Lancet Haematology最新文献

{"title":"Ask, do not tell: consulting a patient advisory board to understand unmet needs of patients with GVHD in Europe.","authors":"Hélène Schoemans, Elke Stienissen, Kathy Goris, Susan K Stewart, Meredith Cowden, Kristina Arnahoutova, Steven Z Pavletic, Hildegard Greinix, Anna Barata, Lorna Warwick, Natacha Bolanos, Isabel Barbosa, Guy Bouguet, Isabelle Lhenry-Yvon, Riikka-Leena Manninen, Simona Pavukova, Guy Tavori, Marleen van Amerongen, Anita Lawitschka, Kirk R Schultz, Daniel Wolff, Anne Herrmann","doi":"10.1016/S2352-3026(24)00357-0","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00357-0","url":null,"abstract":"<p><p>Graft-versus-host disease (GVHD) is a complication of hematopoietic cell transplantation (HCT) that has a low chance of complete remission and a substantial effect on morbidity and mortality. To better understand how to improve the field of GVHD research, management, and care, the cGVHD Eurograft Initiative organised a European community advisory board of patient advocates, with the assistance of the Lymphoma Coalition, to identify unmet needs. We present the results of this project in this Viewpoint, which identify unmet GVHD needs from the patient advocates' perspectives and provide five key actionable themes to improve GVHD management and care. The identified themes were: the need for reliable and tailored information, increased patient empowerment, access to professional dedicated care, optimal emotional support, and attention to the financial implications of GVHD, with improved communication as an overarching theme. This first step in patient-centred research opens the way to future collaborative initiatives by merging stakeholder perspectives to strive for better care and outcomes after HCT by addressing the most pertinent patient needs.</p>","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 3","pages":"e214-e223"},"PeriodicalIF":15.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568581","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":"Epistemic limitations of measurable residual disease in haematological malignancies.","authors":"Benjamin Chin-Yee, Lucie Laplane, Pierre Sujobert","doi":"10.1016/S2352-3026(25)00002-X","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00002-X","url":null,"abstract":"<p><p>The growing use of measurable residual disease (MRD) assays across haematology-oncology creates an urgent need for clinicians and researchers to reflect on the biological and clinical rationale of this class of biomarkers. In this Viewpoint, we critically examine two premises behind MRD's use in haematology-oncology, focusing on its biological plausibility as a predictive biomarker and surrogate endpoint, and the evidence needed for it to influence decision making in haematological cancers. Examining these premises leads us to advocate for the establishment of more robust biological and clinical evidence to ensure the clinically useful and safe application of MRD. Although achieving the eradication of cancer cells in the form of undetectable MRD seems an attractive goal in haematology-oncology, we highlight the epistemic limitations of this biomarker and need for more clinical evidence to guide its effective use.</p>","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 3","pages":"e224-e229"},"PeriodicalIF":15.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568589","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":"Long-term neurodevelopmental outcomes in infants with haemolytic disease of the newborn treated with darbepoetin.","authors":"Derek P de Winter, Isabelle M C Ree, Masja de Haas, E J T Joanne Verweij, Enrico Lopriore, Jeanine M M van Klink","doi":"10.1016/S2352-3026(25)00030-4","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00030-4","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 3","pages":"e169-e170"},"PeriodicalIF":15.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568593","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":"Do we need another TKI for chronic myeloid leukaemia?","authors":"Henrik Hjorth-Hansen","doi":"10.1016/S2352-3026(24)00373-9","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00373-9","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 3","pages":"e167-e168"},"PeriodicalIF":15.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568587","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":"Global paediatric haematopoietic stem-cell transplantation training and access: providing strategies to overcome challenges.","authors":"Candice Laverne Hendricks, Manuela Spadea, Justin Rudolph Du Toit, Carmelo Gurnari, Rafaella Muratori, Valentine Jimenez, Vera Radici, Sheila Torrado, Nihar Desai, Azada Ibrahimova, Ibrahim N Muhsen, Julio Ropero, Nico Gagelmann, Claire Horgan","doi":"10.1016/S2352-3026(25)00042-0","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00042-0","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 3","pages":"e178-e179"},"PeriodicalIF":15.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568592","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":"Vodobatinib for patients with Philadelphia chromosome-positive chronic myeloid leukaemia resistant or intolerant to multiple lines of previous therapy: an open-label, multicentre, phase 1/2 trial.","authors":"Jorge E Cortes, Dong-Wook Kim, Tapan Saikia, Navin Khattry, Krishnakumar Rathnam, Yesid Alvarado, Guy Hannah, Srinivas K Tantravahi, Jane F Apperley, Aude Charbonnier, Valentin García-Gutiérrez, Alessandro Lucchesi, Delia Dima, Árpád Illés, Viola M Popov, Elisabetta Abruzzese, Arijit Nag, Shashikant Apte, Talha Badar, Siu-Long Yao, Unnati Saxena, Jayasree Sreenivasan, Sandeep Inamdar, Geetanjali Chimote, Franck E Nicolini","doi":"10.1016/S2352-3026(24)00354-5","DOIUrl":"10.1016/S2352-3026(24)00354-5","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Resistance or intolerance to the available tyrosine kinase inhibitors (TKIs) remains a treatment challenge for patients with chronic myeloid leukaemia. We aimed to report the safety, antileukaemic activity, and pharmacokinetics of oral vodobatinib, a novel selective BCR::ABL1 TKI, in patients with Philadelphia chromosome-positive (Ph-positive) chronic myeloid leukaemia who previously received at least three TKIs, including ponatinib and asciminib.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This open-label, multicentre, phase 1/2 trial was conducted at 28 clinical sites across ten countries (Belgium, France, Hungary, India, Italy, Romania, South Korea, Spain, UK, and the USA). Patients aged 18 years or older with Ph-positive chronic myeloid leukaemia or acute lymphoblastic leukaemia (eligible only for the phase 1 study), and an Eastern Cooperative Oncology Group performance status of 2 or lower were eligible. Phase 1 included patients who previously received at least three TKIs or had no other available treatment options. Phase 2 required patients to have treatment resistance or intolerance (or both) with loss of response to at least three TKIs and previous ponatinib use. A key exclusion criterion for both phases was presence of the Thr315Ile mutation. Patients self-administered oral vodobatinib (12-240 mg) once per day for each 28-day treatment cycle and for up to 60 months (ie, 65 cycles) unless patient discontinuation due to adverse events, progressive disease, lost to follow-up, or death. The primary endpoints were to determine the maximum tolerated dose (based on dose-limiting toxicities in phase 1) and antileukaemic activity of vodobatinib (ie, major cytogenetic response for chronic-phase and major haematological response for accelerated-phase or blast-phase in phase 2). Assessment of vodobatinib safety, activity, and pharmacokinetics were determined based on the pooled analysis of data from the phase 1 and 2 studies. This trial is registered with ClinicalTrials.gov, NCT02629692 (active). At data cutoff (July 15, 2023), phase 2 enrolment was closed early on June 22, 2023, due to recruitment-related challenges.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;78 patients were enrolled and received at least one vodobatinib dose (safety and efficacy analysis set). Between April 6, 2017, and June 20, 2023, phase 1 enrolled 58 patients and phase 2 enrolled 20 patients between March 3, 2020, and March 29, 2023. We included 66 (85%) patients with chronic-phase, eight (10%) with accelerated-phase, and four (5%) with blast-phase chronic myeloid leukaemia. 43 (55%) of 78 patients were male and 35 (45%) were female. The median age was 59·0 years (IQR 47·0-66·0). The median follow-up was 22·3 months (IQR 11·1-43·9). Two patients receiving vodobatinib 240 mg had dose-limiting toxicities (one had grade 3 dyspnoea and the other had grade 2 fluid overload), thus the 204 mg dose was considered to be the maximum tolerated dose. 73 (94%) patients had one ","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":" ","pages":"e201-e213"},"PeriodicalIF":15.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143392246","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":"Expanding treatment options for warm autoimmune haemolytic anaemia.","authors":"Sigbjørn Berentsen","doi":"10.1016/S2352-3026(24)00349-1","DOIUrl":"10.1016/S2352-3026(24)00349-1","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":" ","pages":"e84-e85"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142972862","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":"Pioneering a new field of computational pharmacophenomics to unlock the life-saving potential of existing medicines.","authors":"David C Fajgenbaum, Sally Nijim, Grant Mitchell, Matej Macak, Chris Bizon, Alexander Tropsha, David Koslicki","doi":"10.1016/S2352-3026(24)00278-3","DOIUrl":"10.1016/S2352-3026(24)00278-3","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e94-e96"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256971","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":"Improved survival and enhanced quality of life through anaemia correction in lower risk myelodysplastic syndromes: meaningful insights from an EUMDS Registry study.","authors":"Pasquale Niscola","doi":"10.1016/S2352-3026(24)00355-7","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00355-7","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e88-e90"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256964","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":"Position statement on the diagnosis and management of acute leukaemia and aggressive lymphomas in pregnancy.","authors":"Georgia Mills, Antonia Shand, Debra Kennedy, Sandra Lowe, Victoria Bilsland, Briony Cutts, Bruce McBride, Wendy Brown, Srinivas Bolisetty, Eva A Wegner, Giselle Kidson-Gerber","doi":"10.1016/S2352-3026(24)00309-0","DOIUrl":"10.1016/S2352-3026(24)00309-0","url":null,"abstract":"<p><p>Haematological malignancies affect 12·5 in 100 000 pregnancies. Over the past two decades, the number of haematological malignancies in pregnancy has substantially increased. Life-threatening haematological malignancies in pregnancy, such as acute leukaemia and aggressive lymphomas, pose a unique therapeutic challenge: clinicians must consider both maternal and fetal wellbeing, aiming to deliver optimal curative therapy for the patient and a successful pregnancy outcome. A multidisciplinary approach to disease management is paramount, and there are currently no clinical practice guidelines available. An Australasian working group, including representatives from haematology, obstetric medicine, clinical teratology, radiology, nuclear medicine, maternal-fetal medicine, and a patient representative, was established to develop this position statement, which is based on a combination of current evidence and expert consensus. We provide recommendations for diagnosis and staging, imaging safety in pregnancy, therapy in pregnancy incorporating a multidisciplinary approach, supportive care, oncofertility, and pregnancy and birth management. This Viewpoint was reviewed and endorsed by the councils of the Society of Obstetric Medicine of Australia and New Zealand, the Haematology Society of Australia and New Zealand, and the Haematology in Obstetric and Women's Health Collaborative.</p>","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e151-e162"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256972","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}