Lancet Haematology最新文献

{"title":"Hereditary methaemoglobinaemia in the 12th-century Japanese national treasure artwork Yamai no Sōshi.","authors":"Bingwen Eugene Fan, Hanna Osawa","doi":"10.1016/S2352-3026(25)00175-9","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00175-9","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e492-e493"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561567","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hookworm should be considered as a cause of iron deficiency.","authors":"Philip Murphy","doi":"10.1016/S2352-3026(25)00161-9","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00161-9","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e488"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cardiac arrest linked to myeloid sarcoma after haematopoietic stem-cell transplantation.","authors":"Dennis Christoph Harrer, Patricia Mester, Stephan Schmid, Atik Baborie, Markus J Riemenschneider, Alexander Fuchs, Daniel Wolff, Hendrik Poeck, Wolfgang Herr, Martina Müller, Vlad Pavel","doi":"10.1016/S2352-3026(25)00102-4","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00102-4","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e560"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Allogeneic haematopoietic cell transplantation in peripheral T-cell lymphoma: recommendations from the EBMT Practice Harmonisation and Guidelines Committee.","authors":"Gandhi Damaj, Ali Bazarbachi, Philipp Berning, Anne-Ségolène Cottereau, Christopher P Fox, Charalampia Kyriakou, Kate Cwynarski, Pier-Luigi Zinzani, Olivier Tournilhac, Wojiech Jurczak, Imke E Karsten, Fengrong Wang, Ambroise Marcais, Alina Tanase, Stéphanie Nguyen-Quoc, Yasmina Serroukh, Mahmoud Aljurf, David Beauvais, Gerald Wulf, Anna Sureda, Paolo Corradini, Olivier Hermine, Mehdi Hamadani, Francesco Onida, Anna Lisa Ruggeri, Isabel Sanchez-Ortega, Ibrahim Yakoub-Agha, Peter Dreger, Norbert Schmitz","doi":"10.1016/S2352-3026(25)00073-0","DOIUrl":"10.1016/S2352-3026(25)00073-0","url":null,"abstract":"<p><p>Allogeneic haematopoietic cell transplantation (HCT) is a potentially curative therapy for peripheral T-cell lymphoma; however, to date, there are no standardised and detailed guidelines for its application. To address gaps in clinical practice, the European Society for Blood and Marrow Transplantation (EBMT) Practice Harmonisation and Guidelines Committee convened an international expert meeting in Lille (France) on Sept 30 and Oct 1, 2024. EBMT performed funding acquisition. The chairs invited experts in T-cell lymphoma and HCT, who were organised in two work packages devoted to peripheral T-cell lymphoma and transplantation. A literature search was performed beforehand, and results were presented by subgroups of two or three experts, then discussed collectively in structured discussions, and consensus-building exercises. The recommendations presented in this Review were developed and approved based on current evidence, expert opinion, and clinical experience. These recommendations should help to further establish allogeneic HCT as the standard of care for eligible patients with refractory or relapsed peripheral T-cell lymphoma, leveraging its curative potential through the graft-versus-lymphoma effect.</p>","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e542-e554"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561563","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction to Lancet Haematol 2024; 11: e521-29.","authors":"","doi":"10.1016/S2352-3026(25)00174-7","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00174-7","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e489"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561565","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Off-the-shelf induced pluripotent stem-cell-derived natural killer-cell therapy in relapsed or refractory B-cell lymphoma: a multicentre, open-label, phase 1 study.","authors":"Paolo Strati, Januario Castro, Aaron Goodman, Veronika Bachanova, Manali Kamdar, Farrukh T Awan, Scott R Solomon, Lilly Wong, Carol Wong, Deepa Patel, Cara Bickers, Wei Zhao, Zahid Bashir, Bahram Valamehr, Rebecca L Elstrom, Krish Patel","doi":"10.1016/S2352-3026(25)00142-5","DOIUrl":"10.1016/S2352-3026(25)00142-5","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Natural killer-cell therapies are limited by donor cell sourcing and dose-to-dose variability. FT516 is an induced pluripotent stem cell (iPSC)-derived natural killer-cell therapy expressing high-affinity, non-cleavable CD16 to optimise antibody-dependent cellular cytotoxicity in combination with therapeutic monoclonal antibody. We aimed to assess the safety of FT516 in patients with relapsed or refractory B-cell lymphoma.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This multicentre, open-label, phase 1 study was conducted at eight research centres in the USA. Eligible patients were aged 18 years or older, had B-cell lymphoma expected to express CD20, with relapsed or refractory disease following at least one previous systemic therapy including anti-CD20 antibody, had measurable disease, and had no treatment options expected to improve survival. Participants received fludarabine (30 mg/m&lt;sup&gt;2&lt;/sup&gt; for 3 days on days -5 to -3) and cyclophosphamide (500 mg/m&lt;sup&gt;2&lt;/sup&gt; for 3 days on days -5 to -3) or bendamustine (90 mg/m&lt;sup&gt;2&lt;/sup&gt; for 2 days on days -4 and -3) combined with rituximab at 375 mg/m&lt;sup&gt;2&lt;/sup&gt; on day -4 or obinutuzumab 1000 mg replaced rituximab in patients with follicular lymphoma during dose expansion. FT516 was administered intravenously at escalating doses, ranging from 3 × 10&lt;sup&gt;7&lt;/sup&gt; to 9 × 10&lt;sup&gt;8&lt;/sup&gt; cells per dose on days 1, 8, and 15, with IL-2 (6 million units) administered subcutaneously 2-4 h after each FT516 dose. The primary endpoint was safety, including dose-limiting toxicity and maximum tolerated dose. Safety was analysed in all patients who received at least one dose of FT516. Patients with acute myeloid leukaemia were also enrolled and will be reported elsewhere. This study was registered with ClinicalTrials.gov, NCT04023071, and is completed.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;From Oct 11, 2019, to Nov 28, 2022, 56 patients were enrolled, 55 of whom received FT516. 32 (58%) patients were male, 23 (42%) were female, and 43 (78%) were White. The maximum FT516 cell dose (9 × 10&lt;sup&gt;8&lt;/sup&gt; cells per dose for three doses per 28-day cycle) was tolerated and identified as the recommended phase 2 dose. No dose-limiting toxicities were reported. Cytokine release syndrome was reported in one (2%) patient and was grade 1; neurotoxicity was not observed. Most common adverse events grade 3 or worse were neutropenia (in 46 [84%] patients), thrombocytopenia (20 [36%]), and anaemia (15 [27%]). There were no treatment-related deaths. Objective response was observed in 32 (58%) of 55 patients.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interpretation: &lt;/strong&gt;Our findings suggest that cell therapy using iPSC-derived, gene-modified natural killer cells in combination with monoclonal antibody and IL-2 is safe and active in B-cell malignancies and might address limitations of currently available immune-cell therapies, including manufacturing time, heterogeneity, access, and cost.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Funding: &lt;/strong&gt;Fate Therapeutics.&lt;/","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e505-e515"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561569","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study.","authors":"Rachael F Grace, Göksel Leblebisatan, Yesim Aydinok, Şule Ünal, John D Grainger, Jessica Zhang, Linda Smallwood, Emily de León, Brian D Jamieson","doi":"10.1016/S2352-3026(25)00107-3","DOIUrl":"10.1016/S2352-3026(25)00107-3","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Avatrombopag is an oral thrombopoietin receptor agonist (TPO-RA), taken with food without any specific restrictions, approved for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The aim of this study was to assess the efficacy, safety, tolerability, and pharmacokinetic and pharmacodynamic profile of avatrombopag for children and adolescents with persistent and chronic primary ITP.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;AVA-PED-301 is a global, multicentre, randomised, double-blind, placebo-controlled, parallel-group, phase 3b study. Children and adolescents aged ≥1 to &lt;18 years with a confirmed diagnosis of primary ITP for at least 6 months and an insufficient response to a previous treatment were randomly assigned (3:1) to avatrombopag (aged ≥6 years: 20 mg oral tablet once daily; aged ≥1 to &lt;6 years: 10 mg oral suspension once daily) or matching placebo. Doses were titrated to maintain a platelet count of 50-150 × 10&lt;sup&gt;9&lt;/sup&gt; cells per L. The core-phase (12-week double-blind treatment period) full analysis set included all randomly assigned patients; efficacy analysis was performed based on randomised study drug. The safety analysis set included all patients who received at least one dose of study drug in each study phase. The primary endpoint was durable platelet response (proportion of patients with at least six out of eight weekly platelet counts ≥50 × 10&lt;sup&gt;9&lt;/sup&gt; cells per L during the last 8 weeks of the 12-week treatment period in the absence of rescue therapy); the alternative primary endpoint was platelet response (proportion of patients with at least two consecutive platelet assessments ≥50 × 10&lt;sup&gt;9&lt;/sup&gt; cells per L over the 12-week treatment period in the absence of rescue therapy). The trial core phase is complete and was registered with ClinicalTrials.gov (NCT04516967).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;Between March 2, 2021, and Aug 2, 2023, 83 children were screened, with 75 randomly assigned to avatrombopag (n=54; 24 [44%] female; 48 [89%] White) or placebo (n=21; 12 [57%] female; 15 [71%] White). 15 (28%) patients in the avatrombopag group met the primary endpoint of durable platelet response versus no (0%) patients in the placebo group (difference in response rate 28% [95% CI 16-40]; p=0·0077); 44 (81%) patients in the avatrombopag group met the alternative primary endpoint of platelet response versus no (0%) patients in the placebo group (difference in response rate 81% [71-92]; p&lt;0·0001). The most common adverse events across treatment groups were petechiae (n=20), epistaxis (n=16), and headache (n=14). Serious adverse events were reported in five (9%) patients in the avatrombopag group and one (5%) patient in the placebo group. No deaths, thromboembolic events, or grade 3 or higher bleeding events were reported.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Interpretation: &lt;/strong&gt;Avatrombopag is an effective oral treatment for children and a","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":" ","pages":"e494-e504"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144209990","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy or convenience? Subcutaneous blinatumomab as a promising new treatment for B-cell acute lymphoblastic leukaemia.","authors":"Emily Curran","doi":"10.1016/S2352-3026(25)00169-3","DOIUrl":"10.1016/S2352-3026(25)00169-3","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e485-e487"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561566","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Off-the-shelf iPSC natural killer-cell therapy: a new chapter for B-cell lymphoma?","authors":"Tobias Tix, Kai Rejeski","doi":"10.1016/S2352-3026(25)00166-8","DOIUrl":"10.1016/S2352-3026(25)00166-8","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 7","pages":"e484-e485"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561570","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bexmarilimab plus azacitidine for high-risk myelodysplastic syndrome and relapsed or refractory acute myeloid leukaemia: results from the dose-escalation part of a multicentre, single-arm, phase 1/2 trial.","authors":"Mika Kontro, Anthony Selwyn Stein, Marja Pyörälä, Johanna Rimpiläinen, Timo Siitonen, Arno Ylitalo, Marie-Louise Fjällskog, Juho Jalkanen, Sofia Aakko, Inka Pawlitzky, Maija Hollmén, Naval Daver","doi":"10.1016/S2352-3026(25)00103-6","DOIUrl":"10.1016/S2352-3026(25)00103-6","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Bexmarilimab blocks Clever-1 on macrophages to enhance antigen presentation and T cell activation. Because Clever-1 is expressed by myeloid leukaemia cells, bexmarilimab may combat leukaemia and influence the tumour microenvironment to augment the effectiveness of standard-of-care therapy in patients with myelodysplastic syndrome and acute myeloid leukaemia. The aim of this study was to determine the safety of bexmarilimab in combination with standard-of-care treatment in myelodysplastic syndrome and acute myeloid leukaemia and to identify the recommended dose for expansion of bexmarilimab in combination with standard of care.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;The phase 1 dose-escalation part of this multicentre, single-arm, phase 1/2 study was done at six centres in Finland and the USA. Patients aged 18 years or older (Eastern Cooperative Oncology Group performance status of 2 or less) with myelodysplastic syndrome (2016 WHO) with a Revised International Prognostic Scoring System (IPPS-R) score of 3·5 or more for USA (3·0 for the European Union), chronic myelomonocytic leukaemia (2016 WHO) with 10-19% marrow blasts, myelodysplastic syndrome or chronic myelomonocytic leukaemia with no response to or disease progression during hypomethylating agent treatment, or relapsed or refractory acute myeloid leukaemia were treated with escalating doses of bexmarilimab (1·0 mg/kg, 3·0 mg/kg, and 6·0 mg/kg, intravenous, once weekly, 28-day cycle) in combination with azacitidine, administered as per label. Here we report the phase 1 part of the study, for which the primary outcome was safety (the incidence and frequency of dose limiting toxicities and the frequency and severity of adverse events) as well as the determination of the maximum tolerated dose and recommended expansion dose for the phase 2 part using a Bayesian optimal interval design. All patients receiving at least one dose of bexmarilimab were included in safety analyses, and those with a post-baseline activity assessment were included in activity analyses. This trial is registered with ClinicalTrials.gov (NCT05428969) and EudraCT (2021-002104-12) databases. Phase 2 of the study is ongoing in patients with myelodysplastic syndrome with no response to hypomethylating agent.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;Between June 2, 2022, and Dec 7, 2023, 33 patients (14 with myelodysplastic syndrome, 19 with relapsed or refractory acute myeloid leukaemia) were enrolled in phase 1; no patients with chronic myelomonocytic leukaemia were identified. 19 (58%) patients were male and 14 (42%) were female, and 24 (73%) patients were non-Hispanic ethnicity, and eight (24%) were White. Median follow-up time for all patients was 6·2 months (IQR 3·5-10·7). The maximum tolerated dose was not reached, and the recommended expansion dose for phase 2 was established as 6·0 mg/kg in patients with myelodysplastic syndrome with no response to hypomethylating agents. There were no dose-limiting to","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":" ","pages":"e516-e528"},"PeriodicalIF":15.4,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144192379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}