University of Medicine and Pharmacy Gr T Popa Iasi, Pulmonary Disease University Hospital, Department of Internal Medicine II- Pulmonary Disease, 30 Dr I Cihac Str, 700115 Iasi, Romania.
Expert Opin Biol Ther. 2010 Sep;10(9):1357-65. doi: 10.1517/14712598.2010.510507.
Pulmonary alveolar proteinosis (PAP) is a rare disease in which the abnormalities in surfactant metabolism are caused most often by impairments of GM-CSF pathway at different levels in different disease subsets (congenital, secondary, acquired/idiopathic) and for which there are only few, costly invasive therapeutic methods.
This review discusses these impairments, and their pathogenic and clinical consequences along with potential corrective therapies such as exogenous inhaled GM-CSF.
Among the PAP disease subsets, in autoimmune PAP the GM-CSF autoantibodies play a major role in disease pathogenesis and their deleterious pulmonary effects can be blocked efficaciously with inhaled GM-CSF.
In PAP correction of the abnormalities of the GM-CSF pathway represent a plausible approach demonstrated to be efficacious also in the case of inhaled GM-CSF used for autoimmune PAP.
肺泡蛋白沉积症（PAP）是一种罕见的疾病，其中表面活性剂代谢异常通常是由不同疾病亚组（先天性、继发性、获得性/特发性）中 GM-CSF 途径在不同水平的损伤引起的，并且只有少数昂贵的侵入性治疗方法。
本篇综述讨论了这些损伤及其发病机制和临床后果，以及潜在的矫正治疗方法，如外源性吸入 GM-CSF。
在 PAP 疾病亚组中，自身免疫性 PAP 中的 GM-CSF 自身抗体在疾病发病机制中起主要作用，用吸入 GM-CSF 可以有效地阻断其对肺部的有害影响。
在 PAP 中，GM-CSF 途径的异常纠正代表了一种合理的方法，在使用吸入 GM-CSF 治疗自身免疫性 PAP 的情况下也被证明是有效的。
