Haematologica最新文献_第4页

A phase II trial of azacitidine with ipilimumab, nivolumab, or ipilimumab and nivolumab in previously untreated myelodysplastic syndrome. 阿扎胞苷联合伊匹单抗、纳武单抗或伊匹单抗和纳武单抗治疗未经治疗的骨髓增生异常综合征的II期临床试验。

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-02-20 DOI: 10.3324/haematol.2024.286559

Ian M Bouligny, Guillermo Montalban-Bravo, Koji Sasaki, Naval Daver, Elias Jabbour, Yesid Alvarado, Courtney D DiNardo, Farhad Ravandi, Gautam Borthakur, Prithviraj Bose, Naveen Pemmaraju, Steven Kornblau, Tapan Kadia, Lucia Masarova, Koichi Takahashi, Michael Andreeff, Alexandre Bazinet, Hui Yang, Rashmi Kanagal-Shamanna, Chitra Hosing, Sherry Pierce, Meghan Meyer, Xuelin Huang, Guillermo Garcia-Manero

引用次数: 0

Increased RhoA pathway activation downstream of αIIbβ3/SRC contributes to heterozygous Bernard Soulier syndrome. α ib β3/SRC下游RhoA通路激活增加有助于杂合性Bernard Soulier综合征。

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-03-06 DOI: 10.3324/haematol.2024.286424

Larissa Lordier, Christian A Di Buduo, Alexandre Kauskot, Nathalie Balayn, Cécile Lavenu-Bombled, Francesco Baschieri, Valérie Proulle, Cecilia P Marin Oyarzun, Francesca Careddu, Ida Biunno, Tudor Manoliu, Philippe Rameau, Isabelle Plo, Nicolas Papadopoulos, Stefan Constantinescu, William Vainchenker, Guillaume Nam Nguyen, Paola Ballerini, Remi Favier, Alessandra Balduini, Hana Raslova

{"title":"Increased RhoA pathway activation downstream of αIIbβ3/SRC contributes to heterozygous Bernard Soulier syndrome.","authors":"Larissa Lordier, Christian A Di Buduo, Alexandre Kauskot, Nathalie Balayn, Cécile Lavenu-Bombled, Francesco Baschieri, Valérie Proulle, Cecilia P Marin Oyarzun, Francesca Careddu, Ida Biunno, Tudor Manoliu, Philippe Rameau, Isabelle Plo, Nicolas Papadopoulos, Stefan Constantinescu, William Vainchenker, Guillaume Nam Nguyen, Paola Ballerini, Remi Favier, Alessandra Balduini, Hana Raslova","doi":"10.3324/haematol.2024.286424","DOIUrl":"10.3324/haematol.2024.286424","url":null,"abstract":"Bernard Soulier syndrome (BSS) is a severe bleeding disorder with moderate to severe thrombocytopenia, giant platelets, and platelet dysfunction, caused by biallelic mutations in GP1BA, GP1BB, or GP9 genes. We generated induced pluripotent stem cells (iPSC) from a BSS patient with a novel heterozygous GP1BA p.N103D mutation, resulting in moderate macrothrombocytopenia. The mutation does not affect megakaryocyte (MK) differentiation or GPIb-GPIX complex expression but reduces affinity to von Willebrand factor (VWF). It induces increased signaling independent of VWF and αIIbβ3-mediated outside-in signaling, causing a profound defect in proplatelet formation after adhesion on fibrinogen. Pre-activation of αIIbβ3 integrin and heightened stress fiber formation linked to RhoA pathway overactivation were observed, likely due to increased phosphorylation of SRC at Y419 downstream of GPIbα. Dasatinib, a SRC inhibitor, restored stress fiber formation. Using a 3D bone marrow model to mimic platelet release under flow, we demonstrated that the ROCK1/2 inhibitor Y27632 increased platelet number and restored platelet size in GPIbαN103D MK, as well as in MK from two other patients with heterozygous GP1BA mutations (p.L160P and p.N150S). However, Y27632 had no additional effect on platelet generation from MK of two patients with biallelic BSS, suggesting a distinct molecular mechanism in biallelic cases.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1596-1609"},"PeriodicalIF":8.2,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12208166/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143566566","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}

引用次数: 0

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2024-12-12 DOI: 10.3324/haematol.2024.286152

Silvia García Adrián, Claudia Iglesias Pérez, Alberto Carmona-Bayonas, Laura Ortega Morán, Jaime Rubio Pérez, Purificación Martínez Del Prado, Eva Martínez De Castro, Fernando Neria, Isaura Fernández Pérez, Marta García De Herreros, Marta Carmona Campos, Ignacio García Escobar, Rut Porta Balanyà, David Marrupe González, Paula Jiménez Fonseca, María Esperanza Guirao García, Manuel Sánchez Cánovas, José Muñoz Langa, Pedro Pérez Segura, Ma José Méndez Vidal, Andrés J Muñoz Martín, Spanish Society Of Medical Oncology

{"title":"Cancer-related thrombosis: impact of biological sex on the risk of rethrombosis and bleeding.","authors":"Silvia García Adrián, Claudia Iglesias Pérez, Alberto Carmona-Bayonas, Laura Ortega Morán, Jaime Rubio Pérez, Purificación Martínez Del Prado, Eva Martínez De Castro, Fernando Neria, Isaura Fernández Pérez, Marta García De Herreros, Marta Carmona Campos, Ignacio García Escobar, Rut Porta Balanyà, David Marrupe González, Paula Jiménez Fonseca, María Esperanza Guirao García, Manuel Sánchez Cánovas, José Muñoz Langa, Pedro Pérez Segura, Ma José Méndez Vidal, Andrés J Muñoz Martín, Spanish Society Of Medical Oncology","doi":"10.3324/haematol.2024.286152","DOIUrl":"10.3324/haematol.2024.286152","url":null,"abstract":"Patients with cancer have a higher risk of re-thrombosis and bleeding secondary to anticoagulant treatment than have individuals without cancer. Given the lack of specific clinical trials, the decision regarding the optimal duration of treatment must consider multiple factors, including sex. The current study used data from the international, prospective TESEO Registry that includes consecutive patients diagnosed with cancer-associated thrombosis. Between July 2018 and December 2022, 2,823 patients were included in the TESEO Registry, 1,351 (48%) of whom were female. The most common venous thromboembolic event (VTE) in both sexes was pulmonary embolism, with an incidence of 58.0% among men and 54.3% in women (P=0.045). After a median follow-up of 6.9 months (interquartile range, 1.9-14.4), the re-thrombosis rate at the end of follow-up was 10.0% in males and 15.0% in females (P=0.14). The location of the primary tumor in the gastrointestinal tract was associated with a greater risk of re-thrombosis, whereas sex had no significant impact. Men had twice as many major bleeds. Additional risk factors for major bleeding included situations of risk due to tumor site or thrombocytopenia, as well as the presence of active tumor bleeding at the time of VTE diagnosis. Overall survival was longer among women. Given the higher incidence of major bleeding among men, sex should be deemed a relevant factor when deciding the duration of anticoagulant treatment in cancer patients.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1513-1522"},"PeriodicalIF":8.2,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12208159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812935","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}

引用次数: 0

The improved prognosis of FLT3-internal tandem duplication but not tyrosine kinase domain mutations in acute myeloid leukemia in the era of targeted therapy: a real-world study using large-scale electronic health record data. 靶向治疗时代急性髓性白血病flt3 -内部串联重复而非酪氨酸激酶结构域突变的预后改善：一项使用大规模电子健康记录数据的现实世界研究

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-02-06 DOI: 10.3324/haematol.2024.286695

Matthew Schwede, Gladys Rodriguez, Vanessa E Kennedy, Solomon Henry, Douglas Wood, Gabriel N Mannis, Ravindra Majeti, Jonathan H Chen, Eran Bendavid, Tian Yi Zhang

引用次数: 0

Oncogenic NTRK3 mutations exhibit differential sensitivity to tropomyosin receptor kinase inhibitors in patients with acute myeloid leukemia. 致瘤性NTRK3突变在急性髓性白血病患者中对原肌球蛋白受体激酶抑制剂表现出不同的敏感性。

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-02-13 DOI: 10.3324/haematol.2024.285572

Sunil K Joshi, Ariane Huang, Janét Pittsenbarger, Ujwal Shinde, Camilo Posso, Paul D Piehowski, Sara J C Gosline, Richard D Press, Marina A Gritsenko, Chelsea Hutchinson, Karl K Weitz, Kevin Watanabe-Smith, Nicola Long, Karin D Rodland, Jeffrey W Tyner, Brian J Druker, Cristina E Tognon

引用次数: 0

Venetoclax in combination with hypomethylating agents in newly diagnosed and relapsed CBFβ::MYH11⁺ acute myeloid leukemia. Venetoclax联合低甲基化药物治疗新诊断和复发的CBFβ::MYH11+急性髓性白血病

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-02-20 DOI: 10.3324/haematol.2024.286836

Ziyu Feng, Xiaohui Hu, Weiyang Li, Ting Xu, Yang Xu, Shengli Xue, Huiying Qiu, Xiaowen Tang, Yue Han, Suning Chen, Aining Sun, Yanming Zhang, Depei Wu, Ying Wang

引用次数: 0

Plasma exchange for hyperammonemia-induced reduced consciousness after PEG-asparaginase in an adult patient with acute lymphoblastic leukemia. 血浆置换治疗急性淋巴细胞白血病成人患者经peg -天冬酰胺酶治疗后高氨血症诱导的意识降低。

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-03-13 DOI: 10.3324/haematol.2024.286972

Felix Klingler, Anika Forstreuter, Luisa Stegat, Sofie Kämereit, Felix Ullrich, Florian Udonta, Christian Krebs, Dominik Wichmann, Carsten Bokemeyer, Nicola Goekbuget, Walter Fiedler, Franziska Modemann

引用次数: 0

New insights into the generation and function of megakaryocytes in health and disease. 巨核细胞在健康和疾病中的产生和功能的新见解。

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-03-27 DOI: 10.3324/haematol.2024.287236

Changhong Du, Jun Chen, Junping Wang

{"title":"New insights into the generation and function of megakaryocytes in health and disease.","authors":"Changhong Du, Jun Chen, Junping Wang","doi":"10.3324/haematol.2024.287236","DOIUrl":"10.3324/haematol.2024.287236","url":null,"abstract":"Megakaryocytes are canonically viewed as specialized hematopoietic cells that merely produce platelets and that are generated through a series of hematopoietic stem and progenitor cells in the bone marrow. Despite their essential physiological functions, the generation and function of megakaryocytes remain incompletely understood. Recent studies, mostly in mice, have begun to redefine the cellular hierarchy of megakaryopoiesis, and shed new light on the alternative routes and mechanisms of megakaryopoiesis. Moreover, the conception of megakaryocytes as a homogeneous cell population with the sole purpose of platelet production is being challenged. Indeed, megakaryocytes are being shown to be a heterogeneous population of cells with distinct routes of differentiation and versatile functions. In particular, megakaryocytes show abilities reminiscent of immune cells, and are increasingly considered as the root dictating the hemostatic and immune functions of platelets in various physiopathological conditions. Furthermore, although megakaryocytes are well known as a component of the bone marrow niche, maintaining hematopoietic stem cells during homeostasis, the newly identified properties of megakaryocytes further indicate that they may be key supporters of stressed or diseased hematopoietic stem cells during myeloablative injury, aging and hematopoietic malignancy. Therefore, the generation and function of megakaryocytes are more diverse than we previously thought. Here, we review the recent literature that expands our views of megakaryocyte differentiation and heterogeneity, as well as the functions of megakaryocytes, with special focuses on their immune functions and supporting roles for stressed or diseased hematopoietic stem cells.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"1500-1512"},"PeriodicalIF":8.2,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12208170/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143718804","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}

引用次数: 0

Patients with high-risk acute promyelocytic leukemia need maintenance therapy for 1 year - the CONS. 高危急性早幼粒细胞白血病患者需要一年的维持治疗。

IF 8.2 1区医学

Haematologica Pub Date : 2025-07-01 Epub Date: 2025-04-03 DOI: 10.3324/haematol.2025.287418

Harry J Iland

引用次数: 0

Should we move past erythropoietin-stimulating agent monotherapy in lower-risk myelodysplastic syndromes? 在低风险骨髓增生异常综合征中，我们是否应该放弃促红细胞生成素单药治疗？

IF 10.1 1区医学

Haematologica Pub Date : 2025-06-26 DOI: 10.3324/haematol.2025.288157

Lee Mozessohn,Rena Buckstein

引用次数: 0