Expert Review of Hematology最新文献

{"title":"The prognostic power of MMP-9 in diffuse large B-cell lymphoma.","authors":"Aspasia Koudouna, Vasiliki Bartzi, Alexandros Gkiokas, Mavra Papadatou-Gigante, Annita-Ioanna Gkioka, George Oikonomou, Theodoros P Vassilakopoulos, Marie-Christine Kyrtsonis","doi":"10.1080/17474086.2025.2541002","DOIUrl":"https://doi.org/10.1080/17474086.2025.2541002","url":null,"abstract":"","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":""},"PeriodicalIF":2.1,"publicationDate":"2025-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144741741","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emergency care for children with sickle cell disease: a focus on pain and fever.","authors":"Rawan Korman, Dunia Hatabah, Claudia R Morris","doi":"10.1080/17474086.2025.2538537","DOIUrl":"10.1080/17474086.2025.2538537","url":null,"abstract":"<p><strong>Introduction: </strong>Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 100,000 individuals in the United States and millions worldwide, characterized by acute vaso-occlusive pain episodes (VOEs) and other complications that frequently necessitate emergency department (ED) visits.</p><p><strong>Areas covered: </strong>Despite therapeutic advancements, ED care remains a major concern, often cited by patients as the area of healthcare most in need of improvement. National guidelines have been established to ensure ideal emergency SCD care and management, however, these guidelines do not address barriers or facilitators that affect implementation in the complex ED setting. This review examines current diagnostic and management approaches for common SCD complications requiring ED utilization, particularly fever and pain in pediatric patients. It highlights the challenges children with SCD face in emergency care and the existing knowledge gaps. Despite guidelines recommending timely, individualized pain treatment, implementation remains inconsistent, resulting in prolonged suffering and increased hospitalizations.</p><p><strong>Expert opinion: </strong>Future research should focus on enhancing guideline adherence, reducing disparities, and developing targeted therapies. Novel biomarkers could improve early diagnosis, while standardized severity scoring systems may optimize triage and treatment decisions. Advancing biomarker research and investigational therapies beyond traditional supportive care holds promise for improving SCD management and patient outcomes.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-23"},"PeriodicalIF":2.1,"publicationDate":"2025-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144706889","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An observational study of disease management in adult patients with polycythemia vera: results from a large U.S. claims database.","authors":"Andrew Kuykendall, Lucy Bellamy, Lopa Desai, Omari Morrison, Larisa Gofman, Arturo Molina","doi":"10.1080/17474086.2025.2538542","DOIUrl":"10.1080/17474086.2025.2538542","url":null,"abstract":"<p><strong>Background: </strong>Polycythemia vera (PV) is characterized by erythrocytosis and an increased risk of thrombotic events (TEs). Currently, standard-of-care therapies for PV have limitations, which indicate the need to understand real-world treatment patterns and treatment burden in PV.</p><p><strong>Research design and methods: </strong>This retrospective observational study analyzed real-world claims data in adult patients with PV using the Komodo Health claims database (2016-2022) in the United States. Burdensome treatment was classified as patients receiving ≥ 3 phlebotomies (PHLs) within a 6-month period and/or high-dose hydroxyurea (HU) ≥ 1,000 mg per day.</p><p><strong>Results: </strong>Of 44,766 treated patients (mean age: 65 years; 64% male), 55% received burdensome treatment, which included frequent PHL (33%), high-dose HU (17%), or a combination of both (frequent PHL + high-dose HU, 5%). PHL and HU were the most common first-line treatments (PHL, 71%; HU, 27%), and 87% of patients initiating treatment with PHL monotherapy never advanced to another therapy regimen. TEs occurred in 16% of the treated patients.</p><p><strong>Conclusions: </strong>These data suggest a substantial proportion of patients with PV receive burdensome treatments, with 55% of treated patients receiving frequent PHL and/or high-dose HU, highlighting need for therapy optimization. However, inherent limitations of using claims data should be taken into consideration.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-8"},"PeriodicalIF":2.3,"publicationDate":"2025-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144689735","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mesenchymal stromal cells for the prophylaxis of graft-versus-host disease after hematopoietic stem cell transplantation: a meta-analysis of randomized controlled trials.","authors":"Sushmitha Rameshbabu, Hariniska Jayaraman Kannan, Mudit Moondra, Seema Nabil Nimer, Roshan Afshan, Sean Ghose, Muhammad Faisal Aslam, Raef Nizar Ali, Anuj Timshina, Shiny Teja Kolli, Muhammad Ayyan","doi":"10.1080/17474086.2025.2535422","DOIUrl":"10.1080/17474086.2025.2535422","url":null,"abstract":"<p><strong>Background: </strong>Mesenchymal stromal cells (MSCs) have emerged as a potential alternative therapeutic strategy for the prophylaxis of graft-versus-host disease (GVHD) in patients undergoing hematopoietic stem cell transplantation (HSCT).</p><p><strong>Research design and methods: </strong>This meta-analysis included eight randomized controlled trials (RCTs) involving 570 patients. The primary outcomes assessed were overall survival (OS), the development of acute GVHD(aGVHD), and chronic GVHD (cGVHD). The statistical analysis was performed using Review Manager (RevMan 5.4) with a random-effects model.</p><p><strong>Results: </strong>The meta-analysis showed a significant improvement in overall survival in the MSC group compared to the control group (RR 1.12; 95% CI: 1.02-1.23), with no evidence of heterogeneity (I² = 0%). MSC prophylaxis was associated with a significant reduction in the incidence of aGVHD (RR 0.67; 95% CI: 0.40-0.83) and cGVHD (RR 0.65; 95% CI: 0.49-0.87). However, no significant difference was found between the MSC and control groups regarding primary disease relapse (RR 1.00; 95% CI:0.73-1.38) or the incidence of infections (RR 0.80; 95% CI:0.57-1.11). In terms of patients with at least one adverse event, no statistically significant difference was observed between the two groups (RR 1.10; 95% CI: 0.74-1.63).</p><p><strong>Conclusions: </strong>MSC prophylaxis significantly improves overall survival and reduces the incidence of both aGVHD and cGVHD in HSCT patients, without increasing the risk of relapse, infections, or adverse events, indicating its potential as a safe and effective intervention for GVHD management. Further large-scale, multicenter RCTs are needed to validate or refute the current findings.</p><p><strong>Registration: </strong>This review has been registered with theInternational Prospective Register of Systematic Reviews (PROSPERO)(CRD42024569358).</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-8"},"PeriodicalIF":2.3,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144689737","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hematopoietic cell transplantation for leukocyte adhesion deficiency: prevention of graft-versus-host-disease.","authors":"Motoi Yamashita, Takahiro Kamiya, Hirokazu Kanegane","doi":"10.1080/17474086.2025.2538543","DOIUrl":"https://doi.org/10.1080/17474086.2025.2538543","url":null,"abstract":"<p><strong>Introduction: </strong>Leukocyte adhesion deficiency (LAD) is a rare genetic disorder that impairs leukocyte migration, leading to severe immune dysfunction and recurrent infections. Although allogeneic hematopoietic cell transplantation (HCT) remains the primary curative treatment for severe LAD, it is complicated by a high incidence of graft-versus-host-disease (GVHD).</p><p><strong>Areas covered: </strong>This narrative review outlines the key factors influencing GVHD development in patients with LAD-I, the most common LAD subtype, undergoing HCT. It explores established and emerging strategies for preventing GVHD, focusing on their effectiveness and outcomes. The literature search was conducted using PubMed to identify studies reporting HCT for LAD published 1989-2025.</p><p><strong>Expert opinion: </strong>Conventional GVHD prophylaxis regimens, primarily involving calcineurin inhibitors, have proven insufficient in preventing GVHD in high-risk populations. Among patients undergoing haploidentical HCT, post-transplantation cyclophosphamide has shown efficacy in preventing GVHD, although these results were based on limited cases. Graft manipulation techniques such as CD34⁺ selection have also been explored. However, these approaches are often associated with high graft failure rate and poor survival. Alemtuzumab, which is used in conditioning regimens, has shown promise in lowering GVHD incidence. Further studies are essential to optimize GVHD prophylaxis and improve survival outcomes in patients with LAD undergoing HCT.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-9"},"PeriodicalIF":2.3,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144689736","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Review of laboratory methods used for analysis of von Willebrand factor and for diagnosis of related diseases.","authors":"Jürgen Patzke, Nikolaus B Binder, Mariona Bono, François Depasse, Matthias Germer, Michael Heins, Martina Leplatoni, Stephan Schwers, Michael Spannagl, Peter L Turecek","doi":"10.1080/17474086.2025.2525461","DOIUrl":"https://doi.org/10.1080/17474086.2025.2525461","url":null,"abstract":"<p><strong>Introduction: </strong>Von Willebrand factor (VWF) is a large multimeric protein present in the blood. Its most important and best characterized function is to control bleeding in primary hemostasis, which is triggered by different biophysical mechanisms and protein-receptor interactions involving different domains of VWF. Many different diseases related to VWF, most importantly von Willebrand disease comprising different types and sub-types, require diagnosis, laboratory analysis of concentration, and function of VWF.</p><p><strong>Areas covered: </strong>As several different specific functions of VWF are physiologically relevant, several different assays are needed. The aim of this article is to provide a comprehensive overview of all relevant assays together with a description of how each assay is technically designed. Furthermore, guidance is given for choosing the right and validated assay methods.</p><p><strong>Expert opinion: </strong>This information includes technical requirements, analytical performance data, references to relevant guidelines and other guidance documents, and reference standards. Also, information on availability and the type of the assay is provided, such as automated or manual, in vitro diagnostics or research use only. Relation to the clinical use of these assays, as well as performance, and result interpretation is covered in a second article in the same issue of this journal by the same authors.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-15"},"PeriodicalIF":2.3,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144559617","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world treatment patterns and outcomes in people with von Willebrand disease treated prophylactically with recombinant von Willebrand factor in the United States.","authors":"Jonathan C Roberts, Maissaa Janbain, Jessica R Marden, Sanjana Sundaresan, Elyse Swallow, Natalia Nieto, Bethany Jones, Jorge Caicedo","doi":"10.1080/17474086.2025.2504956","DOIUrl":"10.1080/17474086.2025.2504956","url":null,"abstract":"<p><strong>Background: </strong>People with von Willebrand disease (VWD) experience increased bleeding and decreased quality of life; those with a severe bleeding phenotype may benefit from prophylactic treatment. This retrospective chart review evaluated real-world effectiveness of prophylaxis with recombinant von Willebrand factor (rVWF) in all subtypes of VWD.</p><p><strong>Research design and methods: </strong>People aged ≥12 years with a confirmed VWD diagnosis from US health care centers who received either routine or intermittent (for menorrhagia) prophylactic rVWF treatment were included. Eligibility criteria included availability of medical records ≥ 6 months pre- (baseline period) and post-rVWF initiation (rVWF treatment period). Annualized bleed rate (ABR), healthcare resource utilization (HCRU), and treatment patterns were the main outcomes of interest and were compared between both periods.</p><p><strong>Results: </strong>Of 30 participants across 11 sites, 23 (76.7%) received routine rVWF prophylaxis for a mean duration of 2.9 years. Treatment is ongoing in most participants. ABR and total and bleed-related inpatient visits and number of surgeries decreased during the rVWF treatment period versus the baseline period.</p><p><strong>Conclusions: </strong>Participants receiving routine rVWF prophylaxis in this study experienced reduced ABR and HCRU versus the baseline period, indicating that rVWF prophylaxis may result in improved outcomes in people with VWD across all subtypes.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"547-560"},"PeriodicalIF":2.1,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144076900","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment patterns and blood count control in 10,112 patients with polycythemia vera.","authors":"Naveen Pemmaraju, Jingbo Yu, Anupama Vasudevan, Husain Qureshi, Evan Braunstein, Aleksander Chojecki","doi":"10.1080/17474086.2025.2520316","DOIUrl":"10.1080/17474086.2025.2520316","url":null,"abstract":"<p><strong>Background: </strong>Elevated blood counts in polycythemia vera (PV) are associated with increased thrombotic risk, which contributes to morbidity and mortality.</p><p><strong>Research design and methods: </strong>This retrospective study describes treatment patterns and blood count control in patients with PV managed at community oncology practices (January 2014-February 2023; Integra Precision Q database).</p><p><strong>Results: </strong>Of a total 10,112 patients, most received phlebotomy (68.1%) or hydroxyurea (HU; 28.2%) as initial treatment, with median follow-up of 32.1 (IQR, 13.5-58.5) months and 31.5 (IQR, 16.8-54.9) months, respectively. Changing treatment was less common than remaining on initial treatment despite 67.8% of patients on phlebotomy and 30.4% on HU having elevated hematocrit (≥45%) after 1 year of treatment. In contrast, 85.4% of patients who switched to ruxolitinib from HU achieved hematocrit < 45% after 1 year, and fewer required phlebotomy during ruxolitinib treatment than with HU treatment (RUX, 29.3%; HU, 53.5%). Additionally, 54.2% of patients who switched to ruxolitinib achieved white blood cell counts < 11 × 10⁹/L, and 57.5% achieved platelet counts ≤ 400 × 10⁹/L after 1 year of ruxolitinib treatment.</p><p><strong>Conclusions: </strong>This real-world evidence highlights the importance of considering alternative therapies for patients whose initial treatment regimen does not provide adequate clinical benefit.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"529-536"},"PeriodicalIF":2.3,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144283176","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world analysis of extended half-life product posology in hemophilia A: results from a retrospective analysis of medical chart and claims data.","authors":"Cedric Hermans, John Waller, Steven R Lentz","doi":"10.1080/17474086.2025.2520312","DOIUrl":"10.1080/17474086.2025.2520312","url":null,"abstract":"<p><strong>Background: </strong>Extended half-life (EHL) products have changed the treatment landscape of patients with hemophilia as patients maintain protective FVIII levels with minimal occurrence of spontaneous bleeding.</p><p><strong>Research design and methods: </strong>Two independent datasets from the multi-country patient record form (PRF)-based study and PicnicHealth/Komodo Health study were analyzed to understand the real-world use of EHL products for patients with hemophilia A (HA).</p><p><strong>Results: </strong>Most patients receiving EHL prophylaxis were on per-label dosing. Those receiving individualized dosing had moderate to severe disease. EHL dose adjustment occurred in 20% of patients of which 14.3% experienced a second dose adjustment. Treatment efficacy was mainly monitored via annualized bleeding rate (ABR) with quality of life, tolerability, and treatment efficacy being the primary considerations for Healthcare Personnel when selecting treatment. Switching to a different EHL or standard half-life (SHL) product did not significantly reduce median ABR while switching to non-factor therapy significantly reduced median ABR from 5.2 to 0.94, <i>p</i> < 0.0001. Patients on individualized dosing had higher ABRs than those on per-label dosing both before index EHL treatment and while on EHL treatment, whereas individualization via dose adjustment was associated with significant median ABR reduction (<i>p</i> < 0.0001).</p><p><strong>Conclusions: </strong>Individualization may support improved outcomes in patients unable to achieve satisfactory outcomes on a per-label dosing regimen.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"537-545"},"PeriodicalIF":2.3,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144316300","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SYK identified by bioinformatics analysis promotes the proliferation of multiple myeloma.","authors":"Ju Deng, Peichun Li, Shuo Li, Fengting Liang, Minglin Hong, Ting Zhang, Yanhong Tan, Fanggang Ren, Yaofang Zhang, Zhifang Xu, Hongwei Wang","doi":"10.1080/17474086.2025.2505724","DOIUrl":"10.1080/17474086.2025.2505724","url":null,"abstract":"<p><strong>Background: </strong>Despite recent advancements, the pathogenesis of multiple myeloma (MM) remains incompletely elucidated, with relapse and therapy resistance persisting as major clinical challenges, underscoring the imperative to identify novel therapeutic targets.</p><p><strong>Research design and methods: </strong>Differentially expressed genes were initially screened from the GSE6477 and GSE6691 datasets. Subsequent functional annotation and pathway enrichment analyses were conducted utilizing the DAVID bioinformatics platform. A protein-protein interaction network was constructed via the STRING database, followed by module analysis and hub genes identification through CytoHubba plugin. The biological significance of candidate genes was ultimately validated through ex vivo cellular functional assays and in vivo xenograft tumorigenesis experiments in murine models.</p><p><strong>Results: </strong>Bioinformatics analysis identified spleen tyrosine kinase (SYK) as the most prognostically significant candidate gene (<i>p</i> = 0.027). The SYK-specific inhibitor BAY61-3606 demonstrated time- (<i>p</i> < 0.05) and dose- (<i>p</i> < 0.01) dependent inhibition of MM cell viability, concomitant induction of G2/M phase cell cycle arrest (<i>p</i> < 0.001), and significant promotion of apoptosis (<i>p</i> < 0.05). In vivo experiments utilizing MM xenograft models demonstrated that BAY61-3606 administration significantly attenuated tumor growth kinetics (<i>p</i> < 0.05).</p><p><strong>Conclusions: </strong>Our findings establish SYK as a therapeutic target in MM, thereby facilitating the development of innovative treatment strategies.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"569-583"},"PeriodicalIF":2.3,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093287","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}