Therapeutic efficacy of granulocyte-macrophage colony-stimulating factor patients with idiopathic acquired alveolar proteinosis

Abstract

Alveolar proteinosis (AP) is characterized by excessive surfactant accumulation, and most cases are of unknown etiology. Standard therapy for AP is whole-lung lavage, which may not correct the underlying defect. Because the hematopoietic cytokine granulocyte-macrophage colony-stimulating factor (GM-CSF) is required for normal surfactant homeostasis, we evaluated the therapeutic activity of GM-CSF in patients with idiopathic AP. Fourteen patients received 5 μg/kg/d GM-CSF for 6 to 12 wk with serial monitoring of the alveolar-arterial oxygen gradient ([A-a]Do2), diffusing capacity of carbon monoxide, computed tomographic scans, and exercise testing. Patients not responding to 5 μg/kg/d GM-CSF ..