重组人粒细胞-巨噬细胞集落刺激因子雾化吸入治疗自身免疫性肺泡蛋白沉积症的专家共识(2022年版)

Consensus statement on inhaled recombinant human granulocyte-macrophage colony-stimulating factor for autoimmune pulmonary alveolar proteinosis(2022)

自身免疫性肺泡蛋白沉积症(autoimmune pulmonary alveolar proteinosis,aPAP)与机体内产生抗粒细胞-巨噬细胞集落刺激因子(granulocyte-macrophage colony-stimulating factor,GM-CSF)抗体有关,是肺泡蛋白沉积症最常见的类型,约占90%。重组人GM-CSF雾化吸入可有效治疗aPAP,但尚未获得我国批准,属于超适应证和超给药途径给药。肺泡蛋白沉积症共识专家组采用推荐分级的评估、制定和评价(grading of recommendation assessment,development and evaluation,GRADE)方法系统评估国内外专家应用GM-CSF雾化吸入治疗aPAP的经验,内容包括GM-CSF雾化吸入治疗aPAP的适用人群、GM-CSF雾化吸入治疗的给药方案、妊娠和哺乳期患者用药、药物不良反应和使用GM-CSF雾化吸入治疗的注意事项,并给出循证推荐建议。本专家共识有助于规范我国GM-CSF雾化吸入药治疗的临床应用,促进aPAP相关基础与临床研究。

Autoimmune pulmonary alveolar proteinosis(aPAP)is the most common(around 90%)type of pulmonary alveolar proteinosis,characterized by the presence of granulocyte-macrophage colony-stimulating factor(GM-CSF)autoantibody.Accumulating evidence has demonstrated the effectiveness of inhaled recombinant human GM-CSF for aPAP.Such a therapy has not been approved in China yet and has been classified off-label and unusual route of administration.Our expert consensus group evaluated the current knowledge with the methods of grading of recommendation assessment,development and evaluation(GRADE).The current evidence-based consensus statement covers the suitable population for inhaled GM-CSF therapy,details of prescription,usage during pregnancy and lactation,adverse effects and other suggestions in clinical usage.This consensus statement provides a clinical practice guideline of inhaled GM-CSF for aPAP in China and hopefully will accelerate basic and clinical research of aPAP.