Abstract
PAP is a rare disorder, characterized by accumulation of lipoproteinaceous material within alveolar spaces.The clinical course of PAP ranges from spontaneous remission to respiratory failure and whole lung lavage (WLL) currently remains the therapeutic standard.
Methods: Clinical assessment was performed by HRCT scan, BAL, lung biopsy, pulmonary function tests, serum markers (LDH,CYFRA,CEA,NSE) and GM-CSF autoantibodies.
Results: We enrolled 76 PAP patients from 1989 to 2011, divided as follows: Idiopathic PAP (n=70) of which the autoimmune nature was confirmed in 76%, Secondary PAP (n=3, 4%), Congenital PAP (n=1, 1.3%) and PAP-like disorders (n=2, 2.7%). The idiopathic PAP patients showed a prevalence of males (72.8%) over females, with a mean age at diagnosis of 40.7±13. A smoking history (current or former smokers) occurred in 68.5%, with a mean of 27pack/years. The most common presenting symptom was dyspnea, alone (27 patients) or in combination with cough (16 patients). 4 patients were completely asymptomatic. Intercurrent infections were present in 14 cases. Mean GMAbs level was 261±240 μg/mL (n.v. <3 μg/mL). From our whole series, 43 patients were submitted to WLL: 30 (43%) received 1 WLL, whereas 13 (30%) received 2 or more WLLs. Comparison of pulmonary function testing data between not lavaged and lavaged PAP patients showed a significant difference for the TLCO% pred (mean value 60.3±18.4 vs 46±13.7, p=0.011) and for the A-aO2 gradient (35±17vs 45±15.8 mmHg, p=0.043).
Conclusion: The establishment of a reference center for PAP in Italy has allowed to gather and well characterize one of the largest series of PAP patients worldwide.
Funding: AIFA (FARM7MCPK4), eRARE (EuPAPNet).
- © 2011 ERS