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Update: The molecular spectrum of virus-associated high-grade B-cell non-Hodgkin lymphomas 最新进展:与病毒相关的高级别 B 细胞非霍奇金淋巴瘤的分子谱
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-20 DOI: 10.1016/j.blre.2024.101172
H. Witte , A. Künstner , N. Gebauer
{"title":"Update: The molecular spectrum of virus-associated high-grade B-cell non-Hodgkin lymphomas","authors":"H. Witte ,&nbsp;A. Künstner ,&nbsp;N. Gebauer","doi":"10.1016/j.blre.2024.101172","DOIUrl":"10.1016/j.blre.2024.101172","url":null,"abstract":"<div><p>The vast spectrum of aggressive B-cell non-Hodgkin neoplasms (B-NHL) encompasses several infrequent entities occurring in association with viral infections, posing diagnostic challenges for practitioners. In the emerging era of precision oncology, the molecular characterization of malignancies has acquired paramount significance. The pathophysiological comprehension of specific entities and the identification of targeted therapeutic options have seen rapid development. However, owing to their rarity, not all entities have undergone exhaustive molecular characterization.</p><p>Considerable heterogeneity exists in the extant body of work, both in terms of employed methodologies and the scale of cases studied. Presently, therapeutic strategies are predominantly derived from observations in diffuse large B-cell lymphoma (DLBCL), the most prevalent subset of aggressive B-NHL. Ongoing investigations into the molecular profiles of these uncommon virus-associated entities are progressively facilitating a clearer distinction from DLBCL, ultimately paving the way towards individualized therapeutic approaches.</p><p>This review consolidates the current molecular insights into aggressive and virus-associated B-NHL, taking into consideration the recently updated 5th edition of the WHO classification of hematolymphoid tumors (WHO-5HAEM) and the International Consensus Classification (ICC). Additionally, potential therapeutically targetable susceptibilities are highlighted, offering a comprehensive overview of the present scientific landscape in the field.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X24000055/pdfft?md5=b6fed54c1013340b4eafb52f8bc52263&pid=1-s2.0-S0268960X24000055-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139509835","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
When anticoagulation management in atrial fibrillation becomes difficult: Focus on chronic kidney disease, coagulation disorders, and cancer 当心房颤动的抗凝管理变得困难时:关注慢性肾病、凝血功能障碍和癌症
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-14 DOI: 10.1016/j.blre.2024.101171
Panteleimon E. Papakonstantinou , Vasiliki Kalogera , Dimitrios Charitos , Dimitrios Polyzos , Dimitra Benia , Athina Batsouli , Konstantinos Lampropoulos , Sotirios Xydonas , Dhiraj Gupta , Gregory Y.H. Lip
{"title":"When anticoagulation management in atrial fibrillation becomes difficult: Focus on chronic kidney disease, coagulation disorders, and cancer","authors":"Panteleimon E. Papakonstantinou ,&nbsp;Vasiliki Kalogera ,&nbsp;Dimitrios Charitos ,&nbsp;Dimitrios Polyzos ,&nbsp;Dimitra Benia ,&nbsp;Athina Batsouli ,&nbsp;Konstantinos Lampropoulos ,&nbsp;Sotirios Xydonas ,&nbsp;Dhiraj Gupta ,&nbsp;Gregory Y.H. Lip","doi":"10.1016/j.blre.2024.101171","DOIUrl":"10.1016/j.blre.2024.101171","url":null,"abstract":"<div><p>Anticoagulation<span><span> therapy (AT) is fundamental in atrial fibrillation<span><span> (AF) treatment but poses challenges in implementation, especially in AF populations with elevated </span>thromboembolic and bleeding risks. Current guidelines emphasize the need to estimate and balance thrombosis and bleeding risks for all potential candidates of </span></span>antithrombotic therapy<span><span>. However, administering oral AT raises concerns in specific populations, such as those with chronic kidney disease (CKD), </span>coagulation disorders<span><span>, and cancer due to lack of robust data. These groups, excluded from large direct oral anticoagulants trials, rely on observational studies, prompting physicians to adopt individualized management strategies based on case-specific evaluations. The scarcity of evidence and specific guidelines underline the need for a tailored approach, emphasizing regular reassessment of risk factors and </span>anticoagulation drug doses. This narrative review aims to summarize evidence and recommendations for challenging AF clinical scenarios, particularly in the long-term management of AT for patients with CKD, coagulation disorders, and cancer.</span></span></span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139469408","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Harnessing multi-source data for individualized care in Hodgkin Lymphoma 利用多源数据为霍奇金淋巴瘤患者提供个性化治疗
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-14 DOI: 10.1016/j.blre.2024.101170
Susan K. Parsons , Angie Mae Rodday , Jenica N. Upshaw , Carlton D. Scharman , Zhu Cui , Yenong Cao , Yun Kyoung Ryu Tiger , Matthew J. Maurer , Andrew M. Evens
{"title":"Harnessing multi-source data for individualized care in Hodgkin Lymphoma","authors":"Susan K. Parsons ,&nbsp;Angie Mae Rodday ,&nbsp;Jenica N. Upshaw ,&nbsp;Carlton D. Scharman ,&nbsp;Zhu Cui ,&nbsp;Yenong Cao ,&nbsp;Yun Kyoung Ryu Tiger ,&nbsp;Matthew J. Maurer ,&nbsp;Andrew M. Evens","doi":"10.1016/j.blre.2024.101170","DOIUrl":"10.1016/j.blre.2024.101170","url":null,"abstract":"<div><p><span>Hodgkin lymphoma is a rare, but highly curative form of cancer, primarily afflicting adolescents and young adults. Despite multiple seminal trials over the past twenty years, there is no single consensus-based </span>treatment approach beyond use of multi-agency chemotherapy with curative intent. The use of radiation continues to be debated in early-stage disease, as part of combined modality treatment, as well as in salvage, as an important form of consolidation. While short-term disease outcomes have varied little across these different approaches across both early and advanced stage disease, the potential risk of severe, longer-term risk has varied considerably.</p><p>Over the past decade novel therapeutics have been employed in the retrieval setting in preparation to and as consolidation after autologous stem cell transplant. More recently, these novel therapeutics have moved to the frontline setting, initially compared to standard-of-care treatment and later in a direct head-to-head comparison combined with multi-agent chemotherapy.</p><p><span>In 2018, we established the HoLISTIC Consortium, bringing together disease and methods experts to develop clinical decision models based on individual patient data to guide providers, patients, and caregivers in decision-making. In this review, we detail the steps we followed to create the master database of individual patient data from patients treated over the past 20 years, using principles of data science. We then describe different methodological approaches we are taking to clinical decision making, beginning with clinical prediction tools at the time of diagnosis, to multi-state models, incorporating treatments and their response. Finally, we describe how simulation modeling can be used to </span><em>estimate</em> risks of late effects, based on cumulative exposure from frontline and salvage treatment.</p><p>The resultant database and tools employed are <em>dynamic</em> with the expectation that they will be updated as better and more complete information becomes available.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139464046","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Disparity in hematological malignancies: From patients to health care professionals 血液恶性肿瘤的差异:从患者到医护人员
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-08 DOI: 10.1016/j.blre.2024.101169
Xiaoyi Chen , Mihir Shukla , Shella Saint Fleur-Lominy
{"title":"Disparity in hematological malignancies: From patients to health care professionals","authors":"Xiaoyi Chen ,&nbsp;Mihir Shukla ,&nbsp;Shella Saint Fleur-Lominy","doi":"10.1016/j.blre.2024.101169","DOIUrl":"10.1016/j.blre.2024.101169","url":null,"abstract":"<div><p><span><span>In the recent few decades, outcomes in patients diagnosed with </span>hematological malignancies<span> have been steadily improving. However, the improved prognosis does not distribute equally among patients from different backgrounds. Besides cancer biology, demographic and geographic disparities have been found to impact </span></span>overall survival<span> significantly. Specifically, patients from underrepresented minorities including Black and Hispanics, and those with uninsured status, having low socioeconomic status, or from rural areas have had worse outcomes historically, which is uniformly true across all major subtypes of hematological malignancies. Similar discrepancy is also seen in the health care professional field, where a gender gap and a disproportionally low representation of health care providers from underrepresented minorities have been long existing. Thus, a comprehensive strategy to mitigate disparity in the health care system is needed to achieve equity in health care.</span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139412089","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
A paradox of choice: Sequencing therapy in relapsed/refractory diffuse large B-cell lymphoma 选择的悖论:复发/难治性弥漫性大B细胞淋巴瘤的测序治疗。
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-01 DOI: 10.1016/j.blre.2023.101140
Taylor R. Brooks , Paolo F. Caimi
{"title":"A paradox of choice: Sequencing therapy in relapsed/refractory diffuse large B-cell lymphoma","authors":"Taylor R. Brooks ,&nbsp;Paolo F. Caimi","doi":"10.1016/j.blre.2023.101140","DOIUrl":"10.1016/j.blre.2023.101140","url":null,"abstract":"<div><p>The available treatments for relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) have experienced a dramatic change since 2017. Incremental advances in basic and translational science over several decades have led to innovations in immune-oncology. These innovations have culminated in eight separate approvals by the US Food and Drug Administration for the treatment of patients with R/R DLBCL over the last 10 years. High-dose therapy and autologous stem cell transplant (HDT-ASCT) remains the standard of care for transplant-eligible patients who relapse after an initial remission. For transplant-ineligible patients or for those who relapse following HDT-ASCT, multiple options exist. Monoclonal antibodies targeting CD19, antibody-drug conjugates, bispecific antibodies, immune effector cell products, and other agents with novel mechanisms of action are now available for patients with R/R DLBCL. There is increasing use of chimeric antigen receptor (CAR) T-cells as second-line therapy for patients with early relapse of DLBCL or those who are refractory to initial chemoimmunotherapy. The clinical benefits of these strategies vary and are influenced by patient and disease characteristics, as well as the type of prior therapy administered. Therefore, there are multiple clinical scenarios that clinicians might encounter when treating R/R DLBCL. An optimal sequence of drugs has not been established, and there is no evidence-based consensus on how to best order these agents. This abundance of choices introduces a paradox: proliferating treatment options are initially a boon to patients and providers, but as choices grow further they no longer liberate. Rather, more choices make the management of R/R DLBCL more challenging due to lack of direct comparisons among agents and a desire to maximize patient outcomes. Here, we provide a review of recently-approved second- and subsequent-line agents, summarize real-world data detailing the use of these medicines, and provide a framework for sequencing therapy in R/R DLBCL.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X23001017/pdfft?md5=017ece5e4ab2e983af224f8bb953f889&pid=1-s2.0-S0268960X23001017-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"72212076","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East 输血依赖性β-地中海贫血(TDT)的治疗:来自中东的专家见解和实践综述。
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-01 DOI: 10.1016/j.blre.2023.101138
Amal El-Beshlawy , Hany Dewedar , Salwa Hindawi , Salam Alkindi , Azza A. Tantawy , Mohamed A. Yassin , Ali T. Taher
{"title":"Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East","authors":"Amal El-Beshlawy ,&nbsp;Hany Dewedar ,&nbsp;Salwa Hindawi ,&nbsp;Salam Alkindi ,&nbsp;Azza A. Tantawy ,&nbsp;Mohamed A. Yassin ,&nbsp;Ali T. Taher","doi":"10.1016/j.blre.2023.101138","DOIUrl":"10.1016/j.blre.2023.101138","url":null,"abstract":"<div><p>β-Thalassemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management and devising solutions to address challenges attributed to resource limitations. The region has also participated in the majority of clinical trials and development programs of iron chelators and more novel ineffective erythropoiesis-targeted therapy. In this review, we provide a practical overview of management for patients with transfusion-dependent β-thalassemia, primarily driven by such experiences, with the aim of transferring knowledge to colleagues in other regions facing similar challenges.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X23000991/pdfft?md5=bbdd1ac89881b56132dc0258a258a5f4&pid=1-s2.0-S0268960X23000991-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49694303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
A personalized, organ-based approach to the treatment of chronic steroid-refractory graft-versus-host disease 治疗慢性类固醇难治性移植物抗宿主病的个性化、基于器官的方法
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-01 DOI: 10.1016/j.blre.2023.101142
Hanaa Fatoum , Robert Zeiser , Shahrukh K. Hashmi
{"title":"A personalized, organ-based approach to the treatment of chronic steroid-refractory graft-versus-host disease","authors":"Hanaa Fatoum ,&nbsp;Robert Zeiser ,&nbsp;Shahrukh K. Hashmi","doi":"10.1016/j.blre.2023.101142","DOIUrl":"10.1016/j.blre.2023.101142","url":null,"abstract":"<div><p>Chronic graft-versus-host-disease (cGvHD) remains the leading cause of morbidity among transplant recipients. The efficacy of second-line treatments varies widely based on many factors, including wide differences in the organ overall response-rate response and in the current era where multiple agents are approved, and optimal sequencing of drugs based on organ ORR is unknown. We aimed to evaluate outcomes based on ORRs to the most common agents for the treatment of steroid-refractory/steroid-dependent cGvHD by conducting a systematic literature review. A total of 387 studies were evaluated for the ORRs of 12 cGvHD treatments. The highest skin ORR was observed to be 77% though some agents had an acceptable ORR. Most agents had an ocular response ranging from 17 to 50% Some agents resulted in a GI ORR of ≥88%. Rituximab showed the best response for musculoskeletal-GvHD. In the case of lung-GvHD (bronchiolitis obliterans syndrome [BOS]), negligible response was observed in patients treated with various agents. No clinically meaningful responses to treatments were reported for genital-GvHD. Most GvHD trials are focused on the ORR and partial response rates (PRR). The evidence for optimal agents for each organ is limited, and therefore, our study results are striking for differences in organ-ORR yields for a clinically meaningful difference. Thus, a personalized organ-based approach to the selection of therapeutic agents in cGvHD could result in favorable outcomes.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X23001030/pdfft?md5=f71104c627612e349c961bc6377df227&pid=1-s2.0-S0268960X23001030-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135671432","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Access to CAR T-cell therapy: Focus on diversity, equity and inclusion 获得CAR T细胞治疗:关注多样性、公平性和包容性。
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-01 DOI: 10.1016/j.blre.2023.101136
Maria S. Odstrcil , Catherine J. Lee , Catherine Sobieski , Daniel Weisdorf , Daniel Couriel
{"title":"Access to CAR T-cell therapy: Focus on diversity, equity and inclusion","authors":"Maria S. Odstrcil ,&nbsp;Catherine J. Lee ,&nbsp;Catherine Sobieski ,&nbsp;Daniel Weisdorf ,&nbsp;Daniel Couriel","doi":"10.1016/j.blre.2023.101136","DOIUrl":"10.1016/j.blre.2023.101136","url":null,"abstract":"<div><p>Chimeric antigen receptor T-cell (CAR T-cell) therapy has revolutionized the treatment of hematologic malignancies in patients with relapsed or refractory disease without other treatment options. However, only a very small proportion of patients with an indication for CAR T-cell can access the treatment. The imbalance between supply and demand is magnified in minority and vulnerable populations. Limited access is multifactorial and in part a result of factors directly related to the cellular product such as cost, complex logistics and manufacturing limitations. On the other hand, the impact of diversity, equity, and inclusion (DEI) and their social and structural context are also key to understanding access barriers in cellular therapy and health care in general. CAR T-cell therapy provides us with a new opportunity to better understand and prioritize this gap, a key step towards proactively and strategically addressing access.</p><p>The aim of this review is to provide an analysis of the current state of access to CAR T therapy with a focus on the influence of DEI. We will cover aspects related to the cellular product and the inseparable context of social and structural determinants. Identifying and addressing barriers is necessary to ensure equitable access to this and all future novel therapies.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X23000978/pdfft?md5=bd93a2af570618aafab41b4f6949eb07&pid=1-s2.0-S0268960X23000978-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49685497","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Optimal management of chemotherapy-induced thrombocytopenia with thrombopoietin receptor agonists 血小板生成素受体激动剂对化疗诱导的血小板减少症的最佳治疗。
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-01 DOI: 10.1016/j.blre.2023.101139
Hanny Al-Samkari
{"title":"Optimal management of chemotherapy-induced thrombocytopenia with thrombopoietin receptor agonists","authors":"Hanny Al-Samkari","doi":"10.1016/j.blre.2023.101139","DOIUrl":"10.1016/j.blre.2023.101139","url":null,"abstract":"<div><p>Chemotherapy-induced thrombocytopenia (CIT) is a common complication of antineoplastic therapy, resulting in antineoplastic therapy dose reductions, treatment delays, treatment discontinuation, and morbid bleeding events. Despite several decades of research into thrombopoietic growth factors in CIT, there are presently no available U.S. FDA- or EMA-approved agents to treat CIT. However, a respectable body of evidence has been published evaluating the thrombopoietin receptor agonists (TPO-RAs) for the management and prevention of CIT in patients with solid tumors, and critical studies are ongoing with the TPO-RAs romiplostim and avatrombopag. When employed in the appropriate patient population and used properly, TPO-RAs can successfully and safely manage CIT for extended periods of time with minimal apparent risks. This comprehensive review discusses the evidence for TPO-RAs in CIT in patients with solid tumors, provides detailed guidance for their use in the clinic, and discusses ongoing essential clinical trials in management of CIT.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X23001005/pdfft?md5=82954a7b0fa89cdef1ff28f986dec6b6&pid=1-s2.0-S0268960X23001005-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71429537","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
Haematopoietic stem cell health in sickle cell disease and its implications for stem cell therapies and secondary haematological disorders 镰状细胞病的造血干细胞健康及其对干细胞治疗和继发性血液病的影响。
IF 7.4 2区 医学
Blood Reviews Pub Date : 2024-01-01 DOI: 10.1016/j.blre.2023.101137
Vishaka Gorur , Kamil R. Kranc , Miguel Ganuza , Paul Telfer
{"title":"Haematopoietic stem cell health in sickle cell disease and its implications for stem cell therapies and secondary haematological disorders","authors":"Vishaka Gorur ,&nbsp;Kamil R. Kranc ,&nbsp;Miguel Ganuza ,&nbsp;Paul Telfer","doi":"10.1016/j.blre.2023.101137","DOIUrl":"10.1016/j.blre.2023.101137","url":null,"abstract":"<div><p>Gene modification of haematopoietic stem cells (HSCs) is a potentially curative approach to sickle cell disease (SCD) and offers hope for patients who are not eligible for allogeneic HSC transplantation. Current approaches require in vitro manipulation of healthy autologous HSC prior to their transplantation. However, the health and integrity of HSCs may be compromised by a variety of disease processes in SCD, and challenges have emerged in the clinical trials of gene therapy. There is also concern about increased susceptibility to haematological malignancies during long-term follow up of patients, and this raises questions about genomic stability in the stem cell compartment. In this review, we evaluate the evidence for HSC deficits in SCD and then discuss their potential causation. Finally, we suggest several questions which need to be addressed in order to progress with successful HSC manipulation for gene therapy in SCD.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X2300098X/pdfft?md5=dabf831677e606bcad138d17e20f9c44&pid=1-s2.0-S0268960X2300098X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71429536","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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
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