Blood Reviews最新文献_第9页

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":"62 ","pages":"Article 101129"},"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":"62 ","pages":"Article 101118"},"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

Using artificial intelligence to improve body iron quantification: A scoping review 利用人工智能提高人体铁含量：范围界定综述。

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2023.101133

Abdulqadir J. Nashwan , Ibraheem M. Alkhawaldeh , Nour Shaheen , Ibrahem Albalkhi , Ibrahim Serag , Khalid Sarhan , Ahmad A. Abujaber , Alaa Abd-Alrazaq , Mohamed A. Yassin

引用次数: 0

Haploidentical transplants with a G-CSF/ATG-based protocol: Experience from China 基于G-CSF/ atg的单倍体移植:中国的经验

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-11-01 DOI: 10.1016/j.blre.2022.101035

Zheng-Li Xu, Xiao-Jun Huang

引用次数: 2

Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias: Shedding new light on an old enzyme 激活丙酮酸激酶作为罕见溶血性贫血的治疗选择:对一种旧酶的新认识

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.blre.2023.101103

Myrthe J. van Dijk , Jonathan R.A. de Wilde , Marije Bartels , Kevin H.M. Kuo , Andreas Glenthøj , Minke A.E. Rab , Eduard J. van Beers , Richard van Wijk

{"title":"Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias: Shedding new light on an old enzyme","authors":"Myrthe J. van Dijk , Jonathan R.A. de Wilde , Marije Bartels , Kevin H.M. Kuo , Andreas Glenthøj , Minke A.E. Rab , Eduard J. van Beers , Richard van Wijk","doi":"10.1016/j.blre.2023.101103","DOIUrl":"10.1016/j.blre.2023.101103","url":null,"abstract":"<div>Novel developments in therapies for various hereditary hemolytic anemias reflect the pivotal role of pyruvate kinase (PK), a key enzyme of glycolysis, in red blood cell (RBC) health. Without PK catalyzing one of the final steps of the Embden-Meyerhof pathway, there is no net yield of adenosine triphosphate (ATP) during glycolysis, the sole source of energy production required for proper RBC function and survival. In hereditary hemolytic anemias, RBC health is compromised and therefore lifespan is shortened. Although our knowledge on glycolysis in general and PK function in particular is solid, recent advances in genetic, molecular, biochemical, and metabolic aspects of hereditary anemias have improved our understanding of these diseases. These advances provide a rationale for targeting PK as therapeutic option in hereditary hemolytic anemias other than PK deficiency. This review summarizes the knowledge, rationale, (pre)clinical trials, and future advances of PK activators for this important group of rare diseases.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"61 ","pages":"Article 101103"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10003901","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: 1

Do anemia treatments improve quality of life and physical function in patients with myelodysplastic syndromes (MDS)? A systematic review 贫血治疗能改善骨髓增生异常综合征(MDS)患者的生活质量和身体功能吗?系统回顾

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.blre.2023.101114

Allison Mo , Matthew Poynton , Erica Wood , Jake Shortt , Susan J. Brunskill , Carolyn Doree , Josie Sandercock , Nicholas Saadah , Edwin Luk , Simon J. Stanworth , Zoe McQuilten

{"title":"Do anemia treatments improve quality of life and physical function in patients with myelodysplastic syndromes (MDS)? A systematic review","authors":"Allison Mo , Matthew Poynton , Erica Wood , Jake Shortt , Susan J. Brunskill , Carolyn Doree , Josie Sandercock , Nicholas Saadah , Edwin Luk , Simon J. Stanworth , Zoe McQuilten","doi":"10.1016/j.blre.2023.101114","DOIUrl":"10.1016/j.blre.2023.101114","url":null,"abstract":"<div>Anemia is common in Myelodysplastic Syndromes (MDS). Different anemia treatments have been tested in clinical studies, but the full impact on patients' health-related quality of life (HRQoL) and physical function is unknown. The main aim of this review was to assess whether improvements in anemia are associated with changes in HRQoL/physical function.Twenty-six full-text publications were identified, enrolling 2211 patients: nine randomized trials (RCTs), fourteen non-randomized studies of interventions and three cross-sectional studies. Interventions included: growth factors/erythropoiesis-stimulating agents (n = 14), red cell transfusion (n = 9), erythroid maturation agents (n = 1), or a combination (n = 2). Five RCTs reported no changes in HRQoL despite erythroid response to the intervention, raising the question of whether anemia treatment alone can effectively improve HRQoL. Many studies were considered at high risk of bias for assessing HRQoL. There is a pressing need for future clinical trials to better define the nature of the relationship between anemia and HRQoL/functional outcomes.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"61 ","pages":"Article 101114"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10007256","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

Central nervous system prophylaxis in diffuse large B-cell lymphoma: What does the evidence tell us? 弥漫性大B细胞淋巴瘤的中枢神经系统预防：证据告诉我们什么？

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.blre.2023.101101

Jeffrey Lantz , Craig A. Portell , Emily C. Ayers

{"title":"Central nervous system prophylaxis in diffuse large B-cell lymphoma: What does the evidence tell us?","authors":"Jeffrey Lantz , Craig A. Portell , Emily C. Ayers","doi":"10.1016/j.blre.2023.101101","DOIUrl":"10.1016/j.blre.2023.101101","url":null,"abstract":"<div>Secondary involvement of the central nervous system (CNS) by diffuse large b-cell lymphoma (DLBCL) is a rare yet often catastrophic event for DLBCL patients. As standard first-line therapy for DLBCL with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) does not cross the blood-brain barrier, one approach to lessen the risk of CNS relapse has been to include additional agents, primarily methotrexate, directed at the CNS with standard R-CHOP although the timing, dose, and mode of administration differs widely across treating physicians. This practice derives from decades of non-randomized, often retrospective data with inconsistent outcomes. The current available tools and risk models are imprecise in their ability to predict which patients are truly at risk of secondary CNS relapse and more recent, large-scale real-world analyses call into question these longstanding practices. In a field lacking any prospective, randomized studies, this review synthesizes the available data investigating the utility of CNS prophylaxis in patients with DLBCL receiving 1st line therapy.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"61 ","pages":"Article 101101"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9985826","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

Future prospects for the clinical transfusion of pig red blood cells 猪红细胞临床输血的前景展望

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.blre.2023.101113

Yevgen Chornenkyy , Takayuki Yamamoto , Hidetaka Hara , Sean R. Stowell , Ionita Ghiran , Simon C. Robson , David K.C. Cooper

{"title":"Future prospects for the clinical transfusion of pig red blood cells","authors":"Yevgen Chornenkyy , Takayuki Yamamoto , Hidetaka Hara , Sean R. Stowell , Ionita Ghiran , Simon C. Robson , David K.C. Cooper","doi":"10.1016/j.blre.2023.101113","DOIUrl":"10.1016/j.blre.2023.101113","url":null,"abstract":"<div>Transfusion of allogeneic human red blood cell (hRBCs) is limited by supply and compatibility between individual donors and recipients. In situations where the blood supply is constrained or when no compatible RBCs are available, patients suffer. As a result, alternatives to hRBCs that complement existing RBC transfusion strategies are needed. Pig RBCs (pRBCs) could provide an alternative because of their abundant supply, and functional similarities to hRBCs. The ability to genetically modify pigs to limit pRBC immunogenicity and augment expression of human ‘protective’ proteins has provided major boosts to this research and opens up new therapeutic avenues. Although deletion of expression of xenoantigens has been achieved in genetically-engineered pigs, novel genetic methods are needed to introduce human ‘protective’ transgenes into pRBCs at the high levels required to prevent hemolysis and extend RBC survival in vivo. This review addresses recent progress and examines future prospects for clinical xenogeneic pRBC transfusion.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"61 ","pages":"Article 101113"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10119900","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: 1

Artificial intelligence in sickle disease 镰状病中的人工智能

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.blre.2023.101102

Ahmed Adel Elsabagh , Mohamed Elhadary , Basel Elsayed , Amgad Mohamed Elshoeibi , Khaled Ferih , Rasha Kaddoura , Salam Alkindi , Awni Alshurafa , Mona Alrasheed , Abdullah Alzayed , Abdulrahman Al-Abdulmalek , Jaffer Abduljabber Altooq , Mohamed Yassin

引用次数: 4

Primary and secondary immune thrombocytopenia (ITP): Time for a rethink 原发性和继发性免疫性血小板减少症（ITP）：是时候重新思考了

IF 7.4 2区医学

Blood Reviews Pub Date : 2023-09-01 DOI: 10.1016/j.blre.2023.101112

Tomás José González-López , Drew Provan , Abelardo Bárez , Angel Bernardo-Gutiérrez , Silvia Bernat , Daniel Martínez-Carballeira , Isidro Jarque-Ramos , Inmaculada Soto , Reyes Jiménez-Bárcenas , Fernando Fernández-Fuertes

{"title":"Primary and secondary immune thrombocytopenia (ITP): Time for a rethink","authors":"Tomás José González-López , Drew Provan , Abelardo Bárez , Angel Bernardo-Gutiérrez , Silvia Bernat , Daniel Martínez-Carballeira , Isidro Jarque-Ramos , Inmaculada Soto , Reyes Jiménez-Bárcenas , Fernando Fernández-Fuertes","doi":"10.1016/j.blre.2023.101112","DOIUrl":"10.1016/j.blre.2023.101112","url":null,"abstract":"<div>There are not many publications that provide a holistic view of the management of primary and secondary ITP as a whole, reflecting the similarities and differences between the two. Given the lack of major clinical trials, we believe that comprehensive reviews are much needed to guide the diagnosis and treatment of ITP today. Therefore, our review addresses the contemporary diagnosis and treatment of ITP in adult patients. With respect to primary ITP we especially focus on establishing the management of ITP based on the different and successive lines of treatment. Life-threatening situations, “bridge therapy” to surgery or invasive procedures and refractory ITP are also comprehensively reviewed here. Secondary ITP is studied according to its pathogenesis by establishing three major differential groups: Immune Thrombocytopenia due to Central Defects, Immune Thrombocytopenia due to Blocked Differentiation and Immune Thrombocytopenia due to Defective Peripheral Immune Response. Here we provide an up-to-date snapshot of the current diagnosis and treatment of ITP, including a special interest in addressing rare causes of this disease in our daily clinical practice. The target population of this review is adult patients only and the target audience is medical professionals.</div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"61 ","pages":"Article 101112"},"PeriodicalIF":7.4,"publicationDate":"2023-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10006747","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