Acute myeloid leukemia (AML) is the most common indication for allogeneic hematopoietic cell transplantation (alloHCT), yet graft-versus-host disease (GVHD) remains a major post-transplant complication. Conditioning regimens, particularly reduced-intensity approaches, are critical in optimizing outcomes. This subgroup analysis of the phase 3 MC-FludT.14/L trial compared treosulfan-fludarabine with reduced-intensity busulfan-fludarabine in 352 AML patients (aged 31-70) undergoing alloHCT. The primary endpoint was 24-month event-free survival (EFS); secondary endpoints included overall survival (OS), GVHD incidence, relapse/progression, and non-relapse mortality (NRM). Treosulfan compared to busulfan demonstrated superiority: 24-month EFS was 65% vs. 53% (p = 0.01), and OS was 73% vs. 65%. EFS benefits were consistent across AML risk categories and notably higher in patients with hematopoietic cell transplantation comorbidity index >2 (62% vs. 42%, p = 0.02). Treosulfan also showed lower NRM and relapse rates. GVHD outcomes favored treosulfan, with a significantly lower incidence of extensive chronic GVHD at 24 months (15.1% vs. 28.1%, p = 0.01). GVHD-free and relapse-free survival was also improved (53% vs. 40%, p = 0.02). The safety profile was more favorable with treosulfan. These findings support treosulfan-fludarabine as a more effective and safer conditioning regimen than busulfan-fludarabine for AML patients undergoing alloHCT, particularly those at higher risk.
急性髓性白血病（AML）是同种异体造血细胞移植（allogeneic hematopoietic cell transplantation, alloHCT）最常见的适应症，然而移植物抗宿主病（graft- anti -host disease， GVHD）仍然是移植后的主要并发症。调理方案，特别是降低强度的方法，是优化结果的关键。第三阶段mc - flut的亚组分析。在352例接受同种异体hct的AML患者（年龄31-70岁）中，14/L的试验比较了treosulan -fludarabine和降低强度的busulan -fludarabine。主要终点为24个月无事件生存期（EFS）；次要终点包括总生存期（OS）、GVHD发病率、复发/进展和非复发死亡率（NRM）。曲硫丹与丁硫丹相比表现出优越性：24个月的EFS为65%对53% (p = 0.01)， OS为73%对65%。EFS的益处在AML风险类别中是一致的，并且在造血细胞移植合并症指数>2的患者中明显更高（62%对42%，p = 0.02）。曲硫芬也显示出较低的NRM和复发率。GVHD结果倾向于曲硫丹，24个月时广泛慢性GVHD的发生率显著降低（15.1%比28.1%,p = 0.01）。无gvhd和无复发生存率也有所提高（53% vs. 40%, p = 0.02）。曲硫丹的安全性更佳。这些发现支持treosulan -fludarabine作为一种比busulfan-fludarabine更有效和更安全的治疗方案用于接受同种异体hct的AML患者，特别是那些高风险患者。
{"title":"GVHD and GRFS in patients with acute myeloid leukemia undergoing allogeneic HCT with treosulfan- versus reduced-intensity busulfan-based conditioning: a subgroup analysis of a randomized phase 3 trial.","authors":"Friedrich Stölzel,Matthias Stelljes,Dietrich Wilhelm Beelen,Miroslaw Markiewicz,Péter Reményi,Thomas Luft,Fabio Ciceri,Eva-Maria Wagner-Drouet,Christian Junghanss,Christoph Scheid,Francesca Patriarca,Gerard Socié,Inken Hilgendorf,Alessandro Rambaldi,Kerstin Schaefer-Eckart,Domenico Russo,Gerald Wulf,Bertram Glass,Hélène Labussiere-Wallet,Gernot Stuhler,Maria Bieniaszewska,Jochen Casper,Ernst Holler,Fabio Benedetti,Anna Paola Iori,Desiree Kunadt,Jelena Mihajlović,Bettina Schild,Imran Khan,Xieran Li,Wolfgang Bethge","doi":"10.3324/haematol.2025.288770","DOIUrl":"https://doi.org/10.3324/haematol.2025.288770","url":null,"abstract":"Acute myeloid leukemia (AML) is the most common indication for allogeneic hematopoietic cell transplantation (alloHCT), yet graft-versus-host disease (GVHD) remains a major post-transplant complication. Conditioning regimens, particularly reduced-intensity approaches, are critical in optimizing outcomes. This subgroup analysis of the phase 3 MC-FludT.14/L trial compared treosulfan-fludarabine with reduced-intensity busulfan-fludarabine in 352 AML patients (aged 31-70) undergoing alloHCT. The primary endpoint was 24-month event-free survival (EFS); secondary endpoints included overall survival (OS), GVHD incidence, relapse/progression, and non-relapse mortality (NRM). Treosulfan compared to busulfan demonstrated superiority: 24-month EFS was 65% vs. 53% (p = 0.01), and OS was 73% vs. 65%. EFS benefits were consistent across AML risk categories and notably higher in patients with hematopoietic cell transplantation comorbidity index >2 (62% vs. 42%, p = 0.02). Treosulfan also showed lower NRM and relapse rates. GVHD outcomes favored treosulfan, with a significantly lower incidence of extensive chronic GVHD at 24 months (15.1% vs. 28.1%, p = 0.01). GVHD-free and relapse-free survival was also improved (53% vs. 40%, p = 0.02). The safety profile was more favorable with treosulfan. These findings support treosulfan-fludarabine as a more effective and safer conditioning regimen than busulfan-fludarabine for AML patients undergoing alloHCT, particularly those at higher risk.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"74 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146072992","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 : 2026-01-29DOI: 10.3324/haematol.2025.300099
Jae-Ho Yoon,Kyoung Il Min,Daehun Kwag,Gi-June Min,Sung-Soo Park,Silvia Park,Sung-Eun Lee,Byung-Sik Cho,Ki-Seong Eom,Yoo-Jin Kim,Hee-Je Kim,Chang-Ki Min,Seok-Goo Cho,Seok Lee
Not available.
不可用。
{"title":"Frontline ponatinib plus hyper-CVAD over imatinib in adults with Ph-positive acute lymphoblastic leukemia: real-world efficacy and risks of early ponatinib dose reduction.","authors":"Jae-Ho Yoon,Kyoung Il Min,Daehun Kwag,Gi-June Min,Sung-Soo Park,Silvia Park,Sung-Eun Lee,Byung-Sik Cho,Ki-Seong Eom,Yoo-Jin Kim,Hee-Je Kim,Chang-Ki Min,Seok-Goo Cho,Seok Lee","doi":"10.3324/haematol.2025.300099","DOIUrl":"https://doi.org/10.3324/haematol.2025.300099","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"93 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146073143","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 : 2026-01-29DOI: 10.3324/haematol.2025.288891
Leora Boussi,Jan Philipp Bewersdorf,Yiwen Liu,Rory M Shallis,Luis E Aguirre,Rebecca P Bystrom,Andrius Zucenka,Sylvain Garciaz,Daniel J DeAngelo,Richard M Stone,Marlise R Luskin,Jacqueline S Garcia,Eric S Winer,Evan C Chen,Martha Wadleigh,Guillaume Berton,Kelly Ling,Amer M Zeidan,Eytan M Stein,Shai Shimony,Aaron D Goldberg,Maximilian Stahl
Abnormalities in chromosomes 5 and 7 are frequently identified in acute myeloid leukemia (AML), particularly enriched in therapy-and myelodysplasia-related disease, and confer an adverse prognosis. Given the high risk of relapse, allogeneic stem cell transplant (allo-SCT) is typically recommended for patients achieving complete remission (CR) following induction chemotherapy. We currently lack prospective data to decide whether intensive chemotherapy (IC) versus hypomethylating agent+venetoclax (HMA+ven) is the superior frontline treatment approach for these patients. Hence, we performed a retrospective study in a large cohort of patients with AML and deletion 7 (-7) and/or deletion 5 or 5q (-5/del5q) comparing outcomes between IC-versus HMA+ven-treated patients. Remission rates after IC and HMA+ven were found to be comparable (43% vs 52%, p=0.2). When adjusting for patient and disease characteristics in multivariable analysis (MVA), treatment with IC vs HMA+ven did not significantly impact overall survival (OS) (HR 1.02, p=0.9202), while age at diagnosis (HR 1.02, p=0.0324), prior myeloid disease (HR 1.42, p=0.0266), monosomal karyotype (HR 1.48, p=0.029), complex karyotype (HR 1.61, p=0.0156), and KRAS mutations (HR 2.21, p=0.0063) were associated with inferior survival. There was also no difference in OS in patients age 60-75 years by treatment strategy (7.8 vs 6.4 months, p=0.56), motivating future randomized trials of IC versus HMA+ven in this older population to inform optimal therapy. Importantly, OS was significantly improved in patients undergoing allo-SCT irrespective of frontline therapy, and allo-SCT consolidation was the most important predictor of long-term survival in MVA (HR 0.36, p.
{"title":"Hypomethylating agents plus venetoclax versus intensive chemotherapy in acute myeloid leukemia with chromosome 5 and 7 abnormalities.","authors":"Leora Boussi,Jan Philipp Bewersdorf,Yiwen Liu,Rory M Shallis,Luis E Aguirre,Rebecca P Bystrom,Andrius Zucenka,Sylvain Garciaz,Daniel J DeAngelo,Richard M Stone,Marlise R Luskin,Jacqueline S Garcia,Eric S Winer,Evan C Chen,Martha Wadleigh,Guillaume Berton,Kelly Ling,Amer M Zeidan,Eytan M Stein,Shai Shimony,Aaron D Goldberg,Maximilian Stahl","doi":"10.3324/haematol.2025.288891","DOIUrl":"https://doi.org/10.3324/haematol.2025.288891","url":null,"abstract":"Abnormalities in chromosomes 5 and 7 are frequently identified in acute myeloid leukemia (AML), particularly enriched in therapy-and myelodysplasia-related disease, and confer an adverse prognosis. Given the high risk of relapse, allogeneic stem cell transplant (allo-SCT) is typically recommended for patients achieving complete remission (CR) following induction chemotherapy. We currently lack prospective data to decide whether intensive chemotherapy (IC) versus hypomethylating agent+venetoclax (HMA+ven) is the superior frontline treatment approach for these patients. Hence, we performed a retrospective study in a large cohort of patients with AML and deletion 7 (-7) and/or deletion 5 or 5q (-5/del5q) comparing outcomes between IC-versus HMA+ven-treated patients. Remission rates after IC and HMA+ven were found to be comparable (43% vs 52%, p=0.2). When adjusting for patient and disease characteristics in multivariable analysis (MVA), treatment with IC vs HMA+ven did not significantly impact overall survival (OS) (HR 1.02, p=0.9202), while age at diagnosis (HR 1.02, p=0.0324), prior myeloid disease (HR 1.42, p=0.0266), monosomal karyotype (HR 1.48, p=0.029), complex karyotype (HR 1.61, p=0.0156), and KRAS mutations (HR 2.21, p=0.0063) were associated with inferior survival. There was also no difference in OS in patients age 60-75 years by treatment strategy (7.8 vs 6.4 months, p=0.56), motivating future randomized trials of IC versus HMA+ven in this older population to inform optimal therapy. Importantly, OS was significantly improved in patients undergoing allo-SCT irrespective of frontline therapy, and allo-SCT consolidation was the most important predictor of long-term survival in MVA (HR 0.36, p.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"73 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146073146","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 : 2026-01-29DOI: 10.3324/haematol.2025.300275
Mathias Castonguay,John F Seymour
Not available.
不可用。
{"title":"Transforming mantle cell lymphoma: the journey across eras.","authors":"Mathias Castonguay,John F Seymour","doi":"10.3324/haematol.2025.300275","DOIUrl":"https://doi.org/10.3324/haematol.2025.300275","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"93 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146072991","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 : 2026-01-29DOI: 10.3324/haematol.2025.288518
Joseph M Cannova,Muriel R Battaglia,Gregory W Roloff,Sinan Cetin,Michael Tallarico,Wendy Stock,Anand A Patel,Olatoyosi Odenike,Richard A Larson,Michael J Thirman,Mariam T Nawas,Peng Wang,Melissa Y Tjota,Jeremy P Segal,Girish Venkataraman,Adam S DuVall,Michael W Drazer
Not available.
不可用。
{"title":"Hematologic complications in patients exposed to poly-ADP ribose polymerase inhibitors.","authors":"Joseph M Cannova,Muriel R Battaglia,Gregory W Roloff,Sinan Cetin,Michael Tallarico,Wendy Stock,Anand A Patel,Olatoyosi Odenike,Richard A Larson,Michael J Thirman,Mariam T Nawas,Peng Wang,Melissa Y Tjota,Jeremy P Segal,Girish Venkataraman,Adam S DuVall,Michael W Drazer","doi":"10.3324/haematol.2025.288518","DOIUrl":"https://doi.org/10.3324/haematol.2025.288518","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"104 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146073140","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 : 2026-01-29DOI: 10.3324/haematol.2025.288773
Pedro Garrido-Rodríguez,Belén De la Morena-Barrio,Carlos Bravo-Pérez,Rosa Cifuentes-Riquelme,José Padilla,Esther Navarro,María Llamas-López,Antonia Miñano,Raúl Teruel,Agustín Rodríguez-Alen,Francisco Velasco,María J Blanco,María F López-Fernández,Beatriz Fernández-Pérez,Susana Asenjo,José R González-Porras,Vicente Vicente,María L Lozano,María E De la Morena-Barrio,Javier Corral
Not available.
不可用。
{"title":"De novo mutations in antithrombin deficiency: high frequency and heterogeneous mechanisms.","authors":"Pedro Garrido-Rodríguez,Belén De la Morena-Barrio,Carlos Bravo-Pérez,Rosa Cifuentes-Riquelme,José Padilla,Esther Navarro,María Llamas-López,Antonia Miñano,Raúl Teruel,Agustín Rodríguez-Alen,Francisco Velasco,María J Blanco,María F López-Fernández,Beatriz Fernández-Pérez,Susana Asenjo,José R González-Porras,Vicente Vicente,María L Lozano,María E De la Morena-Barrio,Javier Corral","doi":"10.3324/haematol.2025.288773","DOIUrl":"https://doi.org/10.3324/haematol.2025.288773","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"281 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146073147","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 : 2026-01-29DOI: 10.3324/haematol.2025.289079
Jiani N Chai,Paul Dennis Simonson,Kameisha Gulgar,Frank Costanzo,David S Jayabalan,Mark Bustoros,Giorgio Ga Inghirami,Julia T Geyer,Mateo Mejia Saldarriaga
Not available.
不可用。
{"title":"Morphologic and immunophenotypic characterization of lymphocytosis following BCMAtargeted CAR-T cell therapy in relapsed/refractory multiple myeloma.","authors":"Jiani N Chai,Paul Dennis Simonson,Kameisha Gulgar,Frank Costanzo,David S Jayabalan,Mark Bustoros,Giorgio Ga Inghirami,Julia T Geyer,Mateo Mejia Saldarriaga","doi":"10.3324/haematol.2025.289079","DOIUrl":"https://doi.org/10.3324/haematol.2025.289079","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"72 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146073122","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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