GM-CSF pathway correction in pulmonary alveolar proteinosis

Expert Opin Biol Ther. 2010 Sep;10(9):1357-65. doi: 10.1517/14712598.2010.510507.

Abstract

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.

Publication types

  • Review

MeSH terms

  • Administration, Inhalation
  • Animals
  • Autoantibodies / metabolism
  • Granulocyte-Macrophage Colony-Stimulating Factor / administration & dosage*
  • Granulocyte-Macrophage Colony-Stimulating Factor / immunology
  • Granulocyte-Macrophage Colony-Stimulating Factor / metabolism
  • Humans
  • Immunologic Factors / administration & dosage*
  • Pulmonary Alveolar Proteinosis / drug therapy*
  • Pulmonary Alveolar Proteinosis / immunology
  • Pulmonary Alveolar Proteinosis / pathology
  • Signal Transduction / drug effects*
  • Treatment Outcome

Substances

  • Autoantibodies
  • Immunologic Factors
  • Granulocyte-Macrophage Colony-Stimulating Factor