Diffuse parenchymal lung disease caused by surfactant deficiency: dramatic improvement by azithromycin

Abstract

Pulmonary surfactant deficiency caused by mutations in ABCA3 (ATP-binding cassette transporter of the A subfamily, member 3) gene results in diffuse parenchymal lung disease (DPLD) in children. So far, systemic steroids are the main treatment, with however limited efficacy. We report the case of a young boy showing a dramatic long-term improvement of respiratory disease by low-dose azithromycin (AZM) with no side effect after 6 years of treatment. Cellular and molecular studies are ongoing to progress in the understanding of the mechanisms involved. On behalf of the National Reference Center for rare lung diseases in France (Respirare, http://www.respirare.fr), clinical studies on AZM in various forms of DPLD in children have been initiated and should provide information on the types of paediatric DPLD that may benefit from this treatment.

Figure 1

Chest CT of the patient at the age of 6 years showed bilateral ground glass opacities, interlobular septal thickening and some pulmonary cysts (A). CT at the age of 12 years (after 6 years of azithromycin) indicated partial clearing of ground glass attenuation without appearance of new cysts or alveolar condensation (B).