Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome.

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 is the major pathophysiological event in all of these inherited metabolic storage diseases.

journal_name

Curr Opin Hematol

authors

Krivit W,Aubourg P,Shapiro E,Peters C

doi

10.1097/00062752-199911000-00004

keywords:

subject

Has Abstract

pub_date

1999-11-01 00:00:00

pages

377-82

issue

6

eissn

1065-6251

issn

1531-7048

journal_volume

6

pub_type

杂志文章,评审
  • Methods and progress in assessing the quality of life effects of supportive care with erythropoietin therapy.

    abstract::Anemia is a common disorder in patients with cancer and can be caused by the disease itself or by cancer-related therapy. The cardinal symptom of anemia, fatigue, is the most commonly reported symptom in patients with cancer and has profound effects on patient well-being and quality of life. Until recently, blood tran...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-200205000-00010

    authors: Yount S,Lai JS,Cella D

    更新日期:2002-05-01 00:00:00

  • Novel strategies for steroid-refractory acute graft-versus-host disease.

    abstract:PURPOSE OF REVIEW:Graft-versus-host disease is one of the commonest complications of allogeneic bone marrow or peripheral blood stem cell transplantation. This review will cover advances in the pathophysiology of graft-versus-host disease and new agents under investigation for the treatment of this disorder. Patients d...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/01.moh.0000148763.12733.bb

    authors: Bolaños-Meade J,Vogelsang GB

    更新日期:2005-01-01 00:00:00

  • MicroRNAs in endothelial cell homeostasis and vascular disease.

    abstract:PURPOSE OF REVIEW:Since the first discovery of microRNAs (miRNAs) in 1993, the involvement of miRNAs in different aspects of vascular disease has emerged as an important research field. In this review, we summarize the fundamental roles of miRNAs in controlling endothelial cell functions and their implication with seve...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0000000000000424

    authors: Fernández-Hernando C,Suárez Y

    更新日期:2018-05-01 00:00:00

  • Unexpected functions of nuclear factor-κB during germinal center B-cell development: implications for lymphomagenesis.

    abstract:PURPOSE OF REVIEW:B-cell tumors originating from the transformation of germinal center B cells frequently harbor genetic mutations, leading to constitutive activation of the nuclear factor-κB (NF-κB) signaling pathway. The present review highlights recent insights into the roles of separate NF-κB transcription factors ...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0000000000000160

    authors: Klein U,Heise N

    更新日期:2015-07-01 00:00:00

  • Rho signaling and mechanical control of vascular development.

    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

    authors: Mammoto A,Mammoto T,Ingber DE

    更新日期:2008-05-01 00:00:00

  • The platelet proteome.

    abstract:PURPOSE OF REVIEW:The proteome is the pool of proteins expressed at a given time and circumstance. The word 'proteomics' summarizes several technologies for visualization, quantitation and identification of these proteins. Recent advances in these techniques are helping to elucidate platelet processes which are relevan...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0b013e32832e9dc6

    authors: Senzel L,Gnatenko DV,Bahou WF

    更新日期:2009-09-01 00:00:00

  • Targeting myeloproliferative neoplasms with JAK inhibitors.

    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

    authors: Pardanani A,Tefferi A

    更新日期:2011-03-01 00:00:00

  • Pathophysiology of transfusion-related acute lung injury.

    abstract:PURPOSE OF REVIEW:The purpose of this review is to summarize the recent experimental and clinical literature on the pathogenesis of transfusion-related acute lung injury (TRALI). RECENT FINDINGS:In both experimental and clinical TRALI, an immune priming step is generally necessary to produce lung injury. Experimental ...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0b013e32833c07d3

    authors: Looney MR,Gilliss BM,Matthay MA

    更新日期:2010-09-01 00:00:00

  • Epstein-Barr virus and lymphoproliferative disease.

    abstract::The Epstein-Barr virus is a ubiquitous human herpesvirus that is associated with an increasing number of human malignancies. Among these are Epstein-Barr virus-associated lymphoproliferative diseases in immunocompromised patients, a spectrum of mainly B-cell diseases that range from polyclonal lymphoproliferative dise...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-199901000-00005

    authors: Mosier DE

    更新日期:1999-01-01 00:00:00

  • Endothelial cell disease: emerging knowledge from cerebral cavernous malformations.

    abstract:PURPOSE OF REVIEW:Endothelial cells dysfunctions are crucial determinants of several human diseases. We review here the most recent reports on endothelial cell defects in cerebral cavernous malformations (CCMs), particularly focusing on adherens junctions. CCM is a vascular disease that affects specifically the venous ...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0000000000000338

    authors: Lampugnani MG,Malinverno M,Dejana E,Rudini N

    更新日期:2017-05-01 00:00:00

  • Clonal diversity of the stem cell compartment.

    abstract:PURPOSE OF REVIEW:Hematopoietic stem cells are functionally heterogeneous even when isolated as phenotypically homogenous populations. How this heterogeneity is generated is incompletely understood. Several models have been formulated to explain the generation of diversity. All of these assume the existence of a single...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/01.moh.0000231421.00407.65

    authors: Muller-Sieburg CE,Sieburg HB

    更新日期:2006-07-01 00:00:00

  • The use of granulocyte colony-stimulating factor for treatment of autoimmune neutropenia.

    abstract::The advent of better diagnostic tools, molecular techniques, and cytokine therapies have come together at the end of the 20th century to provide an improved outlook for patients with autoimmune neutropenia. Severe chronic neutropenia is no longer idiopathic in most cases, and growth factor therapy can be safely offere...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-200105000-00007

    authors: Smith MA,Smith JG

    更新日期:2001-05-01 00:00:00

  • Recent advances in the biology and treatment of childhood acute lymphoblastic leukemia.

    abstract::The molecular analysis of genetic alterations in acute lymphoblastic leukemia has led to the development of molecular diagnostic techniques that have improved the biologic characterization of newly diagnosed patients. Modern treatment protocols rely on this information to tailor the intensity of therapy to the risk of...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-199704040-00003

    authors: Rubnitz JE,Pui CH

    更新日期:1997-07-01 00:00:00

  • The use of hematopoietic growth factors in treating HIV infection.

    abstract::Human immunodeficiency virus infection causes multilineage hematopoietic defects. Defects in the production and function of CD4+ helper cells have been the focus of the majority of HIV research, but anemia, neutropenia, and thrombocytopenia are significant clinical problems as well. Bone marrow suppression is the dose...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-199502030-00012

    authors: Miles S

    更新日期:1995-05-01 00:00:00

  • Objectives and limitations of bloodless medical care.

    abstract:PURPOSE OF REVIEW:To outline the rationale, limitations, and execution of bloodless medical and surgical programs, highlighting characteristics that contribute to successful outcomes. RECENT FINDINGS:Clinical experiences with patients who refuse blood transfusions for religious reasons have provided valuable lessons a...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/01.moh.0000245692.32085.bd

    authors: Shander A,Goodnough LT

    更新日期:2006-11-01 00:00:00

  • Hypereosinophilia.

    abstract::Recent advances in our understanding of hypereosinophilia have related particularly to the definition of criteria for distinguishing between eosinophilic leukemia and the 'idiopathic' hypereosinophilic syndromes. In this article, leukemogenic mechanisms have been identified in a number of subtypes of eosinophilic leuk...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-200001000-00005

    authors: Bain BJ

    更新日期:2000-01-01 00:00:00

  • Platelets, leukocytes, and coagulation.

    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

    authors: Bouchard BA,Tracy PB

    更新日期:2001-09-01 00:00:00

  • Ras-related GTP-binding proteins and leukocyte signal transduction.

    abstract::Many aspects of leukocyte function are regulated by both heterotrimeric and Ras-related GTP-binding proteins, but there is little definite information about their roles in the specialized processes utilized by leukocytes for cell killing. Recent progress in understanding the regulation of the phagocyte NADPH oxidase b...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章

    doi:

    authors: Bokoch GM,Knaus UG

    更新日期:1994-01-01 00:00:00

  • Acute leukemia.

    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:

    authors: Ganser A,Hoelzer D

    更新日期:1994-07-01 00:00:00

  • Microtransplantation: clinical applications and mechanisms.

    abstract:PURPOSE OF REVIEW:Microtransplantation (MST) has been successfully used to manage patients with hematological malignancies, especially older patients with acute myeloid leukemia (AML). Recent clinical applications and mechanistic studies of MST are reviewed. RECENT FINDINGS:MST improves complete remission rates which ...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0000000000000470

    authors: Cai B,Guo M,Ai H

    更新日期:2018-11-01 00:00:00

  • Angiogenesis: vascular remodeling of the extracellular matrix involves metalloproteinases.

    abstract::Endothelial cell invasion is an essential event during angiogenesis (the formation of new blood vessels). This process involves the degradation of the extracellular matrix, the basement membrane, and interstitial stroma, and is governed by the activation of matrix metalloproteinases. However, the contribution of matri...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-200303000-00007

    authors: Heissig B,Hattori K,Friedrich M,Rafii S,Werb Z

    更新日期:2003-03-01 00:00:00

  • Biologic functions of blood group antigens.

    abstract::In the past few years, we have learned a great deal about the biologic function of structures bearing blood group antigens. Some blood group antigen-bearing proteins function as major transport channels within the erythrocyte membrane; these include the anion transporter (band 3: Diego and Wright antigens), the water ...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-199603060-00013

    authors: Mudad R,Telen MJ

    更新日期:1996-11-01 00:00:00

  • Molecular basis of erythrocyte membrane disorders.

    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

    authors: Gallagher PG,Ferriera JD

    更新日期:1997-03-01 00:00:00

  • Endothelial cell biology of Endoglin in hereditary hemorrhagic telangiectasia.

    abstract:PURPOSE OF REVIEW:Mutations in the Endoglin (Eng) gene, an auxiliary receptor in the transforming growth factor beta (TGFβ)-superfamily signaling pathway, are responsible for the human vascular disorder hereditary hemorrhagic telangiectasia (HHT) type 1, characterized in part by blood vessel enlargement. A growing body...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0000000000000419

    authors: Sugden WW,Siekmann AF

    更新日期:2018-05-01 00:00:00

  • D-dimer for the diagnosis of venous thromboembolism.

    abstract::D-dimer, a breakdown product of cross-linked fibrin has been extensively evaluated as a diagnostic test for acute venous thromboembolism. Rapid, highly sensitive D-dimer assays are now available that are suitable for testing in the emergency setting. Preliminary studies suggest that when using a highly sensitive D-dim...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-200009000-00007

    authors: Anderson DR,Wells PS

    更新日期:2000-09-01 00:00:00

  • Neutrophilic dermatosis: disease mechanism and treatment.

    abstract:PURPOSE OF REVIEW:The purpose of this review is to describe the physiopathological and therapeutic aspects of neutrophilic dermatosis, taking into account their most frequent associated conditions. RECENT FINDINGS:In autoinflammatory syndromes featuring neutrophilic dermatosis, the role of interleukin-1 and tumor necr...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0000000000000100

    authors: Maalouf D,Battistella M,Bouaziz JD

    更新日期:2015-01-01 00:00:00

  • Recent advances in multiple myeloma.

    abstract::Multiple myeloma is the second most common hematologic malignancy, with approximately 15,000 new cases each year in the United States. Our understanding of the pathophysiology underlying myeloma continues to expand, but the cause of this plasma cell dyscrasia remains unclear. Though controversy remains regarding a pos...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/00062752-200007000-00007

    authors: Sjak-Shie NN,Vescio RA,Berenson JR

    更新日期:2000-07-01 00:00:00

  • Bleeding complications with platelet glycoprotein IIb/IIIa receptor antagonists.

    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

    authors: Madan M,Blankenship JC,Berkowitz SD

    更新日期:1999-09-01 00:00:00

  • Guidelines for newborn screening of primary immunodeficiency diseases.

    abstract:PURPOSE OF REVIEW:Technical possibilities to screen for inborn errors of immune function at the neonatal stage have been rapidly progressing, whereas the guidelines that apply for the evaluation of benefits and concerns on expanding screening panels have not been broadly discussed for primary immunodeficiency diseases ...

    journal_title:Current opinion in hematology

    pub_type: 杂志文章,评审

    doi:10.1097/MOH.0b013e32835a9130

    authors: Borte S,von Döbeln U,Hammarström L

    更新日期:2013-01-01 00:00:00

  • Human stem cell assays in immune-deficient mice.

    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

    authors: Dick JE

    更新日期:1996-11-01 00:00:00