Jouneau Stéphane, Chauvin Pierre, Lederlin Mathieu, Painvin Benoît, Kerjouan Mallorie
Service de Pneumologie, Centre de Référence Constitutif pour les Maladies Rares Pulmonaires, CHU de Rennes, IRSET UMR 1085, Université de Rennes, 2 rue Henri Le Guilloux, 35033, Rennes, France.
IRSET UMR 1085, Université de Rennes, Rennes, France.
Drugs. 2025 Aug 27. doi: 10.1007/s40265-025-02228-3.
Pulmonary alveolar proteinosis is suspected when a "crazy paving" pattern is observed on a chest CT scan. This diagnosis is confirmed by the presence of eosinophilic extracellular material that shows positive staining with Periodic Acid Schiff on bronchoalveolar lavage samples. The autoimmune form of pulmonary alveolar proteinosis is confirmed by detecting anti-granulocyte-macrophage colony-stimulating factor antibodies in the patient's serum. The historical first-line treatment for autoimmune pulmonary alveolar proteinosis is whole lung lavage, which should only be performed in expert centers. It remains the preferred treatment for patients experiencing respiratory failure, especially at the time of diagnosis. Inhaled granulocyte-macrophage colony-stimulating factor supplementation with molgramostim or sargramostim is now considered a first-line treatment in the international guidelines for autoimmune pulmonary alveolar proteinosis, following the positive results of recent randomized placebo-controlled studies. Rituximab and plasmapheresis can be prescribed as third- and fourth-line treatments, respectively. Lung transplantation may be considered for eligible patients experiencing terminal respiratory failure. A deeper understanding of the pathogenesis of autoimmune pulmonary alveolar proteinosis has opened up new therapeutic avenues, such as the use of PPARγ agonists or statins.
当胸部CT扫描观察到“铺路石征”时，怀疑患有肺泡蛋白沉积症。支气管肺泡灌洗样本中存在嗜酸性细胞外物质，且对过碘酸希夫染色呈阳性，可确诊该疾病。通过检测患者血清中的抗粒细胞巨噬细胞集落刺激因子抗体，可确诊自身免疫性肺泡蛋白沉积症。自身免疫性肺泡蛋白沉积症的传统一线治疗方法是全肺灌洗，且仅应在专业中心进行。对于出现呼吸衰竭的患者，尤其是在诊断时，全肺灌洗仍是首选治疗方法。近期随机安慰剂对照研究取得了阳性结果，吸入用粒细胞巨噬细胞集落刺激因子补充剂（如莫拉司亭或沙格司亭）现在被国际指南视为自身免疫性肺泡蛋白沉积症的一线治疗方法。利妥昔单抗和血浆置换可分别作为三线和四线治疗方法。对于终末期呼吸衰竭的合格患者，可考虑进行肺移植。对自身免疫性肺泡蛋白沉积症发病机制的更深入了解开辟了新的治疗途径，如使用PPARγ激动剂或他汀类药物。
