Expert Review of Hematology最新文献_第2页

Real-world treatment patterns and outcomes in people with von Willebrand disease treated prophylactically with recombinant von Willebrand factor in the United States. 在美国用重组血管性血友病因子预防性治疗血管性血友病患者的现实世界治疗模式和结果

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-05-20 DOI: 10.1080/17474086.2025.2504956

Jonathan C Roberts, Maissaa Janbain, Jessica R Marden, Sanjana Sundaresan, Elyse Swallow, Natalia Nieto, Bethany Jones, Jorge Caicedo

{"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":"Background: 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.Research design and methods: 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.Results: 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.Conclusions: 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.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"547-560"},"PeriodicalIF":2.3,"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}

引用次数: 0

Treatment patterns and blood count control in 10,112 patients with polycythemia vera. 真性红细胞增多症10112例的治疗模式和血细胞计数控制。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-06-26 DOI: 10.1080/17474086.2025.2520316

Naveen Pemmaraju, Jingbo Yu, Anupama Vasudevan, Husain Qureshi, Evan Braunstein, Aleksander Chojecki

{"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":"Background: Elevated blood counts in polycythemia vera (PV) are associated with increased thrombotic risk, which contributes to morbidity and mortality.Research design and methods: 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).Results: 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.Conclusions: This real-world evidence highlights the importance of considering alternative therapies for patients whose initial treatment regimen does not provide adequate clinical benefit.","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}

引用次数: 0

SYK identified by bioinformatics analysis promotes the proliferation of multiple myeloma. 生物信息学分析发现SYK促进多发性骨髓瘤的增殖。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-05-20 DOI: 10.1080/17474086.2025.2505724

Ju Deng, Peichun Li, Shuo Li, Fengting Liang, Minglin Hong, Ting Zhang, Yanhong Tan, Fanggang Ren, Yaofang Zhang, Zhifang Xu, Hongwei Wang

{"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":"Background: 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.Research design and methods: 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.Results: Bioinformatics analysis identified spleen tyrosine kinase (SYK) as the most prognostically significant candidate gene (p = 0.027). The SYK-specific inhibitor BAY61-3606 demonstrated time- (p < 0.05) and dose- (p < 0.01) dependent inhibition of MM cell viability, concomitant induction of G2/M phase cell cycle arrest (p < 0.001), and significant promotion of apoptosis (p < 0.05). In vivo experiments utilizing MM xenograft models demonstrated that BAY61-3606 administration significantly attenuated tumor growth kinetics (p < 0.05).Conclusions: Our findings establish SYK as a therapeutic target in MM, thereby facilitating the development of innovative treatment strategies.","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}

引用次数: 0

Real-world analysis of extended half-life product posology in hemophilia A: results from a retrospective analysis of medical chart and claims data. 血友病a中延长半衰期产品病理学的实际分析：来自医疗图表和索赔数据的回顾性分析结果。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-06-24 DOI: 10.1080/17474086.2025.2520312

Cedric Hermans, John Waller, Steven R Lentz

{"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":"Background: 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.Research design and methods: 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).Results: 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, p < 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 (p < 0.0001).Conclusions: Individualization may support improved outcomes in patients unable to achieve satisfactory outcomes on a per-label dosing regimen.","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}

引用次数: 0

In the `prophylaxis era´ people with hemophilia still need major orthopedic surgery. 在“预防时代”，血友病患者仍然需要进行大型骨科手术。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-05-18 DOI: 10.1080/17474086.2025.2508506

Emerito Carlos Rodriguez-Merchan

{"title":"In the `prophylaxis era´ people with hemophilia still need major orthopedic surgery.","authors":"Emerito Carlos Rodriguez-Merchan","doi":"10.1080/17474086.2025.2508506","DOIUrl":"10.1080/17474086.2025.2508506","url":null,"abstract":"Introduction: A non-negligible percentage of people with hemophilia (PWH) eventually develop hemophilic arthropathy in what we might call the 'prophylaxis era'.Areas covered: Due to hemophilic arthropathy, some of the patients suffering from it will eventually require major orthopedic surgery when conservative treatment has failed. The purpose of this article has been to analyze the articles published in PubMed during the period 2020-2025, in order to detect what type of major orthopedic surgery interventions are performed in PWH during the 'prophylaxis era'. On 22 April 202522 April 2025, using 'hemophilia orthopedic surgery' from 1 January 2020 to 22 April 2025 as keywords, 373 articles were found in PubMed. Of these, 50 were analyzed because they were directly related to the title of this article (inclusion criterion). The remaining 323 were excluded because they were not directly related to the title of this article.Expert opinion: Although great progress has been made in prophylaxis therapy in PWH, some patients still suffer from severe painful and disabling hemophilic arthropathy requiring major orthopedic surgery. This suggests that there is still much room for improvement in prophylaxis therapy in PWH. Multidisciplinary teams treating PWH should redouble their efforts to eliminate hemophilic arthropathy.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"501-507"},"PeriodicalIF":2.3,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144076792","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}

引用次数: 0

Proactive measures to reduce maternal mortality and post-partum hemorrhage in sub-Saharan Africa: the hematology perspective. 减少撒哈拉以南非洲孕产妇死亡率和产后出血的积极措施：血液学观点。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 DOI: 10.1080/17474086.2025.2527348

Bilgimol Chumappumkal Joseph, Esther Chioma Emenalom, Patrícia Silva, Annette von Drygalski

引用次数: 0

Anti-tissue factor pathway inhibitors for hemophilia: are these treatments the answer to overcoming current treatment limitations? 血友病抗组织因子途径抑制剂：这些治疗方法是克服当前治疗局限性的答案吗？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-06-14 DOI: 10.1080/17474086.2025.2519002

Johnny Mahlangu

{"title":"Anti-tissue factor pathway inhibitors for hemophilia: are these treatments the answer to overcoming current treatment limitations?","authors":"Johnny Mahlangu","doi":"10.1080/17474086.2025.2519002","DOIUrl":"10.1080/17474086.2025.2519002","url":null,"abstract":"Introduction: Non-factor therapies were developed to address the shortcomings of clotting factor concentrates (CFCs) used for hemophilia bleed management. These CFC unmet needs include high treatment burden, immunogenicity, inconsistent hemostatic cover, poor treatment outcomes, and musculoskeletal progression despite adequate prophylactic treatment. Anti-tissue factor pathway inhibitors (anti-TFPIs) that have completed Phase 3 clinical studies are concizumab and marstacimab. The role of these anti-TFPIs in the hemophilia treatment armamentarium remains unclear.Areas covered: This review critically appraises data published in PubMed, World of Science, and peer-reviewed congress presentations to determine whether anti-TFPIs merely supplement current treatment options or represent a disruptive shift in the treatment paradigm for hemophilia. It underscores the unmet needs of replacement therapies and compares the pharmacokinetic, efficacy, and safety data of anti-TFPIs and selected FVIII and FIX products.Expert opinion: As hemophilia treatment goals continue to evolve, the role of currently developed anti-TFPIs is still not fully defined. This review comprehensively summarizes the clinical trial data, which shows that anti-TFPIs are not intended to replace the standard of care CFCs but to expand the therapeutic arsenal for patients with hemophilia treated with these therapeutic agents.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"521-527"},"PeriodicalIF":2.3,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144293624","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}

引用次数: 0

Clonal hematopoiesis meets an autoinflammatory disease: the new paradigm of VEXAS syndrome. 克隆造血遇到自身炎症性疾病：VEXAS综合征的新范式。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-05-30 DOI: 10.1080/17474086.2025.2508505

Martina Fiumara, Raffaella Molteni, Gianluca Scorpio, Alessandro Tomelleri, Gregorio Maria Bergonzi, Samuele Ferrari, Marco Matucci-Cerinic, Simone Cenci, Lorenzo Dagna, Fabio Ciceri, Elisa Diral, Corrado Campochiaro

{"title":"Clonal hematopoiesis meets an autoinflammatory disease: the new paradigm of VEXAS syndrome.","authors":"Martina Fiumara, Raffaella Molteni, Gianluca Scorpio, Alessandro Tomelleri, Gregorio Maria Bergonzi, Samuele Ferrari, Marco Matucci-Cerinic, Simone Cenci, Lorenzo Dagna, Fabio Ciceri, Elisa Diral, Corrado Campochiaro","doi":"10.1080/17474086.2025.2508505","DOIUrl":"10.1080/17474086.2025.2508505","url":null,"abstract":"Introduction: VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is an acquired autoinflammatory disorder caused by somatic mutations in the UBA1 gene. Predominantly affecting males over 50, the disease presents with systemic inflammation, hematologic abnormalities, and features of clonal hematopoiesis, with nearly half of patients developing myelodysplastic syndromes (MDS). The interaction between inflammation and clonal expansion defines disease progression, emphasizing the need for a comprehensive understanding of its pathogenesis and management.Areas covered: This review discusses the clinical spectrum, genetic landscape, and pathogenic mechanisms of VEXAS syndrome. The correlation between UBA1 mutations and disease severity is explored, alongside the role of clonal hematopoiesis and inflammatory pathways. Current treatments, including corticosteroids, immunosuppressants, JAK inhibitors, and azacitidine, are evaluated for efficacy and limitations. The potential of allogeneic hematopoietic stem cell transplantation (allo-HSCT) as a curative approach is also addressed. Literature search was conducted from January 2020 to present using PubMed and Scopus databases to identify relevant studies.Expert opinion: VEXAS syndrome reflects a complex interaction between autoinflammation and clonal hematopoiesis. While targeted therapies offer symptomatic control, responses remain variable. Future strategies should focus on genotype-driven, personalized treatments and optimizing allo-HSCT protocols to improve patient outcomes and offer disease-modifying potential.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"509-519"},"PeriodicalIF":2.3,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110418","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}

引用次数: 0

Benefits of primary prophylaxis with letermovir in patients after allogeneic hematopoietic stem cell transplantation for hematologic malignancies. 恶性血液病患者异体造血干细胞移植后初级预防使用莱替莫的益处。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-07-01 Epub Date: 2025-05-23 DOI: 10.1080/17474086.2025.2509876

Martyna Włodarczyk, Agata Wieczorkiewicz-Kabut, Anna Armatys, Anna Koclęga, Anna Kopińska, Izabela Noster, Krzysztof Woźniczka, Patrycja Zielińska, Grzegorz Helbig

{"title":"Benefits of primary prophylaxis with letermovir in patients after allogeneic hematopoietic stem cell transplantation for hematologic malignancies.","authors":"Martyna Włodarczyk, Agata Wieczorkiewicz-Kabut, Anna Armatys, Anna Koclęga, Anna Kopińska, Izabela Noster, Krzysztof Woźniczka, Patrycja Zielińska, Grzegorz Helbig","doi":"10.1080/17474086.2025.2509876","DOIUrl":"10.1080/17474086.2025.2509876","url":null,"abstract":"Background: Introduction of letermovir (LMV) as prophylaxis for cytomegalovirus (CMV) infection has decreased the number of clinically significant CMV infections (cs-CMVi) in allogeneic hematopoietic stem cell transplantation (HSCT) recipients. However, long-term, real-world data on LMV's impact on post-transplant outcome remain scarce.Research design and methods: The aim of our study was to evaluate clinical outcome of 93 CMV-seropositive patients who received LMV prophylaxis and to compare them to 168 LMV-free recipients.Results: CMV reactivation was less frequently observed in LMV group if compared to LMV-free control. Twelve patients (17%) and 71 (42%) reactivated CMV in LMV-treated and LMV-free patients, respectively. The cumulative incidence of cs-CMVi was lower in LMV group compared to control [37% vs. 63%]. The incidence of severe acute graft-versus-host disease (aGVHD) was also lower in LMV-treated patients (6% vs. 21%). Overall survival, non-relapse mortality and progression-free survival at 24 months were comparable. No risk factors for post-transplant CMV reactivation were identified in LMV group, whereas unrelated donor, donor-negative/recipient-positive CMV-serostatus, and presence of severe aGVHD were associated with higher risk of CMV reactivation in LMV-free control.Conclusions: LMV as CMV primary prophylaxis has a beneficial effect on post HSCT outcome decreasing the incidence of severe aGVHD and cs-CMV reactivation.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"561-568"},"PeriodicalIF":2.3,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144127068","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}

引用次数: 0

Impact of HLA eplets mismatch load on outcomes of outpatient haploidentical-related stem cell transplantation from peripheral blood after reduced intensity conditioning. HLA小体错配负荷对门诊单倍体相关外周血干细胞移植低强度调理后结果的影响

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-29 DOI: 10.1080/17474086.2025.2524522

José Carlos Jaime-Pérez, Magali Irais Ureño Segura, Adriana Domínguez-Villanueva, Nidia Karina Moncada-Saucedo, Sandra Iveth Mendoza-Ibarra, David Gómez-Almaguer

{"title":"Impact of HLA eplets mismatch load on outcomes of outpatient haploidentical-related stem cell transplantation from peripheral blood after reduced intensity conditioning.","authors":"José Carlos Jaime-Pérez, Magali Irais Ureño Segura, Adriana Domínguez-Villanueva, Nidia Karina Moncada-Saucedo, Sandra Iveth Mendoza-Ibarra, David Gómez-Almaguer","doi":"10.1080/17474086.2025.2524522","DOIUrl":"10.1080/17474086.2025.2524522","url":null,"abstract":"Background: Molecular typing before hematopoietic stem cell transplantation (HSCT) at the eplet level is an emerging method to optimize HLA matching.Materials and methods: Patients who received outpatient haploidentical HSCT (Haplo-HSCT) from a sibling after reduced intensity conditioning (RIC) were studied. HLA class I and II mismatched eplets (MEs) load was determined using HLAMatchmaker software. A cutoff > or < 10 MEs was used to assess outcomes.Results: 114 patients were studied. A locus B MEs load > 10 was associated with decreased overall survival (OS), (p = 0.049), and increased graft failure (GF) (p = 0.004). Mortality was higher with > 10 MEs in HLA-I locus B (p = 0.025), and HLA-II DRB1 (p = 0.026); chronic GVHD was higher with > 10 MEs in DRB1 (p = 0.009). Anti-HLA donor-specific antibodies (DSA) were more frequent in recipients with > 10 MEs load at locus C (p = 0.021) and DRB1 (p = 0.002). MEs load > 10 at locus C and DRB1 were associated with anti-HLA DSA. Infection (p = 0.012), DSA (p = 0.014), and relapse (p = 0.001) were associated with lower OS, while multitransfusion (p = 0.035), aGVHD (p = 0.035) and infection (p = 0.021) with reduced event-free survival (EFS).Conclusion: HLA MEs load > 10 in locus B was associated with reduced OS, and in locus DRB1 with higher death rate and cGVHD after outpatient sibling haplo-HSCT using RIC.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-9"},"PeriodicalIF":2.3,"publicationDate":"2025-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144474372","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}

引用次数: 0