Expert Review of Hematology最新文献_第7页

Challenges faced by women and girls with hemophilia/Carriers: an expert call for closing the diagnosis gap. 患有血友病或携带血友病的妇女和女童面临的挑战：专家呼吁缩小诊断差距。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-05-13 DOI: 10.1080/17474086.2026.2672096

Ramya Ramakrishnan, Miguel A Escobar, Lakshmi V Srivaths

{"title":"Challenges faced by women and girls with hemophilia/Carriers: an expert call for closing the diagnosis gap.","authors":"Ramya Ramakrishnan, Miguel A Escobar, Lakshmi V Srivaths","doi":"10.1080/17474086.2026.2672096","DOIUrl":"10.1080/17474086.2026.2672096","url":null,"abstract":"Introduction: Women and girls (WG) carrying factor 8/9 gene variant can present with hemophilia with low factor VIII/IX levels and bleeding phenotype similar to men and boys with additional gynecological bleeding, or as hemophilia carriers (HC) with normal factor levels with/without bleeding. This review aims to address the gender gap in diagnosing WG with hemophilia (WGwH)/HC and discusses ways to improve diagnosis for appropriate and timely management of their unique challenges.Areas covered: This review summarizes available evidence published in Pubmed, regarding the various challenges in WGwH/HC screening and diagnosis. Patient-related challenges in recognizing their bleeding manifestations and provider-related factors causing delay in diagnosis are outlined. Barriers encountered by providers in screening, evaluation, and diagnosis including utilization of screening tools, laboratory diagnosis with factor assays and genetic testing are discussed.Expert opinion: The diagnosis gap in WGwH/HC can be narrowed by improving patient and provider knowledge, and by developing expert recommendations for uniform screening, laboratory, and genetic testing. Creating international WGwH/HC registries, outreach, twinning programs with developing countries or centers of excellence can further help improve understanding of the unique global challenges in diagnosing these patients. Research efforts can further help mitigate existing gaps and improve overall care in WGwH/HC.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"697-703"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147856181","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

ADAMTS13 activity and clinical outcomes: a targeted literature review in congenital thrombotic thrombocytopenic purpura and other related thrombotic conditions. ADAMTS13活性和临床结果：先天性血栓性血小板减少性紫癜和其他相关血栓性疾病的针对性文献综述

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-05-28 DOI: 10.1080/17474086.2026.2678350

Harneet Kaur, Annie Berkley, Victoria Shodimu, Oliver Burn, Isobel Munro

{"title":"ADAMTS13 activity and clinical outcomes: a targeted literature review in congenital thrombotic thrombocytopenic purpura and other related thrombotic conditions.","authors":"Harneet Kaur, Annie Berkley, Victoria Shodimu, Oliver Burn, Isobel Munro","doi":"10.1080/17474086.2026.2678350","DOIUrl":"10.1080/17474086.2026.2678350","url":null,"abstract":"Introduction: Severe ADAMTS13 deficiency causes congenital thrombotic thrombocytopenic purpura (cTTP), a potentially life-threatening thrombotic microangiopathy. Considering ADAMTS13 regulates thrombosis, ADAMTS13 activity might correlate with clinical outcomes in cTTP; however, evidence evaluating this relationship remains limited.Areas covered: We conducted a targeted literature review examining associations between ADAMTS13 activity and clinical outcomes and/or treatment patterns in cTTP. Given the ultra-rarity of cTTP and extremely few cTTP-specific studies, we reviewed findings from TTP and non-TTP populations for contextual insight. The 34 included studies (MEDLINE database search: 2014-2024) statistically assessed relationships between ADAMTS13 activity and outcomes including mortality, cardiovascular events, organ damage, renal signs, or treatment frequency/patterns.Expert opinion: Lower ADAMTS13 activity appears associated with increased mortality and cardiovascular risk in non-TTP populations, providing exploratory guidance for potential protective thresholds in cTTP. Only one cTTP study met inclusion criteria, and it did not evaluate activity-outcome associations. With only four studies in immune-mediated/unspecified TTP, evidence is insufficient to draw conclusions regarding ADAMTS13 activity thresholds, prognostic value or therapeutic targets in cTTP. Future research should prioritize cTTP-specific studies, including evaluating prophylaxis and temporal peak/trough activity, to define actionable ADAMTS13 activity thresholds to guide personalized management and prevent breakthrough events or long-term complications in cTTP.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"757-765"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148028610","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

Social challenges and support needs of patients with hemophilia in Pakistan: a qualitative exploratory study. 巴基斯坦血友病患者的社会挑战和支持需求：一项定性探索性研究。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-05-21 DOI: 10.1080/17474086.2026.2676241

Hassan Raza, Umme Habiba, Sidra Rasool, Umera Saleem, Saima Farhan, Muhammad Asif Naveed, Khansa Qamar

{"title":"Social challenges and support needs of patients with hemophilia in Pakistan: a qualitative exploratory study.","authors":"Hassan Raza, Umme Habiba, Sidra Rasool, Umera Saleem, Saima Farhan, Muhammad Asif Naveed, Khansa Qamar","doi":"10.1080/17474086.2026.2676241","DOIUrl":"10.1080/17474086.2026.2676241","url":null,"abstract":"Background: Hemophilia significantly impacts both physical health and psychosocial well-being. In Pakistan, limited resources, social stigma, and inadequate support systems often worsen these burdens. This study explored the lived experiences and support needs of adult patients in thisresource-constrained setting.Research design and methods: We conducted a qualitative study with 30 men (ages 20-45) at the Sundas Foundation in Lahore. Data from semi-structured interviews in Urdu were transcribed, translated, and analyzed using thematic analysis.Results: Four key themes emerged: (1) widespread social isolation and stigma (reported by 28/30 participants); (2) the family unit as the primary source of emotional and daily support; (3) significant educational and career barriers caused by physical limitations (25/30); and (4) an urgent need for decentralized care to ease travel and financial pressures (27/30). Younger participants increasingly relied on digital peer-support for practical treatment advice and emotional connection.Conclusions: Adults with hemophilia in Pakistan face deep-seated social and structural hurdles. Improving quality of life requires shifting toward community-based services, raising public awareness, and embedding psychosocial support into routine clinical care. While limited by its single-center design, this study highlights critical areas for policy intervention.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"793-797"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147962675","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

Bleeding Disorders Research Collaborative: an opportunity for impact. 出血性疾病研究合作：影响的机会。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-08-06 DOI: 10.1080/17474086.2026.2688871

Maria E Santaella, Sammie Valadez, Esmeralda Vázquez, Halli Benasutti, Samantha A Carlson, Donna DiMichele, Keri L Norris, Fiona Robinson, Leonard A Valentino, Michelle L Witkop, Michael Recht

{"title":"Bleeding Disorders Research Collaborative: an opportunity for impact.","authors":"Maria E Santaella, Sammie Valadez, Esmeralda Vázquez, Halli Benasutti, Samantha A Carlson, Donna DiMichele, Keri L Norris, Fiona Robinson, Leonard A Valentino, Michelle L Witkop, Michael Recht","doi":"10.1080/17474086.2026.2688871","DOIUrl":"10.1080/17474086.2026.2688871","url":null,"abstract":"Introduction: The National Research Blueprint proposes a Bleeding Disorders Research Collaborative (BDRC) centering people, priorities, and principles. Driven by community-identified unmet needs, the National Bleeding Disorders Foundation (NBDF) coordinated a five-year co-creation endeavor engaging a multitude of diverse stakeholders.Areas covere: Seven multidisciplinary working groups built upon previous NBDF State of the Science Summit conclusions to draft a blueprint of the research and development, workforce, infrastructure, community engagement, policy, Lived Experience Expert, and health equity, diversity, and inclusion elements of the novel research collaborative. Implementing their proposal will engage the entire inheritable bleeding disorders community mobilizing all its expertise, resources, champions, and partners. Education, training, and mentorship will be key in empowering diverse research teams working together in respectful partnership.Expert opinion: The proposed BDRC has the potential to transform inheritable bleeding disorders research through its collaborative infrastructure centering lived experience expertise and health equity, diversity, and inclusion to efficiently advance health justice for the entire community.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"19 sup1","pages":"7-22"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148684033","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

Rilzabrutinib for persistent or chronic immune thrombocytopenia (ITP): a Bruton tyrosine kinase inhibitor that can target multiple pathophysiological pathways. 利扎布替尼治疗持续性或慢性免疫性血小板减少症（ITP）：一种布鲁顿酪氨酸激酶抑制剂，可靶向多种病理生理途径。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-05-13 DOI: 10.1080/17474086.2026.2672113

Ahmed Mjali, Sylvain Audia, Yoshitaka Miyakawa, Waleed Ghanima

{"title":"Rilzabrutinib for persistent or chronic immune thrombocytopenia (ITP): a Bruton tyrosine kinase inhibitor that can target multiple pathophysiological pathways.","authors":"Ahmed Mjali, Sylvain Audia, Yoshitaka Miyakawa, Waleed Ghanima","doi":"10.1080/17474086.2026.2672113","DOIUrl":"10.1080/17474086.2026.2672113","url":null,"abstract":"Introduction: Immune thrombocytopenia (ITP) is characterized by reduced platelet production and platelet destruction. Despite the availability of numerous treatments, many patients develop treatment resistance, experience relapse, or suffer from persistent symptoms such as fatigue, highlighting the need for innovative immune-modulating techniques. This review highlights the chemical properties, clinical efficacy and safety evidence for rilzabrutinib.Areas covered: Bruton tyrosine kinase (BTK) is a promising therapeutic target in ITP as it is essential for B-cell receptor signaling, Fcγ receptor-mediated platelet clearance, and inflammatory amplification. Rilzabrutinib is a reversible, covalent oral BTK inhibitor developed to treat immune-mediated disorders. It targets several pathogenic immunological pathways. In the phase 2 LUNA 2 trial and the subsequent randomized, placebo-controlled, phase 3 LUNA 3 study, rilzabrutinib was clinically evaluated in patients with persistent or chronic ITP. The treatment resulted in durable platelet responses in extensively pretreated patients, decreased bleeding episodes, and led to significant improvements in patient-reported fatigue. The safety profile was generally good with adverse events considered mild to moderate.Expert opinion: The approval of rilzabrutinib for persistent and chronic ITP represents an important addition to the therapeutic armamentarium for the disease. The drug's full potential will be further defined in future studies.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"685-696"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147863878","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

Centering Lived Experience Experts and health equity in the Bleeding Disorders Research Collaborative. 以出血性疾病研究合作中的生活经验专家和健康公平为中心。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-08-06 DOI: 10.1080/17474086.2026.2688873

Sammie Valadez, Maria E Santaella, Samantha A Carlson, Erin Burke Cirelli, Fiona Robinson, Raymond W Stanhope, Esmeralda Vázquez, Keri L Norris

引用次数: 0

Plain language summary: a phase 2/3 study of plasminogen, human-tvmh for the treatment of children and adults with type 1 plasminogen deficiency. 简单的语言总结：一项2/3期研究，用于治疗1型纤溶酶原缺乏症的儿童和成人。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-06-01 DOI: 10.1080/17474086.2026.2678346

Amy D Shapiro, Charles Nakar, Joseph M Parker, Karen Thibaudeau, Roberto Crea, Per Morten Sandset

引用次数: 0

Apparent plateaus during iron chelation in transfusion-dependent β-thalassemia: interpretation and clinical implications. 输血依赖性β-地中海贫血铁螯合过程中的明显高原：解释和临床意义。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-05-12 DOI: 10.1080/17474086.2026.2672115

Sophia Delicou, Constantina Aggeli, Iliana Mani, Christos Savvidis, Aikaterini Xydaki, Maria Moraki, John Koskinas

{"title":"Apparent plateaus during iron chelation in transfusion-dependent β-thalassemia: interpretation and clinical implications.","authors":"Sophia Delicou, Constantina Aggeli, Iliana Mani, Christos Savvidis, Aikaterini Xydaki, Maria Moraki, John Koskinas","doi":"10.1080/17474086.2026.2672115","DOIUrl":"10.1080/17474086.2026.2672115","url":null,"abstract":"Introduction: Transfusion-dependent β-thalassemia (TDT) has become a chronic, survivable condition, but transfusional iron overload remains a central driver of cardiac, hepatic, and endocrine morbidity. Clinicians increasingly encounter patients whose iron indices 'stall' despite seemingly appropriate chelation, creating uncertainty about whether the problem is correctable (iron balance, adherence, dosing) or reflects a harder biological limitation in iron mobilization.Areas covered: A narrative review of pivotal randomized trials, prospective cohorts, and major observational MRI datasets evaluating deferoxamine, deferiprone, deferasirox, and combination regimens in TDT was performed. Literature was identified via structured searches of PubMed and key hematology/cardiometabolic journals (2000-February 2026), prioritizing primary studies with MRI liver iron concentration (LIC) and cardiac T2* endpoints, and supplemented by regulatory prescribing information for safety monitoring.Expert opinion/commentary: Many 'plateaus' are predictable steady-state iron balance-ongoing transfusional iron input matches achievable chelator-mediated excretion-and can be addressed by quantifying iron intake, verifying adherence, and rationally intensifying therapy. A second category, a hypothesized 'biological ceiling,' may arise when stored iron becomes less accessible (slow-turnover pools, tissue remodeling, altered export). Clinicians can disentangle these mechanisms by integrating transfusion iron input calculations with serial multi-organ MRI trajectories, then tailoring chelator strategy to the dominant iron compartment and organ risk.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"747-756"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147835791","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

Lessons from warfarin management during the COVID-19 pandemic: from crisis to an opportunity to revolutionize telemedicine for chronic disease care. COVID-19大流行期间华法林管理的经验教训：从危机到慢性病护理远程医疗革命的机遇

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-06-25 DOI: 10.1080/17474086.2026.2692435

Guilan Wu, Shaojun Jiang, Jinhua Zhang

{"title":"Lessons from warfarin management during the COVID-19 pandemic: from crisis to an opportunity to revolutionize telemedicine for chronic disease care.","authors":"Guilan Wu, Shaojun Jiang, Jinhua Zhang","doi":"10.1080/17474086.2026.2692435","DOIUrl":"10.1080/17474086.2026.2692435","url":null,"abstract":"Introduction: Corona virus disease 2019 (COVID-19) had a dramatic impact on the daily management of warfarin. To improve the quality of warfarin anticoagulation management in the outbreak setting while reducing the risk of patient exposure to COVID-19, researchers around the world have conducted many explorations and practices on warfarin management models.Areas covered: This review analyzes the factors affecting the management of warfarin by COVID-19, summarizes the current situation of warfarin management in the epidemic environment, introduces the characteristics and limitations of feasible warfarin management strategies, and provides a reference for the selection of warfarin management plans in clinical practice. From before 31 May 2025, we conducted a literature search using the PubMed database to identify relevant studies.Expert opinion: Warfarin management faced major challenges during the epidemic, exposing the vulnerability of its management system. At the same time, this state of affairs has driven improvements and innovations in related programs, facilitating the use of telemedicine technology in anticoagulation therapy. More importantly, this change is expected to inspire legislators and policy makers to introduce policies to extend telemedicine to a wider range of chronic disease management areas, thereby improving the overall quality and efficiency of healthcare services.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-7"},"PeriodicalIF":2.8,"publicationDate":"2026-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148293937","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

Improving multidisciplinary tailored care models for bleeding disorders. 改进出血性疾病的多学科定制护理模式。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-06-01 Epub Date: 2026-04-07 DOI: 10.1080/17474086.2026.2655968

Tinatin Muradashvili, Robert F Sidonio

{"title":"Improving multidisciplinary tailored care models for bleeding disorders.","authors":"Tinatin Muradashvili, Robert F Sidonio","doi":"10.1080/17474086.2026.2655968","DOIUrl":"10.1080/17474086.2026.2655968","url":null,"abstract":"Introduction: Bleeding disorder care has advanced rapidly, but care delivery has not evolved at the same pace. While multidisciplinary models transformed outcomes in hemophilia, many patients with diverse bleeding phenotypes continue to face delayed diagnosis, fragmented care, and vulnerable transitions across the life span.Areas covered: This review explores how multidisciplinary care can be adapted to meet the changing needs of people with inherited and acquired bleeding disorders of different phenotypes at different life stages. A targeted literature search was conducted through 1 February 2026, including peer-reviewed studies, registry analyses, consensus guidance, and quality improvement reports. We summarize emerging tailored care models and examine structural factors influencing access, continuity, and effectiveness. Evidence was identified through a narrative review of peer-reviewed literature, registry analyses, consensus guidance, and quality-improvement reports.Expert opinion: The future of bleeding disorder care lies in flexible, tailored multidisciplinary models rather than uniform approaches. Progress will depend on redesigning care delivery, adopting patient-centered outcome measures, and prioritizing equity and scalability. Over time, care is expected to shift toward integrated, hybrid systems that support earlier diagnosis, sustained engagement, and safer long-term management.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"585-599"},"PeriodicalIF":2.8,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147607951","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