title: Therapy options in pulmonary alveolar proteinosis.
abstract: Pulmonary alveolar proteinosis is a rare condition characterized by the accumulation of lipoproteinaceous material within the airspaces, resulting in impaired gas transfer, and clinical manifestations ranging from asymptomatic to severe respiratory failure. To the best of the authors' knowledge, there are only a few conditions whose natural history has been so dramatically changed by the influence of advances in basic science, clinical medicine, and translational research in therapeutic approaches. Whole-lung lavage is the current standard of care and it plays a critical role as a modifier factor of the natural history of proteinosis. That notwithstanding, the identification of autoantibodies neutralizing granulocyte-macrophage colony-stimulating factor in serum and lung of patients affected by the form of proteinosis previously referred to as idiopathic, has opened the way to novel therapeutic options, such as supplementation of exogenous granulocyte-macrophage colony-stimulating factor, or strategies aimed at reducing the levels of the autoantibodies. The aim of this paper is to provide an updated review of the current therapeutic approach to proteinosis.
authors.affiliation: SC Pneumologia, Fondazione IRCCS, Policlinico San Matteo, Piazza Golgi 1, 27100 Pavia, Italy. m.luisetti@smatteo.pv.it
authors.forename: Maurizio
authors.initials: M
authors.lastname: Luisetti
authors.forename: Zamir
authors.initials: Z
authors.lastname: Kadija
authors.forename: Francesca
authors.initials: F
authors.lastname: Mariani
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Rodi
authors.forename: Ilaria
authors.initials: I
authors.lastname: Campo
authors.forename: Bruce C
authors.initials: BC
authors.lastname: Trapnell
mesh_terms: D000818:Animals; D001323:Autoantibodies / Q000276:immunology*; D018893:Bronchoalveolar Lavage / Q000379:methods*; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000008:administration & dosage* / Q000276:immunology; D006801:Humans; D011649:Pulmonary Alveolar Proteinosis / Q000276:immunology / Q000503:physiopathology / Q000628:therapy*; D012131:Respiratory Insufficiency / Q000209:etiology; D012720:Severity of Illness Index
chemical_list: D001323:Autoantibodies; D016178:Granulocyte-Macrophage Colony-Stimulating Factor