Efficacy of Long-Term Use of Azithromycin in the Management of Cystic Fibrosis in Pediatric Patients with or Without Pseudomonas aeruginosa: A Systematic Review and Meta-Analysis Article.

PMID 40282944 | PMCID PMC12029021 | DOI 10.3390/medicina61040653 · Medicina (Kaunas, Lithuania) · 2025

Background and Objectives: In the present systematic review and meta-analysis, we aimed to discover the overall efficacy of azithromycin in children with cystic fibrosis (CF) and with or without Pseudomonas aeruginosa infection, specifically regarding its effect on respiratory parameters such as forced expiratory volume in 1 s (FEV1) and forced vital capacity (FVC) in addition to its effect on exacerbations and the need to use additional antibiotics. Materials and Method: We conducted this systematic review and meta-analysis by searching for all eligible articles on PubMed, Web of Science, and Scopus published between inception and September 2024. We used the following search strategy for our searching process: "Cystic fibrosis" AND "Azithromycin" and "Children" OR "Pediatric" OR "Infant". We conducted the meta-analysis by pooling the mean difference (MD) and comparing the continuous variables and odds ratio (OR) for dichotomous variables at 95% confidence intervals (CI), at a p-value of 0.05. Results: Azithromycin was observed to be associated with increased FEV1 compared with the control, showing an MD of 1.91 (95% CI: 1.09, 2.74, p < 0.00001) and non-significant heterogeneity. However, no significant difference was observed between azithromycin and control groups regarding FVC with MD = 0.62 (95% CI: -0.01, 1.25, p = 0.06). Compared with the control group, azithromycin was significantly associated with lower risk and a lower number of exacerbations, with OR = 0.48 (95% CI: 0.34, 0.67, p < 0.0001) and MD = -0.82 (95% CI: -1.32, -0.33, p = 0.001), respectively, with non-significant heterogeneity. Regarding the need for new antibiotic usage, azithromycin showed a significantly lower need, with OR = 0.35 (95% CI: 0.13, 0.94, p = 0.04), I2 = 75%, p = 0.02. No significant difference was observed between both groups regarding hospitalization rate, with OR = 0.88 (95% CI: 0.55, 1.4, p = 0.59). Conclusions: This systematic review and meta-analysis showed the efficacy of azithromycin in pediatric patients with CF, as it improved lung function by increasing FEV1, reduced exacerbations of CF, which is the most common symptom of CF that leads to mortality, and reduced the number of antibiotics that needed to be administered to patients with CF, which reduces the risk of antibiotic resistance. Therefore, the long-term use of azithromycin is recommended for pediatric patients with CF as part of their treatment regimen.