Trial reportAmerican journal of respiratory and critical care medicine2018
Azithromycin for Early Pseudomonas Infection in Cystic Fibrosis. The OPTIMIZE Randomized Trial.
Trial report in American journal of respiratory and critical care medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT02054156 (OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis), which is not on this map. Cited by 52 papers, 6 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
OPTIMIZing Treatment for Early Pseudomonas Aeruginosa Infection in Cystic Fibrosis: The OPTIMIZE Multicenter, Placebo-Controlled, Double-Blind, Randomized Trial
Who cites it
52 citing papers in PubMed, 6 syntheses or guidelines pooled it, 98 citations in OpenAlex.
- A systematic review and meta-analysis of the treatment modalities available for children afflicted from cystic fibrosis.BMC pediatrics · 2025Pooled it
- Efficacy of Long-Term Use of Azithromycin in the Management of Cystic Fibrosis in Pediatric Patients with or WithoutMedicina (Kaunas, Lithuania) · 2025Pooled it
- Macrolide antibiotics (including azithromycin) for cystic fibrosis.The Cochrane database of systematic reviews · 2024Pooled it
- Inhaled anti-pseudomonal antibiotics for long-term therapy in cystic fibrosis.The Cochrane database of systematic reviews · 2022Pooled it
- Safety of azithromycin in pediatrics: a systematic review and meta-analysis.European journal of clinical pharmacology · 2020Pooled it
- Treatments for preventing recurrence of infection with Pseudomonas aeruginosa in people with cystic fibrosis.The Cochrane database of systematic reviews · 2019Pooled it
- Trial
- Association of Intensity of Antipseudomonal Antibiotic Therapy With Risk of Treatment-Emergent Organisms in Children With Cystic Fibrosis and Newly Acquired Pseudomonas Aeruginosa.Clinical infectious diseases : an official publication of the Infectious Diseases Society of America · 2021Trial
- Intravenous versus oral antibiotics for eradication of Pseudomonas aeruginosa in cystic fibrosis (TORPEDO-CF): a randomised controlled trial.The Lancet. Respiratory medicine · 2020Trial
- Systematic development and optimization of a microfluidic formulation protocol for liposomal azithromycin.RSC pharmaceutics · 2026Article
- Specialized Pro-Resolving Lipid Mediators in Pulmonary Diseases: Molecular and Therapeutic Implications.Molecules (Basel, Switzerland) · 2025Review
- In Vitro and In Vivo Antibacterial and Antibiofilm Activity of Zinc Sulfate (ZnSOAntibiotics (Basel, Switzerland) · 2025Article
- Antibiotic treatment of bacterial lung infections in cystic fibrosis.European journal of pediatrics · 2024Review
- Statistical approaches for the integration of external controls in a cystic fibrosis clinical trial: a simulation and an application.American journal of epidemiology · 2024Article
- Therapeutic Interventions forJournal of clinical medicine · 2024Review
- Current and future therapeutic approaches of CFTR and airway dysbiosis in an era of personalized medicine.Journal of family medicine and primary care · 2024Review
- Microevolution, reinfection and highly complex genomic diversity in patients with sequential isolates of Mycobacterium abscessus.Nature communications · 2024Article
- Limited effects of azithromycin on the oropharyngeal microbiome in children with CF and early pseudomonas infection.BMC microbiology · 2023Article
- Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis.The Cochrane database of systematic reviews · 2023Review
- Diagnosis and Management of Cystic Fibrosis Exacerbations.Seminars in respiratory and critical care medicine · 2023Review
Corrections and comments
- Commented on by
- Commented on by
- Commented on by
- Erratum issued
Authors and funding
16 authors at 11 institutions in 1 country.
Funding
Abstract
rationaleNew isolation of Pseudomonas aeruginosa (Pa) is generally treated with inhaled antipseudomonal antibiotics such as tobramycin inhalation solution (TIS). A therapeutic approach that complements traditional antimicrobial therapy by reducing the risk of pulmonary exacerbation and inflammation may ultimately prolong the time to Pa recurrence.
objectivesTo test the hypothesis that the addition of azithromycin to TIS in children with cystic fibrosis and early Pa decreases the risk of pulmonary exacerbation and prolongs the time to Pa recurrence.
methodsThe OPTIMIZE (Optimizing Treatment for Early Pseudomonas aeruginosa Infection in Cystic Fibrosis) trial was a multicenter, double-blind, randomized, placebo-controlled, 18-month trial in children with CF, 6 months to 18 years of age, with early Pa. Azithromycin or placebo was given 3× weekly with standardized TIS. MEASUREMENTS AND MAIN
resultsThe primary endpoint was the time to pulmonary exacerbation requiring antibiotics and the secondary endpoint was the time to Pa recurrence, in addition to other clinical and safety outcomes. A total of 221 participants (111 placebo, 110 azithromycin) out of a planned 274 were enrolled. Enrollment was stopped early by the NHLBI because the trial had reached the prespecified interim boundary for efficacy. The risk of pulmonary exacerbation was reduced by 44% in the azithromycin group as compared with the placebo group (hazard ratio, 0.56; 95% confidence interval, 0.37-0.83; P = 0.004). Weight increased by 1.27 kg in the azithromycin group compared with the placebo group (95% confidence interval, 0.01-2.52; P = 0.046). No significant differences were seen in microbiological or other clinical or safety endpoints.
conclusionsAzithromycin was associated with a significant reduction in the risk of pulmonary exacerbation and a sustained improvement in weight, but had no impact on microbiological outcomes in children with early Pa. Clinical trial registered with clinicaltrials.gov (NCT02054156).
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.