{"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}
{"title":"Designing and conducting a clinical trial in blood and marrow transplantation","authors":"Michael J. Martens , Yan Gao , 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}
{"title":"What should be done and what should be avoided when comparing two treatments?","authors":"Florie Brion Bouvier , 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}
{"title":"Financial toxicity in patients with leukemia undergoing hematopoietic stem cell transplantation: A systematic review","authors":"Orrin Pail , 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}
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 , Johannes Schetelig , Hein Putter , 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}
{"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}
{"title":"Take a spin: Apheresis in the care of adult leukaemia patients","authors":"Jing Ai","doi":"10.1016/j.beha.2023.101467","DOIUrl":"10.1016/j.beha.2023.101467","url":null,"abstract":"<div><p><span><span><span>Apheresis is an automated process to separate the whole blood of a patient or a donor, collect or remove specific blood components, and return the remaining back to the individual. Apheresis is an integral part of blood and marrow transplantation and has been increasingly utilized in novel cellular therapies for a variety of </span>blood disorders<span><span>. This review uses clinical cases to highlight the multiple roles of apheresis in the care of adult leukaemia patients, including therapeutic leukapheresis in hyperleukocytosis, mobilized peripheral blood hematopoietic progenitor cell collection in donors, mononucleated cell collection in preparation of </span>donor lymphocyte infusion or </span></span>chimeric antigen receptor </span>T cells<span> manufacture, and extracorporeal photopheresis<span> in the treatment<span> of graft versus host diseases.</span></span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101467"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9683353","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}
Zoë C. Wong , Laura W. Dillon , Christopher S. Hourigan
{"title":"Measurable residual disease in patients undergoing allogeneic transplant for acute myeloid leukemia","authors":"Zoë C. Wong , Laura W. Dillon , Christopher S. Hourigan","doi":"10.1016/j.beha.2023.101468","DOIUrl":"10.1016/j.beha.2023.101468","url":null,"abstract":"<div><p><span>The most common indication for allogeneic hematopoietic cell<span> transplant (alloHCT) is maintenance of remission after initial treatment for patients with </span></span>acute myeloid leukemia<span><span> (AML). Loss of remission, relapse, remains however the most frequent cause of alloHCT failure. There is strong evidence that detectable persistent disease burden (“measurable residual disease”, MRD) </span>in patients<span> with AML in remission prior to alloHCT is associated with increased risk of post-transplant relapse. MRD status as a summative assessment of response to pre-transplant therapy may allow superior patient-personalized risk stratification compared with models solely incorporating pre-treatment variables. An optimal methodology for AML MRD detection has not yet been established, but molecular methods such as DNA-sequencing may have additional prognostic utility compared to current approaches. There is growing evidence that intervention on AML MRD positivity may improve post-transplant outcomes. New initiatives will generate actionable data on the clinical utility of AML MRD testing for patients undergoing alloHCT.</span></span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101468"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10291441/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9767733","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":"Allogeneic hematopoietic cell transplantation in acute myeloid leukemia","authors":"Gray Magee, Brittany Knick Ragon","doi":"10.1016/j.beha.2023.101466","DOIUrl":"10.1016/j.beha.2023.101466","url":null,"abstract":"<div><p>Allogeneic hematopoietic cell transplantation<span><span> (HCT) is a curative treatment modality for select patients with </span>acute myeloid leukemia (AML), functioning as a restorative agent following intensified chemo- and/or radiotherapy and also engendering the disease-directed immunologic threat of graft-versus-leukemia effect. Advancements in conditioning regimen intensity, donor availability, and supportive care have broadened the eligibility for allogeneic HCT, reduced rates of transplant related mortality, and improved outcomes over time. There are still obstacles to transplant in AML, offering opportunities for ongoing discovery, including poor recipient fitness, insufficient donor availability for certain populations, and limited access to care. Relapse remains the most common cause of treatment failure and a high priority area of investigative efforts. Post-transplant maintenance and novel applications of cellular therapeutics are expected to usher in a new era of promise for successful HCT in AML and will aim to overcome the remaining barriers impeding favorable outcomes for these patients.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101466"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9688814","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":"Dermatologic complications in transplantation and cellular therapy for acute leukemia","authors":"Shahab Babakoohi , Stephanie L. Gu , Hamid Ehsan , Alina Markova","doi":"10.1016/j.beha.2023.101464","DOIUrl":"10.1016/j.beha.2023.101464","url":null,"abstract":"<div><p><span><span>Adoptive cellular immunotherapy, mainly hematopoietic </span>stem cell transplant<span><span> and CAR-T cell therapy have revolutionized treatment of patients with </span>acute leukemia<span><span>. Indications and inclusion criteria for these treatments have expanded in recent years. While these therapies are associated with significant improvements in disease response and overall survival, patients may experience adverse events from associated chemotherapy conditioning, engraftment, </span>cytokine storm<span><span><span>, supportive medications, and post-transplant maintenance targeted therapies. Supportive oncodermatology is a growing specialty to manage </span>cutaneous toxicities resulting from the anti-cancer therapies. In this review, we summarize diagnosis and management of the common cutaneous adverse events including </span>drug eruptions, graft-versus-host disease, neoplastic and paraneoplastic complications </span></span></span></span>in patients undergoing cellular therapies.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 2","pages":"Article 101464"},"PeriodicalIF":2.1,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10291442/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10071511","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}