Inhaled granulocyte-macrophage colony stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis - a six month phase II randomized study with 24 months of follow-up
机构：[1]Chinese Acad Med Sci & Peking Union Med Coll, Peking Union Med Coll Hosp, Dept Pulm & Crit Care Med, Beijing, Peoples R China[2]Nanjing Univ, Dept Pulm & Crit Care Med, Affiliated Drum Tower Hosp, Med Sch, Nanjing, Jiangsu, Peoples R China[3]Chinese Acad Med Sci & Peking Union Med Coll, Peking Union Med Coll Hosp, Dept Radiol, Beijing, Peoples R China[4]Capital Med Univ, Beijing Tongren Hosp, Emergency Ctr, Beijing, Peoples R China首都医科大学附属北京同仁医院首都医科大学附属同仁医院[5]Univ Alberta, Fac Med & Dent, Dept Biochem, Edmonton, AB, Canada
Background: Treatment of autoimmune pulmonary alveolar proteinosis (aPAP) by inhaled granulocyte-macrophage colony stimulating factor (GM-CSF) is considered safe and effective. Evidence of benefit from GM-CSG inhalation for mild to moderate aPAP patients is limited. Methods: In this multicenter, randomized, open-labeled clinical trial, 36 aPAP patients with mild to moderate disease severity were randomized into either the GM-CSF treatment group or control group. Inhaled GM-CSF was prescribed for 6 months, and patients received follow-up for another 18 months without treatment. Physiological features of the patients were analyzed. Results: There were 36 patients (19 in the treatment group, 17 in the control group) included. There were no significant differences in the primary endpoints as measured by the change of alveolar arterial oxygen gradient (A-aDO(2)) from the baseline values to the values obtained during treatment or during the following 18-month non-treatment observation period [control group vs. treatment group: 0.51 +/- 12.09 mmHg vs. -0.35 +/- 13.76 mmHg,p = 0.848 (3 month); 1.85 +/- 11.21 mmHg vs. 7.31 +/- 8.81 mmHg,p = 0.146 (6 months); 6.05 +/- 11.14 mmHg vs. 6.61 +/- 10.64 mmHg,p = 0.899 (24 months)]). Percentage of diffusion capacity predicted (DLCO%) and percentage of total lung capacity predicted (TLC%), however, were significantly improved in the treatment group by the end of the study (P = 0.010 and 0.027). St. George Respiratory questionnaire (SGRQ) scores were better after 6 months treatment with GM-CSF compared to the control group, and the benefits of treatment were maintained throughout the observation period. No severe side effects were observed during the study. Conclusion: Six months of inhaled GM-CSF treatment had no effect on the alveolar-arterial oxygen gradient in patients with mild to moderate pulmonary alveolar proteinosis. There were changes in some clinical or laboratory measures, but no clinically important changes were noted at the end of study. (Clinical Trial Registry: NCT02243228, Registered on September 17, 2014, https://www.clinicaltrials.gov/ct2/show/NCT02243228?term=NCT0224322 8&draw=2&rank=1)
基金：
National Key Research and Development Program of China [2016YFC0901502]; National Nature Science Foundation of ChinaNational Natural Science Foundation of China (NSFC) [81570061]; Chinese Academy of Medical Sciences (CAMS) Initiative for Innovative Medicine [2017-12 M-2-001, 2018-12 M01-003]
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外文
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中科院(CAS)分区：
出版当年[2019]版：
大类|2 区医学
小类|3 区遗传学3 区医学：研究与实验
最新[2025]版：
大类|2 区医学
小类|2 区遗传学2 区医学：研究与实验
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出版当年[2018]版：
Q2GENETICS & HEREDITYQ2MEDICINE, RESEARCH & EXPERIMENTAL
最新[2023]版：
Q2GENETICS & HEREDITYQ2MEDICINE, RESEARCH & EXPERIMENTAL
第一作者机构：[1]Chinese Acad Med Sci & Peking Union Med Coll, Peking Union Med Coll Hosp, Dept Pulm & Crit Care Med, Beijing, Peoples R China
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推荐引用方式(GB/T 7714)：
Tian Xinlun,Yang Yanli,Chen Lulu,et al.Inhaled granulocyte-macrophage colony stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis - a six month phase II randomized study with 24 months of follow-up[J].ORPHANET JOURNAL OF RARE DISEASES.2020,15(1):doi:10.1186/s13023-020-01450-4.
APA：
Tian, Xinlun,Yang, Yanli,Chen, Lulu,Sui, Xin,Xu, Wenshuai...&Xu, Kai-Feng.(2020).Inhaled granulocyte-macrophage colony stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis - a six month phase II randomized study with 24 months of follow-up.ORPHANET JOURNAL OF RARE DISEASES,15,(1)
MLA：
Tian, Xinlun,et al."Inhaled granulocyte-macrophage colony stimulating factor for mild-to-moderate autoimmune pulmonary alveolar proteinosis - a six month phase II randomized study with 24 months of follow-up".ORPHANET JOURNAL OF RARE DISEASES 15..1(2020)
