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How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future
Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by complement‐mediated intravascular haemolysis, severe thrombophilia and bone marrow failure. While for patients with bone marrow failure the treatment follows that of immune‐mediated aplastic anaemia, that of classic, haemolytic PNH is ba...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291300/ https://www.ncbi.nlm.nih.gov/pubmed/34355382 http://dx.doi.org/10.1111/bjh.17753 |
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author | Risitano, Antonio Maria Peffault de Latour, Régis |
author_facet | Risitano, Antonio Maria Peffault de Latour, Régis |
author_sort | Risitano, Antonio Maria |
collection | PubMed |
description | Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by complement‐mediated intravascular haemolysis, severe thrombophilia and bone marrow failure. While for patients with bone marrow failure the treatment follows that of immune‐mediated aplastic anaemia, that of classic, haemolytic PNH is based on anti‐complement medication. The anti‐C5 monoclonal antibody eculizumab has revolutionized treatment, resulting in control of intravascular haemolysis and thromboembolic risk, with improved long‐term survival. Novel strategies of complement inhibition are emerging. New anti‐C5 agents reproduce the safety and efficacy of eculizumab, with improved patient convenience. Proximal complement inhibitors have been developed to address C3‐mediated extra‐vascular haemolysis and seem to improve haematological response. |
format | Online Article Text |
id | pubmed-9291300 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-92913002022-07-20 How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future Risitano, Antonio Maria Peffault de Latour, Régis Br J Haematol Reviews Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by complement‐mediated intravascular haemolysis, severe thrombophilia and bone marrow failure. While for patients with bone marrow failure the treatment follows that of immune‐mediated aplastic anaemia, that of classic, haemolytic PNH is based on anti‐complement medication. The anti‐C5 monoclonal antibody eculizumab has revolutionized treatment, resulting in control of intravascular haemolysis and thromboembolic risk, with improved long‐term survival. Novel strategies of complement inhibition are emerging. New anti‐C5 agents reproduce the safety and efficacy of eculizumab, with improved patient convenience. Proximal complement inhibitors have been developed to address C3‐mediated extra‐vascular haemolysis and seem to improve haematological response. John Wiley and Sons Inc. 2021-08-05 2022-01 /pmc/articles/PMC9291300/ /pubmed/34355382 http://dx.doi.org/10.1111/bjh.17753 Text en © 2021 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Reviews Risitano, Antonio Maria Peffault de Latour, Régis How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future |
title | How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future |
title_full | How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future |
title_fullStr | How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future |
title_full_unstemmed | How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future |
title_short | How we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future |
title_sort | how we(’ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future |
topic | Reviews |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291300/ https://www.ncbi.nlm.nih.gov/pubmed/34355382 http://dx.doi.org/10.1111/bjh.17753 |
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