Current Research in Translational Medicine最新文献

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Extracorporeal membrane oxygenation (ECMO) during aplasia: A bridge towards myopericarditis recovery after autologous hematopoietic stem cell transplant for systemic sclerosis and recent Coronarovirus disease (COVID-19) vaccination 再生障碍期体外膜氧合(ECMO):自体造血干细胞移植治疗系统性硬化症和近期接种冠状病毒病(COVID-19)疫苗后心肌炎康复的桥梁
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-04-09 DOI: 10.1016/j.retram.2024.103449
Carlotta Cacciatore , Mathilde Baudet , Estelle Jean , Simona Presente , Marylou Para , Romain Sonneville , Dimitri Arangalage , Nassim Ait Abdallah , Flore Sicre de Fontbrune , Pedro Henrique Prata , Benjamin Crichi , Baptiste Hervier , Nathalie Parquet , Gilles Soulat , Elie Mousseaux , Richard K Burt , Dominique Farge
{"title":"Extracorporeal membrane oxygenation (ECMO) during aplasia: A bridge towards myopericarditis recovery after autologous hematopoietic stem cell transplant for systemic sclerosis and recent Coronarovirus disease (COVID-19) vaccination","authors":"Carlotta Cacciatore ,&nbsp;Mathilde Baudet ,&nbsp;Estelle Jean ,&nbsp;Simona Presente ,&nbsp;Marylou Para ,&nbsp;Romain Sonneville ,&nbsp;Dimitri Arangalage ,&nbsp;Nassim Ait Abdallah ,&nbsp;Flore Sicre de Fontbrune ,&nbsp;Pedro Henrique Prata ,&nbsp;Benjamin Crichi ,&nbsp;Baptiste Hervier ,&nbsp;Nathalie Parquet ,&nbsp;Gilles Soulat ,&nbsp;Elie Mousseaux ,&nbsp;Richard K Burt ,&nbsp;Dominique Farge","doi":"10.1016/j.retram.2024.103449","DOIUrl":"https://doi.org/10.1016/j.retram.2024.103449","url":null,"abstract":"<div><p>Systemic sclerosis (SSc) is a rare autoimmune disease (AD), characterised by early diffuse vasculopathy, activation of the immune response and progressive skin and internal organ fibrosis. In severe progressive diffuse SSc (dSSc), autologous hematopoietic stem cell transplantation (aHSCT) improves survival, despite its own risk of complications and transplant related mortality (TRM).</p><p>We present herein the case of a dSSc patient undergoing aHSCT with low dose cyclophosphamide conditioning and sudden acute myopericarditis and cardiogenic shock, four weeks after a second mRNA SARS-CoV-2 vaccine (Pfizer) injection. Four days of extracorporeal membrane oxygenation (ECMO) support during the aplasia period, allowed to observe full cardiac function recovery and progressive SSc rehabilitation with sustained disease response at 30 months follow-up. This report illustrates, for the first time to our knowledge, that ECMO can be indicated despite aplasia during aHSCT and successfully used as a bridge towards heart function recovery in highly selected and fragile AD patients. We review the factors that may contribute to endothelial and myocardial stunning and acute reversible cardiac failure in SSc and aggravate intrinsic endothelial injury during the aHSCT procedure. These classically include: cyclophosphamide drug toxicity, viral infections and autoimmune activation with disease flair per se. In the COVID-19 pandemic times, acute myocarditis due to recent viral infection or mRNA vaccine per se, must also be considered.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 3","pages":"Article 103449"},"PeriodicalIF":4.1,"publicationDate":"2024-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2452318624000126/pdfft?md5=b5ba308a940814fa81061f5e19e1c50e&pid=1-s2.0-S2452318624000126-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140605626","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}
引用次数: 0
Validation of analytical methods for the production of expanded γδ T lymphocytes useful for therapeutic purposes 验证生产用于治疗目的的扩增γδ T 淋巴细胞的分析方法
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-03-05 DOI: 10.1016/j.retram.2024.103445
Fabio Morandi, Martina Della Lastra, Federico Zara, Irma Airoldi
{"title":"Validation of analytical methods for the production of expanded γδ T lymphocytes useful for therapeutic purposes","authors":"Fabio Morandi,&nbsp;Martina Della Lastra,&nbsp;Federico Zara,&nbsp;Irma Airoldi","doi":"10.1016/j.retram.2024.103445","DOIUrl":"10.1016/j.retram.2024.103445","url":null,"abstract":"<div><p>The use of γδ T lymphocytes as advanced therapeutic medicinal product has attracted much interest in the last years. Indeed, such cells are an ideal tool for the reconstitution of the immune system in patients receiving hematopoietic stem cell transplantation, due to their MHC-independent anti-tumor and anti-viral activities. We have here setup a protocol for the production of pure and functional γδ T lymphocytes, expanded from healthy donors’ mononuclear cells, and validated the analytical methods to identify them and to analyze their potency. Next, we performed stability studies to ensure that the cell product (γδ T cells) can be used after freezing and thawing. Notably, such protocol can be promptly translated to GMP-facility, since it has been designed using only clinical grade reagents.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 4","pages":"Article 103445"},"PeriodicalIF":4.1,"publicationDate":"2024-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140087661","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}
引用次数: 0
Sofosbuvir (+) daclatasvir (+) ribavirin in Egyptian patients with hepatitis C virus: Therapeutic outcomes and the prognostic role of natural killer cells 索非布韦(+)达卡他韦(+)利巴韦林治疗埃及丙型肝炎病毒感染者:治疗效果和自然杀伤细胞的预后作用
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-03-01 DOI: 10.1016/j.retram.2024.103443
Ahmed B. Zaid , Shimaa K. Almady , Samah M. Awad , Mona G. Elabd , Sara A. Saied , AbdulRahman A Saied , Alshimaa M Elmalawany
{"title":"Sofosbuvir (+) daclatasvir (+) ribavirin in Egyptian patients with hepatitis C virus: Therapeutic outcomes and the prognostic role of natural killer cells","authors":"Ahmed B. Zaid ,&nbsp;Shimaa K. Almady ,&nbsp;Samah M. Awad ,&nbsp;Mona G. Elabd ,&nbsp;Sara A. Saied ,&nbsp;AbdulRahman A Saied ,&nbsp;Alshimaa M Elmalawany","doi":"10.1016/j.retram.2024.103443","DOIUrl":"https://doi.org/10.1016/j.retram.2024.103443","url":null,"abstract":"<div><h3>Background</h3><p>One of the prominent causes of chronic liver disease worldwide is the hepatitis C virus (HCV). HCV believed that innate immunity contributes to a sustained virological response (SVR) to the treatment of Sofosbuvir (SOF) (+) Daclatasvir (DCV) (+) Ribavirin (RBV). This study aimed to evaluate the impact of SOF (+) DCV (+) RBV therapy and persistent HCV infection on the subset of natural killer cells (NK) in HCV genotype four patients from Egypt.</p></div><div><h3>Materials and Methods</h3><p>One hundred and ten patients with persistent HCV infections requiring SOF (+) DCV (+) RBV therapy were grouped, and a flow cytometry (FCM) study of the NK cell subset in peripheral blood was performed. The assessment was performed before and after three and/or six months of the cessation of viral suppression therapy when a patient had a long-term viral response (SVR). One hundred and ten volunteers from the National Liver Institute<sup>’</sup>s (NLI) blood bank were selected as controls.</p></div><div><h3>Results</h3><p>Patients with chronic HCV infection before therapy had considerably lower CD16<sup>+</sup> and CD3<sup>−</sup> CD56<sup>+</sup> cells than controls. Their levels increase during SOF (+) DCV (+) RBV therapy. In patients with SVR during treatment, CD16<sup>+</sup> and CD3<sup>–</sup> CD56<sup>+</sup> cells increased significantly compared to those who did not get SVR. Furthermore, CD56<sup>+</sup> cells were significantly higher in patients with persistent infection before treatment than controls but diminished with the response to treatment.</p></div><div><h3>Conclusion</h3><p>: NK cell activation following SOF (+) DCV (+) RBV therapy and polarization to cytotoxicity occurred early in HCV antiviral therapy and was elevated in the respondents. Our data illustrated that establishing an inhibitory cytotoxic NK profile is related to therapeutic outcomes.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 3","pages":"Article 103443"},"PeriodicalIF":4.1,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140042812","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}
引用次数: 0
In vivo and in vitro effects of cord blood hematopoietic stem and progenitor cell (HSPC) expansion using valproic acid and/or nicotinamide 使用丙戊酸和/或烟酰胺扩增脐带血造血干细胞和祖细胞(HSPC)的体内和体外效应
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-03-01 DOI: 10.1016/j.retram.2024.103444
Emine Begum Gencer , Hasan Yalim Akin , Selami Kocak Toprak , Eylul Turasan , Mahsa Yousefzadeh , Pinar Yurdakul-Mesutoglu , Murat Cagan , Mehmet Murat Seval , Doruk Cevdi Katlan , Klara Dalva , Mehmet Sinan Beksac , Meral Beksac
{"title":"In vivo and in vitro effects of cord blood hematopoietic stem and progenitor cell (HSPC) expansion using valproic acid and/or nicotinamide","authors":"Emine Begum Gencer ,&nbsp;Hasan Yalim Akin ,&nbsp;Selami Kocak Toprak ,&nbsp;Eylul Turasan ,&nbsp;Mahsa Yousefzadeh ,&nbsp;Pinar Yurdakul-Mesutoglu ,&nbsp;Murat Cagan ,&nbsp;Mehmet Murat Seval ,&nbsp;Doruk Cevdi Katlan ,&nbsp;Klara Dalva ,&nbsp;Mehmet Sinan Beksac ,&nbsp;Meral Beksac","doi":"10.1016/j.retram.2024.103444","DOIUrl":"https://doi.org/10.1016/j.retram.2024.103444","url":null,"abstract":"<div><h3>Background</h3><p>High self-renewal capacity and most permissive nature of umbilical cord blood (CB) results with successful transplant outcomes but low hematopoietic stem and progenitor cell (HSPC) counts limits wider use. In order to overcome this problem <em>ex vivo</em> expansion with small molecules such as Valproic acid (VPA) or Nicotinamide (NAM) have been shown to be effective. To the best of our knowledge, the combinatory effects of VPA and NAM on HSPC expansion has not been studied earlier. The aim of this study was to analyze <em>ex vivo</em> and <em>in vivo</em> efficacy of VPA and NAM either alone or in combination in terms of expansion and engraftment.</p></div><div><h3>Methods</h3><p>A total of 44 CB units were included in this study. To determine the <em>ex vivo</em> and <em>in vivo</em> efficacy, human CB CD34+ cells were expanded with VPA and/or NAM and colony forming unit (CFU) assay was performed on expanded HSPC. Xenotransplantation was performed simultaneously by intravenous injection of expanded HSPC to <em>NOD-SCID</em> gamma (NSG) mice (<em>n</em> = 22). Significance of the difference between the expansion groups or xenotransplantation models was analyzed using <em>t</em>-test, Mann-Whitney, ANOVA or Kruskal-Wallis tests as appropriate considering the normality of distributions and the number of groups analyzed.</p></div><div><h3>Results</h3><p><em>In vitro</em> CD34+ HSPC expansion fold relative to cytokines-only was significantly higher with VPA compared to NAM [2.23 (1.07–5.59) <em>vs</em> 1.48 (1.00–4.40); <em>p</em> &lt; 0.05]. Synergistic effect of VPA+NAM has achieved a maximum relative expansion fold at 21 days (D21) of incubation [2.95 (1.00–11.94)]. There was no significant difference between VPA and VPA+NAM D21 (<em>p</em> = 0.44). Fold number of colony-forming unit granulocyte-macrophage (CFU-GM) colonies relative to the cytokine-only group was in favor of NAM compared to VPA [1.87 (1.00–3.59) <em>vs</em> 1.00 (1.00–1.81); <em>p</em> &lt; 0.01]. VPA+NAM D21 [1.62 (1.00–2.77)] was also superior against VPA (<em>p</em> &lt; 0.05). There was no significant difference between NAM and VPA+NAM D21. Following human CB34+ CB transplantation (CBT) in the mouse model, fastest <em>in vivo</em> leukocyte recovery was observed with VPA+NAM expanded cells (6 ± 2 days) and the highest levels of human CD45 chimerism was detectable with VPA-expanded CBT (VPA: 5.42 % at day 28; NAM: 2.45 % at day 31; VPA+NAM 1.8 % at day 31).</p></div><div><h3>Conclusion</h3><p>Our study results suggest using VPA alone, rather than in combination with NAM or NAM alone, to achieve better and faster expansion and engraftment of CB HSPC.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 3","pages":"Article 103444"},"PeriodicalIF":4.1,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140042813","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}
引用次数: 0
Role of next-generation sequencing in acquired amegakaryocytic thrombocytopenic purpura 新一代测序技术在获得性巨核细胞性血小板减少性紫癜中的作用
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-03-01 DOI: 10.1016/j.retram.2024.103441
Lorenzo Lazzari , Lucia Bongiovanni , Paola Ronchi , Gregorio Maria Bergonzi , Camilla Gariazzo , Elisa Diral , Fabio Ciceri , Andrea D'Alessio , Maurilio Ponzoni
{"title":"Role of next-generation sequencing in acquired amegakaryocytic thrombocytopenic purpura","authors":"Lorenzo Lazzari ,&nbsp;Lucia Bongiovanni ,&nbsp;Paola Ronchi ,&nbsp;Gregorio Maria Bergonzi ,&nbsp;Camilla Gariazzo ,&nbsp;Elisa Diral ,&nbsp;Fabio Ciceri ,&nbsp;Andrea D'Alessio ,&nbsp;Maurilio Ponzoni","doi":"10.1016/j.retram.2024.103441","DOIUrl":"10.1016/j.retram.2024.103441","url":null,"abstract":"","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 1","pages":"Article 103441"},"PeriodicalIF":4.1,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139590521","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}
引用次数: 0
Application of chimeric antigen receptor therapy beyond oncology: A bibliometric and visualized analysis 嵌合抗原受体疗法在肿瘤学之外的应用:文献计量与可视化分析
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-02-06 DOI: 10.1016/j.retram.2024.103442
Linxin Yang , Jinshen He , Jiahao Liu , Tianjian Xie , Qi Tang
{"title":"Application of chimeric antigen receptor therapy beyond oncology: A bibliometric and visualized analysis","authors":"Linxin Yang ,&nbsp;Jinshen He ,&nbsp;Jiahao Liu ,&nbsp;Tianjian Xie ,&nbsp;Qi Tang","doi":"10.1016/j.retram.2024.103442","DOIUrl":"10.1016/j.retram.2024.103442","url":null,"abstract":"<div><h3>Purpose</h3><p>Chimeric antigen receptor therapy beyond oncology has gained increasing attention. While a substantial number of publications have emerged in recent years, there has been a paucity of conducted bibliometric studies. Our objective is to systematically summarize and visually analyze the literature in the field of chimeric antigen receptors therapy beyond oncology and explore hotspots in this field.</p></div><div><h3>Methods</h3><p>Web of Science Core Collection was selected as the data source, and the data was retrieved on December 23, 2022, according to the search strategy. CiteSpace 6.1.R6 and Vosviewer 1.6.18 were used to analyze publications and explore research hotspots and directions.</p></div><div><h3>Results</h3><p>A total of 338 publications written by 1832 authors from 516 institutions in 42 countries/regions were selected for the analysis. The number of publications is steadily increasing annually. The United States emerged as the primary contributor, and University of Pennsylvania was the leading institution. Frontiers in Immunology boasted the highest number of published papers. Kitchen SG, Riley JL, and Scott DW were the most productive researchers in this field. The keyword cluster analysis identified HIV, autoimmune diseases, transplant related diseases and COVID-19 as the primary focus areas within the realm of chimeric antigen receptor therapy beyond oncology.</p></div><div><h3>Conclusion</h3><p>The advancement of chimeric antigen receptor therapy beyond oncology has witnessed rapid progress in recent years. We have explored the hotspots and research directions in this field. It is hoped that this study could provide references and directions for future clinical researches.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 3","pages":"Article 103442"},"PeriodicalIF":4.1,"publicationDate":"2024-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139769994","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}
引用次数: 0
The clinical applications of immunosequencing 免疫测定的临床应用
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-01-12 DOI: 10.1016/j.retram.2024.103439
B. Bardwell , J. Bay , Z. Colburn
{"title":"The clinical applications of immunosequencing","authors":"B. Bardwell ,&nbsp;J. Bay ,&nbsp;Z. Colburn","doi":"10.1016/j.retram.2024.103439","DOIUrl":"10.1016/j.retram.2024.103439","url":null,"abstract":"<div><p>Technological advances in high-throughput sequencing have opened the door for the interrogation of adaptive immune responses at unprecedented scale. It is now possible to determine the sequences of antibodies or T-cell receptors produced by individual B and T cells in a sample. This capability, termed immunosequencing, has transformed the study of both infectious and non-infectious diseases by allowing the tracking of dynamic changes in B and T cell clonal populations over time. This has improved our understanding of the pathology of cancers, autoimmune diseases, and infectious diseases. However, to date there has been only limited clinical adoption of the technology. Advances over the last decade and on the horizon that reduce costs and improve interpretability could enable widespread clinical use. Many clinical applications have been proposed and, while most are still undergoing research and development, some methods relying on immunosequencing data have been implemented, the most widespread of which is the detection of measurable residual disease. Here, we review the diagnostic, prognostic, and therapeutic applications of immunosequencing for both infectious and non-infectious diseases.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 3","pages":"Article 103439"},"PeriodicalIF":4.1,"publicationDate":"2024-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139461405","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}
引用次数: 0
Cytogenetics in the management of hematological malignancies: An overview of alternative technologies for cytogenetic characterization 血液恶性肿瘤治疗中的细胞遗传学:细胞遗传学特征描述替代技术概述
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-01-12 DOI: 10.1016/j.retram.2024.103440
Valentin Lestringant , Hélène Guermouche-Flament , Mélanie Jimenez-Pocquet , Jean-Baptiste Gaillard , Dominique Penther
{"title":"Cytogenetics in the management of hematological malignancies: An overview of alternative technologies for cytogenetic characterization","authors":"Valentin Lestringant ,&nbsp;Hélène Guermouche-Flament ,&nbsp;Mélanie Jimenez-Pocquet ,&nbsp;Jean-Baptiste Gaillard ,&nbsp;Dominique Penther","doi":"10.1016/j.retram.2024.103440","DOIUrl":"10.1016/j.retram.2024.103440","url":null,"abstract":"<div><p>Genomic characterization is an essential part of the clinical management of hematological malignancies for diagnostic, prognostic and therapeutic purposes. Although CBA and FISH are still the gold standard in hematology for the detection of CNA and SV, some alternative technologies are intended to complement their deficiencies or even replace them in the more or less near future. In this article, we provide a technological overview of these alternatives. CMA is the historical and well established technique for the high-resolution detection of CNA. For SV detection, there are emerging techniques based on the study of chromatin conformation and more established ones such as RTMLPA for the detection of fusion transcripts and RNA-seq to reveal the molecular consequences of SV. Comprehensive techniques that detect both CNA and SV are the most interesting because they provide all the information in a single examination. Among these, OGM is a promising emerging higher-solution technique that offers a complete solution at a contained cost, at the expense of a relatively low throughput per machine. WGS remains the most adaptable solution, with long-read approaches enabling very high-resolution detection of CAs, but requiring a heavy bioinformatics installation and at a still high cost. However, the development of high-resolution genome-wide detection techniques for CAs allows for a much better description of chromoanagenesis. Therefore, we have included in this review an update on the various existing mechanisms and their consequences and implications, especially prognostic, in hematological malignancies.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 3","pages":"Article 103440"},"PeriodicalIF":4.1,"publicationDate":"2024-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2452318624000035/pdfft?md5=e7561bfa72b241b6f1f7d35dd90d8c94&pid=1-s2.0-S2452318624000035-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139475391","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}
引用次数: 0
Luspatercept for the treatment of congenital sideroblastic anemia: Two case reports 鲁帕特罗治疗先天性红细胞性贫血:两例报告
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2024-01-12 DOI: 10.1016/j.retram.2024.103438
Yuanyuan Shao, Li He, Shaoxue Ding, Rong Fu
{"title":"Luspatercept for the treatment of congenital sideroblastic anemia: Two case reports","authors":"Yuanyuan Shao,&nbsp;Li He,&nbsp;Shaoxue Ding,&nbsp;Rong Fu","doi":"10.1016/j.retram.2024.103438","DOIUrl":"10.1016/j.retram.2024.103438","url":null,"abstract":"<div><p><span>Congenital sideroblastic anemia<span> (CSA) is a group of disorders caused by different genetic mutations that result in low iron utilization and ineffective erythropoiesis<span><span>. Current treatments are limited, and some patients do not respond to </span>vitamin B6<span> therapy. Luspatercept is a novel erythropoietic maturation agent approved for adult β-thalassemia and Myelodysplastic syndromes with ring sideroblasts (MDS-RS) associated with ineffective erythropoiesis. Here we report 2 patients with CSA due to mutations in </span></span></span></span><em>ALAS2</em> and <em>SLC25A38</em><span> genes who became unresponsive after a period of treatment with vitamin B6 and iron chelators but achieved transfusion independence and a markedly reduced spleen after combination with luspatercept.</span></p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 1","pages":"Article 103438"},"PeriodicalIF":4.1,"publicationDate":"2024-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139461442","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}
引用次数: 0
Autologous intraarterial pancreatic bone-marrow mononuclear cells infusion in T2D patients: Changes on beta-cells function, insulin resistance, and inflammatory marker 自体胰腺动脉内骨髓单核细胞输注治疗 T2D 患者:β 细胞功能、胰岛素抵抗和炎症标志物的变化
IF 4.1 4区 医学
Current Research in Translational Medicine Pub Date : 2023-12-29 DOI: 10.1016/j.retram.2023.103437
Farid Kurniawan , Imam Subekti , Em Yunir , Dante Saksono Harbuwono , Dyah Purnamasari , Tri Juli Edi Tarigan , Wismandari Wisnu , Dicky Levenus Tahapary , Syahidatul Wafa , Cindy Astrella , Eunike Vania Christabel , Anna Mira Lubis , Ika Prasetya Wijaya , Birry Karim , Mohamad Syahrir Azizi , Indrati Suroyo , Sahat Matondang , Krishna Pandu Wicaksono , Dewi Wulandari , Iqbal Fasha , Pradana Soewondo
{"title":"Autologous intraarterial pancreatic bone-marrow mononuclear cells infusion in T2D patients: Changes on beta-cells function, insulin resistance, and inflammatory marker","authors":"Farid Kurniawan ,&nbsp;Imam Subekti ,&nbsp;Em Yunir ,&nbsp;Dante Saksono Harbuwono ,&nbsp;Dyah Purnamasari ,&nbsp;Tri Juli Edi Tarigan ,&nbsp;Wismandari Wisnu ,&nbsp;Dicky Levenus Tahapary ,&nbsp;Syahidatul Wafa ,&nbsp;Cindy Astrella ,&nbsp;Eunike Vania Christabel ,&nbsp;Anna Mira Lubis ,&nbsp;Ika Prasetya Wijaya ,&nbsp;Birry Karim ,&nbsp;Mohamad Syahrir Azizi ,&nbsp;Indrati Suroyo ,&nbsp;Sahat Matondang ,&nbsp;Krishna Pandu Wicaksono ,&nbsp;Dewi Wulandari ,&nbsp;Iqbal Fasha ,&nbsp;Pradana Soewondo","doi":"10.1016/j.retram.2023.103437","DOIUrl":"10.1016/j.retram.2023.103437","url":null,"abstract":"<div><h3>Background</h3><p>Type 2 diabetes (T2D) is a progressive disease. Many drugs<span> currently being used for the management of T2D have minimal effect on pancreatic beta cells regeneration. Cell-based therapies might provide potential benefits in this aspect.</span></p></div><div><h3>Methods</h3><p>A pilot study in five T2D patients with 12 months follow-up was performed to evaluate the effect of autologous bone marrow mononuclear stem cells (BM-MNCs) infusion into pancreatic arteries on the insulin requirement, beta-cell function, insulin resistance, and systemic inflammatory marker (CRP).</p></div><div><h3>Results</h3><p>The primary endpoint, a 50 % reduction of total insulin doses from baseline, was not achieved in this study. However, a trend of increasing fasting C-peptide (<em>p</em> = 0.07) and C-peptide 60′ (<em>p</em> = 0.07) and 90′ (<em>p</em><span> = 0.07) after a mixed-meal tolerance test was observed 12 months post-infusion compared to baseline levels. A similar result was observed for the homeostatic model assessment of beta cell function (HOMA1-B), an index for beta cell function. No improvement was observed for insulin resistance measured by homeostasis model assessment of insulin resistance (HOMA1-IR) and systemic inflammatory parameter.</span></p></div><div><h3>Conclusion</h3><p>Intraarterial pancreatic autologous BM-MNCs infusion might potentially improve beta cell function in T2D patients, although further study is needed to confirm this finding.</p></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"72 2","pages":"Article 103437"},"PeriodicalIF":4.1,"publicationDate":"2023-12-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139068872","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}
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