GM-CSF pathway correction in pulmonary alveolar proteinosis.

IMPORTANCE OF THE FIELD

Pulmonary alveolar proteinosis (PAP) is a rare disease in which the abnormalities in surfactant metabolism are caused most often by impairments of GM-CSF pathway at different levels in different disease subsets (congenital, secondary, acquired/idiopathic) and for which there are only few, costly invasive therapeutic methods.

AREAS COVERED IN THIS REVIEW

This review discusses these impairments, and their pathogenic and clinical consequences along with potential corrective therapies such as exogenous inhaled GM-CSF.

WHAT THE READER WILL GAIN

Among the PAP disease subsets, in autoimmune PAP the GM-CSF autoantibodies play a major role in disease pathogenesis and their deleterious pulmonary effects can be blocked efficaciously with inhaled GM-CSF.

TAKE HOME MESSAGE

In PAP correction of the abnormalities of the GM-CSF pathway represent a plausible approach demonstrated to be efficacious also in the case of inhaled GM-CSF used for autoimmune PAP.