{"title":"Transplantation and cellular therapy in acute leukemia","authors":"Michael Grunwald, Belinda Avalos, 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}
{"title":"The analysis of multiple outcomes, multiple variables and variables selection in hematopoietic cell transplantation studies","authors":"Soyoung Kim , Xi Fang , 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}
{"title":"Hodgkin lymphoma: Focus on evolving treatment paradigms","authors":"Joseph L. Roswarski , Dan L. Longo","doi":"10.1016/j.beha.2023.101510","DOIUrl":"https://doi.org/10.1016/j.beha.2023.101510","url":null,"abstract":"<div><p><span><span>Hodgkin lymphoma (HL) is a highly curable B-cell </span>malignancy<span> of germinal center origin. Biologically it is a </span></span>hematologic malignancy<span> that is highly dependent on the immune microenvironment and utilizes immune escape through upregulation of the programmed-death ligands on the neoplastic cells. Despite being highly curable, consensus is lacking nationally and internationally about the optimal approach to management, particularly in limited-stage disease. The addition of brentuximab vedotin<span> and checkpoint inhibitors for the management of HL has led to a rapidly changing treatment landscape. Further studies should be done to include these novel agents at all stages of disease to determine improvements in frontline cure rates and long-term toxicity.</span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 4","pages":"Article 101510"},"PeriodicalIF":2.1,"publicationDate":"2023-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91987019","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}
{"title":"Cytogenetics and genomics in pediatric acute lymphoblastic leukaemia","authors":"Željko Antić , Jana Lentes , Anke K. Bergmann","doi":"10.1016/j.beha.2023.101511","DOIUrl":"https://doi.org/10.1016/j.beha.2023.101511","url":null,"abstract":"<div><p><span><span><span>The last five decades have witnessed significant improvement in diagnostics, treatment and management of children with </span>acute lymphoblastic leukaemia<span> (ALL). These advancements have become possible through progress in our understanding of the genetic and biological background of ALL, resulting in the introduction of risk-adapted treatment and novel therapeutic targets, e.g., </span></span>tyrosine kinase inhibitors for </span><em>BCR::ABL1</em><span>-positive ALL. Further advances in the taxonomy of ALL and the discovery of new genetic biomarkers and therapeutic targets, as well as the introduction of targeted and immunotherapies into the frontline treatment protocols, may improve management and outcome of children with ALL. In this review we describe the current developments in the (cyto)genetic diagnostics and management of children with ALL, and provide an overview of the most important advances in the genetic classification of ALL. Furthermore, we discuss perspectives resulting from the development of new techniques, including artificial intelligence (AI).</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 4","pages":"Article 101511"},"PeriodicalIF":2.1,"publicationDate":"2023-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49716024","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}
Yi Ning , Yanming Zhang , Michael A. Kallen , Ashkan Emadi , Maria R. Baer
{"title":"Cytogenetics and molecular genetics of myelodysplastic neoplasms","authors":"Yi Ning , Yanming Zhang , Michael A. Kallen , Ashkan Emadi , 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}
{"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}
{"title":"Immunopathogenic mechanisms and modulatory approaches to graft-versus-host disease prevention in acute myeloid leukaemia","authors":"Yifan Pang , 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}
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 , Jaime M. Preussler , Christa L. Meyer , Katie Schoeppner , Jennifer A. Sees Coles , Amber Ruffin , Meggan McCann , Steven M. Devine , 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}
{"title":"Diagnosis and management of mature B-cell lymphomas in children, adolescents, and young adults","authors":"Stanton Goldman , 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}