精准医疗下的个体化治疗策略-肺泡蛋白沉积症治疗进展

Individualized treatment strategies under precision medical treatment-progress in the treatment of pulmonary alveolar proteinosis

肺泡蛋白沉积症是一种罕见的临床综合征,以肺泡和细支气管腔内过碘酸雪夫染色阳性的富磷脂蛋白物质蓄积为特征,导致肺通气功能障碍。主要分为3型:自身免疫性、先天性、继发性。目前全肺灌洗术仍然是临床主要的治疗方法,IgG型抗粒细胞-单核细胞集落刺激因子(GM-CSF)替代治疗、利妥昔单抗、血浆置换、肺移植等是新兴的治疗方法,但大多数研究仅限于病例报道,通常样本数量有限,证据水平不足,具体的治疗应按照不同的病因分型,选择不同的治疗方案,进行个体化治疗。

Pulmonary alveolar proteinosis is a rare clinical syndrome,which was first reported by Rosen in 1958.It is characterized by the accumulation of phospholipid-rich proteins(PAP)that are positive forpositivperiodic acid-Schiffstaining in the alveolar and bronchial lumens,leading to pulmonary ventilatory dysfunction.It was divided into three types:autoimmune PAP,congenital PAP and secondary PAP.Wholelung lavages is still the cornerstone of treatment,and GMCSF therapy,rituximab,plasma exchange and lung transplantation are emerging treatments.Most studies are in case series,often with limited patient numbers,so the level of evidence is low.Specific treatment should be classified according to different etiological factors,different treatment schemes should be chosen,and individual treatment should be carried out.