血液科学(英文)最新文献

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Exosome miRNAs profiling in serum and prognostic evaluation in patients with multiple myeloma. 多发性骨髓瘤患者血清外泌体mirna谱分析和预后评估。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000160
Teng Fang, Hao Sun, Xiyue Sun, Yi He, Peixia Tang, Lixin Gong, Zhen Yu, Lanting Liu, Shiyi Xie, Tingyu Wang, Zhenshu Xu, Shuhua Yi, Gang An, Yan Xu, Guoqing Zhu, Lugui Qiu, Mu Hao
{"title":"Exosome miRNAs profiling in serum and prognostic evaluation in patients with multiple myeloma.","authors":"Teng Fang,&nbsp;Hao Sun,&nbsp;Xiyue Sun,&nbsp;Yi He,&nbsp;Peixia Tang,&nbsp;Lixin Gong,&nbsp;Zhen Yu,&nbsp;Lanting Liu,&nbsp;Shiyi Xie,&nbsp;Tingyu Wang,&nbsp;Zhenshu Xu,&nbsp;Shuhua Yi,&nbsp;Gang An,&nbsp;Yan Xu,&nbsp;Guoqing Zhu,&nbsp;Lugui Qiu,&nbsp;Mu Hao","doi":"10.1097/BS9.0000000000000160","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000160","url":null,"abstract":"<p><p>MicroRNAs (MiRNAs) carried by exosomes play pivotal roles in the crosstalk between cell components in the tumor microenvironment. Our study aimed at identifying the expression profile of exosomal miRNAs (exo-miRNAs) in the serum of multiple myeloma (MM) patients and investigating the regulation networks and their potential functions by integrated bioinformatics analysis. Exosomes in serum from 19 newly diagnosed MM patients and 9 healthy donors were isolated and the miRNA profile was investigated by small RNA sequencing. Differential expression of exo-miRNAs was calculated and target genes of miRNAs were predicted. CytoHubba was applied to identify the hub miRNAs and core target genes. The LASSO Cox regression model was used to develop the prognostic model, and the ESTIMATE immune score was calculated to investigate the correlation between the model and immune status in MM patients. The top six hub differentially expressed serum exo-miRNAs were identified. 513 target genes of the six hub exo-miRNAs were confirmed to be differentially expressed in MM cells in the Zhan Myeloma microarray dataset. Functional enrichment analysis indicated that these target genes were mainly involved in mRNA splicing, cellular response to stress, and deubiquitination. 13 core exo-miRNA target genes were applied to create a novel prognostic signature to provide risk stratification for MM patients, which is associated with the immune microenvironment of MM patients. Our study comprehensively investigated the exo-miRNA profiles in MM patients. A novel prognostic signature was constructed to facilitate the risk stratification of MM patients with distinct outcomes.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/4d/42/bs9-5-196.PMC10400059.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006850","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Diagnosis and management of adult central nervous system leukemia. 成人中枢神经系统白血病的诊断与治疗。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000162
Siyu Liu, Ying Wang
{"title":"Diagnosis and management of adult central nervous system leukemia.","authors":"Siyu Liu,&nbsp;Ying Wang","doi":"10.1097/BS9.0000000000000162","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000162","url":null,"abstract":"<p><p>Central nervous system leukemia (CNSL) is a prominent infiltration reason for therapy failing in acute leukemia. Recurrence rates and the prognosis have alleviated with current prophylactic regimens. However, the accurate stratification of relapse risk and treatment regimens for relapsed or refractory patients remain clinical challenges yet to be solved. Recently, with hematopoietic stem cell transplantation (HSCT) and chimeric antigen receptor-T (CAR-T) cellular therapy showing encouraging effects in some CNSL patients, advances in treating CNSL have already been reported. The development of molecular targeted agents as well as antibody-based drugs will provide patients with more personalized treatment. This article summarized recent research developments about risk factors, diagnosis, prevention, and treatment in adults with CNSL.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/70/47/bs9-5-141.PMC10400053.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006852","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
MXRA7 is involved in megakaryocyte differentiation and platelet production. MXRA7参与巨核细胞分化和血小板产生。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000167
Zhenjiang Sun, Benfang Wang, Ying Shen, Kunpeng Ma, Ting Wang, Yiqiang Wang, Dandan Lin
{"title":"MXRA7 is involved in megakaryocyte differentiation and platelet production.","authors":"Zhenjiang Sun,&nbsp;Benfang Wang,&nbsp;Ying Shen,&nbsp;Kunpeng Ma,&nbsp;Ting Wang,&nbsp;Yiqiang Wang,&nbsp;Dandan Lin","doi":"10.1097/BS9.0000000000000167","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000167","url":null,"abstract":"<p><p>Matrix remodeling is a critical process in hematopoiesis. The biology of MXRA7, as a matrix remodeling associated gene, has still not been reported in hematopoietic process. Public databases showed that MXRA7 expressed in hematopoietic stem cells, suggesting that it may be involved in hematopoiesis. We found that the amounts of megakaryocytes were lower in bone marrow and spleen from <i>Mxra7</i><sup>-/-</sup> mice compared with that from wild-type mice. Knock-out of MXRA7 also reduced the amount of platelet in peripheral blood and affected the function of platelets. Knock-out of MXRA7 inhibited hematopoietic stem/progenitor cells differentiate to megakaryocytes possibly through down-regulating the expression of <i>GATA-1</i> and <i>FOG-1</i>. Moreover, knockdown of MXRA7 in MEG-01 cells could inhibit the cell proliferation and cell apoptosis. Knockdown of MXRA7 inhibited the differentiation of MEG-01 cells and proplatelet formation through suppressing the ERK/MAPK signaling pathway and the expression of β-tubulin. In conclusion, the current study demonstrated the potential significance of MXRA7 in megakaryocyte differentiation and platelet production. The novel findings proposed a new target for the treatment of platelet-related diseases, and much more investigations are guaranteed to dissect the mechanisms of MXRA7 in megakaryocyte differentiation and platelet production.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400050/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9953313","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
An investigation of long-term outcome of rabbit anti-thymocyte globulin and cyclosporine therapy for pediatric severe aplastic anemia. 兔抗胸腺细胞球蛋白联合环孢素治疗小儿重度再生障碍性贫血的远期疗效观察。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000157
Lixian Chang, Mingchen Yan, Jingliao Zhang, Binghang Liu, Li Zhang, Ye Guo, Jing Sun, Yang Wan, Meihui Yi, Yang Lan, Yuli Cai, Yuanyuan Ren, Haihui Zheng, Aoli Zhang, Zhenyu Li, Jian Wang, Yingrui Li, Xiaofan Zhu
{"title":"An investigation of long-term outcome of rabbit anti-thymocyte globulin and cyclosporine therapy for pediatric severe aplastic anemia.","authors":"Lixian Chang,&nbsp;Mingchen Yan,&nbsp;Jingliao Zhang,&nbsp;Binghang Liu,&nbsp;Li Zhang,&nbsp;Ye Guo,&nbsp;Jing Sun,&nbsp;Yang Wan,&nbsp;Meihui Yi,&nbsp;Yang Lan,&nbsp;Yuli Cai,&nbsp;Yuanyuan Ren,&nbsp;Haihui Zheng,&nbsp;Aoli Zhang,&nbsp;Zhenyu Li,&nbsp;Jian Wang,&nbsp;Yingrui Li,&nbsp;Xiaofan Zhu","doi":"10.1097/BS9.0000000000000157","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000157","url":null,"abstract":"<p><p>Children with severe aplastic anemia (SAA) face heterogeneous prognoses after immunosuppressive therapy (IST). There are few models that can predict the long-term outcomes of IST for these patients. The objective of this paper is to develop a more effective prediction model for SAA prognosis based on clinical electronic medical records from 203 children with newly diagnosed SAA. In the early stage, a novel model for long-term outcomes of SAA patients with IST was developed using machine-learning techniques. Among the indicators related to long-term efficacy, white blood cell count, lymphocyte count, absolute reticulocyte count, lymphocyte ratio in bone-marrow smears, C-reactive protein, and the level of IL-6, IL-8 and vitamin B12 in the early stage are strongly correlated with long-term efficacy (<i>P</i> < .05). Taken together, we analyzed the long-term outcomes of rabbit anti-thymocyte globulin and cyclosporine therapy for children with SAA through machine-learning techniques, which may shorten the observation period of therapeutic effects and reduce treatment costs and time.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a4/e8/bs9-5-180.PMC10400069.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9953316","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Post-transcriptional regulation of erythropoiesis. 红细胞生成的转录后调控。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000159
Yanan Li, Haihang Zhang, Bin Hu, Pan Wang, Wei Wang, Jing Liu
{"title":"Post-transcriptional regulation of erythropoiesis.","authors":"Yanan Li,&nbsp;Haihang Zhang,&nbsp;Bin Hu,&nbsp;Pan Wang,&nbsp;Wei Wang,&nbsp;Jing Liu","doi":"10.1097/BS9.0000000000000159","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000159","url":null,"abstract":"<p><p>Erythropoiesis is a complex, precise, and lifelong process that is essential for maintaining normal body functions. Its strict regulation is necessary to prevent a variety of blood diseases. Normal erythropoiesis is precisely regulated by an intricate network that involves transcription levels, signal transduction, and various epigenetic modifications. In recent years, research on post-transcriptional levels in erythropoiesis has expanded significantly. The dynamic regulation of splicing transitions is responsible for changes in protein isoform expression that add new functions beneficial for erythropoiesis. RNA-binding proteins adapt the translation of transcripts to the protein requirements of the cell, yielding mRNA with dynamic translation efficiency. Noncoding RNAs, such as microRNAs and lncRNAs, are indispensable for changing the translational efficiency and/or stability of targeted mRNAs to maintain the normal expression of genes related to erythropoiesis. N6-methyladenosine-dependent regulation of mRNA translation plays an important role in maintaining the expression programs of erythroid-related genes and promoting erythroid lineage determination. This review aims to describe our current understanding of the role of post-transcriptional regulation in erythropoiesis and erythroid-associated diseases, and to shed light on the physiological and pathological implications of the post-transcriptional regulation machinery in erythropoiesis. These may help to further enrich our understanding of the regulatory network of erythropoiesis and provide new strategies for the diagnosis and treatment of erythroid-related diseases.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400058/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006851","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Effect of COVID-19 pandemic on blood transfusion service: an experience from a regional blood transfusion center. COVID-19大流行对输血服务的影响:来自区域输血中心的经验。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000161
Sanjay K Thakur, Anil K Sinha, Dinesh K Negi, Sompal Singh
{"title":"Effect of COVID-19 pandemic on blood transfusion service: an experience from a regional blood transfusion center.","authors":"Sanjay K Thakur,&nbsp;Anil K Sinha,&nbsp;Dinesh K Negi,&nbsp;Sompal Singh","doi":"10.1097/BS9.0000000000000161","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000161","url":null,"abstract":"<p><p>The unforeseen and uncertain life-threatening situation of the COVID-19 pandemic dramatically affected all areas of the human daily work schedule. This study was designed to assess the impact of the COVID-19 pandemic on blood transfusion services and discuss the adopted confrontation measures for uninterrupted blood supply during the pandemic situation. The data on blood donation, blood component preparation, and issue from January 2019 to December 2022 were collected from the inventory registers of the RBTC, Delhi, India. Compared to the non-pandemic year 2019, during the year 2020, all variables decreased gradually. The observed maximum decrease in variables such as blood collection (-79.16%) in the month of October, blood issue (-71.61%) in the month of August, random donor platelets (RDP) preparation (-98.09%) in the month of October, RDP issue (-86.08%) in the month of September, fresh frozen plasma (FFP) preparation (-100%) in the month of October, and FFP issue (-96.08%) in the month of July with an annual decrease of -45.52%, -42.87%, -33.00%, -59.79%, -40.98%, and -54.48%, respectively, as compared to year 2019. Compared to year 2020, in year 2021, the annual increase in blood collection, blood issue, FFP preparation, FFP issue, RDP preparation, and RDP issue was +50.20%, +21.68%, +65.31%, +78.52%, +116.23%, and +213.30%, respectively. Our study results show that the COVID-19 pandemic has significantly affected blood transfusion services at our blood bank. The adopted coping strategies to maintain the safe and uninterrupted blood transfusion chain at our blood bank gave us lessons for future preparedness if faced with a similar situation.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400046/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006853","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Erratum: Benefit of rituximab maintenance is associated with Follicular Lymphoma International Prognostic Index in patients with follicular lymphoma. 在滤泡性淋巴瘤患者中,利妥昔单抗维持的获益与滤泡性淋巴瘤国际预后指数相关。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000164
{"title":"Erratum: Benefit of rituximab maintenance is associated with Follicular Lymphoma International Prognostic Index in patients with follicular lymphoma.","authors":"","doi":"10.1097/BS9.0000000000000164","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000164","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1097/BS9.0000000000000144.].</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400060/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9953311","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Erratum: Development of Auer bodies from giant inclusions associated with rough endoplasmic reticulum in acute promyelocytic leukemia. 勘误:急性早幼粒细胞白血病中与粗内质网相关的巨包涵体的发育。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000163
{"title":"Erratum: Development of Auer bodies from giant inclusions associated with rough endoplasmic reticulum in acute promyelocytic leukemia.","authors":"","doi":"10.1097/BS9.0000000000000163","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000163","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1097/BS9.0000000000000145.].</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400052/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9953312","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Ginsenoside Rg1 improves anti-tumor efficacy of adoptive cell therapy by enhancing T cell effector functions. 人参皂苷Rg1通过增强T细胞效应物功能提高过继细胞治疗的抗肿瘤效果。
血液科学(英文) Pub Date : 2023-07-01 DOI: 10.1097/BS9.0000000000000165
Yue Liu, Lingna An, Chengfei Yang, Xiaoqi Wang, Ruihao Huang, Xi Zhang
{"title":"Ginsenoside Rg1 improves anti-tumor efficacy of adoptive cell therapy by enhancing T cell effector functions.","authors":"Yue Liu,&nbsp;Lingna An,&nbsp;Chengfei Yang,&nbsp;Xiaoqi Wang,&nbsp;Ruihao Huang,&nbsp;Xi Zhang","doi":"10.1097/BS9.0000000000000165","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000165","url":null,"abstract":"<p><p>Adoptive cell therapy (ACT) has emerged with remarkable efficacies for tumor immunotherapy. Chimeric antigen receptor (CAR) T cell therapy, as one of most promising ACTs, has achieved prominent effects in treating malignant hematological tumors. However, the insufficient killing activity and limited persistence of T cells in the immunosuppressive tumor microenvironment limit the further application of ACTs for cancer patients. Many studies have focused on improving cytotoxicity and persistence of T cells to achieve improved therapeutic effects. In this study, we explored the potential function in ACT of ginsenoside Rg1, the main pharmacologically active component of ginseng. We introduced Rg1 during the in vitro activation and expansion phase of T cells, and found that Rg1 treatment upregulated two T cell activation markers, CD69 and CD25, while promoting T cell differentiation towards a mature state. Transcriptome sequencing revealed that Rg1 influenced T cell metabolic reprogramming by strengthening mitochondrial biosynthesis. When co-cultured with tumor cells, Rg1-treated T cells showed stronger cytotoxicity than untreated cells. Moreover, adding Rg1 to the culture endowed CAR-T cells with enhanced anti-tumor efficacy. This study suggests that ginsenoside Rg1 provides a potential approach for improving the anti-tumor efficacy of ACT by enhancing T cell effector functions.</p>","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400057/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006854","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
CAR-T cell therapy in myeloma: hopes and hurdles. CAR-T细胞治疗骨髓瘤:希望与障碍。
血液科学(英文) Pub Date : 2023-04-01 DOI: 10.1097/BS9.0000000000000148
Jean Luc Harousseau
{"title":"CAR-T cell therapy in myeloma: hopes and hurdles.","authors":"Jean Luc Harousseau","doi":"10.1097/BS9.0000000000000148","DOIUrl":"https://doi.org/10.1097/BS9.0000000000000148","url":null,"abstract":"Immune therapy is a new avenue in the treatment of multiple myeloma (MM). The naked anti-38 antibodies daratumumab and isatuximab are already used in frontline therapy after excellent results have been achieved in relapsed/refractory MM (RRMM).The second step was the development of immune therapies targeting B cell maturation antigen (BCMA). Genetically mod- ified autologous chimeric antigen receptor (CAR)-T cell therapy BCMA-directed were initially tested in heavily pretreated patients with RRMM exposed to the 3 main therapeutic classes, immunomodulatory drugs (iMiDs), proteasome inhibitors, and anti-CD38 antibodies (triple-class exposed). Efficacy results were unprecedented in this context and Idecabtagene vicleucel (ide-cel or Abecma) was the first BCMA-directed CAR-T cell therapy approved in MM by both Federal Drug Administration (FDA) and European Medicines Agency (EMA). This approval was based on the results of the KarMMa Phase 2 study in 128 patients with RRMM who had previously received a median number of 6 lines of therapy (LOT). 1 The response rate (RR) was 73%, including 33% of complete response (CR) or better and 26% negative min- imal residual disease (MRD). However, median progression-free survival (PFS) was only 8.8 months. The Cartitude 1 Phase1b/2 evaluated ciltacabtagene autoleucel (cilta-cel) another CAR-T cell therapy with 2 BCMA-targeting single domain antibodies in 97 heavily pretreated patients with RRMM. 2 Results were recently updated and with a median follow-up of 27.7 months, 3 the results were even more impressive with a 98% RR, including 82.5% CR or better, and 92% negative MRD in evaluable patients. The 27-month PFS was 55%. With these results, Cilta-cel (Carvykti) was","PeriodicalId":67343,"journal":{"name":"血液科学(英文)","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10205213/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9530769","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
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