Introduction and objectives Asthma is the most common chronic disease of childhood (GINA 2015). For pre-school children whose asthma symptoms are not well controlled on inhaled corticosteroids, limited options are available for further treatment. Here, we evaluated the safety of once-daily (QD) tiotropium Respimat® (tioR) in patients aged 1–5 years with symptomatic persistent asthma.

Methods A Phase II/III, randomised, double-blind, placebo-controlled, parallel-group trial (NCT01634113) of tioR 5 μg, tioR 2.5 μg or placebo Respimat® (pboR), administered QD in the afternoon for 12 weeks, each as add-on to usual maintenance therapy. Safety data, including post hoc analysis of a composite exacerbation end point derived from adverse events (AEs), are reported.

Results No AEs leading to treatment discontinuation or death were reported. The proportion of patients with any AEs was higher with pboR (73.5%) than with tioR 5 μg (58.1%) and 2.5 μg (55.6%). Two patients each in the tioR 5 μg (6.5%) and pboR (5.9%) groups were reported with drug-related AEs. Three patients, all in the pboR group, were reported with serious AEs. Asthma exacerbation/worsening was reported by fewer patients in the tioR 5 μg and tioR 2.5 μg groups compared with the pboR group (Table).

Conclusion Once-daily tiotropium Respimat® add-on to maintenance therapy is well tolerated and may reduce exacerbations in pre-school children with symptomatic persistent asthma.