Expert Review of Hematology最新文献_第6页

Review, conceptual model, and gap analysis of transfusion burden in hematological diseases. 血液病中输血负担的回顾、概念模型和差距分析。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-06-18 DOI: 10.1080/17474086.2026.2667750

Jennifer Lord-Bessen, Esther Natalie Oliva, Mrudula B Glassberg, Hong Xiao, Jessica Jordan, Gale Harding, Ahmed Hnoosh

{"title":"Review, conceptual model, and gap analysis of transfusion burden in hematological diseases.","authors":"Jennifer Lord-Bessen, Esther Natalie Oliva, Mrudula B Glassberg, Hong Xiao, Jessica Jordan, Gale Harding, Ahmed Hnoosh","doi":"10.1080/17474086.2026.2667750","DOIUrl":"10.1080/17474086.2026.2667750","url":null,"abstract":"Introduction: Hematological diseases affect millions of people worldwide, and many of these patients require blood transfusions. Although the potential adverse clinical outcomes associated with transfusions are well documented, the patient perspective of transfusion burden in hematological diseases is not. This perspective is critical, as health-related quality of life scores alone cannot fully capture patients' experiences.Areas covered: A comprehensive review was conducted to understand the patient perspective of transfusion burden in hematological diseases. This study included a targeted literature review of peer-reviewed articles conducted in Embase and MEDLINE from January 2012-September 2024 (stage 1), a preliminary conceptual model (stage 2), a review of patient-reported outcome measures (PROMs) included in clinical trials and label claims (stage 3), and a gap analysis of existing PROMs (stage 4).Expert opinion: This study identified four key concepts and two existing PROMs that were relevant to transfusion burden in hematological diseases. Transfusions impact patients economically, physically, socially, and emotionally. The study identified an unmet need: existing PROMs lack specificity to transfusion burden, lack generalizability to all hematological diseases, and do not reflect all four key concepts identified. A new PROM is needed to better understand the patient perspective of transfusion burden in hematological diseases.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"767-778"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147928005","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

Lived Experience Expert, community engagement, policy, and health equity, diversity, and inclusion elements of the Bleeding Disorders Research Collaborative. 出血性疾病研究协作的生活经验专家、社区参与、政策和健康公平、多样性和包容要素。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-08-06 DOI: 10.1080/17474086.2026.2688870

Kyle A Davis, Nathan Mermilliod, Jeremy Griffin, Samantha A Carlson, Erin Burke Cirelli, Michelle Conde, Donna DiMichele, Alexis Dinno, Michael Glenzer, Roshni Kulkarni, Regina Legere, Marissa Melton, Keri L Norris, Richard Pezzillo, Michael Recht, Fiona Robinson, Maria E Santaella, Nathan Schaefer, Raymond W Stanhope, Sammie Valadez, Leonard A Valentino, Esmeralda Vázquez, Mosi Williams, Michelle Witkop, Shannon L Carpenter

{"title":"Lived Experience Expert, community engagement, policy, and health equity, diversity, and inclusion elements of the Bleeding Disorders Research Collaborative.","authors":"Kyle A Davis, Nathan Mermilliod, Jeremy Griffin, Samantha A Carlson, Erin Burke Cirelli, Michelle Conde, Donna DiMichele, Alexis Dinno, Michael Glenzer, Roshni Kulkarni, Regina Legere, Marissa Melton, Keri L Norris, Richard Pezzillo, Michael Recht, Fiona Robinson, Maria E Santaella, Nathan Schaefer, Raymond W Stanhope, Sammie Valadez, Leonard A Valentino, Esmeralda Vázquez, Mosi Williams, Michelle Witkop, Shannon L Carpenter","doi":"10.1080/17474086.2026.2688870","DOIUrl":"10.1080/17474086.2026.2688870","url":null,"abstract":"Background: The U.S. National Bleeding Disorders Foundation charged seven multidisciplinary working groups (WGs) with developing a National Research Blueprint (NRB) for a novel Bleeding Disorders Research Collaborative (BDRC) firmly rooted in health equity, diversity, and inclusion (HEDI) and centering the knowledge of people living with inheritable bleeding disorders, the Lived Experience Experts (LEEs).Research design and methods: The HEDI, LEE, Community Engagement, and Policy WGs met virtually to develop recommendations for BDRC design and operationalization.Results: Engaging, empowering, and elevating guidance and practices to ground BDRC research and operations in HEDI principles are detailed. The full potential of integrating LEE knowledge throughout research conduct and BDRC governance is explored, potential barriers identified, and solutions proposed. Investing in relationships, trust, and transparency are elaborated as keys to meeting community members wherever they are on a research engagement continuum and facilitating desired progression. Expert advocacy and partnerships will be essential to securing policies and funding supporting the BDRC.Conclusions: The proposed community-inspired BDRC has the potential to catalyze a paradigm shift in U.S. inheritable bleeding disorders research and ultimately advance health equity for all.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"19 sup1","pages":"23-42"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148684019","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

Infrastructure and workforce development processes inspired by Lived Experience Experts and grounded in health equity for the Bleeding Disorders Research Collaborative. 基础设施和劳动力发展过程受到生活经验专家的启发，并以出血性疾病研究协作的卫生公平为基础。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-08-06 DOI: 10.1080/17474086.2026.2688869

Lynn Malec, Moses E Miles, Lauren E Amos, Samantha A Carlson, Donna DiMichele, Iftikhar Haider, Andra H James, Keri L Norris, Kristin Paulyson-Nuñez, Michael Recht, Fiona Robinson, Maria E Santaella, Raymond W Stanhope, Sammie Valadez, Michelle L Witkop, Ziva Mann, Amy D Shapiro, Margaret V Ragni

{"title":"Infrastructure and workforce development processes inspired by Lived Experience Experts and grounded in health equity for the Bleeding Disorders Research Collaborative.","authors":"Lynn Malec, Moses E Miles, Lauren E Amos, Samantha A Carlson, Donna DiMichele, Iftikhar Haider, Andra H James, Keri L Norris, Kristin Paulyson-Nuñez, Michael Recht, Fiona Robinson, Maria E Santaella, Raymond W Stanhope, Sammie Valadez, Michelle L Witkop, Ziva Mann, Amy D Shapiro, Margaret V Ragni","doi":"10.1080/17474086.2026.2688869","DOIUrl":"10.1080/17474086.2026.2688869","url":null,"abstract":"Background: Accelerating research advancing health equity for the entire inheritable bleeding disorders community requires a new approach. It must be firmly rooted in health equity, diversity, and inclusion (HEDI) and center the knowledge of people living with inheritable bleeding disorders, the Lived Experience Experts (LEEs). The National Bleeding Disorders Foundation charged seven multidisciplinary working groups (WGs) with developing a National Research Blueprint (NRB) for this Bleeding Disorders Research Collaborative (BDRC).Research design and methods: The Infrastructure and Workforce WGs, in collaboration with the HEDI and LEE WGs, met virtually to develop recommendations for BDRC operationalization.Results: A progressive network of elements and processes capacitating diverse community-prioritized research ideas into successfully completed BDRC projects is proposed. Essential components for launch, iterative evolution, effective conduct, and accountability are described. Sharing resources and expertise and embedding research in inheritable bleeding disorders care will create synergistic efficiencies. Education and training to grow and empower interdisciplinary research teams, including LEEs and HEDI champions as valued members, are detailed. Shared leadership integrating LEE and HEDI expertise throughout will provide dynamic governance.Conclusions: BDRC infrastructure and workforce development must start small and grow iteratively in partnership with the many organizations that share its vision of health justice.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"19 sup1","pages":"43-62"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148683947","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

Changes in circular RNA expression in acute chest syndrome and vaso-occlusive crisis in sickle cell disease: analysis of a public RNA-seq cohort. 镰状细胞病急性胸综合征和血管闭塞危象中环状RNA表达的变化：一项公开RNA序列队列分析

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-05-09 DOI: 10.1080/17474086.2026.2671173

Alawi Habara

{"title":"Changes in circular RNA expression in acute chest syndrome and vaso-occlusive crisis in sickle cell disease: analysis of a public RNA-seq cohort.","authors":"Alawi Habara","doi":"10.1080/17474086.2026.2671173","DOIUrl":"10.1080/17474086.2026.2671173","url":null,"abstract":"Background: Sickle cell disease (SCD) is a monogenic hemoglobinopathy characterized by recurrent vaso-occlusive crises (VOCs) that affect any organ and progress to multi-organ failure. Acute chest syndrome (ACS) is a major complication and leading cause of death. This study explored whole-blood circRNA signatures as candidate molecular markers for VOC and ACS and examined their functional relevance through miRNA mapping and pathway enrichment.Research design and methods: A publicly available total RNA-seq dataset (GSE139912) was analyzed for circRNA expression at baseline (n = 12), VOC (n = 10), and ACS (n = 11).Results: Significant circRNA dysregulation was identified in ACS vs. baseline and VOC vs. baseline. An exploratory panel of the top 20 upregulated and 16 downregulated circRNAs in ACS was summarized as a composite circRNA score. The unchanged score increased stepwise across clinical states, with mean differences of 1.60 for VOC vs. baseline and 1.08 for ACS vs. VOC. Standardized effect sizes were Cohen's d = 2.89 and 1.61, respectively. VOC showed intermediate scores between baseline and ACS. Enrichment analyses suggested immune and inflammatory involvement, including interleukin signaling and PI3K/AKT/MAPK-related cascades.Conclusions: These findings support circRNA expression as a source of candidate biomarkers for ACS and VOC, although validation in independent multicenter cohorts is required.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"779-791"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147835830","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

The hunt for a cure: modern medical approaches for immune thrombocytopenia. 寻找治疗方法：免疫性血小板减少症的现代医学方法。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-05-26 DOI: 10.1080/17474086.2026.2678348

Debbie Jiang, David J Kuter

{"title":"The hunt for a cure: modern medical approaches for immune thrombocytopenia.","authors":"Debbie Jiang, David J Kuter","doi":"10.1080/17474086.2026.2678348","DOIUrl":"10.1080/17474086.2026.2678348","url":null,"abstract":"Introduction: Splenectomy and stem cell transplant have historically provided long-term remissions that seemingly cure a subset of patients with immune thrombocytopenia (ITP). Dozens of modern medical therapies with better safety profiles have revolutionized ITP treatment and several can induce sustained response off treatment (SROT) and delay relapse, providing hope for disease modification and cure.Areas covered: In this review, we consider how ITP cure might be defined, review key pathophysiologic mechanisms relevant for disease modification, discuss historical approaches, and explore modern therapies that hold promise. Articles were identified based on the authors' expertise. This review on potential ITP cure focuses on pathophysiologic studies that shed light on determinants of immunologic complexity, persistence of autoimmune memory, and treatment resistance or refractoriness and clinical studies that report on long-term remission and relapse rates, prioritizing clinical trials and large retrospective studies, where available.Expert opinion: While SROT and deep immunologic disruption are possible with modern therapies, it remains unknown whether this translates to a meaningful alteration of ITP trajectory. As the field strives for cure, identifying predictive biomarkers, clinical trials incorporating longer follow-up, better understanding of the pathophysiologic underpinnings of autoimmune memory, and balancing patient-focused considerations of risk and toxicity are critical.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"705-719"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147980857","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

T-cell acute lymphoblastic leukemia in adults: current treatment approaches and future perspectives. 成人t细胞急性淋巴细胞白血病：目前的治疗方法和未来的展望。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-06-03 DOI: 10.1080/17474086.2026.2678349

Lorenzo Celona, Eleonora Boscaro, Giulia Berutto, Davide Stella, Camilla Gariazzo, Federica Catania, Carlotta Zavatto, Chiara Dellacasa, Carolina Secreto, Elisa Santambrogio, Irene Urbino, Chiara Frairia, Valentina Giai, Stefano D'Ardia, Roberto Freilone, Alessandro Busca, Ernesta Audisio, Marco Cerrano

{"title":"T-cell acute lymphoblastic leukemia in adults: current treatment approaches and future perspectives.","authors":"Lorenzo Celona, Eleonora Boscaro, Giulia Berutto, Davide Stella, Camilla Gariazzo, Federica Catania, Carlotta Zavatto, Chiara Dellacasa, Carolina Secreto, Elisa Santambrogio, Irene Urbino, Chiara Frairia, Valentina Giai, Stefano D'Ardia, Roberto Freilone, Alessandro Busca, Ernesta Audisio, Marco Cerrano","doi":"10.1080/17474086.2026.2678349","DOIUrl":"10.1080/17474086.2026.2678349","url":null,"abstract":"Introduction: The therapeutic landscape of acute lymphoblastic leukemia (ALL) in adults has greatly changed thanks to the introduction of response-adapted pediatric-inspired regimens and, in B-ALL, the advent of immunotherapy. More recently, the better understanding of genetics, the encouraging results of new agents and the evidence of activity of monoclonal antibodies and CAR-T cells are opening the road for meaningful advances in T-ALL.Areas covered: We discuss the current therapeutic scenario of T-ALL in adults, including the basis of relevant chemotherapy backbones and a summary of recent biological advances with potential clinical implications. Besides, we summarize data of new drugs explored in the relapsed/refractory (R/R) setting and how they are moving frontline, including a focus of the emerging role of immunotherapy.Expert opinion: The availability of effective agents like venetoclax, bortezomib and daratumumab may lead to improved frontline regimens, which will be tailored according to patient's genetics and drug response profile, and relevant role of CAR-T cell is foreseen, possibly beyond R/R setting. While several issues will require further studies, including central nervous system prophylaxis and treatment optimization in young adults and older patients, these advances will ultimately lead to improved efficacy, reduced toxicities and limited indications of allo-HCT.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"721-745"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147981428","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

Research agenda and related workforce requirements inspired by Lived Experience Experts and grounded in health equity for the Bleeding Disorders Research Collaborative. 研究议程和相关的劳动力要求，由生活经验专家启发，并以出血性疾病研究协作的卫生公平为基础。

IF 2.8 4区医学

Expert Review of Hematology Pub Date : 2026-07-01 Epub Date: 2026-08-06 DOI: 10.1080/17474086.2026.2688867

Jill M Johnsen, Tyler W Buckner, Glaivy Batsuli, Samantha A Carlson, Erin Burke Cirelli, Donna DiMichele, Veronica H Flood, Andrew D Johnson, Meadow Heiman, Justine Kaplan, Barbara A Konkle, Bernard Khor, Emily W Lankau, Gallia G Levy, Keri L Norris, Glenn F Pierce, Nicholas A Popp, Michael Recht, Jonathan C Roberts, Fiona Robinson, Maria E Santaella, Jordan A Shavit, Raymond W Stanhope, Sammie Valadez, Michelle L Witkop, Lynn Malec, Amy D Shapiro

{"title":"Research agenda and related workforce requirements inspired by Lived Experience Experts and grounded in health equity for the Bleeding Disorders Research Collaborative.","authors":"Jill M Johnsen, Tyler W Buckner, Glaivy Batsuli, Samantha A Carlson, Erin Burke Cirelli, Donna DiMichele, Veronica H Flood, Andrew D Johnson, Meadow Heiman, Justine Kaplan, Barbara A Konkle, Bernard Khor, Emily W Lankau, Gallia G Levy, Keri L Norris, Glenn F Pierce, Nicholas A Popp, Michael Recht, Jonathan C Roberts, Fiona Robinson, Maria E Santaella, Jordan A Shavit, Raymond W Stanhope, Sammie Valadez, Michelle L Witkop, Lynn Malec, Amy D Shapiro","doi":"10.1080/17474086.2026.2688867","DOIUrl":"10.1080/17474086.2026.2688867","url":null,"abstract":"Background: Inheritable bleeding disorders (BDs) research has not historically reflected the diversity or needs of the entire community. The National Bleeding Disorders Foundation charged seven multidisciplinary working groups (WGs) with developing a U.S. National Research Blueprint (NRB) for a Bleeding Disorders Research Collaborative (BDRC) inspired by Lived Experience Experts (LEE) and grounded in health equity, diversity, and inclusion (HEDI).Research design and methods: The Research and Development and Workforce WGs, in collaboration with the HEDI and LEE WGs, met virtually and in-person to develop recommendations for BDRC operationalization.Results: An agenda of 327 feasible research priorities spanning nine main topics, each with four to six scientific areas of interest is proposed. It captures the hope that new diagnostic and therapeutic technologies and innovative research approaches enriched by LEE and HEDI expertise may advance health equity for all. Key constraints of time, expertise, funding, resources, and diversity were identified as important barriers to the capacitation of the interdisciplinary research workforce required to successfully achieve this research. Mentorship, partnership, training and education, collaboration, and advocacy solutions to these barriers are proposed.Conclusions: The BDRC seeks to capacitate a diverse, inclusive, collaborative workforce, and effectively accelerate research that advances health equity for all.","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"19 sup1","pages":"63-79"},"PeriodicalIF":2.8,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148683992","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

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