{"title":"Clinical characteristics and therapeutic determinants of RUNX1::RUNX1T1 differ from those of CBFB::MYH11 acute myeloid leukemia.","authors":"Miao Yang,Wenting Wang,Guangji Zhang,Shaowei Qiu,Bingcheng Liu,Yingchang Mi,Ying Wang,Jianxiang Wang,Hui Wei","doi":"10.3324/haematol.2024.287293","DOIUrl":"https://doi.org/10.3324/haematol.2024.287293","url":null,"abstract":"Core binding factor acute myeloid leukemia (CBF-AML) includes RUNX1::RUNX1T1 and CBFB::MYH11 AML. To investigate whether they should be regarded as distinct entities and treated separately, we retrospectively analyzed 536 patients with CBF-AML aged 60 years or younger. For CBFB::MYH11 AML, no outcome differences were observed between standard-dose (SD) and intermediate-dose (ID) cytarabine induction, with 5-year overall survival (OS) and relapse-free survival (RFS) at 86.4% vs. 85.3% (P=0.99) and 74.1% vs. 68.4% (P=0.93), respectively. However, ID induction improved the outcomes of RUNX1::RUNX1T1 AML, with 5-year OS and RFS rates of 77.7% vs. 60.3% (P.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"31 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143893266","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}
HaematologicaPub Date : 2025-04-30DOI: 10.3324/haematol.2025.287364
Simona Manni,Laura Iaffaldano,Marika Guercio,Pietro Merli,Francesca Del Bufalo,Marco Becilli,Mattia Algeri,Daria Pagliara,Rita De Vito,Gerhard Zugmaier,Biagio De Angelis,Concetta Quintarelli,Franco Locatelli
{"title":"Truncated form of human CD19 antigen as a suicide gene to control T-cell alloreactivity: ΔCD19.","authors":"Simona Manni,Laura Iaffaldano,Marika Guercio,Pietro Merli,Francesca Del Bufalo,Marco Becilli,Mattia Algeri,Daria Pagliara,Rita De Vito,Gerhard Zugmaier,Biagio De Angelis,Concetta Quintarelli,Franco Locatelli","doi":"10.3324/haematol.2025.287364","DOIUrl":"https://doi.org/10.3324/haematol.2025.287364","url":null,"abstract":"Donor lymphocyte infusion (DLI) is employed to either treat or prevent relapse in patients with hematologic malignancies, as well as to accelerate recovery of adaptive immunity, after allogeneic hematopoietic stem cell transplantation (allo-HSCT). With the increased use of DLI, there is renewed interest in the development of approaches able to prevent graft-versus-host disease (GvHD) reaction. In this study, we describe a novel and effective safety switch represented by the truncated form of the human CD19 antigen (ΔCD19) used to transduce T lymphocytes (hΔCD19 Tcells). We demonstrated that the exposure of ΔCD19-T-cells to an anti-hCD19-hCD3 T-cell bispecific T-cell engager (BiTE) molecule (structurally identical to blinatumomab, an agent largely used in the treatment of B-cell acute lymphoblastic leukemia) resulted into a prompt elimination of hCD19+/CD3+ cells both in vitro and in an in vivo animal model of mice developing a xenograft reaction mimicking GvHD after infusion of in vitro-activated/expanded human T cells. Importantly, the administration of the anti-hCD19-hCD3 BiTE molecule in the animal model, from one hand led to the improvement of signs and symptoms of GvHD, as well as of the overall-survival of the mice, and from the other, after a drug washout, was associated with the resurge of ΔCD19-T-cells without reoccurrence of GvHD. Our study provides evidence that the ΔCD19 suicide gene used in combination with an anti-hCD19-hCD3 T-cell BiTE molecule could represent a valid and effective strategy to control GvHD occurring after the infusion of donor T lymphocytes.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"11 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143893276","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}
HaematologicaPub Date : 2025-04-30DOI: 10.3324/haematol.2024.287090
Lok Lam Ngai,Tom Reuvekamp,Diana Hanekamp,Fleur Janssen,Laura Oudshoorn-van Marsbergen,Jannemieke Carbaat-Ham,Maaike A M Hofland,Mona M H E Fayed,Angèle Kelder,Willemijn J Scholten,Alexander N Snel,Costa Bachas,Jesse M Tettero,Dimitri A Breems,Thomas Fischer,Bjørn T Gjertsen,Laimonas Griškevičius,Gunnar Juliusson,Arjan A Van de Loosdrecht,Johan A Maertens,Markus G Manz,Thomas Pabst,Jakob R Passweg,Kimmo Porkka,Peter J M Valk,Patrycja Gradowska,Bob Löwenberg,Gert J Ossenkoppele,David C De Leeuw,Jacqueline Cloos
{"title":"Different features of acute myeloid leukemia stem cell quantification in intensively treated patients.","authors":"Lok Lam Ngai,Tom Reuvekamp,Diana Hanekamp,Fleur Janssen,Laura Oudshoorn-van Marsbergen,Jannemieke Carbaat-Ham,Maaike A M Hofland,Mona M H E Fayed,Angèle Kelder,Willemijn J Scholten,Alexander N Snel,Costa Bachas,Jesse M Tettero,Dimitri A Breems,Thomas Fischer,Bjørn T Gjertsen,Laimonas Griškevičius,Gunnar Juliusson,Arjan A Van de Loosdrecht,Johan A Maertens,Markus G Manz,Thomas Pabst,Jakob R Passweg,Kimmo Porkka,Peter J M Valk,Patrycja Gradowska,Bob Löwenberg,Gert J Ossenkoppele,David C De Leeuw,Jacqueline Cloos","doi":"10.3324/haematol.2024.287090","DOIUrl":"https://doi.org/10.3324/haematol.2024.287090","url":null,"abstract":"In acute myeloid leukemia, the burden of CD34+CD38- leukemia stem cells (LSC) has prognostic value at diagnosis and after induction chemotherapy. Since different methods of LSC quantification have been proposed, we determined the prognostic value on overall survival and incidence of relapse of these methods across ELN2017 risk groups, using data from the HOVON-SAKK132 trial. In addition, we have evaluated the optimal number of acquired white blood cells for accurate LSC detection and the prognostic value of individual LSC markers. Results show that acquiring 1 million white blood cells is essential for accurate LSC-negativity assessment. Among different LSC markers, CD44 overexpression on CD34+CD38- cells was the only insignificant marker in our panel. Testing the impact of several published variations on the analysis for LSC assessments on prognostic value for overall survival and cumulative incidence of relapse, showed marginal differences, demonstrating the robust prognostic value of LSC burden. For further clinical implementation, the optimal LSC assessment may differ among ELN risk groups. In conclusion, LSC burden is a robust prognostic factor and insight in the different methods of LSC definition can facilitate the clinical implementation.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"8 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143893277","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}
HaematologicaPub Date : 2025-04-30DOI: 10.3324/haematol.2024.286457
Gilad Shamir,Ory Rouvio,Anat Reiner-Benaim
{"title":"Prognostic and biological characteristics associated with multiple myeloma presenting only with anemia.","authors":"Gilad Shamir,Ory Rouvio,Anat Reiner-Benaim","doi":"10.3324/haematol.2024.286457","DOIUrl":"https://doi.org/10.3324/haematol.2024.286457","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"44 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143893267","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":"Optimizing olverembatinib dose in chronic phase chronic myeloid leukemia.","authors":"Xiaoshuai Zhang,Yunfan Yang,Bingcheng Liu,Xin Du,Xiaodong Wang,Huanling Zhu,Lu Yu,Zongru Li,Shasha Zhao,Linhua Yang,Yanping Ma,Li Meng,Yanqing Zhang,Guohui Li,Lijie Yang,Baohong Wang,Xuehong Ran,Jian Huang,Na Gao,Qin Wen,Yan Wen,Yuxia Zhao,Yu Zhu,Yanqiu Han,Zhenfang Liu,Xin Du,Jianyu Weng,Robert Peter Gale,Li Zhou,Yanli Zhang,Qian Jiang","doi":"10.3324/haematol.2024.287116","DOIUrl":"https://doi.org/10.3324/haematol.2024.287116","url":null,"abstract":"Optimizing olverembatinib dose in people with chronic phase chronic myeloid leukemia (CML) is important to increase safety without compromising efficacy. We designed a multi-center retrospective study comparing safety and efficacy of olverembatinib between the recommended dose of 40 mg every other day (QOD; N = 216) and a reduced dose of 30 mg QOD (N = 66) in subjects failing other tyrosine kinase-inhibitors (TKIs). The cohorts were similar in baseline co-variates and adjusted for by propensity score matching (PSM). There were no significant differences in cytogenetic and molecular responses, as well as outcomes between the 2 dose cohorts. However, the proportion of subjects receiving the original olverembatinib dose at the last follow-up was significantly higher in the 30 mg cohort (64% [95%Confidence Interval [CI], 53, 75%] versus 44% [37,51%]; p = 0.004). Also, the proportion of subjects receiving a reduced dose or permanently discontinuing because of adverse event was significantly lower in the 30 mg cohort (21% [9, 33%] versus 41% [34, 48%]; p = 0.003). In summary, olverembatinib, 30 mg QOD starting dose is as effective as a 40 mg starting dose but better tolerated in persons with chronic phase CML failing other TKIs.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"121 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143893278","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}
HaematologicaPub Date : 2025-04-24DOI: 10.3324/haematol.2025.287509
Kjersti Lia,Rasmus Rask Kragh Jørgensen,Per Wikman,Bente L Wold,Ninja Övergaard,Øystein Fluge,Unn-Merete Fagerli,Hanne Bersvendsen,Idun B Bø,Sameer Bhargava,Daniel Molin,Alexander Fosså
{"title":"A simplified frailty score predicts outcome in curatively treated older patients with classical Hodgkin lymphoma.","authors":"Kjersti Lia,Rasmus Rask Kragh Jørgensen,Per Wikman,Bente L Wold,Ninja Övergaard,Øystein Fluge,Unn-Merete Fagerli,Hanne Bersvendsen,Idun B Bø,Sameer Bhargava,Daniel Molin,Alexander Fosså","doi":"10.3324/haematol.2025.287509","DOIUrl":"https://doi.org/10.3324/haematol.2025.287509","url":null,"abstract":"Older patients with classical Hodgkin lymphoma (cHL) have lower tolerance and inferior outcomes after standard chemotherapy regimens. To identify patient-derived indicators of frailty associated with outcome, we retrospectively analyzed patient and disease characteristics, treatment and outcome in a Norwegian population-based cohort of older (≥60 years) patients with cHL diagnosed 2000-2015. We included 279 patients (median age 69 years, range 60-90) treated with curative intent, defined as any typical cHL regimen with ≥50% standard doxorubicin dose in the first cycle. Treatment-related mortality was 8%, median progression-free (PFS) and overall survival (OS) were 7.1 years (95% confidence interval [CI] 5.0-9.3) and 8.7 years (95%CI 7.0-10.4), respectively, in the Norwegian cohort. Multivariable analyses identified patient-derived prognostic factors working independently of stage, histology and international prognostic score. We derived a frailty index ranging from 0-3 with one point each for age ≥70 years, Eastern Cooperative Oncology Group status ≥2 and a Cumulative illness rating scale in geriatrics score ≥8. Patients were categorized as fit (score 0, 34% of patients), unfit (score 1-2, 60%) and frail (score 3, 7%), with 5-year PFS of 74%, 49% and 11%, and 5-year OS of 86%, 52%, and 22% respectively. The proposed frailty score was validated in an external cohort of 792 similarly selected patients from the Swedish Lymphoma Register, where comorbidities were scored based on the Charlson comorbidity index (0-2 versus 3 or higher). In this comprehensive study, we develop a frailty score for elderly cHL patients to inform clinical decisions and prospective trials evaluating selective therapies for older patients.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"141 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143872048","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}
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