Pub Date : 2026-01-22DOI: 10.3324/haematol.2025.288956
Mikkael A Sekeres,Amer M Zeidan,Valeria Santini,Rami S Komrokji,Pierre Fenaux,Michael R Savona,Yazan F Madanat,David Valcárcel,Antoine Regnault,Kristin Creel,Libo Sun,Ying Wan,Shyamala Navada,Tymara Berry,Faye Feller,Uwe Platzbecker,María Díez-Campelo,Esther Natalie Oliva
Red blood cell (RBC) transfusions for anemia associated with lower-risk myelodysplastic syndromes/neoplasms (LR-MDS) often contribute to reduced quality of life (QOL). Thus, reduction in RBC transfusion dependency (TD) is a primary therapeutic goal. Imetelstat is a firstin-class, competitive telomerase inhibitor approved to treat certain adult patients with LR-MDS with RBC-TD anemia who have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents. In the phase III IMerge study (NCT02598661), treatment with imetelstat resulted in clinically meaningful, statistically significant increases in the primary endpoint of ≥8-week RBC transfusion independence (TI) versus placebo. Because patients with LR-MDS experience detrimental effects on numerous facets of QOL (physical, emotional, social, and functional), these exploratory analyses assessed patient-reported outcomes using the Functional Assessment of Chronic Illness Therapy-Fatigue, Quality of Life in Myelodysplasia Scale, and Functional Assessment of Cancer Therapy-Anemia questionnaires as part of the phase III IMerge study. Nominal P values were reported. Fewer imetelstat-treated patients experienced deterioration in fatigue and more imetelstat-treated patients experienced sustained improvement in fatigue and QOL versus placebo. In the imetelstat group, 8-week, 24-week, and 1-year RBC-TI responders had sustained improvements in predefined significance thresholds versus nonresponders for fatigue (70%, 73%, and 88%, respectively, vs. 37%, 41%, and 44%, respectively; P.
{"title":"Imetelstat improves patient-reported outcomes and quality of life in lower-risk myelodysplastic syndromes: results from the phase III IMerge study.","authors":"Mikkael A Sekeres,Amer M Zeidan,Valeria Santini,Rami S Komrokji,Pierre Fenaux,Michael R Savona,Yazan F Madanat,David Valcárcel,Antoine Regnault,Kristin Creel,Libo Sun,Ying Wan,Shyamala Navada,Tymara Berry,Faye Feller,Uwe Platzbecker,María Díez-Campelo,Esther Natalie Oliva","doi":"10.3324/haematol.2025.288956","DOIUrl":"https://doi.org/10.3324/haematol.2025.288956","url":null,"abstract":"Red blood cell (RBC) transfusions for anemia associated with lower-risk myelodysplastic syndromes/neoplasms (LR-MDS) often contribute to reduced quality of life (QOL). Thus, reduction in RBC transfusion dependency (TD) is a primary therapeutic goal. Imetelstat is a firstin-class, competitive telomerase inhibitor approved to treat certain adult patients with LR-MDS with RBC-TD anemia who have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents. In the phase III IMerge study (NCT02598661), treatment with imetelstat resulted in clinically meaningful, statistically significant increases in the primary endpoint of ≥8-week RBC transfusion independence (TI) versus placebo. Because patients with LR-MDS experience detrimental effects on numerous facets of QOL (physical, emotional, social, and functional), these exploratory analyses assessed patient-reported outcomes using the Functional Assessment of Chronic Illness Therapy-Fatigue, Quality of Life in Myelodysplasia Scale, and Functional Assessment of Cancer Therapy-Anemia questionnaires as part of the phase III IMerge study. Nominal P values were reported. Fewer imetelstat-treated patients experienced deterioration in fatigue and more imetelstat-treated patients experienced sustained improvement in fatigue and QOL versus placebo. In the imetelstat group, 8-week, 24-week, and 1-year RBC-TI responders had sustained improvements in predefined significance thresholds versus nonresponders for fatigue (70%, 73%, and 88%, respectively, vs. 37%, 41%, and 44%, respectively; P.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"4 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146015412","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":"Abrupt ferritin increases as a marker of cancer in transfusion-dependent thalassemia.","authors":"Elena Chatzikalil,Polyxeni Delaporta,Ilona Binenbaum,Vasiliki Chouliara,Dimitra Kyriakopoulou,Sofirela Berdalli,Konstantinos Bistas,Maria-Ioanna Chatzieleftheriou,Euthalia-Faidra Agiomavriti-Stefanopoulou,Antonis Kattamis","doi":"10.3324/haematol.2025.288434","DOIUrl":"https://doi.org/10.3324/haematol.2025.288434","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"71 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968351","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}
We studied platelet recovery in 93 patients with myelofibrosis, following an allogeneic hemopoietic stem cell transplant (HSCT). The primary end point of the study was achieving a platelet count of 50x10^9/L within day +100, which occurred in 62 patients (67%) and predicted 5 year non relapse mortality (5% vs 55%; p=0.0009) and 5 year actuarial survival (85% vs 38%; p=.
我们研究了93例同种异体造血干细胞移植（HSCT）后骨髓纤维化患者的血小板恢复情况。该研究的主要终点是在100天内达到50x10^9/L的血小板计数，62例患者（67%）实现了这一目标，并预测了5年非复发死亡率（5% vs 55%, p=0.0009）和5年精算生存率（85% vs 38%, p=0.0009）。
{"title":"Platelet recovery delay and survival in patients with myelofibrosis undergoing allogeneic hemopoietic stem cell transplantation.","authors":"Federica Sora',Andrea Bacigalupo,Sabrina Giammarco,Elisabetta Metafuni,Filippo Frioni,Eugenio Galli,Maria Assunta Limongiello,Simona Sica,Patrizia Chiusolo","doi":"10.3324/haematol.2025.288371","DOIUrl":"https://doi.org/10.3324/haematol.2025.288371","url":null,"abstract":"We studied platelet recovery in 93 patients with myelofibrosis, following an allogeneic hemopoietic stem cell transplant (HSCT). The primary end point of the study was achieving a platelet count of 50x10^9/L within day +100, which occurred in 62 patients (67%) and predicted 5 year non relapse mortality (5% vs 55%; p=0.0009) and 5 year actuarial survival (85% vs 38%; p=.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"51 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968699","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}
Pub Date : 2026-01-15DOI: 10.3324/haematol.2025.300189
Martin S Tallman
Not available.
不可用。
{"title":"Don't forget about thrombosis in acute promyelocytic leukemia.","authors":"Martin S Tallman","doi":"10.3324/haematol.2025.300189","DOIUrl":"https://doi.org/10.3324/haematol.2025.300189","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"141 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968695","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}
Pub Date : 2026-01-15DOI: 10.3324/haematol.2025.288700
Mikkel R Simonsen,Toby A Eyre,Eliza A Hawkes,Tarec C El-Galaly
Older patients with lymphoma represent a growing, heterogeneous population whose care is challenged by diverse outcomes, limited evidence, and one-dimensional age definitions. Historically, arbitrary age thresholds such as ≥60 or ≥80 years have guided treatment decisions, yet they fail to capture the biological and functional diversity of aging and can limit opportunities for cure and progress. Current practice relies on arbitrary dose reductions in old age, such as RminiCHOP, despite limited data for optimal intensity and benefit-risk trade-offs. Likewise, novel agents and combination therapies frequently demonstrate discrepant efficacy and safety across age groups, but systematic attempts to optimize dose for the older patients are rarely prioritized. When it comes to clinical trials, documenting benefit of new therapies is more challenging in older patients due to high background mortality that complicates interpretation of overall and progression-free survival and may led to underpowered trials. Moreover, prognostic models developed in younger populations have limited applicability in older patients, as they overlook the broader range of clinically relevant outcomes in older patients, including treatment-related mortality, functional decline, and quality of life. Pre-therapeutic geriatric assessments are prognostic, but their predictive capability remains to be demonstrated in prospective trials before use as treatment decision support tools. Addressing these challenges requires reframing of "old age" to a multidimensional construct, incorporating geriatric assessment, patient preferences, and biological age. More inclusive trial designs, dedicated dose-finding in older patients, and development of holistic, predictive models are critical to advance care. Without this, progress risks stalling for a growing group of our patients.
{"title":"Older patients with lymphoma: navigating a landscape of clinical controversies and barriers to innovation.","authors":"Mikkel R Simonsen,Toby A Eyre,Eliza A Hawkes,Tarec C El-Galaly","doi":"10.3324/haematol.2025.288700","DOIUrl":"https://doi.org/10.3324/haematol.2025.288700","url":null,"abstract":"Older patients with lymphoma represent a growing, heterogeneous population whose care is challenged by diverse outcomes, limited evidence, and one-dimensional age definitions. Historically, arbitrary age thresholds such as ≥60 or ≥80 years have guided treatment decisions, yet they fail to capture the biological and functional diversity of aging and can limit opportunities for cure and progress. Current practice relies on arbitrary dose reductions in old age, such as RminiCHOP, despite limited data for optimal intensity and benefit-risk trade-offs. Likewise, novel agents and combination therapies frequently demonstrate discrepant efficacy and safety across age groups, but systematic attempts to optimize dose for the older patients are rarely prioritized. When it comes to clinical trials, documenting benefit of new therapies is more challenging in older patients due to high background mortality that complicates interpretation of overall and progression-free survival and may led to underpowered trials. Moreover, prognostic models developed in younger populations have limited applicability in older patients, as they overlook the broader range of clinically relevant outcomes in older patients, including treatment-related mortality, functional decline, and quality of life. Pre-therapeutic geriatric assessments are prognostic, but their predictive capability remains to be demonstrated in prospective trials before use as treatment decision support tools. Addressing these challenges requires reframing of \"old age\" to a multidimensional construct, incorporating geriatric assessment, patient preferences, and biological age. More inclusive trial designs, dedicated dose-finding in older patients, and development of holistic, predictive models are critical to advance care. Without this, progress risks stalling for a growing group of our patients.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"101 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968700","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}
Pub Date : 2026-01-15DOI: 10.3324/haematol.2025.288892
Brenna Park-Egan,Catherine L Hough,Patrick G Lyons,Laura F Newell
Not available.
不可用。
{"title":"Performance of commonly used risk triage tools in predicting clinical deterioration among hospitalized hematopoietic stem cell transplant recipients.","authors":"Brenna Park-Egan,Catherine L Hough,Patrick G Lyons,Laura F Newell","doi":"10.3324/haematol.2025.288892","DOIUrl":"https://doi.org/10.3324/haematol.2025.288892","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"30 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968696","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}
Pub Date : 2026-01-15DOI: 10.3324/haematol.2025.288786
Nigel H Russell,Katie D Lewis
Not available.
不可用。
{"title":"All patients with acute myeloid leukemia and FLT3-ITD should be transplanted in first remission. Also in the era of tyrosine kinase inhibitors? - the CON.","authors":"Nigel H Russell,Katie D Lewis","doi":"10.3324/haematol.2025.288786","DOIUrl":"https://doi.org/10.3324/haematol.2025.288786","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"17 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968353","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}
Pub Date : 2026-01-15DOI: 10.3324/haematol.2025.287809
Bruno Palhais,Nitesh D Sharma,Igor Fijalkowski,Tim Pieters,Dieter Deforce,Filip Van Nieuwerburgh,Pieter Mestdagh,Panagiotis Ntziachristos,Ksenia Matlawska-Wasowska,Pieter Van Vlierberghe
Understanding the molecular mechanisms underlying T-cell acute lymphoblastic leukemia (T-ALL) is essential for developing more effective therapeutic strategies. Despite therapeutic advances, the role of RNA-binding proteins in the pathogenesis of T-ALL remains poorly understood. Here, we investigate the RNA-binding protein Quaking (QKI), identifying it as a key regulator of splicing with tumor-suppressive properties in T-ALL. Through the analysis of two independent pediatric T-ALL cohorts, we demonstrate that QKI expression is frequently reduced in T-ALL, particularly within the HOXA subtype, and this reduction correlates with poor overall and event-free survival. Using T-ALL cell lines, we show that QKI depletion induces widespread splicing alterations, with numerous events corroborated in patient samples. Transcriptome profiling indicates that QKI downregulation leads to broad changes in gene expression, notably affecting pathways related to cell cycle progression, cholesterol homeostasis, and epithelial-mesenchymal transition. Functional assays demonstrate that QKI overexpression in T-ALL cells significantly reduces cell proliferation, induces G0/G1 cell cycle arrest, and limits leukemia progression and dissemination, ultimately improving survival in xenograft models. Together, these findings provide compelling evidence that QKI functions as a regulator of RNA splicing with tumor-suppressive activity in T-ALL.
{"title":"QKI dysregulation induces extensive splicing changes in T-cell acute lymphoblastic leukemia.","authors":"Bruno Palhais,Nitesh D Sharma,Igor Fijalkowski,Tim Pieters,Dieter Deforce,Filip Van Nieuwerburgh,Pieter Mestdagh,Panagiotis Ntziachristos,Ksenia Matlawska-Wasowska,Pieter Van Vlierberghe","doi":"10.3324/haematol.2025.287809","DOIUrl":"https://doi.org/10.3324/haematol.2025.287809","url":null,"abstract":"Understanding the molecular mechanisms underlying T-cell acute lymphoblastic leukemia (T-ALL) is essential for developing more effective therapeutic strategies. Despite therapeutic advances, the role of RNA-binding proteins in the pathogenesis of T-ALL remains poorly understood. Here, we investigate the RNA-binding protein Quaking (QKI), identifying it as a key regulator of splicing with tumor-suppressive properties in T-ALL. Through the analysis of two independent pediatric T-ALL cohorts, we demonstrate that QKI expression is frequently reduced in T-ALL, particularly within the HOXA subtype, and this reduction correlates with poor overall and event-free survival. Using T-ALL cell lines, we show that QKI depletion induces widespread splicing alterations, with numerous events corroborated in patient samples. Transcriptome profiling indicates that QKI downregulation leads to broad changes in gene expression, notably affecting pathways related to cell cycle progression, cholesterol homeostasis, and epithelial-mesenchymal transition. Functional assays demonstrate that QKI overexpression in T-ALL cells significantly reduces cell proliferation, induces G0/G1 cell cycle arrest, and limits leukemia progression and dissemination, ultimately improving survival in xenograft models. Together, these findings provide compelling evidence that QKI functions as a regulator of RNA splicing with tumor-suppressive activity in T-ALL.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"40 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968349","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}
Pub Date : 2026-01-15DOI: 10.3324/haematol.2025.288876
Ivy E Abraham,Garth H Rauscher,Jerry Luo,Sarah Monick,Madelyn Burkart,Peter Doukas,Ahmed Aleem,Nepheli Raptis,Ami Dave,Andrew Wilmington,Mark Debettencourt,Michelle Nwachukwu,Juwairiyyah Fatima,Amani Erra,Gauri Shankar,Stephanie B Tsai,Melissa Larson,Maryam Zia,John Quigley,Jessica K Altman,Wendy Stock,Anand Patel,Irum Khan
Not available.
不可用。
{"title":"Clinical and sociodemographic predictors of intensive care unit admission following chemotherapy in acute myeloid leukemia.","authors":"Ivy E Abraham,Garth H Rauscher,Jerry Luo,Sarah Monick,Madelyn Burkart,Peter Doukas,Ahmed Aleem,Nepheli Raptis,Ami Dave,Andrew Wilmington,Mark Debettencourt,Michelle Nwachukwu,Juwairiyyah Fatima,Amani Erra,Gauri Shankar,Stephanie B Tsai,Melissa Larson,Maryam Zia,John Quigley,Jessica K Altman,Wendy Stock,Anand Patel,Irum Khan","doi":"10.3324/haematol.2025.288876","DOIUrl":"https://doi.org/10.3324/haematol.2025.288876","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"40 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145968697","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}
京公网安备 11010802042870号
京ICP备2023020795号-1
扫码关注我们
求助内容：
应助结果提醒方式：