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Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges

Pulmonary Alveolar Proteinosis (PAP) is a rare syndrome characterized by pulmonary surfactant accumulation within the alveolar spaces. It occurs with a reported prevalence of 0.1 per 100,000 individuals and in distinct clinical forms: autoimmune (previously referred to as the idiopathic form, repres...

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Autores principales: Campo, Ilaria, Kadija, Zamir, Mariani, Francesca, Paracchini, Elena, Rodi, Giuseppe, Mojoli, Francesco, Braschi, Antonio, Luisetti, Maurizio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2012
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3415123/
https://www.ncbi.nlm.nih.gov/pubmed/22958344
http://dx.doi.org/10.1186/2049-6958-7-4
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author Campo, Ilaria
Kadija, Zamir
Mariani, Francesca
Paracchini, Elena
Rodi, Giuseppe
Mojoli, Francesco
Braschi, Antonio
Luisetti, Maurizio
author_facet Campo, Ilaria
Kadija, Zamir
Mariani, Francesca
Paracchini, Elena
Rodi, Giuseppe
Mojoli, Francesco
Braschi, Antonio
Luisetti, Maurizio
author_sort Campo, Ilaria
collection PubMed
description Pulmonary Alveolar Proteinosis (PAP) is a rare syndrome characterized by pulmonary surfactant accumulation within the alveolar spaces. It occurs with a reported prevalence of 0.1 per 100,000 individuals and in distinct clinical forms: autoimmune (previously referred to as the idiopathic form, represents the vast majority of PAP cases, and is associated with Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) auto-antibodies; GMAbs), secondary (is a consequence of underlying disorders), congenital (caused by mutations in the genes encoding for the GM-CSF receptor), and PAP-like syndromes (disorders associated with surfactant gene mutations). The clinical course of PAP is variable, ranging from spontaneous remission to respiratory failure. Whole lung lavage (WLL) is the current standard treatment for PAP patients and although it is effective in the majority of cases, disease persistence is not an unusual outcome, even if disease is well controlled by WLL. In this paper we review the therapeutic strategies which have been proposed for the treatment of PAP patients and the progress which has been made in the understanding of the disease pathogenesis.
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spelling pubmed-34151232012-08-13 Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges Campo, Ilaria Kadija, Zamir Mariani, Francesca Paracchini, Elena Rodi, Giuseppe Mojoli, Francesco Braschi, Antonio Luisetti, Maurizio Multidiscip Respir Med Review Pulmonary Alveolar Proteinosis (PAP) is a rare syndrome characterized by pulmonary surfactant accumulation within the alveolar spaces. It occurs with a reported prevalence of 0.1 per 100,000 individuals and in distinct clinical forms: autoimmune (previously referred to as the idiopathic form, represents the vast majority of PAP cases, and is associated with Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) auto-antibodies; GMAbs), secondary (is a consequence of underlying disorders), congenital (caused by mutations in the genes encoding for the GM-CSF receptor), and PAP-like syndromes (disorders associated with surfactant gene mutations). The clinical course of PAP is variable, ranging from spontaneous remission to respiratory failure. Whole lung lavage (WLL) is the current standard treatment for PAP patients and although it is effective in the majority of cases, disease persistence is not an unusual outcome, even if disease is well controlled by WLL. In this paper we review the therapeutic strategies which have been proposed for the treatment of PAP patients and the progress which has been made in the understanding of the disease pathogenesis. BioMed Central 2012-06-11 /pmc/articles/PMC3415123/ /pubmed/22958344 http://dx.doi.org/10.1186/2049-6958-7-4 Text en Copyright ©2012 Campo et al.; licensee BioMed Central Ltd. http://creativecommons.org/licenses/by/2.0 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Campo, Ilaria
Kadija, Zamir
Mariani, Francesca
Paracchini, Elena
Rodi, Giuseppe
Mojoli, Francesco
Braschi, Antonio
Luisetti, Maurizio
Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges
title Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges
title_full Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges
title_fullStr Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges
title_full_unstemmed Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges
title_short Pulmonary alveolar proteinosis: diagnostic and therapeutic challenges
title_sort pulmonary alveolar proteinosis: diagnostic and therapeutic challenges
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3415123/
https://www.ncbi.nlm.nih.gov/pubmed/22958344
http://dx.doi.org/10.1186/2049-6958-7-4
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