American Journal of Hematology最新文献

{"title":"Tissue-Resident Myeloid and Histiocytic Cells in Health and Disease: Novel Emerging Concepts.","authors":"Peter Valent,Johann Wojta,Petri T Kovanen,Olivier Hermine,Falko Fend,Karl Sotlar,Hildegard Greinix,Klaus Geissler,Karin Hartmann,Juliana Schwaab,Marco Herling,Laura Boccuni,Lukas Kazianka,Max Vincent John,Wolfgang R Sperr,Carina Zierfuss,Alexandar Tzankov,Christian Sillaber,Milen Minkov,Gregor Hoermann,Matthew Collin,Hans-Peter Horny,Torsten Haferlach,Maria Sibilia,Julien Haroche,Paul La Rosée,Alberto Orfao,Michel Arock","doi":"10.1002/ajh.70062","DOIUrl":"https://doi.org/10.1002/ajh.70062","url":null,"abstract":"Although all myeloid cells are considered to derive from hematopoietic stem cells, the cells in each myeloid lineage are heterogeneous populations, and their distribution and functions vary, depending on underlying physiologic and pathologic processes, age, sex, and genetic and epigenetic signatures. In general, myeloid cells can be separated into circulating and tissue-resident cells. Tissue-resident myeloid cells can further be divided into cells derived from circulating monocytes, circulating stem cells, or local tissue-restricted stem or progenitor cells. Depending on underlying diseases and co-morbidities, the phenotype, function, and distribution of these cells may change substantially. In this article, we discuss new developments in the field and related emerging concepts around tissue-resident myeloid cells and their role and function in reactive and clonal disorders. Cell types reviewed in depth in this article include monocytes, macrophages, histiocytes, dendritic cells, and tissue mast cells, with a focus on inflammatory disease processes, vascular pathologies, solid tumors, and hematopoietic malignancies. Moreover, the current article provides an update on patient-related and disease-related diagnostic and prognostic variables, multi-parametric prognostic scoring systems, and therapeutic options and algorithms in these neoplasms. Finally, our article provides an overview on the emerging role and impact of precision medicine approaches, translational research, and artificial intelligence in the diagnosis, prognostication, and management of monocytic, histiocytic, and mast cell disorders.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"72 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145035793","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":"Association Between Molecular Profile and Outcome After Intensive Chemotherapy in Patients > 60 Years With Secondary and/or Adverse Cytogenetics Acute Myeloid Leukemia-A FILO Study.","authors":"Corentin Orvain,Anne Bouvier,Audrey Bidet,Pierre-Yves Dumas,Sarah Bertoli,Lamya Haddaoui,Odile Blanchet,Sylvain Thépot,Aline Tanguy-Schmidt,Damien Luque Paz,Arnaud Pigneux,Eric Delabesse,Christian Recher,Mathilde Hunault","doi":"10.1002/ajh.70076","DOIUrl":"https://doi.org/10.1002/ajh.70076","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"131 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145035807","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":"Similar Outcome With Haploidentical, Matched Sibling, or Matched Unrelated Donor Hematopoietic Cell Transplantation for Adult Patients With Adverse-Risk TP53-Mutated Acute Myeloid Leukemia in First Remission: A Comparative Study From the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.","authors":"Yuhua Ru,Jia Chen,Allain-Thibeault Ferhat,Hélène Labussière-Wallet,Tobias Gedde-Dahl,Nicolaus Kröger,Gérard Socié,Ibrahim Yakoub-Agha,Matthias Eder,Stephan Mielke,Depei Wu,Mohamad Mohty,Fabio Ciceri,Norbert-Claude Gorin","doi":"10.1002/ajh.70069","DOIUrl":"https://doi.org/10.1002/ajh.70069","url":null,"abstract":"Given the dismal prognosis for patients with TP53-mutated acute myeloid leukemia (AML), the optimal donor for those undergoing allogeneic hematopoietic cell transplantation (allo-HCT) remains unclear. We retrospectively analyzed adult patients with TP53-mutated AML who underwent first allo-HCT in CR1 between 2010 and 2021. Outcomes were compared among using a haploidentical donor (Haplo), a matched sibling donor (MSD), and a 10/10 matched unrelated donor (MUD). The analysis comprised 451 patients, including 86 Haplo, 117 MSD, and 248 MUD. Patients in the three groups were transplanted during a similar period. Haplo, MSD, and MUD groups experienced similar incidences of Day 180 Grades II-IV aGVHD (30.9% vs. 23.6% vs. 28.3%), Grades III-IV aGVHD (13.6% vs. 10.1% vs. 9.1%), 2-year cGVHD (28.9.% vs. 30.9% vs. 25.6%) and extensive cGVHD (10.9% vs. 16.1% vs. 13.3%). By multivariate analysis, the outcomes were similar in the three groups. The MSD group was associated with a similar 2-year overall survival (OS: 33.9%; p = 0.799), leukemia-free survival (LFS: 30.5%; p = 0.956), relapse incidence (RI: 54.2%; p = 0.497), non-relapse mortality (NRM: 15.3%; p = 0.368), and GVHD-free, relapse-free survival (GRFS: 21.8%, p = 0.957) when compared with the Haplo group (2-year OS: 46.7%, LFS: 37.4%, RI: 40.8%, NRM: 21.7%, GRFS: 25.7%). The MUD group also experienced a similar 2-year OS (36.9%; p = 0.892), LFS (31%; p = 0.904), RI (50.8%; p = 0.521), NRM (18.2%; p = 0.368) and GRFS (21.9%; p = 0.383) when compared with the Haplo group. In conclusion, outcomes of patients with TP53-mutated AML undergoing allo-HCT from a haploidentical donor were comparable to those from an MSD or 10/10 MUD HCT.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"1 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145025306","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":"CHEK2 Germline Variants in Early-Onset and Familial Myeloproliferative Neoplasms.","authors":"Oscar Borsani,Elisabetta Molteni,Daniela Pietra,Anna Gallì,Virginia Valeria Ferretti,Silvia Catricalà,Ettore Rizzo,Luca Malcovati,Elisa Rumi","doi":"10.1002/ajh.70072","DOIUrl":"https://doi.org/10.1002/ajh.70072","url":null,"abstract":"Of 313 patients with early-onset or familial MPN, 7 (2.2%) patients had pathogenic/likely pathogenic (P/LP) germline heterozygous loss of function mutations in CHEK2. The presence of CHEK2 variants was associated with a familial history of malignancies and a higher risk of leukemic evolution, reinforcing the hypothesis of CHEK2 variants as tumor predisposing risk allele.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"37 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145026025","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 Cytoreduction on Blinatumomab Outcomes for Relapsed or Refractory B-ALL With High Disease Burden.","authors":"Jose Tinajero,Kim Nguyen,Dat Ngo,Vaibhav Agrawal,Haoyue Shan,Jianying Zhang,Paul Koller,Hoda Pourhassan,Lindsey Murphy,Yazeed Samara,Shukaib Arslan,Stephen Forman,Anthony Stein,Guido Marcucci,Vinod Pullarkat,Ibrahim Aldoss","doi":"10.1002/ajh.70057","DOIUrl":"https://doi.org/10.1002/ajh.70057","url":null,"abstract":"Blinatumomab is approved for the treatment of relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL). Studies have correlated pre-blinatumomab high disease burden (HDB) [> 50% bone marrow blasts (BMB)] with lower response rates and increased risk for toxicities, including cytokine release syndrome (CRS) and neurotoxicity (NT). While the administration of pre-blinatumomab cytoreductive therapy is an appealing approach, larger studies validating the beneficial effect of this strategy in patients with HDB are lacking. We retrospectively analyzed 148 adult patients with R/R B-ALL treated with blinatumomab. Patients were grouped as low disease burden (LDB, n = 55), HDB without cytoreduction (n = 41), and HDB with cytoreduction (n = 52). The median age for the cohort was 40 years; the majority were males (63%) and Hispanic (73%), and the most common ALL subtype was Ph-like (40%). Patients with HDB with cytoreduction had a significantly higher rate of prior alloHCT (p = 0.041) and more lines of prior therapy (p = 0.006) compared to the other cohorts. Compared to patients with HDB, those with LDB had significantly higher response rates to blinatumomab (p < 0.0001), while rates of CRS and NT were not significantly different. Among patients with HDB, cytoreductive therapy was associated with a significantly lower rate of CRS compared to those who did not receive cytoreduction (p = 0.038). However, cytoreduction had no significant impact on treatment response, MRD negativity, or NT. Patients with B-ALL and HDB remain a challenging population for blinatumomab therapy; while cytoreduction may improve safety by reducing CRS, novel strategies are needed to enhance treatment efficacy.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"164 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145025311","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":"European Myeloma Network Consensus Statement on Functional High-Risk Multiple Myeloma.","authors":"Sueh-Li Lim,Monika Engelhardt,Evangelos Terpos,Francesca Gay,Niels W C J Van de Donk,Herman Einsele,Pieter Sonneveld,Martin Kaiser,Mario Boccadoro,Andrew Spencer","doi":"10.1002/ajh.70070","DOIUrl":"https://doi.org/10.1002/ajh.70070","url":null,"abstract":"Multiple myeloma (MM) is an incurable blood cancer characterized by clonal bone marrow plasmacytosis, hypercalcemia, renal failure, anemia, and osteolytic bone disease. Approximately 20% of NDMM patients, not predicted to have high-risk disease at diagnosis, progress early, despite optimal induction +/- ASCT and lenalidomide maintenance, and are subsequently categorized as functional high-risk (FHR) disease. Standardized risk-stratification models incorporate biomarkers of tumor burden, existence of high-risk cytogenetics, with the presence/absence of plasma cell leukemia/extramedullary disease to attribute high-risk at diagnosis; however, depth/duration of response to novel agent-based induction (NA-IND) as dynamic markers of disease risk have not been defined. However, irrespective of diagnostic risk-stratification, response to NA-IND may be the single most effective method of identifying patients whose FHR biology portends an unacceptably short overall survival (OS). In this EMN consensus statement, we define FHR-MM as disease progression within 18 months of commencement of first-line therapy in the absence of high-risk cytogenetics, discuss the underlying disease biology, and strategies to improve outcomes for these patients.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"32 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145026020","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":"Spirometry Interpretation in Children With Sickle Cell Disease: GLI Global Versus Race-Specific Normative Equations.","authors":"Jacob McCoy,David Wilson,Melanie Kirby-Allen,Isaac Odame,Hartmut Grasemann","doi":"10.1002/ajh.70071","DOIUrl":"https://doi.org/10.1002/ajh.70071","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"53 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145025309","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":"Splenic Iron Overload Influence on Lumbar Spine BMD Reproducibility in β-Thalassemia.","authors":"Joseph Klim,Ahmad Machmouchi,Farouk Kabbara,Sacha El Khoury,Nicole Charbel,Maya Rahme,Maya Charafeddine,Ali Taher,Ghada El-Hajj Fuleihan","doi":"10.1002/ajh.70067","DOIUrl":"https://doi.org/10.1002/ajh.70067","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"28 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145018279","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":"Endothelial Activation and Stress Index Serves as a Predictor for Survival in Lymphoma-Associated Hemophagocytic Lymphohistiocytosis: A Retrospective Multicenter Cohort Study of Jiangsu Cooperative Lymphoma Group.","authors":"Yanping Liu,Yimin Ren,Ling Gao,Shanza Ahmed,Xuzhang Lu,Bingzong Li,Chunling Wang,Liang Yu,Miao Sun,Yun Zhuang,Yuqing Miao,Haiwen Ni,Xiaoyan Xie,Xiaofeng Shi,Jingyan Xu,Yunping Zhang,Min Zhao,Min Xu,Wanchuan Zhuang,Weiying Gu,Guoqiang Lin,Haiying Hua,Jianfeng Zhu,Maozhong Xu,Tao Jia,Ping Liu,Lijia Zhai,Tongtong Zhang,Huirong Shan,Qiudan Shen,Lei Fan,Jianyong Li,Wenyu Shi,Yi Miao","doi":"10.1002/ajh.70063","DOIUrl":"https://doi.org/10.1002/ajh.70063","url":null,"abstract":"Lymphoma-associated hemophagocytic lymphohistiocytosis (LA-HLH) is a life-threatening hyperinflammatory syndrome, and hierarchical management based on a prognostic model is important. The endothelial activation and stress index (EASIX) score has demonstrated prognostic utility in recipients of allogeneic stem cell transplantation and chimeric antigen receptor (CAR) T-cell therapy. However, its role in LA-HLH remains unestablished. We conducted a multicenter retrospective analysis of patients with LA-HLH from 28 medical centers to explore the prognostic impacts of EASIX in LA-HLH. EASIX was calculated using baseline lactate dehydrogenase, serum creatinine, and platelet counts. A total of 490 patients with LA-HLH were included and stratified by EASIX quartiles (Q1-Q4). Patients with a higher EASIX score had significantly inferior 2-month survival and overall survival, according to the Kaplan-Meier analysis (log-rank p < 0.001). In multivariable analyses, after adjustment for age, gender, lymphoma type, splenomegaly, bone marrow infiltration, lymphoma status (treatment-naïve versus relapsed/refractory), hemoglobin, absolute neutrophil count, serum ferritin levels, and aspartate aminotransferase, the highest EASIX quartile (Q4) exhibited a 7.01-fold risk of death compared to the lowest quartile (Q1) (Hazard ratio [HR] = 7.01, 95% confidence interval [CI]: 3.98-12.36; p < 0.001). Additionally, the restricted cubic splines (RCS) analysis illustrated an increase in the risk of mortality with an increasing EASIX score. Our findings support EASIX being a robust, universally accessible prognostic marker for LA-HLH, strongly associated with early mortality risk. This index can be used to stratify the risk levels of patients with LA-HLH and predict their survival outcomes.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"16 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145008785","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":"Comorbidities in Mild WAS/XLT Require Lifelong Follow-Up and Consideration of Definitive Treatment.","authors":"Coralie Mallebranche,Charline Miot,Mickael Alligon,Despina Moshous,Benedicte Neven,Vincent Barlogis,Virginie Courteille,Bénédicte Bruno,Marie Ouachée-Chardin,Dalila Adjaoud,Yves Delneste,Olivier Bouaziz,Capucine Picard,Alain Fischer,Nizar Mahlaoui,Isabelle Pellier, ","doi":"10.1002/ajh.70061","DOIUrl":"https://doi.org/10.1002/ajh.70061","url":null,"abstract":"Loss of function mutations in the gene encoding WASP (Wiskott-Aldrich syndrome protein) result in Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia-XLT (WAS/XLT). The clinical severity of the disease can be assessed using the WAS clinical severity score. Typically, patients with a score of 3 or less at 2 years of age are considered to have mild WAS/XLT disease. However, patients with a low score in the first 2 years of life can still experience life-threatening complications, and there are no agreed guidelines for the management of these patients with mild WAS/XLT. We analyzed data on WAS/XLT patients from the French National Reference Centre for Primary Immunodeficiencies registry. At 10 December 2021, data were available for 261 patients, 170 of whom had mild WAS/XLT. The median age of these patients at last follow-up was 15.8 years (range 2.0-60.4). Overall survival at 40 years was 73% in mild WAS patients versus 65% in severe WAS patients (p = 0.43). In the mild WAS population, prior to Hematopoietic Stem Cell Transplantation or gene therapy, 38.2% of patients progressed to a WAS severity score of 4-5 after the age of 2. Remarkably, no deaths were reported in 45 HSCTs performed since 2010, regardless of initial severity, type and HLA compatibility of the transplant, and age at diagnosis, with a median follow-up of 3.2 years (0-11.5) after transplant. As complications can occur throughout life, our study supports definitive treatment for all patients when available, including those with mild forms.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"25 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145008786","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}