British Journal of Haematology最新文献

{"title":"Efficacy and safety of high doses of eltrombopag for treating adult immune thrombocytopenia: Results from a multicentre retrospective cohort study.","authors":"Salomé Dahan, Stéphanie Guillet, Olivier Fain, Delphine Gobert, Stéphane Cheze, Florent Broca, Bertrand Godeau, Marc Michel","doi":"10.1111/bjh.70728","DOIUrl":"10.1111/bjh.70728","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1937-1940"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674144/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148589564","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":"Clinical trials portfolio and regulatory outcomes of ixazomib in multiple myeloma: A systematic review and meta-analysis.","authors":"Veer Shah, Darshi Shah, Payton Utzman, Karan Shah, Srivatsan Swaminathan, Maria Mainou, Rajshekhar Chakraborty, Aaron Goodman, Samer Al Hadidi, Amar H Kelkar, Edward R Scheffer Cliff, Ghulam Rehman Mohyuddin","doi":"10.1111/bjh.70757","DOIUrl":"10.1111/bjh.70757","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1882-1885"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674204/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148808105","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":"Salvage treatment patterns in multiple myeloma patients progressing after lenalidomide maintenance: A real-life study from European Myeloma Network (EMN) Italy.","authors":"Gregorio Barilà, Angela Grassi, Valeria Ruocco, Carmine Liberatore, Edoardo Olivari, Martina Tinelli, Francesca Fazio, Sonia Morè, Emanuele Favero, Claudio Salvatore Cartia, Nicola Sgherza, Concetta Conticello, Roberto Mina, Martina Gherardini, Giuseppe Mele, Massimo Gentile, Anna Furlan, Massimiliano Arangio Febbo, Maria Livia Del Giudice, Ombretta Annibali, Bernardo Rossini, Edoardo Scomazzon, Marco Talarico, Enrica Antonia Martino, Claudio De Magistris, Francesca Fioritoni, Anna Maria Cafro, Laura Paris, Sara Pezzatti, Roberta Della Pepa, Paola Boggione, Giuseppe Pietrantuono, Valeria Tomarchio, Francesco Vassallo, Maria Luisa Pioltelli, Francesco Pisani, Antonia Cagnetta, Lorenzo De Paoli, Gabriele Buda, Elisabetta Antonioli, Monica Galli, Alberto Tosetto, Pellegrino Musto, Elena Zamagni, Francesca Patriarca, Silvia Mangiacavalli, Renato Zambello, Massimo Offidani, Maria Teresa Petrucci","doi":"10.1111/bjh.70751","DOIUrl":"10.1111/bjh.70751","url":null,"abstract":"<p><p>The extensive use of lenalidomide (Len) in the first-line treatment of multiple myeloma (MM) has resulted in an increased frequency of patients who have been exposed or are refractory to Len (Len-R) at first relapse. However, the outcome of Len-R patients treated at first relapse after autologous haematopoietic stem cell transplantation (AHSCT) remains unclear. The aim of this real-life study is to evaluate the effectiveness of the regimens licensed in Italy and used in a cohort of patients who relapsed during Len maintenance after AHSCT. We collected 306 patients with these features. Considering the different regimens used, most patients received isatuximab-carfilzomib-dexamethasone (Isa-KD) (45.4%) followed by daratumumab-pomalidomide-dexamethasone (D-PD) (19.3%) and daratumumab-bortezomib-dexamethasone (D-VD) (12.4%). Overall, a reduced progression-free survival (PFS) and overall survival (OS) were observed in patients with high-risk disease features at relapse, including high lactate dehydrogenase (LDH) (p = 0.0003 and p < 0.0001), International Staging System (ISS) ≥2 (p < 0.0001 and p < 0.0001) and early relapse (<12 months from maintenance initiation) (p = 0.0017 and p = 0.0009). Considering the type of treatment, anti-cluster of differentiation 38 (CD38) monoclonal antibodies (anti-CD38 MoAb)-based regimen displayed improved survival compared to the other treatment, with Isa-KD reporting the longest PFS (22.8 months). Our data in a homogenous cohort of Len-R patients treated at first relapse demonstrated that the anti-CD38 MoAb-based combinations, particularly Isa-KD, represented the standard of care before the approval of anti-B cell maturation antigen (BCMA) immunotherapy.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1704-1714"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674119/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148786124","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":"Glofitamab in combination with immunochemotherapy in patients with relapsed/refractory B-cell non-Hodgkin lymphoma: Phase 1b dose-escalation study.","authors":"William Townsend, Nilanjan Ghosh, Martin Hutchings, Michael Dickinson, Monica Tani, Armando Santoro, Michael Crump, Amitkumar Mehta, Reinhard Marks, Martin Weisser, Martin Barrett, Chun Wu, Derrick Kaufman, Katharina Lechner, Pauline Baumlin, Pontus Lundberg, Max S Topp","doi":"10.1111/bjh.70741","DOIUrl":"10.1111/bjh.70741","url":null,"abstract":"<p><p>Glofitamab was investigated with obinutuzumab/rituximab plus cyclophosphamide, doxorubicin, vincristine and prednisone (G/R-CHOP) in patients with relapsed/refractory B-cell non-Hodgkin lymphoma (B-NHL) who had ≥1 prior obinutuzumab/rituximab-containing therapy (NCT03467373). The primary end-point was to determine the optimal biological dose of glofitamab with R-CHOP. Using a modified continual reassessment method, glofitamab dose escalation was administered from cycle (C) 2 day (D)8 (70 μg, 1800 μg, 10 mg, 30 mg), with G/R- CHOP for 6-8 21-day cycles. The 70 and 1800 μg cohorts received fixed-dose glofitamab; the 10 and 30 mg cohorts received step-up dosing (C2D8, 2.5 mg; C2D15, 10 mg; C3D8+, 30 mg). Patients completing induction could receive glofitamab every 8 weeks for ≤2 years. Thirty-one patients with follicular lymphoma (including transformed), mantle cell lymphoma or marginal zone lymphoma received treatment. The most common adverse events (AEs) were anaemia and fatigue (each 58.1%). Grade 3/4 AEs occurred in 25 (80.6%) patients (serious AEs: 23 [74.2%]; grade 5 AEs: 4 [12.9%; mostly infections]). Sixteen (51.6%) patients experienced cytokine release syndrome (grade 1/2, n = 12 [38.7%]; grade 3/4, n = 4 [12.9%]). Complete metabolic response rate was 83.9%. Median progression-free survival was not reached after 44.2 months follow-up. Glofitamab plus G/R-CHOP showed promising benefit in relapsed/refractory B-NHL, supporting future investigation of glofitamab (2.5/10/30 mg) with R-CHOP in untreated diffuse large B-cell lymphoma.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1651-1663"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674135/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148786064","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":"External validation of the AAA+ survival model in a large Chinese cohort with essential thrombocythaemia.","authors":"Lin Zhang, Wenjing Gu, Huan Dong, Xiaofan Liu, Feng Xue, Yunfei Chen, Wei Liu, Mankai Ju, Ting Sun, Xinyue Dai, Yanmei Xu, Anqi Zhang, Xinmiao Qu, Shuang Ma, Renchi Yang, Rongfeng Fu, Lei Zhang","doi":"10.1111/bjh.70764","DOIUrl":"10.1111/bjh.70764","url":null,"abstract":"<p><p>Triple A Plus (AAA+) is a recently proposed survival prediction model for essential thrombocythaemia (ET) intended for settings in which next-generation sequencing is unavailable. However, independent validation in non-Western populations remains limited. A retrospective cohort of 995 consecutive patients diagnosed with ET at a single centre between 2001 and 2022 was screened. Primary external validation was performed in 850 patients who had complete baseline variables required for the original AAA+ calculation. In the external-validation cohort (n = 850; deaths = 116), AAA+ demonstrated strong discrimination for overall survival (C-index 0.892, 95% confidence interval 0.866-0.917). Time-dependent analyses indicated superior discrimination across follow-up, with 5- and 10-year area under the curve values of 0.902 and 0.912, respectively, exceeding those of AAA (0.873 and 0.894) and International Prognostic Score for Essential Thrombocythemia (0.759 and 0.857). For 10-year all-cause mortality, decision-curve analysis based on locally recalibrated absolute risks showed that AAA+ provided favourable net benefit across most clinically relevant thresholds. Diagnostic period-adjusted analyses showed higher hazards of death in intermediate- and high-risk patients than in low-risk patients, with no clear evidence of temporal heterogeneity or monotonic attenuation of the AAA+ risk gradient over time. AAA+ exhibited robust risk stratification and good discrimination for overall survival in this large Chinese ET cohort.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1725-1734"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148786102","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":"Exploratory research in immune TTP and other ultra-rare diseases-Balancing statistical idealism with pragmatic discovery.","authors":"Shruti Chaturvedi","doi":"10.1111/bjh.70644","DOIUrl":"10.1111/bjh.70644","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1960-1961"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148434299","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":"Leveraging paired germline and somatic analysis to improve the classification of DDX41 variants.","authors":"Maajid Mohi Ud Din Malik","doi":"10.1111/bjh.70789","DOIUrl":"10.1111/bjh.70789","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1966-1967"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148786115","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":"Reply to Correspondence: Leveraging paired germline and somatic analysis to improve the classification of DDX41 variants.","authors":"Terri Patricia McVeigh, Jamshid S Khorashad","doi":"10.1111/bjh.70788","DOIUrl":"10.1111/bjh.70788","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1968-1969"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148981669","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":"Patterns of presentation and outcomes in stage IV Hodgkin lymphoma: A report from the Children's Oncology Group (COG) AHOD1331 trial.","authors":"Dana L Casey, Lindsay A Renfro, Yue Wu, Kathleen McCarten, Qinglin Pei, Sarah A Milgrom, Jennifer A Belsky, Steve Cho, Frank G Keller, Angela Punnett, David C Hodgson, Bradford S Hoppe, Kara M Kelly, Sharon M Castellino","doi":"10.1111/bjh.70625","DOIUrl":"10.1111/bjh.70625","url":null,"abstract":"<p><p>Outcomes for high-risk paediatric classic Hodgkin lymphoma (cHL) improved with the addition of brentuximab vedotin (Bv) to doxorubicin, vincristine, etoposide, prednisone and cyclophosphamide (AVEPC) chemotherapy. We evaluated the prognostic implications of distant extra-nodal sites contributing to stage IV disease. On AHOD1331 (NCT02166463), patients aged 2-21 years with high-risk cHL (stage IIB with bulk/IIIB/IVA/IVB) were randomized to 5 cycles of doxorubicin, bleomycin, vincristine, etoposide, prednisone and cyclophosphamide (ABVE-PC) versus Bv-AVEPC. Patients with stage IV disease, determined by protocol-defined and centrally reviewed Positron Emission Tomography - Computed Tomography (PET-CT), were identified. Baseline characteristics and progression-free survival (PFS) were compared by pattern of stage IV involvement (lung, bone, bone marrow, multiple sites and others). A total of 340 patients (median age 15 years) with stage IV disease were included. Of these patients, 173 (51%) received ABVE-PC and 167 (49%) Bv-AVEPC. PFS was highest with lung-only involvement (4-year PFS 88.6%) and lowest with bone marrow-only involvement (4-year PFS 71.9%). Compared to ABVE-PC, Bv-AVEPC improved PFS among all stage IV patients (hazard ratio [HR] 0.53, 90% confidence interval (CI) 0.33-0.85) and in the lung-only and bone-only subgroups. However, no PFS benefit was observed in those with bone marrow-only involvement. Among paediatric patients with stage IV cHL, Bv-AVEPC improved PFS across most patterns of stage IV disease, except in those with bone marrow involvement. These results will guide efforts to improve outcomes among children with stage IV cHL.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1746-1753"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13386275/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148409738","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":"Treatment sequence with gilteritinib and allogeneic haematopoietic stem cell transplantation in relapsed/refractory FMS-like tyrosine kinase 3 (FLT3)-mutated acute myeloid leukaemia patients: A multicentre real-world study.","authors":"Desiree Kunadt, Sven Zukunft, Christoph Röllig, Stephan Metzelder, Andreas Burchert, Eva Wagner-Drouet, Oliver Kriege, Christoph Schliemann, Matthias Stelljes, Julian Ronnacker, Wolfgang Bethge, Jan Frederic Weller, Björn Steffen, Gesine Bug, Khouloud Kouidri, Claudia Baldus, Lars Fransecky, Elisa Sala, Uwe Platzbecker, Klaus Metzeler, Sabine Kayser, Inken Hilgendorf, Andreas Hochhaus, Tim H Brümmendorf, Edgar Jost, Anna Dorottya Doleschall, Thomas Schroeder, Jennifer Kaivers, Lambros Kordelas, Udo Holtick, Julia M Unglaub, Tim Sauer, Paul Sebastian Jäger, Guido Kobbe, Elena Stauffer, Johanna Tischer, Tobias Herold, Normann Steiner, David Nachbaur, Nicolaus Kröger, Jochen J Frietsch, Sabrina Kraus, Hermann Einsele, Mathias Hänel, Judith Schaffrath, William Krüger, Anne Brandenburg, Tobias A W Holderried, Laura Reinhardt, Markus Maulhardt, Christian Thiede, Martin Bornhäuser, Johannes Schetelig, Jan M Middeke, Friedrich Stölzel","doi":"10.1111/bjh.70735","DOIUrl":"10.1111/bjh.70735","url":null,"abstract":"<p><p>Patients with relapsed or refractory acute myeloid leukaemia (r/r AML) harbouring FMS-like tyrosine kinase 3 (FLT3) mutations generally have poor prognosis and limited treatment options. The second-generation FLT3 inhibitor gilteritinib provides better disease control compared to standard relapse therapies and was approved based on two randomized trials. We analysed its efficacy and safety in a real-world cohort of r/r AML patients undergoing gilteritinib treatment including allogeneic haematopoietic stem cell transplantation (alloHSCT). Data were collected from all consecutive r/r AML patients with FLT3-internal tandem duplication and/or FLT3-tyrosine kinase domain mutations receiving gilteritinib at 25 centres. Response and survival were analysed using descriptive and correlative statistics. A total of 156 patients were analysed, 55.1% with relapse after previous alloHSCT, and 46.2% with prior FLT3 inhibitor exposure. The overall complete remission/incomplete remission (CR/Cri) rate was 45.3%, with a median overall survival (OS) of 10.0 months. Patients receiving gilteritinib only had a 1-year OS of 34%, while those with consolidating alloHSCT had a 1-year OS of 76%-92%, depending on gilteritinib maintenance post-transplant. Resuming gilteritinib post-transplant led to an 85.7% CR/CRi rate. Our real-world data confirm that gilteritinib is an effective single-agent therapy for r/r AML with FLT3 mutations, with best outcomes when used as a bridge to transplant and post-transplant maintenance.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":"1783-1796"},"PeriodicalIF":3.6,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13674134/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148786113","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}