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Moving forward from spleen response as an endpoint in randomized controlled trials in myelofibrosis. 从脾脏反应作为骨髓纤维化随机对照试验的终点向前推进。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-25 DOI: 10.3324/haematol.2025.288712
Ruben Van Dijck,John-John B Schnog,Peter A W Te Boekhorst
{"title":"Moving forward from spleen response as an endpoint in randomized controlled trials in myelofibrosis.","authors":"Ruben Van Dijck,John-John B Schnog,Peter A W Te Boekhorst","doi":"10.3324/haematol.2025.288712","DOIUrl":"https://doi.org/10.3324/haematol.2025.288712","url":null,"abstract":"Anticancer drugs should make patients live longer and/or feel better. Ideally, endpoints of cancer randomized controlled trials (RCTs) should demonstrate that a drug leads to an increase in overall survival (OS) and/or improvement in quality of life (QOL). With the aim of including smaller patient numbers, running shorter trials and thus getting new drugs to patients faster, cancer RCTs increasingly use (putative) surrogate endpoints. However, changes in surrogate endpoints often do not reliably predict improvements in OS and/or QOL. Furthermore, especially in later lines of cancer treatments or in cancer with a short life expectancy, use of surrogates does hardly speed up the availability of novel therapies but increases the advent of costly toxic drugs with uncertain benefit, thereby harming both patients and society. In myelofibrosis (MF) spleen response has extensively been used as a surrogate for clinical outcome. In this review we argue that there is no convincing evidence for the use of spleen response or other surrogate endpoints in MF, and that the use of surrogate endpoints in MF RCTs should be avoided altogether.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"41 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145134036","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}
引用次数: 0
Rapid and durable recovery of immune effector cells in myelofibrosis patients treated with momelotinib. 莫米洛替尼治疗骨髓纤维化患者免疫效应细胞的快速持久恢复。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-25 DOI: 10.3324/haematol.2025.288515
Jasmine Makker,Kyaw Htin Thaw,Amna Sheikh,Llywelyn Cadman-Davies,Andrea Duminuco,Elena Torre,Margherita Panetta,Conor Stanley,Hugues De Lavallade,Natalia Curto-Garcia,Priya Sriskandarajah,Jennifer O'Sullivan,Deepti H Radia,Claire Woodley,Benissa Narciso,Susan Asirvatham,Bethan Psaila,Shahram Kordasti,Claire Harrison,Patrick Harrington
{"title":"Rapid and durable recovery of immune effector cells in myelofibrosis patients treated with momelotinib.","authors":"Jasmine Makker,Kyaw Htin Thaw,Amna Sheikh,Llywelyn Cadman-Davies,Andrea Duminuco,Elena Torre,Margherita Panetta,Conor Stanley,Hugues De Lavallade,Natalia Curto-Garcia,Priya Sriskandarajah,Jennifer O'Sullivan,Deepti H Radia,Claire Woodley,Benissa Narciso,Susan Asirvatham,Bethan Psaila,Shahram Kordasti,Claire Harrison,Patrick Harrington","doi":"10.3324/haematol.2025.288515","DOIUrl":"https://doi.org/10.3324/haematol.2025.288515","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"44 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145134078","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}
引用次数: 0
The co-inheritance of two ITGB3 variants with additive detrimental effects on platelets leads to variant Glanzmann thrombasthenia. 两种对血小板有害的ITGB3变异的共同遗传导致变异型Glanzmann血栓缺失。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-25 DOI: 10.3324/haematol.2025.288369
Nina Arndt,Emanuela Falcinelli,Ana Zamora-Canovas,Ana Sanchez Fuentes,Ana Marin-Quilez,Maria Del Mar Nieto-Hernandez,Jose Rivera,Paolo Gresele,Loredana Bury
{"title":"The co-inheritance of two ITGB3 variants with additive detrimental effects on platelets leads to variant Glanzmann thrombasthenia.","authors":"Nina Arndt,Emanuela Falcinelli,Ana Zamora-Canovas,Ana Sanchez Fuentes,Ana Marin-Quilez,Maria Del Mar Nieto-Hernandez,Jose Rivera,Paolo Gresele,Loredana Bury","doi":"10.3324/haematol.2025.288369","DOIUrl":"https://doi.org/10.3324/haematol.2025.288369","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"57 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145134037","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}
引用次数: 0
Alterations in calreticulin and CD47 expression dynamics in myeloid malignancies: therapeutic and prognostic implications in myelodysplastic syndromes and myeloproliferative neoplasms. 骨髓恶性肿瘤中钙调钙蛋白和CD47表达动态的改变:骨髓增生异常综合征和骨髓增生性肿瘤的治疗和预后意义
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-25 DOI: 10.3324/haematol.2025.288166
Kristian Boasman,Claire L Walker,Matthew J Simmonds,Ciro R Rinaldi
{"title":"Alterations in calreticulin and CD47 expression dynamics in myeloid malignancies: therapeutic and prognostic implications in myelodysplastic syndromes and myeloproliferative neoplasms.","authors":"Kristian Boasman,Claire L Walker,Matthew J Simmonds,Ciro R Rinaldi","doi":"10.3324/haematol.2025.288166","DOIUrl":"https://doi.org/10.3324/haematol.2025.288166","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"41 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145133934","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}
引用次数: 0
Enhancing myeloablative fludarabine-busulfan conditioning with total marrow irradiation in high-risk myeloid disease. 全骨髓照射增强高危髓系疾病的清髓性氟达拉滨-布磺胺调节作用。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-25 DOI: 10.3324/haematol.2025.288307
Fernando Molina,Alberto Mussetti,Anna Sureda
{"title":"Enhancing myeloablative fludarabine-busulfan conditioning with total marrow irradiation in high-risk myeloid disease.","authors":"Fernando Molina,Alberto Mussetti,Anna Sureda","doi":"10.3324/haematol.2025.288307","DOIUrl":"https://doi.org/10.3324/haematol.2025.288307","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"11 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145134034","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}
引用次数: 0
Venetoclax plus hypomethylating agents as a bridge to transplant or donor lymphocyte infusion in relapsed/refractory acute myeloid leukemia. Venetoclax联合低甲基化药物作为复发/难治性急性髓性白血病移植或供体淋巴细胞输注的桥梁。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-18 DOI: 10.3324/haematol.2025.288178
Kathelijn Verdeyen,Tom Reuvekamp,Walter J F M Van der Velden,Bas J Wouters,Peter A Von dem Borne,Anna Van Rhenen,Birgit I Lissenberg-Witte,Daniëlle Van Lammeren,Geerte L Van Sluis,Eva De Jongh,Roel B Fiets,Maarten F Corsten,Arie C Van der Spek,Anke M Gerrits,Esther R Van Bladel,Lidwine W Tick,Okke De Weerdt,Bregje Van Zaane,Marjan J Cruijsen,Eduardus F M Posthuma,Catharina H M J Van Elssen,Saskia K Klein,Arjan A Van de Loosdrecht,David C De Leeuw
{"title":"Venetoclax plus hypomethylating agents as a bridge to transplant or donor lymphocyte infusion in relapsed/refractory acute myeloid leukemia.","authors":"Kathelijn Verdeyen,Tom Reuvekamp,Walter J F M Van der Velden,Bas J Wouters,Peter A Von dem Borne,Anna Van Rhenen,Birgit I Lissenberg-Witte,Daniëlle Van Lammeren,Geerte L Van Sluis,Eva De Jongh,Roel B Fiets,Maarten F Corsten,Arie C Van der Spek,Anke M Gerrits,Esther R Van Bladel,Lidwine W Tick,Okke De Weerdt,Bregje Van Zaane,Marjan J Cruijsen,Eduardus F M Posthuma,Catharina H M J Van Elssen,Saskia K Klein,Arjan A Van de Loosdrecht,David C De Leeuw","doi":"10.3324/haematol.2025.288178","DOIUrl":"https://doi.org/10.3324/haematol.2025.288178","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"49 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145078135","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}
引用次数: 0
Brentuximab vedotin plus nivolumab as bridging therapy to CAR T-cells in relapsed/refractory primary mediastinal B-cell lymphoma. Brentuximab vedotin联合nivolumab作为CAR - t细胞桥接疗法治疗复发/难治性原发性纵隔b细胞淋巴瘤。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-18 DOI: 10.3324/haematol.2025.287672
Armando Santoro,Ciro Maria Improta,Barbara Sarina,Daniele Mannina,Chiara De Philippis,Daniela Taurino,Jacopo Mariotti,Monica Balzarotti,Stefania Bramanti
{"title":"Brentuximab vedotin plus nivolumab as bridging therapy to CAR T-cells in relapsed/refractory primary mediastinal B-cell lymphoma.","authors":"Armando Santoro,Ciro Maria Improta,Barbara Sarina,Daniele Mannina,Chiara De Philippis,Daniela Taurino,Jacopo Mariotti,Monica Balzarotti,Stefania Bramanti","doi":"10.3324/haematol.2025.287672","DOIUrl":"https://doi.org/10.3324/haematol.2025.287672","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"4 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145078137","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}
引用次数: 0
Response to Comment on: Selection of unrelated donors for allogeneic transplantation using post-transplant cyclophosphamide in acute lymphoblastic leukemia: an analysis by the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. 对《选择非亲属供体进行急性淋巴细胞白血病移植后环磷酰胺异体移植:欧洲血液和骨髓移植学会急性白血病工作组的分析》评论的回应。
IF 7.9 1区 医学
Haematologica Pub Date : 2025-09-18 DOI: 10.3324/haematol.2025.289110
Jaime Sanz, Mohamad Mohty, Fabio Ciceri
{"title":"Response to Comment on: Selection of unrelated donors for allogeneic transplantation using post-transplant cyclophosphamide in acute lymphoblastic leukemia: an analysis by the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.","authors":"Jaime Sanz, Mohamad Mohty, Fabio Ciceri","doi":"10.3324/haematol.2025.289110","DOIUrl":"https://doi.org/10.3324/haematol.2025.289110","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":7.9,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145080261","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}
引用次数: 0
From myeloma to POEMS: extending the potential of T-cell redirecting therapies. 从骨髓瘤到POEMS:扩展t细胞重定向治疗的潜力。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-18 DOI: 10.3324/haematol.2025.288466
Joselle Cook,Angela Dispenzieri
{"title":"From myeloma to POEMS: extending the potential of T-cell redirecting therapies.","authors":"Joselle Cook,Angela Dispenzieri","doi":"10.3324/haematol.2025.288466","DOIUrl":"https://doi.org/10.3324/haematol.2025.288466","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"73 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145078134","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}
引用次数: 0
Anti-HLA class I IgG subclasses skew platelet activation mechanisms in transfusion refractoriness. 抗hla I类IgG亚类在输血难治性中扭曲血小板激活机制。
IF 10.1 1区 医学
Haematologica Pub Date : 2025-09-18 DOI: 10.3324/haematol.2025.287677
Adèle Couvidou,Mathieu Wald,Catherine Angénieux,Juliana Pires-Marafon,Marie-Joëlle Apithy,Catherine Humbrecht,Luc-Matthieu Fornecker,Nicolas Congy-Jolivet,Arnaud Dupuis,Jérôme Rollin,Blandine Maître
{"title":"Anti-HLA class I IgG subclasses skew platelet activation mechanisms in transfusion refractoriness.","authors":"Adèle Couvidou,Mathieu Wald,Catherine Angénieux,Juliana Pires-Marafon,Marie-Joëlle Apithy,Catherine Humbrecht,Luc-Matthieu Fornecker,Nicolas Congy-Jolivet,Arnaud Dupuis,Jérôme Rollin,Blandine Maître","doi":"10.3324/haematol.2025.287677","DOIUrl":"https://doi.org/10.3324/haematol.2025.287677","url":null,"abstract":"Patients requiring recurrent platelet transfusions are subject to platelet transfusion refractoriness (PTR), a therapeutic failure due to rapid clearance of transfused platelets from the circulation. One major cause of PTR is the presence in the recipient of multiple IgG directed against allogeneic HLA Class-I (HLA-I) molecules expressed by the donor platelets. Strikingly, the presence of anti-HLA-I IgG does not necessarily correlate with PTR, thus questioning the role of the antibody properties themselves. Using blood of HLA-I alloimmunized patients with or without PTR, we identified the subclasses of their anti-HLA-I IgG. We found the distribution of these subclasses to be different according to patients, with IgG1 being predominant in non-PTR patients while IgG1 in combination with IgG2 or IgG3 were detected in PTR patients. To understand the mechanisms associated with PTR, we used human chimeric pan-HLA-I IgG1, IgG2, or IgG3 antibodies and assessed the functional implications of these human IgG subclasses on platelet activation. We showed that each subclass led to platelet aggregation, P-selectin exposure and Annexin V binding. However, we found that the mechanisms of platelet activation differed between subclasses. Specifically, we discovered that pan-HLA-I hIgG2-induced platelet activation was CD32a dependent, while hIgG1- and hIgG3-induced platelet activation relied on complement recruitment. Hence, this study may have direct implications for hierarchizing pathogenic anti-HLA-I alloantibodies in highly polyimmunized patients and be a valuable aid in selecting suitable treatments, particularly by streamlining the search for functionally compatible platelet components.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"1 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145078138","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}
引用次数: 0
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