Blood Reviews最新文献

{"title":"The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators","authors":"Tine M.H.J. Goedhart ,&nbsp;A. Janssen ,&nbsp;Ron A.A. Mathôt ,&nbsp;Marjon H. Cnossen ,&nbsp;for the OPTI-CLOT Study Group and SYMPHONY Consortium","doi":"10.1016/j.blre.2023.101098","DOIUrl":"10.1016/j.blre.2023.101098","url":null,"abstract":"<div><p>Clinical guidelines and expert groups recommend the use of pharmacokinetic (PK)-guided dosing of factor replacement therapy for the treatment of bleeding disorders, especially for patients with hemophilia. Although PK-guided dosing is increasingly applied, it is generally not considered standard clinical practice. The aim of this scoping review is to map barriers and facilitators for the implementation of PK-guided dosing in clinical practice and to identify knowledge gaps. A literature search was performed and 110 articles were included that describe PK-guided dosing in patients with bleeding disorders, mostly hemophilia A. We defined two overarching themes, efficacy and feasibility, and discuss five topics within each theme. For each topic, barriers, facilitators and knowledge gaps were described. Although consensus was found with regard to some topics, contradicting reports were found for others, especially with respect to the efficacy of PK-guided dosing. These contradictions highlight the need for future research to elucidate current ambiguities.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10001449","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}

{"title":"Diamond-Blackfan anemia in adults: In pursuit of a common approach for a rare disease","authors":"Deena Iskander ,&nbsp;Noémi B.A. Roy ,&nbsp;Elspeth Payne ,&nbsp;Emma Drasar ,&nbsp;Kelly Hennessy ,&nbsp;Yvonne Harrington ,&nbsp;Chrysi Christodoulidou ,&nbsp;Anastasios Karadimitris ,&nbsp;Leisa Batkin ,&nbsp;Josu de la Fuente","doi":"10.1016/j.blre.2023.101097","DOIUrl":"10.1016/j.blre.2023.101097","url":null,"abstract":"<div><p><span>Diamond-Blackfan anemia (DBA) is a rare bone marrow failure syndrome, usually caused by loss-of function variants in genes encoding </span>ribosomal proteins<span>. The hallmarks of DBA are anemia, congenital anomalies<span> and cancer predisposition. Although DBA usually presents in childhood, the prevalence in later life is increasing due to an expanding repertoire of implicated genes, improvements in genetic<span> diagnosis and increasing life expectancy. Adult patients uniquely suffer the manifestations of end-organ damage caused by the disease and its treatment, and transition to adulthood poses specific issues in disease management. To standardize and optimize care for this rare disease, in this review we provide updated guidance on the diagnosis and management of DBA, with a specific focus on older adolescents and adults. Recommendations are based upon published literature and our pooled clinical experience from three centres in the United Kingdom (U·K.). Uniquely we have also solicited and incorporated the views of affected families, represented by the independent patient organization, DBA U.K.</span></span></span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10058328","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}

{"title":"Cardiovascular and haematological pathology in myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS): A role for viruses","authors":"Jean M. Nunes ,&nbsp;Douglas B. Kell ,&nbsp;Etheresia Pretorius","doi":"10.1016/j.blre.2023.101075","DOIUrl":"10.1016/j.blre.2023.101075","url":null,"abstract":"<div><p>ME/CFS is a debilitating chronic condition that often develops after viral or bacterial infection. Insight from the study of Long COVID/Post Acute Sequelae of COVID-19 (PASC), the post-viral syndrome associated with SARS-CoV-2 infection, might prove to be useful for understanding pathophysiological mechanisms of ME/CFS. Disease presentation is similar between the two conditions, and a subset of Long COVID patients meet the diagnostic criteria for ME/CFS. Since Long COVID is characterized by significant vascular pathology – including endothelial dysfunction, coagulopathy, and vascular dysregulation – the question of whether or not the same biological abnormalities are of significance in ME/CFS arises. Cardiac abnormalities have for a while now been documented in ME/CFS cohorts, with recent studies demonstrating major deficits in cerebral blood flow, and hence vascular dysregulation. A growing body of research is demonstrating that ME/CFS is accompanied by platelet hyperactivation, anomalous clotting, a procoagulant phenotype, and endothelial dysfunction. Endothelial damage and dysregulated clotting can impair substance exchange between blood and tissues, and result in hypoperfusion, which may contribute to the manifestation of certain ME/CFS symptoms. Here we review the ME/CFS literature to summarize cardiovascular and haematological findings documented in patients with the condition, and, in this context, briefly discuss the potential role of previously-implicated pathogens. Overall, cardiac and haematological abnormalities are present within ME/CFS cohorts. While atherosclerotic heart disease is not significantly associated with ME/CFS, suboptimal cardiovascular function defined by reduced cardiac output, impaired cerebral blood flow, and vascular dysregulation are, and these abnormalities do not appear to be influenced by deconditioning. Rather, these cardiac abnormalities may result from dysfunction in the (autonomic) nervous system. Plenty of recently published studies are demonstrating significant platelet hyperactivity and endothelial dysfunction in ME/CFS, as well as anomalous clotting processes. It is of particular importance to determine to what extent these cardiovascular and haematological abnormalities contribute to symptom severity, and if these two systems can be targeted for therapeutic purposes. Viral reservoirs of herpesviruses exist in ME/CFS, and most likely contribute to cardiovascular and haematological dysfunction directly or indirectly. This review highlights the potential of studying cardiac functioning, the vasculature, and coagulation system in ME/CFS.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10027292/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9733455","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS)","authors":"Jan Philipp Bewersdorf ,&nbsp;Zhuoer Xie ,&nbsp;Rafael Bejar ,&nbsp;Uma Borate ,&nbsp;Jacqueline Boultwood ,&nbsp;Andrew M. Brunner ,&nbsp;Rena Buckstein ,&nbsp;Hetty E. Carraway ,&nbsp;Jane E. Churpek ,&nbsp;Naval G. Daver ,&nbsp;Matteo Giovanni Della Porta ,&nbsp;Amy E. DeZern ,&nbsp;Pierre Fenaux ,&nbsp;Maria E. Figueroa ,&nbsp;Steven D. Gore ,&nbsp;Elizabeth A. Griffiths ,&nbsp;Stephanie Halene ,&nbsp;Robert P. Hasserjian ,&nbsp;Christopher S. Hourigan ,&nbsp;Tae Kon Kim ,&nbsp;Amer M. Zeidan","doi":"10.1016/j.blre.2023.101072","DOIUrl":"https://doi.org/10.1016/j.blre.2023.101072","url":null,"abstract":"<div><p><span><span>Biological events that contribute to the pathogenesis of myelodysplastic syndromes/neoplasms (MDS) are becoming increasingly characterized and are being translated into rationally designed therapeutic strategies. Herein, we provide updates from the first International Workshop on MDS (iwMDS) of the International Consortium for MDS (icMDS) detailing recent advances in understanding the genetic landscape of MDS, including </span>germline<span> predisposition, epigenetic<span><span> and immune dysregulation<span>, the complexities of clonal hematopoiesis progression to MDS, as well as novel </span></span>animal models<span><span> of the disease. Connected to this progress is the development of novel therapies targeting specific molecular alterations, the innate immune system, and </span>immune checkpoint inhibitors. While some of these agents have entered </span></span></span></span>clinical trials (e.g., splicing modulators, IRAK1/4 inhibitors, anti-CD47 and anti-TIM3 antibodies, and cellular therapies), none have been approved for MDS. Additional preclinical and clinical work is needed to develop a truly individualized approach to the care of MDS patients.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49857534","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}

{"title":"How do we improve the translation of new evidence into the practice of hematopoietic cell transplantation and cellular therapy?","authors":"Mark Juckett ,&nbsp;Christopher Dandoy ,&nbsp;Zachariah DeFilipp ,&nbsp;Tamila L. Kindwall-Keller ,&nbsp;Stephen R. Spellman ,&nbsp;Celalettin Ustun ,&nbsp;Bryce M. Waldman ,&nbsp;Daniel J. Weisdorf ,&nbsp;William A. Wood ,&nbsp;Mary M. Horowitz ,&nbsp;Linda J. Burns ,&nbsp;Nandita Khera","doi":"10.1016/j.blre.2023.101079","DOIUrl":"10.1016/j.blre.2023.101079","url":null,"abstract":"<div><p>The field of hematopoietic cell transplantation<span><span><span> and cell therapy (HCT/CT) is advancing rapidly to bring an ever-expanding collection of potentially curative therapies to patients with malignant and non-malignant </span>diseases<span>. The impact of these therapies depends on our ability to implement them as new evidence becomes available to advance the quality of care. There is often a long delay between evidence development and adoption of therapies based on that evidence into clinical practice. In this review, we describe the potential factors based on an implementation framework that could act as facilitators or barriers to adoption of therapies in the context of HCT/CT. We highlight two examples, the first to showcase the efforts to improve the efficiency of adoption of new findings and accelerate improvement in care of HCT/CT patients and the second to discuss the challenges in real world implementation of chimeric antigen receptor </span></span>T cell therapy. We conclude by reviewing strategies to improve translation of evidence and ways to measure their success.</span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10330269/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9763283","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prospects of early therapeutic interventions for indolent adult T-cell leukemia/lymphoma based on the chronic lymphocytic leukemia progression model","authors":"Akihiro Ohmoto ,&nbsp;Shigeo Fuji","doi":"10.1016/j.blre.2023.101057","DOIUrl":"10.1016/j.blre.2023.101057","url":null,"abstract":"<div><p><span>Adult T-cell leukemia/lymphoma (ATLL) has aggressive clinical behaviors, and improving its prognosis is a great challenge. A disease progression model from asymptomatic human T-cell leukemia virus type 1 carrier to aggressive-type ATLL has been proposed, and indolent ATLL comprising a smoldering or favorable chronic type is located at the midpoint. Even the most favorable smoldering type has a 4-year overall survival rate of &lt;60%. Although watchful waiting is pervasive </span>in patients<span> with indolent ATLL, early therapeutic intervention is discussed among hematologists. Indolent ATLL was once termed T-cell-derived chronic lymphocytic leukemia<span> (CLL). Unlike indolent ATLL, several molecular-targeted agents at the initial treatment have dramatically improved CLL prognosis. Recent studies on CLL have revealed a similar progression model involving premalignant monoclonal B-cell lymphocytosis (MBL). In particular, individuals with high-count MBL have an increased lymphoma risk. Considering the unsatisfactory long-term prognosis of indolent ATLL, further treatment strategies, including precision medicine, are warranted.</span></span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10092372","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}

{"title":"Beyond current treatment of Fanconi Anemia: What do advances in cell and gene-based approaches offer?","authors":"Elena Martínez-Balsalobre ,&nbsp;Jean-Hugues Guervilly ,&nbsp;Jenny van Asbeck-van der Wijst ,&nbsp;Ana Belén Pérez-Oliva ,&nbsp;Christophe Lachaud","doi":"10.1016/j.blre.2023.101094","DOIUrl":"10.1016/j.blre.2023.101094","url":null,"abstract":"<div><p><span>Fanconi anemia<span><span><span> (FA) is a rare inherited disorder that mainly affects the bone marrow. This condition causes decreased production of all types of blood cells. FA is caused by a defective repair of DNA interstrand crosslinks and to date, mutations in over 20 genes have been linked to the disease. Advances in science and </span>molecular biology have provided new insight between FA </span>gene mutations<span> and the severity of clinical manifestations. Here, we will highlight the current and promising therapeutic options for this rare disease. The current standard treatment for FA patients is </span></span></span>hematopoietic stem cell<span><span><span> transplantation, a treatment associated to exposure to radiation or chemotherapy, immunological complications, plus opportunistic infections from prolonged immune incompetence or increased risk of morbidity. New arising treatments include gene addition therapy, </span>genome editing<span> using CRISPR-Cas9 nuclease, and hematopoietic stem cell generation from </span></span>induced pluripotent stem cells. Finally, we will also discuss the revolutionary developments in mRNA therapeutics as an opportunity for this disease.</span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9737220","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}

{"title":"Advances in NK cell therapy for hematologic malignancies: NK source, persistence and tumor targeting.","authors":"Aimee Merino,&nbsp;Joseph Maakaron,&nbsp;Veronika Bachanova","doi":"10.1016/j.blre.2023.101073","DOIUrl":"10.1016/j.blre.2023.101073","url":null,"abstract":"<div><p><span><span>Natural Killer (NK) cells yield promise in therapy of hematologic malignancies. The clinical experience with adoptively transferred allogeneic NK cells over past two decades has revealed safety and minimal risk of </span>CRS<span> or ICANS. Unlike </span></span>T cells<span><span> which have to be genetically altered to avoid graft vs host disease (GVHD), HLA mismatched NK cells can be infused without GVHD risk. This makes them ideal for the development of off-the-shelf products. In this review we focus on NK biology relevant to the cancer therapy, the trajectory of NK therapeutics for leukemia, lymphoma, and </span>myeloma<span>; and advantages of the NK cell platform. We will also discuss novel methods to enhance NK cell targeting, persistence, and function in the tumor microenvironment. The future of NK cell therapy depends on novel strategies to realize these qualities.</span></span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10109725","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}

{"title":"Nodal peripheral T-cell lymphoma: Chemotherapy-free management, are we there yet?","authors":"Sharina C. Macapagal ,&nbsp;N. Nora Bennani","doi":"10.1016/j.blre.2023.101071","DOIUrl":"10.1016/j.blre.2023.101071","url":null,"abstract":"<div><p><span>Peripheral T-cell lymphomas (PTCLs) are a diverse and uncommon type of lymphoid malignancies with a dismal prognosis. Recent advances in genomic studies have shown recurring mutations that are changing our knowledge of the disease's molecular genetics<span><span> and pathogenesis. As such, new targeted therapies and treatments to improve disease outcomes are currently being explored. In this review, we discussed the current understanding of the nodal PTCL biology with potential therapeutic implications and gave our insights on the promising novel therapies that are currently under study such as </span>immunotherapy, </span></span>chimeric antigen receptor<span> T-cell therapy, and oncolytic virotherapy.</span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9736115","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}

{"title":"‘Secondary’ acute lymphoblastic/lymphocytic leukemia - done playing second fiddle?","authors":"Yosef Joseph Rene Amel Riazat-Kesh ,&nbsp;John Mascarenhas ,&nbsp;Michal Bar-Natan","doi":"10.1016/j.blre.2023.101070","DOIUrl":"10.1016/j.blre.2023.101070","url":null,"abstract":"<div><p>Acute lymphoblastic/lymphocytic leukemia (ALL) occurring post-cancer diagnosis (secondary ALL - sALL) is increasingly recognized as a discrete entity, constituting up to as much as 5–10% of all new ALL diagnoses, and carrying its own biologic, prognostic and therapeutic significance.</p><p>In this review, we will outline the history and current state of research into sALL. We will explore the evidence for differences underlining its existence as a distinct subgroup, as well as examining what might be driving such differences etiologically, including prior chemotherapy. We will examine these distinctions on population-, chromosomal-, and molecular-levels, and we will consider whether they translate to differences in clinical outcome, and whether they do - or should - warrant differences in treatment selection.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9788331","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}