A new study published in Lancet Respiratory Medicine provides preliminary evidence that tiotropium, a long-acting anticholinergic drug used for chronic obstructive pulmonary disease (COPD), is safe and effective in preschool-age children with asthma symptoms not well controlled by inhaled corticosteroids.

“Treatment options for the management of persistent asthmatic symptoms in the preschool patient population are scarce and are often associated with safety concerns,” the authors noted. “Data on the safety and efficacy of potential asthma medications in very young children (age <=5 years) are scarce, mainly because of the few available assessments of efficacy parameters and no consensus over diagnostic criteria.”

In a 12-week randomized trial, Dr. Elianne Vrijlandt from the University of Groningen, the Netherlands, and colleagues assessed the safety and efficacy of tiotropium (2.5 or 5 micrograms via the Respimat inhaler) or placebo, added to inhaled corticosteroids with or without additional controller medication. The participants were 102 children (ages 1 to 5) with persistent asthma symptoms.

Tiotropium had a “favorable safety profile, similar to that of placebo,” they reported, and reduced the risk of asthma exacerbations compared with placebo. Asthma exacerbations were reported in 10 of 34 (29%) children on placebo, compared with five of 36 (14%) on the lower tiotropium dose and two of 31 (6%) on the higher tiotropium dose.

Tiotropium, however, had no apparent effect on daytime asthma symptom scores. “The changes in adjusted weekly mean combined daytime asthma symptom scores between baseline and week 12 were not significantly different between any of the groups,” the investigators wrote.

“Our results on asthma exacerbation risk provide an important starting point for future trials, suggesting that tiotropium might be a complementary treatment strategy for managing young children with persistent asthmatic symptoms – a population for whom treatment options are scarce and often associated with safety concerns,” Dr. Vrijlandt and colleagues concluded.

Dr. Alvaro Cruz from the Program for the Control of Asthma, Federal University of Bahai School of Medicine in Salvador, Brazil, commented that the study team had to “overcome the problem of being unable to perform spirometry in most children, which reduced the certainty of asthma diagnoses and caused an inability to use the usual lung function endpoints to measure efficacy."

This “ambitious” study brings “much-needed preliminary evidence of the safety and efficacy of a new controller medication to add on to inhaled corticosteroids in young children with uncontrolled asthma,” he wrote. The number of patients in the study was small, and further research is needed to confirm the potential of tiotropium to lower the risk of asthma exacerbations in young children.