Cystic fibrosis is a severe genetic disorder-the most commonly recessively inherited disorder in white populations. Treatment is limited, and aimed at counteracting chronic respiratory infection. The antibiotic azithromycin has anti-inflammatory properties potentially beneficial in cystic fibrosis. Mark Rosenthal from the Royal Brompton Hospital, London, UK, and colleagues investigated the effect of azithromycin on cystic fibrosis after encouraging results from previous pilot studies.
41 children with cystic fibrosis, aged 8-18 years took part in the study. They were randomly allocated azithromycin or placebo for six months, and then switched to either treatment or placebo for a further six months after a two-month 'washout' period. Differences in forced expiratory volume in one second's exhalation (FEV1), measurement of immune response, and quality-of-life assessments were compared between azithromycin and placebo.
Children given azithromycin had a modest improvement in FEV1 of around 5%; however there was wide variation, with 13 children improving and 5 deteriorating by 13%. There was no difference in quality-of-life assessment between azithromycin and placebo; the drug was well tolerated with few reported side effects.
Mark Rosenthal Comments: "A 4-6 month trial of azithromycin is safe, comparatively inexpensive, and could greatly benefit the lung function of patients who do not respond adequately to conventional treatment for cystic fibrosis. The mechanism of benefit remains unknown."
Contact: Dr Mark Rosenthal, Royal Brompton Hospital,
Sydney Street,
London, SW3 6NP, UK;
T)44-207-352-8121;
F)44-207-351-8473;
E)M.Rosenthal@rbh.nthames.nhs.uk
Journal
The Lancet