American Journal of Hematology最新文献

{"title":"Ex Vivo IL-15-Stimulated NK Cells as Adoptive Cell Therapy in Haploidentical Transplantation for Pediatric Leukemia","authors":"Carmen Mestre-Durán,&nbsp;Odelaisy León-Triana,&nbsp;Halin Bareke,&nbsp;Lidia Pertíñez,&nbsp;Laura Clares-Villa,&nbsp;David Bueno,&nbsp;Daniel Lozano-Ojalvo,&nbsp;Alfonso Navarro-Zapata,&nbsp;Luisa Sisinni,&nbsp;Isabel Badell-Serra,&nbsp;Marta González-Vicent,&nbsp;Cristina Beléndez,&nbsp;Pilar Palomo-Moraleda,&nbsp;Yasmina Mozo,&nbsp;Víctor Galán,&nbsp;Mikel Fernández Artázcoz,&nbsp;Carlos Echecopar,&nbsp;Mercedes Gasior,&nbsp;Karima Al-Akioui,&nbsp;Jordi Cano-Ochando,&nbsp;Pilar Guerra-García,&nbsp;María Eugenia Fernández-Santos,&nbsp;Cristina Aguirre-Portolés,&nbsp;Antonio Pérez-Martínez","doi":"10.1002/ajh.70023","DOIUrl":"10.1002/ajh.70023","url":null,"abstract":"<p>A Phase I/II clinical trial was conducted in pediatric patients with high-risk malignancies to assess the safety and efficacy of NK cell infusion post-haplo-HSCT. Patients received either alloreactive NK cells from KIR/HLA-mismatched donors or IL-15-stimulated NK cells from KIR/HLA-matched donors, across three dose-escalating cohorts. The IL15-NK cell infusion product demonstrated enhanced activating receptor expression and functional capacity. Increased NKG2D expression was observed in NK cells from patients receiving IL15-NK cells. One-year follow-up showed accelerated immune reconstitution in both the allo-NK and IL15-NK cell arms. IL15-NK recipients exhibited a pro-inflammatory cytokine profile (IL-2, IL-4, IL-6, IFN-γ). Elevated CD69 expression was associated with a higher risk of transplant-related complications.\u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure>\u0000 </p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1898-1901"},"PeriodicalIF":9.9,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.70023","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144797046","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hypomethylating Agent and Venetoclax Combination Is a Safe and Effective Alternative to Intensive Chemotherapy in Older (≥ 70 Years) Patients With Newly Diagnosed Favorable Risk Acute Myeloid Leukemia","authors":"Somedeb Ball,&nbsp;Akriti G. Jain,&nbsp;Najla Al Ali,&nbsp;Luis E. Aguirre,&nbsp;Jan Philipp Bewersdorf,&nbsp;Andrew Kent,&nbsp;Ameera Rose,&nbsp;Alexander Hayden,&nbsp;Alexa Siddon,&nbsp;Jill Lykon,&nbsp;Ellen Madarang,&nbsp;David M. Swoboda,&nbsp;Eric Padron,&nbsp;Kendra Sweet,&nbsp;David A. Sallman,&nbsp;Jeffrey Lancet,&nbsp;Hetty E. Carraway,&nbsp;Justin Watts,&nbsp;Amer Zeidan,&nbsp;Daniel A. Pollyea,&nbsp;Rami S. Komrokji","doi":"10.1002/ajh.70031","DOIUrl":"10.1002/ajh.70031","url":null,"abstract":"&lt;p&gt;The European LeukemiaNet (ELN) 2017 guidelines defined favorable risk acute myeloid leukemia (AML) as cases with specific genetic characteristics, such as &lt;i&gt;t&lt;/i&gt;(8;21), inv (16), &lt;i&gt;NPM1&lt;/i&gt;, and biallelic &lt;i&gt;CEBPA&lt;/i&gt; mutations [&lt;span&gt;1&lt;/span&gt;]. Younger patients with favorable risk AML experience improved outcomes with intensive chemotherapy (IC), but long-term survival remains dismal in older (&gt; 60 years of age) adults with AML [&lt;span&gt;2&lt;/span&gt;]. Advanced age, multiple comorbidities, and poor performance status significantly increase the risk of treatment-related adverse events and early mortality with IC in older patients with AML. The less intensive treatment option of hypomethylating agent and venetoclax combination (HMA-Ven) is approved for use in newly diagnosed AML in patients who are IC-ineligible due to advanced age (75 years or older) or coexisting conditions. The randomized phase III trial (VIALE-A) comparing azacitidine-Ven to azacitidine monotherapy demonstrated significant improvement in overall survival (OS) but excluded patients with favorable risk cytogenetics [&lt;span&gt;3&lt;/span&gt;]. Hence, it is unclear if HMA-Ven is an effective alternative to IC in all patients aged 70 years or more with favorable risk AML.&lt;/p&gt;&lt;p&gt;In this multicenter retrospective study, adult patients aged 70 years or older, with newly diagnosed AML treated at the H. Lee Moffitt Cancer Center, University of Colorado, Cleveland Clinic, Yale Cancer Center, and Sylvester Comprehensive Cancer Center from January 2004 till December 2021, were considered for inclusion. Patients with newly diagnosed favorable risk AML (ELN 2017) [&lt;span&gt;1&lt;/span&gt;], who received IC, HMA-Ven, or HMA alone were eligible. The study protocol was approved by the institutional review boards of all participating institutions. Clinical and genomic data on eligible patients were collected by review of electronic medical records. Response to treatment was assessed per ELN 2017 guidelines [&lt;span&gt;1&lt;/span&gt;]. Composite complete remission (cCR) rate included cases with complete remission (CR) and complete remission with incomplete hematologic recovery (CRi). For patients experiencing a CR or CRi, the relapse-free survival (RFS) was calculated from the time of remission until relapse or death due to any cause. We calculated OS from the time of AML diagnosis. Following the univariate Cox regression analysis of relevant factors for RFS and OS, factors with a &lt;i&gt;p&lt;/i&gt; &lt; 0.10 were included in a multivariate Cox regression model to identify independent predictors of RFS and OS. All statistical analyses were conducted in SPSS statistics (v.29).&lt;/p&gt;&lt;p&gt;Our study included 111 patients with newly diagnosed favorable risk AML (Figure 1A). IC was the frontline treatment in 57 (51%) patients, whereas 29 (26%) received HMA-Ven and 25 (23%) patients were treated with HMA monotherapy. Patients in the IC group were younger than patients in other treatment groups (median age [range] at diagnosis- IC vs. HMA-Ven vs.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1920-1923"},"PeriodicalIF":9.9,"publicationDate":"2025-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.70031","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144791877","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcome of Patients With Acute Myeloid Leukemias or Myelodysplastic Syndromes After Relapsing From Allogeneic Stem Cell Transplantation: The GITMO AML/MDS-Relapse Registry Study","authors":"Malagola Michele,&nbsp;Castagna Luca,&nbsp;Matranga Domenica,&nbsp;Radici Vera,&nbsp;Farina Mirko,&nbsp;Galli Marco,&nbsp;Accorsi Buttini Eugenia,&nbsp;Rubini Vicky,&nbsp;Alati Caterina,&nbsp;Bassi Simona,&nbsp;Biffi Alessandra,&nbsp;Borghero Carlo,&nbsp;Busca Alessandro,&nbsp;Nozzoli Chiara,&nbsp;Carella Angelo Michele,&nbsp;Cavattoni Irene,&nbsp;Cerretti Raffaella,&nbsp;Chiusolo Patrizia,&nbsp;Cimminiello Michele,&nbsp;Cuoghi Angela,&nbsp;De Gobbi Marco,&nbsp;Vincenzo Federico,&nbsp;Galieni Piero,&nbsp;Iori Anna Paola,&nbsp;Olivieri Attilio,&nbsp;Parma Matteo,&nbsp;Patriarca Francesca,&nbsp;Pavone Vincenzo,&nbsp;Picardi Alessandra,&nbsp;Piras Eugenia,&nbsp;Polverelli Nicola,&nbsp;Prezioso Lucia,&nbsp;Rambaldi Benedetta,&nbsp;Scalisi Elvira,&nbsp;Selleri Carmine,&nbsp;Skert Cristina,&nbsp;Spina Alessandro,&nbsp;Tecchio Cristina,&nbsp;Oldani Elena,&nbsp;Eliana Degrandi,&nbsp;Domenico Russo,&nbsp;Martino Massimo","doi":"10.1002/ajh.70030","DOIUrl":"10.1002/ajh.70030","url":null,"abstract":"<p>\u0000 \u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure>\u0000 </p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1902-1905"},"PeriodicalIF":9.9,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.70030","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144786531","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and Safety of the Third-Generation Tyrosine Kinase Inhibitor Olverembatinib in Combination With Inotuzumab Ozogamicin for the Treatment of Adult Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Patients With Refractory/Relapsed Disease or Persistent Minimal Residual Disease Bridging to Hematopoietic Stem Cell Transplantation","authors":"Xiaoyu Zhang,&nbsp;Yigeng Cao,&nbsp;Jialin Wei,&nbsp;Weihua Zhai,&nbsp;Qiaoling Ma,&nbsp;Chen Liang,&nbsp;Xin Chen,&nbsp;Wenbin Cao,&nbsp;Donglin Yang,&nbsp;Aiming Pang,&nbsp;Yi He,&nbsp;Sizhou Feng,&nbsp;Mingzhe Han,&nbsp;Rongli Zhang,&nbsp;Erlie Jiang","doi":"10.1002/ajh.70026","DOIUrl":"10.1002/ajh.70026","url":null,"abstract":"&lt;p&gt;Despite significant advancements in the treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL), particularly the development of tyrosine kinase inhibitors (TKIs), the prognosis of refractory/relapsed (R/R) or persistent minimal residual disease (MRD)-positive Ph + ALL remains poor. For these patients, allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the main approach for long-term disease-free survival [&lt;span&gt;1&lt;/span&gt;]. However, post-transplantation relapses remain a major challenge. The remission depth is strongly associated with HSCT outcomes, highlighting the importance of achieving complete molecular remission (CMR) prior to transplantation [&lt;span&gt;2&lt;/span&gt;]. The combination of TKIs and immune therapies has been the mainstay of therapy for Ph + ALL. The advent of targeted therapies such as the CD22 monoclonal antibody inotuzumab ozogamicin (INO) and the CD19 bispecific T-cell Engager (Blinatumomab, BiTE) has renewed interest in strategies to enhance disease remission while minimizing the cytotoxic effects of conventional chemotherapy [&lt;span&gt;3, 4&lt;/span&gt;]. These advancements offer promising avenues for improving outcomes in high-risk patients.&lt;/p&gt;&lt;p&gt;Olverembatinib, currently the only third-generation TKI available in mainland China, has demonstrated significant efficacy in treating chronic myeloid leukemia (CML) patients with the T315I mutation who are resistant to other TKIs [&lt;span&gt;5&lt;/span&gt;]. However, its role in the treatment of R/R or MRD-positive Ph + ALL, particularly in heavily pretreated patients bridging to transplantation, remains underexplored. To address this gap, we conducted a prospective study to evaluate the efficacy and safety of combining olverembatinib with INO as a bridging therapy prior to transplantation. This approach aims to achieve deeper molecular remission, improve transplantation outcomes, while minimizing treatment-related toxicity in this high-risk patient population.&lt;/p&gt;&lt;p&gt;We conducted two open-label, single-center, investigator-initiated phase II studies in Ph/BCR-ABL1+ ALL patients using this combined treatment but with distinct patient populations. The first study (NCT05603156) enrolled patients with persistent MRD after at least three rounds of chemotherapy, while the second study (ChiCTR2200061432) focused on refractory/relapsed patients. Apart from requirements of primary disease diagnosis and evaluation, patients were required to be older than 16 years and have ≥ 20% of blasts positive for CD22 expression. In both studies, eligible patients received therapy including olverembatinib (40 mg QOD, d1-28) combined with INO (0.6 mg/m&lt;sup&gt;2&lt;/sup&gt;, d1, d8 per 28-day cycle). Bone marrow MRD was assessed at the end of each treatment cycle. Enrolled patients received a maximum of two treatment cycles before proceeding to HSCT. Post-transplant maintenance treatment with olverembatinib was administered after adequate hematopoietic recovery was achieved under physicia","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1924-1928"},"PeriodicalIF":9.9,"publicationDate":"2025-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.70026","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144786530","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CD19 Directed CAR T Therapy for Transformed Follicular Lymphoma: A CIBMTR Analysis","authors":"Swetha Kambhampati Thiruvengadam,&nbsp;Kwang Woo Ahn,&nbsp;Jinalben Patel,&nbsp;Qinghua Lian,&nbsp;Mark Hertzberg,&nbsp;Narendranath Epperla,&nbsp;Leland Metheny,&nbsp;Sanghee Hong,&nbsp;Tania Jain,&nbsp;Mahmoud Aljurf,&nbsp;Amer Beitinjaneh,&nbsp;John Vaughn,&nbsp;Ajay Gopal,&nbsp;Madiha Iqbal,&nbsp;Baldeep Wirk,&nbsp;Shivaprasad Manjappa,&nbsp;Carolina Oliver,&nbsp;Razan Mohty,&nbsp;Mazyar Shadman,&nbsp;Cameron Turtle,&nbsp;Mehdi Hamadani,&nbsp;Alex F. Herrera","doi":"10.1002/ajh.70027","DOIUrl":"10.1002/ajh.70027","url":null,"abstract":"<div>\u0000 \u0000 <p>Transformed follicular lymphoma (tFL) is typically associated with chemotherapy resistance and a poor prognosis. There are limited data regarding outcomes after CD19-directed chimeric antigen receptor T-cell (CAR T) therapy in relapsed/refractory (R/R) tFL. A total of 923 adult patients with R/R tFL who received commercial CD19 CAR T therapy between 2017 and 2023 were identified in the Center for International Blood and Marrow Transplant Research registry. Median age was 64 years (range: 30–86) and median prior lines of therapy was 4 (range: 1–18). Most patients (78%) received axicabtagene ciloleucel, with 67% of patients having resistant disease at the time of CAR T infusion. At a median follow-up of 25 months (range: 1–72) from CAR T infusion, the 2-year overall survival (OS) was 57% (95% CI: 53–60) and progression-free survival (PFS) was 43% (95% CI: 40–47). The 2-year cumulative incidences of relapse or progression (rel/prog) and non-relapse mortality (NRM) were 47% (95% CI: 44–51) and 9% (95% CI: 7–11), respectively. The overall response rate to CAR T was 76%, with a complete response rate of 63%. Grade ≥ 3 cytokine release syndrome (CRS) was observed in 7.1% and grade ≥ 3 immune effector cell–associated neurologic syndrome (ICANS) in 21.6% of patients. Multivariable analysis suggested that resistant disease status at the time of CAR T, use of bridging therapy, and high comorbidity index ≥ 3 were associated with inferior PFS and OS. Older age ≥ 60 significantly increased the risk of NRM. Our study suggests that CD19 CAR T is effective and safe for tFL.</p>\u0000 </div>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1803-1812"},"PeriodicalIF":9.9,"publicationDate":"2025-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144783270","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":"The Impact of Tirabrutinib Monotherapy for the Treatment of Bing-Neel Syndrome: A Multicenter Retrospective Study","authors":"Masuho Saburi,&nbsp;Taro Masunari,&nbsp;Noriko Fukuhara,&nbsp;Yuichiro Inagaki,&nbsp;Arika Shimura,&nbsp;Naoto Imoto,&nbsp;Yuta Hasegawa,&nbsp;Masao Hagihara,&nbsp;Nobuhiko Kobayashi,&nbsp;Hanae Kumekawa,&nbsp;Hideyuki Nakazawa,&nbsp;Kana Miyazaki,&nbsp;Toshiro Kawakita,&nbsp;Takahiro Isshiki,&nbsp;Atsushi Katsube,&nbsp;Shin Fujisawa,&nbsp;Yuichi Horigome,&nbsp;Yusuke Koba,&nbsp;Fumiaki Jinnouchi,&nbsp;Naohiro Sekiguchi","doi":"10.1002/ajh.70029","DOIUrl":"10.1002/ajh.70029","url":null,"abstract":"&lt;p&gt;Waldenström macroglobulinemia (WM) is a rare lymphoproliferative disorder, and Bing-Neel syndrome (BNS) is characterized by the infiltration of WM cells into the central nervous system (CNS) without large cell transformation. Two retrospective studies demonstrated that BNS occurs in approximately 1% of WM patients and may develop at any stage of the disease [&lt;span&gt;1, 2&lt;/span&gt;]. Although a standard treatment strategy has yet to be established for BNS, drugs that cross the blood–brain barrier are commonly used. With conventional chemotherapy regimens, the 3-year overall survival (OS) rate for patients with BNS was reported to be 60%, and a significant number of deaths occurred within 2 years of the diagnosis of BNS, 75% of which were attributed to its progression [&lt;span&gt;2&lt;/span&gt;]. Castillo et al. [&lt;span&gt;3&lt;/span&gt;] examined 28 BNS patients treated with the Bruton's tyrosine kinase inhibitor (BTKi), ibrutinib, and showed symptomatic improvement in 86% of patients and a 2-year event-free survival (EFS) rate of 80%. A recent multicenter study was performed on BNS treated by the second-generation BTKi, zanubrutinib, and the findings obtained indicated its high efficacy and manageable toxicities [&lt;span&gt;4&lt;/span&gt;]. Tirabrutinib, a second-generation BTKi developed in Japan, has been authorized for the treatment of WM [&lt;span&gt;5&lt;/span&gt;] as well as primary CNS lymphoma [&lt;span&gt;6&lt;/span&gt;]; however, evidence for the efficacy and safety of tirabrutinib for BNS in real-world clinical practice is limited. Therefore, we conducted a multicenter retrospective study to examine the outcomes of BNS treated with tirabrutinib.&lt;/p&gt;&lt;p&gt;Patients with BNS treated with at least one dose of tirabrutinib were retrospectively enrolled in this study, which included previously published case reports with extended follow-ups and re-evaluations (the Supporting Information). A definitive diagnosis of BNS was defined by any of the following criteria: atypical lymphocytes, lymphoplasmacytic cells, or plasma cells detected by cytology in the CSF, the confirmation of clonal B-cells in the CSF by flow cytometry, the infiltration of atypical lymphocytes, lymphoplasmacytic cells, or plasma cells in the histopathological diagnosis of CNS tissue, or the detection of the &lt;i&gt;MYD88&lt;/i&gt;\u0000 &lt;sup&gt;\u0000 &lt;i&gt;L265P&lt;/i&gt;\u0000 &lt;/sup&gt; mutation in the CSF. Additionally, a probable diagnosis of BNS was applied to patients who did not fulfill the criteria for a definitive diagnosis of BNS but were deemed appropriate for a diagnosis of BNS based on the exclusion of other diseases, such as only evidence of brain masses, leptomeningeal enhancement, or abnormal spinal cord signals using magnetic resonance imaging (MRI). A definitive diagnosis and probable diagnosis were equally evaluated in the present study. This study was approved by the centralized Ethics Review Committee of the National Hospital Organization Disaster Medical Center, the lead research institution, and by the collaborat","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1912-1915"},"PeriodicalIF":9.9,"publicationDate":"2025-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.70029","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144763124","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"History of Thrombosis at High Altitude Associates With Increased Erythropoietin.","authors":"Ricardo Amaru,Josef Prchal,Mireya Carrasco,Silvia Mancilla,Teddy Quispe,Julieta Luna,Juan Carlos Valencia,Daniela Paton,Victor R Gordeuk","doi":"10.1002/ajh.70025","DOIUrl":"https://doi.org/10.1002/ajh.70025","url":null,"abstract":"In Bolivian Aymara with erythrocytosis, elevated erythropoietin strongly associates with history of thrombosis. Hypoxia and iron deficiency predict elevated erythropoietin, but they do not have a direct relationship with thrombosis history. Source: Artwork by Nadia Gordeuk.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"26 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144737257","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":"Eculizumab is Associated With Increased Infection Rates and Infection Related Mortality in Children With Thrombotic Microangiopathy After HCT","authors":"Michelle L. Schoettler,&nbsp;Robert Lisac,&nbsp;Joel Ofori,&nbsp;Erin Frost,&nbsp;Wayne Liang,&nbsp;Suhag Parikh,&nbsp;Shanmuganathan Chandrakasan,&nbsp;Taylor Fitch,&nbsp;Jeremy Obordo,&nbsp;Kathleen Spencer,&nbsp;Satheesh Chonat,&nbsp;Adrianna Westbrook,&nbsp;Kirsten M. Williams","doi":"10.1002/ajh.70022","DOIUrl":"10.1002/ajh.70022","url":null,"abstract":"<div>\u0000 \u0000 <p>Complement C5 inhibition can be used to treat hematopoietic cell transplant-associated thrombotic microangiopathy (TA-TMA) but may impact infectious organism clearance, impeding opsonization and lysis. The infectious risks of eculizumab exposure after hematopoietic cell transplant (HCT) are unknown. In this single center, retrospective case–control study, we included allogeneic HCT recipients transplanted from January 2019 to January 2023. All patients with TA-TMA treated with eculizumab from day 14–180 post HCT were identified as cases. Controls were matched using an exact matching procedure on maximum grade 3–4 acute GVHD and intensive care unit admission. Cases and controls were matched on index date (first day of eculizumab therapy), and infections from 1 year of index date were captured. Among 31 pairs (62 patients), the rate of bacteremia was 8.49 times higher (95% CI 4.4, 16.4), tissue specific bacterial infections 6.22 times higher (95% CI 2.28, 17.17), and viral infections 3.16 times higher (95% CI 1.90, 5.25) in eculizumab exposed children compared to matched controls. After adjusting for the number of immune suppressive medications, steroid days exposed, and steroid refractory GVHD, all infection rates remained significantly higher among eculizumab exposed patients. In sensitivity models excluding an outlier and altering the study follow up time to 3 and 6 months, rates of infections remained significantly higher in the eculizumab cohort. Infection related mortality was significantly higher in the eculizumab exposed patients than controls; 1-yr mortality was 45% vs 19% respectively. This study suggests infectious complications are increased with eculizumab treatment in the HCT setting, though additional studies are needed to validate these findings.</p>\u0000 </div>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1779-1791"},"PeriodicalIF":9.9,"publicationDate":"2025-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144737255","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":"Distinct Characteristics of Lymphoid and Myeloid Clonal Hematopoiesis in World Trade Center First Responders","authors":"Myvizhi Esai Selvan,&nbsp;Pei-Fen Kuan,&nbsp;Xiaohua Yang,&nbsp;John Mascarenhas,&nbsp;Robert J. Klein,&nbsp;Benjamin J. Luft,&nbsp;Paolo Boffetta,&nbsp;Zeynep H. Gümüş","doi":"10.1002/ajh.70014","DOIUrl":"10.1002/ajh.70014","url":null,"abstract":"<p>CHIP in WTC first responders: Study design.\u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure>\u0000 </p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1873-1877"},"PeriodicalIF":9.9,"publicationDate":"2025-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.70014","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144726538","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes of Post-Transplant Rh-GCSF and Decitabine Maintenance Therapy in Patients With High-Risk Myeloid Neoplasm","authors":"Mathias Palmer,&nbsp;Anmol Baranwal,&nbsp;Rami Basmaci,&nbsp;Khalil Hassan,&nbsp;Jade Kutzke,&nbsp;Gabriel Bartoo,&nbsp;Hemant Murthy,&nbsp;James Foran,&nbsp;Abhishek A. Mangaonkar,&nbsp;Mehrdad Hefazi,&nbsp;Aasiya Matin,&nbsp;William J. Hogan,&nbsp;Mark Litzow,&nbsp;Mithun Vinod Shah,&nbsp;Hassan B. Alkhateeb","doi":"10.1002/ajh.70018","DOIUrl":"10.1002/ajh.70018","url":null,"abstract":"<p>\u0000 \u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure>\u0000 </p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"100 10","pages":"1891-1894"},"PeriodicalIF":9.9,"publicationDate":"2025-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.70018","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144720242","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}