Hydroxychloroquine in children with interstitial (diffuse parenchymal) lung diseases

Summary Hydroxychloroquine (HCQ) is one of the drugs frequently used for the treatment of interstitial lung disease (ILD) in children (chILD). This use is off‐label and studies to analyze the effect and safety of HCQ in chILD are lacking. Therefore, a literature research on the usage of chloroquine (CQ) and HCQ in these conditions was done. Eighty‐five case reports and small series in the period from 1984 to 2013 were identified in which children with different diagnoses of ILD were treated with CQ or HCQ, sometimes in combination with other medication including steroids. A favorable response to HCQ or CQ was reported in 35 cases, whereas in the other cases the effect was negative or not clear. The dose of HCQ used was between 5 and 10 mg/kg body weight/day (bw/d). No pharmacokinetic studies have been done. The side effect profile in children seemed to be similar to that in adults. Most often gastrointestinal symptoms were reported. Three patients were found developing retinal changes during the treatment with CQ, whereas in none of the patients treated with HCQ retinal changes were reported. Based on retrospective case reports and small series likely to be reported with bias, the use of HCQ in chILD might be classified as safe. As no prospective data on efficacy and safety of HCQ in chILD are available, systematic collection is necessary. This may be achieved by web‐based registers like the European Management Platform for Childhood Interstitial Lung Diseases. Prospective and controlled investigations of HCQ in patients with chILD are mandatory. Pediatr Pulmonol. 2015; 50:410–419. © 2014 Wiley Periodicals, Inc.