American Journal of Hematology最新文献

{"title":"Thromboembolic Events Are Increased After Splenectomy in Postmenopausal Women","authors":"Qi Feng, Yi Mu, Christopher Kabrhel, Bernard Rosner, Rulla M. Tamimi, James B. Bussel","doi":"10.1002/ajh.27714","DOIUrl":"https://doi.org/10.1002/ajh.27714","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"55 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144328531","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":"Luspatercept in Transfusion‐Dependent β‐Thalassemia: The Benefit Is Real, and So Are the Risks","authors":"Khaled M. Musallam","doi":"10.1002/ajh.27745","DOIUrl":"https://doi.org/10.1002/ajh.27745","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"12 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144319752","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":"Efficacy and Safety of Hetrombopag Versus Thrombopoietin in Promoting Platelet Engraftment After Allogeneic Hematopoietic Stem Cell Transplantation: A Prospective, Multicenter, Randomized Controlled Clinical Trial","authors":"Yimei Feng, Linhui Wang, Mingqiang Ren, Dan Wang, Tao Lang, Jianchuan Deng, Shifeng Lou, Hai Yi, Liangliang Ma, Hongyun Xing, Pengcheng He, Xiaoning Wang, Sanbin Wang, Jishi Wang, Han Yao, Ting Chen, Jia Liu, Lu Zhao, Yuqing Liu, Xiaoqi Wang, Li Li, Shuhan Tang, Lidan Zhu, Jia Liu, Shichun Gao, Huanfeng Liu, Lu Wang, Peiyan Kong, Lei Gao, Xi Zhang","doi":"10.1002/ajh.27746","DOIUrl":"https://doi.org/10.1002/ajh.27746","url":null,"abstract":"Allogeneic hematopoietic stem cell transplantation (allo‐HSCT) is pivotal for hematological malignancies but faces challenges in delayed platelet engraftment, poor graft function (PGF), and bleeding risks. Hetrombopag, a thrombopoietin receptor agonist, was evaluated to enhance platelet recovery posttransplant. Patients undergoing allo‐HSCT were randomized on Day 3 Post‐Infusion into low‐dose (2.5 mg/day), high‐dose (5 mg/day) hetrombopag groups, or a control group receiving thrombopoietin (300 U/kg/day). Endpoints included platelet/neutrophil engraftment time, PGF incidence, transfusion needs, and safety. Among 212 analyzed patients, platelet engraftment was faster in hetrombopag groups (median 13 days) versus controls (15 days; <jats:italic>p</jats:italic> = 0.001), with no dose‐dependent difference (<jats:italic>p</jats:italic> = 0.821). Neutrophil engraftment was also accelerated (13 vs. 15 days; <jats:italic>p</jats:italic> = 0.002). PGF incidence was lower in hetrombopag groups (5.04%) versus controls (13.7%; <jats:italic>p</jats:italic> = 0.042). The experimental group required fewer platelet transfusions (<jats:italic>p</jats:italic> = 0.021), had reduced gastrointestinal bleeding, and lower costs. Secondary platelet recovery failure showed no intergroup differences. Hetrombopag safely accelerates platelet and neutrophil engraftment, reduces PGF risk, and decreases transfusion dependency post‐allo‐HSCT. Low‐dose hetrombopag demonstrated efficacy equivalent to high‐dose, offering a cost‐effective strategy to improve transplant outcomes.Trial Registration: ChiCTR2200057764.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"8 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144319318","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":"MRD and NK‐Cell Chimerism Predict Transplant Outcomes in Allogeneic Hematopoietic Stem Cell Transplantation Recipients","authors":"Xing Chen, Huihong Huang, Weihao Chen, Yeqian Zhao, Chuanhe Jiang, Luxiang Wang, Zengkai Pan, Jiayu Huang, Zilu Zhang, Haiyang Lu, Yan Sheng, Xiangqin Weng, Yanmin Zhao, Yang Cao, Xiaoxia Hu","doi":"10.1002/ajh.27750","DOIUrl":"https://doi.org/10.1002/ajh.27750","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"42 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144319750","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":"Comparison of Frontline Therapies in Older Adults Age ≥ 80 Years With Chronic Lymphocytic Leukemia (CLL): A Mayo Clinic and Danish Nation-Wide Study","authors":"Mazie Tsang, Paul J. Hampel, Kari G. Rabe, Christian Brieghel, Wei Ding, Jose F. Leis, Talal Hilal, Saad S. Kenderian, Yucai Wang, Eli Muchtar, Amber B. Koehler, Daniel L. Van Dyke, Curtis A. Hanson, Min Shi, Susan L. Slager, Neil E. Kay, Henrik Hjalgrim, Carsten U. Niemann, Sameer A. Parikh, Emelie C. Rotbain","doi":"10.1002/ajh.27747","DOIUrl":"https://doi.org/10.1002/ajh.27747","url":null,"abstract":"&lt;p&gt;People with chronic lymphocytic leukemia (CLL) have a median age at diagnosis of around 70 years [&lt;span&gt;1&lt;/span&gt;]. With an average time to first treatment of 4–5 years from diagnosis, it is estimated that a third of all people with CLL will be ≥ 80 years old when they become symptomatic and receive frontline therapy [&lt;span&gt;1&lt;/span&gt;]. People ≥ 80 years old often have comorbidities and unfavorable disease characteristics (like unmutated IGHV and 17p deletion) associated with lower survival [&lt;span&gt;2&lt;/span&gt;]. Despite efforts to include advanced age in the eligibility criteria, landmark CLL clinical trials that led to the approval of ibrutinib and venetoclax primarily included participants &lt; 75 years old, making it challenging to apply their findings to older patients. To help close this knowledge gap, we conducted a multi-center retrospective cohort study with data spanning nearly three decades to describe treatment outcomes for patients treated for CLL in the frontline setting at ages ≥ 80 years.&lt;/p&gt;\u0000&lt;p&gt;Using the Mayo Clinic CLL Database and the Danish Lymphoid-lineage Cancer Research (DALY-CARE) data (Danish Cohort), we conducted a retrospective cohort study of patients who were ≥ 80 years old at the time of first CLL treatment between January 1995 and November 2022 (Mayo Clinic) and January 2008 and March 2022 (Denmark). The Mayo Clinic CLL Database contains records of consenting patients diagnosed with CLL who were seen at Mayo Clinic in Rochester, MN. The Danish Cohort includes all Danish citizens diagnosed with CLL, with data available from time of birth or immigration, enabled by linkage of personal-level data across national administrative and clinical quality registers using a unique personal identification number. We extracted information about clinical characteristics, patient demographics, and all lines of treatment (Table S1). We calculated overall survival (OS) from the date of first treatment to the date of death or date last known to be alive. OS was displayed using the Kaplan–Meier method. Time to next treatment (TTNT) was calculated from the date of first treatment to earliest date of (a) date of second treatment, (b) date of death, or (c) date last known to be alive. TTNT was visualized using the cumulative incidence method with competing risk of death. Univariable Cox proportional hazards models were used to analyze the association between type of treatment and time to events (i.e., OS or TTNT); results were reported as hazard ratios (HR) and 95% confidence intervals (CI). Analyses were conducted using statistical software SAS 9.4 (SAS Institute Inc., Cary, NC, USA) and R statistical software versions 4.2.0 and 4.3.2. The Mayo Clinic Institutional Review Board and the Danish Health Data Authority and National Ethics Committee (approvals P-2020-561 and 1804410, respectively) approved this study.&lt;/p&gt;\u0000&lt;p&gt;We identified a total of 653 patients aged ≥ 80 years at the time of frontline CLL treatment (216 from Mayo Clinic and 437 fr","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"35 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144311848","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":"Hereditary Elliptocytosis Resulting From Heterozygosity for β Spectrin Tandil.","authors":"María-Angustias Molina-Arrebola,Barbara J Bain","doi":"10.1002/ajh.27744","DOIUrl":"https://doi.org/10.1002/ajh.27744","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"232 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144311575","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":"Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): 2025 Update on Diagnosis, Pathophysiology, Risk Assessment, and Management","authors":"Shai Shimony, Marlise R. Luskin, Naseema Gangat, Nicole R. LeBoeuf, Angela M. Feraco, Andrew A. Lane","doi":"10.1002/ajh.27737","DOIUrl":"https://doi.org/10.1002/ajh.27737","url":null,"abstract":"Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, aggressive hematologic neoplasm that involves multiple organs, including the skin, bone marrow and blood, lymph nodes, and central nervous system. Violaceous tumors or plaques appearing in sun-exposed areas are characteristic and present in most patients. The disease typically affects adult men aged 60–70 years, although children and younger adults may also be affected.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"92 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144305171","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":"Digital Necrosis as Presenting Symptom of Polycythemia Vera","authors":"Charly Edmiston, Brett J. Carroll, Jason A. Freed","doi":"10.1002/ajh.27742","DOIUrl":"https://doi.org/10.1002/ajh.27742","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"12 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144290049","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":"Integrative Genomic and Transcriptomic Analysis Reveals Targetable Vulnerabilities in Angioimmunoblastic T‐Cell Lymphoma","authors":"Alyssa Bouska, Weiwei Zhang, Sunandini Sharma, Harald Holte, Rauf A. Shah, Waseem G. Lone, Mahfuza Afroz Soma, Ruimeng Yang, Xuxiang Liu, Syed Mehmood, Ravneet Singh Chawla, Luca Vincenzo Cappelli, Danilo Fiore, Qiang Gong, Tayla B. Heavican‐Foral, Jeffrey J. Cannatella, Catalina Amador, Aiza Arif, Lynette M. Smith, Soon Thye Lim, Choon Kiat Ong, Andrew L. Feldman, Ming‐Qing Du, Anamarija M. Perry, Laurence de Leval, Timothy C. Greiner, Kai Fu, Gunhild Trøen, Daniel Vodák, Sigve Nakken, Jan Delabie, David Weinstock, Stefano Pileri, Antonella Laginestra, KyeongJin Kim, Utpal Pajvani, Julie M. Vose, Dennis D. Weisenburger, Steven M. Horwitz, Sandeep Dave, Joseph Khoury, Giorgio Inghirami, Wing C. Chan, Javeed Iqbal","doi":"10.1002/ajh.27736","DOIUrl":"https://doi.org/10.1002/ajh.27736","url":null,"abstract":"Nodal follicular helper T‐cell (T&lt;jats:sub&gt;FH&lt;/jats:sub&gt;) lymphoma of the angioimmunoblastic (AITL) subtype has a dismal prognosis. Using whole‐exome sequencing (&lt;jats:italic&gt;n&lt;/jats:italic&gt; = 124), transcriptomic (&lt;jats:italic&gt;n&lt;/jats:italic&gt; = 78), and methylation (&lt;jats:italic&gt;n&lt;/jats:italic&gt; = 40) analysis, we identified recurrent mutations in known epigenetic drivers (&lt;jats:italic&gt;TET2, DNMT3A, IDH2&lt;/jats:italic&gt;&lt;jats:sup&gt;&lt;jats:italic&gt;R172&lt;/jats:italic&gt;&lt;/jats:sup&gt;) and novel ones (&lt;jats:italic&gt;TET3, KMT2D&lt;/jats:italic&gt;). &lt;jats:italic&gt;TET2, IDH2&lt;/jats:italic&gt;&lt;jats:sup&gt;&lt;jats:italic&gt;R172&lt;/jats:italic&gt;&lt;/jats:sup&gt;, &lt;jats:italic&gt;DNMT3A&lt;/jats:italic&gt; co‐mutated AITLs had poor prognosis (&lt;jats:italic&gt;p&lt;/jats:italic&gt; &lt; 0.0001). Genes regulating T‐cell receptor (TCR) signaling (&lt;jats:italic&gt;CD28, PLCG1, VAV1&lt;/jats:italic&gt;, &lt;jats:italic&gt;FYN&lt;/jats:italic&gt;) or activation (&lt;jats:italic&gt;RHOA&lt;/jats:italic&gt;&lt;jats:sup&gt;&lt;jats:italic&gt;G17V&lt;/jats:italic&gt;&lt;/jats:sup&gt;) or regulators of the PI3K‐pathway (PIK(3)C members, &lt;jats:italic&gt;PTEN, PHLPP1, PHLPP2&lt;/jats:italic&gt;) were mutated. &lt;jats:italic&gt;CD28&lt;/jats:italic&gt; mutation/fusion was associated with poor prognosis (&lt;jats:italic&gt;p&lt;/jats:italic&gt; = 0.02). WES of purified, neoplastic T‐cell (CD3&lt;jats:sup&gt;+&lt;/jats:sup&gt;PD1&lt;jats:sup&gt;+&lt;/jats:sup&gt;) demonstrated high concordance with whole tumor biopsies and validated the presence of &lt;jats:italic&gt;TET2&lt;/jats:italic&gt; and &lt;jats:italic&gt;DNMT3A&lt;/jats:italic&gt; in tumor and non‐lymphoid cells, but other mutations (&lt;jats:italic&gt;CD28&lt;/jats:italic&gt;, &lt;jats:italic&gt;RHOA&lt;/jats:italic&gt;&lt;jats:sup&gt;&lt;jats:italic&gt;G17V&lt;/jats:italic&gt;&lt;/jats:sup&gt;, &lt;jats:italic&gt;IDH2&lt;/jats:italic&gt;&lt;jats:sup&gt;&lt;jats:italic&gt;R172&lt;/jats:italic&gt;&lt;/jats:sup&gt;, &lt;jats:italic&gt;PLCG1&lt;/jats:italic&gt;) in neoplastic cells. Integrated DNA‐methylation and mRNA expression analysis revealed epigenetic alterations in genes regulating TCR, cytokines, PI3K‐signaling, and apoptosis. RNA‐seq analysis identified fusion transcripts regulating TCR‐activation (8%), revealed a restricted TCR‐repertoire (α = 87%, β = 72%), and showed the presence of Epstein–Barr virus transcriptome (73%). GEP demonstrated the association of B‐cells or dendritic cells in the tumor milieu with prognosis (&lt;jats:italic&gt;p&lt;/jats:italic&gt; &lt; 0.01). RNA‐seq and WES analysis of 12 AITL‐patient‐derived‐xenografts (PDX) showed that bi‐allelic &lt;jats:italic&gt;TET2&lt;/jats:italic&gt; and &lt;jats:italic&gt;DNMT3A&lt;/jats:italic&gt; mutations or sub‐clonal mutations (&lt;jats:italic&gt;PLCG1, PHLPP2&lt;/jats:italic&gt;) propagated in sequential passages, and gene signatures related to T&lt;jats:sub&gt;FH&lt;/jats:sub&gt; and T&lt;jats:sub&gt;CM&lt;/jats:sub&gt; (central‐memory) were well‐maintained through passages. Gene expression signatures associated with late PDX passages (3rd–5th) were enriched with proliferation and metabolic reprogramming‐related genes and predicted prognosis in an independent AITL series. Low &lt;jats:italic&gt;PHLPP2&lt;/jats:italic&gt; mRNA expression predicted poor prognosis (&lt;jats:italic&gt;p&lt;/jats:italic&gt; = 0.05) and enginee","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"31 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144278262","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":"Poor Engraftment After Posttransplant Cyclophosphamide Graft-Versus-Host DISAESE Prophylaxis in Patients With Myelofibrosis.","authors":"Haesook T Kim, Deborah J Liney, Kristen Rizza, Corey S Cutler, John Koreth, Sarah Nikiforow, Roman M Shapiro, Amar H Kelkar, Mahasweta Gooptu, Rizwan Romee, Catherine J Wu, Joseph H Antin, Jerome Ritz, Robert J Soiffer, Vincent T Ho","doi":"10.1002/ajh.27741","DOIUrl":"https://doi.org/10.1002/ajh.27741","url":null,"abstract":"<p><p>Patients with myelofibrosis (MF) often have impaired engraftment after allogeneic hematopoietic cell transplantation (alloHCT). To determine whether posttransplant cyclophosphamide (PTCY) exerts a detrimental impact on engraftment, we compared clinical outcomes of all patients receiving PTCY (N = 49) and no-PTCY (N = 89) regimens as GVHD prophylaxis after first alloHCT for MF from 2016 to 2023. Median age was 64 in both groups, and baseline characteristics were balanced except that the PTCY group received more haploidentical transplants and were more likely to have secondary MF. Among engrafted individuals, median time to neutrophil engraftment was D + 18 for PTCY and D + 15 for no-PTCY (p < 0.0001). Median time to platelet recovery was D + 33.5 and D + 26 for PTCY and no-PTCY, respectively (p = 0.0006). The graft failure rate was higher for PTCY but did not reach statistical significance (27% for PTCY and 15% for non-PTCY, p = 0.095). The need for donor lymphocyte infusion, CD34 boost, or second transplant was significantly higher for PTCY (33% vs. 13% at 1-year, respectively, p = 0.004). Most cases of graft failure in both cohorts were due to prolonged cytopenias in the absence of relapse. While T-cell chimerism at D + 30 and D + 100 was robust after PTCY, the proportion of patients with granulocyte chimerism ≥ 90% at Day + 100 was significantly lower in PTCY (76% vs. 98.2%, respectively, p = 0.0028). All other outcomes were similar except for a significantly lower chronic GVHD rate in PTCY compared with no-PTCY (p = 0.0002). GVHD prophylaxis with PTCY in patients with MF is associated with delayed engraftment, lower donor granulocyte chimerism, and increased need for additional donor cell infusions after transplant without compromising survival.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":10.1,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144281996","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}