Abstract:
INTRODUCTION:Inhibitors against factor VIII (FVIII), which develop in around 20-30% of patients with severe hemophilia A, represent a significant complication of on-demand or prophylactic FVIII therapy. Currently, the main treatment option for inhibitor patients is eradication using immune tolerance induction (ITI) therapy. Areas covered: This article reviews inhibitor eradication in clinically complicated hemophilia A patients. The benefits and disadvantages of ITI therapy are discussed, with reference to the new nonfactor replacement agents such as emicizumab, which are becoming available. Expert commentary: At present, ITI therapy is the method of choice for all inhibitor patients, but as alternative treatment options become increasingly available, this may change in the future. Despite the development of nonfactor replacement agents, ITI therapy is likely to maintain a key role in the management of inhibitor patients, although changes in ITI therapy regimens including the timing of ITI initiation, the dosing regimen, the management of thrombotic risk or safety, laboratory assessment of ITI outcomes, and maintenance treatment after ITI success, may be necessary.
journal_name
Expert Rev Hematoljournal_title
Expert review of hematologyauthors
Oldenburg J,Young G,Santagostino E,Escuriola Ettingshausen Cdoi
10.1080/17474086.2018.1521718subject
Has Abstractpub_date
2018-11-01 00:00:00pages
857-862issue
11eissn
1747-4086issn
1747-4094journal_volume
11pub_type
杂志文章,评审abstract::Many patients with acute myeloid leukemia will eventually develop refractory or relapsed disease. In the absence of standard therapy for this population, there is currently an urgent unmet need for novel therapeutic agents. Targeted therapy with small molecule inhibitors represents a new therapeutic intervention that ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2014.932687
更新日期:2014-08-01 00:00:00
abstract:INTRODUCTION:Hodgkin's lymphoma (HL) is largely a curable disease with excellent prognosis. The standard of care in patients with relapsed disease has been to try salvage chemotherapy followed by an autologous stem cell transplantation (ASCT). Managing the patients who relapse after ASCT, is challenging. With the appro...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1449637
更新日期:2018-04-01 00:00:00
abstract:INTRODUCTION:In the last decade, the availability of new drugs for the treatment of Multiple Myeloma (MM) significantly improved patients' outcomes, but also raised attention towards a new spectrum of adverse events. Recently, four novel agents with different mechanisms of action (carfilzomib, elotuzumab, daratumumab a...
journal_title:Expert review of hematology
pub_type: 杂志文章,meta分析,评审
doi:10.1080/17474086.2017.1284584
更新日期:2017-03-01 00:00:00
abstract:INTRODUCTION:Recent advances and drug approvals in the last decade have substantially changed the landscape of relapsed and refractory multiple myeloma (RRMM), which not only improved outcomes for patients but also increased complexity of treatment decisions. The approvals are based on randomized studies of novel agent...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1804356
更新日期:2020-09-01 00:00:00
abstract::β-thalassemias are caused by nearly 300 mutations of the β-globin gene, leading to a low or absent production of adult hemoglobin (HbA). Two major therapeutic approaches have recently been proposed: gene therapy and induction of fetal hemoglobin (HbF) with the objective of achieving clinically relevant levels of Hbs. ...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1586/ehm.13.24
更新日期:2013-06-01 00:00:00
abstract::Introduction: Oral anticoagulants (OACs) are established treatments to reduce thromboembolic risk in patients with atrial fibrillation (AF). Due to their mechanisms of action, they are associated with increased bleeding risk. Evaluation with bleeding risk scores may be useful to identify patients at high risk. However...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2019.1647779
更新日期:2019-10-01 00:00:00
abstract:INTRODUCTION:Hypogonadism is the most frequently reported endocrine complication, affecting 40%-80% of thalassemia major (TM) patients. The prevalence and severity of hypogonadism in TM varies among studies, depending on patients' age, genotype, transfusion frequency and starting age and efficiency of iron chelation. A...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1398080
更新日期:2017-12-01 00:00:00
abstract::Primary mediastinal large B-cell lymphoma (PMBCL) is a unique B-cell lymphoma variant that arises from a putative thymic medulla B cell. It constitutes 2-4% of non-Hodgkin lymphomas and occurs most frequently in young females. PMBCL is characterized by a diffuse proliferation of medium-to-large B cells associated with...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2015.994604
更新日期:2015-04-01 00:00:00
abstract::The incidence of non-Hodgkin's lymphoma (NHL) is increasing among all age groups, with a median age at diagnosis of 67 years. With the increase in the geriatric population, there is a need for the development and validation of treatment strategies for NHL for these patients. Therapy in elderly patients is affected by ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.09.7
更新日期:2009-04-01 00:00:00
abstract:BACKGROUND:Hb H disease is an alpha-thalassemia (α-thal) syndrome characterized by chronic hemolytic anemia that occurs when three of total four α-globin genes lost their function due to completely deletions or different kind of mutations. OBJECTIVE:We here described 66 patients who have been diagnosed for Hb H diseas...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1586/17474086.2016.1107470
更新日期:2016-01-01 00:00:00
abstract::The fms-like tyrosine kinase 3 (FLT3) plays an important role in both normal and malignant hematopoiesis. Activating mutations in the FLT3 receptor can be detected in approximately 30% of acute myeloid leukemias (AMLs) and are associated with a distinctly poor clinical outcome for patients. There are now several class...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.1.2.153
更新日期:2008-12-01 00:00:00
abstract::Hematopoietic stem cells can be mobilized out of the bone marrow into the blood for the reconstitution of hematopoiesis following high-dose therapy. Methods to improve mobilization efficiency and yields are rapidly emerging. Traditional methods include chemotherapy with or without myeloid growth factors. Plerixafor, a...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.09.54
更新日期:2009-12-01 00:00:00
abstract::Myeloid sarcoma (MS) is an extramedullary myeloid neoplasm characterized by proliferation of myeloblasts which can occur in any organ or site. Bronchial and pulmonary involvement, however, is uncommon. We describe a case of bronchial MS in an 81-year-old female with a history of high-grade myelodysplastic syndrome; sh...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1586/17474086.2015.1044747
更新日期:2015-08-01 00:00:00
abstract::Major advances have significantly improved the outcome of mantle cell lymphoma (MCL). Incorporation of rituximab to CHOP regimen, the adoption of high dose cytarabine with frontline autologous stem cell transplantation in young patients, maintenance rituximab or bortezomib based chemotherapy in elderly patients, impro...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2016.1134309
更新日期:2016-03-01 00:00:00
abstract::Despite significant improvements in treatment modalities over the 10 years, the clinical course of patients with follicular lymphoma (FL) remains heterogeneous. Thus, prognostic indexes are still required to direct treatment choices and for the design of clinical trials. Investigators have conducted a variety of studi...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.09.34
更新日期:2009-10-01 00:00:00
abstract::Asparaginase has been a mainstay of therapy in the treatment of acute lymphoblastic leukemia since the 1970s. There are two major preparations available and FDA approved in the United States today, one derived from Escherichia coli and the other from Erwinia chrysanthemi. Erwinia asparaginase is antigenically distinct...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2016.1142370
更新日期:2016-03-01 00:00:00
abstract:INTRODUCTION:Guidelines provide recommendations on the management of multiple myeloma (MM), but there are no standard algorithms for the choice and sequencing of treatments. As a result, there is widespread variation in the interpretation and implementation of these guidelines. Areas covered: This review will cover: th...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1437345
更新日期:2018-03-01 00:00:00
abstract::In chronic lymphocytic leukemia (CLL), the most prevalent lymphoid malignancy in western countries, patients have a median age at diagnosis of 72 years. In the last few years, there has been remarkable progress in understanding the biology of CLL, the detection of molecular prognostic factors and the development of mo...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2013.814845
更新日期:2013-08-01 00:00:00
abstract::Anti-CD20 monoclonal antibodies (mAbs), rituximab, ofatumumab and obinutuzumab, have a significant impact in the treatment of chronic lymphocytic leukemia (CLL), particularly in combination with chemotherapy. Over the last few years, several new mAbs have been developed and investigated in CLL. The most promising newe...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2014.963048
更新日期:2014-12-01 00:00:00
abstract::Chronic immune thrombocytopenia (ITP) is an autoimmune disorder characterized by a low platelet count that has persisted for more than 12 months. Patients with severe, symptomatic disease may have significant morbidity and require treatment. Historically, the underlying cause of ITP was believed to be accelerated plat...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1586/ehm.11.25
更新日期:2011-06-01 00:00:00
abstract::Introduction: There is emerging evidence linking the commensal gut microbiota with the development of cardiovascular disease and arterial thrombosis. In immunothrombosis, the host clotting system protects against the dissemination of invading microbes, not considering the huge number of microbes that interact with hos...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2019.1627191
更新日期:2019-07-01 00:00:00
abstract::Hemophilia A is a congenital, recessive, X-linked bleeding disorder that is managed with infusions of plasma-derived or recombinant factor (F) VIII. The primary considerations in FVIII replacement therapy today are the: 1) immunogenicity of FVIII concentrates, 2) role of longer-acting FVIII products, 3) prophylactic u...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2014.899896
更新日期:2014-06-01 00:00:00
abstract::Introduction: Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, consumption thrombocytopenia, and organ injury. TTP pathophysiology is based on a severe ADAMTS13 deficiency, the specific von Willebrand factor (VWF)-cl...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2019.1611423
更新日期:2019-06-01 00:00:00
abstract::Early evidence has already shown that activated platelets play a role in the intrinsic pathway of coagulation, but the mechanism remained unclear. The finding that the dense granules of platelets contain polyphosphate (polyP), which has procoagulant effects, resolved this missing link between activated platelets and c...
journal_title:Expert review of hematology
pub_type: 评论,杂志文章
doi:10.1586/ehm.10.26
更新日期:2010-06-01 00:00:00
abstract:INTRODUCTION:Iliopsoas muscle hemorrhage in patients with hemophilia is a serious complication with relevant related morbidity. AREAS COVERED:A Cochrane Library and PubMed (MEDLINE) search of studies related to iliopsoas hematomas in hemophilia was analyzed. Pain in the hip joint area and femoral nerve involvement in ...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1787146
更新日期:2020-08-01 00:00:00
abstract::Sickle cell disease (SCD) is a severe genetic disorder of hemoglobin causing vaso-occlusion. Patients suffer severe anemia, strokes, renal failure, pulmonary compromise and shortened life expectancy. Over 90,000 people in the USA have SCD, and the options for therapy are limited and only partially effective. With the ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.12.20
更新日期:2012-06-01 00:00:00
abstract::Key paper evaluation: Craddock C, et al. Combination Lenalidomide and Azacitidine: A Novel Salvage Therapy in Patients Who Relapse After Allogeneic Stem-Cell Transplantation for Acute Myeloid Leukemia. J Clin Oncol. 2019; 37: 580-8. Allogeneic hematopoietic stem cell transplant (allo-HSCT) is the only potentially cura...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2019.1635005
更新日期:2019-08-01 00:00:00
abstract:INTRODUCTION:Human term placenta is comprised of various tissues from which different cells can be obtained, including hematopoietic stem cells and mesenchymal stem/stromal cells (MSCs). Areas covered: This review will discuss the possibility to incorporate placental tissue cells in cord blood banking. It will discuss ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1483717
更新日期:2018-08-01 00:00:00
abstract:INTRODUCTION:Most patients with Hodgkin lymphoma (HL) enjoy durable remissions following front-line treatment but 30% of patients are refractory or relapse after first line therapy. Salvage chemotherapy followed by autologous stem cell transplantation (ASCT) can cure an additional 50-55% of relapsing patients but new t...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1313701
更新日期:2017-05-01 00:00:00
abstract::Objectives: CD43 can be useful in routine flow cytometry. We conducted a systematic review aiming to describe when CD43 is used by flow cytometry in malignant hematology and to determine its value in these settings. Methods: Systematic review of MEDLINE (search 'CD43' AND 'flow cytometry,' starting in 2010). Results: ...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2021.1856653
更新日期:2021-01-01 00:00:00