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ABSTRACT
Introduction
Pulmonary alveolar proteinosis (PAP) is a heterogeneous group of rare diseases characterized by the abnormal production and impaired degradation of pulmonary surfactant as a result of malfunctioning of alveolar macrophages. This is due to the downstream dysregulation of the GM-CSF pathway, which can be caused by specific autoantibodies (autoimmune, aPAP formerly known as idiopathic iPAP), direct injury to alveolar macrophages (e.g. by toxic inhaled agents.), or by genetic defects (hereditary or congenital PAP). Few pharmacotherapy options are currently available to treat this disease.
Area covered
The authors discuss the exogenous administration of GM-CSF, rituximab, and the potential role of cholesterol lowering medications in this review. The authors, furthermore, provide their opinion on the available pharmacotherapeutic options and give their future perspectives.
Expert opinion
Inhaled GM-CSF remains the most commonly used therapy in patients with iPAP but other inhaled therapies such as PPARγ activators should be considered, especially in patients who are partially responsive or unresponsive to traditional treatments.
Article Highlights
Pulmonary alveolar proteinosis is a rare interstitial lung disease with a large range of etiologies, from idiopathic to secondary to chemical aggressions.
It is characterized by the malfunctioning of the pulmonary surfactant which is caused by abnormalities in its composition
These can be caused by multiple factors and the main pathogenic feature is represented by the impaired GM-CSF pathway
Anti GM-CSF autoantibodies are detected in autoimmune PAP and have recently evoled as a diagnostic test
Exogenous GM-CSF given via subcutaneous or inhalation routes is the main pharmacotherapeutic approach currently available for autoimmune PAP
Other potential pharmacotherapies might be represented by pioglitazone, rituximab or statins
This box summarizes key points contained in the article.
Declaration of interest
S Antoniu has received congress fees from Chiesi Farmaceutici and Angellini. R Rajnoveanu has received both congress and speaker’s fees from and has served on the advisory boards of Novartis, Chiesi Farmaceutici, AstraZeneca, GlaxoSmithKline, Sandoz and Boehringer Ingelheim. M Grigore has received congress and speaker’s fees from Antibiotice SA, Gedeon Richter, Theramex, ADPharma, Pharma Promotion Srl and Dr Reddy’s Laboratories. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Reviewer Disclosures
Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.