Blood Reviews最新文献_第2页

Unmet needs in hemophilic arthropathy. 血友病关节病未满足的需求。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-05-21 DOI: 10.1016/j.blre.2025.101304

Yesim Dargaud, Sebastien Lobet, Nathalie Roussel, Leonard A Valentino

引用次数: 0

Patient-reported outcomes - the missing link to advancing light chain (AL) amyloidosis clinical research. 患者报告的结果-推进轻链（AL）淀粉样变性临床研究的缺失环节。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-05-20 DOI: 10.1016/j.blre.2025.101303

Tobias Dittrich, Lina Weinert, Anita D'Souza

引用次数: 0

The critical role of platelets in venous thromboembolism: Pathogenesis, clinical status, and emerging therapeutic strategies. 血小板在静脉血栓栓塞中的关键作用：发病机制、临床状况和新兴的治疗策略。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-05-14 DOI: 10.1016/j.blre.2025.101302

Shuang Liu, Yan Shen, Jiayi Chen, Zheng Ruan, Li Hua, Kankan Wang, Xiaodong Xi, Jianhua Mao

{"title":"The critical role of platelets in venous thromboembolism: Pathogenesis, clinical status, and emerging therapeutic strategies.","authors":"Shuang Liu, Yan Shen, Jiayi Chen, Zheng Ruan, Li Hua, Kankan Wang, Xiaodong Xi, Jianhua Mao","doi":"10.1016/j.blre.2025.101302","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101302","url":null,"abstract":"Venous thromboembolism (VTE), encompassing deep vein thrombosis (DVT) and pulmonary embolism (PE), is a complex vascular disorder with high morbidity and mortality, driven by Virchow's Triad: blood stasis, hypercoagulability, and endothelial injury. VTE is now recognized as an inflammatory process involving multiple components. Platelets are involved in the process of VTE, contributing to thrombosis initiation, progression, resolution and recurrence through coagulation activation, and interactions with immune and endothelial cells. Anticoagulation remains the cornerstone of VTE treatment; however, antiplatelet agents like aspirin have demonstrated therapeutic potential, particularly following major orthopedic surgeries. Furthermore, emerging platelet-targeted therapies and biomarkers offer new opportunities for improving VTE diagnosis and treatment. This review explores the evolving role of platelets in VTE pathophysiology, assesses current antiplatelet strategies, and highlights novel therapeutic approaches. Advancing platelet research in VTE may lead to safer, more effective interventions, optimizing outcomes for patients with this life-threatening condition.","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101302"},"PeriodicalIF":6.9,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144096012","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Navigating the dynamic landscape of lower-risk MDS: Advances and emerging insights. 导航低风险MDS的动态景观：进展和新兴见解。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-05-14 DOI: 10.1016/j.blre.2025.101301

Alain Mina, Yazan Madanat, Yasmin Abaza, Amer M Zeidan

{"title":"Navigating the dynamic landscape of lower-risk MDS: Advances and emerging insights.","authors":"Alain Mina, Yazan Madanat, Yasmin Abaza, Amer M Zeidan","doi":"10.1016/j.blre.2025.101301","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101301","url":null,"abstract":"Myelodysplastic syndromes/neoplasms (MDS) are a group of clonal myeloid malignancies characterized by ineffective hematopoiesis, cytopenias, and an increased risk of transformation to acute myeloid leukemia (AML). In lower-risk (LR) MDS, as defined by the revised and molecular international prognostic scoring systems (IPSS-R and IPSS-M), anemia is often the predominant clinical manifestation. Treatment strategies have traditionally focused on supportive care, including transfusion support and erythropoiesis stimulating agents (ESAs). While allogeneic hematopoietic stem cell transplantation remains the only potentially curative option for select patients, LR-MDS remain otherwise incurable with current therapies. With the exception of lenalidomide which was approved in 2005 in USA, therapeutic advancements in LR-MDS have stalled for almost 15 years. Progress has been limited by the disease's inherent complexity, indolent nature, and significant heterogeneity, as well as challenges in clinical trial design and execution. Recent advances in gene sequencing and molecular analyses have significantly increased our understanding of disease biology. These insights, coupled with collaborative efforts across the academic community, have led to meaningful shifts in classification, prognostication, and response assessment paradigms in LR-MDS. This evolution has led to a number of approvals, including luspatercept approved in 2020, and imetelstat, which was approved in 2024 in USA. As the therapeutic landscape of LR-MDS continues to evolve, there is growing optimism that these recent milestones will pave the way for further advancements and improved patient outcomes. Next set of studies should focus on the optimal sequencing and combinations of existing agents, as well as moving forward novel effective agents.","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101301"},"PeriodicalIF":6.9,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144227776","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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 new era of primary immune thrombocytopenia management in adults: A narrative review of current and emerging treatments. 成人原发性免疫性血小板减少症管理的新时代：对当前和新兴治疗方法的叙述回顾。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-05-09 DOI: 10.1016/j.blre.2025.101300

Tomás José González-López, Drew Provan

引用次数: 0

Suppression to removal, an emerging therapeutic approach for AL amyloidosis: A comprehensive review with early human data and pharmacokinetics of CAEL-101 antibody. 抑制去除，AL淀粉样变性的一种新兴治疗方法：早期人类数据和CAEL-101抗体药代动力学的综合综述

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-05-09 DOI: 10.1016/j.blre.2025.101299

Anam Ashfaque, Yara Shatnawi, Shahzad Raza, Diana Basali, Jack Khouri, Louis Williams, Sandra Mazzoni, Christy Samaras, Hamza Hassan, Utkarsh Acharya, Chakra Chaulagain, Faiz Anwer

{"title":"Suppression to removal, an emerging therapeutic approach for AL amyloidosis: A comprehensive review with early human data and pharmacokinetics of CAEL-101 antibody.","authors":"Anam Ashfaque, Yara Shatnawi, Shahzad Raza, Diana Basali, Jack Khouri, Louis Williams, Sandra Mazzoni, Christy Samaras, Hamza Hassan, Utkarsh Acharya, Chakra Chaulagain, Faiz Anwer","doi":"10.1016/j.blre.2025.101299","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101299","url":null,"abstract":"Light chain (AL amyloidosis) is a rare disorder characterized by the deposition of misfolded light chains in various organs, causing progressive organ damage. Current therapeutic agents do not remove amyloid aggregates already present in the organs, which are the major determinants of morbidity and mortality. Therefore, drugs targeting amyloid fibrils are currently being investigated. This article provides a comprehensive review of a novel, fibril-directed antibody, CAEL-101 (Anselamimab), which removes fibrillary aggregates from the organs, restoring organ function. Overall, CAEL-101 has demonstrated a favorable toxicity profile and improved organ responses, with faster treatment response time than current therapies in phase I/II trials, and is expected to have promising outcomes in the ongoing phase III studies. Combining anti-plasma cell dyscrasia (suppression) and fibril-directed agents (removal) is a novel therapeutic approach, providing optimism for organ function recovery and enhanced quality of life and survival, especially in patients with severe diseases.","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101299"},"PeriodicalIF":6.9,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144096009","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies. 镰状细胞病新药物治疗临床结果的证据和差距：一项系统的文献综述，突出临床试验和现实世界研究的见解。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-04-25 DOI: 10.1016/j.blre.2025.101298

Mohamed Yassin, Caterina Minniti, Nirmish Shah, Salam Alkindi, Fateen Ata, Mohammed Qari, Abdullah Al Zayed, Jaffer Altooq, Mona Al Rasheed, Maria Domenica Capellini

{"title":"Evidence and gaps in clinical outcomes of novel pharmacologic therapies for sickle cell disease: A systematic literature review highlighting insights from clinical trials and real-world studies.","authors":"Mohamed Yassin, Caterina Minniti, Nirmish Shah, Salam Alkindi, Fateen Ata, Mohammed Qari, Abdullah Al Zayed, Jaffer Altooq, Mona Al Rasheed, Maria Domenica Capellini","doi":"10.1016/j.blre.2025.101298","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101298","url":null,"abstract":"This systematic review aims to summarise the clinical outcomes of l-glutamine, crizanlizumab, and voxelotor in the treatment of sickle cell disease (SCD) based on clinical trials and real-world data and to identify any gaps in the observations. The review identified 97 studies reporting data until 31 May 2024. A pivotal phase III study of l-glutamine showed that patients treated with l-glutamine had a 25 % reduction in pain crises and 33 % fewer hospital days compared to placebo. l-glutamine was generally well tolerated with minimal side effects. Real-world studies of l-glutamine emphasize patient adherence and obstacles to medication accessibility and approval as key concerns. In the SUSTAIN study, a 5-mg/kg dose of crizanlizumab reduced the occurrence of vaso-occlusive crises (VOCs) and hospitalizations by 45 % and 41 %, respectively. Real-world studies of crizanlizumab showed a reduction in complicated VOC events. The high discontinuation rate and results of the STAND trial led to a significant decrease in the use of crizanlizumab. The HOPE trial demonstrated a 51 % improvement in hemoglobin response and a reduction in hemolytic markers in patients treated with voxelotor. While some real-world studies have reported a decrease in VOCs and hospitalizations, the results are inconsistent and not conclusive. Further studies are needed to assess the impact of these novel therapies on end-organ-specific complications of SCD.","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101298"},"PeriodicalIF":6.9,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143998774","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Transplant-acquired allergy in HCT-recipients: Reference for clinical management. hct受者移植获得性过敏：临床管理参考。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-04-11 DOI: 10.1016/j.blre.2025.101289

Akihiro Ohmoto, Yoshiyuki Yamada, Shigeo Fuji

引用次数: 0

Practical guidance on the prevention and management of infection in multiple myeloma patients: A case-based approach 预防和管理多发性骨髓瘤患者感染的实用指南：基于病例的方法。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-04-11 DOI: 10.1016/j.blre.2025.101287

A. Kannan , K. Jeffrey , S. Misbah , K. Ramasamy

引用次数: 0

Navigating acute myeloid leukemia towards better outcomes: Treatment pathways and challenges for patients ineligible for intensive chemotherapy. 引导急性髓系白血病走向更好的结局：不适合强化化疗的患者的治疗途径和挑战。

IF 6.9 2区医学

Blood Reviews Pub Date : 2025-04-09 DOI: 10.1016/j.blre.2025.101288

Raffaele Palmieri, Anna Candoni, Francesco Di Raimondo, Giuseppe Rossi, Massimo Breccia, Fabrizio Pane, Paola Volpicelli, Benedetta Neri, Paola Finsinger, Morena Caira, Felicetto Ferrara

{"title":"Navigating acute myeloid leukemia towards better outcomes: Treatment pathways and challenges for patients ineligible for intensive chemotherapy.","authors":"Raffaele Palmieri, Anna Candoni, Francesco Di Raimondo, Giuseppe Rossi, Massimo Breccia, Fabrizio Pane, Paola Volpicelli, Benedetta Neri, Paola Finsinger, Morena Caira, Felicetto Ferrara","doi":"10.1016/j.blre.2025.101288","DOIUrl":"https://doi.org/10.1016/j.blre.2025.101288","url":null,"abstract":"Acute myeloid leukemia (AML) is an aggressive hematologic malignancy that affects primarily older individuals. Patients ineligible to receive intensive standard chemotherapy followed by consolidation with/without hematopoietic stem cell transplant have a suboptimal prognosis. In recent years, significant advances have been made in the AML field leading to the development of new anti-leukemic approaches, including lower-intensity therapies specifically developed for patients who are ineligible for intensive chemotherapy. As the available options for this hard-to-manage and historically undertreated patient category are increasing, selecting the best treatment for each patient is crucial and ever more challenging. Accordingly, accurate patient evaluation is required to guide this decision-making process. There is currently no consensus on how to evaluate patients' fitness status, and the available tools that were originally developed for this purpose might not be adequate in the setting of the new treatment options. In this review we describe current management of AML patients unfit for intensive chemotherapy, aiming to highlight current challenges and suggest possible strategies for an accurate therapeutic selection. For this purpose, we will first provide an overview of epidemiology and classification of AML, and then move to current anti-leukemic treatments for unfit patients and the tools used for evaluating patient eligibility for a specific treatment. Finally, we will suggest possible measures to improve the management of AML patients in the era of novel lower-intensity regimens.","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":" ","pages":"101288"},"PeriodicalIF":6.9,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065222","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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