Expert Review of Hematology最新文献_第8页

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

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

Is the early use of ketamine effective as an opioid sparing adjunct for treating pain in sickle cell disease patients? 早期使用氯胺酮作为阿片类药物保留辅助治疗镰状细胞病患者疼痛有效吗？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-11 DOI: 10.1080/17474086.2025.2500605

Vineetha Reddy Nallagatla, Natasha M Archer

引用次数: 0

Treating sickle cell disease in resource-limited sub-Saharan Africa: recent strategies and recommendations in addressing the gaps for the provision of evidence-based management. 在资源有限的撒哈拉以南非洲治疗镰状细胞病：在提供循证管理方面弥补差距的最新战略和建议。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-11 DOI: 10.1080/17474086.2025.2500599

Miguel Brito, Catarina Ginete, Akinyemi Ofakunrin, Ijeoma Diaku-Akinwumi, Baba Psalm Duniya Inusa

{"title":"Treating sickle cell disease in resource-limited sub-Saharan Africa: recent strategies and recommendations in addressing the gaps for the provision of evidence-based management.","authors":"Miguel Brito, Catarina Ginete, Akinyemi Ofakunrin, Ijeoma Diaku-Akinwumi, Baba Psalm Duniya Inusa","doi":"10.1080/17474086.2025.2500599","DOIUrl":"10.1080/17474086.2025.2500599","url":null,"abstract":"Introduction: The management of Sickle cell disease (SCD) in sub-Saharan Africa (SSA) suffers from the lack of universal infant and population screening, inadequate access to standard treatment and poor public health prioritization amidst unstable political systems.Areas covered: The state of evidencebased management of SCD in SSA was investigated including sustainability of international funding agencies.Expert opinion: Current efforts are fragmentary along languages lines; sometimes driven by the funder's objectives and not the national agenda. The review highlighted the role of internal and external partnerships such as SPARCO, ARISE, CONSA, as well as technology-based support for the implementation of evidence-based care for SCD. We advocate for increased funding to implement SCD comprehensive care in line with the WHO SCD Framework for Primary, Secondary, Tertiary and Specialist Comprehensive Care at state and national level. To achieve this objective, it is important that SCD, as a leading non-communicable disease in Africa, be mandated as a standing agenda for the National Council of Ministers at the African Union, WHO and other regional bodies in Africa.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"447-462"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143964168","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

Kompetitive allele specific PCR (KASP) based genotyping of sickle gene in the selected sub-ethnic tribal population of Gujarat and Madhya Pradesh. 基于竞争等位基因特异性PCR （KASP）的古吉拉特邦和中央邦亚民族部落人群镰刀基因分型研究

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-08 DOI: 10.1080/17474086.2025.2499530

Urvi Budhbhatti, Bhumika Prajapati, Bhagirath Dave, Aman Tripathi, Chaitanya Joshi, Madhvi Joshi

{"title":"Kompetitive allele specific PCR (KASP) based genotyping of sickle gene in the selected sub-ethnic tribal population of Gujarat and Madhya Pradesh.","authors":"Urvi Budhbhatti, Bhumika Prajapati, Bhagirath Dave, Aman Tripathi, Chaitanya Joshi, Madhvi Joshi","doi":"10.1080/17474086.2025.2499530","DOIUrl":"10.1080/17474086.2025.2499530","url":null,"abstract":"Background: Indian tribal population is more vulnerable to sickle cell disease (SCD), an autosomal recessive disease caused by a single A>T transversion mutation in the HBB gene. Current study aims to develop a novel Kompetitive allele specific PCR (KASP) assay for genotyping of sickle gene in selected tribal communities of India.Patients and methods: The study was conducted from 2021 to 2023 involving 583 self-declared healthy individuals from four tribal communities, i.e. Korku, Kol, and Sahariya of Madhya Pradesh and Dongri bhil of Gujarat. The sickle cell genotyping was performed using KASP and PCR-RFLP methods and further validated by Sanger sequencing. Various hematological and biochemical parameters were also studied and compared for their association with identified genotypes.Results: Prevalence of sickle cell trait (SCT) was observed as 4.67%, 9.33%, and 16.41% in Kol, Korku, and Dongri bhil, respectively. The SCD prevalence of 0.7% was observed in Dongri bhil. The KASP assay reflects as a novel, high-throughput, and cost-effective method for effective screening. RBC, folic acid, fetal Hb (HBF), and bilirubin have shown significant association with sickle gene mutation.Conclusions: KASP assay offers a rapid, precise, and cost-effective method for screening in tribal populations, which can help in early identification, management, and elimination of disease.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"491-499"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143981915","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

How can we improve healthcare outcomes for hemophilia B patients with inhibitors? 我们如何改善使用抑制剂的B型血友病患者的医疗结果？

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-04-29 DOI: 10.1080/17474086.2025.2499524

Kristin N Maher, Allison P Wheeler

引用次数: 0

To enhance the detection of aplastic anemia in primary care settings: a population-based study in Italy. 加强在初级保健机构再生障碍性贫血的检测：在意大利的一项基于人群的研究。

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-05-10 DOI: 10.1080/17474086.2025.2500604

Francesco Lapi, Ettore Marconi, Iacopo Cricelli, Adriana Masotti, Alessandro Rossi, Claudio Cricelli

{"title":"To enhance the detection of aplastic anemia in primary care settings: a population-based study in Italy.","authors":"Francesco Lapi, Ettore Marconi, Iacopo Cricelli, Adriana Masotti, Alessandro Rossi, Claudio Cricelli","doi":"10.1080/17474086.2025.2500604","DOIUrl":"10.1080/17474086.2025.2500604","url":null,"abstract":"Background: Aplastic Anemia (AA) is a rare, life-threatening condition featured by hypocellular bone marrow without leukemia or myelodysplastic syndromes. Early diagnosis is vital because of the effectiveness of the existing treatments. This study examined AA epidemiology and clinical correlates to aid general practitioners (GPs) in recognizing potential AA cases.Research design and methods: The Italian Health Search database (HSD) was used. A cohort study on individuals aged 18 years and older registered in the HSD between 1 January 1998, and 31 December 2022, estimated the prevalence and incidence rate of AA. Cases were operationally classified as 'certain,' 'probable,' and 'possible.' A case-control study was conducted to examine the clinical correlates of AA.Results: The cumulative prevalence was 3.8-4.8 per 100,000. The AA incidence rates for certain, certain/probable, and certain/probable/possible diagnoses were 0.3, 0.7, and 6 cases/million person-years, respectively. Increased infections (OR = 2.5), higher comorbidity burden (Charlson Index 1: OR = 2.14; 2+: OR = 2.43), and immunosuppressants use (OR = 14.9) were strongly associated with an AA diagnosis.Conclusions: Our findings indicate that AA is often underdiagnosed in primary care, but these data could help raise the suspicion of AA. Efforts are needed to utilize GPs' healthcare records for early AA identification and to enhance GP-hematologist collaboration.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"463-469"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143989716","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

Vasculopathy among children and adolescents with sickle cell disease: the crosstalk with annexin A1, vitamin D, and myocardial iron overload. 儿童和青少年镰状细胞病患者的血管病变：与膜联蛋白A1、维生素D和心肌铁超载的串扰

IF 2.3 4区医学

Expert Review of Hematology Pub Date : 2025-06-01 Epub Date: 2025-04-18 DOI: 10.1080/17474086.2025.2495670

Mohsen Saleh ElAlfy, Eman Abdel Rahman Ismail, Sara Mostafa Makkeyah, Ahmed Samir, Dina Husseiny Salama, Nanis Mohammed Salah Eldin, Dina Mohamed Fathy ElMaghraby, Nada Ayman Gad, Marwa Fath Ahmed Ali, Fatma Soliman Elsayed Ebeid

{"title":"Vasculopathy among children and adolescents with sickle cell disease: the crosstalk with annexin A1, vitamin D, and myocardial iron overload.","authors":"Mohsen Saleh ElAlfy, Eman Abdel Rahman Ismail, Sara Mostafa Makkeyah, Ahmed Samir, Dina Husseiny Salama, Nanis Mohammed Salah Eldin, Dina Mohamed Fathy ElMaghraby, Nada Ayman Gad, Marwa Fath Ahmed Ali, Fatma Soliman Elsayed Ebeid","doi":"10.1080/17474086.2025.2495670","DOIUrl":"10.1080/17474086.2025.2495670","url":null,"abstract":"Background: Annexin A1 plays an important role in myocardial defense against ischemia-reperfusion injury. We aimed to evaluate the role of annexin A1 as a potential marker of vasculopathy in children and adolescents with sickle cell disease (SCD) and its relation to myocardial iron content (MIC) and vitamin D status.Research design and methods: Forty-one patients with SCD were compared with 40 age- and sex-matched healthy controls, and underwent assessment of serum annexin A1, vitamin D, Doppler echocardiography and cardiac magnetic resonance (CMR).Results: Six (14.6%) SCD patients had cardiac disease, five (12.2%) had abnormal MIC (≥1.16) and 10 (24.4%) had pulmonary hypertension risk. Annexin A1 levels were significantly lower among patients with SCD compared with healthy controls (p < 0.001). SCD patients with pulmonary hypertension risk, evidence of diastolic dysfunction, and nephropathy as well as those with serum ferritin ≥ 2500 µg/L and vitamin D deficiency had lower Annexin A1 levels than those without. Serum annexin A1 levels were negatively correlated to urinary albumin creatinine ratio (UACR) and Tei index while positively correlated to vitamin D among SCD patients.Conclusions: Annexin A1 could be a promising marker of vasculopathy and may provide a biochemical explanation for vitamin D deficiency in SCD.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"481-490"},"PeriodicalIF":2.3,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144001681","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