American Journal of Hematology最新文献

{"title":"Cytomolecular Mechanisms of Relapse and Post‐Relapse Outcomes After Frontline FLT3 Inhibitor‐Based Therapy in FLT3‐Mutated AML","authors":"Sankalp Arora, Sanam Loghavi, Naval Daver, Farhad Ravandi, Courtney D. DiNardo, Tapan M. Kadia, Gautam Borthakur, Elias Jabbour, Musa Yilmaz, Ghayas C. Issa, Justin McCormack, Omer Karrar, Sherry Pierce, Hagop Kantarjian, Nicholas J. Short","doi":"10.1002/ajh.70053","DOIUrl":"https://doi.org/10.1002/ajh.70053","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"301 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144928249","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":"Sialyl‐T Antigen: A Novel Red Blood Cell Determinant for Plasmodium falciparum Invasion","authors":"Mahmoud Mikdar, Estela Shabani, Christof Grüring, Mudit Chaand, Usheer Kanjee, Jonathan M. Goldberg, Slim Azouzi, Jacob A. Tennessen, Brendan Elsworth, Cyrianne Keutcha, Natasha S. Barteneva, John G. Doench, Manoj T. Duraisingh","doi":"10.1002/ajh.70037","DOIUrl":"https://doi.org/10.1002/ajh.70037","url":null,"abstract":"Malaria continues to pose significant health challenges globally despite advances in control measures. <jats:italic>Plasmodium falciparum</jats:italic>, the parasite responsible for most severe malaria cases, uses multiple redundant invasion pathways to enter the red blood cell (RBC) during the blood stage of infection. Through a combination of RNA interference screening in erythroid cells and validation by CRISPR/Cas9‐mediated knockout in primary human hematopoietic stem cells, we identified the glycosyltransferase Core 1 Synthase Glycoprotein‐N‐Acetylgalactosamine 3‐Beta‐Galactosyltransferase 1 (C1GALT1) as a novel host determinant for <jats:italic>P. falciparum</jats:italic> invasion. Analyses of C1GALT1‐deficient cultured reticulocytes and RBCs with the glycophorin A/B‐null M<jats:sub>k</jats:sub>M<jats:sub>k</jats:sub> blood group phenotype demonstrated that the C1GALT1‐dependent α(2‐3) sialic acid structures within mucin‐type O‐glycans are crucial for efficient invasion of both sialic acid‐dependent and sialic acid‐independent <jats:italic>P. falciparum</jats:italic> strains, but not the primate malaria parasite <jats:italic>Plasmodium knowlesi</jats:italic>. However, different <jats:italic>P. falciparum</jats:italic> parasite strains exhibit variable dependencies on distinct sialic acid configurations on the RBC surface. Overall, our findings highlight a key role for RBC glycans in malaria infection.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"26 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144919391","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 Outcomes of Transplant Ineligible Older Adults With Multiple Myeloma Treated With Novel Regimens: A Population‐Based Retrospective Cohort Study","authors":"Nadine Abdallah, Hira Mian, Hsien Seow, Gregory P. Pond, Anastasia Gayowsky, Alissa Visram, Luciano J. Costa, Tanya M. Wildes, Rafael Fonseca, Charlotte Pawlyn, Gordon Cook, Shaji Kumar, Rajshekhar Chakraborty","doi":"10.1002/ajh.70042","DOIUrl":"https://doi.org/10.1002/ajh.70042","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"13 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144919393","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":"Developmental Origins of the Heterogeneous Hematopoiesis","authors":"Shaokang Mo, Kuangyu Yen","doi":"10.1002/ajh.70050","DOIUrl":"https://doi.org/10.1002/ajh.70050","url":null,"abstract":"Hematopoietic stem and progenitor cells (HSPCs) are initially generated during embryogenesis, laying the foundation for the hematopoietic system. These embryonic HSPCs exhibit functional heterogeneity, which is essential for their diverse roles in both fetal and adult hematopoiesis. Growing evidence suggests that this heterogeneity arises from hemogenic endothelium (HE), a specialized subset of endothelial precursors with hematopoietic potential. Additionally, studies show that hematopoietic stem cells (HSCs) and multipotent hematopoietic progenitors (MPPs) emerge independently from HE, with notable contributions from non‐endothelial sources. This review explores the spatiotemporal heterogeneity of HE and discusses how this diversity contributes to the emergence of HSPCs. We propose an integrated framework that refines current models by highlighting the regional and temporal variations in HE that drive HSPC diversification. By synthesizing current knowledge, this review offers novel insights into the origins of hematopoietic diversity and the factors that govern HE differentiation.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"1 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144915675","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":"Impact of Donor Type on Outcomes After Allogeneic Hematopoietic Cell Transplantation in Myelofibrosis: A Study on Behalf of the Chronic Malignancies Working Party of the EBMT","authors":"Juan Carlos Hernández‐Boluda, Vipul Sheth, Simona Iacobelli, Linda Koster, Nicolaus Kröger, Patrizia Chiusolo, Thomas Schroeder, Marie Robin, Massimiliano Gambella, Didier Blaise, Henrik Sengeloev, Jakob Passweg, Mattias Stelljes, Robert Zeiser, Ibrahim Yakoub‐Agha, Andrew Clark, Urpu Salmenniemi, Piero Galieni, Werner Rabisch, Kavita Raj, Joanna Drozd‐Sokolowska, Giorgia Battipaglia, Nicola Polverelli, Tomasz Czerw, Donal P. McLornan","doi":"10.1002/ajh.70049","DOIUrl":"https://doi.org/10.1002/ajh.70049","url":null,"abstract":"Selecting the optimal donor is crucial for optimizing results of allogeneic hematopoietic cell transplantation (allo‐HCT). We analyzed outcomes based on donor type in 2809 myelofibrosis (MF) patients undergoing first allo‐HCT between 2015 and 2021 at EBMT centers. Study outcomes included overall survival (OS), progression‐free survival (PFS), relapse, non‐relapse mortality (NRM), engraftment, and graft‐versus‐host disease (GvHD). Four groups were compared: matched sibling donor (MSD, <jats:italic>n</jats:italic> = 742), matched unrelated donor (MUD, <jats:italic>n</jats:italic> = 1401), mismatched unrelated donor (MMUD, <jats:italic>n</jats:italic> = 379) and haploidentical donor (HD, <jats:italic>n</jats:italic> = 287). After a median follow‐up of 33.5 months, 3‐year OS rates were 65.8%, 61.5%, 53.2%, and 57.7% for MSD, MUD, MMUD, and HD, respectively. Multivariable analyses (MSD as reference) showed that donor type significantly correlated with OS (HR: 1.63 for MMUD, HR: 1.42 for HD), PFS (HR: 1.38 for MMUD), NRM (HR: 1.73 for MMUD, HR: 1.47 for HD), engraftment (HR: 0.72 for MMUD, HR: 0.40 for HD), grade 2–4 acute GvHD (HR: 1.53 for MUD, HR: 1.69 for MMUD, HR: 1.49 for HD), and extensive chronic GvHD (HR: 0.77 for MUD, HR: 0.65 for HD). Donor type was not associated with relapse risk. In patients over 60 years, correlations between donor type and outcomes were consistent with those in the overall study population. In summary, with current practices, MF patients receiving MSD or MUD grafts achieve comparable outcomes. In contrast, MMUD and HD transplants have worse OS due to increased NRM. MMUD transplants have a higher risk of GvHD than HD transplants, but this difference seems to disappear with post‐transplant cyclophosphamide.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"27 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144919395","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":"Narsoplimab Results in Excellent Survival in Adults and Children With Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy (TA‐TMA)","authors":"Michelle L. Schoettler, Sunil Kumar Pusarla, Narinder Nangia, Miguel‐Angel Perales, Rafael F. Duarte, Andreas Grauer, Alessandro Rambaldi","doi":"10.1002/ajh.70044","DOIUrl":"https://doi.org/10.1002/ajh.70044","url":null,"abstract":"Inappropriate complement activation is a key driver of hematopoietic cell transplant‐associated thrombotic microangiopathy (TA‐TMA). Treatment with narsoplimab, an inhibitor of MASP‐2, the effector enzyme of the lectin pathway, resulted in a response rate of 61% in a Phase 2 clinical trial in adults with TA‐TMA. Given these promising results, a global expanded access program (EAP) was established facilitating compassionate use treatment. Herein, we report the survival of children (&lt; 16 years old) and adults (≥ 16 years old) enrolled in the EAP from October 2017 to October 2023 (<jats:italic>n</jats:italic> = 136). Among children (<jats:italic>n</jats:italic> = 50), the majority underwent allogeneic HCT (<jats:italic>n</jats:italic> = 44); 37 were considered high‐risk (HR) and 30 (81.1%) had organ dysfunction at the time of TA‐TMA diagnosis. One‐year overall survival (OS) in pediatric allogeneic recipients with HR TA‐TMA who received narsoplimab as first‐line therapy (<jats:italic>n</jats:italic> = 12) was 75.0% and 56.2% in those who received treatment as ≥ second‐line (<jats:italic>n</jats:italic> = 25, 20 refractory to eculizumab). One‐year OS in the six children with a solid tumor who underwent an autologous HCT was 80%. Among the 86 adults, 84 received an allogeneic HCT, 65 had HR TA‐TMA and, among them, 57 (87.7%) had organ dysfunction at diagnosis. One‐year OS in allogeneic adults with HR TA‐TMA who received narsoplimab as first line therapy (<jats:italic>n</jats:italic> = 49) was 58.0%, and 40.5% in those who received narsoplimab as ≥ second line therapy (<jats:italic>n</jats:italic> = 16). There were no concerning safety signals. In this real‐world study enriched with patients with severe TA‐TMA, survival was excellent in children and adults, supporting the use of narsoplimab.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"37 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144919394","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":"Outcomes and Patterns of Relapse of NPM1 Mutated Acute Myeloid Leukemia Treated With Venetoclax Based Therapies","authors":"Wei‐Ying Jen, Sanam Loghavi, Alexandre Bazinet, Alex Bataller, Ian Bouligny, Naval G. Daver, Ghayas C. Issa, Naszrin Arani, Emmanuel Almanza Huante, Abhishek Maiti, Guillermo Montalban‐Bravo, Gautam Borthakur, Nicholas J. Short, Sherry Pierce, Elias Jabbour, Guillermo Garcia‐Manero, Farhad Ravandi, Hagop M. Kantarjian, Courtney D. DiNardo, Tapan M. Kadia","doi":"10.1002/ajh.70046","DOIUrl":"https://doi.org/10.1002/ajh.70046","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"23 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144911194","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":"Innovation Alone Is Not Enough: The Urgent Need for Equitable Access in Thalassemia Care","authors":"Ali T. Taher","doi":"10.1002/ajh.70047","DOIUrl":"https://doi.org/10.1002/ajh.70047","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"31 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144911075","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":"Contemporary Outcomes of Adults With Acute Lymphoblastic Leukemia With KMT2A Rearrangements Treated With Immunotherapy‐Based Regimens","authors":"Hannah Goulart, Elias Jabbour, Nicholas Short, Nitin Jain, Ghayas Issa, Himachandana Atluri, Alex Bataller, Tapan Kadia, Naval Daver, Courtney DiNardo, Rebecca Garris, Jayastu Senapati, Farhad Ravandi, Hagop Kantarjian","doi":"10.1002/ajh.70048","DOIUrl":"https://doi.org/10.1002/ajh.70048","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"1 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144906054","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":"Classic Hairy Cell Leukemia With MAP2K1 Mutation: Diagnosis and Targeted Therapy","authors":"Andrea Duminuco, Alessandra Venanzi, Alessia Santi, Alessandro Mancini, Alessandra Romano, Enrico Tiacci","doi":"10.1002/ajh.70043","DOIUrl":"https://doi.org/10.1002/ajh.70043","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"50 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144899268","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}