Better approach for autoimmune pulmonary alveolar proteinosis treatment: inhaled or subcutaneous granulocyte-macrophage colony-stimulating factor: a meta-analyses

Peng Chen,Gaohong Sheng,Yanqiu Wei,Xiaolei Cao,Hui-Lan Zhang,Jiaojiao Chu

doi:10.1186/s12931-018-0862-4

Abstract

BackgroundAutoimmune pulmonary alveolar proteinosis (aPAP) is a rare pulmonary disease caused by functional deficiency of granulocyte-macrophage colony-stimulating factor (GM-CSF). GM-CSF therapy in aPAP has been reported effective in some studies. This meta-analyses aimed to evaluate whether GM-CSF therapy, including inhaled and subcutaneous GM-CSF have therapeutic effect in aPAP patients.MethodsWe analyzed 10 studies searched from PubMed, EmBase, Web of Science, Wiley Online Library and Cochrane Collaboration databases to evaluate the pooled effects of GM-CSF treatment in aPAP patients.ResultsTen observational studies involving 115 aPAP patients were included. The pooled analyses of response rate (81%, p < 0.001), relapse rate (22%, p = 0.009), PaO2 (13.76 mmHg, p < 0.001) and P(A-a)O2 (19.44 mmHg, p < 0.001) showed that GM-CSF treatment was effective on aPAP patients. Further analyses showed that inhaled GM-CSF treatment was more effective than subcutaneous GM-CSF therapy, including a higher response rate (89% vs. 71%, p = 0.023), more improvements in PaO2 (21.02 mmHg vs. 8.28 mmHg, p < 0.001) and P(A-a)O2 (19.63 mmHg vs. 9.15 mmHg, p < 0.001).ConclusionsAs two routes of exogenous GM-CSF treatment, inhaled and subcutaneous were both proven to have effect on aPAP patients. Furthermore, inhaled GM-CSF therapy showed a higher response rate, more improvements on PaO2 and P(A-a)O2 than subcutaneous GM-CSF treatment in aPAP patients, suggesting inhaled GM-CSF therapy could have more benefits on aPAP patients. Therefore, GM-CSF therapy, especially inhaled GM-CSF, might be a promising therapeutic option in treating aPAP.

Highlights

Autoimmune pulmonary alveolar proteinosis is a rare pulmonary disease caused by functional deficiency of granulocyte-macrophage colony-stimulating factor (GM-CSF)
Response rate was significantly higher in the high initial dose. This meta-analyses including 10 studies enrolling 115 patients aimed to evaluate whether GM-CSF therapy including inhaled and subcutaneous routes were effective on Autoimmune pulmonary alveolar proteinosis (aPAP) patients
Pooled analyses of available studies suggests that treatment of GM-CSF were effective on aPAP patients with response rate (81%)

Summary

Introduction

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare pulmonary disease caused by functional deficiency of granulocyte-macrophage colony-stimulating factor (GM-CSF). Approximate 90% PAP cases were autoimmune pulmonary alveolar proteinosis (aPAP) characterized by elevated levels of antibodies against granulocyte-macrophage colony-stimulating factor (GM-CSF) in blood and alveoli, and the antibodies could not be detected in health controls [6,7,8,9]. Some studies reported that inhaled or subcutaneous GM-CSF administration was effective in aPAP patients. It is debatable whether either or both routes of GM-CSF therapy is effective, and further, which route could achieve better effect

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