Abstract:
PURPOSE OF REVIEW:Bone marrow transplantation is the only curative treatment for severe thalassemia. Since its successful first report in 1981, more than 4000 patients with this disease worldwide underwent bone marrow transplantation. The purpose of this review is to update the most recent reports of matched sibling donor hematopoietic stem cell transplantation in thalassemia. RECENT FINDINGS:Advanced and improved transplant techniques result in the improved outcomes in those transplants from a matched sibling donor with transplant-related mortality less than 5%. Class 3 patients aged at least 7 years and liver enlargement at least 5 cm have a very high risk of graft rejection and regimen-related toxicity. This subset of patients require innovative approaches to overcome the morbidity and mortality. Those include the addition of hydroxyurea, azathioprine, and fludarabine as preconditioning to busulfan, thiotepa, and cyclophosphamide. Novel conditioning consisting of pretransplant immunosuppression with two cycles of fludarabine and dexamethasone followed by reduced intensity conditioning with fludarabine, busulfan, and thymoglobulin has been developed. SUMMARY:Bone marrow transplantation in young low-risk (class 1 and 2) patients should be performed as soon as possible. For class 3 severe thalassemia, novel conditioning regimens have been developed to overcome graft rejection and regimen-related toxicity. Hematopoietic stem cell transplantation in adults who have been well chelated should be offered with clinical trials.
journal_name
Curr Opin Hematoljournal_title
Current opinion in hematologyauthors
Issaragrisil S,Kunacheewa Cdoi
10.1097/MOH.0000000000000286subject
Has Abstractpub_date
2016-11-01 00:00:00pages
508-514issue
6eissn
1065-6251issn
1531-7048journal_volume
23pub_type
杂志文章,评审abstract::Recent studies have improved our understanding of the cytogenesis, biology, and therapy of chronic lymphocytic leukemia (CLL). This review highlights this recent progress reported over the past year. We have improved our understanding of the cytogenetic abnormalities in CLL and soon may see identification of new tumor...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199807000-00003
更新日期:1998-07-01 00:00:00
abstract:PURPOSE OF REVIEW:Inconsistent results regarding the clinical efficacy of granulocyte transfusions for the treatment or prophylaxis of life-threatening infections in neutropenic patients have been attributed to insufficient number of transfused neutrophils. Since the introduction of granulocyte colony-stimulating facto...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000203
更新日期:2016-01-01 00:00:00
abstract:PURPOSE OF REVIEW:The advent of tyrosine kinase inhibitors (TKI) has remarkably changed the treatment of chronic myeloid leukemia (CML). Patients treated in chronic phase can enjoy an expected survival similar to the general population. In the last few years we have found that some patients with an excellent therapeuti...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000224
更新日期:2016-03-01 00:00:00
abstract:PURPOSE OF REVIEW:Adoptive T-cell therapy has become one of the most exciting fields of cancer therapy in the past few years. In this article, we describe a method which combines adoptive T-cell therapy with antibody therapy by arming T cells from cord blood, normal patients, and cancer patients with bispecific antibod...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000176
更新日期:2015-11-01 00:00:00
abstract:PURPOSE OF REVIEW:The presence of the Philadelphia chromosome causing the fusion between BCR to ABL1 in B cell precursor acute lymphoblastic leukemias (ALLs) was associated with a particularly bad prognosis, which has been markedly improved with the addition of imatinib to chemotherapy. Recent genomic studies have lead...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000050
更新日期:2014-07-01 00:00:00
abstract:PURPOSE OF REVIEW:To review the data on angiogenesis related to exosomes secreted by tumor cells in hematologic neoplasia and to elucidate the role of exosomes and exosomal miRNA in the bone marrow microenvironment, especially under hypoxic conditions. RECENT FINDINGS:Cross-talk between bone marrow tumor cells and sur...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000235
更新日期:2016-05-01 00:00:00
abstract:PURPOSE OF REVIEW:Despite parenteral artesunate therapy, the fatality rate of cerebral malaria remains high. Adjunctive therapy targeting the underlying pathophysiology of cerebral malaria may further improve the clinical outcome. Endothelial activation and dysfunction is a central process in the pathogenesis of cerebr...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e328345a4cf
更新日期:2011-05-01 00:00:00
abstract:PURPOSE OF REVIEW:Neutrophils priming has been long studied in vitro. Recent studies describe it in vivo. In pathophysiological conditions, complex, heterogeneous characteristics of priming are described in the last few years. RECENT FINDINGS:Priming can occur systemically when insults such as sepsis or trauma result ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000471
更新日期:2019-01-01 00:00:00
abstract:PURPOSE OF REVIEW:Core binding factor acute myeloid leukemia (CBF-AML) corresponds to two distinct subtypes of AML characterized by recurrent favorable chromosome translocations, namely t(8;21) and inv(16)/t(16;16). Given the relatively good outcome of patients with CBF-AML, when treated with intensive chemotherapy inc...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e3283257b18
更新日期:2009-03-01 00:00:00
abstract::Bone marrow transplantation protocols for inherited metabolic storage diseases are unique for each disorder treated. Differences depend also upon how old the patient was when onset occurred and rate of progression of disease. Treatment is directed to prevent or ameliorate the inexorable neurological deterioration that...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199911000-00004
更新日期:1999-11-01 00:00:00
abstract:PURPOSE OF REVIEW:To examine the current literature regarding retrievable inferior vena cava filters and to discuss the appropriate indications for their clinical use. RECENT FINDINGS:Permanent filters have been shown to be effective, but have a number of long-term complications such as filter thrombosis or migration....
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/01.moh.0000239707.63168.ee
更新日期:2006-09-01 00:00:00
abstract:PURPOSE OF REVIEW:In response to agonists produced at vascular lesions, platelets release a host of components from their three granules: dense core, alpha, and lysosome. This releasate activates other platelets, promotes wound repair, and initiates inflammatory responses. Although widely accepted, the specific mechani...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e328309ec74
更新日期:2008-09-01 00:00:00
abstract:PURPOSE OF REVIEW:Graft-versus-host disease (GVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic cell transplantation (HCT), despite improvements in our understanding of its pathophysiology as well as the generation of new monoclonal antibodies, immunomodulatory chemotherapy, cellular ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e3283319a6f
更新日期:2009-11-01 00:00:00
abstract:PURPOSE OF REVIEW:Over the last years, long non-coding RNAs (lncRNAs) have emerged as putative regulators of malignant hematopoietic development. Here, we review recent literature on the involvement of lncRNAs in leukemia, including their role in driving or sustaining disease and their potential impact on diagnosis, cl...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000354
更新日期:2017-07-01 00:00:00
abstract:PURPOSE OF REVIEW:In the postnatal life, hematopoietic stem cell (HSC) niches are specialized microenvironments in the bone marrow that are essential for the maintenance and function of HSCs. The purpose of this review is to discuss the concept of HSC niche in light of recent studies that broaden its complexity and bet...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e32833a25d8
更新日期:2010-07-01 00:00:00
abstract:PURPOSE OF REVIEW:Type 1 von Willebrand disease (VWD) is the most common form of VWD, but has remained [corrected] the least well understood. Recent work is changing this situation. This review summarizes recent analysis of the genetic basis of the disease. RECENT FINDINGS:Linkage analysis demonstrates that dominantly...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e32826f4b41
更新日期:2007-09-01 00:00:00
abstract::Disorders of the erythrocyte membrane, such as hereditary spherocytosis, hereditary elliptocytosis, and hereditary pyropoikilocytosis, are characterized by heterogeneity in their clinical and laboratory manifestations. Recent work has shown that these disorders also exhibit marked genetic heterogeneity. Multiple genet...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199704020-00009
更新日期:1997-03-01 00:00:00
abstract::This overview concentrates on familial and congenital polycythemias in the context of other polycythemic disorders, with emphasis on those with established molecular lesions. Recent advances in the regulation of erythropoiesis, as they may relate to polycythemic states, are discussed as a background for those well-def...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199903000-00008
更新日期:1999-03-01 00:00:00
abstract::Commitment of hematopoietic cells to the erythroid lineage involves the actions of several transcription factors, including TAL1, LMO2, and GATA-2. The differentiation of committed erythroid progenitor cells involves other transcription factors, including NF-E2 and EKLF. Upon binding erythropoietin, the principal regu...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200203000-00002
更新日期:2002-03-01 00:00:00
abstract:PURPOSE OF REVIEW:The review describes recent advances in our understanding of mechanisms leading to development of neutralizing antibodies following factor VIII (FVIII) replacement therapy for hemophilia A. Novel interventions with translational potential to lessen the incidence of these deleterious immune responses a...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e328356ed37
更新日期:2012-09-01 00:00:00
abstract::The bulk of antigens that are presented by major histocompatibility complex (MHC) class I molecules are processed in the cytosol. Therefore, the cellular protein degradation machinery is thought to play a major role in antigen processing. For example, there is clear evidence that the ubiquitin-proteasome pathway, the ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200101000-00003
更新日期:2001-01-01 00:00:00
abstract::Matching for human leukocyte antigen (HLA) loci is the most crucial factor in the selection of bone marrow donors. The techniques utilized to assign HLA type have evolved significantly since the application of polymerase chain reaction-based methodologies. A significant challenge for DNA typing methodologies is to be ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199811000-00012
更新日期:1998-11-01 00:00:00
abstract::Following on the heels of multiple failed clinical trials of adjunctive therapeutic agents in sepsis, the positive outcome of a recent phase III study using activated protein C (APC) has led to a renewed optimism in targeted biotherapies for this syndrome. A growing body of data (both preclinical and clinical) suggest...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200209000-00005
更新日期:2002-09-01 00:00:00
abstract::Over the past year, substantial progress was made toward understanding transcriptional control of red cell differentiation. Complementary DNAs encoding two novel erythroid-restricted transcription factors--globin locus control region regulatory factor NF-E2 and CACC-binding protein EKLF--were cloned and characterized....
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1994-03-01 00:00:00
abstract:PURPOSE OF REVIEW:Mortality and morbidity associated with leukemia are largely due to frequently occurring cytopenias or the dysfunction of normal blood cells in patients. Our knowledge of how normal blood cells degenerate in response to leukemic cell infiltration has been quite limited. This review summarizes recent f...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000253
更新日期:2016-07-01 00:00:00
abstract::Considerable data now support the hypothesis that platelets actively regulate the propagation of coagulation by (1) expressing specific, high-affinity receptors for coagulation proteases, zymogens, and cofactors; (2) protecting the bound coagulation enzymes from inactivation/inhibition; (3) restricting coagulant activ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200109000-00001
更新日期:2001-09-01 00:00:00
abstract:PURPOSE OF REVIEW:In addition to the crucial role of vascular endothelial growth factor (VEGF)A in vessel development, it has become apparent that the VEGF signaling pathway (VSP) plays an important role during adulthood in the maintenance of tissue homeostasis in normal physiological processes, as well as in pathologi...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e32833865e6
更新日期:2010-05-01 00:00:00
abstract::: Hematopoietic stem cells (HSCs) are a unique population of cells with the remarkable ability to replenish themselves through self-renewal and to give rise to differentiated cell lineages. Though having been discovered more than 50 years ago, and having been widely used in bone marrow transplantation to treat blood d...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000506
更新日期:2019-07-01 00:00:00
abstract:PURPOSE OF REVIEW:This review summarizes recent advances in the application of minimal residual disease (MRD) testing in childhood acute lymphoblastic leukemia (ALL). RECENT FINDINGS:Polymerase chain reaction amplification of antigen receptor genes, one of the two main methods to study MRD in ALL, could be made more r...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e3283543d5c
更新日期:2012-07-01 00:00:00
abstract:PURPOSE OF REVIEW:The 'mobilization' of hematopoietic stem cells (HSCs) out of the bone marrow and into the peripheral blood is used clinically to obtain HSCs for transplantation. Although generally successful, mobilization protocols remain imperfect and the mechanisms involved are not fully understood. This review dis...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e3283606090
更新日期:2013-07-01 00:00:00