Hematology. American Society of Hematology. Education Program最新文献_第2页

Approach to the patient with suspected hypereosinophilic syndrome. 疑似嗜酸性粒细胞增多综合征患者的治疗方法。

IF 3 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000367

Amy D Klion

{"title":"Approach to the patient with suspected hypereosinophilic syndrome.","authors":"Amy D Klion","doi":"10.1182/hematology.2022000367","DOIUrl":"https://doi.org/10.1182/hematology.2022000367","url":null,"abstract":"Hypereosinophilic syndromes (HES) are a heterogenous group of rare disorders with clinical manifestations ranging from fatigue to life-threatening endomyocardial fibrosis and thromboembolic events. Given the broad differential diagnosis of HES, a comprehensive approach is needed to identify potential secondary (treatable) causes and define end-organ manifestations. Classification by clinical HES subtype is also useful in terms of assessing prognosis and guiding therapy. Corticosteroids remain the mainstay of initial therapy in the setting of acute, life-threatening PDGFR mutation-negative HES. Whereas the recent availability of eosinophil-targeted therapies with extraordinary efficacy and little apparent toxicity is changing the treatment paradigm, especially for idiopathic HES and overlap syndromes, questions remain unanswered regarding the choice of agent, impact of combination therapies, and long-term effects of eosinophil depletion. This review provides a case-based discussion of the differential diagnosis of HES, including the classification by clinical HES subtype. Treatment options are reviewed, including novel eosinophil-targeted agents recently approved for the treatment of HES and/or other eosinophil-associated disorders. Primary (myeloid) disorders associated with hypereosinophilia are not be addressed in depth in this review.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"47-54"},"PeriodicalIF":3.0,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9821533/pdf/hem.2022000367.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10557805","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: 7

Thrombosis and anticoagulation: clinical issues of special importance to hematologists who practice in Asia. 血栓和抗凝:对在亚洲执业的血液学家特别重要的临床问题。

IF 3 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000383

Kochawan Boonyawat, Pantep Angchaisuksiri

{"title":"Thrombosis and anticoagulation: clinical issues of special importance to hematologists who practice in Asia.","authors":"Kochawan Boonyawat, Pantep Angchaisuksiri","doi":"10.1182/hematology.2022000383","DOIUrl":"https://doi.org/10.1182/hematology.2022000383","url":null,"abstract":"There are clinical issues of special importance and practice variation in the management of venous thromboembolism (VTE) and in the use of anticoagulants among hematologists who practice in Asia. In Asian-inherited thrombophilia, coagulation is disturbed due to loss-of-function mutations of protein S and protein C causing protein S and protein C deficiencies, whereas the gain-of-function factor V Leiden and prothrombin G20210A mutations are almost absent. Thrombophilia screening is not recommended in patients with VTE patients who have major provoking factors. However, it can be considered in unprovoked young patients with VTE who have a strong family history of VTE. Cancer is the most important acquired risk factor for VTE in Asians. Limited cancer screening at the initial presentation of unprovoked VTE is appropriate, especially in the elderly. Direct oral anticoagulants have been shown to have similar efficacy and reduce risk of major bleeding, including intracranial hemorrhage and bleeding requiring hospitalization, compared with warfarin. Most clinical trials evaluating therapies for treatment and prevention of VTE have included small numbers of Asian patients. Despite this lack of evidence, direct oral anticoagulants have been increasingly used in Asia for cancer-associated thrombosis. Individualized assessment of thrombotic and bleeding risks should be used for all hospitalized Asian patients when deciding on pharmacologic thromboprophylaxis. More research is needed to understand the factors that contribute to risks of VTE and anticoagulant-associated bleeding in Asian patients as these may differ from Western populations.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"474-480"},"PeriodicalIF":3.0,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9820496/pdf/hem.2022000383.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10557808","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: 2

New investigational combinations for higher-risk MDS. 针对高风险 MDS 的新研究组合。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000351

Kristin L Koenig, Uma Borate

{"title":"New investigational combinations for higher-risk MDS.","authors":"Kristin L Koenig, Uma Borate","doi":"10.1182/hematology.2022000351","DOIUrl":"10.1182/hematology.2022000351","url":null,"abstract":"Myelodysplastic syndromes (MDS) are typically a hematologic malignancy of older adults characterized by dysplastic hematopoiesis, cytopenia(s), and risk of acute myeloid leukemia transformation. The treatment approach to MDS depends largely on risk stratification of an individual's disease, most commonly using the Revised International Prognostic Scoring System, which takes into account peripheral blood cytopenias and bone marrow blast percentage and cytogenetics. The current standard of care for patients with higher-risk MDS (HR-MDS) includes hypomethylating agents (HMAs), decitabine and azacitidine, and allogenic stem cell transplant for patients able to undergo this therapy. However, leukemic transformation remains a significant challenge, and outcomes with these current therapies are still dismal. There are several novel therapies in development aiming to improve upon the outcomes of single-agent HMA therapy using combination strategies with HMAs. Here we discuss the current standard of care for HR-MDS treatment and explore some of the most promising combination therapies coming out of the pipeline for HR-MDS.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"368-374"},"PeriodicalIF":2.9,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9820246/pdf/hem.2022000351.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10557811","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

Dyskeratosis congenita and telomere biology disorders. 先天性角化不良和端粒生物学障碍。

IF 3 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000394

Sharon A Savage

{"title":"Dyskeratosis congenita and telomere biology disorders.","authors":"Sharon A Savage","doi":"10.1182/hematology.2022000394","DOIUrl":"https://doi.org/10.1182/hematology.2022000394","url":null,"abstract":"Numerous genetic discoveries and the advent of clinical telomere length testing have led to the recognition of a spectrum of telomere biology disorders (TBDs) beyond the classic dyskeratosis congenita (DC) triad of nail dysplasia, abnormal skin pigmentation, and oral leukoplakia occurring with pediatric bone marrow failure. Patients with DC/TBDs have very short telomeres for their age and are at high risk of bone marrow failure, cancer, pulmonary fibrosis (PF), pulmonary arteriovenous malformations, liver disease, stenosis of the urethra, esophagus, and/or lacrimal ducts, avascular necrosis of the hips and/or shoulders, and other medical problems. However, many patients with TBDs do not develop classic DC features; they may present in middle age and/or with just 1 feature, such as PF or aplastic anemia. TBD-associated clinical manifestations are progressive and attributed to aberrant telomere biology caused by the X-linked recessive, autosomal dominant, autosomal recessive, or de novo occurrence of pathogenic germline variants in at least 18 different genes. This review describes the genetics and clinical manifestations of TBDs and highlights areas in need of additional clinical and basic science research.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"637-648"},"PeriodicalIF":3.0,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9821046/pdf/hem.2022000394.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10851370","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: 15

Available and emerging therapies for bona fide advanced systemic mastocytosis and primary eosinophilic neoplasms. 治疗真正的晚期系统性肥大细胞增多症和原发性嗜酸性粒细胞肿瘤的现有疗法和新兴疗法。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000368

Jason Gotlib

{"title":"Available and emerging therapies for bona fide advanced systemic mastocytosis and primary eosinophilic neoplasms.","authors":"Jason Gotlib","doi":"10.1182/hematology.2022000368","DOIUrl":"10.1182/hematology.2022000368","url":null,"abstract":"The historically poor prognosis of patients with advanced systemic mastocytosis (AdvSM) and primary eosinophilic neoplasms has shifted to increasingly favorable outcomes with the discovery of druggable targets. The multikinase/KIT inhibitor midostaurin and the highly selective KIT D816V inhibitor avapritinib can elicit marked improvements in measures of mast cell (MC) burden as well as reversion of MC-mediated organ damage (C-findings) and disease symptoms. With avapritinib, the achievement of molecular remission of KIT D816V and improved survival compared with historical therapy suggests a potential to affect disease natural history. BLU-263 and bezuclastinib are KIT D816V inhibitors currently being tested in trials of AdvSM. In the new World Health Organization and International Consensus Classifications, the category of \"myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase (TK) gene fusions\" is inclusive of rearrangements involving PDGFRA, PDGFRB, FGFR1, JAK2, FLT3, and ETV6::ABL1. While the successful outcomes with imatinib in FIP1L1::PDGFRA-positive cases and PDGFRB-rearranged neoplasms have become the \"poster children\" of these disorders, the responses of the other TK-driven neoplasms to small-molecule inhibitors are more variable. The selective FGFR inhibitor pemigatinib, approved in August 2022, is a promising therapy in aggressive FGFR1-driven diseases and highlights the role of such agents in bridging patients to allogeneic transplantation. This review summarizes the data for these approved and investigational agents and discusses open questions and future priorities regarding the management of these rare diseases.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"34-46"},"PeriodicalIF":2.9,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9821059/pdf/hem.2022000368.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10276262","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

Fitness and frailty in myeloma. 骨髓瘤患者的体质和虚弱。

IF 2.9 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000346

Charlotte Pawlyn, Abdullah M Khan, Ciara L Freeman

{"title":"Fitness and frailty in myeloma.","authors":"Charlotte Pawlyn, Abdullah M Khan, Ciara L Freeman","doi":"10.1182/hematology.2022000346","DOIUrl":"10.1182/hematology.2022000346","url":null,"abstract":"As the aging population grows, so too does the number of well-tolerated antimyeloma therapies. Physicians will see an increasing volume of patients for subsequent lines of therapy, which could now extend this relationship for over a decade. For younger patients, treatment choices are infrequently impacted by concerns of fitness, but instead about effecting the deepest, most durable response. Older adults, in contrast, are more likely to experience under- than overtreatment, and therefore more objective (and ideally straightforward) ways to evaluate their fitness and ability to tolerate therapy will increasingly assist in decision-making. Post hoc analyses categorizing the fitness of trial patients in the modern treatment era globally demonstrate that even in highly selected populations, those that are recategorized as less fit or frail are consistently at higher risk of inferior outcomes and increased toxicities. Real-world data are comparatively lacking but do demonstrate that most patients with myeloma are not representative of those enrolled on clinical trials, generally more heavily burdened by comorbidities and more likely to be categorized as \"less than fit.\" Simultaneously, the number of therapeutic options open to patients in the relapsed setting continues to grow, now including T-cell engagers and cellular therapies, with their unique toxicity profiles. The aim of this review is to summarize the available data, highlight some of the approaches possible to easily assess fitness and how results might inform treatment selection, and illustrate ways that patients' condition can be optimized rather than lead to exclusion from the more complex therapies newly available.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"337-348"},"PeriodicalIF":2.9,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9820647/pdf/hem.2022000346.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10276264","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

Optimal approach to T-cell ALL. t细胞ALL的最佳治疗方法。

IF 3 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000337C

Kristen M O'Dwyer

{"title":"Optimal approach to T-cell ALL.","authors":"Kristen M O'Dwyer","doi":"10.1182/hematology.2022000337C","DOIUrl":"https://doi.org/10.1182/hematology.2022000337C","url":null,"abstract":"T-lineage acute lymphoblastic leukemia (T-ALL) is curable for most children and adolescent and young adult patients with contemporary frontline chemotherapy regimens. During the past decade, improved survival rates have resulted from the optimization of frontline chemotherapy regimens, the use of minimal residual disease (MRD) assessment for evaluating a patient's risk for relapse, and the intensification of treatment based on the persistence of MRD. Optimization of initial therapy is critical because relapsed T-ALL after initial intensive chemotherapy is incurable for most adult patients. Current T-ALL salvage chemotherapy regimens are minimally effective, and unlike in B-cell ALL, there are no approved antibody therapies or chimeric antigen receptor T-cell therapies for relapsed disease. Immunotherapy and small-molecule inhibitors are beginning to be tested in relapsed T-ALL and have the potential to advance the treatment. Until effective salvage strategies are discovered, however, intensive frontline therapy is required for cure. In this article I review the current frontline chemotherapy regimens for adult patients with T-ALL, summarize the novel targeted and immune therapeutics currently in early-phase clinical trials, and outline how these therapies are helping to define an optimal approach for T-ALL.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"197-205"},"PeriodicalIF":3.0,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9821176/pdf/hem.2022000337.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10499514","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: 1

Acute pain episodes, acute chest syndrome, and pulmonary thromboembolism in pregnancy. 妊娠期急性疼痛发作、急性胸综合征和肺血栓栓塞。

IF 3 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000376

Eugenia Vicky Asare, Michael R DeBaun, Edeghonghon Olayemi, Theodore Boafor, Samuel A Oppong

{"title":"Acute pain episodes, acute chest syndrome, and pulmonary thromboembolism in pregnancy.","authors":"Eugenia Vicky Asare, Michael R DeBaun, Edeghonghon Olayemi, Theodore Boafor, Samuel A Oppong","doi":"10.1182/hematology.2022000376","DOIUrl":"https://doi.org/10.1182/hematology.2022000376","url":null,"abstract":"Pregnancy in women with sickle cell disease (SCD) is a life-threatening condition. In both high- and low-income countries, there is an 11-fold increased risk of maternal death and a 4-fold increased risk of perinatal death. We highlight the epidemiology of SCD-specific and obstetric complications commonly seen during pregnancy in SCD and propose definitions for acute pain and acute chest syndrome (ACS) episodes during pregnancy. We conducted a systematic review of the recent obstetric and hematology literature using full research articles published within the last 5 years that reported outcomes in pregnant women with SCD. The prevalence of acute pain episodes during pregnancy ranged between 4% and 75%. The prevalence of ACS episodes during pregnancy ranged between 4% and 13%. The estimated prevalence of pulmonary thromboembolism in women with SCD during pregnancy is approximately 0.5 to 1%. ACS is the most common cause of death and is often preceded by acute pain episodes. The most crucial time to develop these complications in pregnancy is during the third trimester and postpartum period. In a pooled analysis from studies in low- and middle-income settings, maternal death in women with SCD is approximately 2393 and 4300 deaths per 100 000 live births with and without multidisciplinary care, respectively. In comparison, in the US and northern Europe, the general maternal mortality rate is approximately 23.8 and 8 deaths per 100 000 live births, respectively. A multidisciplinary SCD obstetrics care approach reduces maternal and perinatal morbidity and mortality in low- and middle-income countries.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"388-407"},"PeriodicalIF":3.0,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9821602/pdf/hem.2022000376.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10499975","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: 1

In 2022, which is preferred: haploidentical or cord transplant? 2022年，单倍体同卵还是脐带移植?

IF 3 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000327

Arnon Nagler, Mohamad Mohty

{"title":"In 2022, which is preferred: haploidentical or cord transplant?","authors":"Arnon Nagler, Mohamad Mohty","doi":"10.1182/hematology.2022000327","DOIUrl":"https://doi.org/10.1182/hematology.2022000327","url":null,"abstract":"Allogeneic hematopoietic stem cell transplantation is the treatment of choice for high-risk hematological malignancies such as acute myeloid and lymphocytic leukemia, myelodysplastic syndrome, and myeloproliferative disorders. Alternative donor transplantation from either haploidentical (haplo-SCT) or cord blood donor (CBT) is an established therapeutic alternative for patients who need transplants but lack a human leukocyte antigen-matched donor. Although haplo-SCT (mainly non-T-cell-depleted haplo-SCT with posttransplant cyclophosphamide) is increasing while CBT is decreasing worldwide (Figure 1), recent developments in CBT, especially cord blood expansion and other strategies to improve engraftment and immune reconstitution post-CBT, make CBT still a valuable option. This article discusses the 2 options based on the currently available data, focusing on adults, and tries to give some clues to help the transplant physician choose a haploidentical vs a cord blood donor. Given the limited numbers of published or ongoing well-designed randomized controlled trials comparing haplo-SCT to CBT and the overall similar clinical results in the available, mostly registry-based, and single-center studies, with substantial heterogeneity and variability, the decision to perform haplo-SCT or CBT in a given patient depends not only on the patient, disease, and donor characteristics and donor availability (although most if not all patients should have in principle an alternative donor) but also on the transplant physician's discretion and, most importantly, the center's experience and preference and ongoing protocols and strategies.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"64-73"},"PeriodicalIF":3.0,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9820258/pdf/hem.2022000327.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10501737","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: 6

Cold AIHA and the best treatment strategies. 冷AIHA和最佳治疗策略。

IF 3 3区教育学

Hematology. American Society of Hematology. Education Program Pub Date : 2022-12-09 DOI: 10.1182/hematology.2022000369

Jenny McDade Despotovic, Taylor Olmsted Kim

{"title":"Cold AIHA and the best treatment strategies.","authors":"Jenny McDade Despotovic, Taylor Olmsted Kim","doi":"10.1182/hematology.2022000369","DOIUrl":"https://doi.org/10.1182/hematology.2022000369","url":null,"abstract":"Cold-reactive autoimmune hemolytic anemia (AIHA) is rare among the hemolytic anemias. It results when 1 of a variety of processes causes the generation of immunoglobulin M (IgM) autoantibodies against endogenous erythrocytes, resulting in complement activation and predominantly intravascular hemolysis. Cold AIHA is typically a primary lymphoproliferative disorder with marrow B-cell clones producing pathogenic IgM. More rarely, secondary cold AIHA (cAIHA) can develop from malignancy, infection, or other autoimmune disorders. However, in children cAIHA is typically post infection, mild, and self-limited. Symptoms include a sequelae of anemia, fatigue, and acrocyanosis. The severity of disease is variable and highly dependent on the thermal binding range of the autoantibody. In adults, treatment has most commonly focused on reducing antibody production with rituximab-based regimens. The addition of cytotoxic agents to rituximab improves response rates, but at the expense of tolerability. Recent insights into the cause of cold agglutinin disease as a clonal disorder driven by complement form the basis of newer therapeutic options. While rituximab-based regimens are still the mainstay of therapy, options have now expanded to include complement-directed treatments and other B-cell-directed or plasma-cell-directed therapies.","PeriodicalId":12973,"journal":{"name":"Hematology. American Society of Hematology. Education Program","volume":"2022 1","pages":"90-95"},"PeriodicalIF":3.0,"publicationDate":"2022-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9821124/pdf/hem.2022000369.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10501742","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: 2