Table 1—

Summary of studies examining the clinical effectiveness of macrolide therapy in cystic fibrosis

First author [ref.]Study designAzithromycin regimenAdults/ children nLength of studyBenefit?Adverse effects (treatment arm)
Southern 23Meta-analysisAzithromycin296≤6 months+↑FEV1No serious events
Tramper-Stranders 24Prospective, open-label5–10 mg·kg−1 o.d.0/1003 (0.9–7) yrs↑FEV1 in year 1 and ↓FEV1 in years 2 and 3Macrolide-resistant Staphylococcus aureus (100%)
Clement 25Multicentric, randomised, double-blind, placebo-controlled250–500 mg three times weekly82#12 monthsFEV1 unchanged; ↓exacerbation rate/number oral antibioticsNo difference in minor adverse events between groups
Hansen 26Observational cohort250 mg o.d.45/012 months↑FEV1+FVC; ↑weight; ↓mucoid Pseudomonas aeruginosaTinnitus (n = 2)
Saiman 27Multicentric, randomised, double-blind, placebo-controlled250 or 500 mg three times weekly18524 weeks↑FEV1; ↓exacerbation rate; ↑weight; ↑adverse events↑Nausea (17% more); ↑diarrhoea (15% more); ↑wheezing (13% more)
Pirzada 28Retrospective250 mg o.d.20/021 months↑FEV1+FVC; ↓i.v. antibiotics; ↑weightNot specifically mentioned; one subject withdrew because of abdominal pain
Equi 29Randomised, double-blind, placebo-controlled, crossover250–500 mg o.d.0/41Two 6-month blocks↑FEV1; ↓oral antibioticsSubjects reported no significant side-effects (LFT results abnormal (n = 1))
Wolter 30Randomised, double-blind, placebo-controlled250 mg o.d.60/03 months↓FEV1 decline rate; ↑QoL score; ↓ i.v. antibioticsNo difference between groups (urticaria (n = 1), neutropenia (n = 1), rash (n = 1))
  • FEV1: forced expiratory volume in one second; FVC: forced vital capacity; LFT: liver function test; QoL: quality of life. #: mostly children; : all chronically infected with Pseudomonas; +: follow-up.