-adjusted indirect comparison with the phase III VIALE-A trial (NCT02993523) showed comparable median OS (14.8 months vs. 14.9 months; p = 0.6). The GIMEMA AML2320 trial confirmed the efficacy of VEN/HMAs in a prospective, real-world unfit population. Early remission was associated with improved outcomes.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1735-1745"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148786062","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":"First-line hetrombopag combined with immunosuppressive therapy for paediatric severe aplastic anaemia: A prospective randomized controlled trial.","authors":"Lingling Fu, Bixi Yang, Ruixin Wang, Hongmin Li, Hui Chen, Jie Ma","doi":"10.1111/bjh.70636","DOIUrl":"10.1111/bjh.70636","url":null,"abstract":"<p><p>Although the thrombopoietin receptor agonist hetrombopag (HPAG) improves response in adults with severe aplastic anaemia (SAA), its efficacy in paediatric patients remains unclear. In this single-centre, prospective trial, 54 children with newly diagnosed SAA were randomized to receive standard immunosuppressive therapy (IST) alone (n = 27) or combined with HPAG (IST+HPAG, n = 27). The primary end-point was complete response (CR) rate (CRR) at 6 months. Baseline characteristics were balanced. At 6 months, the CRRs were comparable (59.3% vs. 62.9%, p = 0.948) and overall response (OR) rates (ORRs) identical (85.2%) between HPAG+IST and IST groups. At 12 months, ORRs remained identical (77.8%) and CRRs were 62.9% vs. 74.1% (p = 0.875). Time to response showed no significant differences. In children older than 9 years, the 3-month CRR was significantly higher with IST+HPAG (44.4% vs. 27.3%, p = 0.049). Eight grade ≥3 adverse events (primarily elevated liver enzymes) occurred in the IST+HPAG group, all manageable without treatment discontinuation. Rates of clonal events were comparable (23.8% vs. 26.3%). Adding HPAG to first-line IST did not significantly improve early or sustained haematological response rates in paediatric SAA. Routine upfront combination therapy is not warranted for most patients.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1754-1761"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148838677","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":"Bruton tyrosine kinase inhibitors plus chemoimmunotherapy in previously untreated younger mantle cell lymphoma patients: Single-centre clinical outcomes.","authors":"Kelly Chung, Amanda Chron, Anita Kumar, Lisa Modelevsky, Ashlee Joseph, Lynnette Henshaw","doi":"10.1111/bjh.70732","DOIUrl":"10.1111/bjh.70732","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1874-1877"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148688357","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":"Optimizing post-transplant tyrosine kinase inhibitor maintenance in Philadelphia chromosome-positive acute lymphoblastic leukaemia: A risk-adapted framework.","authors":"Satoshi Nishiwaki","doi":"10.1111/bjh.70713","DOIUrl":"10.1111/bjh.70713","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1901-1906"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148534550","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":"De novo JAK2-mutated acute myeloid leukaemia: Clinical outcomes and molecular features.","authors":"Luca Guarnera, Emiliano Fabiani, Eleonora Lumia, Paola Ricci, Nicole Lelli, Federico Moretti, Elisa Meddi, Raffaele Palmieri, Enrico Attardi, Luca Maurillo, Adriano Venditti, Maria Teresa Voso","doi":"10.1111/bjh.70746","DOIUrl":"10.1111/bjh.70746","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1913-1917"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148786156","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":"Second primary cancers in lymphoplasmacytic lymphoma/Waldenström macroglobulinaemia-cumulative burden without generalized excess cancer risk.","authors":"Lars Munksgaard, Lars Moeller Pedersen, Lise Mette Rahbek Gjerdrum","doi":"10.1111/bjh.70740","DOIUrl":"10.1111/bjh.70740","url":null,"abstract":"<p><p>Second primary cancers (SPCs) are a survivorship concern in lymphoplasmacytic lymphoma/Waldenström macroglobulinaemia (LPL/WM), but estimates may be influenced by competing mortality and surveillance. We assessed cumulative incidence, relative risk and predictors of SPCs. We studied 521 patients diagnosed with LPL/WM in Region Zealand, Denmark, during 2000-2023. Cancers diagnosed >6 months before or after diagnosis were classified as prior malignancies and SPCs, respectively. Cumulative incidence was estimated with death as a competing event. Standardized incidence ratios (SIRs) and absolute excess risks (AERs) used regional rates. SPC predictors were evaluated using Fine-Gray and Cox models. Median age was 72 years and median follow-up was 8.1 years. Prior malignancy was present in 64 patients (12.3%); 68 (13.1%) developed an SPC. Cumulative incidence was 9.3% at 5 years and 14.6% at 10 years; 166 patients (31.9%) died without a preceding SPC. With 1-year latency, overall cancer risk was not increased (SIR 1.09, 95% confidence interval [CI] 0.84-1.40; AER 21.3 per 10 000 person-years). Haematological cancers showed borderline excess risk, driven by myelodysplastic syndrome. No covariates were associated with SPC risk. SPCs are clinically relevant in LPL/WM, although excess risk appears selective rather than broadly increased. Long-term surveillance should consider myeloid malignancies.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1684-1693"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674139/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148700247","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":"Total body irradiation is associated with increased burden of cerebral microbleeds in haematopoietic stem cell transplant recipients.","authors":"Jure Hederih, Asad Charania, Camelia Vladescu, Ira Lacej, Richard Szydlo, Jiri Pavlu, Renuka Palanicawandar, Jose Ros-Soto, Andrew Innes, Dragana Milojkovic, Eduardo Olavarria, Jane Apperley, Adam Waldman, Nichola Cooper","doi":"10.1111/bjh.70747","DOIUrl":"10.1111/bjh.70747","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1923-1927"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674133/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148719778","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":"Loss of DNA methylation is not associated with venetoclax-hypomethylating agents synergy in acute myeloid leukaemia cells.","authors":"Aida Selimović-Pašić, Lisa Haglund, Maike Bensberg, Júlia Goldmann, Padraig D'arcy, Sandra Hellberg, Colm E Nestor","doi":"10.1111/bjh.70729","DOIUrl":"10.1111/bjh.70729","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1907-1912"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674118/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148596946","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":"Risk stratification, treatment patterns and survival outcomes in newly diagnosed multiple myeloma patients: A real-world analysis from the Chinese Myeloma Committee.","authors":"Xuelin Dou, Aijun Liao, Jun Luo, Chunrui Li, Ling Qin, Yuping Zhong, Liru Wang, Jinlei Qi, Yang Liu, Nan Peng, Lei Wen, Sinan Gu, Lifen Kuang, Jia Song, Zhongqing Li, Maigeng Zhou, Rong Fu, Juan Li, Juan Du, Jin Lu","doi":"10.1111/bjh.70712","DOIUrl":"10.1111/bjh.70712","url":null,"abstract":"<p><p>We analysed 1384 newly diagnosed multiple myeloma patients from 10 representative centres of the Chinese Myeloma Committee to provide an important modern real-world reference. Survival outcomes were verified by the National Mortality Surveillance System to ensure data accuracy. The median age was 62 years, confirming a younger age at onset in Chinese patients than in Western populations. Treatment patterns have evolved significantly: Lenalidomide, bortezomib and dexamethasone (RVD) became the predominant induction regimen (51.8%), and upfront autologous haematopoietic stem cell transplantation (AHSCT) rates in patients aged ≤65 years rose to 40.0% (vs. 20.0% in our previous multicentre study). In a subcohort of patients from two out of the 10 sites, the median progression-free survival was 44.2 months. In the overall multicentre cohort, the median survival was not reached during a median follow-up of 46.6 months, with an estimated 4-year survival rate of 66.4%. Using the 2025 International Myeloma Society (IMS) / International Myeloma Working Group (IMWG) Consensus Genomic Staging (CGS), 26.2% of patients were classified as high risk. Patients harbouring ≥2 CGS high-risk factors (3.8%) had significantly inferior prognosis. This study validates the 2025 CGS in an Asian cohort and suggests that the widespread adoption of modern triplets is associated with improved real-world survival, while highlighting the distinct poor outcomes in patients with multiple CGS risk factors.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1694-1703"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148589583","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":"Comparison of outcomes after DA-EPOCH-R or GMALL high-intensity therapy for front-line treatment of Burkitt lymphoma: A retrospective cohort.","authors":"Eduard Schulz, Florian Posch, Kristina Friedrich, Peter Neumeister, Katharina T Prochazka","doi":"10.1111/bjh.70730","DOIUrl":"10.1111/bjh.70730","url":null,"abstract":"<p><p>In this retrospective cohort of adult Burkitt lymphoma, DA-EPOCH-R showed no signal of inferior survival or relapse risk compared with GMALL-based high-intensity therapy. Outcomes were driven mainly by established prognostic factors, particularly age, advanced stage and Burkitt lymphoma International Prognostic Index.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1870-1873"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674111/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148583226","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}