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Taking a low dose of the antibiotic azithromycin for six months reduces symptoms for patients with the chronic lung condition primary ciliary dyskinesia (PCD), according to research presented at the 2019 European Respiratory Society (ERS) International Congress.
PCD is a rare inherited disease that affects people from birth. It causes coughing, a build-up of mucus in the lungs and frequent chest and ear infections that can lead to permanent lung damage and hearing loss.
The new findings come from a ‘gold-standard’ randomised controlled clinical trial comparing the therapy to a placebo in patients across Europe. It is the first trial of its kind to demonstrate an effective therapy for PCD.
The study was presented by Dr Helene Kobbernagel from the Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine, Copenhagen University Hospital – Rigshospitalet, Denmark. She said: “Because it is a rare disease, there is a lack of good evidence on how to treat children and adults to relieve symptoms and prevent longer-term damage.”
Dr Kobbernagel and her colleagues wanted to see if they could use azithromycin as a form of ‘maintenance therapy’ to keep infections at bay over an extended period, hopefully improving symptoms and reducing infections in PCD patients.
The study included 90 patients, ranging in age from seven- to 50-years-old, who were being treated at six hospitals across Europe. Forty-nine of the patients were randomly assigned to take the antibiotic for six months, while the other 41 took a placebo.
All patients were checked for symptoms, the presence of infection-causing bacteria in their sputum, lung function, hearing and quality-of-life.
Patients taking azithromycin suffered an average (mean) 0.63 episodes of symptoms that were serious enough to require treatment during the study. Among those taking the placebo, patients suffered an average of 1.37 such episodes. This equates to a 50 per cent reduction in episodes in patients taking the treatment. People taking the antibiotic also had fewer infection-causing bacteria in their sputum samples, but they were more likely to suffer with mild diarrhoea.
Dr Kobbernagel said: “Our results show that azithromycin is safe for patients with PCD and that it could offer an effective maintenance therapy, reducing ill-health and helping children and adults get on with their daily lives.”
Researchers did not find any measurable difference in longer-term measures such as lung function and hearing, but they say a longer study might be needed to see whether the treatment has any effect on these.
Dr Kobbernagel added: “We need to know if it’s safe for patients to continue taking the drug for longer than six months and whether it can prevent irreversible lung damage, but this trial is an important first step.”