Blood Reviews最新文献_第5页

Revolutionising healing: Gene Editing's breakthrough against sickle cell disease 彻底改变治疗：基因编辑防治镰状细胞病的突破。

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-07 DOI: 10.1016/j.blre.2024.101185

Marija Dimitrievska , Dravie Bansal , Marta Vitale , John Strouboulis , Annarita Miccio , Kypros H. Nicolaides , Sara El Hoss , Panicos Shangaris , Joanna Jacków-Malinowska

{"title":"Revolutionising healing: Gene Editing's breakthrough against sickle cell disease","authors":"Marija Dimitrievska , Dravie Bansal , Marta Vitale , John Strouboulis , Annarita Miccio , Kypros H. Nicolaides , Sara El Hoss , Panicos Shangaris , Joanna Jacków-Malinowska","doi":"10.1016/j.blre.2024.101185","DOIUrl":"10.1016/j.blre.2024.101185","url":null,"abstract":"<div>Recent advancements in gene editing illuminate new potential therapeutic approaches for Sickle Cell Disease (SCD), a debilitating monogenic disorder caused by a point mutation in the β-globin gene. Despite the availability of several FDA-approved medications for symptomatic relief, allogeneic hematopoietic stem cell transplantation (HSCT) remains the sole curative option, underscoring a persistent need for novel treatments. This review delves into the growing field of gene editing, particularly the extensive research focused on curing haemoglobinopathies like SCD. We examine the use of techniques such as CRISPR-Cas9 and homology-directed repair, base editing, and prime editing to either correct the pathogenic variant into a non-pathogenic or wild-type one or augment fetal haemoglobin (HbF) production. The article elucidates ways to optimize these tools for efficacious gene editing with minimal off-target effects and offers insights into their effective delivery into cells. Furthermore, we explore clinical trials involving alternative SCD treatment strategies, such as LentiGlobin therapy and autologous HSCT, distilling the current findings. This review consolidates vital information for the clinical translation of gene editing for SCD, providing strategic insights for investigators eager to further the development of gene editing for SCD.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"65 ","pages":"Article 101185"},"PeriodicalIF":7.4,"publicationDate":"2024-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X24000183/pdfft?md5=db509693927e17ab2cf19d48178db412&pid=1-s2.0-S0268960X24000183-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140141181","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

Heartbreaker: Detection and prevention of cardiotoxicity in hematological malignancies 心碎者检测和预防血液恶性肿瘤的心脏毒性

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101166

Azin Vakilpour , Bénédicte Lefebvre , Catherine Lai , Marielle Scherrer-Crosbie

{"title":"Heartbreaker: Detection and prevention of cardiotoxicity in hematological malignancies","authors":"Azin Vakilpour , Bénédicte Lefebvre , Catherine Lai , Marielle Scherrer-Crosbie","doi":"10.1016/j.blre.2023.101166","DOIUrl":"10.1016/j.blre.2023.101166","url":null,"abstract":"<div>Cancer survivors are at significant risk of cardiovascular (CV) morbidity and mortality; patients with hematologic malignancies have a higher rate of death due to heart failure compared to all other cancer subtypes. The majority of conventional hematologic cancer treatments is associated with increased risk of acute and long-term CV toxicity. The incidence of cancer therapy induced CV toxicity depends on the combination of patient characteristics and on the type, dose, and duration of the therapy. Early diagnosis of CV toxicity, appropriate referral, more specific cardiac monitoring follow-up and timely interventions in target patients can decrease the risk of CV adverse events, the interruption of oncological therapy, and improve the patient's prognosis. Herein, we summarize the CV effects of conventional treatments used in hematologic malignancies with a focus on definitions and incidence of the most common CV toxicities, guideline recommended early detection approaches, and preventive strategies before and during cancer treatments.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"64 ","pages":"Article 101166"},"PeriodicalIF":7.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139063488","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

Platelet factor 4(PF4) and its multiple roles in diseases 血小板因子4(PF4)及其在疾病中的多重作用

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101155

Zhiyan Liu , Longtu Li , Hanxu Zhang , Xiaocong Pang , Zhiwei Qiu , Qian Xiang , Yimin Cui

{"title":"Platelet factor 4(PF4) and its multiple roles in diseases","authors":"Zhiyan Liu , Longtu Li , Hanxu Zhang , Xiaocong Pang , Zhiwei Qiu , Qian Xiang , Yimin Cui","doi":"10.1016/j.blre.2023.101155","DOIUrl":"10.1016/j.blre.2023.101155","url":null,"abstract":"<div>Platelet factor 4 (PF4) combines with heparin to form an antigen that could produce IgG antibodies and participate in heparin-induced thrombocytopenia (HIT). PF4 has attracted wide attention due to its role in novel coronavirus vaccine-19 (COVID-9)-induced immune thrombotic thrombocytopenia (VITT) and cognitive impairments. The electrostatic interaction between PF4 and negatively charged molecules is vital in the progression of VITT, which is similar to HIT. Emerging evidence suggests its multiple roles in hematopoietic and angiogenic inhibition, platelet coagulation interference, host inflammatory response promotion, vascular inhibition, and antitumor properties. The emerging pharmacological effects of PF4 may help deepen the exploration of its mechanism, thus accelerating the development of targeted therapies. However, due to its pleiotropic properties, the development of drugs targeting PF4 is at an early stage and faces many challenges. Herein, we discussed the characteristics and biological functions of PF4, summarized PF4-mediated signaling pathways, and discussed its multiple roles in diseases to inform novel approaches for successful clinical translational research.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"64 ","pages":"Article 101155"},"PeriodicalIF":7.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138441734","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

Practical considerations in clinical application of WHO 5th and ICC classification schemes for acute myeloid leukemia 急性髓系白血病WHO第5和ICC分型方案临床应用的实践思考。

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101156

Daniel J. Chandra , Curtis A. Lachowiez , Sanam Loghavi

引用次数: 0

Corrigendum to “Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias: Shedding new light on an old enzyme” [Blood Rev. 2023 Sep:61:101103] “激活丙酮酸激酶作为罕见溶血性贫血的治疗选择:对一种旧酶的新认识”的勘误表[Blood Rev. 2023 Sep:61:101103]。

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101160

Myrthe J. van Dijk , Jonathan R.A. de Wilde , Marije Bartels , Kevin H.M. Kuo , Andreas Glenthøj , Minke A.E. Rab , Eduard J. van Beers , Richard van Wijk

引用次数: 0

Down syndrome and leukemia: An insight into the disease biology and current treatment options 唐氏综合症和白血病:对疾病生物学和当前治疗方案的洞察。

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101154

Sonali P. Barwe, E. Anders Kolb, Anilkumar Gopalakrishnapillai

{"title":"Down syndrome and leukemia: An insight into the disease biology and current treatment options","authors":"Sonali P. Barwe, E. Anders Kolb, Anilkumar Gopalakrishnapillai","doi":"10.1016/j.blre.2023.101154","DOIUrl":"10.1016/j.blre.2023.101154","url":null,"abstract":"<div>Children with Down syndrome (DS) have a 10- to 20-fold greater predisposition to develop acute leukemia compared to the general population, with a skew towards myeloid leukemia (ML-DS). While ML-DS is known to be a subtype with good outcome, patients who relapse face a dismal prognosis. Acute lymphocytic leukemia in DS (DS-ALL) is considered to have poor prognosis. The relapse rate is high in DS-ALL compared to their non-DS counterparts. We have a better understanding about the mutational spectrum of DS leukemia. Studies using animal, embryonic stem cell- and induced pluripotent stem cell-based models have shed light on the mechanism by which these mutations contribute to disease initiation and progression. In this review, we list the currently available treatment strategies for DS-leukemias along with their outcome with emphasis on challenges with chemotherapy-related toxicities in children with DS. We focus on the mechanisms of initiation and progression of leukemia in children with DS and highlight the novel molecular targets with greater success in preclinical trials that have the potential to progress to the clinic.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"64 ","pages":"Article 101154"},"PeriodicalIF":7.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138453213","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

Chromosomal defects in multiple myeloma 多发性骨髓瘤的染色体缺陷

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2024.101168

Sarah E. Clarke , Kathryn A. Fuller , Wendy N. Erber

{"title":"Chromosomal defects in multiple myeloma","authors":"Sarah E. Clarke , Kathryn A. Fuller , Wendy N. Erber","doi":"10.1016/j.blre.2024.101168","DOIUrl":"10.1016/j.blre.2024.101168","url":null,"abstract":"<div>Multiple myeloma is a plasma cell neoplasm driven by primary (e.g. hyperdiploidy; IGH translocations) and secondary (e.g. 1q21 gains/amplifications; del(17p); MYC translocations) chromosomal events. These are important to detect as they influence prognosis, therapeutic response and disease survival. Currently, cytogenetic testing is most commonly performed by interphase fluorescence in situ hybridisation (FISH) on aspirated bone marrow samples. A number of variations to FISH methodology are available, including prior plasma cell enrichment and incorporation of immunophenotypic plasma cell identification. Other molecular methods are increasingly being utilised to provide a genome-wide view at high resolution (e.g. single nucleotide polymorphism (SNP) microarray analysis) and these can detect abnormalities in most cases. Despite their wide application at diagnostic assessment, both FISH and SNP-array have relatively low sensitivity, limiting their use for identification of prognostically significant low-level sub-clones or for disease monitoring. Next-generation sequencing is increasingly being used to detect mutations and new FISH techniques such as by flow cytometry are in development and may address some of the current test limitations. Here we review the primary and secondary cytogenetic aberrations in myeloma and discuss the range of techniques available for their assessment.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"64 ","pages":"Article 101168"},"PeriodicalIF":7.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X24000018/pdfft?md5=4cbbd86c290549e3b2d6d8e05c96d26e&pid=1-s2.0-S0268960X24000018-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139096351","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

Optimizing high dose melphalan 优化大剂量美法仑

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101162

Gunjan Shah, Sergio Giralt, Parastoo Dahi

{"title":"Optimizing high dose melphalan","authors":"Gunjan Shah, Sergio Giralt, Parastoo Dahi","doi":"10.1016/j.blre.2023.101162","DOIUrl":"10.1016/j.blre.2023.101162","url":null,"abstract":"<div>Melphalan, has been a major component of myeloma therapy since the 1950s. In the context of hematopoietic cell transplantation (HCT), high dose melphalan (HDM) is the most common conditioning regimen used due to its potent anti-myeloma effects and manageable toxicities. Common toxicities associated with HDM include myelosuppression, gastrointestinal issues, and mucositis. Established approaches to reduce these toxicities encompass dose modification, nausea prophylaxis with 5HT3 receptor antagonists, cryotherapy, amifostine use, and growth factors. Optimization of melphalan exposure through personalized dosing and its combination with other agents like busulfan, or bendamustine show promise. Propylene glycol-free melphalan (Evomela) represents a novel formulation aiming to enhance drug stability and reduce adverse effects. This review explores strategies to enhance the efficacy and mitigate the toxicity of HDM in multiple myeloma. Future directions involve exploring these strategies in clinical trials to improve the safety and efficacy of HDM, thereby enhancing outcomes for multiple myeloma patients undergoing autologous HCT.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"64 ","pages":"Article 101162"},"PeriodicalIF":7.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138576170","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

From microscope to micropixels: A rapid review of artificial intelligence for the peripheral blood film 从显微镜到微像素:外周血膜人工智能的快速回顾。

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101144

Bingwen Eugene Fan , Bryan Song Jun Yong , Ruiqi Li , Samuel Sherng Young Wang , Min Yi Natalie Aw , Ming Fang Chia , David Tao Yi Chen , Yuan Shan Neo , Bruno Occhipinti , Ryan Ruiyang Ling , Kollengode Ramanathan , Yi Xiong Ong , Kian Guan Eric Lim , Wei Yong Kevin Wong , Shu Ping Lim , Siti Thuraiya Binte Abdul Latiff , Hemalatha Shanmugam , Moh Sim Wong , Kuperan Ponnudurai , Stefan Winkler

{"title":"From microscope to micropixels: A rapid review of artificial intelligence for the peripheral blood film","authors":"Bingwen Eugene Fan , Bryan Song Jun Yong , Ruiqi Li , Samuel Sherng Young Wang , Min Yi Natalie Aw , Ming Fang Chia , David Tao Yi Chen , Yuan Shan Neo , Bruno Occhipinti , Ryan Ruiyang Ling , Kollengode Ramanathan , Yi Xiong Ong , Kian Guan Eric Lim , Wei Yong Kevin Wong , Shu Ping Lim , Siti Thuraiya Binte Abdul Latiff , Hemalatha Shanmugam , Moh Sim Wong , Kuperan Ponnudurai , Stefan Winkler","doi":"10.1016/j.blre.2023.101144","DOIUrl":"10.1016/j.blre.2023.101144","url":null,"abstract":"<div>Artificial intelligence (AI) and its application in classification of blood cells in the peripheral blood film is an evolving field in haematology. We performed a rapid review of the literature on AI and peripheral blood films, evaluating the condition studied, image datasets, machine learning models, training set size, testing set size and accuracy. A total of 283 studies were identified, encompassing 6 broad domains: malaria (n = 95), leukemia (n = 81), leukocytes (n = 72), mixed (n = 25), erythrocytes (n = 15) or Myelodysplastic syndrome (MDS) (n = 1). These publications have demonstrated high self-reported mean accuracy rates across various studies (95.5% for malaria, 96.0% for leukemia, 94.4% for leukocytes, 95.2% for mixed studies and 91.2% for erythrocytes), with an overall mean accuracy of 95.1%. Despite the high accuracy, the challenges toward real world translational usage of these AI trained models include the need for well-validated multicentre data, data standardisation, and studies on less common cell types and non-malarial blood-borne parasites.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"64 ","pages":"Article 101144"},"PeriodicalIF":7.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138453214","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

Unraveling germline predisposition in hematological neoplasms: Navigating complexity in the genomic era 揭示血液学肿瘤的种系易感性:在基因组时代导航复杂性。

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-03-01 DOI: 10.1016/j.blre.2023.101143

Joaquín Jerez , Marta Santiago

引用次数: 0