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 available therapies - hydroxyurea, blood transfusion, hydration and pain medicines - patients continue to suffer the long-term complications of the disease. This review focuses on the pathogenesis of SCD and the role of fetal hemoglobin in disrupting the polymerization of sickle hemoglobin. The authors review the compounds that induce fetal hemoglobin: hydroxyurea, which is currently US FDA approved, and the histone deacetylase inhibitors and discuss their role in the treatment of SCD and other β-hemoglobinopathies.
journal_name
Expert Rev Hematoljournal_title
Expert review of hematologyauthors
Okam MM,Ebert BLdoi
10.1586/ehm.12.20subject
Has Abstractpub_date
2012-06-01 00:00:00pages
303-11issue
3eissn
1747-4086issn
1747-4094journal_volume
5pub_type
杂志文章,评审abstract::Allogeneic hematopoietic stem cell transplantation is still the only potentially curative treatment for patients with myelodysplastic syndromes. Improvements in donor selection, supportive care and the introduction of reduced-intensity conditioning have led to a decrease in early transplant mortality. However, relapse...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.11.58
更新日期:2011-12-01 00:00:00
abstract::Although guidelines exist to deal with some aspects of platelet transfusion practice, many important clinical issues have not been addressed in large randomized controlled trials (RCTs). Slichter et al. conducted a RCT of prophylactic platelet transfusions to determine the effects of the dose of platelets on clinical ...
journal_title:Expert review of hematology
pub_type: 评论,杂志文章
doi:10.1586/ehm.10.36
更新日期:2010-08-01 00:00:00
abstract:INTRODUCTION:Diffuse large B-cell lymphoma (DLBCL) is a molecularly heterogeneous disease defined by different cellular origins and mechanisms of oncogenic activation. Approximately 10% of DLBCL cases harbor a MYC rearrangement and this has been associated with a more aggressive clinical course following standard thera...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2016.1182858
更新日期:2016-07-01 00:00:00
abstract:INTRODUCTION:While associated life-threatening and fatal bleeding events are less frequent with the direct factor Xa inhibitors compared to vitamin K antagonists, significant concern surrounding management of major bleeds and urgent periprocedural interruption of these agents exists among clinicians. Andexanet alfa is ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1532287
更新日期:2018-11-01 00:00:00
abstract:INTRODUCTION:Mitochondria are cellular organelles that perform numerous bioenergetic, biosynthetic, and regulatory functions and play a central role in iron metabolism. Extracellular iron is taken up by cells and transported to the mitochondria, where it is utilized for synthesis of cofactors essential to the function ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2016.1268047
更新日期:2017-01-01 00:00:00
abstract:OBJECTIVES:We analyzed hemoglobin (Hb) levels and degree of anemia in relation to genotype in patients with hemoglobin H (Hb H) disease, thereby providing a scientific basis for the prevention and treatment of Hb H disease in the Guangxi region of China. METHODS:Hb analysis was conducted in 615 patients using high per...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1803736
更新日期:2020-09-01 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:Activation of the hemostatic system can occur in many clinical conditions. However, a systemic and strong activation of coagulation complicating clinical settings such as sepsis, trauma or malignant disease may result in the occurrence disseminated intravascular coagulation (DIC). Areas covered: This artic...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1500173
更新日期:2018-08-01 00:00:00
abstract::Ofatumumab is a humanized second-generation monoclonal antibody with the affinity to a transmembrane protein CD20. In in vitro studies, it exhibits higher efficacy towards chronic lymphocytic leukemia (CLL) cells compared to rituximab, and it can be explained by the fact that its epitope on the target CD20 protein is ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2015.1037736
更新日期:2015-06-01 00:00:00
abstract::Tumor lysis syndrome (TLS) is a rare but potentially life-threatening complication of neoplasms, preferentially hematological malignancies. Well known since at least ninety years ago, this condition can be misdiagnosed and incorrectly managed due to rapid onset of symptoms, sometimes overlapping with cancer-derived cl...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2016.1127156
更新日期:2016-01-01 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:Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are considered indolent lymphocytic malignancies, more often requiring active surveillance rather than intervention. Despite the indolent nature of CLL/SLL, treatment is likely indicated in a patients' lifetime. Recent changes in the t...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1270203
更新日期:2017-02-01 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::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:Ferumoxytol is a superparamagnetic molecule originally developed as a contrast agent for magnetic resonance imaging. Elemental iron is contained within the carbohydrate core and is released slowly after infusion allowing a large dose of iron to be administered in a short period of time. Ferumoxytol, origin...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1518712
更新日期:2018-10-01 00:00:00
abstract:INTRODUCTION:Multiple myeloma (MM) is characterized by notable inter-patient and intra-clonal heterogeneity that is gradually decoded over the last decade. Despite the deeper and better understanding of its biology and the development of novel therapeutic strategies that have prolonged overall survival, MM still retain...
journal_title:Expert review of hematology
pub_type: 杂志文章,meta分析,评审
doi:10.1080/17474086.2018.1537776
更新日期:2018-11-01 00:00:00
abstract:INTRODUCTION:Peripheral T cell lymphomas (PTCL) are a heterogenous group of lymphoproliferative disorders which are generally not curable with conventional chemotherapy and associated with inferior outcomes. Pralatrexate is a novel folate analog, the first FDA approved drug) for the treatment of relapsed/refractory (R/...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1756257
更新日期:2020-06-01 00:00:00
abstract:INTRODUCTION:In sickle cell disease (SCD), hemoglobin S (HbS) red blood cells (RBCs) are characteristically deformed and inflexible. Often breaking down in the circulation, they exhibit increased adhesive properties with the endothelium and activated neutrophils and platelets, increasing the risk of occlusion of the mi...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1758555
更新日期:2020-06-01 00:00:00
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::Hemopoietic stem-cell transplant patients may require intensive blood component support. Complications of transfusions include transmission of viral and bacterial infections, transfusion-associated graft-versus-host disease and transfusion-related acute lung injury. Alloimmunization to red cell antigens may cause diff...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.11.14
更新日期:2011-04-01 00:00:00
abstract:INTRODUCTION:Pulmonary hypertension (PH) develops in a significant number of patients with sickle cell disease (SCD), resulting in increased morbidity and mortality. This review focuses on PH pathophysiology, risk stratification, and new recommendations for screening and treatment for patients with SCD. Areas covered: ...
journal_title:Expert review of hematology
pub_type: 杂志文章,meta分析,评审
doi:10.1080/17474086.2017.1364989
更新日期:2017-10-01 00:00:00
abstract:INTRODUCTION:Treatment of elderly patients with acute leukemia is challenging. Older age is associated with increased risk of treatment-related toxicity. Currently, no consensus exists regarding optimal therapy in this patient population. Areas covered: The following review is a comprehensive summary of various therape...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1382345
更新日期:2017-11-01 00:00:00
abstract:INTRODUCTION:Myeloproliferative neoplasms (MPNs) are associated with a variety of symptoms and signs which cause major morbidity for the patients. The disorders are associated with increased incidence of thromboembolic and hemorrhagic events which can lead to complications and shortened life expectancy. Areas covered: ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1410433
更新日期:2018-01-01 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:Ruxolitinib is a potent and selective JAK1/JAK2 inhibitor that has shown superiority as compared to available conventional chemotherapies, in terms of reduction in splenomegaly and improvement of symptoms and quality of life. Areas covered: Data published about overall survival in the major randomized spon...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1273766
更新日期:2017-02-01 00:00:00
abstract:INTRODUCTION:Large granular lymphocytic leukemia (LGLL) is a low grade lymphoproliferative disorder characterized by the clonal proliferation of large granular lymphocytes (LGL) and recognised by the WHO. The diagnosis and management of these patients is challenging due to the limited information from prospective studi...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1284585
更新日期:2017-03-01 00:00:00
abstract::Thromboembolic diseases require anticoagulation for their prevention and treatment. New oral anticoagulants, specifically direct factor Xa and thrombin inhibitors, were developed to overcome the limitations of conventional anticoagulants. Their benefit has been demonstrated using fixed doses without laboratory-guided ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.11.79
更新日期:2012-02-01 00:00:00
abstract::Over two-thirds of newly diagnosed multiple myeloma are over 65 years. The treatment goals for the non-transplant-eligible patients should be to prolong survival by achieving the best response, while ensuring quality of life. New upfront treatment combinations based on first generation of novel proteasome inhibitors a...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2014.864230
更新日期:2014-02-01 00:00:00
abstract:INTRODUCTION:Purine analogs made dramatic improvements for patients with hairy cell leukemia (HCL), but patients often relapse, require multiple treatments, and may become refractory. Major developments in treatment of relapsed/refractory HCL occurred with discovery of disease biology. New agents increase the complexit...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1819231
更新日期:2020-10-01 00:00:00
abstract::Introduction: The genomic landscape of Waldenström macroglobulinemia (WM) is characterized by recurrent MYD88 (MYD88L265P) and CXCR4 mutations (CXCR4MUT), detected in 90% and 30% of cases, respectively. The role of CXCR4MUT in clinical features and outcomes to therapy in WM patients is evolving. Areas covered: We perf...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2019.1649132
更新日期:2019-10-01 00:00:00