Blood Reviews最新文献_第2页

Autologous stem cell transplantation in AL amyloidosis: Muddy waters AL 淀粉样变性的自体干细胞移植：浑水摸鱼。

IF 6.9 2区医学

Blood Reviews Pub Date : 2024-11-01 DOI: 10.1016/j.blre.2024.101228

Patrick Hagen , Anita D'Souza

引用次数: 0

Advancing CAR T-cell therapies: Preclinical insights and clinical translation for hematological malignancies 推进 CAR T 细胞疗法：血液恶性肿瘤的临床前研究和临床转化。

IF 6.9 2区医学

Blood Reviews Pub Date : 2024-11-01 DOI: 10.1016/j.blre.2024.101241

Arun K. Arunachalam, Céline Grégoire, Beatriz Coutinho de Oliveira, Jan Joseph Melenhorst

{"title":"Advancing CAR T-cell therapies: Preclinical insights and clinical translation for hematological malignancies","authors":"Arun K. Arunachalam, Céline Grégoire, Beatriz Coutinho de Oliveira, Jan Joseph Melenhorst","doi":"10.1016/j.blre.2024.101241","DOIUrl":"10.1016/j.blre.2024.101241","url":null,"abstract":"<div><div>Chimeric antigen receptor (CAR) T-cell therapy has achieved significant success in achieving durable and potentially curative responses in patients with hematological malignancies. CARs are tailored fusion proteins that direct T cells to a specific antigen on tumor cells thereby eliciting a targeted immune response. The approval of several CD19-targeted CAR T-cell therapies has resulted in a notable surge in clinical trials involving CAR T cell therapies for hematological malignancies. Despite advancements in understanding response mechanisms, resistance patterns, and adverse events associated with CAR T-cell therapy, the translation of these insights into robust clinical efficacy has shown modest outcomes in both clinical trials and real-world scenarios. Therefore, the assessment of CAR T-cell functionality through rigorous preclinical studies plays a pivotal role in refining therapeutic strategies for clinical applications. This review provides an overview of the various in vitro and animal models used to assess the functionality of CAR T-cells. We discuss the findings from preclinical research involving approved CAR T-cell products, along with the implications derived from recent preclinical studies aiming to optimize the functionality of CAR T-cells. The review underscores the importance of robust preclinical evaluations and the need for models that accurately replicate human disease to bridge the gap between preclinical success and clinical efficacy.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101241"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142301815","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

Current challenges in conditioning regimens for MDS transplantation 当前 MDS 移植调节方案面临的挑战

IF 6.9 2区医学

Blood Reviews Pub Date : 2024-07-10 DOI: 10.1016/j.blre.2024.101223

A.B. Notarantonio , M. Robin , M. D'Aveni

{"title":"Current challenges in conditioning regimens for MDS transplantation","authors":"A.B. Notarantonio , M. Robin , M. D'Aveni","doi":"10.1016/j.blre.2024.101223","DOIUrl":"10.1016/j.blre.2024.101223","url":null,"abstract":"<div><p>Myelodysplastic syndrome (MDS) is a very heterogeneous clonal disorder. Patients with “higher-risk” MDS, defined by specific recurrent genetic abnormalities, have a poor prognosis because of a high risk of progression to secondary acute myeloid leukemia with low chemosensitivity. Allogeneic hematopoietic stem cell transplantation remains the only treatment that offers durable disease control because the donor immune system allows graft-versus-MDS effects. In terms of preparation steps before transplantation, targeting the malignant clone by increasing the conditioning regimen intensity is still a matter of intense debate. MDS is mainly diagnosed in older patients, and high toxicity related to common myeloablative conditioning regimens has been reported. Efforts to include new drugs in the conditioning regimen to achieve the best malignant clone control without increasing toxicity have been made over the past 20 years. We summarized these retrospective and prospective studies and evaluated the limitations of the available evidence to delineate the ideal conditioning regimen.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"67 ","pages":"Article 101223"},"PeriodicalIF":6.9,"publicationDate":"2024-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X24000560/pdfft?md5=b28710cbdba2c6aa48108ac802eca6b2&pid=1-s2.0-S0268960X24000560-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141700122","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

Immune thrombocytopenia: Pathophysiology and impacts of Romiplostim treatment 免疫性血小板减少症：病理生理学和罗米波司汀治疗的影响。

IF 6.9 2区医学

Blood Reviews Pub Date : 2024-06-20 DOI: 10.1016/j.blre.2024.101222

{"title":"Immune thrombocytopenia: Pathophysiology and impacts of Romiplostim treatment","authors":"","doi":"10.1016/j.blre.2024.101222","DOIUrl":"10.1016/j.blre.2024.101222","url":null,"abstract":"<div><p>Immune thrombocytopenia (ITP) is an autoimmune bleeding disease caused by immune-mediated platelet destruction and decreased platelet production. ITP is characterized by an isolated thrombocytopenia (<100 × 10<sup>9</sup>/L) and increased risk of bleeding. The disease has a complex pathophysiology wherein immune tolerance breakdown leads to platelet and megakaryocyte destruction. Therapeutics such as corticosteroids, intravenous immunoglobulins (IVIg), rituximab, and thrombopoietin receptor agonists (TPO-RAs) aim to increase platelet counts to prevent hemorrhage and increase quality of life. TPO-RAs act via stimulation of TPO receptors on megakaryocytes to directly stimulate platelet production. Romiplostim is a TPO-RA that has become a mainstay in the treatment of ITP. Treatment significantly increases megakaryocyte maturation and growth leading to improved platelet production and it has recently been shown to have additional immunomodulatory effects in treated patients. This review will highlight the complex pathophysiology of ITP and discuss the usage of Romiplostim in ITP and its ability to potentially immunomodulate autoimmunity.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"67 ","pages":"Article 101222"},"PeriodicalIF":6.9,"publicationDate":"2024-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X24000559/pdfft?md5=1c8588478182d414ca3ddab3c9877c79&pid=1-s2.0-S0268960X24000559-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141473052","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

Dismantling relapsed/refractory mantle cell lymphoma 解构复发/难治套细胞淋巴瘤

IF 6.9 2区医学

Blood Reviews Pub Date : 2024-06-13 DOI: 10.1016/j.blre.2024.101221

引用次数: 0

Thrombotic events associated with immune checkpoint inhibitors and novel antithrombotic strategies to mitigate bleeding risk 与免疫检查点抑制剂相关的血栓事件以及降低出血风险的新型抗血栓策略。

IF 6.9 2区医学

Blood Reviews Pub Date : 2024-06-07 DOI: 10.1016/j.blre.2024.101220

{"title":"Thrombotic events associated with immune checkpoint inhibitors and novel antithrombotic strategies to mitigate bleeding risk","authors":"","doi":"10.1016/j.blre.2024.101220","DOIUrl":"10.1016/j.blre.2024.101220","url":null,"abstract":"<div><p><span>Although immunotherapy<span><span><span> is expanding treatment options for cancer patients, the prognosis of advanced cancer remains poor, and these patients must contend with both cancers and cancer-related thrombotic events. In particular, </span>immune checkpoint inhibitors are associated with an increased risk of atherosclerotic thrombotic events. Given the fundamental role of platelets in atherothrombosis, co-administration of </span>antiplatelet<span><span> agents is always indicated. Platelets are also involved in all steps of cancer progression. Classical antithrombotic drugs<span> can cause inevitable hemorrhagic side effects due to blocking </span></span>integrin<span><span> β3 bidirectional signaling, which regulates simultaneously thrombosis and hemostasis. Meanwhile, many promising new targets are emerging with minimal </span>bleeding risk and desirable anti-tumor effects. This review will focus on the issue of thrombosis during immune checkpoint inhibitor treatment and the role of </span></span></span></span>platelet activation<span> in cancer progression as well as explore the mechanisms by which novel antiplatelet therapies may exert both antithrombotic and antitumor effects without excessive bleeding risk.</span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"67 ","pages":"Article 101220"},"PeriodicalIF":6.9,"publicationDate":"2024-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141322131","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

Gut microbiome composition and dysbiosis in immune thrombocytopenia: A review of literature 免疫性血小板减少症的肠道微生物组组成和菌群失调：文献综述。

IF 6.9 2区医学

Blood Reviews Pub Date : 2024-06-06 DOI: 10.1016/j.blre.2024.101219

{"title":"Gut microbiome composition and dysbiosis in immune thrombocytopenia: A review of literature","authors":"","doi":"10.1016/j.blre.2024.101219","DOIUrl":"10.1016/j.blre.2024.101219","url":null,"abstract":"<div><p><span>Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder characterized by excessive reticuloendothelial platelet destruction and inadequate compensatory platelet production. However, the pathogenesis of ITP is relatively complex, and its exact mechanisms and etiology have not been definitively established. The </span>gut microbiome<span><span><span><span>, namely a diverse community of symbiotic microorganisms residing in the gastrointestinal system, affects health through involvement in human metabolism, </span>immune modulation, and maintaining physiological balance. Emerging evidence reveals that the gut </span>microbiome<span><span> composition differs in patients with ITP compared to healthy individuals, which is related with platelet count, disease duration, and response to treatment. These findings suggest that the </span>microbiome and </span></span>metabolome profiles of individuals could unveil a new pathway for aiding diagnosis, predicting prognosis, assessing treatment response, and formulating personalized therapeutic approaches for ITP. However, due to controversial reports, definitive conclusions cannot be drawn, and further investigations are needed.</span></p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"67 ","pages":"Article 101219"},"PeriodicalIF":6.9,"publicationDate":"2024-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141307541","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

Endothelial injury and dysfunction with emerging immunotherapies in multiple myeloma, the impact of COVID-19, and endothelial protection with a focus on the evolving role of defibrotide 多发性骨髓瘤新兴免疫疗法导致的内皮损伤和功能障碍、COVID-19 的影响以及内皮保护（重点关注去纤维化药物不断演变的作用

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-06-03 DOI: 10.1016/j.blre.2024.101218

Clifton C. Mo , Edward Richardson , Eleonora Calabretta , Francesco Corrado , Mehmet H. Kocoglu , Rebecca M. Baron , Jean Marie Connors , Massimo Iacobelli , Lee-Jen Wei , Aaron P. Rapoport , Maribel Díaz-Ricart , José M. Moraleda , Carmelo Carlo-Stella , Paul G. Richardson

{"title":"Endothelial injury and dysfunction with emerging immunotherapies in multiple myeloma, the impact of COVID-19, and endothelial protection with a focus on the evolving role of defibrotide","authors":"Clifton C. Mo , Edward Richardson , Eleonora Calabretta , Francesco Corrado , Mehmet H. Kocoglu , Rebecca M. Baron , Jean Marie Connors , Massimo Iacobelli , Lee-Jen Wei , Aaron P. Rapoport , Maribel Díaz-Ricart , José M. Moraleda , Carmelo Carlo-Stella , Paul G. Richardson","doi":"10.1016/j.blre.2024.101218","DOIUrl":"10.1016/j.blre.2024.101218","url":null,"abstract":"<div><p>Patients with multiple myeloma (MM) were among the groups impacted more severely by the COVID-19 pandemic, with higher rates of severe disease and COVID-19-related mortality. MM and COVID-19, plus post-acute sequelae of SARS-CoV-2 infection, are associated with endothelial dysfunction and injury, with overlapping inflammatory pathways and coagulopathies. Existing treatment options for MM, notably high-dose therapy with autologous stem cell transplantation and novel chimeric antigen receptor (CAR) T-cell therapies and bispecific T-cell engaging antibodies, are also associated with endothelial cell injury and mechanism-related toxicities. These pathologies include cytokine release syndrome (CRS) and neurotoxicity that may be exacerbated by underlying endotheliopathies. In the context of these overlapping risks, prophylaxis and treatment approaches mitigating the inflammatory and pro-coagulant effects of endothelial injury are important considerations for patient management, including cytokine receptor antagonists, thromboprophylaxis with low-molecular-weight heparin and direct oral anticoagulants, and direct endothelial protection with defibrotide in the appropriate clinical settings.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"66 ","pages":"Article 101218"},"PeriodicalIF":7.4,"publicationDate":"2024-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X24000511/pdfft?md5=8ce6ec6a02e5d22fd5262aa7ded0991e&pid=1-s2.0-S0268960X24000511-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141275333","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

Factor B inhibitor iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria 治疗阵发性夜间血红蛋白尿的 B 因子抑制剂 iptacopan。

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-05-25 DOI: 10.1016/j.blre.2024.101210

Bo Xu , Bo Kang , Jixiang Chen , Shaoqian Li , Jiecan Zhou

{"title":"Factor B inhibitor iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria","authors":"Bo Xu , Bo Kang , Jixiang Chen , Shaoqian Li , Jiecan Zhou","doi":"10.1016/j.blre.2024.101210","DOIUrl":"10.1016/j.blre.2024.101210","url":null,"abstract":"<div><p>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with a variety of manifestations. Currently, the methods for treating PNH include anti-C5 treatments (eculizumab and ravulizumab) and pegcetacoplan (a targeted C3 inhibitor). On December 5, 2023, the US FDA approved a factor B inhibitor called Fabhalta® (iptacopan), previously known as LNP023, for the treatment of adult patients with PNH, including those who have previously received anti-C5 therapy. The main objective of this review was to elucidate the clinical efficacy and safety of the newly approved factor B inhibitor, iptacopan. Iptacopan plays a proximal role in the alternative complement pathway to control extravascular hemolysis mediated by C3b and intravascular hemolysis mediated by terminal complement. The recommended dosage is 200 mg orally twice daily. The 24-week results of the pivotal phase III open-label trial, APPLY-PNH, demonstrated that among PNH patients who had previously received anti-C5 therapy, 51/60 (estimated percentages 82%) of patients in the iptacopan group showed an increase in hemoglobin of ≥2 g/dL compared to 0/35 (estimated percentages 2%) in the standard treatment group, also, 69% of iptacopan-treated patients achieved hemoglobin levels ≥12 g/dL, while no patients in the standard treatment group reached this level (both <em>p</em> < 0.001). The 48-week results were similar to those observed at 24 weeks. The most common adverse events were headache, infection and diarrhea. There were almost no clinical breakthrough hemolysis. Trials evaluating the long-term safety and efficacy of iptacopan are currently recruiting.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"66 ","pages":"Article 101210"},"PeriodicalIF":7.4,"publicationDate":"2024-05-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141176913","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

Cancer cytogenetics in a genomics world: Wedding the old with the new 基因组学世界中的癌症细胞遗传学：新旧结合

IF 7.4 2区医学

Blood Reviews Pub Date : 2024-05-07 DOI: 10.1016/j.blre.2024.101209

Jorune Balciuniene , Yi Ning , Hillard M. Lazarus , Vania Aikawa , Sarina Sherpa , Yanming Zhang , Jennifer J.D. Morrissette

{"title":"Cancer cytogenetics in a genomics world: Wedding the old with the new","authors":"Jorune Balciuniene , Yi Ning , Hillard M. Lazarus , Vania Aikawa , Sarina Sherpa , Yanming Zhang , Jennifer J.D. Morrissette","doi":"10.1016/j.blre.2024.101209","DOIUrl":"10.1016/j.blre.2024.101209","url":null,"abstract":"<div><p>Since the discovery of the Philadelphia chromosome in 1960, cytogenetic studies have been instrumental in detecting chromosomal abnormalities that can inform cancer diagnosis, treatment, and risk assessment efforts. The initial expansion of cancer cytogenetics was with fluorescence in situ hybridization (FISH) to assess submicroscopic alterations in dividing or non-dividing cells and has grown into the incorporation of chromosomal microarrays (CMA), and next generation sequencing (NGS). These molecular technologies add additional dimensions to the genomic assessment of cancers by uncovering cytogenetically invisible molecular markers. Rapid technological and bioinformatic advances in NGS are so promising that the idea of performing whole genome sequencing as part of routine patient care may soon become economically and logistically feasible. However, for now cytogenetic studies continue to play a major role in the diagnostic testing and subsequent assessments in leukemia with other genomic studies serving as complementary testing options for detection of actionable genomic abnormalities. In this review, we discuss the role of conventional cytogenetics (karyotyping, chromosome analysis) and FISH studies in hematological malignancies, highlighting the continued clinical utility of these techniques, the subtleties and complexities that are relevant to treating physicians and the unique strengths of cytogenetics that cannot yet be paralleled by the current high-throughput molecular technologies. Additionally, we describe how CMA, optical genome mapping (OGM), and NGS detect abnormalities that were beyond the capacity of cytogenetic studies and how an integrated approach (broad molecular testing) can contribute to the detection of actionable targets and variants in malignancies. Finally, we discuss advances in the field of genomic testing that are bridging the advantages of individual (single) cell based cytogenetic testing and broad genomic testing.</p></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"66 ","pages":"Article 101209"},"PeriodicalIF":7.4,"publicationDate":"2024-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141023017","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