Blood Reviews最新文献

{"title":"Tailoring oral anticoagulant treatment in the era of multi-drug therapies for PAH and CTEPH","authors":"Jean Escal ,&nbsp;Geraldine Poenou ,&nbsp;Xavier Delavenne ,&nbsp;Souad Bezzeghoud ,&nbsp;Valentine Mismetti ,&nbsp;Marc Humbert ,&nbsp;David Montani ,&nbsp;Laurent Bertoletti","doi":"10.1016/j.blre.2024.101240","DOIUrl":"10.1016/j.blre.2024.101240","url":null,"abstract":"<div><div>The use of oral anticoagulants in the management of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) presents distinct therapeutic challenges and benefits. In PAH, the benefits of oral anticoagulation are uncertain, with studies yielding mixed results on their efficacy and safety. Conversely, oral anticoagulants are a cornerstone in the treatment of CTEPH, where their use is consistently recommended to prevent recurrent thromboembolic events. The choice between vitamin K antagonists (VKAs) and direct oral anticoagulants (DOACs) remains a significant clinical question, as each type presents advantages and potential drawbacks. Furthermore, drug-drug interactions (DDIs) with concomitant PAH and CTEPH treatments complicate anticoagulant management, necessitating careful consideration of individual patient regimens. This review examines the current evidence on oral anticoagulant use in PAH and CTEPH and discusses the implications of DDIs within a context of multi-drug treatments, including targeted drugs in PAH.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101240"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142156777","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}

{"title":"Measurable residual disease (MRD)-testing in haematological cancers: A giant leap forward or sideways?","authors":"Qiujin Shen ,&nbsp;Xiaowen Gong ,&nbsp;Yahui Feng ,&nbsp;Yu Hu ,&nbsp;Tiantian Wang ,&nbsp;Wen Yan ,&nbsp;Wei Zhang ,&nbsp;Saibing Qi ,&nbsp;Robert Peter Gale ,&nbsp;Junren Chen","doi":"10.1016/j.blre.2024.101226","DOIUrl":"10.1016/j.blre.2024.101226","url":null,"abstract":"<div><div>Measurable residual disease (MRD)-testing is used in many haematological cancers to estimate relapse risk and to direct therapy. Sometimes MRD-test results are used for regulatory approval. However, some people including regulators wrongfully believe results of MRD-testing are highly accurate and of proven efficacy in directing therapy. We review MRD-testing technologies and evaluate the accuracy of MRD-testing for predicting relapse and the strength of evidence supporting efficacy of MRD-guided therapy. We show that at the individual level MRD-test results are often an inaccurate relapse predictor. Also, no convincing data indicate that increasing therapy-intensity based on a positive MRD-test reduces relapse risk or improves survival. We caution against adjusting therapy-intensity based solely on results of MRD-testing.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101226"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142010021","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}

{"title":"Clinical applications of circulating tumor DNA in hematological malignancies: From past to the future","authors":"Jun-Ying Li,&nbsp;Li-Ping Zuo,&nbsp;Jian Xu,&nbsp;Chun-Yan Sun","doi":"10.1016/j.blre.2024.101237","DOIUrl":"10.1016/j.blre.2024.101237","url":null,"abstract":"<div><div>Liquid biopsy, particularly circulating tumor DNA (ctDNA), has drawn a lot of attention as a non- or minimal-invasive detection approach for clinical applications in patients with cancer. Many hematological malignancies are well suited for serial and repeated ctDNA surveillance due to relatively high ctDNA concentrations and high loads of tumor-specific genetic and epigenetic abnormalities. Progress of detecting technology in recent years has improved sensitivity and specificity significantly, thus broadening and strengthening the potential utilities of ctDNA including early diagnosis, prognosis estimation, treatment response evaluation, minimal residual disease monitoring, targeted therapy selection, and immunotherapy surveillance. This manuscript reviews the detection methodologies, clinical application and future challenges of ctDNA in hematological malignancies, especially for lymphomas, myeloma and leukemias.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101237"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142252509","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}

{"title":"Longitudinal clinical manifestations of Fanconi anemia: A systematized review","authors":"Alex Hoover ,&nbsp;Lucie M. Turcotte ,&nbsp;Rachel Phelan ,&nbsp;Crystal Barbus ,&nbsp;Arpana Rayannavar ,&nbsp;Bradley S. Miller ,&nbsp;Erin E. Reardon ,&nbsp;Nicole Theis-Mahon ,&nbsp;Margaret L. MacMillan","doi":"10.1016/j.blre.2024.101225","DOIUrl":"10.1016/j.blre.2024.101225","url":null,"abstract":"<div><div>Fanconi anemia (FA) is a rare and complex inherited genetic disorder characterized by impaired DNA repair mechanisms leading to genomic instability. Individuals with FA have increased susceptibility to congenital anomalies, progressive bone marrow failure, leukemia and malignant tumors, endocrinopathies and other medical issues. In recent decades, steadily improved approaches to hematopoietic cell transplantation (HCT), the only proven curative therapy for the hematologic manifestations of FA, have significantly increased the life expectancy of affected individuals, illuminating the need to understand the long-term consequences and multi-organ ramifications. Utilizing a systematized review approach with narrative synthesis of each primary issue and organ system, we shed light on the challenges and opportunities for optimizing the care and quality of life for individuals with FA and identify knowledge gaps informing future research directions.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101225"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141899019","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}

{"title":"Corrigendum to “Measurable residual disease (MRD)-testing in haematological cancers: A giant leap forward or sideways?”[BLOOD REVIEWS, 9 August 2024, https://doi.org/10.1016/j.blre.2024.101226]","authors":"Qiujin Shen ,&nbsp;Xiaowen Gong ,&nbsp;Yahui Feng ,&nbsp;Yu Hu ,&nbsp;Tiantian Wang ,&nbsp;Wen Yan ,&nbsp;Wei Zhang ,&nbsp;Saibing Qi ,&nbsp;Robert Peter Gale ,&nbsp;Junren Chen","doi":"10.1016/j.blre.2024.101239","DOIUrl":"10.1016/j.blre.2024.101239","url":null,"abstract":"","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101239"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142147014","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}

{"title":"From MGUS to multiple myeloma: Unraveling the unknown of precursor states","authors":"Gil Hevroni ,&nbsp;Mounika Vattigunta ,&nbsp;Dickran Kazandjian ,&nbsp;David Coffey ,&nbsp;Benjamin Diamond ,&nbsp;Francesco Maura ,&nbsp;James Hoffman ,&nbsp;Ola Landgren","doi":"10.1016/j.blre.2024.101242","DOIUrl":"10.1016/j.blre.2024.101242","url":null,"abstract":"<div><div>In the 1960s, through laboratory-based investigations of peripheral blood partnered with detailed clinical annotations, Dr. Waldenström described a condition he called “benign monoclonal gammopathy”. These patients were asymptomatic with a detectable monoclonal protein, and did not meet imaging and laboratory criteria for multiple myeloma. In 1978, through observational retrospective review of medical records, Dr. Kyle observed that not all cases of monoclonal gammopathy were benign. He introduced the term monoclonal gammopathy of undetermined significance (MGUS) to describe a condition that may potentially progress to multiple myeloma (MM), highlighting clinical inability in predicting which patients might progress. In 1980, Drs. Kyle and Greipp described 6 cases which did not fit the definitions of MGUS or MM, and they remained asymptomatic after at least 5 years of follow-up; they were proposed to have smoldering multiple myeloma (SMM). Over time, SMM was defined by arbitrary numerical values (≥10 % plasma cells in the bone marrow and serum M-protein concentration ≥ 3 g/dL). Numerous clinical scores have been developed to define high-risk groups for progression to MM. Current statistical models for progression provide only average risk scores, offering limited clinical utility since the risk of progression at an individual level remains unknown. Physician-scientists are focusing on emerging technologies, such as whole genome sequencing, tumor microenvironment analysis, and single-cell RNA sequencing, to understand precursor states at a molecular level. The overarching goal of these technologies is to better characterize monoclonal gammopathy and other myeloma precursor states. This will enable clinicians to provide more precise, individualized risk assessments and ultimately improve patient outcomes. This review outlines the history of MM precursor states, current definitions, challenges in risk stratification models, and the role of emerging technologies in enhancing predictions and outcomes.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101242"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142402126","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}

{"title":"Addressing the surge of infections by multidrug-resistant Enterobacterales in hematopoietic cell transplantation","authors":"Mobil Akhmedov ,&nbsp;J. Luis Espinoza","doi":"10.1016/j.blre.2024.101229","DOIUrl":"10.1016/j.blre.2024.101229","url":null,"abstract":"<div><div>Patients undergoing hematopoietic cell transplantation (HCT) have an increased risk of developing severe infections. In recent years, bloodstream infections caused by Gram-negative bacteria have been increasingly reported among HCT recipients, and many of these infections are caused by bacterial strains of the <em>Enterobacterales</em> order. Among these pathogens, particularly concerning are the multidrug-resistant <em>Enterobacterales</em> (MDRE), such as Extended Spectrum β-lactamase-producing <em>Enterobacterales</em> and Carbapenem-resistant <em>Enterobacterales</em>, since infections caused by these pathogens are difficult to treat due to the limited antimicrobial options and are associated with worse transplant outcomes. We summarized the evidence from studies published in PubMed and Scopus on the burden of MDRE infections in HCT recipients, and strategies for the management and prevention of these infections, including strict adherence to recommended infection control practices and multidisciplinary antimicrobial stewardship, the use of probiotics, and fecal microbiota transplantation, are also discussed.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101229"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142115237","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}

{"title":"Autologous stem cell transplantation in AL amyloidosis: Muddy waters","authors":"Patrick Hagen ,&nbsp;Anita D'Souza","doi":"10.1016/j.blre.2024.101228","DOIUrl":"10.1016/j.blre.2024.101228","url":null,"abstract":"<div><div>Immunoglobulin light chain (AL) amyloidosis is a malignant plasma cell dyscrasia causing multi-organ morbidity. High dose melphalan and autologous stem cell transplantation (ASCT) is a preferred consolidation approach and is safe with improved patient selection criteria. With the advent of bortezomib and daratumumab based induction therapy, nearly all patients can achieve deep hematological responses but follow up for daratumumab based induction is short. Consequently, the traditional approach of induction followed by ASCT is called into question. Given the multi-organ involvement of AL, endpoints beyond depth of response and hematological progression free survival (PFS) are important. Major organ dysfunction PFS (MOD-PFS) adds to PFS and is a composite endpoint of PFS, renal and cardiac organ progression, and overall survival. It is currently unknown which consolidative approach (ASCT or non-ASCT) will generate improved outcomes across the MOD-PFS spectrum a question the recently opened S2213 trial will attempt to answer.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"68 ","pages":"Article 101228"},"PeriodicalIF":6.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142047605","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}

{"title":"Advancing CAR T-cell therapies: Preclinical insights and clinical translation for hematological malignancies","authors":"Arun K. Arunachalam,&nbsp;Céline Grégoire,&nbsp;Beatriz Coutinho de Oliveira,&nbsp;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}

{"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 (&lt;100 × 10⁹/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}