Advances in cell and gene therapy最新文献_第9页

Advances in transplantation and gene therapy in transfusion-dependent β-thalassemia 输血依赖性β -地中海贫血的移植和基因治疗进展

Advances in cell and gene therapy Pub Date : 2018-11-09 DOI: 10.1002/acg2.25

Emanuele Angelucci, Syed A. Abutalib

引用次数: 2

Targeting reservoirs of HIV replication in lymphoid follicles with cellular therapies to cure HIV 用细胞疗法靶向淋巴滤泡中的HIV复制库来治愈HIV

Advances in cell and gene therapy Pub Date : 2018-11-06 DOI: 10.1002/acg2.27

Pamela J. Skinner

引用次数: 3

Emerging role of CAR T cell therapy in multiple myeloma CAR - T细胞治疗在多发性骨髓瘤中的新作用

Advances in cell and gene therapy Pub Date : 2018-10-25 DOI: 10.1002/acg2.22

Ranjit Nair, Krina Patel

引用次数: 2

Genetics, prognosis, and transplantation for myelofibrosis 骨髓纤维化的遗传学、预后和移植

Advances in cell and gene therapy Pub Date : 2018-10-18 DOI: 10.1002/acg2.24

H. Joachim Deeg, Rachel Salit, Bart L. Scott, Janghee Woo

{"title":"Genetics, prognosis, and transplantation for myelofibrosis","authors":"H. Joachim Deeg, Rachel Salit, Bart L. Scott, Janghee Woo","doi":"10.1002/acg2.24","DOIUrl":"10.1002/acg2.24","url":null,"abstract":"Primary myelofibrosis (PMF), polycythemia vera (PV), and essential thrombocythemia (ET) are chronic disorders that may extend over years or decades. Therapy tends to be conservative, but once marrow fibrosis and peripheral cytopenias become the dominant characteristics, prognosis is poor. Hematopoietic cell transplantation (HCT) is the only treatment with curative potential, leading to survival in remission in 35%-70% of patients. However, HCT is associated with risks, and despite the development of numerous risk scoring systems, optimal timing of HCT remains controversial. The identification of “driver mutations” in JAK2, MPL1, and CALR, and prognostically relevant additional mutations, may assist in the decision-making process. While patients with type 1 CALR mutations generally have a superior prognosis, absence of all driver mutations is associated with inferior outcome. Mutations in ASXL1, SRSF2, IDH1/2, and EZH2 are linked to more rapid disease progression and, along with biallelic TP53 mutations, leukemic transformation. The strongest risk factor is the presence of multiple mutations. By MIPSS70 criteria, considering mutations, median survival was 27 years for the best risk group, but 2.3 years for the highest-risk group. The presence of nondriver mutations, particularly in the absence of CALR mutations, and association with adverse cytogenetics, should lead to consideration of HCT. But the role of mutations has to be assessed in the context of the overall presentation. Unfortunately, risk factors that affect the natural history of the disease also impact post-HCT outcome. Needed are innovative transplant strategies, also including pre-HCT and post-HCT adjuvant therapy.","PeriodicalId":72084,"journal":{"name":"Advances in cell and gene therapy","volume":"1 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1002/acg2.24","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41937476","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Dawn of chimeric antigen receptor T cell therapy in non-Hodgkin Lymphoma 嵌合抗原受体T细胞治疗非霍奇金淋巴瘤的曙光

Advances in cell and gene therapy Pub Date : 2018-10-07 DOI: 10.1002/acg2.23

Karlo Perica, M. Lia Palomba, Renier J. Brentjens

引用次数: 1

Return of gemtuzumab ozogamicin in acute myeloid leukemia—Is it for everyone with CD33+ disease? 吉妥珠单抗-奥佐格米星治疗急性粒细胞白血病的疗效——它适用于所有CD33+疾病患者吗？

Advances in cell and gene therapy Pub Date : 2018-09-24 DOI: 10.1002/acg2.21

Prajwal Boddu, Farhad Ravandi

引用次数: 0

Advances in transplantation for lymphomas resulting from CIBMTR lymphoma working committee's research portfolio: A five-year report (2013-2018) CIBMTR淋巴瘤工作委员会研究组合的淋巴瘤移植进展:五年报告(2013-2018)

Advances in cell and gene therapy Pub Date : 2018-08-30 DOI: 10.1002/acg2.17

Mehdi Hamadani

{"title":"Advances in transplantation for lymphomas resulting from CIBMTR lymphoma working committee's research portfolio: A five-year report (2013-2018)","authors":"Mehdi Hamadani","doi":"10.1002/acg2.17","DOIUrl":"10.1002/acg2.17","url":null,"abstract":"The Center for International Blood and Marrow Transplant Research (CIBMTR) is a research collaboration between the National Marrow Donor Program (NMDP)/Be The Match and the Medical College of Wisconsin (MCW). The CIBMTR collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide to increase survival and enrich quality of life for patients. The observation research program within CIBMTR is organized into 15 working committees. This review is aiming to highlight the observational research studies published by the CIBMTR Lymphoma Working committee over the last 5 years (2013-18) and to summarize how these studies have impacted the field by helping inform clinical practice in scenarios where prospective data from high quality randomized trials were not available or where owing to the rarity of a particular transplant indication such data were unlikely to be generated, outside the setting of a large observational research database. The salient findings reviewed include; (a) studies supporting role of autologous HCT in diffuse large B-cell lymphoma (DLBCL) patients with sensitive relapse of disease within 1 year of diagnosis, (b) role of autologous HCT vs allogeneic HCT in follicular lymphoma patients with early therapy failure, (c) prognostic scoring system development for classical Hodgkin lymphoma and DLBCL patients with prior autograft failure, (d) defining the role of alternative donor transplantation in lymphomas, (e) evaluating appropriate conditioning regimens for HCT in lymphoma, and (f) outcomes of HCT in rare lymphoid malignancies.","PeriodicalId":72084,"journal":{"name":"Advances in cell and gene therapy","volume":"1 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1002/acg2.17","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25469499","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Mesenchymal stromal cell infusions for acute graft-versus-host disease: Rationale, data, and unanswered questions 骨髓基质细胞输注治疗急性移植物抗宿主病：原理、数据和未回答的问题

Advances in cell and gene therapy Pub Date : 2018-08-14 DOI: 10.1002/acg2.14

Amara Seng, Neil Dunavin

引用次数: 3

HLA typing—A case-based approach to donor selection HLA分型-基于病例的供体选择方法

Advances in cell and gene therapy Pub Date : 2018-08-13 DOI: 10.1002/acg2.16

Neema P. Mayor, Bronwen E. Shaw

{"title":"HLA typing—A case-based approach to donor selection","authors":"Neema P. Mayor, Bronwen E. Shaw","doi":"10.1002/acg2.16","DOIUrl":"10.1002/acg2.16","url":null,"abstract":"In the early years of hematopoietic cell transplantation (HCT), matching of patients and donors was poorly understood and complications related to genetic (especially HLA) mismatching were profound, resulting in a very high incidence of fatal graft-versus-host disease (GVHD). This essentially limited the choice of a donor to a sibling, and as the understanding of HLA improved, defined the donor as an HLA-identical sibling. As patient outcomes improved, the use of an unrelated donor (URD) became more common. Later, with the advent of URD registries and improved HLA typing techniques, the use of an URD overtook that of an HLA-identical sibling in frequency both in Europe and in the United States (US). Increasing confidence in our ability to manage and prevent the immune complications of HCT led to the use of HLA-mismatched donors being expanded, in the setting of umbilical cord blood (UCB) and haploidentical transplantation. In 2018, it is unusual for a patient not to go to transplant due to the lack of a suitable donor option. In this manuscript, we describe the impact of genetic factors, in particular HLA, and the technologies for typing these, on the outcomes of HCT. We highlight both well-accepted donor selection practices and newer or more controversial advances in donor selection.","PeriodicalId":72084,"journal":{"name":"Advances in cell and gene therapy","volume":"1 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1002/acg2.16","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"50822197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Method comparison study of peripheral blood CD34+ count performed on an Abbott CELL-DYN Sapphire hematology analyzer versus flow cytometry reference procedure (modified ISHAGE) 雅培CELL-DYN蓝宝石血液学分析仪与流式细胞术参考程序(改进的ISHAGE)外周血CD34+计数的比较研究

Advances in cell and gene therapy Pub Date : 2018-08-13 DOI: 10.1002/acg2.15

Scott T. Avecilla, Cheryl Goss, Steven M. Marionneaux, Donald R. Wright, Tyler D. Leiva, Jo-ann Tonon, Katherine M. Smith, Peter Maslak

{"title":"Method comparison study of peripheral blood CD34+ count performed on an Abbott CELL-DYN Sapphire hematology analyzer versus flow cytometry reference procedure (modified ISHAGE)","authors":"Scott T. Avecilla, Cheryl Goss, Steven M. Marionneaux, Donald R. Wright, Tyler D. Leiva, Jo-ann Tonon, Katherine M. Smith, Peter Maslak","doi":"10.1002/acg2.15","DOIUrl":"10.1002/acg2.15","url":null,"abstract":"CD34+ cell enumeration is a critical parameter used to determine the timing of apheresis collections of hematopoietic progenitor cell products (HPC(A)). Automated hematology analyzers equipped with flow cytometry capabilities may be a solution to the problem of limited access to standard flow cytometry testing. We compared CD34+ cell enumeration using a reference flow cytometry procedure employing modified International Society of Hematotherapy and Graft Engineering (ISHAGE) analysis with a hematology analyzer/flow cytometer hybrid (CELL DYN (CD)Sapphire) using a sequential gating analysis designed to emulate the ISHAGE gating strategy. CD34+ cell values obtained from the ISHAGE and CD Sapphire analysis was plotted and compared in a linear regression analysis which showed a high degree of correlation (R2 = 0.96). No statistically significant (P = 0.53) differences in CD34+ cell enumeration values were observed between the flow cytometer and automated hematology analyzer using manual analysis schema. We have demonstrated that an automated hematology analyzer equipped with a flow module can provide CD34+ cell enumeration results in the peripheral blood for clinical decision algorithms without the need for a dedicated flow cytometry laboratory.","PeriodicalId":72084,"journal":{"name":"Advances in cell and gene therapy","volume":"1 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2018-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1002/acg2.15","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37057791","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 1