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Best Practice & Research Clinical Haematology最新文献

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Graft versus Leukemia in 2023 2023年移植物对抗白血病
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-06-10 DOI: 10.1016/j.beha.2023.101476
Zane Chiad, Aleksander Chojecki
{"title":"Graft versus Leukemia in 2023","authors":"Zane Chiad,&nbsp;Aleksander Chojecki","doi":"10.1016/j.beha.2023.101476","DOIUrl":"10.1016/j.beha.2023.101476","url":null,"abstract":"<div><p>Allogeneic hematopoietic stem cell transplantation<span><span> (HSCT) is commonly utilized in the management of leukemia across multiple subtypes. Graft versus leukemia<span> (GVL) is a critical component of successful transplantation and involves donor cells eradicating residual leukemia within the recipient. Graft versus host disease (GVHD) by contrast is a common complication of the transplantation process in which donor cells identify the recipient's various organ systems as foreign, thereby leading to a multitude of </span></span>organ toxicities that can be described as autoimmune in nature. As both GVL and GVHD are mediated by a similar mechanism, these processes are felt to occur in tandem with one another. Here, we review the allogeneic HCT process in the context of GVL.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101476"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10458628","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
Editorial Board / Aims & Scope 编辑委员会/目标与范围
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-08-21 DOI: 10.1016/S1521-6926(23)00065-8
{"title":"Editorial Board / Aims & Scope","authors":"","doi":"10.1016/S1521-6926(23)00065-8","DOIUrl":"https://doi.org/10.1016/S1521-6926(23)00065-8","url":null,"abstract":"","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101504"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49771236","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
Targeted cellular therapy for treatment of relapsed or refractory leukemia 靶向细胞疗法治疗复发或难治性白血病
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-05-25 DOI: 10.1016/j.beha.2023.101481
Lauren D. Scherer , Rayne H. Rouce
{"title":"Targeted cellular therapy for treatment of relapsed or refractory leukemia","authors":"Lauren D. Scherer ,&nbsp;Rayne H. Rouce","doi":"10.1016/j.beha.2023.101481","DOIUrl":"10.1016/j.beha.2023.101481","url":null,"abstract":"<div><p><span>While the mainstay of treatment for high-risk or relapsed, refractory leukemia has historically revolved around allogeneic </span>hematopoietic stem cell<span><span><span> transplant (allo-HSCT), targeted immunotherapies have emerged as a promising therapeutic option, especially given the poor prognosis of patients who relapse after allo-HSCT. Novel cellular immunotherapies that harness the cytotoxic abilities of the immune system in a targeted manner (often called “adoptive” cell therapy), have changed the way we treat r/r </span>hematologic malignancies and continue to change the treatment landscape given the rapid evolution of these powerful, yet sophisticated precision therapies that often offer a less toxic alternative to conventional </span>salvage therapies<span><span>. Importantly, adoptive cell therapy can be allo-HSCT-enabling or a therapeutic option for patients in whom transplantation has failed or is contraindicated. A solid understanding of the core concepts of adoptive cell therapy is necessary for stem cell transplant physicians, nurses and ancillary staff given its proximity to the transplant field as well as its inherent complexities that require specific expertise in compliant manufacturing, clinical application, and risk mitigation. Here we will review use of targeted cellular therapy for the treatment of r/r leukemia, focusing on </span>chimeric antigen receptor T-cells (CAR T-cells) given the remarkable sustained clinical responses leading to commercial approval for several hematologic indications including leukemia, with brief discussion of other promising investigational cellular immunotherapies and special considerations for sustainability and scalability.</span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101481"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10458631","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
Describing and analyzing complex disease history in retrospective studies 在回顾性研究中描述和分析复杂的病史
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-06-10 DOI: 10.1016/j.beha.2023.101483
Ariane Boumendil , Myriam Labopin
{"title":"Describing and analyzing complex disease history in retrospective studies","authors":"Ariane Boumendil ,&nbsp;Myriam Labopin","doi":"10.1016/j.beha.2023.101483","DOIUrl":"10.1016/j.beha.2023.101483","url":null,"abstract":"<div><p><span>Blood-related diseases<span><span> are complex diseases with diverse origins, treatments and prognosis. In </span>haematology studies, investigators are interested in multiple outcomes and multiple prognostic variables that may change value over the course of follow-up. These time-dependent variables can be of different nature. Time-dependent events such as treatment with haematopoeitic </span></span>stem cell transplant<span> (HCT) and acute or chronic graft-versus-host disease (GVHD) typically interact with outcomes respectively after diagnosis or HCT. Longitudinal measurement such as immune response do influence survival after HCT. Effect of these time-dependent variables on outcomes can be investigated using different approaches, such as time-dependent Cox regression, landmark analysis, multi-state models or joint modelisation. In this paper we review basic principles of these different approaches using examples from haematological studies.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101483"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10458633","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
Preface of special edition “Biostatistics in clinical haematology” 《临床血液学生物统计学》特刊前言
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-06-08 DOI: 10.1016/j.beha.2023.101486
Myriam Labopin, Ariane Boumendil
{"title":"Preface of special edition “Biostatistics in clinical haematology”","authors":"Myriam Labopin,&nbsp;Ariane Boumendil","doi":"10.1016/j.beha.2023.101486","DOIUrl":"10.1016/j.beha.2023.101486","url":null,"abstract":"","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101486"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10101340","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 current landscape: Allogeneic hematopoietic stem cell transplant for acute lymphoblastic leukemia 目前的情况:同种异体造血干细胞移植治疗急性淋巴细胞白血病
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-06-04 DOI: 10.1016/j.beha.2023.101485
Susan Prockop, Franziska Wachter
{"title":"The current landscape: Allogeneic hematopoietic stem cell transplant for acute lymphoblastic leukemia","authors":"Susan Prockop,&nbsp;Franziska Wachter","doi":"10.1016/j.beha.2023.101485","DOIUrl":"10.1016/j.beha.2023.101485","url":null,"abstract":"<div><p><span>One of the consistent features in development of hematopoietic stem cell transplant<span> (HCT) for Acute Lymphoblastic Leukemia (ALL) is the rapidity with which discoveries in the laboratory are translated into innovations in clinical care. Just a few years after murine studies demonstrated that rescue from radiation induced marrow failure is mediated by cellular not </span></span>humoral factors<span><span><span><span>, E. Donnall Thomas reported on the transfer of bone marrow cells into irradiated leukemia patients. This was followed quickly by the first descriptions of Graft versus Leukemia (GvL) effect and </span>Graft versus Host Disease (GvHD). Despite the pivotal nature of these findings, early human transplants were uniformly unsuccessful and identified the challenges that continue to thwart transplanters today – leukemic relapse, regimen related toxicity, and GvHD. While originally only an option for young, fit patients with a matched family donor, expansion of the donor pool to include unrelated donors, umbilical cord blood units, and more recently the growing use of haploidentical donors have all made transplant a more accessible therapy for patients with ALL. Novel agents for conditioning, prevention and </span>treatment of GvHD have improved outcomes and investigators continue to develop novel treatment strategies that balance regimen related toxicity with disease control. Our evolving understanding of how to prevent and treat GvHD and how to prevent relapse are incorporated into novel </span>clinical trials that are expected to further improve outcomes.</span></p><p>Here we review current considerations and future directions for both adult and pediatric patients undergoing HCT for ALL, including indication for transplant, donor selection, cytoreductive regimens, and outcomes.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101485"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10458625","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
Transplantation and cellular therapy in acute leukemia 急性白血病的移植和细胞治疗
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-06-22 DOI: 10.1016/j.beha.2023.101499
Michael Grunwald, Belinda Avalos, Edward Copelan
{"title":"Transplantation and cellular therapy in acute leukemia","authors":"Michael Grunwald,&nbsp;Belinda Avalos,&nbsp;Edward Copelan","doi":"10.1016/j.beha.2023.101499","DOIUrl":"10.1016/j.beha.2023.101499","url":null,"abstract":"","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101499"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10458632","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 analysis of multiple outcomes, multiple variables and variables selection in hematopoietic cell transplantation studies 造血细胞移植研究的多结局、多变量及变量选择分析
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-09-01 Epub Date: 2023-05-24 DOI: 10.1016/j.beha.2023.101478
Soyoung Kim , Xi Fang , Kwang Woo Ahn
{"title":"The analysis of multiple outcomes, multiple variables and variables selection in hematopoietic cell transplantation studies","authors":"Soyoung Kim ,&nbsp;Xi Fang ,&nbsp;Kwang Woo Ahn","doi":"10.1016/j.beha.2023.101478","DOIUrl":"10.1016/j.beha.2023.101478","url":null,"abstract":"<div><p>It is common to study time-to-event data in cancer research such as hematopoietic cell transplantation (HCT) for leukemia. The extensive work has been done for the univariate survival outcome, that is, one event type. However, in practice a subject is often exposed to multiple types of outcomes. In this article, we review various types of right-censored data with multiple outcome types including competing risks data, recurrent event data, and composite endpoints. We also provide hematopoietic cell transplantation data examples.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 3","pages":"Article 101478"},"PeriodicalIF":2.1,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10447944/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10477485","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cytogenetics and molecular genetics of myelodysplastic neoplasms 骨髓增生异常肿瘤的细胞遗传学和分子遗传学
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-08-16 DOI: 10.1016/j.beha.2023.101512
Yi Ning , Yanming Zhang , Michael A. Kallen , Ashkan Emadi , Maria R. Baer
{"title":"Cytogenetics and molecular genetics of myelodysplastic neoplasms","authors":"Yi Ning ,&nbsp;Yanming Zhang ,&nbsp;Michael A. Kallen ,&nbsp;Ashkan Emadi ,&nbsp;Maria R. Baer","doi":"10.1016/j.beha.2023.101512","DOIUrl":"https://doi.org/10.1016/j.beha.2023.101512","url":null,"abstract":"<div><p>According to the 2022 World Health Organization (WHO) Classification (5th edition), the term myelodysplastic neoplasms (abbreviated MDS) has been introduced to replace myelodysplastic syndromes. MDS are a group of clonal hematopoietic stem cell<span><span> diseases<span> characterized by cytopenia(s), dysplasia<span><span> in one or more of lineages, ineffective hematopoiesis, and an increased risk of progression to </span>bone marrow failure or to </span></span></span>acute myeloid leukemia<span><span> (AML). Current NCCN guidelines and recent review articles have provided in depth discussion on the clinical diagnosis and management of MDS. This review will focus on discussion of the WHO and International Consensus Classification (ICC) updates on the role of cytogenetics and molecular genetics in the diagnosis and </span>risk stratification of MDS.</span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 4","pages":"Article 101512"},"PeriodicalIF":2.1,"publicationDate":"2023-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49734338","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 presentation of results from studies in clinical haematology 临床血液学研究结果的介绍
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 Epub Date: 2023-05-06 DOI: 10.1016/j.beha.2023.101472
Richard M. Szydlo
{"title":"The presentation of results from studies in clinical haematology","authors":"Richard M. Szydlo","doi":"10.1016/j.beha.2023.101472","DOIUrl":"10.1016/j.beha.2023.101472","url":null,"abstract":"<div><p>Research is based on trying to find answers to specific questions or to test hypotheses. Studies are thus undertaken to generate data which, with appropriate statistical methods, will help to determine the validity of the science under investigation. The aim of this paper is not to provide answers on which statistical methods to use, but will concentrate on suggesting the best ways of presenting the results of appropriately analysed data. And presentation is the key, because however well conducted and analysed a study may be, incorrect or inappropriate presentation of the findings will severely hamper its publication potential. With illustrative examples, the fundamentals required in the presentation of study objectives, population selection, description of characteristics and missing values, survival analyses, unadjusted analyses, multivariate regression models and matched pair analyses, are presented.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101472"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9676581","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
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