American Journal of Hematology最新文献

{"title":"Reduced-Dose Versus Full-Dose Direct Oral Anticoagulants for Extended Secondary Venous Thromboembolism Prophylaxis in Cancer Patients: A Systematic Review and Meta-Analysis of Randomized Trials.","authors":"Amir Mahmoud,Basant Eltaher,Purva Shah,Ranjini Vengilote,Mariam Mostafa,Amber Afzal,Kristen M Sanfilippo","doi":"10.1002/ajh.70090","DOIUrl":"https://doi.org/10.1002/ajh.70090","url":null,"abstract":"Patients with cancer are at high risk for both recurrent venous thromboembolism (VTE) and anticoagulant-associated bleeding. Direct oral anticoagulants (DOACs) have become a standard option for long-term VTE management; however, the safety and efficacy of reduced-dose regimens in cancer populations remain uncertain. We conducted a systematic review and meta-analysis of randomized controlled trials (RCTs) comparing reduced-dose versus full-dose DOACs in adult patients with active cancer and prior VTE who had completed at least 6 months of therapeutic anticoagulation. Embase and MEDLINE were searched from inception through April 1, 2025. Primary outcomes were recurrent VTE and a composite of major bleeding and clinically relevant non-major bleeding. Pooled risk ratios (RRs) were calculated using a random-effects model. Three RCTs including 2178 patients were eligible. There was no significant difference in recurrent VTE between reduced-dose and full-dose apixaban or rivaroxaban (RR, 0.87; 95% CI, 0.10-7.83; I2 = 0%). The composite bleeding risk was numerically lower with reduced-dose DOACs (RR, 0.77; 95% CI, 0.55-1.06; p = 0.07; I2 = 0%). In a subgroup analysis of cancer-only trials (API-CAT and EVE; n = 2126), reduced-dose apixaban significantly reduced the risk of clinically relevant bleeding (RR, 0.77; 95% CI, 0.60-0.99; p = 0.05) without increasing VTE recurrence. Reduced-dose apixaban or rivaroxaban offers comparable efficacy to full-dose regimens for extended secondary VTE prophylaxis in cancer patients, with a potential reduction in clinically relevant bleeding. These findings support consideration of dose de-escalation in select patients with active cancer.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"45 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145127318","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":"Real World Study on the Best CPX-351 Treatment Duration and Timing for Allogeneic Stem Cell Transplantation.","authors":"Fabio Guolo,Luana Fianchi,Maria Paola Martelli,Federico Lussana,Francesco Grimaldi,Federica Pilo,Michela Rondoni,Carla Filì,Paola Minetto,Debora Capelli,Patrizia Chiusolo,Massimo Breccia,Sara Mastaglio,Massimo Bernardi,Monica Bocchia,Monica Fumagalli,Sara Galimberti,Valentina Mancini,Anna Lisa Piccioni,Luca Maurillo,Nicola Stefano Fracchiolla,Raffaele Palmieri,Calogero Vetro,Cristina Papayannidis,Lorenzo Brunetti,Alessandra Sperotto,Federica Gigli,Patrizia Zappasodi,Antonino Mulé,Caterina Patti,Erika Borlenghi,Michelina Dargenio,Federica Lessi,Marco Cerrano,Daniela Cilloni,Alessandro Isidori,Monia Lunghi,Caterina Alati,Carmela Gurrieri,Carola Riva,Giovanni Marconi,Ivana Lotesoriere,Samuele Gatani,Anna Maria Scattolin,Manuela Caizzi,Salvatore Perrone,Atto Billio,Filippo Gherlinzoni,Francesco Mannelli,Michele Gottardi,Roberto Cairoli,Anna Candoni,Felicetto Ferrara,Livio Pagano,Roberto Massimo Lemoli,Adriano Venditti,Elisabetta Todisco","doi":"10.1002/ajh.70083","DOIUrl":"https://doi.org/10.1002/ajh.70083","url":null,"abstract":"In the registration clinical trial 301 (NCT01696084), CPX-351 has shown to be superior to conventional 3 + 7 in secondary AML (s-AML). However, the optimal duration of treatment, the best timing for allogeneic stem cell transplantation (allo-HSCT), and the activity of CPX-351 in specific s-AML subgroups are unclear. To evaluate these aspects, a total of 513 s-AML patients (median age 65.6 years, 19-79) treated with CPX-351 were retrospectively analyzed. Complete remission (CR) rate after induction was 297/513 (58%), increasing to 340/513 (66%) after cycle 2. Among the 340 responding patients, 118 (34.7%), 137 (40.3%), and 85 (25%) received none, one, or two consolidation cycles of CPX-351, respectively. Overall, 230/513 patients (48.8%) received allo-HSCT. Median follow up was 23.66 months and median overall survival (OS) was 16.23 months. Patients with mutated NPM1 or with ELN 2017 favorable risk (p < 0.05) had a significantly longer OS (p < 0.05). In a landmark analysis, receiving allo-HSCT was associated with a longer survival (Median OS not reached vs. 16.3 months for patients receiving or not receiving allo-HSCT, p < 0.05). Completion of all allowed CPX-351 cycles was beneficial only in patients not proceeding to transplant (p < 0.05), whereas in transplanted patients additional CPX-351 cycles did not improve outcome. Our analysis suggests that also s-AML patients with NPM1 mutations and those belonging to the ELN 2017 favorable risk category benefit from CPX-351. In eligible patients, allo-HSCT should be performed as soon as a CR is achieved, whereas patients not undergoing transplant benefit from a complete CPX-351 schedule.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"40 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145127335","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":"Myeloproliferative Neoplasms‐Unclassifiable (MPN‐U): A Carefully Curated Series of 30 Mayo Clinic Patients","authors":"Rania M. Abdelaziz, Priyansh Faldu, Saubia Fathima, Cinthya J. Zepeda Mendoza, Animesh Pardanani, Rong He, Attilio Orazi, Kaaren K. Reichard, Naseema Gangat, Ayalew Tefferi","doi":"10.1002/ajh.70085","DOIUrl":"https://doi.org/10.1002/ajh.70085","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"48 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145116519","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":"Bleeding Patterns and Clinical Outcomes in Patients With Systemic AL Amyloidosis-Related Acquired Factor X Deficiency.","authors":"Okan Cetin,Andrew Staron,Patric Teodorescu,John Mark Sloan,Vaishali Sanchorawala,Raphael Szalat","doi":"10.1002/ajh.70084","DOIUrl":"https://doi.org/10.1002/ajh.70084","url":null,"abstract":"Bleeding patterns and clinical outcomes in patients with systemic AL amyloidosis-related acquired factor X deficiency.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"21 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145103516","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":"Optical Genome Mapping (OGM): Validation and Clinical Utility in the Cytogenetic Diagnosis of Multiple Myeloma.","authors":"Adela Cisneros,Mar Mallo,Aleix Méndez-López,Carmen Villena,Encarnación Santafé,Jessica Tijero,Maria Abel,Neus Ruiz-Xivillé,Gladys Ibarra,Laura Abril,Anna M Sureda,Lourdes Escoda,Yolanda González-Montes,Xavier Ortín,Jose T Navarro,Albert Oriol,Francesc Solé,Isabel Granada","doi":"10.1002/ajh.70077","DOIUrl":"https://doi.org/10.1002/ajh.70077","url":null,"abstract":"Diagram illustrating the final OGM assessment in relation to targeted FISH analysis. On the right, the final OGM results indicate whether additional alterations were detected, no alterations were observed, genetic diagnosis was improved, or if results were inconclusive, including cases where FISH and OGM showed no concordance and OGM did not reveal any alterations.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"5 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145089777","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":"Recursive Partitioning to Differentiate Acquired From Inherited Bone Marrow Failure Syndromes.","authors":"Eléonore Kaphan,Anouk Walter-Petrich,Lise Larcher,Thierry Leblanc,Benedicte Bruno,Edouard Forcade,Cecile Renard,Arthur Sterin,Michael Loschi,Elodie Lainey,Jean Soulier,Caroline Kannengiesser,Gérard Socié,Régis Peffault de Latour,Mony Fahd,Jérome Lambert,Flore Sicre De Fontbrune","doi":"10.1002/ajh.70064","DOIUrl":"https://doi.org/10.1002/ajh.70064","url":null,"abstract":"Distinguishing inherited bone marrow failure (IBMF) from acquired aplastic anemia (aAA) at diagnosis is a major clinical challenge and is essential for appropriate treatment and patients' counseling. Genetic testing to exclude IBMF is frequently subject to delays of several months and may not be available in all centers and could not be applied to all patients in a large number of countries. Our study aimed to develop a practical scoring system to identify patients who are unlikely to have IBMF. Patients in the training set (N = 150) were classified as having either aAA or IBMF based on genetic results and/or response to immunosuppressive therapy. We retrospectively recorded 33 clinical and laboratory characteristics at the time of diagnosis. The diagnostic algorithm was then constructed using recursive partitioning. This unbiased model handles missing values using conditional inference procedures. Model sensitivity addresses the ability to correctly predict aAA. Three of the variables of interest were selected by the model in the final algorithm: morphological abnormalities, PNH clone (≥ 0.1% on granulocytes), and acute onset of BMF (cytopenia < 1 year). In the training set, the algorithm achieved a sensitivity of 96.2% (IC95%: 91.4%-98.8%) and a specificity of 88.2% (IC95%: 63.6-98.5) in differentiating aAA from IBMF. We then applied this algorithm to a validation set of 465 patients (aAA n = 368 (79.1%); IBMF n = 97 (20.9%)) and obtained similar sensitivity of 95.7% (IC95: 93-97.5) and specificity of 91.8% (IC95%: 84.4-96.4) with a positive predictive value of 97.8% (IC95%: 95.7-99). This efficient and practical scoring system might help physicians to identify patients who do not need genetic screening, allowing prompt start of treatment. Positive predictive value may be improved in specific populations by adding telomere length, AFP and HbF values.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"83 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145068305","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":"Perspectives on Selected Hemophilia Research Presented at ISTH 2025: Nonfactor Replacement Therapies.","authors":"Annette von Drygalski","doi":"10.1002/ajh.70051","DOIUrl":"https://doi.org/10.1002/ajh.70051","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"24 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145068307","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":"Independent Origin of Hairy Cell Leukemia (HCL) From Co-Occurring Clonal Hematopoiesis (CH).","authors":"Alessandra Venanzi,Alessia Santi,Renato Zambello,Claudia Giordano,Alessandra Pucciarini,Alessandro Mancini,Brunangelo Falini,Vincenzo Maria Perriello,Enrico Tiacci","doi":"10.1002/ajh.70060","DOIUrl":"https://doi.org/10.1002/ajh.70060","url":null,"abstract":"Independent Origin of Hairy Cell Leukemia (HCL) From Co-Occurring Clonal Hematopoiesis.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"58 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145043516","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":"Third-Generation Bruton tyrosine kinase Inhibitors for the Treatment of Monoclonal Gammopathy of Thrombotic Significance (MGTS).","authors":"Louise Man,Adam J Kanack,Emily E Mauch,Noah P Splinter,Anand Padmanabhan","doi":"10.1002/ajh.70078","DOIUrl":"https://doi.org/10.1002/ajh.70078","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"87 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145043518","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":"Critical Blue-Green Neutrophilic Inclusions in the Context of CD19 CAR-T Toxicity.","authors":"Vibooshini Ganeshalingam,Hannah Sumasundaram,Elizabeth Cameron,Andrew Cross,Jessica Pearce,Kirollos Salah Kamel","doi":"10.1002/ajh.70075","DOIUrl":"https://doi.org/10.1002/ajh.70075","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"14 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145035806","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}