American Journal of Hematology最新文献

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MRD and NK‐Cell Chimerism Predict Transplant Outcomes in Allogeneic Hematopoietic Stem Cell Transplantation Recipients MRD和NK细胞嵌合预测同种异体造血干细胞移植接受者的移植结果
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-19 DOI: 10.1002/ajh.27750
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
{"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}
引用次数: 0
Comparison of Frontline Therapies in Older Adults Age ≥ 80 Years With Chronic Lymphocytic Leukemia (CLL): A Mayo Clinic and Danish Nation-Wide Study 老年≥80岁慢性淋巴细胞白血病(CLL)一线治疗的比较:梅奥诊所和丹麦全国范围的研究
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-18 DOI: 10.1002/ajh.27747
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
{"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":"<p>People with chronic lymphocytic leukemia (CLL) have a median age at diagnosis of around 70 years [<span>1</span>]. 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 [<span>1</span>]. People ≥ 80 years old often have comorbidities and unfavorable disease characteristics (like unmutated IGHV and 17p deletion) associated with lower survival [<span>2</span>]. 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 < 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.</p>\u0000<p>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.</p>\u0000<p>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}
引用次数: 0
Hereditary Elliptocytosis Resulting From Heterozygosity for β Spectrin Tandil. β Spectrin Tandil杂合性引起的遗传性椭圆细胞增多症。
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-18 DOI: 10.1002/ajh.27744
María-Angustias Molina-Arrebola,Barbara J Bain
{"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}
引用次数: 0
Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): 2025 Update on Diagnosis, Pathophysiology, Risk Assessment, and Management 母浆细胞样树突状细胞肿瘤(BPDCN): 2025年诊断、病理生理学、风险评估和管理的最新进展
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-17 DOI: 10.1002/ajh.27737
Shai Shimony, Marlise R. Luskin, Naseema Gangat, Nicole R. LeBoeuf, Angela M. Feraco, Andrew A. Lane
{"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}
引用次数: 0
Digital Necrosis as Presenting Symptom of Polycythemia Vera 真性红细胞增多症的主要症状是指坏死
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-14 DOI: 10.1002/ajh.27742
Charly Edmiston, Brett J. Carroll, Jason A. Freed
{"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}
引用次数: 0
Integrative Genomic and Transcriptomic Analysis Reveals Targetable Vulnerabilities in Angioimmunoblastic T‐Cell Lymphoma 综合基因组学和转录组学分析揭示了血管免疫母细胞淋巴瘤的可靶向性脆弱性
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-13 DOI: 10.1002/ajh.27736
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
{"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<jats:sub>FH</jats:sub>) lymphoma of the angioimmunoblastic (AITL) subtype has a dismal prognosis. Using whole‐exome sequencing (<jats:italic>n</jats:italic> = 124), transcriptomic (<jats:italic>n</jats:italic> = 78), and methylation (<jats:italic>n</jats:italic> = 40) analysis, we identified recurrent mutations in known epigenetic drivers (<jats:italic>TET2, DNMT3A, IDH2</jats:italic><jats:sup><jats:italic>R172</jats:italic></jats:sup>) and novel ones (<jats:italic>TET3, KMT2D</jats:italic>). <jats:italic>TET2, IDH2</jats:italic><jats:sup><jats:italic>R172</jats:italic></jats:sup>, <jats:italic>DNMT3A</jats:italic> co‐mutated AITLs had poor prognosis (<jats:italic>p</jats:italic> < 0.0001). Genes regulating T‐cell receptor (TCR) signaling (<jats:italic>CD28, PLCG1, VAV1</jats:italic>, <jats:italic>FYN</jats:italic>) or activation (<jats:italic>RHOA</jats:italic><jats:sup><jats:italic>G17V</jats:italic></jats:sup>) or regulators of the PI3K‐pathway (PIK(3)C members, <jats:italic>PTEN, PHLPP1, PHLPP2</jats:italic>) were mutated. <jats:italic>CD28</jats:italic> mutation/fusion was associated with poor prognosis (<jats:italic>p</jats:italic> = 0.02). WES of purified, neoplastic T‐cell (CD3<jats:sup>+</jats:sup>PD1<jats:sup>+</jats:sup>) demonstrated high concordance with whole tumor biopsies and validated the presence of <jats:italic>TET2</jats:italic> and <jats:italic>DNMT3A</jats:italic> in tumor and non‐lymphoid cells, but other mutations (<jats:italic>CD28</jats:italic>, <jats:italic>RHOA</jats:italic><jats:sup><jats:italic>G17V</jats:italic></jats:sup>, <jats:italic>IDH2</jats:italic><jats:sup><jats:italic>R172</jats:italic></jats:sup>, <jats:italic>PLCG1</jats:italic>) 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 (<jats:italic>p</jats:italic> < 0.01). RNA‐seq and WES analysis of 12 AITL‐patient‐derived‐xenografts (PDX) showed that bi‐allelic <jats:italic>TET2</jats:italic> and <jats:italic>DNMT3A</jats:italic> mutations or sub‐clonal mutations (<jats:italic>PLCG1, PHLPP2</jats:italic>) propagated in sequential passages, and gene signatures related to T<jats:sub>FH</jats:sub> and T<jats:sub>CM</jats:sub> (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 <jats:italic>PHLPP2</jats:italic> mRNA expression predicted poor prognosis (<jats:italic>p</jats:italic> = 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}
引用次数: 0
Poor Engraftment After Posttransplant Cyclophosphamide Graft-Versus-Host DISAESE Prophylaxis in Patients With Myelofibrosis. 骨髓纤维化患者移植后植入不良的环磷酰胺移植物抗宿主病预防。
IF 10.1 1区 医学
American Journal of Hematology Pub Date : 2025-06-12 DOI: 10.1002/ajh.27741
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
{"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}
引用次数: 0
Mycosis Fungoides, Sézary Syndrome, and Cutaneous B-Cell Lymphomas: 2025 Update on Diagnosis, Risk-Stratification, and Management 蕈样真菌病、ssamzary综合征和皮肤b细胞淋巴瘤:2025年诊断、风险分层和管理的最新进展
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-10 DOI: 10.1002/ajh.27735
Alexandra C. Hristov, Trilokraj Tejasvi, Ryan A. Wilcox
{"title":"Mycosis Fungoides, Sézary Syndrome, and Cutaneous B-Cell Lymphomas: 2025 Update on Diagnosis, Risk-Stratification, and Management","authors":"Alexandra C. Hristov, Trilokraj Tejasvi, Ryan A. Wilcox","doi":"10.1002/ajh.27735","DOIUrl":"https://doi.org/10.1002/ajh.27735","url":null,"abstract":"Primary cutaneous lymphomas are a rare and heterogeneous group of extranodal lymphomas that require the integration of clinical and histopathologic data for classification and treatment.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"140 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144260227","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}
引用次数: 0
Phase II Trial of Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation Conditioning With Haploidentical Donor Peripheral Blood Stem Cell Transplant 低强度氟达拉滨、美法兰和全身照射治疗单倍体供体外周血干细胞移植的II期试验
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-10 DOI: 10.1002/ajh.27738
Hany Elmariah, Jongphil Kim, Rebecca Gonzalez, Elizabeth DiMaggio, Samer Sansil, Asmita Mishra, Rawan Faramand, Lia Perez, Aleksandr Lazaryan, Abu-Sayeef Mirza, Farhad Khimani, Hien Liu, Jose L. Ochoa-Bayona, Michael Nieder, Fabiana Perna, Nicholas Figura, Timothy J. Robinson, Taiga Nishihori, Claudio Anasetti, Joseph A. Pidala, Nelli Bejanyan
{"title":"Phase II Trial of Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation Conditioning With Haploidentical Donor Peripheral Blood Stem Cell Transplant","authors":"Hany Elmariah, Jongphil Kim, Rebecca Gonzalez, Elizabeth DiMaggio, Samer Sansil, Asmita Mishra, Rawan Faramand, Lia Perez, Aleksandr Lazaryan, Abu-Sayeef Mirza, Farhad Khimani, Hien Liu, Jose L. Ochoa-Bayona, Michael Nieder, Fabiana Perna, Nicholas Figura, Timothy J. Robinson, Taiga Nishihori, Claudio Anasetti, Joseph A. Pidala, Nelli Bejanyan","doi":"10.1002/ajh.27738","DOIUrl":"https://doi.org/10.1002/ajh.27738","url":null,"abstract":"&lt;p&gt;Human leukocyte antigen (HLA) haploidentical (haplo) hematopoietic cell transplantation (HCT) with post-transplant cyclophosphamide (PTCy) has expanded access to this curative therapy with rates of graft-versus-host disease (GVHD) that are similar to HLA matched donor HCT [&lt;span&gt;1&lt;/span&gt;]. The original Johns Hopkins PTCy platform uses a reduced intensity conditioning (RIC) regimen consisting of Fludarabine (Flu) 150 mg/m&lt;sup&gt;2&lt;/sup&gt;, Cy 14.5 mg/kg on Day-6 and-5, total body irradiation (TBI) 200 cGy on Day-1 (Flu/Cy/TBI) and a bone marrow graft (BMT). Though this regimen yields favorable toxicity including rates of non-relapse mortality (NRM) ~10%–15%, severe acute GVHD ~10%, and chronic GVHD ~10%, outcomes have been marred by high rates of disease relapse &gt; 50% [&lt;span&gt;1&lt;/span&gt;].&lt;/p&gt;\u0000&lt;p&gt;While myeloablative conditioning (MAC) regimens can reduce the risk of relapse, older and frail patients cannot tolerate MAC regimens. To improve relapse after RIC haplo HCT with PTCy, investigators at the MD Anderson Cancer Center attempted a conditioning regimen consisting of Flu 160 mg/m&lt;sup&gt;2&lt;/sup&gt;, melphalan (Mel) 100–140 mg/m&lt;sup&gt;2&lt;/sup&gt;, and either Thiotepa 5 mg/kg or TBI 200 cGy [&lt;span&gt;2&lt;/span&gt;]. While they reported low 1-year relapse rates of 19%, NRM was high at 21%, resulting in 1-year disease-free survival (DFS) of 60%.&lt;/p&gt;\u0000&lt;p&gt;As haplo HCT with PTCy is increasingly used, strategies to improve outcomes are essential. We hypothesized that reducing the Mel dose to 70 mg/m&lt;sup&gt;2&lt;/sup&gt; in a Flu/Mel70/TBI regimen followed by haplo peripheral blood stem cell transplant (PBSCT) with PTCy would successfully improve DFS by reducing relapse without a significant increase in NRM compared to Flu/Cy/TBI haplo HCT in patients unfit for MAC.&lt;/p&gt;\u0000&lt;p&gt;This trial, NCT04191187, is single-institution phase II study of Flu/Mel70/TBI haplo PBSCT with PTCy/sirolimus/mycophenolate mofetil (MMF) in patients with high-risk hematologic malignancies. It was approved by the Advarra Institutional Review Board and is compliant with the Declaration of Helsinki.&lt;/p&gt;\u0000&lt;p&gt;Eligible patients were required to be ≥ 55 years or &lt; 55 years with significant comorbidities defined as an HCT comorbidity index (HCT-CI) ≥ 3, and receiving an HLA haplo donor HCT for a hematologic malignancy.&lt;/p&gt;\u0000&lt;p&gt;All patients received conditioning with Flu 30 mg/m&lt;sup&gt;2&lt;/sup&gt;/day from −6 to −2 based on actual body weight, Mel 70 mg/mg&lt;sup&gt;2&lt;/sup&gt; on day-6 based on actual body weight, and TBI 200 cGy on day-1. On day 0, patients received the PBSCT (Figure 1A). The target CD34+ cell dose was 5 × 10&lt;sup&gt;6&lt;/sup&gt; CD34+ cells/kg, with a maximum allowable dose of 7 × 10&lt;sup&gt;6&lt;/sup&gt; CD34+ cells/kg. GVHD prophylaxis consisted of PTCy/tacrolimus/MMF for the first five subjects. The protocol was subsequently modified to follow the PTCy/sirolimus/MMF platform published separately by our group [&lt;span&gt;3&lt;/span&gt;].&lt;/p&gt;\u0000&lt;figure&gt;&lt;picture&gt;\u0000&lt;source media=\"(min-width: 1650px)\" srcset=\"/cms/asset/78a4833a-5adb-42fb-a926-7058ec195f","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"8 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144252847","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}
引用次数: 0
Common Lymphoma in an Uncommon Location 罕见部位的常见淋巴瘤
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-06-09 DOI: 10.1002/ajh.27740
Jordan Carter, Jonathan M. von Reusner, Mark A. Sellmyer, Adam Bagg, Stefan K. Barta
{"title":"Common Lymphoma in an Uncommon Location","authors":"Jordan Carter, Jonathan M. von Reusner, Mark A. Sellmyer, Adam Bagg, Stefan K. Barta","doi":"10.1002/ajh.27740","DOIUrl":"https://doi.org/10.1002/ajh.27740","url":null,"abstract":"FDG avid bilateral ureteral lesions.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"57 12 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144237808","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}
引用次数: 0
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