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Azithromycin for Early Pseudomonas Infection in Cystic Fibrosis. The OPTIMIZE Randomized Trial

Nicole Mayer-Hamblett1,2,3, George Retsch-Bogart4, Margaret Kloster3

  • 11 Department of Pediatrics and.

American Journal of Respiratory and Critical Care Medicine
|June 12, 2018
PubMed
Abstract

Insights

Adding azithromycin to tobramycin inhalation solution (TIS) significantly reduced pulmonary exacerbations in children with cystic fibrosis and early Pseudomonas aeruginosa (Pa) infection. This combination therapy also improved weight gain but did not affect bacterial recurrence.

Area of Science:

  • Pediatric Pulmonology
  • Infectious Diseases
  • Clinical Trials

Background:

  • Pseudomonas aeruginosa (Pa) infection is a significant concern in cystic fibrosis (CF) management.
  • Early Pa infection in CF is typically treated with inhaled antipseudomonal antibiotics like tobramycin inhalation solution (TIS).
  • Complementary therapies reducing pulmonary exacerbations and inflammation may prolong time to Pa recurrence.

Purpose of the Study:

  • To evaluate if adding azithromycin to TIS reduces pulmonary exacerbations and delays Pa recurrence in children with CF and early Pa.
  • To assess the impact of this combination therapy on clinical and safety outcomes.

Main Methods:

  • The OPTIMIZE trial was an 18-month, multicenter, double-blind, randomized, placebo-controlled study.
  • Participants (6 months to 18 years) with early Pa received azithromycin or placebo three times weekly alongside standardized TIS.
  • Primary endpoint: time to pulmonary exacerbation; secondary endpoint: time to Pa recurrence.

Main Results:

  • Enrollment was stopped early due to demonstrated efficacy.
  • Azithromycin significantly reduced the risk of pulmonary exacerbation by 44% (HR, 0.56; P=0.004).
  • A significant increase in weight was observed in the azithromycin group (1.27 kg, P=0.046), with no impact on microbiological outcomes.

Conclusions:

  • Azithromycin combined with TIS significantly reduces pulmonary exacerbations in children with CF and early Pa.
  • This regimen also leads to sustained weight improvement.
  • Azithromycin did not influence microbiological outcomes or time to Pa recurrence in this population.
Keywords:
Pseudomonas aeruginosaclinical trialeradicationpulmonary exacerbation

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