title: [Effectiveness and safety of inhaled granulocyte-macrophage colony-stimulating factor therapy in idiopathic pulmonary alveolar proteinosis].
abstract: OBJECTIVE
  To investigate the effectiveness and safety of inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) therapy in idiopathic pulmonary alveolar proteinosis (PAP).
  
  
  METHODS
  Ten PAP patients were enrolled, who were hospitalized in the Affiliated Drum Tower Hospital of Nanjing University Medical School from January 2012 to January 2014.All the patients were treated with inhaled GM-CSF therapy, high-dose therapy for 12 weeks, GM-CSF 150 µg twice a day on days 1-7, none for days 8-14, 6 cycles, low-dose therapy for 12 weeks, GM-CSF 150 µg inhaled once a day on days 1-7, none for days 8-14, 6 cycles, and followed-up for one year. Physiologic, serologic and radiologic features of the patients were analyzed.
  
  
  RESULTS
  After inhaled therapy, clinical symptoms, oxygenation indexes, pulmonary function of nine patients were improved, and high resolution CT (HRCT) showed ground-glass lesions reduced. After inhaled therapy for 6 months, the average level of arterial oxygen partial pressure (PaO₂) was significantly higher than that before therapy ((75.5 ± 7.0) vs (63.6 ± 8.9) mmHg (1 mmHg=0.133 kPa)), while alveolar-arterial oxygen pressure difference (P(A-a)O₂) was lower than before ((25.1 ± 7.1) vs (41.2 ± 13.5) mmHg) (both P<0.01). Similarly, vital capacity (VC)% predicted, forced vital capacity (FVC)% predicted and carbon monoxide diffusing capacity (DLCO)% predicted all improved after therapy ((77.3 ± 16.6)% vs (63.3 ± 16.6)%), (79.5 ± 17.6)% vs (64.9 ± 17.1)%), (69.4 ± 23.0)% vs (50.0 ± 19.9)%) (all P<0.01). The score of HRCT reduced after therapy for six months (P<0.05). While the levels of serum lactate dehydrogenase (LDH), carcinoembryonic antigen (CEA), CYFRA211 were unchanged. No serious adverse events occurred during observation.
  
  
  CONCLUSIONS
  Inhaled GM-CSF therapy is safe and effective.
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China.
authors.forename: Jingjing
authors.initials: J
authors.lastname: Ding
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China.
authors.forename: Yonglong
authors.initials: Y
authors.lastname: Xiao
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China.
authors.forename: Jinghong
authors.initials: J
authors.lastname: Dai
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China.
authors.forename: Liyun
authors.initials: L
authors.lastname: Miao
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China.
authors.forename: Yuying
authors.initials: Y
authors.lastname: Qiu
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China.
authors.forename: Lulu
authors.initials: L
authors.lastname: Chen
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China.
authors.forename: Hanyi
authors.initials: H
authors.lastname: Jiang
authors.affiliation: Department of Respiratory Medicine, the Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing 210008, China; Email: caihourong2013@163.com.
authors.forename: Hourong
authors.initials: H
authors.lastname: Cai
mesh_terms: D000280:Administration, Inhalation; D002272:Carcinoembryonic Antigen; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D006801:Humans; D008168:Lung; D011649:Pulmonary Alveolar Proteinosis; D014797:Vital Capacity
chemical_list: D002272:Carcinoembryonic Antigen; D016178:Granulocyte-Macrophage Colony-Stimulating Factor