Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder mediated by anti-platelet autoantibodies and antigen-specific T cells (1,2). These immune effectors either destroy platelets peripherally in the spleen and/or impair platelet production in the bone marrow. The most recent pathophysiologic studies have shown that abnormal T cell responses, particularly, defective T regulatory cell (Treg) activity are central to the autoimmune pathogenesis of ITP and various therapeutics can restore these responses. Just in the last 2 years, for example, there is a significant literature on ITP pathophysiology and it points to the concept that the disease is initiated by flawed self-tolerance mechanisms (1). It appears that understanding how to specifically modulate T cells in patients with ITP will undoubtedly lead to effective antigen-specific therapeutics. This issue of AOB will be dedicated to ITP with a series of manuscripts written by leading experts in the field of ITP management and treatment. It now appears that a great deal of new treatments are available for the disease, and we now have a better understanding of how these new treatments may increase platelet counts in ITP. In the first chapter, Branch (3) and colleagues re-visit what is known about the efficacy and mechanism of action of intravenous immunoglobulin (IVIg) treatment for ITP in adults. IVIg has been used for almost 40 years as a therapeutic for the treatment of ITP and was originally found to be an efficacious treatment for pediatric ITP and later for adults suffering from the disorder. The chapter delves into the success of IVIg treatment over the years and highlights its use with other therapeutics. It points out that despite its many years of use, the mechanism of action of IVIg in ITP still remains controversial with many experimentally-supported theories as to its mechanism of action. Even so, the authors suggest that IVIg will likely continue to be a first-line therapy for adult ITP, particularly when patients suffer from bleeding symptoms. In the second chapter Schmidt (4) and colleagues discuss IVIg usage in pediatric patients with ITP. In childhood ITP, morbidity is significant due to the risk of bleeding and there is a reduced health-related quality of life (HRQoL). The authors suggest that IVIg treatment accelerates the remission of thrombocytopenia in newly diagnosed ITP and reduces bleeding symptoms, but there are disadvantages such as side effects and costs. They discuss their recent randomized controlled study (TIKI) that showed that IVIg does not affect the development of chronic ITP. It appears that approximately 60% of children with newly diagnosed ITP have a sustained response to IVIg and this response is associated with long-term remission. They then move into recent molecular and clinical data, which shows that treatment responders can be identified before IVIg administration and this is associated with a transient, self-limiting ITP disease course. They c
{"title":"Focused themed issue on immune thrombocytopenia (ITP)","authors":"J. Semple, R. Kapur","doi":"10.21037/AOB-2021-01","DOIUrl":"https://doi.org/10.21037/AOB-2021-01","url":null,"abstract":"Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder mediated by anti-platelet autoantibodies and antigen-specific T cells (1,2). These immune effectors either destroy platelets peripherally in the spleen and/or impair platelet production in the bone marrow. The most recent pathophysiologic studies have shown that abnormal T cell responses, particularly, defective T regulatory cell (Treg) activity are central to the autoimmune pathogenesis of ITP and various therapeutics can restore these responses. Just in the last 2 years, for example, there is a significant literature on ITP pathophysiology and it points to the concept that the disease is initiated by flawed self-tolerance mechanisms (1). It appears that understanding how to specifically modulate T cells in patients with ITP will undoubtedly lead to effective antigen-specific therapeutics. This issue of AOB will be dedicated to ITP with a series of manuscripts written by leading experts in the field of ITP management and treatment. It now appears that a great deal of new treatments are available for the disease, and we now have a better understanding of how these new treatments may increase platelet counts in ITP. In the first chapter, Branch (3) and colleagues re-visit what is known about the efficacy and mechanism of action of intravenous immunoglobulin (IVIg) treatment for ITP in adults. IVIg has been used for almost 40 years as a therapeutic for the treatment of ITP and was originally found to be an efficacious treatment for pediatric ITP and later for adults suffering from the disorder. The chapter delves into the success of IVIg treatment over the years and highlights its use with other therapeutics. It points out that despite its many years of use, the mechanism of action of IVIg in ITP still remains controversial with many experimentally-supported theories as to its mechanism of action. Even so, the authors suggest that IVIg will likely continue to be a first-line therapy for adult ITP, particularly when patients suffer from bleeding symptoms. In the second chapter Schmidt (4) and colleagues discuss IVIg usage in pediatric patients with ITP. In childhood ITP, morbidity is significant due to the risk of bleeding and there is a reduced health-related quality of life (HRQoL). The authors suggest that IVIg treatment accelerates the remission of thrombocytopenia in newly diagnosed ITP and reduces bleeding symptoms, but there are disadvantages such as side effects and costs. They discuss their recent randomized controlled study (TIKI) that showed that IVIg does not affect the development of chronic ITP. It appears that approximately 60% of children with newly diagnosed ITP have a sustained response to IVIg and this response is associated with long-term remission. They then move into recent molecular and clinical data, which shows that treatment responders can be identified before IVIg administration and this is associated with a transient, self-limiting ITP disease course. They c","PeriodicalId":72211,"journal":{"name":"Annals of blood","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2021-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46962114","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}
: Immune thrombocytopenia (ITP) is a rare heterogeneous autoimmune bleeding disorder that causes a lower than normal circulating platelet count resulting from impaired platelet production and accelerated platelet destruction. Patients with ITP face a complex set of challenges. The multifaceted burden of living with ITP impacts the overall health-related quality of life (HRQoL) of patients and their families. Here, we review the patients’ perspective on unmet needs, and the physical and emotional burden of disease in an attempt to highlight areas where healthcare providers treating ITP can enhance their current approach to managing a patient with this rare disease. Patients want their voices heard and their experiences with ITP acknowledged beyond simply treating the platelet count. Unmet needs identified include knowledge and communication deficiencies, the need for diagnostic improvements, access issues to the most appropriate treatments, and awareness of the extent to which ITP impacts HRQoL both on physical and mental health. ITP leads to fatigue, challenges with daily activities, reduced physical functioning, anxiety, and depression. Aside from the constant risk for serious bleeding, patients experience both physical and emotional consequences living with their disease on a daily basis. Further studies are needed to clarify the nature and source of pain, anxiety, depression, and fatigue reported in both adults and pediatric patients living with ITP. 13
{"title":"Immune thrombocytopenia: the patient’s perspective","authors":"C. Kruse, A. Kruse, J. DiRaimo","doi":"10.21037/aob-20-57","DOIUrl":"https://doi.org/10.21037/aob-20-57","url":null,"abstract":": Immune thrombocytopenia (ITP) is a rare heterogeneous autoimmune bleeding disorder that causes a lower than normal circulating platelet count resulting from impaired platelet production and accelerated platelet destruction. Patients with ITP face a complex set of challenges. The multifaceted burden of living with ITP impacts the overall health-related quality of life (HRQoL) of patients and their families. Here, we review the patients’ perspective on unmet needs, and the physical and emotional burden of disease in an attempt to highlight areas where healthcare providers treating ITP can enhance their current approach to managing a patient with this rare disease. Patients want their voices heard and their experiences with ITP acknowledged beyond simply treating the platelet count. Unmet needs identified include knowledge and communication deficiencies, the need for diagnostic improvements, access issues to the most appropriate treatments, and awareness of the extent to which ITP impacts HRQoL both on physical and mental health. ITP leads to fatigue, challenges with daily activities, reduced physical functioning, anxiety, and depression. Aside from the constant risk for serious bleeding, patients experience both physical and emotional consequences living with their disease on a daily basis. Further studies are needed to clarify the nature and source of pain, anxiety, depression, and fatigue reported in both adults and pediatric patients living with ITP. 13","PeriodicalId":72211,"journal":{"name":"Annals of blood","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2021-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48985556","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}
G. Grisolia, Alessia Pinto, Giorgia Pavan, M. Capuzzo, M. Franchini
The emergence of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic is a global concern, considering both the severity of the disease, with a high mortality rate compared to that of other influenza-like viral illnesses, and the lack of a specific, effective treatment. Pregnant women with coronavirus disease 2019 (COVID-19) represent a further challenge for clinicians. Indeed, although the majority of them are asymptomatic or their SARS-CoV-2 disease has a mild to moderate course, in some cases this viral infection is accompanied by severe respiratory symptoms. In such a critical clinical setting, the already limited therapeutic armamentarium available for COVID-19 patients is further restricted in pregnant women because of the risk of fetal toxicity especially during the first trimester of gestation. Among the treatment options, the use of convalescent plasma has gained increasing interest from investigators in pregnant women, given the initial positive reports on safety and efficacy aspects of this treatment in critically ill COVID-19 patients. However, the literature data are scanty and almost limited to single case reports, considering that pregnant women are usually excluded from trials on convalescent plasma. In this narrative review, we will critically discuss the current literature evidence on the use of hyperimmune plasma during pregnancies complicated by COVID-19.
{"title":"COVID-19 and pregnancy: a narrative review on the use of convalescent plasma","authors":"G. Grisolia, Alessia Pinto, Giorgia Pavan, M. Capuzzo, M. Franchini","doi":"10.21037/AOB-2020-CP-02","DOIUrl":"https://doi.org/10.21037/AOB-2020-CP-02","url":null,"abstract":"The emergence of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic is a global concern, considering both the severity of the disease, with a high mortality rate compared to that of other influenza-like viral illnesses, and the lack of a specific, effective treatment. Pregnant women with coronavirus disease 2019 (COVID-19) represent a further challenge for clinicians. Indeed, although the majority of them are asymptomatic or their SARS-CoV-2 disease has a mild to moderate course, in some cases this viral infection is accompanied by severe respiratory symptoms. In such a critical clinical setting, the already limited therapeutic armamentarium available for COVID-19 patients is further restricted in pregnant women because of the risk of fetal toxicity especially during the first trimester of gestation. Among the treatment options, the use of convalescent plasma has gained increasing interest from investigators in pregnant women, given the initial positive reports on safety and efficacy aspects of this treatment in critically ill COVID-19 patients. However, the literature data are scanty and almost limited to single case reports, considering that pregnant women are usually excluded from trials on convalescent plasma. In this narrative review, we will critically discuss the current literature evidence on the use of hyperimmune plasma during pregnancies complicated by COVID-19.","PeriodicalId":72211,"journal":{"name":"Annals of blood","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2021-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45284307","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}
: Pathogen reduction technology (PRT) has the potential to prevent pathogen transfusion transmission from blood donor to patient by impeding the replication of bacteria, viruses and parasites in blood components. Additionally, PRT can help to guarantee blood safety in challenging situations for blood supply, as in the Ebola or Chikungunya epidemics, or in a scenario full of uncertainties such as the current SARS-CoV-2 pandemic. The Balearic Islands Blood Bank (BIBB) is one of the few blood establishments worldwide with more than 10 years of experience in the routine use of amotosalen/UVA (Intercept Blood System) and riboflavin/UVA-UVB (Mirasol PRT system) for platelets (PLTs), the use of riboflavin/UVA-UVB for plasma and with research experience in riboflavin/UVA-UVB applied to whole blood. Over the years, we have had the opportunity to evaluate PRT from different perspectives, such as clinical and hemovigilance research in adults and children, in vitro studies on PRT effects on PLTs and assessing the financial impact of PRT implementation. PRT methods offer remarkable benefits but also have certain limitations, which are important to bear in mind during the decision-making process for PRT implementation. The purpose of this study is to review the current knowledge on PRT for PLTs drawing on our experience acquired over the last decade.
{"title":"Pathogen reduction of platelets: experience of a single blood bank","authors":"T. Jimenez‐Marco","doi":"10.21037/AOB-2020-PT-03","DOIUrl":"https://doi.org/10.21037/AOB-2020-PT-03","url":null,"abstract":": Pathogen reduction technology (PRT) has the potential to prevent pathogen transfusion transmission from blood donor to patient by impeding the replication of bacteria, viruses and parasites in blood components. Additionally, PRT can help to guarantee blood safety in challenging situations for blood supply, as in the Ebola or Chikungunya epidemics, or in a scenario full of uncertainties such as the current SARS-CoV-2 pandemic. The Balearic Islands Blood Bank (BIBB) is one of the few blood establishments worldwide with more than 10 years of experience in the routine use of amotosalen/UVA (Intercept Blood System) and riboflavin/UVA-UVB (Mirasol PRT system) for platelets (PLTs), the use of riboflavin/UVA-UVB for plasma and with research experience in riboflavin/UVA-UVB applied to whole blood. Over the years, we have had the opportunity to evaluate PRT from different perspectives, such as clinical and hemovigilance research in adults and children, in vitro studies on PRT effects on PLTs and assessing the financial impact of PRT implementation. PRT methods offer remarkable benefits but also have certain limitations, which are important to bear in mind during the decision-making process for PRT implementation. The purpose of this study is to review the current knowledge on PRT for PLTs drawing on our experience acquired over the last decade.","PeriodicalId":72211,"journal":{"name":"Annals of blood","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2021-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"44121000","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}
{"title":"Hybrids and microconversions in RH genes: investigation and implication in transfusion therapy","authors":"Y. Fichou","doi":"10.21037/aob-21-55","DOIUrl":"https://doi.org/10.21037/aob-21-55","url":null,"abstract":"","PeriodicalId":72211,"journal":{"name":"Annals of blood","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46887410","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}
Yuyuan Chen, Jiawei Li, Xujia Zeng, Wenhui Yuan, Yan Xu
Objective: In order to understand the activation mechanism of γδ T cells and their role in tumor immunity and autoimmune diseases. Background: γδ T cells are a conserved population of natural lymphocytes with a variety of structural and functional heterogeneities, accounting for approximately 0.5% to 10% of total peripheral blood lymphocytes in healthy adults. As a “bridge” between innate and acquired immunity, they have an important role in tumor surveillance. Methods: γδ T cells are considered to be effective anti-tumor effector cells, which can kill tumor cells through direct and indirect methods; γδ T cells can secrete a variety of cytokines, such as tumor necrosis factor α (TNF- α ), γ -interferon (IFN- γ ), perforin, etc., thus, they own the ability to kill tumor cells directly, and can also regulate other innate and adaptive immune cells, and then achieve the purpose of indirectly killing tumor cells, thereby establishing anti-tumor immunity. A unique feature of γδ T cells is that they recognize antigens in a non-major histocompatibility complex (MHC)-restricted manner, and they have strong cytotoxicity to a variety of cancer cells, which make them have important clinical application value. Conclusions: In this review, we provide an overview of the activation mechanisms of γδ T cells and their role in tumor immunity and autoimmune diseases. These studies provide insights into γδ T cell function to facilitate more targeted approaches for tumor therapy.
{"title":"γδ T cells and their roles in immunotherapy: a narrative review","authors":"Yuyuan Chen, Jiawei Li, Xujia Zeng, Wenhui Yuan, Yan Xu","doi":"10.21037/aob-21-33","DOIUrl":"https://doi.org/10.21037/aob-21-33","url":null,"abstract":"Objective: In order to understand the activation mechanism of γδ T cells and their role in tumor immunity and autoimmune diseases. Background: γδ T cells are a conserved population of natural lymphocytes with a variety of structural and functional heterogeneities, accounting for approximately 0.5% to 10% of total peripheral blood lymphocytes in healthy adults. As a “bridge” between innate and acquired immunity, they have an important role in tumor surveillance. Methods: γδ T cells are considered to be effective anti-tumor effector cells, which can kill tumor cells through direct and indirect methods; γδ T cells can secrete a variety of cytokines, such as tumor necrosis factor α (TNF- α ), γ -interferon (IFN- γ ), perforin, etc., thus, they own the ability to kill tumor cells directly, and can also regulate other innate and adaptive immune cells, and then achieve the purpose of indirectly killing tumor cells, thereby establishing anti-tumor immunity. A unique feature of γδ T cells is that they recognize antigens in a non-major histocompatibility complex (MHC)-restricted manner, and they have strong cytotoxicity to a variety of cancer cells, which make them have important clinical application value. Conclusions: In this review, we provide an overview of the activation mechanisms of γδ T cells and their role in tumor immunity and autoimmune diseases. These studies provide insights into γδ T cell function to facilitate more targeted approaches for tumor therapy.","PeriodicalId":72211,"journal":{"name":"Annals of blood","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48808774","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}
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