Long-Term Inhaled Granulocyte Macrophage–Colony-Stimulating Factor in Autoimmune Pulmonary Alveolar Proteinosis: Effectiveness, Safety, and Lowest Effective Dose

• Published in: Clinical drug investigation 2014-08, Vol.34 (8), p.553-564
• Main Authors: Papiris, Spyros A., Tsirigotis, Panagiotis, Kolilekas, Likurgos, Papadaki, Georgia, Papaioannou, Andriana I., Triantafillidou, Christina, Papaporfyriou, Anastasia, Karakatsani, Anna, Kagouridis, Konstantinos, Griese, Matthias, Manali, Effrosyni D.
• Format: Article
• Language: English
• Subjects: Adult; Aged; Autoimmune Diseases - diagnosis; Autoimmune Diseases - drug therapy; Cohort Studies; Dose-Response Relationship, Drug; Female; Follow-Up Studies; Granulocyte-Macrophage Colony-Stimulating Factor - administration & dosage; Granulocyte-Macrophage Colony-Stimulating Factor - agonists; Humans; Internal Medicine; Male; Medicine; Medicine & Public Health; Middle Aged; Original Research Article; Pharmacology/Toxicology; Pharmacotherapy; Pulmonary Alveolar Proteinosis - diagnosis; Pulmonary Alveolar Proteinosis - drug therapy; Retrospective Studies; Treatment Outcome; Young Adult
• ISSN: 1173-2563; 1179-1918
• DOI: 10.1007/s40261-014-0208-z

Background and Objectives Granulocyte macrophage–colony-stimulating factor (GM-CSF) causes variable improvement in autoimmune pulmonary alveolar proteinosis (aPAP). Upon response to short-term treatment, patients are divided into responders and non-responders. The aim of this study was to test the hypothesis that long-term inhaled GM-CSF (iGM-CSF) is effective in all patients and that attainment of remission permits gradual de-escalation of the dose to the lowest effective safe dose. Methods Patients were treated with iGM-CSF 250 μg once a day given 4 days on and 4 days off for as long as necessary (the “as far as it takes” protocol). Upon remission, defined as absence of symptoms, oxygen desaturation