Lancet Haematology最新文献

{"title":"Inclusion matters: progress in haematological trials for pregnant individuals.","authors":"The Lancet Haematology","doi":"10.1016/S2352-3026(25)00006-7","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00006-7","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e83"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256966","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":"Isatuximab, bortezomib, lenalidomide, and limited dexamethasone in patients with transplant-ineligible multiple myeloma (REST): a multicentre, single-arm, phase 2 trial.","authors":"Frida Bugge Askeland, Einar Haukås, Tobias S Slørdahl, Anja Klostergaard, Tatjana Alexandersen, Anna Lysén, Pegah Abdollahi, Lene Kongsgaard Nielsen, Emil Hermansen, Fredrik Schjesvold","doi":"10.1016/S2352-3026(24)00347-8","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00347-8","url":null,"abstract":"<p><strong>Background: </strong>Adding anti-CD38 monoclonal antibodies to standard therapies can improve outcomes in patients with multiple myeloma. Long-term treatment with corticosteroids increases the risk of infection. We aimed to evaluate the safety and activity of isatuximab, weekly bortezomib, lenalidomide, and limited dexamethasone in patients with newly diagnosed multiple myeloma ineligible for autologous haematopoietic stem-cell transplantation (HSCT).</p><p><strong>Methods: </strong>REST is an academic, multicentre, single-arm, phase 2 trial of adults with newly diagnosed multiple myeloma and measurable disease as defined by International Myeloma Working Group criteria, ineligible for high-dose melphalan and autologous HSCT, Eastern Cooperative Oncology Group performance status of 0-3 (with 3 only allowed if related to myeloma). In 28-day cycles, patients received isatuximab (10 mg/kg intravenously on days 1, 8, 15, and 22 of cycle 1, and days 1 and 15 of cycles 2-18), bortezomib (1·3 mg/m<sup>2</sup> subcutaneously on days 1, 8, and 15 of cycles 1-8), and lenalidomide (25 mg orally on days 1-21, until progressive disease). Dexamethasone was given 20 mg orally on days 1, 8, 15 and 22, limited to the two first cycles only. The primary endpoint was measurable residual disease (MRD)-negative complete response, assessed by next-generation flow cytometry (sensitivity 1·0 × 10<sup>-5</sup>), during or after 18 cycles of study treatment. MRD was tested in all patients who had at least complete response before cycle 19 and in all patients who had at least very good partial response at cycle 19. All patients enrolled initiated treatment and were included in the analyses. This trial is registered with ClinicalTrials.gov (NCT04939844); the primary endpoint is reported in this Article, and follow-up is ongoing.</p><p><strong>Findings: </strong>Between June 30, 2021 and Jan 19, 2023, we assessed for eligibility and recruited 51 patients (27 [53%] females and 24 [47%] males), with a median age of 77 years (IQR 73·5-80). 39 participants completed 18 cycles of treatment on protocol, of whom two had discontinued treatment but not protocol. At a median follow-up of 27·0 months (IQR 23·0-33·7), MRD-negative complete response was observed in 19 (37% [95% CI 25·3-51·0]) patients, with a median treatment duration of 22 months (IQR 15·2-28·8; range 1·4-35·1). Disease progression or death had occurred in 18 (35%) of 51 patients, and eight (16%) patients had died. During the first 18 cycles of study treatment, the most common adverse events of grade 3 or 4 were neutropenia (28 [55%] patients), infections (21 [41%] patients), and thrombocytopenia (11 [22%] patients). 48 serious adverse events of grade 3 or higher were reported in 27 (53%) patients. A total of 14 (27%) patients discontinued treatment before cycle 19, most commonly because of progressive disease (eight [16%]) and adverse events (four [8%]). Two deaths (one due to pneumonia and one due to sepsis) ","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e120-e127"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256967","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":"Survival and quality of life in patients with lower risk myelodysplastic syndromes exposed to erythropoiesis-stimulating agents: an observational cohort study.","authors":"Hege Kristin Gravdahl Garelius, Timothy Bagguley, Adele Taylor, Pierre Fenaux, David Bowen, Argiris Symeonidis, Moshe Mittelmann, Reinhard Stauder, Jaroslav Čermák, Guillermo Sanz, Saskia Langemeijer, Luca Malcovati, Ulrich Germing, Laurence Sanhes, Maud d'Aveni, Dominic Culligan, Ioannis Kotsianidis, Karin A Koinig, Corine van Marrewijk, Simon Crouch, Theo deWitte, Alexandra Smith, Eva Hellström-Lindberg","doi":"10.1016/S2352-3026(24)00350-8","DOIUrl":"10.1016/S2352-3026(24)00350-8","url":null,"abstract":"<p><strong>Background: </strong>In our previous study on erythropoiesis-stimulating agent (ESA) treatment in lower risk myelodysplastic syndromes from the European MDS (EUMDS) Registry, we showed that patients treated with ESAs had longer survival compared with patients who receive red blood cell transfusion (RBCT). In this study, with a longer follow up time and more patients included, we aimed to assess long-term effects on survival and health-related quality of life (HRQoL) of exposure to ESAs with or without RBCT in patients with lower risk myelodysplastic syndromes.</p><p><strong>Methods: </strong>The EUMDS Registry is a non-interventional, longitudinal, real-world registry prospectively enrolling newly diagnosed patients older than 18 years with lower risk (International Prognostic Scoring System low or intermediate-1) myelodysplastic syndromes from 16 European countries and Israel. The analysis was restricted to patients with haemoglobin concentrations less than 100 g/L enrolled between Jan 1, 2008, and July 1, 2019, with last censoring of data on Dec 31, 2021. Patient management was recorded every 6 months, including treatment, transfusions, and HRQoL. ESA treatment followed local guidelines. The patients were separated into four groups at each study visit: no ESA or RBCT, ESA only, ESA plus RBCT, and RBCT only. The data were analysed longitudinally over time according to ESA and RBCT status during each 6-month interval, using propensity score matching. The main outcomes were median overall survival and leukaemia-free survival, and HRQoL. This study is registered with ClinicalTrials.gov, NCT00600860, as is ongoing.</p><p><strong>Findings: </strong>2448 patients (the ESA-unexposed group [n=1265] and ESA-exposed group [n=1183]) were diagnosed before July 1, 2019; 1520 (62·1%) were male and 928 (37·9%) were female. Median follow-up time was 3·9 years (IQR 1·6-6·5). After applying eligibility criteria and propensity matching, there were 426 patients in the ESA-unexposed group and 744 patients in the ESA-exposed group. Median overall survival in the ESA exposed group was 44·9 months (95% CI 40·2-50·5) compared with 34·8 months (28·6-39·2) in the ESA unexposed group; the absolute difference was 10·1 months (95% CI 2·2-18·0; hazard ratio [HR] 0·70 [95% CI 0·59-0·83]; p<0·0001). Patients without RBCT in the presence or absence of ESA exposure maintained significantly better HRQoL than those with RBCT, irrespective of ESA exposure (linear mixed effect model of EQ-5d-3L index score, RBCT coefficient -0·04 [95% CI -0·06 to 0·03], p<0·0001; linear mixed effect model of VAS, -4·57 [-6·02 to -3·13], p<0·0001).</p><p><strong>Interpretation: </strong>ESA treatment in patients with lower risk myelodysplastic syndromes significantly improves overall survival when started before or early after the onset of regular transfusion therapy. Avoiding RBCT is associated with significantly better HRQoL.</p><p><strong>Funding: </strong>H2020 European Research Council","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e128-e137"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11803517/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256973","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cristóbal Frutos: confronting treatment barriers.","authors":"Ray Cavanaugh","doi":"10.1016/S2352-3026(25)00004-3","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00004-3","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e93"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143257046","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":"Diagnosis and treatment of Burkitt lymphoma in adults: clinical practice guidelines from ERN-EuroBloodNet.","authors":"Vincent Ribrag, Dominique Bron, Grzegorz Rymkiewicz, Dieter Hoelzer, Judit Jørgensen, Aythami de Armas-Castellano, Maria Trujillo-Martín, Pierre Fenaux, Luca Malcovati, Natacha Bolaños, Josep-Maria Ribera, Charles Herbaux, Clémentine Sarkozy, Pier Luigi Zinzani, Jan Walewski, Martine E D Chamuleau","doi":"10.1016/S2352-3026(24)00351-X","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00351-X","url":null,"abstract":"<p><p>Burkitt lymphoma is a rare lymphoma entity that represents less than 5% of adult lymphomas. Although prognosis has improved with dose-dense therapy, Burkitt lymphoma remains an area of clinical and biological research with specificities due to the high incidence of CNS involvement and tumour lysis syndrome in patients with a high tumour burden. Few consensus recommendations are available concerning diagnosis, treatment, and prognostic factors in adult patients. In this Review, a European Reference Network on Rare Haematological Diseases (ERN-EuroBloodNet) expert panel has reviewed recent advances in the management of Burkitt lymphoma in the first-line setting to develop updated evidence-based and expert opinion-based recommendations on the management of this disease. The expert panel consisted of ten clinicians and pathologists involved in the clinical management of Burkitt lymphoma from eight EU member states. Additionally, two haematologists were included to support the systematic review process. A balanced representation was ensured between individuals affiliated and not affiliated with ERN-EuroBloodNet. Together with providing current indications on diagnosis and risk-adapted first-line therapy, the Review contains specific recommendations for the identification and management of important complications of Burkitt lymphoma such as tumour lysis syndrome and CNS-oriented therapy, and recommendations for prognostic assessment to guide treatment. Finally, unresolved questions for Burkitt lymphoma are highlighted, including questions around genetics, imaging, and second-line therapies, along with patient perspective.</p>","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e138-e150"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143257048","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":"2024 ASH Annual Meeting.","authors":"Daniela Marín, Lan-Lan Smith","doi":"10.1016/S2352-3026(25)00005-5","DOIUrl":"https://doi.org/10.1016/S2352-3026(25)00005-5","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e91-e92"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143366241","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":"Sovleplenib in patients with primary or secondary warm autoimmune haemolytic anaemia: results from phase 2 of a randomised, double-blind, placebo-controlled, phase 2/3 study.","authors":"Xin Zhao, Jing Sun, Zhihua Zhang, Miao Chen, Tiejun Gong, Guangsheng He, Yingmei Li, Hong Liu, Fei Li, Xin Li, Hu Zhou, Xiaoqin Wang, Mei Hong, Lei Lei, Hongyan Yin, Xian Luo, Yang Li, Songhua Fan, Xiaojun Guo, Michael M Shi, Weiguo Su, Liansheng Zhang, Bing Han, Fengkui Zhang","doi":"10.1016/S2352-3026(24)00344-2","DOIUrl":"10.1016/S2352-3026(24)00344-2","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Spleen tyrosine kinase inhibitors are potential treatment options for warm autoimmune haemolytic anaemia. This study aimed to assess the preliminary efficacy and safety of sovleplenib-an oral spleen tyrosine kinase inhibitor-in patients with warm autoimmune haemolytic anaemia in China. Here we report on the phase 2 results.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This randomised, double-blind, placebo-controlled, phase 2 part from the phase 2/3 study was conducted at 13 centres in China. Eligible patients, aged 18-75 years, with an Eastern Cooperative Oncology Group (ECOG) performance status of no more than 2, had primary or secondary warm autoimmune haemolytic anaemia (stable underlying disease not requiring drug intervention) with no response to previous glucocorticoid treatment, haemoglobin of less than 100 g/L with active haemolysis, and a positive direct antiglobulin test. The study comprised two periods; patients were randomly assigned (3:1) to receive sovleplenib or placebo at 300 mg orally once a day in the 8-week double-blind period. Upon completion, all patients entered an open-label treatment period for at least 16 weeks and received sovleplenib 300 mg once a day until 24 weeks after the last patient was randomly assigned. The primary endpoint for phase 2 of the trial was overall haemoglobin response rate (haemoglobin ≥100 g/L with an increase of ≥20 g/L from baseline at least once, and haemoglobin not affected by rescue therapy, such as red blood cell transfusions, intravenous immunoglobulin, and glucocorticoids) by week 24. Efficacy analyses in the 0-8 week double-blind period included all patients who were randomly assigned, analysed by intention-to-treat. Safety analysis in the double-blind period included patients in the intention-to-treat population who received at least one dose of the study medication. This phase 2/3 study is registered with ClinicalTrials.gov, NCT05535933, and the phase 3 part is ongoing.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Findings: &lt;/strong&gt;Between Sept 26, 2022, and May 9, 2023, 34 patients were screened and 21 patients (four [19%] male and 17 [81%] female) were enrolled in the study and randomly assigned to receive either sovleplenib (n=16) or placebo (n=5). All 21 patients completed the 0-8-week double-blind treatment and entered the open-label treatment period. The overall haemoglobin response rate was 67% (14 of 21 patients) by week 24, and durable haemoglobin response rate was 48% (ten of 21 patients) by week 24. During the 0-8-week double-blind treatment, 13 (81%) of 16 patients in the sovleplenib group versus five (100%) of five patients taking placebo reported treatment-emergent adverse events (TEAEs), and four (25%) of 16 patients versus four (80%) of five patients reported grade 3 adverse events. Although all 21 patients had a TEAE during the 24-week treatment with sovleplenib, only seven (33%) patients had grade 3 events. The most common grade 3 TEAE was anaemia (four [19%] patients), which was","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":" ","pages":"e97-e108"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142972863","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":"Optimising quadruplet regimens to broaden eligibility in multiple myeloma.","authors":"Tanya M Wildes","doi":"10.1016/S2352-3026(24)00356-9","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00356-9","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 2","pages":"e87-e88"},"PeriodicalIF":15.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256969","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":"Patient reported outcomes with earlier use of chimeric antigen receptor-T cell therapy in multiple myeloma.","authors":"Alissa Visram, Hira Mian","doi":"10.1016/S2352-3026(24)00346-6","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00346-6","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 1","pages":"e6-e8"},"PeriodicalIF":15.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142933197","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":"Oreofe Odejide-aiming to raise the bar in haematology care.","authors":"Tony Kirby","doi":"10.1016/S2352-3026(24)00380-6","DOIUrl":"https://doi.org/10.1016/S2352-3026(24)00380-6","url":null,"abstract":"","PeriodicalId":48726,"journal":{"name":"Lancet Haematology","volume":"12 1","pages":"e10"},"PeriodicalIF":15.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142933196","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}