| Management of Paroxysmal Nocturnal Haemoglobinuria: a personal view. | |
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MedLine Citation:
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PMID: 21517820 Owner: NLM Status: Publisher |
Abstract/OtherAbstract:
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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. |
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Authors:
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Lucio Luzzatto; Giacomo Gianfaldoni; Rosario Notaro |
Publication Detail:
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Type: JOURNAL ARTICLE Date: 2011-4-22 |
Journal Detail:
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Title: British journal of haematology Volume: - ISSN: 1365-2141 ISO Abbreviation: - Publication Date: 2011 Apr |
Date Detail:
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Created Date: 2011-4-26 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 0372544 Medline TA: Br J Haematol Country: - |
Other Details:
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Languages: ENG Pagination: - Citation Subset: - |
Copyright Information:
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© 2011 Blackwell Publishing Ltd. |
Affiliation:
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Istituto Toscano Tumori, Firenze Division of Haematology, Azienda Ospedaliero-Universitaria Careggi, Firenze Core Research Laboratory, Istituto Toscano Tumori, Azienda Ospedaliero-Universitaria Careggi, Firenze, Italy. |
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