Table 4:

Evidence from recent randomized controlled trials of macrolides

Study (year)TreatmentStudy durationNumber of patientsPatient characteristicsSignificant results and outcomes
BAT study (2013) (65)250 mg daily azithromycin v. placebo12-mo treatment
90-d run-out
83≥ × 3 exacerbations/yr ≥ 1 sputum culture with pathogens in preceding yrWith azithromycin v. placebo:
-Fewer exacerbations (0[0 – 1] v. 2[1 – 3])
-Improvement in FEV1% predicted (+1.03% v. −0.1%)
-Improved HRQL
-Well tolerated, despite increased relative risk of diarrhea
-Increased macrolide resistance: 35% in 8 patients at baseline increased to 88% in 20 patients v. 26% in 22 patients
BLESS study (2013) (66)400 mg twice daily erythromycin v. placebo48-wk treatment
4-wk washout
117≥ × 2 exacerbations/yrWith erythromycin v. placebo:
-Fewer exacerbations (76 v. 114)
-Significant reduction in 24-h sputum weight (−5.4 g v. −1.7 g reduction)
-Less decline in postbronchodilator FEV1% predicted (−1.6% v. −4.0%)
-Increased macrolide resistance: 27.7% v. 0.04%
-Well tolerated; 28.8% v. 25.9% reporting AEs
EMBRACE study (2012) (67)500 mg azithromycin 3 times per wk v. placebo6-mo treatment
6-mo follow-up
141≥ × 1 exacerbation/yrWith azithromycin v. placebo:
-62% relative reduction in exacerbation rate during treatment and 42% annually
-Annually, longer time to first exacerbation 239 (190–331) d v. 85 (52–113) d
-Well tolerated, with 59 reported AEs v. 65
-No macrolide resistance testing performed
  • Note: AE = adverse event, FEV1 = forced expiratory volume in the first second, HRQL = health-related quality of life.