Haematologica最新文献_第10页

Survival assessment of warfarin and international normalized ratio exposure in cancer-associated venous thromboembolism: Veterans Affairs healthcare system-based analysis. 华法林和国际标准化比例暴露在癌症相关静脉血栓栓塞的生存评估：退伍军人事务医疗保健系统为基础的分析。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2025.300306

Justine Ryu,Jennifer La,Rushad Patell,Ang Li,Nhan Do,Mary Brophy,Nathanael Fillmore,Jeffrey I Zwicker

{"title":"Survival assessment of warfarin and international normalized ratio exposure in cancer-associated venous thromboembolism: Veterans Affairs healthcare system-based analysis.","authors":"Justine Ryu,Jennifer La,Rushad Patell,Ang Li,Nhan Do,Mary Brophy,Nathanael Fillmore,Jeffrey I Zwicker","doi":"10.3324/haematol.2025.300306","DOIUrl":"https://doi.org/10.3324/haematol.2025.300306","url":null,"abstract":"Venous thromboembolism (VTE) is a common complication among patients with cancer. While randomized clinical trials have established the safety and efficacy of direct oral anticoagulants (DOACs) and low-molecular-weight heparin (LMWH) relative to warfarin for the treatment of cancer-associated thrombosis, emerging evidence suggests that warfarin may be associated with improved overall survival in patients with malignancy. To evaluate survival outcomes among patients with cancer and VTE treated with warfarin compared with other anticoagulants within the Veterans Affairs (VA) Health Care System and analyze the association of international normalized ratio (INR) exposure and overall survival. Among 12 298 propensity-matched patients (mean age 69 years; 97% men), warfarin use was associated with a 16% lower risk of mortality compared with other anticoagulants (hazard ratio [HR], 0.84; (95% CI, 0.80-0.88); P < .001). Median survival was 1457 days in the warfarin group vs 1045 days in the non-warfarin group. Survival benefits were consistent across subgroups defined by tumor type, stage, and demographic characteristics. In landmark analyses the greatest benefit was observed with extended periods in the INR range of 2.5 to 3.0 (HR, 0.81; 95% CI, 0.75-0.87). In this population-based cohort study of patients with cancer and VTE, warfarin use was associated with improved survival compared with other anticoagulants. The greatest benefit was observed among patients who maintained an INR between 2.5 and 3.0.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"99 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147585394","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

Phenotypic evolution of circulating plasma cells from early precursor stages to multiple myeloma. 循环浆细胞从早期前体到多发性骨髓瘤的表型进化。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2025.300105

Martin Pietzsch,Sina Alexandra Beer,Lucca Marco Kimmich,Chiara Windsor,Sarah Gekeler,Britta Besemer,Anna Maria Paczulla Stanger

{"title":"Phenotypic evolution of circulating plasma cells from early precursor stages to multiple myeloma.","authors":"Martin Pietzsch,Sina Alexandra Beer,Lucca Marco Kimmich,Chiara Windsor,Sarah Gekeler,Britta Besemer,Anna Maria Paczulla Stanger","doi":"10.3324/haematol.2025.300105","DOIUrl":"https://doi.org/10.3324/haematol.2025.300105","url":null,"abstract":"Circulating tumour plasma-cells (CTPCs) have emerged as valuable diagnostic and prognostic marker in multiple myeloma (MM), with their presence linked to progression from precursor stages and poorer outcomes in newly diagnosed MM (NDMM). While quantitative CTPC enumeration is increasingly validated, comprehensive phenotypic profiling across disease stages remains lacking. We applied 36-parameter spectral flow cytometry to 113 PBMC samples of MGUS (n=42), SMM (n=22), NDMM (n=15), treated MM (n=24), and healthy controls (n=10), alongside paired bone marrow (BM) samples from six NDMM patients. CTPCs were defined phenotypically as CD45-CD38highCD56+ events without assessment of clonality. Phenotypic profiling of the CD56+ CTPC-like subset across disease stages revealed alterations in canonical and lineage-atypical surface markers. Progression to NDMM was characterised by upregulation of CD38 and CD56 with concomitant loss of B- (CD19), myeloid- (CD14, CD16), and T-cell-associated (CD45RA) markers. In addition, HLA-ABC, interleukin receptor subunits (CD25, CD123), and chemokine receptors (CCR6, CCR7) were upregulated in NDMM. In treated MM, a reversed pattern was observed with lower CD25 and CD123 expression and increased levels of exhaustion markers (TIGIT, PD-1). Paired BM samples showed a different tissue-residency marker expression, characterised by higher CD69 and CCR6 and lower CD138, along with changes in cell-survival related markers, including increased CD25 and CD123. Both BCMA and CD307e were more highly expressed on BM plasma-cells than CTPCs. This study is among the first to provide a comprehensive phenotypic characterisation of CD56+ CTPCs across the MM spectrum, including checkpoint and chemokine receptors, treated disease cases, and paired BM samples for NDMM.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"51 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147585390","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 "Sweet" crosstalk between refractory leukemia cells and vascular niche. 难治性白血病细胞与血管生态位之间的“甜蜜”串扰。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2026.300820

Hui Cheng

引用次数: 0

Diagnostic and therapeutic challenges in rare hematologic entities: monoclonal gammopathy of thrombotic and bleeding significance. 罕见血液学实体的诊断和治疗挑战：单克隆伽玛病的血栓和出血意义。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2025.300196

Despina Fotiou,Darko Antic,Aurelien Delluc,Kristen M Sanfilippo,Evangelos Terpos,Efstathios Kastritis,Vasiliki Gkalea

{"title":"Diagnostic and therapeutic challenges in rare hematologic entities: monoclonal gammopathy of thrombotic and bleeding significance.","authors":"Despina Fotiou,Darko Antic,Aurelien Delluc,Kristen M Sanfilippo,Evangelos Terpos,Efstathios Kastritis,Vasiliki Gkalea","doi":"10.3324/haematol.2025.300196","DOIUrl":"https://doi.org/10.3324/haematol.2025.300196","url":null,"abstract":"Monoclonal gammopathies (MG) encompass a spectrum of clonal B- or plasma-cell disorders characterized by the production of a monoclonal immunoglobulin. While most cases remain asymptomatic, certain clones can elicit organ or tissue injury through distinct pathogenic mechanisms, leading to the concept of Monoclonal Gammopathy of Clinical Significance (MGCS). Among the least recognized but clinically important MGCS entities are Monoclonal Gammopathy of Thrombotic Significance (MGTS) and Monoclonal Gammopathy of Bleeding Significance (MGBS), where the Mprotein directly interferes with hemostatic pathways, resulting in thrombosis or bleeding. These conditions remain underdiagnosed due to heterogeneous clinical presentations and challenges in establishing causal relationships between the paraprotein and hemostatic abnormalities. MGTS and MGBS encompass diverse mechanisms, including cryoprotein formation, complement activation, coagulation factor inhibition, and von Willebrand factor or platelet dysfunction. Currently, there are no standardized diagnostic criteria or evidence-based treatment recommendations, and the role of anti-clonal therapy remains undefined. This perspective outlines an ongoing multinational initiative under the auspices of the ISTH SSC Subcommittee on Cancer-Associated Thrombosis and Hemostasis, aiming to define diagnostic pathways, propose classification and treatment criteria, and identify patients who may benefit from targeted therapies. A unified framework will improve recognition, diagnosis, and management of these rare yet clinically significant entities.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"18 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147585383","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

First-year results of the International Leukemia/Lymphoma Target Board for pediatric relapsed and refractory hematological malignancies. 国际白血病/淋巴瘤目标委员会对儿童复发和难治性血液恶性肿瘤的第一年结果。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2025.300154

Uri Ilan,Judith M Boer,Maaike Luesink,Francisco Bautista,Mattias Hofmans,Aditi Vedi,Bálint Egyed,Birgit Geoerger,Ruta Tuckuviene,Bodil Als-Nielsen,Pablo Velasco,Jose Luis Fuster,Alba Rubio,Sarah K Tasian,Frederik van Delft,Julie A E Irving,Dániel J Erdély,Kjeld Schmiegelow,Barbara De Moerloose,André Baruchel,Monique L Den Boer,Michel C Zwaan

引用次数: 0

Relapsed Hodgkin disease: Should all patients receive an autologous stem cell transplant? - the CON. 复发霍奇金病：所有患者都应该接受自体干细胞移植吗？——骗子。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2026.300566

Alison J Moskowitz

引用次数: 0

Acceptability and feasibility of post-treatment fertility preservation in women previously treated for hematological diseases. 既往接受过血液病治疗的妇女保留治疗后生育能力的可接受性和可行性。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2025.300093

Celine Chalas,Florian Chevillon,Flore Sicre De Fontbrune,Nathalie Dhedin,Alienor Xhaard,Catherine Thieblemont,Pauline Brice,Marion Bendayan,Benedicte Paillusson,Jean-Hugues Dalle,Beatrice Delepine,Mathilde Bourdon,Virginie Barraud-Lange,Catherine Poirot,Nicolas Boissel

引用次数: 0

Relapsed classic Hodgkin lymphoma; should all patients receive an autologous stem cell transplant? - the PRO. 复发的经典霍奇金淋巴瘤；所有患者都应该接受自体干细胞移植吗？- PRO。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2026.300574

Chathuri Abeyakoon,John Kuruvilla

引用次数: 0

Preemptive hematopoietic stem cell transplantation in RUNX1 familial platelet disorder: a shared decision-making framework. 抢先造血干细胞移植治疗RUNX1家族性血小板疾病：共同决策框架

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2025.300403

Timothy S Olson,Katrin Ericson,Joseph H Antin,Laura Babbitt,Monica Babich,Natalie T Deuitch,Courtney DiNardo,Paul J Ford,Esther A Obeng,Brittany L Stewart,Wenbin Xiao,Akiko Shimamura

{"title":"Preemptive hematopoietic stem cell transplantation in RUNX1 familial platelet disorder: a shared decision-making framework.","authors":"Timothy S Olson,Katrin Ericson,Joseph H Antin,Laura Babbitt,Monica Babich,Natalie T Deuitch,Courtney DiNardo,Paul J Ford,Esther A Obeng,Brittany L Stewart,Wenbin Xiao,Akiko Shimamura","doi":"10.3324/haematol.2025.300403","DOIUrl":"https://doi.org/10.3324/haematol.2025.300403","url":null,"abstract":"RUNX1 familial platelet disorder (RUNX1-FPD) is associated with a 35% to 50% lifetime risk of hematologic malignancy (HM), and like all germline HM predisposition syndromes, can only be cured with allogeneic hematopoietic stem cell transplantation (HSCT). Current genetic screening techniques allow for early detection of germline predisposition, and consequently, the opportunity for HSCT before overt development of HM (ie, preemptive HSCT). However, there is not yet a consensus on the use of preemptive HSCT for RUNX1-FPD. Described here is the case of an individual with RUNX1-FPD and a family history of HM who underwent preemptive HSCT. We introduce a shared decision-making framework designed to support individuals with RUNX1-FPD, their families, and their multidisciplinary clinical teams in evaluating whether and when to pursue preemptive HSCT versus continued surveillance. The framework reviews key medical factors that influence HSCT timing decisions, including germline and somatic variants, clonal changes over time, familial history of HM, early morphologic or hematologic features, bleeding-related quality of life impacts, and donor availability. The framework also summarizes the major risks and uncertainties potentially associated with preemptive HSCT while highlighting the associated ethical challenges. Together, the case and framework provide a structured, patient-centered approach for navigating the complex clinical decision of preemptive HSCT. Ongoing collaborative efforts to define cytogenetic and clonal changes preceding malignant transformation in RUNX1-FPD will refine the framework and bolster individualized treatment strategies aimed at preventing HM and improving the quality of life of individuals with RUNX1-FPD.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"18 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147585377","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

Treatment of TP53-mutated myelodysplastic syndrome and acute myeloid leukemia with lowintensity metronomic decitabine and venetoclax. 低强度地西他滨联合维妥乐治疗tp53突变骨髓增生异常综合征和急性髓系白血病。

IF 10.1 1区医学

Haematologica Pub Date : 2026-04-02 DOI: 10.3324/haematol.2026.300534

Mendel Goldfinger,Ioannis Mantzaris,Aditi Shastri,Bradley Rockwell,Yogen Saunthararajah,Shanye Yin,David Levitz,Kira Gritsman,Alejandro R Sica,Noah Kornblum,Lauren C Shapiro,Ridhi Gupta,Stephen Peeke,Nishi Shah,Kith Pradhan,Anne Munoz,Aradhika Dhawan,Jhannine Alyssa Verceles,Karen Fehn,Monica Comas,Lamisha Shah,Yang Shi,Brian A Jonas,Dennis L Cooper,Marina Konopleva,Eric J Feldman,Amit Verma

{"title":"Treatment of TP53-mutated myelodysplastic syndrome and acute myeloid leukemia with lowintensity metronomic decitabine and venetoclax.","authors":"Mendel Goldfinger,Ioannis Mantzaris,Aditi Shastri,Bradley Rockwell,Yogen Saunthararajah,Shanye Yin,David Levitz,Kira Gritsman,Alejandro R Sica,Noah Kornblum,Lauren C Shapiro,Ridhi Gupta,Stephen Peeke,Nishi Shah,Kith Pradhan,Anne Munoz,Aradhika Dhawan,Jhannine Alyssa Verceles,Karen Fehn,Monica Comas,Lamisha Shah,Yang Shi,Brian A Jonas,Dennis L Cooper,Marina Konopleva,Eric J Feldman,Amit Verma","doi":"10.3324/haematol.2026.300534","DOIUrl":"https://doi.org/10.3324/haematol.2026.300534","url":null,"abstract":"Venetoclax (Ven) in combination with hypomethylating agents (HMA) (azacitidine or decitabine) is the standard of care for elderly or unfit patients with acute myeloid leukemia (AML) and is being explored in high-risk myelodysplastic syndrome (HR-MDS). However, currently approved dosing of HMA/Ven is associated with prolonged cytopenias, without a clear improvement in survival for TP53-mutated myeloid malignancies. In order to reduce hospitalizations during COVID, a once-weekly, metronomic schedule of decitabine (0.2 mg/Kg) and venetoclax (400 mg) was developed for patients with MDS and AML. Based on encouraging results, a phase 2 trial was performed. In the current study, we analyzed response rates and survival for all patients with TP53-mutated disease treated on the metronomic schedule. In total, 40 patients with TP53-mutated MDS and AML (26 in a prospective trial and 14 in the retrospective cohort) were included; 26 had HR-MDS and 14 had AML. The median age was 76.5 years, 70% had complex cytogenetics and 82% had bi-allelic TP53 mutations. The ORR for AML (CR+Cri) was 70% and 57% (CR+mCR) for MDS. With a median follow-up of 12.9 months, the median OS for the entire cohort was 11.3 months (11.6 months for AML, 9.9 months for MDS), and median OS in the 31 patients with bi-allelic mutated TP53 was 10.4 months. Transfusion independence was achieved in 58%. Neutropenic fever occurred in 15%, there were no therapy-related fatalities, and the 100-day mortality was 7.5%. A non-cytotoxic metronomic dosing schedule of decitabine/Ven has a low toxicity profile in TP53-mutated myeloid malignancies.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"17 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147585389","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