Successful treatment of pulmonary alveolar proteinosis with whole lung lavage and subcutaneous GM-CSF

Abstract

Abstract Introduction Pulmonary alveolar proteinosis (PAP) is a rare lung disease characterized by the accumulation of a periodic acid-Shiff-positive lipoproteinaceous material (surfactant) in the distal airways. The standard treatment is whole lung lavage (WLL), however, not all patients respond well to WLL. Aim We present an exciting case report on the successful treatment of the rare disease PAP with WLL and subcutaneous granulocyte macrophage colony-stimulating factor (GM-CSF) therapy for 12 weeks. Case study A 20 year-old woman with a 7-month history of progressive shortness of breath was referred to us. Computed tomography of the chest revealed typical geographic ground-glass opacity combined with interlobular septal thickening (crazy paving). Pulmonary function tests showed restriction with severe impairment of diffusion capacity (DLCO 20%). Milky bronchoalveolar lavage fluid (BALF) was detected during bronchoscopy. The cytology of the BALF was consistent with PAP. WLL was performed and subcutaneous GM-CSF was started immediately and continued for 12 weeks. At the end of treatment, the patient's lung function was clearly improved (DLCO 62%). Results and discussion One year after the withdrawal of treatment, the patient remains in good health, with no recurrence of hypoxemia and respiratory symptoms. Different treatment modalities have been applied since PAP was first defined, and WLL has been the preferred first line of treatment. Although our patient‘s condition improved after WLL, her respiratory insufficiency persisted. Consequently, we initiated treatment with subcutaneous GM-CSF. Conclusions WLL significantly improved our patient's condition, and GM-CSF was effective at prolonging pulmonary washout from excess surfactant.