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Best Practice & Research Clinical Haematology最新文献

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Principles of cost-effectiveness studies and their use in haematology 成本效益研究的原则及其在血液学中的应用
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-03-01 DOI: 10.1016/j.beha.2023.101441
Isabelle Durand-Zaleski
{"title":"Principles of cost-effectiveness studies and their use in haematology","authors":"Isabelle Durand-Zaleski","doi":"10.1016/j.beha.2023.101441","DOIUrl":"10.1016/j.beha.2023.101441","url":null,"abstract":"<div><p>Health economics is about providing the population with the maximum health possible under budget constraint. The most common method to present the result of an economic evaluation is the calculation of the incremental cost-effectiveness ratio (ICER). It is defined by the difference in cost between two possible technologies, divided by the difference in their effect. It represents the amount of money required to gain one additional unit of health for the population. Economic evaluations are based upon 1) medical evidence of the health benefits of technologies and 2) the value of resources used to achieve these health benefits. An economic evaluation is one type of information that can be used by policy makers, in combination with data on organisation, financing, and incentives to decide on the adoption of innovative technologies.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 1","pages":"Article 101441"},"PeriodicalIF":2.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9182469","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 lymphoblastic lymphoma in children, adolescents and young adults 儿童、青少年和年轻人淋巴母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 : 2023-03-01 DOI: 10.1016/j.beha.2023.101449
William C. Temple , Stephanie Mueller , Michelle L. Hermiston , Birgit Burkhardt
{"title":"Diagnosis and management of lymphoblastic lymphoma in children, adolescents and young adults","authors":"William C. Temple ,&nbsp;Stephanie Mueller ,&nbsp;Michelle L. Hermiston ,&nbsp;Birgit Burkhardt","doi":"10.1016/j.beha.2023.101449","DOIUrl":"10.1016/j.beha.2023.101449","url":null,"abstract":"<div><p>Lymphoblastic lymphoma (LBL) is the second most common type of non-Hodgkin Lymphoma (NHL) in children, adolescents, and young adults (CAYA), accounting for 25–35% of all cases. T-lymphoblastic lymphoma (T-LBL) comprises 70–80% of cases, while precursor B-lymphoblastic lymphoma (pB-LBL) makes up the remaining 20–25% of cases. Event-free and overall survival (EFS and OS) for paediatric LBL patients both exceed 80% with current therapies. Treatment regimens, especially in T-LBL with large mediastinal tumours, are complex with significant toxicity and long-term complications. Though prognosis overall is good for T-LBL and pB-LBL with upfront therapy, outcomes for patients with relapsed or refractory (r/r) disease remain dismal. Here, we review new understanding about the pathogenesis and biology of LBL, recent clinical results and future directions for therapy, and remaining obstacles to improve outcomes while reducing toxicity.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 1","pages":"Article 101449"},"PeriodicalIF":2.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9175048","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 post-transplant lymphoproliferative disease following solid organ transplantation in children, adolescents, and young adults 儿童、青少年和年轻人实体器官移植后淋巴细胞增生性疾病的诊断和治疗
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-03-01 DOI: 10.1016/j.beha.2023.101446
Jeremy Rubinstein , Keri Toner , Thomas Gross , Birte Wistinghausen
{"title":"Diagnosis and management of post-transplant lymphoproliferative disease following solid organ transplantation in children, adolescents, and young adults","authors":"Jeremy Rubinstein ,&nbsp;Keri Toner ,&nbsp;Thomas Gross ,&nbsp;Birte Wistinghausen","doi":"10.1016/j.beha.2023.101446","DOIUrl":"10.1016/j.beha.2023.101446","url":null,"abstract":"<div><p>Post-transplant Lymphoproliferative Disease (PTLD) remains a major complication of solid organ transplantation (SOT) in pediatric patients. The majority are Epstein-Barr Virus (EBV) driven CD20<sup>+</sup> B-cell proliferations responsive to reduction to immunosuppression and anti-CD20 directed immunotherapy. This review focusses on the epidemiology, role of EBV, clinical presentation, current treatment strategies, adoptive immunotherapy and future research in EBV + PTLD in pediatric patients.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 1","pages":"Article 101446"},"PeriodicalIF":2.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9167733","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: 4
Diagnosis and management of rare paediatric Non-Hodgkin lymphoma 罕见儿童非霍奇金淋巴瘤的诊断和治疗
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2023-03-01 DOI: 10.1016/j.beha.2023.101440
Ana C. Xavier , Ritsuro Suzuki , Andishe Attarbaschi
{"title":"Diagnosis and management of rare paediatric Non-Hodgkin lymphoma","authors":"Ana C. Xavier ,&nbsp;Ritsuro Suzuki ,&nbsp;Andishe Attarbaschi","doi":"10.1016/j.beha.2023.101440","DOIUrl":"10.1016/j.beha.2023.101440","url":null,"abstract":"<div><p>Mature B-cell lymphomas, (B- or T-cell) lymphoblastic lymphomas (LBL), and anaplastic large cell lymphoma (ALCL) correspond to about 90% of all non-Hodgkin lymphoma (NHL) cases occurring in children and adolescents. The remaining 10% encompass a complex group of entities characterized by low/very low incidences, paucity of knowledge in terms of underlying biology in comparison to their adult counterparts, and consequent lack of standardization of care, information on clinical therapeutic efficacy and long-term survival. At the Seventh International Symposium on Childhood, Adolescent and Young Adult NHL, organized on October 20–23, 2022, in New York City, New York, US, we had the opportunity to discuss clinical, pathogenetic, diagnostic, and treatment aspects of certain subtypes of rare B- or T-cell NHL and they will be the topic of this review.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 1","pages":"Article 101440"},"PeriodicalIF":2.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9175040","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: 2
Improving clinical trials in higher-risk myelodysplastic syndromes 改善高危骨髓增生异常综合征的临床试验
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2022-12-01 DOI: 10.1016/j.beha.2022.101406
Mikkael A. Sekeres
{"title":"Improving clinical trials in higher-risk myelodysplastic syndromes","authors":"Mikkael A. Sekeres","doi":"10.1016/j.beha.2022.101406","DOIUrl":"10.1016/j.beha.2022.101406","url":null,"abstract":"<div><p><span>Patients with higher-risk myelodysplastic syndromes (HR-MDS) have poor survival and are in need of more effective therapy options. Hypomethylating agents<span> (HMAs) are the current standard of care and are being studied in combination with a number of novel therapies. Recent evidence, however, has delivered sub-optimal results, prompting the need to revisit patient selection criteria, treatment schedules, and </span></span>clinical endpoints to better inform future studies and steer endpoints towards those that are clinically meaningful to patients.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"35 4","pages":"Article 101406"},"PeriodicalIF":2.1,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10762650","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
Should all CAR-T therapy for acute lymphoblastic leukemia Be consolidated with allogeneic stem cell transplant? 急性淋巴细胞白血病的CAR-T治疗是否应该与异体干细胞移植相结合?
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2022-12-01 DOI: 10.1016/j.beha.2022.101414
Alejandro Marinos, Helen E. Heslop
{"title":"Should all CAR-T therapy for acute lymphoblastic leukemia Be consolidated with allogeneic stem cell transplant?","authors":"Alejandro Marinos,&nbsp;Helen E. Heslop","doi":"10.1016/j.beha.2022.101414","DOIUrl":"10.1016/j.beha.2022.101414","url":null,"abstract":"<div><p>Autologous T cells<span><span> genetically modified with a CD19<span> chimeric antigen receptor<span> are an effective therapy for children and adults with relapsed or refractory acute lymphoblastic leukemia with initial response rates ranging from 70 to 85%. Unfortunately, about half of these responding patients will subsequently relapse raising the question of whether allogeneic hemopoietic </span></span></span>stem cell transplant should be considered as a consolidative therapy. Currently efforts are focused on defining risk factors for relapse to try and develop algorithms predicting which patients may benefit from allogenic transplant.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"35 4","pages":"Article 101414"},"PeriodicalIF":2.1,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10762653","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: 2
Measurable residual disease in acute lymphoblastic leukemia: How low is low enough? 急性淋巴细胞白血病可测量残留病:多低才算足够低?
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2022-12-01 DOI: 10.1016/j.beha.2022.101407
Aaron C. Logan
{"title":"Measurable residual disease in acute lymphoblastic leukemia: How low is low enough?","authors":"Aaron C. Logan","doi":"10.1016/j.beha.2022.101407","DOIUrl":"10.1016/j.beha.2022.101407","url":null,"abstract":"<div><p>Quantification of measurable residual disease (MRD) in acute lymphoblastic leukemia (ALL) is a well-established clinical tool used to risk stratify patients during the course of chemotherapy, immunotherapy, and/or transplant therapy. As technologies evolve, the sensitivity for quantifying exceptionally low disease burden using either next generation sequencing (NGS) or next generation flow cytometry (NGF) has improved. It is now possible to detect MRD and quantify it precisely in patients who would previously have been deemed MRD negative by older, lower sensitivity methods. Persistence or recurrence of ALL disease burden above 10<sup>−4</sup> (0.01%) is accepted as the minimum threshold for making clinical decisions, but with NGS and NGF, clinicians now confront decision-making with disease burdens sometimes quantified to as low as 10<sup>−6</sup> (0.0001%, or one leukemia cell in a million leukocytes). Emerging data suggest these higher sensitivity methods are superior for identifying patients at lowest risk for relapse, but it remains controversial whether to institute therapies such as blinatumomab or chimeric antigen receptor (CAR)-T cells or move patients to allogeneic hematopoietic cell transplant (alloHCT) when they have quantifiable disease burden less than 10<sup>−4</sup>. With additional evidence to facilitate integration of highly sensitive MRD quantification into clinical care and to contextualize MRD within the genotype of individual patients, it will likely be increasingly possible to identify patients able to avoid alloHCT and potentially even de-escalate therapy.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"35 4","pages":"Article 101407"},"PeriodicalIF":2.1,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1521692622000627/pdfft?md5=3eedb7a2c99ca8220459819175d732fb&pid=1-s2.0-S1521692622000627-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10473776","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: 1
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 : 2022-12-01 DOI: 10.1016/S1521-6926(22)00087-1
{"title":"Editorial Board / Aims & Scope","authors":"","doi":"10.1016/S1521-6926(22)00087-1","DOIUrl":"https://doi.org/10.1016/S1521-6926(22)00087-1","url":null,"abstract":"","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"35 4","pages":"Article 101432"},"PeriodicalIF":2.1,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1521692622000871/pdfft?md5=7beefdc458583f541031540b0148b0d4&pid=1-s2.0-S1521692622000871-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138224977","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
Acute GVHD: New approaches to clinical trial monitoring 急性GVHD:临床试验监测的新方法
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2022-12-01 DOI: 10.1016/j.beha.2022.101400
Nikolaos Spyrou, John E. Levine, James L.M. Ferrara
{"title":"Acute GVHD: New approaches to clinical trial monitoring","authors":"Nikolaos Spyrou,&nbsp;John E. Levine,&nbsp;James L.M. Ferrara","doi":"10.1016/j.beha.2022.101400","DOIUrl":"10.1016/j.beha.2022.101400","url":null,"abstract":"<div><p><span>Acute GVHD occurs in nearly 50% of patients receiving hematopoietic </span>cell transplantation<span><span><span> (HCT), and is the major driver of mortality. However, progress in the development of new acute GVHD therapeutics has been slow, in part due to heterogeneity in acute GVHD data collection and interpretation among centers. Herein, we first describe the methods used by the Mount Sinai Acute GVHD International Consortium (MAGIC) to standardize acute GVHD data collection and curation. We then review the utility of serum biomarkers, specifically the MAGIC Algorithm Probability (MAP) that combines two GI biomarkers (ST2 and REG3α) that has been shown to be more accurate than changes in clinical symptom severity after GVHD </span>treatment. We then present preliminary data on the feasibility of a surrogate </span>clinical trial endpoint that combines clinical response and MAP two weeks after treatment. This novel endpoint is an earlier and potentially better predictor of non-relapse mortality than the current gold standard of clinical response four weeks after treatment.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"35 4","pages":"Article 101400"},"PeriodicalIF":2.1,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9818618","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: 2
When will chemotherapy be replaced in upfront induction therapy for adult acute lymphoblastic leukemia (ALL)? 成人急性淋巴细胞白血病(ALL)的前期诱导治疗何时可以替代化疗?
IF 2.1 4区 医学
Best Practice & Research Clinical Haematology Pub Date : 2022-12-01 DOI: 10.1016/j.beha.2022.101404
Anjali Advani
{"title":"When will chemotherapy be replaced in upfront induction therapy for adult acute lymphoblastic leukemia (ALL)?","authors":"Anjali Advani","doi":"10.1016/j.beha.2022.101404","DOIUrl":"10.1016/j.beha.2022.101404","url":null,"abstract":"<div><p><span><span>The treatment of </span>acute lymphoblastic leukemia (ALL) has changed significantly over the last decade. With the approval of novel antibody based therapies in the relapsed/refractory setting, many of these agents are starting to be used in the upfront setting in </span>clinical trials<span> for older patients. These results have been impressive, and further trials are underway. Other targeted therapies (i.e. BCL inhibitors) are being explored. This article will discuss the incorporation of novel agents to “replace” chemotherapy in induction.</span></p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"35 4","pages":"Article 101404"},"PeriodicalIF":2.1,"publicationDate":"2022-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10473773","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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