Stefano Luminari, Maria Elena Nizzoli, Michele Merli, Sara Rattotti, Vittoria Tarantino, Simone Ferrero, Annalisa Talami, Roberta Murru, Marina Deodato, Emanuele Cencini, Giovanni Roti, Carlo Visco, Guilherme Duffles, Michele Spina, Ombretta Annibali, Alessandro Pulsoni, Andrés José María Ferreri, Caterina Stelitano, Elsa Pennese, Emiliano Barbieri, Marzia Varettoni, Gambara Silvia, Marta Coscia, Dario Marino, Tisi Maria Chiara, Maher Nawar, Luigi Marcheselli, Francesco Merli, Luca Arcaini
{"title":"Survival after first relapse in marginal zone lymphomas: Prospective data from the international observational NF10 study.","authors":"Stefano Luminari, Maria Elena Nizzoli, Michele Merli, Sara Rattotti, Vittoria Tarantino, Simone Ferrero, Annalisa Talami, Roberta Murru, Marina Deodato, Emanuele Cencini, Giovanni Roti, Carlo Visco, Guilherme Duffles, Michele Spina, Ombretta Annibali, Alessandro Pulsoni, Andrés José María Ferreri, Caterina Stelitano, Elsa Pennese, Emiliano Barbieri, Marzia Varettoni, Gambara Silvia, Marta Coscia, Dario Marino, Tisi Maria Chiara, Maher Nawar, Luigi Marcheselli, Francesco Merli, Luca Arcaini","doi":"10.1111/bjh.70808","DOIUrl":"https://doi.org/10.1111/bjh.70808","url":null,"abstract":"<p><p>While marginal zone lymphoma (MZL) is generally indolent, outcomes following relapse remain poorly defined. This study utilized the indolent Non Follicular 10 (NF10) prospective database to characterize survival and identify prognostic drivers in patients failing initial systemic therapy. The study analysed 122 patients with relapsed MZL. The primary end-point was progression-free survival from first relapse (2PFS), with survival after relapse (SAR) as the secondary end-point. The median 2PFS was 24 months, with a 2-year 2PFS rate of 49% and a 2-year SAR rate of 66%. Multivariable analysis identified advanced age, progression of disease within 24 months (POD24) and high MZL International Prognostic Index (MZL-IPI) scores as significant predictors of inferior survival. Patients with splenic and disseminated subtypes also demonstrated worse outcomes. Relapse following systemic therapy often marks a transition to an aggressive clinical course. Time to progression and the MZL-IPI are robust tools for risk stratification, highlighting an urgent need for novel therapeutic strategies for high-risk relapsed MZL populations.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2026-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148862815","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}
Elena Vuelta, Alessandro Liquori, Matias Morín, Lucia Soletto, Miguel Ángel Marugal, Marta Santiago-Balsera, Miguel Gallardo, Miguel Ángel Moreno-Pelayo, José Cervera, Alejandra Sanjuan-Pla
{"title":"Functional assessment of inherited myeloid neoplasm-associated SAMD9L germline variants via Monoallelic CRISPR modelling.","authors":"Elena Vuelta, Alessandro Liquori, Matias Morín, Lucia Soletto, Miguel Ángel Marugal, Marta Santiago-Balsera, Miguel Gallardo, Miguel Ángel Moreno-Pelayo, José Cervera, Alejandra Sanjuan-Pla","doi":"10.1111/bjh.70799","DOIUrl":"https://doi.org/10.1111/bjh.70799","url":null,"abstract":"<p><p>While the majority of myeloid neoplasms are sporadic, the increasing application of germline genetic testing has led the World Health Organization to designate 'Myeloid malignancies with germline predisposition' as a distinct clinical entity, carrying major implications for clinical care and research. Germline variants in the Sterile alpha motif domain-containing 9-like (SAMD9L) gene are specifically associated with an increased risk for these malignancies. In this study, we functionally modelled two novel SAMD9L variants-p.N697Y and p.K1294*-alongside two previously reported variants (p.T233N and p.H880Q). We generated heterozygous knock-in cellular models in the Human leukemia (HL-60 myeloid cell line) for each variant using homology-directed repair-based Clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9 (CRISPR/Cas9) gene editing. Functional assays, focused on proliferation and protein translation, confirmed that the p.T233N, p.N697Y and p.K1294* variants all caused a decreased rate of protein translation. These results provide functional evidence to fine-tune the classification of these SAMD9L variants and significantly advance our understanding of the molecular mechanisms by which SAMD9L variants drive inherited myeloid neoplasms.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2026-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148849570","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}
Lingling Fu, Bixi Yang, Ruixin Wang, Hongmin Li, Hui Chen, Jie Ma
{"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":"https://doi.org/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":""},"PeriodicalIF":3.6,"publicationDate":"2026-08-28","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}
R Van Dijck, M Ter Borg, B van der Holt, A E C Broers, M D Hazenberg, J J Cornelissen, N P M Schaap, E de Pater, P A W Te Boekhorst
{"title":"NFкB pathway suppression as a key driver of pacritinib response in high-risk myelofibrosis.","authors":"R Van Dijck, M Ter Borg, B van der Holt, A E C Broers, M D Hazenberg, J J Cornelissen, N P M Schaap, E de Pater, P A W Te Boekhorst","doi":"10.1111/bjh.70811","DOIUrl":"https://doi.org/10.1111/bjh.70811","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2026-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148849556","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}
Luani Barge, Lachlan Webb, Anita Pelecanos, Niamh Waters, Ross Salvaris, Sam Lai, Tessa Potezny, Helen Cashman, Caitlyn Nguyen-Ngo, Denise Lee, Costas K Yannakou, Nada Hamad, Adrien Chauchet, Sylvain Choquet, Cédric Rossi, Adélie Herbin, Lukshe Kanagaratnam, Cyrielle Rodier, Constantine S Tam, Abir Bhattacharyya, Chan Y Cheah, Eliza A Hawkes, Kirk Morris, Eric Durot, Joshua Tobin, Greg Hapgood
{"title":"LTB-TREAT: A validated international prognostic model for time to treatment in low tumour burden follicular lymphoma.","authors":"Luani Barge, Lachlan Webb, Anita Pelecanos, Niamh Waters, Ross Salvaris, Sam Lai, Tessa Potezny, Helen Cashman, Caitlyn Nguyen-Ngo, Denise Lee, Costas K Yannakou, Nada Hamad, Adrien Chauchet, Sylvain Choquet, Cédric Rossi, Adélie Herbin, Lukshe Kanagaratnam, Cyrielle Rodier, Constantine S Tam, Abir Bhattacharyya, Chan Y Cheah, Eliza A Hawkes, Kirk Morris, Eric Durot, Joshua Tobin, Greg Hapgood","doi":"10.1111/bjh.70748","DOIUrl":"https://doi.org/10.1111/bjh.70748","url":null,"abstract":"<p><p>Watchful waiting (WW) in low tumour burden follicular lymphoma (LTB FL) defers treatment. There is a need to develop a model to predict time to treatment (TTT). We aimed to develop and externally validate a model for TTT in LTB FL. We conducted a retrospective analysis across eight centres within the Australasian Lymphoma Alliance. Two hundred and twenty-nine patients were included between 2004 and 2022 with a median follow-up of 5.0 years (range 0.5-17.8 years) and a median age was 64 years. All patients were Group d'Etude des Lymphomes Folliculaires (GELF) negative and managed with WW. Elevated lactate dehydrogenase and >4 nodal areas were independent prognostic factors associated with shorter TTT. These factors formed a prognostic model identifying three groups: low 59% (median TTT 6.34 years), intermediate 36% (median TTT 4.08 years), high risk 5% (median TTT 1.49 years) (p < 0.001). There was a higher incidence of serious complications among risk groups (low 7.5% vs. intermediate 13.3% vs. high 25%; p = 0.096). The prognostic value of the LTB-TREAT model was confirmed in an external validation cohort. The low-risk group is predicted to have a prolonged TTT with WW and a high-risk group is predicted to experience early progression in whom treatment should be considered.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2026-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148823500","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":"Real-world evidence on the safety and effectiveness of thrombopoietin receptor agonists in aplastic anaemia: A descriptive study using Japanese hospital administrative data.","authors":"Kayoko Mizuno, Yutaka Shimazu, Toshiki Fukasawa, Satoru Ito, Yukihiro Nishio, Mami Shimizu, Koji Kawakami","doi":"10.1111/bjh.70796","DOIUrl":"https://doi.org/10.1111/bjh.70796","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2026-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148823525","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}
Edvan Q Crusoé, Glaciano Ribeiro, João Tadeu D Souto Filho, Jayr Schmidt Filho, Natalia Schutz, Milton A F Aranha, Abel Costa, Abrahao Hallack, Fernando V Pericole, Juliana S Lima, Breno Gusmão, Eduardo Flávio O Ribeiro, Jorge P Vaz, Rosane Bittencourt, Rafael Cunha, Danielle Ovigli, Luiza Berg, Ederson R Mattos, Angelo Maiolino, Vania Hungria
{"title":"Real-world outcomes of daratumumab, bortezomib, thalidomide and dexamethasone (Dara-VTd) induction and lenalidomide maintenance in transplant-eligible multiple myeloma.","authors":"Edvan Q Crusoé, Glaciano Ribeiro, João Tadeu D Souto Filho, Jayr Schmidt Filho, Natalia Schutz, Milton A F Aranha, Abel Costa, Abrahao Hallack, Fernando V Pericole, Juliana S Lima, Breno Gusmão, Eduardo Flávio O Ribeiro, Jorge P Vaz, Rosane Bittencourt, Rafael Cunha, Danielle Ovigli, Luiza Berg, Ederson R Mattos, Angelo Maiolino, Vania Hungria","doi":"10.1111/bjh.70775","DOIUrl":"https://doi.org/10.1111/bjh.70775","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2026-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148823460","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}
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