Abstract:
:Paroxysmal nocturnal haemoglobinuria (PNH) is a serious form of acquired haemolytic anaemia with several features that make it unique, including the fact that it is caused by clonal expansion, in the context of bone marrow failure, of a haematopoietic stem cell that has a somatic mutation in a gene crucial for the synthesis of glycosylphosphatidylinositol anchors; and that this also produces a life-threatening acquired thrombophilic state. Until recently, the two only main options for patients with PNH were either allogeneic bone marrow transplantation or supportive management, including blood transfusion: both options require some skill and good patient-doctor collaboration. Since the start of this millennium a major advance has been the introduction of eculizumab, a monoclonal antibody that targets the C5 protein of the complement system: blockade of C5 prevents activation of the complement distal pathway, and thus abrogates the complement-mediated intravascular haemolysis that severely plagues patients with PNH. This review outlines an approach to the management of all three major components of the clinical picture of PNH--namely haemolysis, thrombosis and bone marrow failure--based on the literature and on personal experience. We consider specifically how the use of eculizumab has modified other aspects of the management of PNH, and even the pathophysiology itself of this disease. Finally, we develop a treatment algorithm which others might find helpful.
journal_name
Br J Haematoljournal_title
British journal of haematologyauthors
Luzzatto L,Gianfaldoni G,Notaro Rdoi
10.1111/j.1365-2141.2011.08690.xsubject
Has Abstractpub_date
2011-06-01 00:00:00pages
709-20issue
6eissn
0007-1048issn
1365-2141journal_volume
153pub_type
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journal_title:British journal of haematology
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abstract::The peripheral T-cell neoplasms are a biologically and clinically heterogeneous group of rare disorders that result from clonal proliferation of mature post-thymic lymphocytes. Natural killer (NK) cell neoplasms are included in this group. The World Health Organization classification of haemopoietic malignancies has d...
journal_title:British journal of haematology
pub_type: 杂志文章,实务指引,评审
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更新日期:2011-05-01 00:00:00
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journal_title:British journal of haematology
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abstract::The inoculation of L1210 or L5178Y leukaemia cells decreased the platelet count of the recipient mouse before extensive infiltration of marrow was expected by either tumour line. The decrease was more pronounced after intravenous inoculation of L1210 leukaemia than after intraperitoneal inoculation. Inoculation of L51...
journal_title:British journal of haematology
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journal_title:British journal of haematology
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journal_title:British journal of haematology
pub_type: 杂志文章
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journal_title:British journal of haematology
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journal_title:British journal of haematology
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abstract::Reducing infectious morbidity is an important goal to improve childhood acute lymphoblastic leukaemia (ALL) survival. To explore the impact of chemotherapy reduction on infectious morbidity, we compared outpatient and inpatient infectious morbidity of reduced versus intensive (conventional) chemotherapy. One hundred a...
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journal_title:British journal of haematology
pub_type: 杂志文章
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journal_title:British journal of haematology
pub_type: 杂志文章
doi:10.1046/j.1365-2141.1998.00852.x
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abstract::Patients with solid tumours undergoing high-dose chemotherapy with autologous bone marrow transplantation use an average of 10 units of packed red blood cells (PRBC) while awaiting haemopoietic reconstitution. They are also known to have inappropriately low endogenous erythropoietin levels for their degree of anaemia....
journal_title:British journal of haematology
pub_type: 临床试验,杂志文章
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更新日期:1994-05-01 00:00:00
abstract::An 'artificial' plasma for one-stage factor-VIII assays is made by incubating human plasma with EDTA, to destroy factor VIII, and afterwards removing the anticoagulant by dialysis. Bovine factor V is then added to a given level. In the assay, contact activation is controlled by adding contact product. It was confirmed...
journal_title:British journal of haematology
pub_type: 杂志文章
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abstract::The Australian Leukaemia Study Group has performed a randomized trial of interferon alpha-2A (Roferon-A) as a co-induction agent together with intensive combination chemotherapy and as maintenance following completion of 12 cycles of induction treatment. When used as a co-induction agent, interferon-alpha did not impr...
journal_title:British journal of haematology
pub_type: 临床试验,杂志文章,随机对照试验
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更新日期:1997-04-01 00:00:00
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journal_title:British journal of haematology
pub_type: 杂志文章,meta分析,评审
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更新日期:2013-11-01 00:00:00
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journal_title:British journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1365-2141.1987.tb06157.x
更新日期:1987-12-01 00:00:00
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journal_title:British journal of haematology
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pub_type: 杂志文章
doi:10.1046/j.1365-2141.1996.d01-2683.x
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abstract::Failed surface expression of the complement decay-accelerating factor (DAF) due to mutation of the PIG-A gene is a hallmark of affected paroxysmal nocturnal haemoglobinuria (PNH) blood elements. Previous findings that acute myelogenous leukaemia (AML) blasts evolving in a PNH patient differed from idiopathic AML blast...
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pub_type: 杂志文章,多中心研究,随机对照试验
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journal_title:British journal of haematology
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journal_title:British journal of haematology
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