Therapeutic effect of subcutaneous injection of low dose recombinant human granulocyte-macrophage colony-stimulating factor on pulmonary alveolar proteinosis
Therapeutic effect of subcutaneous injection of low dose recombinant human granulocyte-macrophage colony-stimulating factor on pulmonary alveolar proteinosis
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小剂量重组人粒细胞-巨噬细胞集落刺激因子皮下注射治疗肺泡蛋白沉积症的疗效观察
DOI：
10.1186/s12931-019-1261-1
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发表时间：
2020-01-02
影响因子：
5.8
通讯作者：
Li, Huiping
中科院分区：
文献类型：
--
作者：
Zhang, Fen;Weng, Dong;Li, Huiping
目的观察重组人粒细胞-巨噬细胞集落刺激因子（rhGM-CSF）治疗肺泡蛋白沉积症（PAP）的疗效。其中42例诊断为特发性PAP，24例纳入本研究，20例治疗6个月，17例随访6个月。所有患者均接受GM-CSF 75μg/d皮下注射，qd，疗程1个月。根据胸部CT病灶的变化调整治疗剂量。若病灶吸收，皮下注射GM-CSF 75μg/d，qd和75μg/d，qod，连用2个月和3个月;否则，剂量分别增加至GM-CSF 150μg/d，qd和150μg/d，qod，连用2个月和3个月。前3个月每日1次，后3个月隔日1次。总疗程6个月。停药后，患者再随访6个月。随访截止日期为2019年9月30日。痊愈1例，好转16例，无效3例。1个月评估后，12例患者从治疗第2个月开始接受增加剂量（150μg）。17例患者完成了12个月的随访，其中14例改善。3例CT显示病灶轻度增大。结论小剂量rhGM-CSF（75μg ~ 150μg /d）皮下注射治疗特发性PAP是一种有效的治疗方法。2013年4月16日注册。
ObjectiveTo observe the efficacy of recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) for pulmonary alveolar proteinosis (PAP).Materials and methodsA 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.ResultsTwenty 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.ConclusionSubcutaneous injection of rhGM-CSF at low dose (75μg-150μg /d) is effective treatment for patients with idiopathic PAP.Trial registrationNCT01983657. Registered 16 April 2013.