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Macrolide antibiotics for cystic fibrosis
K W Southern1, P M Barker, A Solis
1Child Health, University of Liverpool, Alder Hey Children's Hospital, Eaton Road, Liverpool, Merseyside, UK, L12 2AP.
Background:
Chronic severe infection with Pseudomonas aeruginosa, affects many people with cystic fibrosis (CF). There is evidence from the laboratory and from other disease processes that macrolide antibiotics, whilst not directly active against Pseudomonas aeruginosa, may have indirect actions against this organism.
Objectives:
We aimed to test the hypotheses that, in people with CF, macrolide antibiotics:(1) improve clinical status compared to placebo or another antibiotic;(2) do not have unacceptable adverse effects. If benefit was demonstrated, we aimed to assess the optimal type, dose and duration of macrolide therapy.
Search Strategy:
We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group trials register comprising references identified from comprehensive electronic database searches, handsearching relevant journals and abstract books of conference proceedings. We contacted principal investigators known to work in the field, previous authors and pharmaceutical companies who manufacture macrolide antibiotics for unpublished or follow-up data (December 2003). Most recent search of the Group's register: January 2004
Selection Criteria:
Published or unpublished randomised controlled trials of macrolide antibiotics compared to placebo, another class of antibiotic or another macrolide antibiotic. Studies comparing regimens of the same macrolide antibiotic at different doses will also be included.
Data Collection And Analysis:
Two reviewers independently extracted data and assessed study quality. Three groups were contacted for missing data and we hope to include these in future reviews.
Main Results:
Searches identified 14 studies, four were included in this review (296 participants). Two studies enrolled adults, one children (a significant number of whom were not colonised with Pseudomonas aeruginosa) and one both adults and children. All the clinical studies reported small but significant improvements in respiratory function with azithromycin versus placebo. Meta-analysis at the one-month and six-month time points demonstrates a significant benefit with respect to relative change in FEV1 (at six months, for n = 104, azithromycin and n = 114, placebo; WMD 5.82% (95% CI 2.45 to 9.20)). The largest study reported a significant increase in mild adverse events (nausea, diarrhoea and wheezing).
Reviewers' Conclusions:
There is clear evidence from these studies of a small but significant improvement in respiratory function following treatment with azithromycin. The largest study employed a three times a week dose and, in this study, treatment with azithromycin was associated with a significant increase in mild adverse events. Further studies are needed to clarify the precise role of azithromycin in the treatment of CF lung disease.
Insights
Macrolide antibiotics like azithromycin offer small but significant improvements in respiratory function for cystic fibrosis patients. However, some mild adverse events were noted, warranting further research into optimal use.
Area of Science:
- Pulmonology
- Infectious Diseases
- Pharmacology
Background:
- Chronic Pseudomonas aeruginosa infection is a major concern for cystic fibrosis (CF) patients.
- Macrolide antibiotics may offer indirect benefits against Pseudomonas aeruginosa in CF, despite lacking direct activity.
Purpose of the Study:
- To evaluate the efficacy of macrolide antibiotics in improving clinical status in CF patients compared to placebo or other antibiotics.
- To assess the safety and adverse effects of macrolide antibiotic use in CF.
- To determine optimal macrolide therapy parameters if benefits are observed.
Main Methods:
- Conducted a systematic review of published and unpublished randomized controlled trials.
- Searched multiple databases and contacted researchers and pharmaceutical companies for data.
- Included studies comparing macrolides to placebo, other antibiotics, or different macrolide regimens.
Main Results:
- Four studies involving 296 participants were included.
- Azithromycin demonstrated small but significant improvements in respiratory function (FEV1) compared to placebo at six months.
- The largest study reported an increase in mild adverse events such as nausea and diarrhea.
Conclusions:
- Azithromycin shows a clear, albeit small, benefit in improving respiratory function in CF patients.
- Mild adverse events were associated with azithromycin use, particularly at a thrice-weekly dosage.
- Further research is required to define the exact role and optimal use of azithromycin in managing CF lung disease.
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