Hematology. American Society of Hematology. Education Program最新文献_第10页

Catastrophic antiphospholipid syndrome: a CAPS-tivating hematologic disease. 灾难性抗磷脂综合征：一种激活caps的血液病。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000544

Brittany M Salter, Mark Andrew Crowther

{"title":"Catastrophic antiphospholipid syndrome: a CAPS-tivating hematologic disease.","authors":"Brittany M Salter, Mark Andrew Crowther","doi":"10.1182/hematology.2024000544","DOIUrl":"10.1182/hematology.2024000544","url":null,"abstract":"Catastrophic antiphospholipid syndrome (CAPS) is a rare but life-threatening form of antiphospholipid syndrome (APS) defined by the rapid onset of large and small vessel thrombosis occurring simultaneously across multiple sites, resulting in multiorgan dysfunction. The presence of underlying immune dysfunction causing activation of coagulation and, in many cases, abnormal complement regulation predisposes these patients to thrombotic events. CAPS is often preceded by triggering factors such as infection, surgery, trauma, anticoagulation discontinuation, and malignancy. Given the high mortality rate, which may exceed 50%, prompt recognition and initiation of management is required. The detection of antiphospholipid antibodies and the histopathologic identification of microvascular ischemia via tissue biopsy are required to diagnose CAPS. However, these patients are often too unwell to obtain results and wait for them. As such, investigations should not delay CAPS therapy, especially if there is strong clinical suspicion. Management of CAPS requires \"triple therapy\" with glucocorticoids, intravenous heparin, therapeutic plasma exchange, and/or intravenous immunoglobulin. Treatment for refractory disease is based on poor-quality evidence but includes anti-CD20 (rituximab) or anticomplement (eculizumab) monoclonal antibodies and other immunosuppressant agents, either alone or in combination. The rarity of this syndrome and the subsequent lack of randomized clinical trials have led to a paucity of high-quality evidence to guide management. Continued international collaboration to expand ongoing CAPS registries will allow a better understanding of the response to newer targeted therapy.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"214-221"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11665682/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791542","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Relapsed/refractory CLL: the role of allo-SCT, CAR-T, and T-cell engagers. 复发/难治性CLL：同种异体细胞移植、CAR-T和t细胞接合物的作用

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000570

Arnon P Kater, Tanya Siddiqi

{"title":"Relapsed/refractory CLL: the role of allo-SCT, CAR-T, and T-cell engagers.","authors":"Arnon P Kater, Tanya Siddiqi","doi":"10.1182/hematology.2024000570","DOIUrl":"10.1182/hematology.2024000570","url":null,"abstract":"Chronic lymphocytic leukemia (CLL) patients who are refractory to both Bruton's tyrosine kinase and B-cell/CLL lymphoma 2 (BCL2) inhibitors face a significant treatment challenge, with limited and short-lasting disease control options. This underscores the urgent need for novel therapeutic strategies. Immunotherapy has emerged as a promising approach to address this unmet need, offering the potential for durable remissions and improved patient outcomes. Historically, allogeneic stem cell transplantation has been used for high-risk CLL patients, demonstrating promising survival rates. However, its applicability is limited by high treatment-related mortality and chronic graft-versus-host disease, especially in older and frail patients. Chimeric antigen receptor (CAR) T-cell therapy is gaining attention for its potential in relapsed/refractory CLL. Early clinical trials have shown that CAR T cells can induce durable remissions, with encouraging overall response rates in heavily pretreated patients. Additionally, bispecific antibodies are being explored as immunotherapeutic strategies, showing promising preclinical and early clinical results in targeting CLL cells effectively. One of the major challenges in CLL treatment with T-cell-based therapies is the acquired T-cell dysfunction observed in patients. To overcome these limitations, strategies such as combining targeted agents with cellular immunotherapies, modifying CAR designs, and incorporating immunomodulatory compounds into the manufacturing process are being investigated. These innovative approaches aim to enhance T-cell engagement and improve outcomes for CLL patients, offering hope for more effective and sustainable treatments in the future.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"474-481"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11665508/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791533","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

von Willebrand disease and heavy menstrual bleeding: when and how to test. 血管性血友病和月经大出血：何时以及如何检测。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000563

Juliana Perez Botero

{"title":"von Willebrand disease and heavy menstrual bleeding: when and how to test.","authors":"Juliana Perez Botero","doi":"10.1182/hematology.2024000563","DOIUrl":"10.1182/hematology.2024000563","url":null,"abstract":"von Willebrand disease (VWD) is a common inherited bleeding disorder caused by von Willebrand factor (VWF) deficiency and is an important cause of heavy menstrual bleeding in young patients. A clinical evaluation using standardized bleeding scores helps determine when screening hemostatic testing is indicated by identifying patients with a moderate or high probability of an inherited bleeding disorder. The diagnosis of VWD is made when VWF levels are under 30 IU/dL or between 30 and 50 IU/dL when there is a positive bleeding history. Activity levels above 100 IU/dL have a high negative predictive value. Multiple factors, including stress from acute bleeding and anemia, pregnancy, and medications, can affect VWF levels, hence testing for VWD is best performed when a person's health is at its baseline level, although this is not always possible in clinical practice. Variation in assay methodologies measuring VWF activity can have a significant impact on the diagnostic evaluation, and it is important for clinicians to be familiar with the limitations of the assay used by their local or reference laboratory. Genetic testing can be useful in establishing the VWD subtype and providing accurate reproductive counseling but is not required to make a diagnosis.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"376-381"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11665653/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791650","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Newborn screening initiatives for sickle cell disease in Africa. 非洲新生儿镰状细胞病筛查倡议。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000548

Obiageli E Nnodu, Chinwe Onyinye Okeke, Hezekiah Alkali Isa

引用次数: 0

Novel clinical care models for patients with sickle cell disease. 镰状细胞病患者的新型临床护理模式

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000586

Pablo Bartolucci

{"title":"Novel clinical care models for patients with sickle cell disease.","authors":"Pablo Bartolucci","doi":"10.1182/hematology.2024000586","DOIUrl":"10.1182/hematology.2024000586","url":null,"abstract":"This educational program outlines the importance of evolving clinical care models in response to increased life expectancy and variability in individual patient experiences, particularly in the context of sickle cell disease (SCD). It emphasizes the need for personalized and adaptive care models, in which the patient should play a central role, and the need for collaborative networks of physicians and caregivers, taking into account the multisystemic nature of the disease. The proposal also discusses the role of personalized medicine and technological advances, highlighting the need for a shared medical record; the balance between rare center expertise and widespread dissemination of knowledge; and the challenges in high- and low-income countries. It emphasizes the need to move toward personalized medicine, given the significant interindividual variability in both follow-up and treatment, and the introduction of more appropriate biomarkers and predictive algorithms to aid decision-making. The proposal includes real-world examples of successful adaptation in clinical care models. It concludes with a summary of the importance and benefits of evolving clinical care models and a future outlook on the evolution of clinical care in response to demographic changes. These proposals are intended to provide a comprehensive overview of the current state and future directions of clinical care models for SCD.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"618-622"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11665723/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791685","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Warfarin and heparin monitoring in antiphospholipid syndrome. 华法林和肝素监测抗磷脂综合征。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000547

Prabal Mittal, Zara Sayar, Hannah Cohen

{"title":"Warfarin and heparin monitoring in antiphospholipid syndrome.","authors":"Prabal Mittal, Zara Sayar, Hannah Cohen","doi":"10.1182/hematology.2024000547","DOIUrl":"10.1182/hematology.2024000547","url":null,"abstract":"Anticoagulation is central to the management of antiphospholipid syndrome (APS), an acquired thrombo-inflammatory disorder characterized by thrombosis (venous, arterial, or microvascular) or pregnancy morbidity, in association with persistent antiphospholipid antibodies (aPL; ie, 1 or more of lupus anticoagulant [LA], anticardiolipin, anti-beta-2- glycoprotein I, IgG, or IgM antibodies). The mainstay of anticoagulation in patients with thrombotic APS is warfarin or an alternative vitamin K antagonist (VKA) and, in certain situations, low-molecular-weight heparin (LMWH) or unfractionated heparin (UFH). Accurate assessment of anticoagulation intensity underpins optimal anticoagulant dosing for thrombus treatment or primary/secondary prevention. In patients with APS on warfarin, the international normalized ratio (INR) may not be representative of anticoagulation intensity due to an interaction between LA and the thromboplastin reagent used in the INR determination. In this review, we summarize the use of warfarin/VKA in patients with APS, along with venous and point-of-care INR monitoring. We also discuss the role and monitoring of LMWH/UFH, including in the anticoagulant refractory setting and during pregnancy.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"192-199"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11665698/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Common variable immunodeficiency: autoimmune cytopenias and advances in molecular diagnosis. 常见的可变免疫缺陷：自身免疫性细胞减少症及其分子诊断进展。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000538

Charlotte Cunningham-Rundles, Jean-Laurent Casanova, Bertrand Boisson

引用次数: 0

Supportive care in myeloma-when treating the clone alone is not enough. 骨髓瘤的支持性治疗——当单独治疗克隆时是不够的。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000579

Sonja Zweegman, Niels W C J van de Donk

引用次数: 0

Demystifying autoimmune HIT: what it is, when to test, and how to treat. 揭开自身免疫HIT的神秘面纱：它是什么，何时检测，以及如何治疗。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000565

Marie Scully, William A Lester

{"title":"Demystifying autoimmune HIT: what it is, when to test, and how to treat.","authors":"Marie Scully, William A Lester","doi":"10.1182/hematology.2024000565","DOIUrl":"10.1182/hematology.2024000565","url":null,"abstract":"Antibodies to platelet factor 4 (PF4) have been primarily linked to classical heparin-induced thrombocytopenia (cHIT). However, during the rollout of the COVID-19 vaccine program a new condition, vaccine-induced thrombocytopenia and thrombosis (VITT), was identified, related to adenoviral-based COVID-19 vaccines. The differences between these 2 conditions, both clinically and in laboratory testing, set the scene for the development of a new rapid anti-PF4 assay that is not linked with heparin (as relevant for cHIT). Concurrently, there has been a reassessment of those cases described as autoimmune HIT. Such scenarios do not follow cHIT, but there is now a clearer differentiation of heparin-dependent and heparin-independent anti-PF4 conditions. The importance of this distinction is the identification of heparin-independent anti-PF4 antibodies in a new subgroup termed VITT-like disorder. Cases appear to be rare, precipitated by infection and in a proportion of cases, orthopaedic surgery, but are associated with high mortality and the need for a different treatment pathway, which includes immunomodulation therapy.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"403-408"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11665499/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791548","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

End-of-life care for people with sickle cell disease: barriers to and facilitators of high-quality care. 镰状细胞病患者的临终护理：高质量护理的障碍和促进因素。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2024-12-06 DOI: 10.1182/hematology.2024000561

Rushil V Patel, Emily E Johnston

{"title":"End-of-life care for people with sickle cell disease: barriers to and facilitators of high-quality care.","authors":"Rushil V Patel, Emily E Johnston","doi":"10.1182/hematology.2024000561","DOIUrl":"10.1182/hematology.2024000561","url":null,"abstract":"End-of-life (EOL) care is a critical part of sickle cell disease (SCD) management. However, barriers to high-quality EOL care remain, including (1) disease-related barriers (prior opioid exposure, risk of vaso-occlusive crises, chronic conditions with conflicting needs, and limitations of receiving disease-directed therapy on hospice); (2) communication-related barriers (challenges of identifying and responding to religious and spiritual concerns, limited health literacy, and previous health care system experience); (3) systemic issues (social determinants of health, structural racism, and mistrust of the medical system). However, palliative care and interdisciplinary collaboration can overcome many of these barriers. In addition, we can improve EOL care by accounting for opioid exposures, multimodal symptom management, and exploring (1) who people want involved in decision-making, (2) the role of religion and spirituality in decision-making, and (3) previous experiences with EOL. Systemic barriers can be addressed through the social determinants of health screening, minimizing financial burdens of care, and building longitudinal relationships with people with SCD. This requires the continued education of SCD providers about primary palliative care and palliative care providers about SCD. With such strategies, high-quality EOL care is possible for this vulnerable population.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2024 1","pages":"355-362"},"PeriodicalIF":2.9,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11665722/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791556","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"教育学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0