LAUSR

for the treatment of childhood interstitial lung disease. However, to our knowledge, there is only one reported case of ABCA3 deficiency that significantly improved after AZM treatment. It is suggested that AZM is effective in treating chronic lung disease because it has various mechanisms of action, such as regulation of the production of inflammatory mediators and control of mucus hypersecretion. A recent study also showed that AZM promotes formation of lamellar bodies in bronchial epithelial cells. This action is probably due to drug-induced phospholipidosis, a side effect; however, this side effect could be beneficial in ABCA3 deficiency. The types of cases for which AZM will be effective and the clinical course of such treated cases remain unclear. In this case, although prednisolone and hydroxychloroquine were ineffective for first 4 months, the patient’s clinical symptoms dramatically improved and KL-6 concentration declined after 1 month of AZM treatment. Considering the change in KL-6 concentration and the patient’s clinical symptoms, we believe that AZM was significantly effective in this case. Moreover, no complications or side effects were observed. The early initiation of AZM treatment is important for ABCA3 deficiency because of its significant efficacy, albeit in some cases, and its long-term safety. Administration of AZM and hydroxychloroquine was approved by the ethical committee, and informed consent was obtained from the patient’s parents. Written informed consent for publication of her clinical details was obtained from the patients’ parents.