Blood Reviews最新文献_第8页

Androgen use in bone marrow failures and myeloid neoplasms: Mechanisms of action and a systematic review of clinical data 雄激素在骨髓衰竭和髓系肿瘤中的应用:作用机制和临床数据的系统回顾

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101132

Alessandro Bosi , Wilma Barcellini , Francesco Passamonti , Bruno Fattizzo

{"title":"Androgen use in bone marrow failures and myeloid neoplasms: Mechanisms of action and a systematic review of clinical data","authors":"Alessandro Bosi , Wilma Barcellini , Francesco Passamonti , Bruno Fattizzo","doi":"10.1016/j.blre.2023.101132","DOIUrl":"10.1016/j.blre.2023.101132","url":null,"abstract":"<div>Despite recent advancements, treatment of cytopenia due to bone marrow failures (BMF) and myeloid neoplasms remains challenging. Androgens promote renewal and maturation of blood cells and may be beneficial in these forms. Here we report a systematic review of androgens use as single agent in hematologic conditions. Forty-six studies, mainly retrospective with various androgen types and doses, were included: 12 on acquired aplastic anemia (AA), 11 on inherited BMF, 17 on myelodysplastic syndromes (MDS), and 7 on myelofibrosis. Responses ranged from 50 to 70% in inherited BMF, 40–50% in acquired AA and MDS, while very limited evidence emerged for myelofibrosis. In acquired AA, response was associated with presence of non-severe disease; in MDS androgens were more effective on thrombocytopenia or mild to moderate anemia, whilst limited benefit was observed for transfusion dependent anemia. Toxicity profile mainly consisted of virilization and liver enzyme elevation, whilst the risk of leukemic evolution remains controversial.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0268960X23000930/pdfft?md5=e0ff73661365a41b146a8ef659a6466b&pid=1-s2.0-S0268960X23000930-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10234224","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

The development of post-transplant cyclophosphamide: Half a century of translational team science 移植后环磷酰胺的发展:半个世纪的转化团队科学

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2022.101034

Paul V. O'Donnell , Richard J. Jones

{"title":"The development of post-transplant cyclophosphamide: Half a century of translational team science","authors":"Paul V. O'Donnell , Richard J. Jones","doi":"10.1016/j.blre.2022.101034","DOIUrl":"10.1016/j.blre.2022.101034","url":null,"abstract":"<div>Close HLA matching of donors and recipients has been the dogma for successful allogeneic blood or marrow transplantation (alloBMT), to limit the complications of graft-versus-host disease (GVHD). However, many patients in need, especially those within certain ethnic groups such as those of African-Americans and Hispanics, remain unable to find a match even with the increased availability of unrelated donors. Over half a century ago, investigators at Johns Hopkins found that cyclophosphamide's immunosuppressive properties made it the ideal replacement for total body irradiation in alloBMT conditioning regimens. They also found it to be the best chemotherapeutic for preventing GVHD in animal models, but its cytotoxic properties scared them from using it clinically in the high doses successful in animal models. Subsequent work showed that cyclophosphamide spared hematopoietic and other stem cells including memory lymphocytes, prompting re-examination at high doses for GVHD prophylaxis. Animal and extensive human studies demonstrated that high-dose post-transplantation cyclophosphamide (PTCy) effectively and safely limited GVHD such that mismatched transplants are now considered standard-of-care worldwide. The beneficial effects of PTCy on GVHD appears to be independent of donor type, graft source, or conditioning regimen intensity.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40496358","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: 2

The next horizon now that everyone has a donor: Precision allogeneic transplantation 现在每个人都有一个供体的下一个前景是:精确的同种异体移植

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2022.100990

Richard J. Jones , Andrea Bacigalupo

{"title":"The next horizon now that everyone has a donor: Precision allogeneic transplantation","authors":"Richard J. Jones , Andrea Bacigalupo","doi":"10.1016/j.blre.2022.100990","DOIUrl":"10.1016/j.blre.2022.100990","url":null,"abstract":"<div>Post-transplant cyclophosphamide (PTCy) allows safe and effective partially matched donor allogeneic blood or marrow transplantation (alloBMT), so that almost everyone in need of the procedure now has a donor. Moreover, PTCy and other recent advances have lowered alloBMT mortality rates to less than half of that seen before the turn of the century, at costs that are substantially less than most newly approved anticancer agents. These advances also make tailoring BMT based on patients' unique diseases and characteristics now feasible for further improving outcomes. Personalizing every aspect of alloBMT, including conditioning, donor, graft type, and post-transplant maintenance is now possible. For example, alloBMT's antitumor activity historically was restricted to the allogeneic graft-versus-tumor effect directed against histocompatibility antigens. However, replacing exhausted immune systems with healthy non-exhausted, non-tolerant ones likely can enhance the activity of novel targeted therapies. The impressive results seen with tyrosine kinase inhibitors after alloBMT for patients with both Ph+ acute lymphoblastic leukemia and FLT/ITD+ acute myeloid leukemia herald the potential of precision BMT.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40569917","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: 2

Aiming for the cure in myeloma: Putting our best foot forward 以治愈骨髓瘤为目标:迈出最好的一步

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101116

Noffar Bar , Ross S. Firestone , Saad Z. Usmani

引用次数: 1

Post-transplantation cyclophosphamide as GVHD prophylaxis in allogenic hematopoietic stem cell transplantation: Recent advances and modification 移植后环磷酰胺在同种异体造血干细胞移植中预防GVHD:最新进展和改进

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101078

Yun Li , Na Wang , Xiaoying Zhang , Yang Cao , Lingfeng Zhang , Aiguo Liu , Yicheng Zhang

引用次数: 1

Relapse after allogeneic transplantation with post-transplant cyclophosphamide: Shattering myths and evolving insight 同种异体环磷酰胺移植后复发:粉碎神话和不断发展的见解

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101093

Shannon R. McCurdy , Leo Luznik

{"title":"Relapse after allogeneic transplantation with post-transplant cyclophosphamide: Shattering myths and evolving insight","authors":"Shannon R. McCurdy , Leo Luznik","doi":"10.1016/j.blre.2023.101093","DOIUrl":"10.1016/j.blre.2023.101093","url":null,"abstract":"<div>Early studies in allogeneic blood or marrow transplantation (alloBMT) demonstrated that HLA-mismatching was protective again relapse. However, benefits in relapse reduction were outweighed by a high risk of graft-versus-host disease (GVHD) when using conventional pharmacological immunosuppression. Post-transplant cyclophosphamide(PTCy)-based platforms abated the risk of GVHD thereby overcoming the negative effects of HLA-mismatching on survival. However, since its inception, PTCy has been shadowed by a reputation for a greater risk of relapse when compared with traditional GVHD prophylaxis. Specifically, whether PTCy reduces the anti-tumor efficacy of HLA-mismatched alloBMT by killing alloreactive T cells has been the subject of debate since the early 2000's. Here we review the many studies demonstrating the potent graft-versus-malignancy (GVM) properties of alloBMT with PTCy. We discuss the laboratory data from PTCy platforms supporting that T regulatory cells may be a major mechanism of prevention of GVHD and that natural killer (NK) cells may be early effectors of GVM. Finally, we propose potential paths to optimize GVM through selecting for class II mismatching and augmenting NK cell activity.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9534016","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

Factor XI inhibitors for the prevention of cardiovascular disease: A new therapeutic approach on the horizon?

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101119

D. Santagata , M.P. Donadini , W. Ageno

引用次数: 0

Combining PTCy and ATG for GvHD prophylaxis in non-malignant diseases 联合PTCy和ATG预防非恶性疾病的GvHD

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2022.101016

Amy E. DeZern , Robert A. Brodsky

引用次数: 3

Current approach to Waldenström macroglobulinemia 目前治疗Waldenström巨球蛋白血症的方法。

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101129

Prashant Kapoor, S. Vincent Rajkumar

{"title":"Current approach to Waldenström macroglobulinemia","authors":"Prashant Kapoor, S. Vincent Rajkumar","doi":"10.1016/j.blre.2023.101129","DOIUrl":"10.1016/j.blre.2023.101129","url":null,"abstract":"<div>Waldenström macroglobulinemia (WM) is a unique CD20+, B-cell non-Hodgkin lymphoma, characterized by lymphoplasmacytic infiltration of the bone marrow and circulating monoclonal immunoglobulin M. The clinical manifestations and outcomes of patients are highly variable. High-level evidence supports integration of monoclonal anti-CD20 antibody, rituximab, to the chemotherapy backbone to treat WM. However, its contemporary management has become more nuanced, with deeper understanding of the pathophysiology and incorporation of Bruton's tyrosine kinase (BTK) inhibitors to the treatment paradigm. Prior knowledge of the patients' MYD88L265P and CXCR4 mutation status may aid in the treatment decision-making. Currently, the two frequently utilized approaches include fixed-duration chemoimmunotherapy and BTK inhibitor-based continuous treatment until progression. Randomized trials comparing these two vastly divergent approaches are lacking. Recent studies demonstrating efficacy of B cell lymphoma-2 (BCL2) inhibitors and non-covalent BTK inhibitors in patients, previously exposed to a covalent BTK inhibitor, are a testament to the rapidly expanding options against WM.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10141367","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

Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline 了解严重先天性血友病A患者的个人目标、偏好和优先事项，为肝脏定向基因治疗提供量身定制的共同决策方法。实用指南

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101118

Giovanni Di Minno , Gaia Spadarella , Nelson Mauro Maldonato , Natascia De Lucia , Giancarlo Castaman , Raimondo De Cristofaro , Cristina Santoro , Flora Peyvandi , Anna Borrelli , Angelo Lupi , Marco Follino , Gerardo Guerrino , Filomena Morisco , Matteo Di Minno

{"title":"Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline","authors":"Giovanni Di Minno , Gaia Spadarella , Nelson Mauro Maldonato , Natascia De Lucia , Giancarlo Castaman , Raimondo De Cristofaro , Cristina Santoro , Flora Peyvandi , Anna Borrelli , Angelo Lupi , Marco Follino , Gerardo Guerrino , Filomena Morisco , Matteo Di Minno","doi":"10.1016/j.blre.2023.101118","DOIUrl":"10.1016/j.blre.2023.101118","url":null,"abstract":"<div>In clinical medicine, shared decision making (SDM) is a well-recognized strategy to enhance engagement of both patients and clinicians in medical decisions. The success of liver-directed gene therapy (GT) to transform severe congenital haemophilia A (HA) from an incurable to a curable disease has launched a shift beyond current standards of treatment. However, GT acceptance remains low in the community of HA persons. We argue for both persons with haemophilia (PWH) and specialists in HA care including clinicians, as needing SDM-oriented educational programs devoted to GT. Here, we provide an ad hoc outline to implement education to SDM and tailor clinician information on GT to individual PWHs. Based on routine key components of SDM: patient priorities; recommendations based on individual risk reduction; adverse effects; drug-drug interactions; alternatives to GT; and ongoing re-assessment of the objectives as risk factors (and individual priorities) change, this approach is finalized to exploit efficacious communication.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":null,"pages":null},"PeriodicalIF":7.4,"publicationDate":"2023-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9946319","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