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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...

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Detalles Bibliográficos
Autores principales: Risitano, Antonio Maria, Peffault de Latour, Régis
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
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.
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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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