Azithromycin for Poorly Controlled Asthma in Children A Randomized Controlled Trial

BACKGROUND: Azithromycin has immunomodulatory actions, and its beneficial effects have been demonstrated in asthmatic adults. Data on children are limited. RESEARCH QUESTION: Does the addition of oral azithromycin to standard therapy in children with poorly controlled asthma improve asthma control compared with standard treatment alone? STUDY DESIGN AND METHODS: This open-label randomized controlled trial included children (5-15 years of age) with poorly controlled asthma defined by Asthma Control Test (ACT) and Childhood Asthma Control Test (CACT) score of <= 19. They were randomized to receive azithromycin (10 mg/kg) three times weekly for 3 months along with standard treatment or standard treatment alone. The primary outcome was the ACT and CACT scores at 3 months. Secondary outcomes were asthma control according to Global Initiative for Asthma (GINA) guidelines, the number of exacerbations, change in spirometry parameters, change in fractional exhaled nitric oxide (F-ENO) level, positive throat swab results, and side effects. RESULTS: The trial included 120 children (89 boys; 60 in each group). The mean +/- SD age was 9.9 +/- 3 years. The baseline parameters were similar between the groups. Mean +/- SD ACT and CACT scores (available for 115 children) at 3 months of intervention were 21.71 +/- 2.17 vs 18.33 +/- 2.19 (P < .001) in the azithromycin and control groups, respectively. The numbers of children with well-controlled asthma according to GINA guidelines were 41 of 56 vs 10 of 56 in the azithromycin and control groups, respectively (P < .001). The median number of exacerbations requiring emergency visit and steroid use were fewer in the azithromycin group: 0 (interquartile range [IQR], 3) vs 1 [IQR, 6]; P < .001). No difference was found in F-ENO level, spirometry parameters, positive throat swab results, and adverse effects between the groups. INTERPRETATION: The use of azithromycin in children with poorly controlled asthma resulted in improved asthma control and reduced exacerbations.