目的:肺泡蛋白沉积症(PAP)是一种罕见的疾病,表现为肺泡巨噬细胞功能障碍和肺泡表面活性蛋白的异常积累。在全国范围内,基于人群的研究,我们调查了台湾PAP的流行病学,并发现了PAP的合并症和预后因素。
方法:来自国家健康保险研究数据库(NHIRD),我们获得了1995年至2013年间台湾所有PAP患者的全面信息.发病率,基线特征合并症,并探讨影响PAP预后的因素。
结果:2000年以后,PAP的年发病率约为每百万人口0.79(范围:0.49-1.17)人,到2013年底,患病率为每百万人口7.96人。总的来说,确定了276例PAP患者,包括177例(64%)和99例(36%)原发性和继发性PAP患者,分别。中位诊断年龄为53.8岁。首次PAP诊断后的中位生存期为9.6年,5年生存率为65.96%。20例(7%)患者在诊断后三个月内接受了全肺灌洗(WLL),与其他患者相比,其生存率明显更好。多变量Cox回归分析显示,年龄较大,辅助PAP,恶性肿瘤与较差的生存率有关,而WLL在诊断后3个月内可能会大大提高生存率。
结论:我们证明了台湾PAP的流行病学,显示几个不良预后因素和WLL的潜在有效性。基于注册的进一步前瞻性研究有必要改善PAP的诊断和治疗。
OBJECTIVE: Pulmonary alveolar proteinosis (PAP) is rare disease manifested as alveolar macrophage dysfunction and abnormal accumulation of surfactant protein in the alveoli. In this nationwide, population-based study, we investigated the epidemiology of PAP in Taiwan, and discovered the comorbidities and prognostic factors of PAP.
METHODS: From the National Health Insurance Research Database (NHIRD), we obtained comprehensive information about all patients of PAP in Taiwan between 1995 and 2013. The incidence, baseline characteristics comorbidities, and prognostic factors of PAP were investigated.
RESULTS: The annual incidence rate of PAP was around 0.79 (range: 0.49-1.17) patients per million people after 2000, and the prevalence rate was 7.96 patients per million people by the end of 2013. In total, 276 patients of PAP were identified, including 177 (64%) and 99 (36%) patients with primary and secondary PAP, respectively. The median age of diagnosis was 53.8 years. The median survival was 9.6 years after the initial PAP diagnosis, and the 5-year survival rate was 65.96%. Twenty (7%) patients received whole lung lavage (WLL) within three months after the diagnosis had significantly better survival compared to the others. Multivariable Cox regression analyses showed that elder age, secondary PAP, and malignancy were associated with poorer survival, while WLL within 3 months of diagnosis might greatly improve the survival.
CONCLUSIONS: We demonstrated the epidemiology of PAP in Taiwan, showing several poor prognostic factors and the potential effectiveness of WLL. Further prospective studies based on registry are warranted to improve the diagnosis and treatment of PAP.