Abstract:
PURPOSE OF REVIEW:In this article, we summarize the recent advances in treating primary immune deficiency (PID) disorders by stem cell transplantation (SCT); we have focused on articles published in the past 2 years since the last major review of SCT for PID. RECENT FINDINGS:Analyses of the outcomes of SCT for PID by specific molecular defect have clarified which conditions are receptive to unconditioned transplants and which require more myeloablative conditioning. Improved outcomes for 'difficult' conditions [adenosine deaminase-severe combined immunodeficiency (ADA-SCID), major histocompatibility complex class II deficiency] and potential advantages of using cord blood as a stem cell source have also been described. Newborn screening for SCID identifies well babies with SCID: the optimal SCT protocol for such young infants remains to be determined. Reduced toxicity conditioning has been successfully used to treat conditions such as Wiskott-Aldrich syndrome and chronic granulomatous disease, offering curative engraftment with reduced transplant-related mortality. Similarly, treating children with familial hemophagocytic lymphohistiocytosis using reduced intensity conditioning SCT results in much improved outcomes. Advances in next generation sequencing have identified new diseases amenable to SCT, such as DOCK8 deficiency, resulting in improved quality of life and protection from malignancy. SUMMARY:Recent studies suggest that further improvements in treating PID with SCT are possible with a greater understanding of the genetics and immunobiology of these diseases, facilitating the matching of donor type and conditioning regimens, or indeed alternative therapies (such as gene therapy) to specific PID disorders.
journal_name
Curr Opin Hematoljournal_title
Current opinion in hematologyauthors
Worth AJ,Booth C,Veys Pdoi
10.1097/MOH.0b013e328365a13bsubject
Has Abstractpub_date
2013-11-01 00:00:00pages
501-8issue
6eissn
1065-6251issn
1531-7048pii
00062752-201311000-00004journal_volume
20pub_type
杂志文章,评审abstract::Within the past year substantial progress has been made in understanding the molecular changes underlying the development of acute leukemia. Development of molecular probes allowed substantial improvements in the detection of minimal residual disease. Pilot studies underlined the correlation between the expression of ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1994-07-01 00:00:00
abstract::Thrombopoietin, also termed the c-Mpl ligand, is a lineage-dominant hematopoietic factor that primarily regulates megakaryopoiesis and thrombopoiesis. Treatment of normal animals with recombinant human megakaryocyte growth and development factor, a truncated molecule of the c-Mpl ligand, which is modified with polyeth...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199805000-00009
更新日期:1998-05-01 00:00:00
abstract::Platelet glycoprotein (GP) IIb/IIIa receptor antagonists are being used with increasing frequency in the settings of percutaneous coronary interventions and acute ischemic syndromes. The development of bleeding complications following GPIIb/IIIa blockade represents a significant limitation to its effectiveness. Baseli...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199909000-00011
更新日期:1999-09-01 00:00:00
abstract:PURPOSE OF REVIEW:The transmembrane protein tissue factor (TF), the initiator of the extrinsic coagulation cascade and a risk factor for thrombotic disease, largely resides on the cell surface in an inactive, encrypted conformation. Whereas TF procoagulant activation, or decryption, is closely associated with the expos...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e32834981de
更新日期:2011-09-01 00:00:00
abstract:PURPOSE OF REVIEW:The role of a patent foramen ovale (PFO) as a risk factor for ischemic stroke has been established in recent years. However, the best therapeutic option to prevent recurrent events is still controversial, with antithrombotic treatment or transcatheter PFO closure being favored by different clinicians....
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e32832d47dd
更新日期:2009-09-01 00:00:00
abstract::Over a decade has passed since the first cases of AIDS were reported in hemophiliacs and transfusion recipients. Studies published over the past year offer an increasingly clear understanding of the extent of the early transfusion-associated AIDS epidemic, and of the effectiveness of progressive donor screening measur...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1994-11-01 00:00:00
abstract:PURPOSE OF REVIEW:Chronic inflammation and disordered lipid metabolism represent hallmarks of atherosclerosis. Considerable evidence has accumulated to suggest that innate immune defense mechanisms might interact with proinflammatory pathways and exacerbate or perhaps even initiate development of arterial plaques. Unti...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/01.moh.0000219662.88409.7c
更新日期:2006-05-01 00:00:00
abstract:PURPOSE OF REVIEW:Immune responses to commensal bacteria and resulting chronic inflammation are hallmarks of inflammatory bowel disease. Considerable evidence has accumulated to suggest that innate immune defense mechanisms interact with pro-inflammatory pathways and exacerbate or perhaps even initiate development of i...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200701000-00010
更新日期:2007-01-01 00:00:00
abstract::Despite the reliance on platelet transfusion support in patients receiving myeloablative therapy, controversies surround platelet transfusion practices. These include the appropriate platelet dose and the threshold at which prophylactic platelet transfusions will be most effective. These issues bear directly on patien...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199911000-00015
更新日期:1999-11-01 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: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:Iron homeostasis and erythropoiesis regulate each other to ensure optimal delivery of oxygen and iron to cells and tissues. Defining the mechanisms of this crosstalk is important for understanding the pathogenesis of common conditions associated with disordered iron metabolism and erythropoiesis. REC...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000132
更新日期:2015-05-01 00:00:00
abstract:PURPOSE OF REVIEW:In this review, we focus on three specific concepts related to platelet transfusion in the neonatal and pediatric population: choice of transfusion threshold; use of ABO-mismatched platelets; transfusion of pathogen-reduced or inactivated platelets. RECENT FINDINGS:Recent trials support the use of lo...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000542
更新日期:2019-11-01 00:00:00
abstract:PURPOSE OF REVIEW:There are three kindlin family members in vertebrates, which have high-sequence homology and a common organization signature with a C-terminal 4.1, ezrin, radixin, moesin (FERM) domain bisected by a pleckstrin-homology domain. Although the cell and tissue distributions of the three kindlins differ, th...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e32832ea389
更新日期:2009-09-01 00:00:00
abstract:PURPOSE OF REVIEW:Hematopoietic stem cells (HSCs) possess two fundamental characteristics, the capacity for self-renewal and the sustained production of all blood cell lineages. The fine balance between HSC expansion and lineage specification is dynamically regulated by the interplay between external and internal stimu...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0000000000000260
更新日期:2016-07-01 00:00:00
abstract:PURPOSE OF REVIEW:To discuss how mechanical cues and Rho signaling contribute to control of vascular development and hematopoiesis. RECENT FINDINGS:Rho guanine trinucleotide phosphatases are ubiquitious regulators of cytoskeletal structure and tension generation. Recent work shows that Rho-dependent mechanical interac...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e3282fa7445
更新日期:2008-05-01 00:00:00
abstract::Allogeneic barriers to transfusion are caused by differences between those portions of the donor and recipient genomes that define the antigenicity and immune response to the transfused cells. Historically, a blood group antigen was identified when an immune response (alloantibody) was detected by hemagglutination in ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200203000-00012
更新日期:2002-03-01 00:00:00
abstract:PURPOSE OF REVIEW:This review focuses on recent advances in the use of immune-based therapy to treat patients with refractory and relapsing acquired thrombotic thrombocytopenic purpura. RECENT FINDINGS:Advances in understanding of the pathophysiology of idiopathic thrombotic thrombocytopenic purpura have provided the ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/01.moh.0000170534.33517.99
更新日期:2005-09-01 00:00:00
abstract::Acute chest syndrome (ACS) is the presence of a new pulmonary infiltrate in combination with fever or respiratory symptoms in a patient with sickle cell disease. ACS is the leading cause of death in sickle cell disease, and many patients suffer from multiple, severe episodes. Age has a striking effect on the clinical ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199803000-00002
更新日期:1998-03-01 00:00:00
abstract:PURPOSE OF REVIEW:While the risks of transfusion-transmitted human immunodeficiency virus, hepatitis C virus, and human T-cell leukemia virus I/II continue to decrease, additional threats to transfusion safety are posed by emerging "new" infectious diseases. RECENT FINDINGS:Following the introduction of nucleic acid t...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200311000-00003
更新日期:2003-11-01 00:00:00
abstract::Advances in platelet transfusion have contributed to improved outcomes in the treatment of patients with cancer and leukemia. However, the optimal strategies to avoid some of the side effects that could result from platelet transfusions remain under investigation. These side effects include the development of refracto...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199603050-00003
更新日期:1996-09-01 00:00:00
abstract:PURPOSE OF REVIEW:Although several clinical studies have investigated the effects of different treatment periods in subjects after a VTE event, the optimal duration of oral anticoagulation is still uncertain. Our review focused on studies that evaluated different risk factors that might be useful to stratify patients i...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/01.moh.0000132310.18883.c0
更新日期:2004-05-01 00:00:00
abstract::Much of our understanding of the organization of the cells that comprise the hematopoietic system and the cellular and molecular mechanisms that regulate their development is derived from mouse models. However, knowledge of the human hematopoietic system and identification of human stem cells have, until recently, bee...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199603060-00002
更新日期:1996-11-01 00:00:00
abstract:PURPOSE OF REVIEW:The role of bone morphogenetic proteins (BMPs) in vasculogenesis is still not well understood, despite many recent developments in this area of research. In this review, we discuss the most recent studies that identify new critical mechanisms through which BMP signaling acts with a focus on angiogenes...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e32832a07d6
更新日期:2009-05-01 00:00:00
abstract::The aim of gene therapy for hemophilia is the stable introduction and expression of a gene encoding functional blood coagulation factor VIII or IX. Although there are as yet no published studies demonstrating long-term expression of therapeutic levels in large animal models of the disease, there have been several repo...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-199809000-00003
更新日期:1998-09-01 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:Intravenous immunoglobulin (IVIg) is an effective treatment for an increasing number of autoimmune and inflammatory conditions. However, IVIg continues to be limited by problems of potential shortages and cost. A number of mechanisms have been described for IVIg, which have been captured in newly emer...
journal_title:Current opinion in hematology
pub_type: 杂志文章
doi:10.1097/MOH.0000000000000609
更新日期:2020-11-01 00:00:00
abstract::FLT3 is the most frequently mutated gene in cases of acute myelogenous leukemia (AML). About 30 to 35% of patients have either internal tandem duplications (ITDs) in the juxtamembrane domain or mutations in the activating loop of FLT3. FLT3 mutations occur in a broad spectrum of FAB subtypes in adult and pediatric AML...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200207000-00003
更新日期:2002-07-01 00:00:00
abstract:PURPOSE OF REVIEW:The discovery of JAK2V617F and other JAK-STAT-activating mutations in BCR-ABL1-negative myeloproliferative neoplasms (MPN) has led to the development of small-molecule ATP-mimetics that inhibit wild-type and mutant JAK. Here, we review the current experience with JAK inhibitors used for the treatment ...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/MOH.0b013e3283439964
更新日期:2011-03-01 00:00:00
abstract::Platelet aggregation, a process mediated by GP IIb-IIIa, is responsible for the occlusive events in thrombosis: indeed, GP IIb-IIIa antagonists are effective in blocking arterial thrombosis. Recent studies have suggested that activated platelets and platelet thrombi can contribute significantly to the initiation and p...
journal_title:Current opinion in hematology
pub_type: 杂志文章,评审
doi:10.1097/00062752-200309000-00006
更新日期:2003-09-01 00:00:00