Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis.

ABSTRACT:

Background

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disease characterized by progressive surfactant accumulation and hypoxemia. It is caused by disruption of granulocyte-macrophage colony-stimulating factor (GM-CSF) signaling, which pulmonary alveolar macrophages require to clear surfactant. Recently, inhaled GM-CSF was shown to improve the partial pressure of arterial oxygen in patients with aPAP.

Methods

In a double-blind, placebo-controlled, three-group trial, we randomly assigned patients with aPAP to receive the recombinant GM-CSF molgramostim (300 μg once daily by inhalation), either continuously or intermittently (every other week), or matching placebo. The 24-week intervention period was followed by an open-label treatment-extension period. The prim

SUBMITTER: Trapnell BC 

PROVIDER: S-EPMC8083051 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature