Transfusion Medicine Reviews最新文献

{"title":"Advances in Acquired Hemophilia A","authors":"Jacqueline N Poston ,&nbsp;Rebecca Kruse-Jarres","doi":"10.1016/j.tmrv.2022.07.001","DOIUrl":"10.1016/j.tmrv.2022.07.001","url":null,"abstract":"<div><p>Acquired Hemophilia A (AHA) is a rare, life-threatening bleeding disorder from autoantibodies against clotting factor VIII. These autoantibodies occur with increasing incidence with advanced age and are often associated with other medical conditions such as autoimmune diseases and malignancy. Not uncommonly, AHA presents as a new bleeding disorder in a person with prior thrombosis or thrombotic risk. Treatment of AHA focuses on managing and preventing bleeding, as well as immunosuppression with the goal to eradicate the autoantibody. Despite current treatment approaches, morbidity, and mortality are high due to complications from bleeding, immunosuppression, and underlying comorbidities. The most pressing needs to improved outcome for this disease are better bleeding prophylaxis in the outpatient setting and reduction of the need for intense immunosuppression. Because of the rare nature of this disease, there is limited prospective data and most treatment standards have been based on case series. The field has recently focused on improved diagnostics and advanced risk stratification, with a potential of tailoring the need and intensity of immunosuppression. Case reports of off label use of emicizumab, a factor FVIII mimetic approved for congenital hemophilia A, suggest emicizumab may provide effective and safe bleeding prophylaxis in the outpatient setting; this could permit reducing immunosuppression and decreasing the risk of treatment related infections. Two ongoing prospective clinical trials of emicizumab will help clarify the safety and efficacy in AHA.</p></div>","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 4","pages":"Pages 215-219"},"PeriodicalIF":4.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10457052","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}

{"title":"Emerging Therapies in Antiphospholipid Syndrome","authors":"Anne Hubben ,&nbsp;Keith R McCrae","doi":"10.1016/j.tmrv.2022.09.002","DOIUrl":"10.1016/j.tmrv.2022.09.002","url":null,"abstract":"<div><p>The antiphospholipid syndrome (APS) is the most common cause of acquired immune-mediated thrombophilia. This syndrome is broadly defined by the presence of arterial or venous thrombosis, or pregnancy morbidity, in the presence of high levels of antiphospholipid antibodies. Despite recognition of this disorder more than 50 years ago, a fundamental unifying pathogenesis has not been determined. Due to this, mechanism-based therapies for APS are not available, and current management following thrombotic events suggests anticoagulation of indeterminate duration, or for obstetric complications, heparin/low molecular weight heparin and aspirin. However, APS is an autoimmune disorder, and several approaches focused on modulating the immune response or its effectors have been employed. Those which have been most extensively studied include hydroxychloroquine, rituximab and eculizumab, an inhibitor of complement C5. In this report, we review in depth, and critique, key clinical studies of these agents. Since all of these studies are small, our conclusions are qualified. However, it appears that hydroxychloroquine may enhance the anticoagulant efficacy of vitamin K antagonists in APS patients, and that rituximab may ameliorate some of the “non-criteria” manifestations of APS. The catastrophic antiphospholipid syndrome (CAPS) is associated with diffuse thrombosis, multi-organ dysfunction, and ∼30% mortality. A high incidence of complement regulatory gene mutations, and compelling data concerning the efficacy of eculizumab in CAPS, suggests an important role for complement in this disorder. However, additional work is needed to clarify the role of complement in non-catastrophic APS, though emerging data suggests that complement inhibition may be effective in preventing thrombosis in these patients as well.</p></div>","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 4","pages":"Pages 195-203"},"PeriodicalIF":4.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9417138","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}

{"title":"Changing Paradigms in ITP Management: Newer Tools for an Old Disease","authors":"Debbie Jiang ,&nbsp;Hanny Al-Samkari ,&nbsp;Sandhya R. Panch","doi":"10.1016/j.tmrv.2022.08.003","DOIUrl":"10.1016/j.tmrv.2022.08.003","url":null,"abstract":"<div><p>Immune thrombocytopenia (ITP) is an autoimmune disease characterized by isolated thrombocytopenia that may be accompanied clinically by bleeding and reduced health-related quality of life (HRQoL). While corticosteroids, splenectomy, and various immunosuppressants (used off-label) have served as historical mainstays of ITP treatment, their use is associated with adverse effects and morbidity. Over the last 15 years, the advent of the thrombopoietin receptor agonists has revolutionized the management of chronic ITP with high response rates, durable responses, and minimal adverse effects in most patients. With four agents now FDA-approved to manage chronic ITP, there is a renewed emphasis on improving HRQoL and minimizing the toxicities associated with traditional therapies. Promising agents with diverse mechanisms of action, ranging from those targeting Bruton's Tyrosine Kinase to the neonatal Fc receptor, are currently under investigation. This review highlights recent landmark clinical trials which have made significant impacts on ITP management and ongoing drug development. In critically analyzing studies of relevance, we illustrate the changing paradigms of ITP management and how the field is advancing beyond traditional therapies.</p></div>","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 4","pages":"Pages 188-194"},"PeriodicalIF":4.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9249103","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}

{"title":"Chop-Chop: The Future of Bacterial Enzymes in Transfusion Medicine","authors":"Erik H. Klontz","doi":"10.1016/j.tmrv.2022.05.003","DOIUrl":"10.1016/j.tmrv.2022.05.003","url":null,"abstract":"<div><p>The discovery of bacterial enzymes with specificity for IgG antibodies has led to breakthroughs in several autoantibody-mediated diseases. Two such enzymes, IdeS and EndoS, degrade IgG by different mechanisms, and have separately shown promise in numerous animal models of autoimmune diseases. Recently, imlifidase (the international nonproprietary name for IdeS) has advanced to clinical trials, where it has performed remarkably well in desensitizing patients to enable kidney transplantation, and in anti-glomerular basement membrane disease. Conversely, it performed poorly in thrombotic thrombocytopenic purpura. This review summarizes the development of antibody-degrading enzymes, with a discussion of key clinical studies involving imlifidase. The future of the field is also discussed, including the use of these enzymes in other diseases, and the potential for re-dosing.</p></div>","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 4","pages":"Pages 246-251"},"PeriodicalIF":4.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10762666","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}

{"title":"Transfusion Medicine in the 21st Century: Beyond Rebalancing the Humors","authors":"Krystalyn E. Hudson ,&nbsp;James C. Zimring","doi":"10.1016/j.tmrv.2022.09.003","DOIUrl":"10.1016/j.tmrv.2022.09.003","url":null,"abstract":"","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 4","pages":"Pages 173-174"},"PeriodicalIF":4.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10397106","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}

{"title":"Plasma Cell Directed Therapy for Immune Thrombotic Thrombocytopenic Purpura (iTTP)","authors":"Melissa Chen ,&nbsp;Jake Shortt","doi":"10.1016/j.tmrv.2022.09.001","DOIUrl":"10.1016/j.tmrv.2022.09.001","url":null,"abstract":"<div><p><span>Immune thrombotic thrombocytopenic purpura (iTTP) is a microangiopathic hemolytic anemia (MAHA) underpinned by autoreactivity against the von Willebrand factor (vWF) cleaving protease, ADAMTS13 (</span><u>a</u> <u>d</u>isintegrin <u>a</u>nd <u>m</u>etalloproteinase with a <u>t</u>hrombo<u>s</u>pondin type 1 motif, member <u>13</u>). Autoantibody mediated ADAMTS13 inhibition leads to the accumulation of ultra-large vWF multimers which activate platelets and endothelium to initiate microvascular thrombosis. In the absence of urgent therapeutic intervention, iTTP is rapidly fatal due to cumulative organ dysfunction including catastrophic neurological and cardiac sequalae. Therapeutic plasma exchange (TPE) is the mainstay of initial therapy and aims to remove pathological autoantibodies and ultra-large vWF multimers while replenishing ADAMTS13. Immunosuppression is an important treatment adjunct, as attainment of remission and successful TPE cessation is strongly associated with suppression of anti-ADAMTS13 antibody production. More recently, caplacizumab, an antibody fragment blocking the interaction between vWF multimers and platelets, has been incorporated into acute TTP management to mitigate end-organ damage while awaiting suppression of anti-ADAMTS13 activity. In most cases, remission is achieved using corticosteroids alone or in combination with the B-cell depleting antibody, rituximab. However, some patients are refractory to front-line immunosuppression in the context of ‘inhibitor boosting’ whereby the exposure to homologous plasma exacerbates the underlying autoimmune flare. As such cases have been observed in the context of likely effective B-cell depletion, it has been hypothesized that plasma cells (ie, terminally differentiated B-cells) may provide a therapy-resistant nidus of anti-ADAMTS13 production as has been demonstrated in other autoimmune disease settings. Autoreactive plasma cells can be targeted by conventional and novel therapeutics, including those developed for malignant plasma cells in the context of multiple myeloma. Here we review the rationale and evidence for plasma cell directed therapy in refractory iTTP, with a focus on the proteasome inhibitor, bortezomib, and the CD38 monoclonal antibody, daratumumab.</p></div>","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 4","pages":"Pages 204-214"},"PeriodicalIF":4.5,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10747123","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}

{"title":"Reflections of Dr. Morris Blajchman (1940-2022)","authors":"","doi":"10.1016/j.tmrv.2022.06.002","DOIUrl":"10.1016/j.tmrv.2022.06.002","url":null,"abstract":"","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 3","pages":"Pages 115-116"},"PeriodicalIF":4.5,"publicationDate":"2022-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0887796322000207/pdfft?md5=5beb61e50f21f07f6a0ce51ccf414509&pid=1-s2.0-S0887796322000207-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46205342","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}

{"title":"Journal Club","authors":"","doi":"10.1016/j.tmrv.2022.06.005","DOIUrl":"https://doi.org/10.1016/j.tmrv.2022.06.005","url":null,"abstract":"","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 3","pages":"Pages 165-172"},"PeriodicalIF":4.5,"publicationDate":"2022-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136472894","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}

{"title":"AABB's response to ‘Gender Inequities in Transfusion Medicine Society Recognition Awards’","authors":"Jay Lewis","doi":"10.1016/j.tmrv.2022.06.006","DOIUrl":"10.1016/j.tmrv.2022.06.006","url":null,"abstract":"","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 3","pages":"Page 164"},"PeriodicalIF":4.5,"publicationDate":"2022-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40646933","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}

{"title":"Production and Quality Assurance of Human Polyclonal Hyperimmune Immunoglobulins Against SARS-CoV-2","authors":"Thierry Burnouf ,&nbsp;Birgit Gathof ,&nbsp;Evan M. Bloch ,&nbsp;Renée Bazin ,&nbsp;Vincenzo de Angelis ,&nbsp;Gopal Kumar Patidar ,&nbsp;Rada M. Grubovic Rastvorceva ,&nbsp;Adaeze Oreh ,&nbsp;Ruchika Goel ,&nbsp;Naomi Rahimi-Levene ,&nbsp;Salwa Hindawi ,&nbsp;Arwa Z. Al-Riyami ,&nbsp;Cynthia So-Osman ,&nbsp;ISBT COVID-19 Convalescent Plasma Working Group (see the Appendix)","doi":"10.1016/j.tmrv.2022.06.001","DOIUrl":"10.1016/j.tmrv.2022.06.001","url":null,"abstract":"<div><p>The coronavirus disease 2019 (COVID-19) pandemic has highlighted the potential therapeutic value of early passive polyclonal immunotherapy using high-titer convalescent plasma (CCP). Human polyclonal hyperimmune immunoglobulin (HIG) has several advantages over CCP. Unlike CCP, HIG can provide standardized and controlled antibody content. It is also subjected to robust pathogen reduction rendering it virally safe and is purified by technologies demonstrated to preserve immunoglobulin neutralization capacity and Fc fragment integrity. This document provides an overview of current practices and guidance for the collection and testing of plasma rich in antibodies against Severe Acute Respiratory Coronavirus 2 (SARS-CoV-2) and its industrial fractionation for the manufacture of quality-assured and safe HIG. Considerations are also given to the production of HIG preparations in low- and middle-income countries.</p></div>","PeriodicalId":56081,"journal":{"name":"Transfusion Medicine Reviews","volume":"36 3","pages":"Pages 125-132"},"PeriodicalIF":4.5,"publicationDate":"2022-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9183240/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10387306","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}