Not much is known on the clinical spectrum, course and treatment options in patients with molecularly defined, congenital PAP due to mutations in the GM-CSF alpha chain. Also the role of long term WLL, which are be very demanding due to the small size of the airways, how to measure clinical response to lavage therapy and the relevance of glucocorticoid therapy have not been reported. Here we present the successful management of a child with a severe congenital PAP caused by the homozygous p.Ser25X mutation in exon 3 of the CSF2RA gene and its follow up for more than a decade. These data may be helpful for future treatment of infants and children with this rare condition.