{"title":"Diagnosis and management of cutaneous lymphomas and lymphoid proliferations in children, adolescents and young adults (CAYA)","authors":"Jamie Stokke , Annabelle Tann , Nader Kim El-Mallawany , Ilske Oschlies","doi":"10.1016/j.beha.2023.101448","DOIUrl":"10.1016/j.beha.2023.101448","url":null,"abstract":"<div><p>Cutaneous lymphomas and lymphoid proliferations (LPD) in children, adolescents, and young adults (CAYA) are a heterogeneous group of lymphoid neoplasms that present formidable diagnostic challenges to clinicians and pathologists alike. Although rare overall, cutaneous lymphomas/LPD occur in real-world settings and awareness of the differential diagnosis, potential complications, and various therapeutic approaches will help ensure the optimal diagnostic work-up and clinical management. Lymphomas/LPD involving the skin can occur as primary cutaneous disease in a patient that characteristically has lymphoma/LPD confined to the skin, or as secondary involvement in patients with systemic disease. This review will comprehensively summarize both primary cutaneous lymphomas/LPD that occur in the CAYA population as well as those CAYA systemic lymphomas/LPD with propensity for secondary cutaneous involvement. Focus on the most common primary entities occurring in CAYA will include lymphomatoid papulosis, primary cutaneous anaplastic large cell lymphoma, mycosis fungoides, subcutaneous panniculitis-like T-cell lymphoma, and hydroa vacciniforme lymphoproliferative disorder.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 1","pages":"Article 101448"},"PeriodicalIF":2.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9175043","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":"Biopathology of childhood, adolescent and young adult non-Hodgkin lymphoma","authors":"Megan S. Lim , Michelle Foley , Lara Mussolin , Reiner Siebert , Suzanne Turner","doi":"10.1016/j.beha.2023.101447","DOIUrl":"10.1016/j.beha.2023.101447","url":null,"abstract":"<div><p>Mature non-Hodgkin lymphomas (NHL) in the childhood, adolescent and young adult (CAYA) population are rare and exhibit unique clinical, immunophenotypic and genetic characteristics. Application of large-scale unbiased genomic and proteomic technologies such as gene expression profiling and next generation sequencing (NGS) have led to enhanced understanding of the genetic basis for many lymphomas in adults. However, studies to investigate the pathogenetic events in CAYA population are relatively sparse. Enhanced understanding of the pathobiologic mechanisms involved in non-Hodgkin lymphomas in this unique population will allow for improved recognition of these rare lymphomas. Elucidation of the pathobiologic differences between CAYA and adult lymphomas will also lead to the design of more rational and much needed, less toxic therapies for this population. In this review, we summarize recent insights gained from the proceedings of the recent 7th International CAYA NHL Symposium held in New York City, New York October 20–23, 2022.</p></div>","PeriodicalId":8744,"journal":{"name":"Best Practice & Research Clinical Haematology","volume":"36 1","pages":"Article 101447"},"PeriodicalIF":2.1,"publicationDate":"2023-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9175044","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":"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}
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 , Stephanie Mueller , Michelle L. Hermiston , 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}
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 , Keri Toner , Thomas Gross , 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}
Ana C. Xavier , Ritsuro Suzuki , Andishe Attarbaschi
{"title":"Diagnosis and management of rare paediatric Non-Hodgkin lymphoma","authors":"Ana C. Xavier , Ritsuro Suzuki , 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}
{"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}
{"title":"Should all CAR-T therapy for acute lymphoblastic leukemia Be consolidated with allogeneic stem cell transplant?","authors":"Alejandro Marinos, 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}
{"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}