Best Practice & Research Clinical Haematology最新文献

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Immunopathogenic mechanisms and modulatory approaches to graft-versus-host disease prevention in acute myeloid leukaemia 急性髓性白血病移植物抗宿主病预防的免疫致病机制和调节途径
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101475
Yifan Pang , Noa G. Holtzman
{"title":"Immunopathogenic mechanisms and modulatory approaches to graft-versus-host disease prevention in acute myeloid leukaemia","authors":"Yifan Pang ,&nbsp;Noa G. Holtzman","doi":"10.1016/j.beha.2023.101475","DOIUrl":"10.1016/j.beha.2023.101475","url":null,"abstract":"<div><p><span>Allogeneic haematopoietic stem cell transplantation<span> (HSCT) remains the only potential cure for intermediate to high-risk acute myeloid leukaemia<span><span> (AML). The therapeutic effect of HSCT is largely dependent on the powerful donor-derived immune response against recipient </span>leukaemia cells, known as graft-versus-leukaemia effect (GvL). However, the donor-derived immune system can also cause acute or chronic damage to normal recipient organs and tissues, in a process known as graft-versus-host disease (GvHD). GvHD is a leading cause of non-relapse mortality in HSCT recipients. There are many similarities and cross talk between the immune pathways of GvL and GvHD. Studies have demonstrated that both processes require the presence of mismatched </span></span></span>alloantigens<span> between the donor and recipient, and activation of immune responses<span> centered around donor T-cells, which can be further modulated by various recipient or donor factors. Dissecting GvL from GvHD to achieve more effective GvHD prevention and enhanced GvL has been the holy grail of HSCT research. In this review, we focused on the key factors that contribute to the immune responses of GvL and GvHD, the effect on GvL with different GvHD prophylactic strategies, and the potential impact of various AML relapse prevention therapy or treatments on GvHD.</span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101475"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10291443/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9767736","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
Reducing barriers of access and care related to hematopoietic cell transplantation and cellular therapy: The mission-driven role of the national marrow donor program 减少与造血细胞移植和细胞治疗相关的获取和护理障碍:国家骨髓捐献者计划的使命驱动作用
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101480
Rafeek A. Yusuf , Jaime M. Preussler , Christa L. Meyer , Katie Schoeppner , Jennifer A. Sees Coles , Amber Ruffin , Meggan McCann , Steven M. Devine , Jeffery J. Auletta
{"title":"Reducing barriers of access and care related to hematopoietic cell transplantation and cellular therapy: The mission-driven role of the national marrow donor program","authors":"Rafeek A. Yusuf ,&nbsp;Jaime M. Preussler ,&nbsp;Christa L. Meyer ,&nbsp;Katie Schoeppner ,&nbsp;Jennifer A. Sees Coles ,&nbsp;Amber Ruffin ,&nbsp;Meggan McCann ,&nbsp;Steven M. Devine ,&nbsp;Jeffery J. Auletta","doi":"10.1016/j.beha.2023.101480","DOIUrl":"10.1016/j.beha.2023.101480","url":null,"abstract":"<div><p><span>The treatment of malignant and nonmalignant hematologic disorders continues to benefit from significant scientific advancement and progress in the use of hematopoietic </span>cell transplantation and cellular therapies. However, barriers associated with receiving these lifesaving treatments and care remain, which necessitate innovative approaches to overcome, so all persons in need can receive these therapies. This article reviews barriers to receiving hematopoietic cell transplantation and cellular therapies, and highlights novel approaches taken by the National Marrow Donor Program in reducing barriers for all patients in need.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101480"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9676580","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: 1
Diagnosis and management of mature B-cell lymphomas in children, adolescents, and young adults 儿童、青少年和年轻人成熟b细胞淋巴瘤的诊断和治疗
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101463
Stanton Goldman , Mitchell S. Cairo
{"title":"Diagnosis and management of mature B-cell lymphomas in children, adolescents, and young adults","authors":"Stanton Goldman ,&nbsp;Mitchell S. Cairo","doi":"10.1016/j.beha.2023.101463","DOIUrl":"10.1016/j.beha.2023.101463","url":null,"abstract":"<div><p><span><span>Mature B-cell lymphoma in children, adolescents and young adults comprises three major histological subtypes including in order of frequency Burkitt, germinal center diffuse large B-cell lymphoma and primary mediastinal B-cell lymphoma. The cure rate of the first two with aggressive short chemotherapy based on clinical grouping is ∼90% in resource rich countries. Recent data has shown that incorporation of </span>immune therapy has enhanced </span>event free survival<span> in advanced patients. Future studies will address the possibility of reducing the burden of chemotherapy by substitution of immune based therapies.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101463"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9676579","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 编委会/目标和范围
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/S1521-6926(23)00052-X
{"title":"Editorial Board / Aims & Scope","authors":"","doi":"10.1016/S1521-6926(23)00052-X","DOIUrl":"https://doi.org/10.1016/S1521-6926(23)00052-X","url":null,"abstract":"","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101491"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49812628","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 state of the science” of childhood, adolescent and young adult Non Hodgkin Lymphoma (NHL) 儿童、青少年和青年非霍奇金淋巴瘤(NHL)的“科学现状”
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101482
Mitchell S. Cairo
{"title":"“The state of the science” of childhood, adolescent and young adult Non Hodgkin Lymphoma (NHL)","authors":"Mitchell S. Cairo","doi":"10.1016/j.beha.2023.101482","DOIUrl":"10.1016/j.beha.2023.101482","url":null,"abstract":"","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101482"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9683344","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
Designing and conducting a clinical trial in blood and marrow transplantation 设计并进行血液和骨髓移植的临床试验
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101471
Michael J. Martens , Yan Gao , Aniko Szabo
{"title":"Designing and conducting a clinical trial in blood and marrow transplantation","authors":"Michael J. Martens ,&nbsp;Yan Gao ,&nbsp;Aniko Szabo","doi":"10.1016/j.beha.2023.101471","DOIUrl":"10.1016/j.beha.2023.101471","url":null,"abstract":"<div><p>Clinical trials<span> form the cornerstone of the science-based approach to improving patient outcomes. A trial needs to be designed and performed carefully to provide valid evidence to inform medical science and to protect the safety and well-being of its participants. The development of a clinical trial involving blood and marrow transplant (BMT) requires special considerations, including the rare disease populations involved and transplant-specific outcomes of interest that necessitate appropriate analysis techniques to evaluate.</span></p><p>This article reviews key considerations and best practices for the design and conduct of a clinical trial in BMT, including the selection of patient population, treatment groups, objectives and endpoints, targeted sample size, statistical analysis strategy, provisions for monitoring patient safety and trial progress, and dissemination of trial results. The practical application of these principles is demonstrated using BMT CTN 1301, a recently completed clinical trial evaluating regimens for chronic graft-versus-host disease prevention in transplant patients.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101471"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9683350","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
What should be done and what should be avoided when comparing two treatments? 在比较两种治疗方法时,应该做什么,应该避免什么?
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101473
Florie Brion Bouvier , Raphaël Porcher
{"title":"What should be done and what should be avoided when comparing two treatments?","authors":"Florie Brion Bouvier ,&nbsp;Raphaël Porcher","doi":"10.1016/j.beha.2023.101473","DOIUrl":"10.1016/j.beha.2023.101473","url":null,"abstract":"<div><p><span>The preferred approach to compare two treatments is a </span>randomized controlled trial (RCT). Indeed, randomization ensures that the groups compared are similar. Well-designed and well-conducted RCTs thus allow to draw causal conclusions on the relative efficacy and safety of treatments compared. However, it is not always possible to conduct RCTs for all clinical questions of interest, and observational data may also be used to infer on the relative effectiveness of treatments. In this review, we present different approaches that allow statistically valid comparisons of the effectiveness of treatments using observational data under some assumptions. Those are based on regression modelling or the propensity score. We also present the principles of target trial emulation.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101473"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9683351","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
Financial toxicity in patients with leukemia undergoing hematopoietic stem cell transplantation: A systematic review 接受造血干细胞移植的白血病患者的财务毒性:一项系统综述
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101469
Orrin Pail , Thomas G. Knight
{"title":"Financial toxicity in patients with leukemia undergoing hematopoietic stem cell transplantation: A systematic review","authors":"Orrin Pail ,&nbsp;Thomas G. Knight","doi":"10.1016/j.beha.2023.101469","DOIUrl":"10.1016/j.beha.2023.101469","url":null,"abstract":"<div><p>Financial toxicity (FT) is a term used to describe the objective financial burden of cancer care including the associated coping behaviors used by patients and their caregivers. FT has been shown to result in both direct financial burdens and in clinically relevant outcomes, such as non-adherence with care, diminished quality of life<span><span>, and even decreased overall survival. Much of the data has been described in solid tumors<span>, with limited investigations in the malignant hematology population. Patients with </span></span>hematologic malignancies<span><span> undergoing hematopoietic stem cell transplantation (HSCT) face a unique financial burden driven by lengthy hospitalizations and acute and chronic morbidity that have downstream implications on their income and costs. In this review, we discuss the prevalence of FT </span>in patients with leukemia who are eligible for HSCT. We review the impact of FT on financial and clinical outcomes and the role of various interventions that have been studied within this population.</span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101469"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9683348","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: 1
Handling missing covariate data in clinical studies in haematology 处理血液学临床研究中缺失的协变量数据
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101477
Edouard F. Bonneville , Johannes Schetelig , Hein Putter , Liesbeth C. de Wreede
{"title":"Handling missing covariate data in clinical studies in haematology","authors":"Edouard F. Bonneville ,&nbsp;Johannes Schetelig ,&nbsp;Hein Putter ,&nbsp;Liesbeth C. de Wreede","doi":"10.1016/j.beha.2023.101477","DOIUrl":"10.1016/j.beha.2023.101477","url":null,"abstract":"<div><p>Missing data are frequently encountered across studies in clinical haematology. Failure to handle these missing values in an appropriate manner can complicate the interpretation of a study's findings, as estimates presented may be biased and/or imprecise. In the present work, we first provide an overview of current methods for handling missing covariate data, along with their advantages and disadvantages. Furthermore, a systematic review is presented, exploring both contemporary reporting of missing values in major haematological journals, and the methods used for handling them. A principal finding was that the method of handling missing data was explicitly specified in a minority of articles (in 76 out of 195 articles reporting missing values, 39%). Among these, complete case analysis and the missing indicator method were the most common approaches to dealing with missing values, with more complex methods such as multiple imputation being extremely rare (in 7 out of 195 articles). An example analysis (with associated code) is also provided using hematopoietic stem cell transplantation data, illustrating the different approaches to handling missing values. We conclude with various recommendations regarding the reporting and handling of missing values for future studies in clinical haematology.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101477"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9688815","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
Cardio-oncology and transplantation for acute myeloid leukemia 急性髓性白血病的心脏肿瘤学和移植
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-06-01 DOI: 10.1016/j.beha.2023.101465
Jai Singh
{"title":"Cardio-oncology and transplantation for acute myeloid leukemia","authors":"Jai Singh","doi":"10.1016/j.beha.2023.101465","DOIUrl":"10.1016/j.beha.2023.101465","url":null,"abstract":"<div><p><span><span>Despite the rapidly evolving treatment landscape for </span>acute myeloid leukemia (AML), allogeneic hematopoietic </span>cell transplantation<span><span> (allo-HCT) remains an important and potentially curative treatment option for many high-risk AML patients. Cardiovascular disease is an important competing risk throughout allo-HCT and a key driver of morbidity and mortality long after treatment. Cardio-oncology is a new discipline in cardiology<span> which provides multidisciplinary care and expertise to complex cancer patients with the aims of optimizing cardiovascular health plus monitoring and treating potential cardiotoxicity related to cancer treatments. As allogeneic HCT techniques get more sophisticated there will be an increase in transplant eligible older patients with a rise in comorbidities including established cardiovascular disease highlighting the need for close collaboration with cardio-oncology specialists from the time of diagnosis through late </span></span>survivorship.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101465"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9683343","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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