Abstract:
:Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder that manifests with hemolytic anemia, thrombosis, and peripheral blood cytopenias. The absence of two glycosylphosphatidylinositol (GPI)-anchored proteins, CD55 and CD59, leads to uncontrolled complement activation that accounts for hemolysis and other PNH manifestations. GPI anchor protein deficiency is almost always due to somatic mutations in phosphatidylinositol glycan class A (PIGA), a gene involved in the first step of GPI anchor biosynthesis; however, alternative mutations that cause PNH have recently been discovered. In addition, hypomorphic germ-line PIGA mutations that do not cause PNH have been shown to be responsible for a condition known as multiple congenital anomalies-hypotonia-seizures syndrome 2. Eculizumab, a first-in-class monoclonal antibody that inhibits terminal complement, is the treatment of choice for patients with severe manifestations of PNH. Bone marrow transplantation remains the only cure for PNH but should be reserved for patients with suboptimal response to eculizumab.
journal_name
Bloodjournal_title
Bloodauthors
Brodsky RAdoi
10.1182/blood-2014-02-522128subject
Has Abstractpub_date
2014-10-30 00:00:00pages
2804-11issue
18eissn
0006-4971issn
1528-0020pii
blood-2014-02-522128journal_volume
124pub_type
杂志文章,评审相关文献
BLOOD文献大全abstract::Surprisingly little is known about the interaction of human blood mononuclear cells with viruses. Here, we show that monocytes are the predominant cell type infected when peripheral blood mononuclear cells are exposed to viruses ex vivo. Remarkably, infection with vesicular stomatitis virus, vaccinia virus, and a vari...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2011-09-379479
更新日期:2012-03-29 00:00:00
abstract::Chronic transfusion of packed red blood cells, in addition to other ongoing treatment with warfarin, acetyl salicylic acid, desferrioxamine, and other supportive measures, was given to a splenectomized hemoglobin E/beta-thalassemia woman with pulmonary arterial hypertension (PHT). Serial measurements of plasma thrombi...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2003-09-3094
更新日期:2004-04-01 00:00:00
abstract::In multiple myeloma (MM), both vascular endothelial (VEGF) and basic fibroblast growth factor (bFGF) promote tumor growth and survival. We have used the novel indolinone BIBF 1000 to study effects of simultaneous inhibition of VEGF, FGF and transforming growth factor-beta on MM cells and their interactions with bone m...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2004-11-4250
更新日期:2006-03-01 00:00:00
abstract::The importance of glutamine (Gln) metabolism in multiple myeloma (MM) cells and its potential role as a therapeutic target are still unknown, although it has been reported that human myeloma cell lines (HMCLs) are highly sensitive to Gln depletion. In this study, we found that both HMCLs and primary bone marrow (BM) C...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2016-01-690743
更新日期:2016-08-04 00:00:00
abstract::L-arginine (L-Arg) plays a central role in several biologic systems including the regulation of T-cell function. L-Arg depletion by myeloid-derived suppressor cells producing arginase I is seen in patients with cancer inducing T-cell anergy. We studied how L-Arg starvation could regulate T-cell-cycle progression. Stim...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2006-06-031856
更新日期:2007-02-15 00:00:00
abstract::The effect of heterologous anti-human platelet antibody on human platelet function was examined in the presence and absence of whole plasma as an in vitro model for antibody-induced immune damage to cells. Heterologous IgG anti-human platelet antibody mediated platelet aggregation and released serotonin from both plat...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1976-07-01 00:00:00
abstract::Thirty-eight children between the ages of 0.8 and 17 years with acute nonlymphoblastic leukemia in first remission induced by chemotherapy were given cyclophosphamide, total body irradiation, and bone marrow transplants from HLA-matched donors. Six patients died of pneumonia, one died of metabolic problems, and one di...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1985-08-01 00:00:00
abstract::Hematologic engraftment and immune reconstitution were examined in patients who received cyclophosphamide and total body irradiation therapy followed by infusion of autologous bone marrow purged with anti-B1 monoclonal antibody (MoAb) and complement as therapy for non-Hodgkin's lymphoma. Hematologic engraftment was pr...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1987-02-01 00:00:00
abstract::Rh(o)(D) immune globulin intravenous (anti-D IGIV) was licensed by the United States Food and Drug Administration (FDA) in March 1995 to treat patients with immune thrombocytopenic purpura (ITP). Anti-D IGIV induces extravascular hemolysis, an expected adverse reaction that is consistent with the presumed mechanism of...
journal_title:Blood
pub_type: 杂志文章,评审
doi:
更新日期:2000-04-15 00:00:00
abstract::Leukocyte rolling is the earliest observable even in their recruitment from the circulation to inflamed tissue. This rolling is mediated largely by interaction between the selectin family of adhesion molecules and their glycosylated ligands. Although the nature of these ligands and their interaction with the selectins...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1998-01-15 00:00:00
abstract::The human peripheral B-cell compartment displays a large population of immunoglobulin M-positive, immunoglobulin D-positive CD27(+) (IgM(+)IgD(+)CD27(+)) "memory" B cells carrying a mutated immunoglobulin receptor. By means of phenotypic analysis, complementarity-determining region 3 (CDR3) spectratyping during a T-in...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2004-01-0346
更新日期:2004-12-01 00:00:00
abstract::Twenty-six patients with progressive Hodgkin's disease after conventional chemotherapy received intensive chemoradiotherapy and autologous bone marrow transplantation (ABMT); 19 also received additional involved-field radiotherapy. Twenty-one patients [81%, 95% confidence intervals (CI) 61% to 94%] attained complete (...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1989-06-01 00:00:00
abstract::High levels of coagulation factor VIII (FVIII) have been associated with cardiovascular disease. Low-density lipoprotein receptor (LDLR) has been recently demonstrated to contribute to FVIII clearance from plasma. The aim of this study was to evaluate 3 single nucleotide polymorphisms in SMARCA4-LDLR gene locus (rs112...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2010-03-277079
更新日期:2010-12-16 00:00:00
abstract::An effective response to extreme hematopoietic stress requires an extreme elevation in hematopoiesis and preservation of hematopoietic stem cells (HSCs). These diametrically opposed processes are likely to be regulated by genes that mediate cellular adaptation to physiologic stress. Herein, we show that heme oxygenase...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2007-12-127621
更新日期:2008-12-01 00:00:00
abstract::The incidence of neutralizing isoantibody formation to infused factor VIII in a cohort of 67 hemophilia A patients, born between January 1, 1971 and April 30, 1990, who had been treated exclusively with lyophilized cryoprecipitate, was 6% (5.3 per 1,000 patient years of observation). The age-dependent cumulative risk ...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1993-06-15 00:00:00
abstract::The CCAAT/enhancer binding protein-alpha (C/EBPalpha) is a transcription factor required for differentiation of myeloid progenitors. In addition to specific DNA binding, C/EBPalpha is also involved in protein-protein interactions, some of which (p21, Cdk2/Cdk4, E2F) appear to be required for inhibition of proliferatio...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2003-02-0477
更新日期:2003-08-15 00:00:00
abstract::Acute graft-versus-host disease (GVHD) is the primary limitation of allogeneic hematopoietic cell transplantation, and once it develops, there are no reliable diagnostic tests to predict treatment outcomes. We hypothesized that 6 previously validated diagnostic biomarkers of GVHD (IL-2 receptor-α; tumor necrosis facto...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2012-01-403063
更新日期:2012-04-19 00:00:00
abstract::The kidney is involved in 70% of patients with immunoglobulin light-chain (AL) amyloidosis, but little is known on progression or reversibility of renal involvement, and criteria for renal response have never been validated. Newly diagnosed patients from the Pavia (n = 461, testing cohort) and Heidelberg (n = 271, val...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2014-04-570010
更新日期:2014-10-09 00:00:00
abstract::Acute myeloid leukemia (AML) is a major unmet medical need. Most patients have poor long-term survival, and treatment has not significantly changed in 40 years. Recently, bispecific antibodies that redirect the cytotoxic activity of effector T cells by binding to CD3, the signaling component of the T-cell receptor, an...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2016-08-735365
更新日期:2017-02-02 00:00:00
abstract::One hundred eight consecutive patients with indolent lymphoproliferative diseases were stratified into chronic lymphocytic leukemia (CLL), stage III and IV well-differentiated lymphocytic lymphoma (WDLL), and stage III and IV follicular lymphoma (FL). Within each stratum, patients were prospectively and randomly assig...
journal_title:Blood
pub_type: 临床试验,杂志文章,随机对照试验
doi:
更新日期:1987-06-01 00:00:00
abstract::The development of alloantibodies against von Willebrand factor (VWF) represents a rare but serious complication of treatment of von Willebrand disease (VWD), occurring in ~5% to 10% of type 3 VWD patients. Affected patients can present with a range of symptoms, including lack or loss of hemostatic response to infused...
journal_title:Blood
pub_type: 杂志文章,评审
doi:10.1182/blood-2012-10-462085
更新日期:2013-08-01 00:00:00
abstract::Dendritic cells (DCs) are specialized antigen-presenting cells that monitor the antigenic environment and activate naive T cells. The role of DCs is not only to sense danger but also to tolerize the immune system to antigens encountered in the absence of maturation/inflammatory stimuli. Indeed, if a naive T cell encou...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2004-03-1211
更新日期:2005-02-01 00:00:00
abstract::After DNA replication, sister chromatids must be untangled, or decatenated, before mitosis so that chromatids do not tear during anaphase. Topoisomerase IIalpha (Topo IIalpha) is the major decatenating enzyme. Topo IIalpha inhibitors prevent decatenation, causing cells to arrest during mitosis. Here we report that acu...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2008-08-175760
更新日期:2009-08-27 00:00:00
abstract::The CD28 family of receptors (CD28, cytotoxic T-lymphocyte-associated antigen 4 [CTLA-4], inducible costimulator [ICOS], program death-1 [PD-1], and B- and T-lymphocyte attenuator [BTLA]) plays a critical role in controlling the adaptive arm of the immune response. While considerable information is available regarding...
journal_title:Blood
pub_type: 杂志文章,评审
doi:10.1182/blood-2004-04-1596
更新日期:2005-01-01 00:00:00
abstract::Regulatory T (Tr) cells have the potential to treat immune-mediated disease, but cloning such cells for study from patients with autoimmune disease has proven difficult. Here, we describe autoantigen-specific, interleukin-10 (IL-10)-secreting Tr cell clones recovered ex vivo from a patient with autoimmune hemolytic an...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2007-07-101345
更新日期:2008-01-15 00:00:00
abstract::Hematopoietic stem cell transplantation (HSCT) is a highly successful treatment for severe congenital immunodeficiencies. However, some studies have suggested that children may experience cognitive difficulties after HSCT. This large-scale study assessed cognitive and behavioral function for the cohort of children tre...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2008-04-151332
更新日期:2008-11-01 00:00:00
abstract::The clonal growth of progenitor cells from myelodysplastic syndromes (MDS) can be subdivided into four growth patterns: (1) normal, (2) no growth or low plating efficiency, (3) low colony and high cluster number, and (4) normal or high colony number with a large number of clusters. The former two (1 and 2) can be refe...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1996-07-01 00:00:00
abstract::The granulocyte-macrophage colony-stimulating factor receptor (GM-CSFR) is a potential target for toxin-directed therapy, because it is overexpressed on many leukemias and solid tumors and apparently not on stem cells. To investigate the potential therapeutic use of GM-CSF toxins, we fused human GM-CSF to truncated fo...
journal_title:Blood
pub_type: 杂志文章
doi:
更新日期:1997-07-01 00:00:00
abstract::Graft-versus-host disease (GVHD) is a major complication of allogeneic bone marrow transplantation (BMT). When GVHD is controlled by T-cell-depleted grafts or immunosuppressants, BM transplant recipients often suffer from an increased rate of leukemic relapse and impaired reconstitution of immunity. Using a mouse BMT ...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood-2003-12-4309
更新日期:2004-09-01 00:00:00
abstract::Stem cell homing into the bone microenvironment is the first step in the initiation of marrow-derived blood cells. It is reported that human severe combined immunodeficient (SCID) repopulating cells home and accumulate rapidly, within a few hours, in the bone marrow and spleen of immunodeficient mice previously condit...
journal_title:Blood
pub_type: 杂志文章
doi:10.1182/blood.v97.10.3283
更新日期:2001-05-15 00:00:00