Expert Review of Hematology最新文献

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Ankle arthrodesis and total ankle replacement in patients with congenital bleeding disorders suffering from severe ankle arthropathy. 严重踝关节病的先天性出血性疾病患者的踝关节固定术和全踝关节置换术。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2024-01-01 Epub Date: 2023-12-27 DOI: 10.1080/17474086.2023.2299301
E Carlos Rodriguez-Merchan
{"title":"Ankle arthrodesis and total ankle replacement in patients with congenital bleeding disorders suffering from severe ankle arthropathy.","authors":"E Carlos Rodriguez-Merchan","doi":"10.1080/17474086.2023.2299301","DOIUrl":"10.1080/17474086.2023.2299301","url":null,"abstract":"<p><strong>Introduction: </strong>In patients with congenital bleeding disorders suffering from severe ankle arthropathy, when conservative treatment and joint-preserving surgical techniques fail, there are two possible non-joint-preserving options: ankle arthrodesis (AA) and total ankle replacement (TAR).</p><p><strong>Areas covered: </strong>The scope and aim of this article was to analyze the current role of AA and TAR in patients with congenital bleeding disorders suffering from severe ankle arthropathy.</p><p><strong>Expert opinion: </strong>In patients with congenital bleeding disorders, both TAR and AA provide good results, mainly in terms of pain relief, although they are not exempt from complications (between 0% and 33% in TAR and between 5% and 23.5% in AA). The current controversy about which of the two surgical techniques, TAR or AA, gives better results, the current literature is not able to resolve it in patients with congenital bleeding disorders. While this question is being answered, my opinion regarding patients with congenital bleeding disorders is that the age of the patient must be taken into account. Given known prosthetic survival rates, the older the patient, the more we might be inclined to indicate TAR. Conversely, AA may be more appropriate for relatively young patients.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"1-8"},"PeriodicalIF":2.8,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138829138","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Should we consider caplacizumab as routine treatment for acute thrombotic thrombocytopenic purpura? An expert perspective on the pros and cons. 是否应考虑将卡普珠单抗作为急性血栓性血小板减少性紫癜的常规治疗方法?从专家角度看利弊。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2024-01-01 Epub Date: 2024-02-20 DOI: 10.1080/17474086.2024.2318347
Inés Gómez Seguí, María Eva Mingot Castellano, Cristina Pascual Izquierdo, Javier de la Rubia
{"title":"Should we consider caplacizumab as routine treatment for acute thrombotic thrombocytopenic purpura? An expert perspective on the pros and cons.","authors":"Inés Gómez Seguí, María Eva Mingot Castellano, Cristina Pascual Izquierdo, Javier de la Rubia","doi":"10.1080/17474086.2024.2318347","DOIUrl":"10.1080/17474086.2024.2318347","url":null,"abstract":"<p><strong>Introduction: </strong>Immune thrombotic thrombocytopenic purpura (iTTP) is a rare and life-threatening disorder. Caplacizumab has been the latest drug incorporated into the initial treatment of acute episodes, allowing for faster platelet recovery and a decrease in refractoriness, exacerbation, thromboembolic events, and mortality. However, caplacizumab is also associated with a bleeding risk and higher treatment costs, which prevent many centers from using it universally.</p><p><strong>Areas covered: </strong>Studies that included iTTP and/or caplacizumab to date were selected for this review using PubMed and MEDLINE platforms. We describe outcomes in the pre-caplacizumab era and after it, highlighting the benefits and risks of its use early in frontline, and also pointing out special situations that require careful management.</p><p><strong>Expert opinion: </strong>It is clear that the availability of caplacizumab has significantly and favorably impacted the management of iTTP patients. Whether this improvement is cost-effective still remains uncertain, and data on long-term sequelae and different healthcare systems will help to clarify this point. In addition, evidence of the bleeding/thrombotic risk of iTTP patients under this drug needs to be better addressed in future studies.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"9-25"},"PeriodicalIF":2.8,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139729424","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
The effect of immunomodulatory drugs on bone metabolism of patients with multiple myeloma. 免疫调节药物对多发性骨髓瘤患者骨代谢的影响。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2024-01-01 Epub Date: 2024-02-12 DOI: 10.1080/17474086.2024.2316090
Yang Liu, Bo Li, Xiaomin Chen, Hao Xiong, Chunlan Huang
{"title":"The effect of immunomodulatory drugs on bone metabolism of patients with multiple myeloma.","authors":"Yang Liu, Bo Li, Xiaomin Chen, Hao Xiong, Chunlan Huang","doi":"10.1080/17474086.2024.2316090","DOIUrl":"10.1080/17474086.2024.2316090","url":null,"abstract":"<p><strong>Introduction: </strong>Immunomodulatory drugs (IMiDs) are widely used in the management of newly diagnosed and relapsed/refractory multiple myeloma patients. These agents show their potential effect on myeloma bone disease (MBD), including inhibition of osteoclasts activity and effects on osteoblasts differentiation. It is unclear whether these effects are direct, which may have an impact on bone formation markers when combined with proteasome inhibitors.</p><p><strong>Areas covered: </strong>This review summarizes the available evidence on the role of IMiDs in microenvironment regulation and their potential effects on bone metabolism. The literature search methodology consisted of searching PubMed for basic and clinical trials using medical subject terms. Included articles were screened and evaluated by the coauthors of this review.</p><p><strong>Expert opinion: </strong>As a therapeutic option, IMiDs directly affect preosteoblast/osteoclast differentiation. The combination of proteasome inhibitors may counteract the short-term up-regulation of osteogenic activity markers, and therefore intravenous zoledronic acid is recommended, however, obtaining a more significant myeloma response will have a long-term positive impact on myeloma bone disease.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"47-54"},"PeriodicalIF":2.8,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139691618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Comparison of blinatumomab and CAR T-cell therapy in relapsed/refractory acute lymphoblastic leukemia: a systematic review and meta-analysis. 复发性/难治性急性淋巴细胞白血病中blinatumomab与CAR T细胞疗法的比较:系统综述与荟萃分析。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2024-01-01 Epub Date: 2024-01-04 DOI: 10.1080/17474086.2023.2298732
Yixin Zhai, Ju Hong, Jinhuan Wang, Yanan Jiang, Wenqi Wu, Yangyang Lv, Jing Guo, Linyan Tian, Huimeng Sun, Yuhang Li, Cheng Li, Hongjie Zhan, Zhigang Zhao
{"title":"Comparison of blinatumomab and CAR T-cell therapy in relapsed/refractory acute lymphoblastic leukemia: a systematic review and meta-analysis.","authors":"Yixin Zhai, Ju Hong, Jinhuan Wang, Yanan Jiang, Wenqi Wu, Yangyang Lv, Jing Guo, Linyan Tian, Huimeng Sun, Yuhang Li, Cheng Li, Hongjie Zhan, Zhigang Zhao","doi":"10.1080/17474086.2023.2298732","DOIUrl":"10.1080/17474086.2023.2298732","url":null,"abstract":"<p><strong>Objectives: </strong>This study evaluated the benefits and risks of patients with refractory or relapsed acute lymphocytic leukemia (R/R ALL) treated with anti-CD19 chimeric antigen receptor (CAR) T-cell therapy and blinatumomab.</p><p><strong>Methods: </strong>PubMed, Web of Science, Embase, and the Cochrane Library were searched for relevant studies.</p><p><strong>Results: </strong>The pooled complete remission (CR) rate and minimal residual disease (MRD) negative rate were 48%, 31% for blinatumomab, and 86% and 80% for CAR T-cell therapy.</p><p><strong>Conclusions: </strong>The CAR T-cell therapy group exhibited a higher likelihood of CR rate than the blinatumomab group in every analysis regardless of adjustment subgroups. CAR T-cell therapy was associated with a significantly prolonged overall survival (OS) and relapse-free survival (RFS) compared with blinatumomab (2-year OS 55% vs 25%; 2-year RFS 40% vs 22%). CAR T-cell therapy was more effective for achieving CR and bridging to allogeneic hematopoietic stem cell transplantation (allo-SCT) than blinatumomab (2-year OS 75% vs. 57%). An emerging role for blinatumomab is as a bridging agent pre-SCT, and for patients who achieve an MRD-negative state pre-SCT, post-SCT outcomes are expected to be the same as CAR-T. For adverse effects (AEs), blinatumomab was associated with a lower rate of grade ≥3 hematological toxicity, CRS, and neurological events.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"67-76"},"PeriodicalIF":2.8,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138884790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Overexpression of SCN5A overcomes ABC transporter-mediated multidrug resistance in acute myeloid leukemia through promoting apoptosis. 过表达 SCN5A 可通过促进细胞凋亡克服急性髓性白血病 ABC 转运体介导的多药耐药性。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2024-01-01 Epub Date: 2024-02-02 DOI: 10.1080/17474086.2024.2305363
Kun Xu, Xian-Xu Zhuang, Xiao-Wei Shi
{"title":"Overexpression of SCN5A overcomes ABC transporter-mediated multidrug resistance in acute myeloid leukemia through promoting apoptosis.","authors":"Kun Xu, Xian-Xu Zhuang, Xiao-Wei Shi","doi":"10.1080/17474086.2024.2305363","DOIUrl":"10.1080/17474086.2024.2305363","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to explore the effect and mechanism of SCN5A overcoming ATP-binding cassette (ABC) transporter-mediated multidrug resistance (MDR) in acute myeloid leukemia (AML) through promoting apoptosis.</p><p><strong>Research design and methods: </strong>The tissues derived from AML patients were divided into Sensitive group and Resistance group according to the presence of drug-resistance. Human AML cell line HL-60 and drug-resistant strain HL-60/ADR were divided into HL-60/ADR-vector group, HL-60/ADR-SCN5A group, HL-60-vector group and HL-60-SCN5A group. RT-qPCR was used to detect the mRNA expression level of SCN5A; MTT assay to assess the survival rate and proliferation level of cells; flow cytometry to determine the apoptosis level; and western blot to check the levels of SCN5A, P-glycoprotein (P-gp), MDR protein 1 (MRP1), MDR gene 1 (MDR1), breast cancer resistance protein (BCRP), Bcl-2-associated X protein (Bax), and B-cell lymphoma 2 (Bcl-2) proteins in cells.</p><p><strong>Results: </strong>SCN5A expressed lowly in drug-resistant AML tissues and cells. Up-regulation of SCN5A inhibited MDR in HL-60 cells, enhanced the chemosensitivity of HL-60/ADR, and increased the apoptosis levels of HL-60 and HL-60/ADR cells. However, over-expression of SCN5A inhibited the expression of MDR-related proteins.</p><p><strong>Conclusions: </strong>SCN5A may overcome ABC transporter-mediated MDR in AML through enhancing the apoptosis and inhibiting the expression of MDR proteins.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"87-94"},"PeriodicalIF":2.8,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139478042","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
National strategic advocacy to manage patients with inherited bleeding disorders in low and lower-middle income countries 中低收入国家管理遗传性出血性疾病患者的国家战略宣传
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2023-12-18 DOI: 10.1080/17474086.2023.2293092
Ampaiwan Chuansumrit, Theera Ruchutrakul, Nongnuch Sirachainan, Pimpun Kitpoka, Monratta Panuwannakorn, Panyu Panburana, Monthon Suwannuraks, Nantana Sri-Udomporn, Chusak Kijkunasathian, Suphaneewan Jaovisidha, Chirawat Utamakul, Rungrote Natesirinilkul, Bunchoo Pongtanakul, Chanchai Traivaree, Patcharee Komvilaisak, Ekawat Suwantaroj, Darintr Sosothikul, Pantep Angchaisuksiri, Ponlapat Rojnuckarin
{"title":"National strategic advocacy to manage patients with inherited bleeding disorders in low and lower-middle income countries","authors":"Ampaiwan Chuansumrit, Theera Ruchutrakul, Nongnuch Sirachainan, Pimpun Kitpoka, Monratta Panuwannakorn, Panyu Panburana, Monthon Suwannuraks, Nantana Sri-Udomporn, Chusak Kijkunasathian, Suphaneewan Jaovisidha, Chirawat Utamakul, Rungrote Natesirinilkul, Bunchoo Pongtanakul, Chanchai Traivaree, Patcharee Komvilaisak, Ekawat Suwantaroj, Darintr Sosothikul, Pantep Angchaisuksiri, Ponlapat Rojnuckarin","doi":"10.1080/17474086.2023.2293092","DOIUrl":"https://doi.org/10.1080/17474086.2023.2293092","url":null,"abstract":"Inherited bleeding disorders (IBDs) including hemophilia, von Willebrand disease, platelet disorders, mucocutaneous bleeding disorders and coagulation factor deficiencies are rarely found and under...","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"9 1","pages":""},"PeriodicalIF":2.8,"publicationDate":"2023-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138714539","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Efficacy/Effectiveness and safety of emicizumab prophylaxis of people with hemophilia A: a systematic review and meta-analysis 对 A 型血友病患者进行埃米珠单抗预防治疗的疗效/有效性和安全性:系统综述和荟萃分析
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2023-12-08 DOI: 10.1080/17474086.2023.2293096
Roberto Lúcio Muniz, R. M. Camelo, M. S. Araújo, M. M. Barbosa, A. Guerra, Francisco de Assis Acúrcio, J. Alvares-Teodoro
{"title":"Efficacy/Effectiveness and safety of emicizumab prophylaxis of people with hemophilia A: a systematic review and meta-analysis","authors":"Roberto Lúcio Muniz, R. M. Camelo, M. S. Araújo, M. M. Barbosa, A. Guerra, Francisco de Assis Acúrcio, J. Alvares-Teodoro","doi":"10.1080/17474086.2023.2293096","DOIUrl":"https://doi.org/10.1080/17474086.2023.2293096","url":null,"abstract":"","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"49 5","pages":""},"PeriodicalIF":2.8,"publicationDate":"2023-12-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138588618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Immune thrombocytopenia: a review on the pathogenetic role of immune cells. 免疫性血小板减少症:免疫细胞致病作用综述。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2023-07-01 Epub Date: 2023-09-08 DOI: 10.1080/17474086.2023.2255750
Forogh Nokhostin, Fatemeh Bakhshpour, Seyed Mohammad Sadegh Pezeshki, Reyhane Khademi, Najmaldin Saki
{"title":"Immune thrombocytopenia: a review on the pathogenetic role of immune cells.","authors":"Forogh Nokhostin, Fatemeh Bakhshpour, Seyed Mohammad Sadegh Pezeshki, Reyhane Khademi, Najmaldin Saki","doi":"10.1080/17474086.2023.2255750","DOIUrl":"10.1080/17474086.2023.2255750","url":null,"abstract":"<p><strong>Introduction: </strong>Immune thrombocytopenia [ITP] is a common bleeding disorder with an isolated platelet count of less than 100 × 10<sup>9</sup>/L.</p><p><strong>Areas covered: </strong>Relevant literature from 2003 to 2022 was retrieved and reviewed from the Google Scholar search engine and PubMed database. Antibodies produced by autoreactive B lymphocytes and the phagocytic function of macrophages are considered the most critical factors in platelet destruction. Also, macrophages present the antigen to T lymphocytes and activate them. Follicular helper T-cells [TFH] play a role in stimulating, differentiating, and activating autoreactive B cells, while cluster of differentiation [CD]-8+ T plays a role in platelet destruction through apoptosis. The classical pathway of the complement system also causes platelet destruction. By inhibiting platelet production, low levels of thrombopoietin and an immune response against megakaryocytes in the bone marrow worsen thrombocytopenia.</p><p><strong>Expert opinion: </strong>T-cell subset changes and an increase in activated autoreactive B cells, in addition to the function of components of the innate immune system [the complement system, dendritic cells, and natural killer cells], play a critical role in the pathogenesis of the ITP. Accurate detection of these changes may lead to developing new therapeutic strategies and identifying better prognostic/diagnostic factors.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":" ","pages":"731-742"},"PeriodicalIF":2.8,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10176701","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Hemophilic arthropathy: how to diagnose subclinical bleeding early and how to orthopedically treat a damaged joint. 嗜血性关节病:如何早期诊断亚临床出血以及如何对受损关节进行骨科治疗。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2023-07-01 Epub Date: 2023-07-04 DOI: 10.1080/17474086.2023.2232547
E Carlos Rodriguez-Merchan
{"title":"Hemophilic arthropathy: how to diagnose subclinical bleeding early and how to orthopedically treat a damaged joint.","authors":"E Carlos Rodriguez-Merchan","doi":"10.1080/17474086.2023.2232547","DOIUrl":"10.1080/17474086.2023.2232547","url":null,"abstract":"<p><strong>Introduction: </strong>It is important to know the current status of hemophilic arthropathy diagnoses, treatments, complications, and outcomes in developed countries.</p><p><strong>Areas covered: </strong>A bibliographic search in PubMed for articles published from 1 January 2019 through 12 June 2023 was performed.</p><p><strong>Expert opinion: </strong>In developed countries with specialized hemophilia treatment centers, primary hematological prophylaxis (started before the age of 2 years and after no more than one joint bleed) has almost completely eliminated the joint-related problems of the disease. The ideal goal of zero hemarthroses can be achieved only with intense and well-dosed prophylaxis: intravenous infusion of coagulation factor - standard half-life or extended half-life; periodic or subcutaneous injections of nonfactor products (emicizumab or fitusiran). However, hemophilic arthropathy continues to occur due to subclinical joint hemorrhages. In one study, 16% of the joints without reported hemarthroses showed signs of previous subclinical bleeding (hemosiderin deposits with/without synovial hypertrophy on magnetic resonance imaging were deemed signs of previous subclinical bleeding), rendering evidence for subclinical bleeding in people with severe hemophilia with lifelong prophylaxis treatment. Subclinical joint hemorrhages can be averted only by employing accurate and tailored prophylaxis.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"16 9","pages":"651-658"},"PeriodicalIF":2.8,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10046131","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Efanesoctocog alfa for the prevention and treatment of bleeding in patients with hemophilia A. 埃法尼酮预防和治疗血友病A患者出血。
IF 2.8 4区 医学
Expert Review of Hematology Pub Date : 2023-07-01 Epub Date: 2023-06-15 DOI: 10.1080/17474086.2023.2223925
Barbara A Konkle
{"title":"Efanesoctocog alfa for the prevention and treatment of bleeding in patients with hemophilia A.","authors":"Barbara A Konkle","doi":"10.1080/17474086.2023.2223925","DOIUrl":"10.1080/17474086.2023.2223925","url":null,"abstract":"<p><strong>Introduction: </strong>Hemophilia A is an inherited bleeding disorder due to a deficiency of coagulation factor VIII (FVIII). Prevention and treatment of bleeding is traditionally through intravenous infusion of a FVIII concentrate. Modifications of recombinant FVIII (rFVIII) with an aim to prolong the half-life have been modest, thought because FVIII is dependent on plasma von Willebrand factor (VWF) for its half-life. Efanesoctocog alfa (ALTUVIIIO), approved by the Federal Drug Administration (FDA) in February 2023, was made independent of endogenous VWF by linking of the FVIII-binding D'D3 domain of VWF to B-domain deleted single chain FVIII.</p><p><strong>Areas covered: </strong>This review will outline the development of efanesoctocog alfa and the pharmacokinetic and safety data from clinical trials, as well as efficacy data from the phase 3 trials. These data formed the basis for the FDA approval.</p><p><strong>Expert opinion: </strong>Efanesoctocog alfa is a new type of FVIII replacement with an extended half-life allowing once weekly dosing to achieve hemostasis and FVIII trough levels of 13-15 IU/dL. This provides a highly effective option for treatment and prevention of bleeding in hemophilia A, where FVIII levels are easily measured. It also provides an option for treatment of bleeding and coverage for surgery with few infusions.</p>","PeriodicalId":12325,"journal":{"name":"Expert Review of Hematology","volume":"16 8","pages":"567-573"},"PeriodicalIF":2.8,"publicationDate":"2023-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9837622","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
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