OBJECTIVE To observe the efficacy of recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) for pulmonary alveolar proteinosis (PAP). MATERIALS AND METHODS A total of 55 patients with PAP were screened at Shanghai Pulmonary Hospital between May 2014 and May 2018. Among these, 42 were diagnosed with idiopathic PAP, 24 were included in this study, 20 were treated for 6 months, and 17 were followed up for additional 6 months. All patients received a subcutaneous injection of 75μg/d GM-CSF qd for 1 month. The therapeutic dose was adjusted according to the changes in the lesions of chest CT. If the lesions were absorbed, subcutaneous injections of 75μg/d GM- CSF qd and 75μg/d GM-CSF qod were given for 2 and 3 months, otherwise, the dose was increased to 150μg/d GM-CSF qd and 150μg/d qod for 2 and 3 months, respectively. All cases were treated once a day in the first 3 months and once every other day in the last 3 months. The total course of treatment was 6 months. After withdrawal, the patients were followed up for another 6 months. The deadline of follow up was September 30, 2019. RESULTS Twenty patients completed the treatment and efficacy evaluation. One patient was completely cured, 16 cases improved, three cases were noneffective. After 1-month evaluation, 12 patients received an increased dose (150μg) from the second month of treatment. Seventeen patients completed the 12-month follow-up, among which fourteen improved. CT showed the lesions were slightly increased in three cases. Economic burden was the following: RMB 7324-15,190 Yuan were required for the 6-month treatment course, which is significantly lower compared to other treatment methods. CONCLUSION Subcutaneous injection of rhGM-CSF at low dose (75μg-150μg /d) is effective treatment for patients with idiopathic PAP. TRIAL REGISTRATION NCT01983657. Registered 16 April 2013.

中文翻译：

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皮下注射低剂量重组人粒细胞-巨噬细胞集落刺激因子对肺泡蛋白沉着的治疗作用。

目的观察重组人粒细胞巨噬细胞集落刺激因子（rhGM-CSF）对肺泡蛋白沉着症（PAP）的疗效。材料与方法2014年5月至2018年5月间，在上海肺科医院筛查55例PAP患者。其中42例确诊为特发性PAP，包括24例，治疗6个月，17例。随访了6个月。所有患者均接受皮下注射75μg/ d GM-CSF qd 1个月。根据胸部CT病变的变化调整治疗剂量。如果病变被吸收，则皮下注射75μg/ d GM-CSF qd和75μg/ d GM-CSF qod，持续2个月和3个月，否则剂量增加至150μg/ d GM-CSF qd和150μg/ d分别停留2个月和3个月。在最初的三个月中，所有病例每天接受一次治疗，在最后的三个月中，每隔一天接受一次治疗。总疗程为6个月。停药后，对患者再随访6个月。随访截止日期为2019年9月30日。结果20例患者完成了治疗和疗效评估。完全治愈1例，好转16例，无效3例。经过1个月的评估，从治疗的第二个月开始，有12名患者接受了增加的剂量（150μg）。17位患者完成了12个月的随访，其中14位得到了改善。CT显示3例病灶轻度增加。经济负担如下：六个月的疗程需要人民币7324-15,190元，相比其他治疗方法要低得多。结论低剂量（75μg-150μg/ d）皮下注射rhGM-CSF可有效治疗特发性PAP。试用注册号NCT01983657。2013年4月16日注册。