Long-term azithromycin decreases exacerbations in primary ciliary dyskinesia

The BESTCILIA trial presented strong evidence that a low dose of azithromycin over a period of 6 months can help prevent exacerbation in primary ciliary dyskinesia (PCD) and reduce pathogenic bacteria in the sputum.

PCD is a rare condition. The signs of this congenital disease start in neonates or young children, and it progresses throughout adulthood. Symptoms include chronic rhinosinusitis, recurrent otitis media, conductive hearing impairment, chronic productive cough, as well as infection and inflammation of the lower respiratory tract [1]. Currently, data on PCD is still scarce.

Dr Helene Kobbernagel (Rigshospitalet Copenhagen, Denmark) presented the results of the randomised controlled, European, multicentre BESTCILIA trial [2]. The trial investigated the efficacy and safety of azithromycin as a maintenance therapy to prevent respiratory exacerbations of PCD. Participants (n=90), aged 7-50 years, were randomised to receive either placebo (n=41) or azithromycin at a dose of 250 mg or 500 mg, depending on their body weight being < or ≥40 kg (n=49) for 3 times a week over 6 months. In addition to the rate of exacerbations, the sputum was investigated for pathogenic bacterial species.

The patients taking azithromycin suffered from about half the amount of exacerbative episodes than the patients receiving placebo (rate ratio 0.46; P=0.006). Furthermore, evidently less pathogenic sputum bacteria were found compared with placebo (rate ratio 0.39; P=0.0002). Values for lung function, hearing levels, or quality of life were not different between the groups. Overall, the treatment was well tolerated, although the patients in the azithromycin group presented with more gastrointestinal symptoms.

“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,” commented Dr Kobbernagel. In her opinion, a next question will be whether a treatment longer than 6 months is still safe and could also help to preclude irreversible lung damage.

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   1. Kobbernagel HE, et al. BMC Pulm Med. 2016;16(1):104.
   2. Kobbernagel HE, et al. RCT5102. ERS 2019, 29 Sept-2 Oct, Madrid, Spain.

Posted on 28 November 201928 March 2024